A Phase 2 interventional study of Haploidentical Bone Marrow Transplantation and Hydroxyurea in Sickle Cell Disease, sponsored by Medical College of Wisconsin. Completed at 32 sites in United States. Open to participants aged 5 Years to 45 Years. Per ClinicalTrials.gov, last updated 2026-07-08.
Sponsored by Medical College of Wisconsin · Phase 2, Interventional, and Treatment
This is a Phase II, single arm, multi-center trial, designed to estimate the efficacy and toxicity of haploidentical bone marrow transplantation (BMT) in patients with sickle cell disease (SCD). Based on their age and entry criteria patients are stratified into two groups: (1) children with severe SCD; and (2) adults with severe SCD.
This study is designed as a Phase II multi-center trial to determine the feasibility of achieving a high rate of event-free survival (EFS) at 2 years post transplant using pre-conditioning hydroxyurea (HU) with a conditioning regimen that consists of a combination of Thymoglobulin/Cyclophosphamide/Fludarabine/Thiotepa with post-grafting high-dose cyclophosphamide in patients with severe SCD who have HLA-haploidentical donors. EFS is defined as survival without a qualifying event. This is a single arm study in which participants will be enrolled into one of two strata. The first stratum will be restricted to children who have stroke and 40 children will be enrolled in this stratum. The second stratum will consist of adult patients with severe sickle cell disease and 40 participants will be enrolled in this stratum.
1,134 studies on the registry are indexed under Anemia, Sickle Cell; 238 are open to participants now.
This study's enrollment of 95 is above the median of 40 across 769 interventional studies indexed under Anemia, Sickle Cell.
Browse Anemia, Sickle Cell studies →Medical College of Wisconsin is the lead sponsor of 548 studies on the registry; 122 are open to participants now.
Of its 75 completed or terminated interventional studies of FDA-regulated products, 57 (76%) have results posted.
Counted across the registry records on this site, refreshed daily.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Adequate physical function as measured by all of the following:
Hepatic function:
Participants must be HLA typed at high resolution using DNA based typing at HLA-A, -B, -C, DRB1, and have available:
An HLA haploidentical first degree relative donor (parents, siblings or half siblings, or children) with 2, 3, or 4 (out of 8) HLA-mismatches who is willing and able to donate bone marrow. A unidirectional mismatch in either the graft versus host or host versus graft direction is considered a mismatch. The donor and recipient must be HLA identical for at least one antigen (using high resolution DNA based typing) at the following genetic loci: HLA-A, HLA-B, HLA-C, and HLA-DRB1. Fulfillment of this criterion shall be considered sufficient evidence that the donor and recipient share one HLA haplotype, and typing of additional family members is not required. Confirmatory donor HLA typing must be completed \< 100 days prior to Segment A enrollment
Inclusion Criteria for Stratum 1: Children Ages 5.00 - 14.99 years of age at enrollment
Participants with sickle cell anemiam disease (Hb SS or Sß° Thalassemia) who have one or more of the following:
Inclusion Criteria for Stratum 2: Adults Ages 15.00 - 45.99 at enrollment
Participants with sickle cell anemia (Hb SS or Sß° Thalassemia) who are 15.00 - 45.99 years of age at enrollment AND who have one or more of the following:
Participants with sickle cell anemia (Hb SS or Sß° Thalassemia) who have one or more of the following:
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
A 0–100 scale of how well someone can carry out everyday activities. 100 is no complaints and no signs of illness; 70 is able to look after yourself but not to work or keep up normal activity; 50 is needing considerable help and frequent medical care.
Studies often ask for 60 or 70 and above. Your doctor gives the score.
The share of blood the heart’s main pumping chamber (the left ventricle) pushes out with each beat, measured by an echocardiogram (heart ultrasound) or a MUGA scan. About 50–70% is normal.
The protein in red blood cells that carries oxygen. A low level is anaemia, which can cause tiredness and breathlessness. Measured in g/dL or g/L (9 g/dL is 90 g/L).
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
How quickly the kidneys clear creatinine, a waste product, from the blood. A study may estimate it from a blood test with age, weight and sex (often by the Cockcroft–Gault formula), or measure it using blood and urine collected over a set time. Given in mL/min; higher is better.
A yellow substance made when red blood cells are broken down, which the liver clears. A high level can mean a problem with the liver or the bile ducts.
Criteria often allow more for people with Gilbert’s syndrome, a harmless inherited condition that raises bilirubin.
Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.
Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.
A biopsy takes a small piece of tissue to examine. “Archival tissue” is a sample already taken, for example at diagnosis, and kept by the hospital.
Whether a stored sample will do depends on when it was taken, how much there is and what the study needs. Ask whether the study accepts your stored sample or needs a new biopsy.
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
A condition in which the immune system attacks the body’s own tissues, such as rheumatoid arthritis, lupus, psoriasis, type 1 diabetes or inflammatory bowel disease.
Immunotherapy studies often leave out active autoimmune disease, and some name exceptions, such as thyroid disease on replacement hormone. Check the exceptions this study lists.
A weakened immune system. It can come from an illness, such as HIV or some blood cancers, or from medicines that turn the immune system down, for example after an organ transplant or for an autoimmune disease; steroids at higher doses count.
Read whether the line is about a condition or about medicines, and which medicines and doses it means.
Periods have stopped for good: usually no period for 12 months in a row without another medical reason, sometimes confirmed by a blood test (FSH).
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Karnofsky performance status (KPS)
A 0–100 scale of how well someone can carry out everyday activities. 100 is no complaints and no signs of illness; 70 is able to look after yourself but not to work or keep up normal activity; 50 is needing considerable help and frequent medical care.
Studies often ask for 60 or 70 and above. Your doctor gives the score.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
LVEF (left ventricular ejection fraction)
The share of blood the heart’s main pumping chamber (the left ventricle) pushes out with each beat, measured by an echocardiogram (heart ultrasound) or a MUGA scan. About 50–70% is normal.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hemoglobin (Hb)
The protein in red blood cells that carries oxygen. A low level is anaemia, which can cause tiredness and breathlessness. Measured in g/dL or g/L (9 g/dL is 90 g/L).
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ULN (upper limit of normal)
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Creatinine clearance (CrCl)
How quickly the kidneys clear creatinine, a waste product, from the blood. A study may estimate it from a blood test with age, weight and sex (often by the Cockcroft–Gault formula), or measure it using blood and urine collected over a set time. Given in mL/min; higher is better.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Bilirubin
A yellow substance made when red blood cells are broken down, which the liver clears. A high level can mean a problem with the liver or the bile ducts.
Criteria often allow more for people with Gilbert’s syndrome, a harmless inherited condition that raises bilirubin.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ALT and AST (liver enzymes)
Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.
Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Biopsy and archival tissue
A biopsy takes a small piece of tissue to examine. “Archival tissue” is a sample already taken, for example at diagnosis, and kept by the hospital.
Whether a stored sample will do depends on when it was taken, how much there is and what the study needs. Ask whether the study accepts your stored sample or needs a new biopsy.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
HIV
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Investigational drug or device
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Clinically significant
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Autoimmune disease
A condition in which the immune system attacks the body’s own tissues, such as rheumatoid arthritis, lupus, psoriasis, type 1 diabetes or inflammatory bowel disease.
Immunotherapy studies often leave out active autoimmune disease, and some name exceptions, such as thyroid disease on replacement hormone. Check the exceptions this study lists.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Immunosuppression
A weakened immune system. It can come from an illness, such as HIV or some blood cancers, or from medicines that turn the immune system down, for example after an organ transplant or for an autoimmune disease; steroids at higher doses count.
Read whether the line is about a condition or about medicines, and which medicines and doses it means.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Postmenopausal
Periods have stopped for good: usually no period for 12 months in a row without another medical reason, sometimes confirmed by a blood test (FSH).
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Contraception
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
A conditioning regimen with Hydroxyurea, rabbit-ATG, Thiotepa, Fludarabine, Cyclophosphamide, Total Body Irradiation, and Mesna will be administered prior to Haploidentical Bone Marrow Transplantation.
Procedure: Haploidentical Bone Marrow Transplantation · Drug: Hydroxyurea · Drug: Rabbit-ATG · Drug: Thiotepa · Drug: Fludarabine · Drug: Cyclophosphamide · Radiation: Total Body Irradiation · Drug: Mesna
Eligible patients with a first degree Human Leukocyte Antigen (HLA)- haploidentical donor will undergo Haploidentical bone marrow transplantation at Day 0 with non T-cell depleted bone marrow. For Graft-vs-Host Disease (GVHD) prophylaxis, patients will be given sirolimus and mycophenolate mofetil beginning on Day +5.
HU will be given daily at 30mg/kg from Day -70 through Day -10.
Also known as: Hydrea, Droxia
Rabbit-ATG (rATG) will be given at 0.5mg/kg on Day -9, and at 2.0mg/kg on Day -8 and Day -7.
Also known as: Thymoglobulin
Thiotepa will be given at 10mg/kg on Day -7
Also known as: Chemo
Fludarabine will be given at 30mg/m2 from Day -6 to Day -2
Also known as: Fludara
Cyclophosphamide will be given at 14.5mg/kg on Day -6 and Day -5, and at 50 mg/kg on Days +3 and +4.
Also known as: Cytoxan®
Total Body Irradiation will be given at 200cGy on Day -1
Also known as: TBI
Mesna will be given at 40mg/kg on Days +3 and +4
Also known as: Mesnex
Percentage of Participants With Two-Year Post-Transplant Event Free Survival (EFS)
EFS is defined as survival without a qualifying event from transplant. Primary graft failure (PGF), secondary graft failure (SGF), second infusion of hematopoietic cells, or death from any cause will count as events for this endpoint. This endpoint was adjudicated by an Endpoint Review Committee. PGF is lack of engraftment on or before Day 42 post-transplant. Engraftment is defined as having greater than or equal to 5% donor cells post-transplant, from any molecular chimerism assessment (e.g., unsorted, myeloid, or T-cell) on a peripheral blood or bone marrow aspirate sample. SGF is defined as \< 5% donor whole blood or myeloid chimerism in peripheral blood or bone marrow beyond day +42 post-transplant in patients with prior documentation of hematopoietic recovery with \>5% donor cells by day +42 post-transplant. Infusion of a second stem cell product will be considered graft rejection, and counted toward primary or, depending on timing of the second infusion, secondary graft rejection
Time frame: 2 years post-transplant
Percentage of Participants With Two-Year Post-Transplant Overall Survival (OS)
Death from any cause will be the event and patients will be censored at the date of last contact or two years post-transplant, whichever comes first.
Time frame: 2 years post-transplant
Number of Participants With Graft Failure
Primary Graft Failure (PGF) is lack of engraftment on or before Day 42 post-transplant. Engraftment is defined as having greater than or equal to 5% donor cells post-transplant, from any molecular chimerism assessment (e.g., unsorted, myeloid, or T-cell) on a peripheral blood or bone marrow aspirate sample. Secondary Graft Failure (SGF) is defined as \< 5% donor whole blood or myeloid chimerism in peripheral blood or bone marrow beyond day +42 post-transplant in patients with prior documentation of hematopoietic recovery with \>5% donor cells by day +42 post-transplant. Infusion of a second stem cell product will be considered graft rejection, and counted toward primary or, depending on timing of the second infusion, secondary graft rejection.
Time frame: 2 Years post-transplant
Percentage of Participants With Disease Recurrence Post-Transplant
Disease Recurrence is defined as primary graft failure, secondary graft failure, or sickle hemoglobin (HbS) \> 70% within 2 years post-transplant. Cumulative Incidence of Disease Recurrence was estimated with a 95% confidence interval using the Aalen-Johansen estimator with death prior to disease recurrence treated as a competing risk. This endpoint was adjudicated by an Endpoint Review Committee.
Time frame: 1 year and 2 years post-transplant
Percentage of Participants With Neutrophil Recovery Post-Transplant
Neutrophil recovery is defined as the first of 3 measurements on different days when the patient has an absolute neutrophil count of ≥500/μL after conditioning. The incidence of neutrophil recovery from transplant will be estimated using the cumulative incidence function with a 95% confidence interval using the Aalen-Johansen estimator with death or second transplant without neutrophil recovery as a competing risk.
Time frame: 42 Days post-transplant
Percentage of Participants With Platelet Recovery Post-Transplant
Platelet recovery is defined as the first day of a minimum of 3 measurements on different days that the patient has achieved a platelet count \> 50,000/μL AND did not receive a platelet transfusion in the previous 7 days. The incidence of platelet recovery from transplant will be estimated using the cumulative incidence function with a 95% confidence interval using the Aalen-Johansen estimator with death or second transplant without platelet recovery as a competing risk.
Time frame: 60 Days, 100 Days post-transplant
Mean Percentage of Donor Chimerism Post-Transplant
A point estimate and confidence interval will be provided for the mean percent donor chimerism at the time points specified including Day 28, Day 100, Day 180, 1 year, and 2 years post-transplant. For each participant's visit with multiple chimerism sources recorded, donor percentage is determined by taking the first non-missing chimerism percentage in the following order of sources: marrow, blood, myeloid, and t-cell.
Time frame: Days 28, 100, and 180 and at 1 and 2 years post-transplant
Percentage of Participants by Donor Chimerism Category Post-Transplant
At Day 28, Day 100, Day 180, 1 year and 2 years post-transplant, the proportions with low chimerism (\<5%), mixed chimerism (5-95%), or full chimerism (\>95%) will be tabulated and described. For each participant's visit with multiple chimerism sources recorded, donor percentage is determined by taking the first non-missing chimerism percentage in the following order of sources: marrow, blood, myeloid, and t-cell.
Time frame: Days 28, 100, and 180 and at 1 and 2 years post-transplant
Number of Participants by Maximum Acute GVHD Grade Post-Transplant
Acute GVHD was graded according to Consensus Criteria with higher grade indicating worse outcomes. Grade I aGVHD is defined as stage 1-2 skin rash and no liver or GI involvement. Grade II is stage 3 skin rash, or stage 1 liver involvement, or stage 1 GI involvement. Grade III is stage 0-3 skin rash, with stage 2-3 liver involvement, or stage 2-3 GI involvement. Grade IV is stage 4 skin rash, liver, or GI involvement. Maximum grade is defined as the maximum grade of acute GVHD (0-IV) that a participant experiences by Day 100 post-transplant.
Time frame: 100 Days post-transplant
Percentage of Participants With Acute GVHD Post-Transplant
Acute GVHD was graded according to Consensus Criteria with higher grade indicating worse outcomes. Grade I aGVHD is defined as stage 1-2 skin rash and no liver or GI involvement. Grade II is stage 3 skin rash, or stage 1 liver involvement, or stage 1 GI involvement. Grade III is stage 0-3 skin rash, with stage 2-3 liver involvement, or stage 2-3 GI involvement. Grade IV is stage 4 skin rash, liver, or GI involvement. The cumulative incidence of acute GVHD grade II-IV and III-IV at Day 100 was estimated using the Aalen-Johansen estimator with 95% confidence intervals, treating death prior to aGVHD as a competing event. Time to aGVHD is defined as time from transplant until onset of grades II-IV and III-IV aGVHD, respectively.
Time frame: 100 Days post-transplant
Number of Participants by Maximum Chronic GVHD Severity Post-Transplant
Chronic GVHD is based on NIH Consensus Criteria (2014 NIH Consensus Criteria) and includes mild, moderate and severe chronic GVHD. Skin/hair, ocular, oral, pulmonary, gastrointestinal, hepatic, genitourinary, musculoskeletal, and hematologic systems were scored on a 0-3 scale to reflect degree of cGVHD involvement. Maximum grade is defined as the maximum grade of chronic GVHD (mild, moderate, or severe) that a participant experiences by 2 years post-transplant.
Time frame: 2 Years post-transplant
Percentage of Participants With Chronic GVHD Post-Transplant
Percentage of participants with chronic GVHD (cGVHD) at will be estimated with 95% confidence intervals for each treatment group using the cumulative incidence estimate with the complementary log-log transformation, treating death prior to cGVHD as a competing event. Chronic GVHD is based on NIH Consensus Criteria (2014 NIH Consensus Criteria) and includes mild, moderate and severe chronic GVHD. Skin/hair, ocular, oral, pulmonary, gastrointestinal, hepatic, genitourinary, musculoskeletal, and hematologic systems were scored on a 0-3 scale to reflect degree of cGVHD involvement. This endpoint considers any cGVHD onset.
Time frame: 6 months, 1 year, 2 years post-transplant
Number of Participants With Complications and Events Post-Transplant
The number of participants experiencing the following complications and events will be tabulated at baseline, 1 year and 2 years post-transplant: Idiopathic pneumonia syndrome (IPS), Veno-occlusive disease (VOD), Various Central Nervous System (CNS) toxicities, Stroke, participants on immunosuppression for GVHD, and significant infections reported (any bacterial/fungal sepsis, Cytomegalovirus (CMV) reactivation with/without clinical disease, adenovirus, Epstein-Barr Virus (EBV)).
Time frame: Baseline, 1 year, and 2 years post-transplant
Number of Participants With Sickle Cell Disease Events of Special Interest Post-Transplant
Participants will be followed for the entire 2 year duration of their time on study for the recurrence of Sickle Cell Disease (SCD) related complications. These SCD related complications are referred to as SCD events of special interest (SCD-EOSI) and will be summarized with frequency. These SCD-EOSI include: pulmonary hypertension, new onset of significant cerebrovascular event, renal function compromise, new onset of avascular necrosis of hip or shoulder, new onset of leg ulceration, new onset of acute chest syndrome, and new onset of painful vaso-occlusive crisis requiring hospitalization or parenteral opioid drug in the outpatient setting.
Time frame: Baseline, 6 months, 1 year, 18 months, and 2 Years post-transplant
Summary of Hematological Outcomes in Percentages
Hematological Outcomes will be summarized with mean and standard deviation at various timepoints. Depending on the measurement, "baseline" could be pre-hydroxyurea conditioning (day -70 pre-transplant) and/or pre-thymoglobulin (day -7 pre-transplant). Measures include: hemoglobin (Hgb) %, reticulocyte count %, and hemoglobin S level %.
Time frame: Baseline, Day 28, Day 100, 6 months, 1, and 2 years post-transplant
Summary of Glomerular Filtration Rate (GFR)
Renal Function Outcomes will be summarized with mean and standard deviation at various timepoints. "Baseline" refers to pre-thymoglobulin (day -7 pre-transplant). Measures include: GFR (mL/min/1.73m2) and Estimated GFR. Estimated GFR is computed using the CKD-EPI Creatinine Equation 2021 for Adults and the Bedside Schwartz 2009 equation for Pediatrics.
Time frame: Baseline, 1, and 2 years post-transplant
Summary of Lung Function in Liters
Lung Function Outcomes will be summarized with mean and standard deviation at various timepoints. "Baseline" refers to pre-hydroxyurea (day -70 pre-transplant). Measures include: FEV1 (L), FVC (L), and TLC (L). All values in this table were reported by study site.
Time frame: Baseline, 1, and 2 years post-transplant
Summary of Cardiac Function
Cardiac Function is measured through tricuspid regurgitant jet velocity (TRJV). It will be summarized with mean and standard deviation at Baseline, 1, and 2 years post-transplant. "Baseline" refers to pre-hydroxyurea (day -70 pre-transplant).
Time frame: Baseline, 1, and 2 years post-transplant
Summary of Six Minute Walk Distance
Six Minute Walk Distance is measured in meters to assess how far a participant can walk in 6 minutes. It will be summarized with mean and standard deviation at Baseline, 1, and 2 years post-transplant. "Baseline" refers to pre-hydroxyurea (day -70 pre-transplant). Change from baseline will also be provided at the 1 and 2 years post-transplant visit.
Time frame: Baseline, 1, and 2 years post-transplant
Mean Patient Reported Quality of Life (QoL) Score
Health-Related Quality of Life (QoL) assessed using the NIH's PROMIS short forms administered to English- and Spanish-speaking patients in the adult stratum. Questions about fatigue (short form 8a), pain interference (short form 8a), and pain intensity (short form 3a) are asked regarding the degree of difficulty in performing activities of daily living such as housework, social activities, and day to day activities. It is scored from 0 to 10 and converted to a standardized T-score with mean 50 and standard deviation 10. A higher T-score represents worse fatigue, pain interfering with more daily activities, and worse pain intensity.
Time frame: Baseline, 1, and 2 years post-transplant
Mean Patient Reported Pain Intensity Score From Pain Diary
Subjects who speak English and are 15 years or older will be asked to use a web-based diary to report pain intensity on a scale from 0 (no pain) to 10 (worst pain). Subject pain intensity score is reported as an aggregate of several AM and PM scores at Baseline, one year, and two years post-transplant timepoints.
Time frame: Baseline, 1, and 2 years post-transplant
Number of Participants by Cause of Death
Cause of Death is tabulated by age strata. Deaths among participants who were not transplanted are included.
Time frame: From Enrollment to 2 Years post-transplant
Number of Participants by Maximum Grade of Infection
Number of participants who reported the Maximum Infection Severity of Grade 2 and Grade 3. Only grade 2 and grade 3 infections occurring post transplantation were reported on the study. Grade 2 and grade 3 infections are defined by the BMT CTN Technical MOP. Higher infection grade indicates worse infection severity. The infection grading criteria are published online (https://bmtctn.net/administrative-manual-procedures-moppolicy-guidelines). Severity of grade 1, 2 and 3 are described for bacterial, fungal, viral, parasitic, and nonmicrobiological infections. For example, grade 2 fungal infections are defined as candida esophagitis, or proven or probably fungal sinusistis confirmed radiologically without orbital, brain or bone involvement. Grade 3 fungal infections are defined as Fungemia including candidemia, Proven or probably invasive fungal infections, Disseminated infections with histoplasmosis, blastomycosis, coccidiomycosis, or Cryptococcus, or Pneumocystis jiroveci pneumonia.
Time frame: From Transplant to 2 Years post-transplant
Frequencies of Infections Categorized by Infection Type
The number of systemic infections is reported. Infections are categorized by infection type. A participant can report multiple types of infections, so the categories are not mutually exclusive for participants. All grade 2 and grade 3 infections, as defined by the BMT CTN Technical MOP, occurring post transplantation were reported on the study.
Time frame: From Transplant to 2 Years post-transplant
Frequency of Hospital Readmissions by Cause
Participants could be readmitted to a hospital for many reasons during follow-up. The causes of each readmission are tabulated by age strata. The protocol-specified scheduled transplant hospitalization is not included in this table.
Time frame: From Transplant to 2 Years post-transplant
Summary of Lactate Dehydrogenase (LDH)
Hematological Outcomes will be summarized with mean and standard deviation at various post-transplant timepoints. Measures in this table include: LDH (U/L)
Time frame: Day 28, Day 100, 6 months, 1, and 2 years post-transplant
Summary of Bilirubin
Hematological Outcomes will be summarized with mean and standard deviation at various timepoints. Baseline timepoint is pre-thymoglobulin (day -7 pre-transplant). Measures in this table include: Bilirubin (mg/dL)
Time frame: Baseline, Day 28, Day 100, 6 months, 1, and 2 years post-transplant
Summary of Serum Ferritin
Hematological Outcomes will be summarized with mean and standard deviation at various timepoints. Baseline refers to pre-thymoglobulin (day -7 pre-transplant). Measures in this table include: Serum Ferritin Level (ng/dL)
Time frame: Baseline pre-thymoglobulin visit (day -7 pre-transplant)
Summary of Creatinine Clearance
Renal Function Outcomes will be summarized with mean and standard deviation at various timepoints. "Baseline" refers to pre-thymoglobulin (day -7 pre-transplant). Measures include: Creatinine Clearance (mL/min).
Time frame: Baseline, 1, and 2 years post-transplant
Summary of Urine Albumin Creatine Ratio
Renal Function Outcomes will be summarized with mean and standard deviation at various timepoints. Measures include: Urine Albumin Creatine Ratio (mg/g).
Time frame: 1 and 2 years post-transplant
Summary of Lung Function Percent Predicted
Lung Function Outcomes will be summarized with mean and standard deviation at various timepoints. "Baseline" refers to pre-hydroxyurea (day -70 pre-transplant). Measures include: FEV1 (percent predicted), FVC (percent predicted), FEV1/FVC (percent predicted), and TLC (percent predicted). All values in this table, including percent predicted, were reported by study site.
Time frame: Baseline, 1, and 2 years post-transplant
Summary of Lung Function Ratio of Liters
Lung Function Outcomes will be summarized with mean and standard deviation at various timepoints. "Baseline" refers to pre-hydroxyurea (day -70 pre-transplant). Measures include: FEV1/FVC (ratio of Liters). All values in this table were reported by study site.
Time frame: Baseline, 1, and 2 years post-transplant
Summary of Lung Function Percentages of Lab Measure
Lung Function Outcomes will be summarized with mean and standard deviation at various timepoints. "Baseline" refers to pre-hydroxyurea (day -70 pre-transplant). Measures include: DLCO (%) and Oxygen Saturation (%). All values in this table were reported by study site.
Time frame: Baseline, 1, and 2 years post-transplant
Summary of Lung Function Ratio of Percent Predicted
Lung Function Outcomes will be summarized with mean and standard deviation at various timepoints. "Baseline" refers to pre-hydroxyurea (day -70 pre-transplant). Measures include: FEV1/FVC (ratio of percent predicted). All values in this table were reported by study site.
Time frame: Baseline, 1, and 2 years post-transplant
Mean Follow-up Days Since Transplant
Average number of post-transplant follow-up days by age strata.
Time frame: From transplant up to 2 years post-transplant
BMT CTN 1507's enrollment was between October 10, 2017 and October 19, 2022 with 36 participating centers in the United States of America. 95 participants enrolled from 28 centers (18 centers enrolled adults and 17 enrolled pediatrics). Of 95 participants, 54 participants enrolled in the adult stratum, and 41 enrolled in the pediatric stratum. The accrual goal was 40 transplanted participants in each age strata. The study was completed on January 29, 2025.
| Milestone | Adult Stratum | Pediatric Stratum |
|---|---|---|
| Started | 54 | 41 |
| Transplanted | 42 | 39 |
| Completed | 38 | 34 |
| Not completed | 16 | 7 |
| Withdrew: Physician decision | 0 | 2 |
| Withdrew: Withdrawal by subject | 4 | 1 |
| Withdrew: Death | 1 | 0 |
| Withdrew: Donor issue | 4 | 0 |
| Withdrew: Psychosocial issues | 1 | 0 |
| Withdrew: Insurance issue | 2 | 0 |
| Withdrew: Participant non-compliance | 1 | 0 |
| Withdrew: Accrual closure | 1 | 0 |
| Withdrew: Lost to follow-up | 2 | 4 |
EFS is defined as survival without a qualifying event from transplant. Primary graft failure (PGF), secondary graft failure (SGF), second infusion of hematopoietic cells, or death from any cause will count as events for this endpoint. This endpoint was adjudicated by an Endpoint Review Committee. PGF is lack of engraftment on or before Day 42 post-transplant. Engraftment is defined as having greater than or equal to 5% donor cells post-transplant, from any molecular chimerism assessment (e.g., unsorted, myeloid, or T-cell) on a peripheral blood or bone marrow aspirate sample. SGF is defined as \< 5% donor whole blood or myeloid chimerism in peripheral blood or bone marrow beyond day +42 post-transplant in patients with prior documentation of hematopoietic recovery with \>5% donor cells by day +42 post-transplant. Infusion of a second stem cell product will be considered graft rejection, and counted toward primary or, depending on timing of the second infusion, secondary graft rejection
| Percentage of Participants | Adult Stratum | Pediatric Stratum |
|---|---|---|
| Percentage of Participants With Two-Year Post-Transplant Event Free Survival (EFS) | 88.0 (73.5 to 94.8) | 79.3 (62.8 to 89.1) |
Death from any cause will be the event and patients will be censored at the date of last contact or two years post-transplant, whichever comes first.
| Percentage of Participants | Adult Stratum | Pediatric Stratum |
|---|---|---|
| Percentage of Participants With Two-Year Post-Transplant Overall Survival (OS) | 95.0 (81.5 to 98.7) | 94.5 (79.7 to 98.6) |
Primary Graft Failure (PGF) is lack of engraftment on or before Day 42 post-transplant. Engraftment is defined as having greater than or equal to 5% donor cells post-transplant, from any molecular chimerism assessment (e.g., unsorted, myeloid, or T-cell) on a peripheral blood or bone marrow aspirate sample. Secondary Graft Failure (SGF) is defined as \< 5% donor whole blood or myeloid chimerism in peripheral blood or bone marrow beyond day +42 post-transplant in patients with prior documentation of hematopoietic recovery with \>5% donor cells by day +42 post-transplant. Infusion of a second stem cell product will be considered graft rejection, and counted toward primary or, depending on timing of the second infusion, secondary graft rejection.
| Participants | Adult Stratum | Pediatric Stratum |
|---|---|---|
| Primary Graft Failure | 2 | 2 |
| Secondary Graft Failure | 1 | 4 |
Disease Recurrence is defined as primary graft failure, secondary graft failure, or sickle hemoglobin (HbS) \> 70% within 2 years post-transplant. Cumulative Incidence of Disease Recurrence was estimated with a 95% confidence interval using the Aalen-Johansen estimator with death prior to disease recurrence treated as a competing risk. This endpoint was adjudicated by an Endpoint Review Committee.
| Percentage of Participants | Adult Stratum | Pediatric Stratum |
|---|---|---|
| Disease Recurrence at 1 year post-transplant | 7.1 (1.8 to 17.6) | 15.4 (6.2 to 28.5) |
| Disease Recurrence at 2 years post-transplant | 9.8 (3.0 to 21.3) | 15.4 (6.2 to 28.5) |
Neutrophil recovery is defined as the first of 3 measurements on different days when the patient has an absolute neutrophil count of ≥500/μL after conditioning. The incidence of neutrophil recovery from transplant will be estimated using the cumulative incidence function with a 95% confidence interval using the Aalen-Johansen estimator with death or second transplant without neutrophil recovery as a competing risk.
| Percentage of Participants | Adult Stratum | Pediatric Stratum |
|---|---|---|
| Percentage of Participants With Neutrophil Recovery Post-Transplant | 92.9 (77.4 to 97.9) | 100.0 (100.0 to 100.0) |
Platelet recovery is defined as the first day of a minimum of 3 measurements on different days that the patient has achieved a platelet count \> 50,000/μL AND did not receive a platelet transfusion in the previous 7 days. The incidence of platelet recovery from transplant will be estimated using the cumulative incidence function with a 95% confidence interval using the Aalen-Johansen estimator with death or second transplant without platelet recovery as a competing risk.
| Percentage of Participants | Adult Stratum | Pediatric Stratum |
|---|---|---|
| 60 Days | 88.1 (72.6 to 95.1) | 81.1 (63.5 to 90.8) |
| 100 Days | 92.9 (77.4 to 97.9) | 91.9 (74.7 to 97.6) |
A point estimate and confidence interval will be provided for the mean percent donor chimerism at the time points specified including Day 28, Day 100, Day 180, 1 year, and 2 years post-transplant. For each participant's visit with multiple chimerism sources recorded, donor percentage is determined by taking the first non-missing chimerism percentage in the following order of sources: marrow, blood, myeloid, and t-cell.
| Percentage of Donor Chimerism | Adult Stratum | Pediatric Stratum |
|---|---|---|
| Day 28 | 93.4 (86.5 to 100.0) | 92.6 (85.0 to 100.0) |
| Day 100 | 92.6 (84.3 to 100.0) | 91.3 (82.7 to 99.9) |
| Day 180 | 96.9 (91.3 to 100.0) | 94.1 (86.8 to 100.0) |
| 1 Year | 99.3 (98.3 to 100.0) | 90.0 (80.4 to 99.6) |
| 2 Years | 97.4 (93.8 to 100.0) | 97.0 (91.6 to 100.0) |
At Day 28, Day 100, Day 180, 1 year and 2 years post-transplant, the proportions with low chimerism (\<5%), mixed chimerism (5-95%), or full chimerism (\>95%) will be tabulated and described. For each participant's visit with multiple chimerism sources recorded, donor percentage is determined by taking the first non-missing chimerism percentage in the following order of sources: marrow, blood, myeloid, and t-cell.
| Percentage of Participants | Adult Stratum | Pediatric Stratum |
|---|---|---|
| Low Chimerism (Donor <5%) at Day 28 | 4.8 | 2.6 |
| Low Chimerism (Donor <5%) at Day 100 | 7.3 | 5.4 |
| Low Chimerism (Donor <5%) at Day 180 | 2.8 | 2.9 |
| Low Chimerism (Donor <5%) at 1 Year | 0.0 | 3.1 |
| Low Chimerism (Donor <5%) at 2 Years | 0.0 | 0.0 |
| Mixed Chimerism (Donor 5-95%) at Day 28 | 7.1 | 15.4 |
| Mixed Chimerism (Donor 5-95%) at Day 100 | 2.4 | 8.1 |
| Mixed Chimerism (Donor 5-95%) at Day 180 | 2.8 | 8.6 |
| Mixed Chimerism (Donor 5-95%) at 1 Year | 5.9 | 15.6 |
| Mixed Chimerism (Donor 5-95%) at 2 Years | 9.4 | 6.9 |
| Full Chimerism (Donor >95%) at Day 28 | 88.1 | 82.1 |
| Full Chimerism (Donor >95%) at Day 100 | 90.2 | 86.5 |
| Full Chimerism (Donor >95%) at Day 180 | 94.4 | 88.6 |
| Full Chimerism (Donor >95%) at 1 Year | 94.1 | 81.3 |
| Full Chimerism (Donor >95%) at 2 Years | 90.6 | 93.1 |
Acute GVHD was graded according to Consensus Criteria with higher grade indicating worse outcomes. Grade I aGVHD is defined as stage 1-2 skin rash and no liver or GI involvement. Grade II is stage 3 skin rash, or stage 1 liver involvement, or stage 1 GI involvement. Grade III is stage 0-3 skin rash, with stage 2-3 liver involvement, or stage 2-3 GI involvement. Grade IV is stage 4 skin rash, liver, or GI involvement. Maximum grade is defined as the maximum grade of acute GVHD (0-IV) that a participant experiences by Day 100 post-transplant.
| Participants | Adult Stratum | Pediatric Stratum |
|---|---|---|
| Grade 0, no aGVHD | 23 | 26 |
| Grade I | 8 | 7 |
| Grade II | 9 | 4 |
| Grade III | 2 | 2 |
| Grade IV | 0 | 0 |
Acute GVHD was graded according to Consensus Criteria with higher grade indicating worse outcomes. Grade I aGVHD is defined as stage 1-2 skin rash and no liver or GI involvement. Grade II is stage 3 skin rash, or stage 1 liver involvement, or stage 1 GI involvement. Grade III is stage 0-3 skin rash, with stage 2-3 liver involvement, or stage 2-3 GI involvement. Grade IV is stage 4 skin rash, liver, or GI involvement. The cumulative incidence of acute GVHD grade II-IV and III-IV at Day 100 was estimated using the Aalen-Johansen estimator with 95% confidence intervals, treating death prior to aGVHD as a competing event. Time to aGVHD is defined as time from transplant until onset of grades II-IV and III-IV aGVHD, respectively.
| Percentage of Participants | Adult Stratum | Pediatric Stratum |
|---|---|---|
| Grades II-IV Acute GVHD | 26.2 (14.0 to 40.2) | 15.5 (6.2 to 28.7) |
| Grades III-IV Acute GVHD | 4.8 (0.9 to 14.4) | 5.2 (0.9 to 15.5) |
Chronic GVHD is based on NIH Consensus Criteria (2014 NIH Consensus Criteria) and includes mild, moderate and severe chronic GVHD. Skin/hair, ocular, oral, pulmonary, gastrointestinal, hepatic, genitourinary, musculoskeletal, and hematologic systems were scored on a 0-3 scale to reflect degree of cGVHD involvement. Maximum grade is defined as the maximum grade of chronic GVHD (mild, moderate, or severe) that a participant experiences by 2 years post-transplant.
| Participants | Adult Stratum | Pediatric Stratum |
|---|---|---|
| None, no chronic GVHD | 33 | 27 |
| Mild | 3 | 4 |
| Moderate | 3 | 5 |
| Severe | 3 | 3 |
Percentage of participants with chronic GVHD (cGVHD) at will be estimated with 95% confidence intervals for each treatment group using the cumulative incidence estimate with the complementary log-log transformation, treating death prior to cGVHD as a competing event. Chronic GVHD is based on NIH Consensus Criteria (2014 NIH Consensus Criteria) and includes mild, moderate and severe chronic GVHD. Skin/hair, ocular, oral, pulmonary, gastrointestinal, hepatic, genitourinary, musculoskeletal, and hematologic systems were scored on a 0-3 scale to reflect degree of cGVHD involvement. This endpoint considers any cGVHD onset.
| Percentage of Participants | Adult Stratum | Pediatric Stratum |
|---|---|---|
| 6 Months | 7.1 (1.8 to 17.6) | 10.5 (3.3 to 22.7) |
| 1 Year | 14.7 (5.9 to 27.3) | 16.0 (6.4 to 29.5) |
| 2 Years | 22.4 (10.9 to 36.4) | 30.6 (16.3 to 46.1) |
The number of participants experiencing the following complications and events will be tabulated at baseline, 1 year and 2 years post-transplant: Idiopathic pneumonia syndrome (IPS), Veno-occlusive disease (VOD), Various Central Nervous System (CNS) toxicities, Stroke, participants on immunosuppression for GVHD, and significant infections reported (any bacterial/fungal sepsis, Cytomegalovirus (CMV) reactivation with/without clinical disease, adenovirus, Epstein-Barr Virus (EBV)).
| Participants | Adult Stratum | Pediatric Stratum |
|---|---|---|
| Idopathic pneumonia Syndrome (IPS) at Baseline — Yes | 0 | 0 |
| Idopathic pneumonia Syndrome (IPS) at Baseline — No | 42 | 39 |
| Idopathic pneumonia Syndrome (IPS) at One Year Post-Transplant — Yes | 1 | 1 |
| Idopathic pneumonia Syndrome (IPS) at One Year Post-Transplant — No | 36 | 34 |
| Idopathic pneumonia Syndrome (IPS) at Two Years Post-Transplant — Yes | 0 | 0 |
| Idopathic pneumonia Syndrome (IPS) at Two Years Post-Transplant — No | 16 | 31 |
| Veno-occlusive disease (VOD) at Baseline — Yes | 0 | 0 |
| Veno-occlusive disease (VOD) at Baseline — No | 42 | 39 |
| Veno-occlusive disease (VOD) at One Year Post-Transplant — Yes | 0 | 0 |
| Veno-occlusive disease (VOD) at One Year Post-Transplant — No | 37 | 35 |
| Veno-occlusive disease (VOD) at Two Years Post-Transplant — Yes | 0 | 0 |
| Veno-occlusive disease (VOD) at Two Years Post-Transplant — No | 16 | 31 |
| Various CNS Toxicities at Baseline — Yes | 0 | 0 |
| Various CNS Toxicities at Baseline — No | 42 | 39 |
| Various CNS Toxicities at One Year Post-Transplant — Yes | 1 | 0 |
| Various CNS Toxicities at One Year Post-Transplant — No | 36 | 35 |
| Various CNS Toxicities at Two Years Post-Transplant — Yes | 0 | 0 |
| Various CNS Toxicities at Two Years Post-Transplant — No | 16 | 31 |
| Stroke at Baseline — Yes | 0 | 2 |
| Stroke at Baseline — No | 42 | 37 |
| Stroke at One Year Post-Transplant — Yes | 0 | 0 |
| Stroke at One Year Post-Transplant — No | 37 | 35 |
| Stroke at Two Years Post-Transplant — Yes | 0 | 0 |
| Stroke at Two Years Post-Transplant — No | 16 | 31 |
| Immunosuppression for GVHD (Prophylaxis and Treatment) at Baseline — Yes | 25 | 37 |
| Immunosuppression for GVHD (Prophylaxis and Treatment) at Baseline — No | 17 | 2 |
| Immunosuppression for GVHD (Prophylaxis and Treatment) at One Year Post-Transplant — Yes | 2 | 20 |
| Immunosuppression for GVHD (Prophylaxis and Treatment) at One Year Post-Transplant — No | 35 | 15 |
| Immunosuppression for GVHD (Prophylaxis and Treatment) at Two Years Post-Transplant — Yes | 1 | 6 |
| Immunosuppression for GVHD (Prophylaxis and Treatment) at Two Years Post-Transplant — No | 15 | 25 |
| Significant Infections (see outcomes description) at Baseline — Yes | 2 | 1 |
| Significant Infections (see outcomes description) at Baseline — No | 40 | 38 |
| Significant Infections (see outcomes description) at One Year Post-Transplant — Yes | 11 | 15 |
| Significant Infections (see outcomes description) at One Year Post-Transplant — No | 26 | 20 |
| Significant Infections (see outcomes description) at Two Years Post-Transplant — Yes | 3 | 6 |
| Significant Infections (see outcomes description) at Two Years Post-Transplant — No | 13 | 25 |
Participants will be followed for the entire 2 year duration of their time on study for the recurrence of Sickle Cell Disease (SCD) related complications. These SCD related complications are referred to as SCD events of special interest (SCD-EOSI) and will be summarized with frequency. These SCD-EOSI include: pulmonary hypertension, new onset of significant cerebrovascular event, renal function compromise, new onset of avascular necrosis of hip or shoulder, new onset of leg ulceration, new onset of acute chest syndrome, and new onset of painful vaso-occlusive crisis requiring hospitalization or parenteral opioid drug in the outpatient setting.
| Participants | Adult Stratum | Pediatric Stratum |
|---|---|---|
| Pulmonary Hypertension at Baseline | 0 | 0 |
| Pulmonary Hypertension at 6 Months Post-Transplant | 0 | 1 |
| Pulmonary Hypertension at 1 Year Post-Transplant | 0 | 0 |
| Pulmonary Hypertension at 18 Months Post-Transplant | 0 | 0 |
| Pulmonary Hypertension at 2 Years Post-Transplant | 0 | 0 |
| New Onset of Significant Cerebrovascular Event at Baseline | 1 | 2 |
| New Onset of Significant Cerebrovascular Event at 6 Months Post-Transplant | 1 | 0 |
| New Onset of Significant Cerebrovascular Event at 1 Year Post-Transplant | 0 | 0 |
| New Onset of Significant Cerebrovascular Event at 18 Months Post-Transplant | 0 | 0 |
| New Onset of Significant Cerebrovascular Event at 2 Years Post-Transplant | 0 | 0 |
| Renal Function Compromise at Baseline | 0 | 0 |
| Renal Function Compromise at 6 Months Post-Transplant | 3 | 1 |
| Renal Function Compromise at 1 Year Post-Transplant | 3 | 1 |
| Renal Function Compromise at 18 Months Post-Transplant | 3 | 0 |
| Renal Function Compromise at 2 Years Post-Transplant | 1 | 0 |
| New Onset of Avascular Necrosis of Hip or Shoulder at Baseline | 3 | 2 |
| New Onset of Avascular Necrosis of Hip or Shoulder at 6 Months Post-Transplant | 0 | 0 |
| New Onset of Avascular Necrosis of Hip or Shoulder at 1 Year Post-Transplant | 0 | 0 |
| New Onset of Avascular Necrosis of Hip or Shoulder at 18 Months Post-Transplant | 4 | 0 |
| New Onset of Avascular Necrosis of Hip or Shoulder at 2 Years Post-Transplant | 0 | 0 |
| New Onset of Leg Ulceration at Baseline | 0 | 0 |
| New Onset of Leg Ulceration at 6 Months Post-Transplant | 0 | 0 |
| New Onset of Leg Ulceration at 1 Year Post-Transplant | 1 | 0 |
| New Onset of Leg Ulceration at 18 Months Post-Transplant | 0 | 0 |
| New Onset of Leg Ulceration at 2 Years Post-Transplant | 1 | 0 |
| New Onset of Acute Chest Syndrome at Baseline | 2 | 2 |
| New Onset of Acute Chest Syndrome at 6 Months Post-Transplant | 0 | 0 |
| New Onset of Acute Chest Syndrome at 1 Year Post-Transplant | 0 | 1 |
| New Onset of Acute Chest Syndrome at 18 Months Post-Transplant | 0 | 0 |
| New Onset of Acute Chest Syndrome at 2 Years Post-Transplant | 0 | 0 |
| New Onset of Painful Vaso-occlusive Crisis at Baseline | 16 | 4 |
| New Onset of Painful Vaso-occlusive Crisis at 6 Months Post-Transplant | 0 | 1 |
| New Onset of Painful Vaso-occlusive Crisis at 1 Year Post-Transplant | 0 | 0 |
| New Onset of Painful Vaso-occlusive Crisis at 18 Months Post-Transplant | 1 | 0 |
| New Onset of Painful Vaso-occlusive Crisis at 2 Years Post-Transplant | 0 | 0 |
Hematological Outcomes will be summarized with mean and standard deviation at various timepoints. Depending on the measurement, "baseline" could be pre-hydroxyurea conditioning (day -70 pre-transplant) and/or pre-thymoglobulin (day -7 pre-transplant). Measures include: hemoglobin (Hgb) %, reticulocyte count %, and hemoglobin S level %.
| percentage of lab measure | Adult Stratum | Pediatric Stratum |
|---|---|---|
| Hgb (%) at Pre-Hydroxyurea Visit | 9.9 ± 1.4 | 9.9 ± 1.5 |
| Hgb (%) at Day 100 Post-Transplant | 11.0 ± 1.7 | 11.0 ± 1.5 |
| Hgb (%) at 6 Months Post-Transplant | 11.7 ± 1.6 | 11.8 ± 1.6 |
| Hgb (%) at 1 Year Post-Transplant | 14.6 ± 14.4 | 11.9 ± 1.8 |
| Hgb (%) at 2 Years Post-Transplant | 12.9 ± 2.3 | 12.1 ± 2.0 |
| Reticulocyte Count (%) at Pre-Hydroxyurea Visit | 10.2 ± 7.7 | 8.4 ± 5.1 |
| Reticulocyte Count (%) at Pre-Thymoglobulin Visit | 8.2 ± 6.2 | 7.9 ± 5.4 |
| Reticulocyte Count (%) at Day 28 Post-Transplant | 1.3 ± 2.3 | 1.6 ± 1.6 |
| Reticulocyte Count (%) at Day 100 Post-Transplant | 3.1 ± 4.6 | 2.7 ± 2.0 |
| Reticulocyte Count (%) at 6 Months Post-Transplant | 3.3 ± 4.5 | 2.4 ± 1.5 |
| Reticulocyte Count (%) at 1 Year Post-Transplant | 2.2 ± 1.4 | 3.1 ± 3.4 |
| Reticulocyte Count (%) at 2 Years Post-Transplant | 1.7 ± 0.7 | 12.1 ± 2.0 |
| Hemoglobin S Level (%) at Pre-Hydroxyurea Visit | 55.1 ± 25.9 | 38.5 ± 24.7 |
| Hemoglobin S Level (%) at Day 28 Post-Transplant | 12.0 ± 14.0 | 12.9 ± 12.6 |
| Hemoglobin S Level (%) at Day 100 Post-Transplant | 23.0 ± 14.7 | 27.1 ± 16.4 |
| Hemoglobin S Level (%) at 6 Months Post-Transplant | 23.0 ± 14.7 | 31.0 ± 11.8 |
| Hemoglobin S Level (%) at 1 Year Post-Transplant | 31.1 ± 14.0 | 34.5 ± 13.8 |
| Hemoglobin S Level (%) at 2 Years Post-Transplant | 30.6 ± 20.0 | 32.1 ± 12.8 |
Renal Function Outcomes will be summarized with mean and standard deviation at various timepoints. "Baseline" refers to pre-thymoglobulin (day -7 pre-transplant). Measures include: GFR (mL/min/1.73m2) and Estimated GFR. Estimated GFR is computed using the CKD-EPI Creatinine Equation 2021 for Adults and the Bedside Schwartz 2009 equation for Pediatrics.
| mL/min/1.73m2 | Adult Stratum | Pediatric Stratum |
|---|---|---|
| GFR (mL/min/1.73m2) at Baseline | 92.5 ± 37.7 | 150.4 ± 57.6 |
| GFR (mL/min/1.73m2) 1-Year Post-Transplant | 58.0 ± 6.8 | 122.6 ± 45.1 |
| GFR (mL/min/1.73m2) 2-Years Post-Transplant | 67.6 ± 13.8 | 108.9 ± 30.4 |
| Estimated GFR at Baseline | 126.9 ± 18.0 | 174.4 ± 58.3 |
| Estimated GFR 1-Year Post-Transplant | 116.5 ± 26.7 | 134.5 ± 45.5 |
| Estimated GFR at 2-Years Post-Transplant | 113.4 ± 29.8 | 131.4 ± 47.1 |
Lung Function Outcomes will be summarized with mean and standard deviation at various timepoints. "Baseline" refers to pre-hydroxyurea (day -70 pre-transplant). Measures include: FEV1 (L), FVC (L), and TLC (L). All values in this table were reported by study site.
| liters (L) | Adult Stratum | Pediatric Stratum |
|---|---|---|
| FEV1 (L) at Baseline | 3.2 ± 0.3 | 1.7 ± 0.8 |
| FEV1 (L) at One Year Post-Transplant | 2.9 ± 0.7 | 1.9 ± 0.7 |
| FEV1 (L) at Two Years Post-Transplant | 2.9 ± 0.6 | 2.2 ± 0.6 |
| FVC (L) at Baseline | 4.1 ± 0.6 | 2.0 ± 1.0 |
| FVC (L) at One Year Post-Transplant | 3.5 ± 0.8 | 2.3 ± 0.9 |
| FVC (L) at Two Years Post-Transplant | 3.6 ± 0.7 | 2.7 ± 0.8 |
| TLC (L) at Baseline | 5.3 ± 0.9 | 3.2 ± 0.0 |
| TLC (L) at One Year Post-Transplant | 87.0 ± 18.4 | 3.3 ± 1.1 |
| TLC (L) at Two Years Post-Transplant | 87.6 ± 11.9 | 3.5 ± 1.1 |
Cardiac Function is measured through tricuspid regurgitant jet velocity (TRJV). It will be summarized with mean and standard deviation at Baseline, 1, and 2 years post-transplant. "Baseline" refers to pre-hydroxyurea (day -70 pre-transplant).
| m/sec | Adult Stratum | Pediatric Stratum |
|---|---|---|
| TRJV Value (m/sec) at Baseline | 1.7 ± 1.1 | 1.1 ± 1.6 |
| TRJV Value (m/sec) at 1-Year Post-Transplant | 1.5 ± 0.9 | 2.1 ± 0.6 |
| TRJV Value (m/sec) at 2-Years Post-Transplant | 1.6 ± 0.8 | 2.0 ± 0.6 |
Six Minute Walk Distance is measured in meters to assess how far a participant can walk in 6 minutes. It will be summarized with mean and standard deviation at Baseline, 1, and 2 years post-transplant. "Baseline" refers to pre-hydroxyurea (day -70 pre-transplant). Change from baseline will also be provided at the 1 and 2 years post-transplant visit.
| meters (m) | Adult Stratum | Pediatric Stratum |
|---|---|---|
| 6 Min Walk Distance (meters) at Baseline | 420.1 ± 170.1 | 447.5 ± 159.9 |
| 6 Min Walk Distance (meters) at 1-Year Post-Transplant | 363.0 ± 139.2 | 386.9 ± 164.6 |
| 6 Min Walk Distance (meters) at 2-Years Post-Transplant | 438.5 ± 212.5 | 449.6 ± 127.3 |
| 6 Min Walk Distance (meters) change from Baseline to One Year Post-Transplant | -29.0 ± 163.8 | -45.2 ± 170.6 |
| 6 Min Walk Distance (meters) change from Baseline to Two Years Post-Transplant | 75.9 ± 339.6 | -38.5 ± 174.9 |
Health-Related Quality of Life (QoL) assessed using the NIH's PROMIS short forms administered to English- and Spanish-speaking patients in the adult stratum. Questions about fatigue (short form 8a), pain interference (short form 8a), and pain intensity (short form 3a) are asked regarding the degree of difficulty in performing activities of daily living such as housework, social activities, and day to day activities. It is scored from 0 to 10 and converted to a standardized T-score with mean 50 and standard deviation 10. A higher T-score represents worse fatigue, pain interfering with more daily activities, and worse pain intensity.
| T-Score | Adult Stratum |
|---|---|
| Fatigue, Baseline | 52.5 ± 11.5 |
| Fatigue, 1 Year Post-transplant | 48.6 ± 10.6 |
| Fatigue, 2 Years Post-transplant | 44.9 ± 11.5 |
| Pain Interference, Baseline | 56.9 ± 10.9 |
| Pain Interference, 1 Year Post-transplant | 50.6 ± 10.7 |
| Pain Interference, 2 Years Post-transplant | 47.4 ± 9.2 |
| Pain Intensity, Baseline | 47.5 ± 11.1 |
| Pain Intensity, 1 Year Post-transplant | 42.1 ± 10.0 |
| Pain Intensity, 2 Years Post-transplant | 38.3 ± 7.9 |
Subjects who speak English and are 15 years or older will be asked to use a web-based diary to report pain intensity on a scale from 0 (no pain) to 10 (worst pain). Subject pain intensity score is reported as an aggregate of several AM and PM scores at Baseline, one year, and two years post-transplant timepoints.
| T-Score | Adult Stratum |
|---|---|
| Pain Intensity at Baseline | 3.4 ± 2.8 |
| Pain Intensity at 1 Year Post-transplant | 1.8 ± 2.6 |
| Pain Intensity at 2 Years Post-transplant | 2.2 ± 2.6 |
| Change in Pain Intensity from Baseline to 1 Year Post-transplant | -0.7 ± 1.6 |
| Change in Pain Intensity from Baseline to 2 Years Post-transplant | -0.6 ± 1.2 |
Cause of Death is tabulated by age strata. Deaths among participants who were not transplanted are included.
| Participants | Adult Stratum | Pediatric Stratum |
|---|---|---|
| Organ Failure | 1 | 1 |
| Acute Respiratory Distress Syndrome | 1 | 0 |
| Hemorrhage | 2 | 0 |
| Infection | 0 | 1 |
Number of participants who reported the Maximum Infection Severity of Grade 2 and Grade 3. Only grade 2 and grade 3 infections occurring post transplantation were reported on the study. Grade 2 and grade 3 infections are defined by the BMT CTN Technical MOP. Higher infection grade indicates worse infection severity. The infection grading criteria are published online (https://bmtctn.net/administrative-manual-procedures-moppolicy-guidelines). Severity of grade 1, 2 and 3 are described for bacterial, fungal, viral, parasitic, and nonmicrobiological infections. For example, grade 2 fungal infections are defined as candida esophagitis, or proven or probably fungal sinusistis confirmed radiologically without orbital, brain or bone involvement. Grade 3 fungal infections are defined as Fungemia including candidemia, Proven or probably invasive fungal infections, Disseminated infections with histoplasmosis, blastomycosis, coccidiomycosis, or Cryptococcus, or Pneumocystis jiroveci pneumonia.
| Participants | Adult Stratum | Pediatric Stratum |
|---|---|---|
| None (No Grade 2 or 3 Infection) | 16 | 16 |
| Grade 2 | 16 | 17 |
| Grade 3 | 10 | 6 |
The number of systemic infections is reported. Infections are categorized by infection type. A participant can report multiple types of infections, so the categories are not mutually exclusive for participants. All grade 2 and grade 3 infections, as defined by the BMT CTN Technical MOP, occurring post transplantation were reported on the study.
| infections | Adult Stratum | Pediatric Stratum |
|---|---|---|
| Bacterial | 41 | 25 |
| Viral | 36 | 33 |
| Fungal | 5 | 3 |
| Protozoal | 0 | 0 |
| Other | 3 | 1 |
Participants could be readmitted to a hospital for many reasons during follow-up. The causes of each readmission are tabulated by age strata. The protocol-specified scheduled transplant hospitalization is not included in this table.
| hospitalizations | Adult Stratum | Pediatric Stratum |
|---|---|---|
| Infection | 39 | 29 |
| Fever | 30 | 28 |
| GVHD | 3 | 13 |
| Nausea/Vomiting | 7 | 2 |
| Diarrhea | 4 | 2 |
| Organ Failure | 4 | 1 |
| Scheduled Procedure | 4 | 2 |
| Vaso-occlusive Pain Crisis (VOC) | 2 | 1 |
| Psychiatric | 2 | 0 |
| Increased Creatinine Grade >= 2 | 2 | 0 |
| Seizure | 1 | 0 |
| Trauma | 1 | 0 |
| Stroke | 1 | 0 |
| Other | 17 | 14 |
Average number of post-transplant follow-up days by age strata.
| days | Adult Stratum | Pediatric Stratum |
|---|---|---|
| Mean Follow-up Days Since Transplant | 753.0 ± 206.0 | 728.9 ± 246.8 |
Hematological Outcomes will be summarized with mean and standard deviation at various post-transplant timepoints. Measures in this table include: LDH (U/L)
| U/L | Adult Stratum | Pediatric Stratum |
|---|---|---|
| LDH (U/L) at Day 28 Post-Transplant Visit | 449.2 ± 254.8 | 435.7 ± 263.8 |
| LDH (U/L) at Day 100 Post-Transplant Visit | 520.3 ± 459.3 | 464.8 ± 238.4 |
| LDH (U/L) at 6 Months Post-Transplant Visit | 550.1 ± 597.2 | 484.5 ± 414.4 |
| LDH (U/L) at 1 Year Post-Transplant Visit | 434.8 ± 495.8 | 313.5 ± 130.6 |
| LDH (U/L) at 2 Years Post-Transplant Visit | 404.0 ± 566.0 | 278.8 ± 82.6 |
Hematological Outcomes will be summarized with mean and standard deviation at various timepoints. Baseline timepoint is pre-thymoglobulin (day -7 pre-transplant). Measures in this table include: Bilirubin (mg/dL)
| mg/dL | Adult Stratum | Pediatric Stratum |
|---|---|---|
| Bilirubin (mg/dL) at Pre-Thymoglobulin visit | 0.8 ± 1.6 | 0.5 ± 0.5 |
| Bilirubin (mg/dL) at Day 28 Post-Transplant visit | 0.3 ± 0.2 | 0.3 ± 0.5 |
| Bilirubin (mg/dL) at Day 100 Post-Transplant visit | 0.2 ± 0.4 | 0.3 ± 0.3 |
| Bilirubin (mg/dL) at 6 Months Post-Transplant visit | 0.2 ± 0.3 | 0.4 ± 1.0 |
| Bilirubin (mg/dL) at 1 Year Post-Transplant visit | 0.2 ± 0.3 | 0.6 ± 1.2 |
| Bilirubin (mg/dL) at 2 Years Post-Transplant visit | 0.2 ± 0.3 | 0.3 ± 0.3 |
Hematological Outcomes will be summarized with mean and standard deviation at various timepoints. Baseline refers to pre-thymoglobulin (day -7 pre-transplant). Measures in this table include: Serum Ferritin Level (ng/dL)
| ng/dL | Adult Stratum | Pediatric Stratum |
|---|---|---|
| Summary of Serum Ferritin | 1205.2 ± 1613.3 | 1617.8 ± 1491.8 |
Renal Function Outcomes will be summarized with mean and standard deviation at various timepoints. "Baseline" refers to pre-thymoglobulin (day -7 pre-transplant). Measures include: Creatinine Clearance (mL/min).
| mL/min | Adult Stratum | Pediatric Stratum |
|---|---|---|
| Creatinine Clearance (mL/min) at Baseline | 129.8 ± 63.9 | 193.3 ± 124.7 |
| Creatinine Clearance (mL/min) at 1-Year Post-Transplant | 148.7 ± 54.9 | 126.6 ± 596.2 |
| Creatinine Clearance (mL/min) at 2-Years Post-Transplant | 130.2 ± 51.2 | 221.9 ± 106.9 |
Renal Function Outcomes will be summarized with mean and standard deviation at various timepoints. Measures include: Urine Albumin Creatine Ratio (mg/g).
| mg/g | Adult Stratum | Pediatric Stratum |
|---|---|---|
| Urine Albumin Creatine Ratio (mg/g) at 1-Year Post-Transplant | 47.0 ± 55.0 | 57.4 ± 73.8 |
| Urine Albumin Creatine Ratio (mg/g) at 2-Years Post-Transplant | 24.4 ± 43.9 | 61.8 ± 92.0 |
Lung Function Outcomes will be summarized with mean and standard deviation at various timepoints. "Baseline" refers to pre-hydroxyurea (day -70 pre-transplant). Measures include: FEV1 (percent predicted), FVC (percent predicted), FEV1/FVC (percent predicted), and TLC (percent predicted). All values in this table, including percent predicted, were reported by study site.
| percent predicted | Adult Stratum | Pediatric Stratum |
|---|---|---|
| FEV1 (percent predicted) at Baseline | 83.6 ± 7.4 | 78.0 ± 23.8 |
| FEV1 (percent predicted) at One Year Post-Transplant | 83.9 ± 18.5 | 84.6 ± 19.0 |
| FEV1 (percent predicted) at Two Years Post-Transplant | 83.3 ± 21.5 | 82.7 ± 22.8 |
| FVC (percent predicted) at Baseline | 91.1 ± 8.2 | 86.0 ± 30.3 |
| FVC (percent predicted) at One Year Post-Transplant | 89.1 ± 18.8 | 87.6 ± 17.6 |
| FVC (percent predicted) at Two Years Post-Transplant | 88.2 ± 22.6 | 87.7 ± 23.4 |
| TLC (percent predicted) at Baseline | 86.6 ± 9.0 | 93.0 ± 0.0 |
| TLC (percent predicted) at One Year Post-Transplant | 79.5 ± 21.2 | 76.4 ± 26.8 |
| TLC (percent predicted) at Two Years Post-Transplant | 82.0 ± 20.8 | 77.4 ± 25.8 |
Lung Function Outcomes will be summarized with mean and standard deviation at various timepoints. "Baseline" refers to pre-hydroxyurea (day -70 pre-transplant). Measures include: FEV1/FVC (ratio of Liters). All values in this table were reported by study site.
| ratio of liters (L) | Adult Stratum | Pediatric Stratum |
|---|---|---|
| FEV1/FVC Ratio at Baseline | 0.8 ± 0.1 | 0.9 ± 0.0 |
| FEV1/FVC Ratio at One Year Post-Transplant | 0.8 ± 0.1 | 0.8 ± 0.2 |
| FEV1/FVC Ratio at Two Years Post-Transplant | 0.8 ± 0.1 | 1.2 ± 0.9 |
Lung Function Outcomes will be summarized with mean and standard deviation at various timepoints. "Baseline" refers to pre-hydroxyurea (day -70 pre-transplant). Measures include: DLCO (%) and Oxygen Saturation (%). All values in this table were reported by study site.
| percent of lab measure | Adult Stratum | Pediatric Stratum |
|---|---|---|
| DLCO (%) at Baseline | 81.8 ± 27.1 | 82.8 ± 18.0 |
| DLCO (%) at One Year Post-Transplant | 71.9 ± 28.7 | 79.4 ± 28.7 |
| DLCO (%) at Two Years Post-Transplant | 71.7 ± 29.9 | 77.0 ± 26.7 |
| Oxygen Saturation (%) at Baseline | 97.0 ± 3.1 | 98.7 ± 1.4 |
| Oxygen Saturation (%) at One Year Post-Transplant | 96.6 ± 4.5 | 97.5 ± 4.5 |
| Oxygen Saturation (%) at Two Years Post-Transplant | 96.3 ± 6.0 | 98.6 ± 1.5 |
Lung Function Outcomes will be summarized with mean and standard deviation at various timepoints. "Baseline" refers to pre-hydroxyurea (day -70 pre-transplant). Measures include: FEV1/FVC (ratio of percent predicted). All values in this table were reported by study site.
| ratio of percent predicted | Adult Stratum | Pediatric Stratum |
|---|---|---|
| FEV1/FVC Percent Predicted at Baseline | 85.4 ± 9.4 | 97.5 ± 3.5 |
| FEV1/FVC Percent Predicted at One Year Post-Transplant | 87.0 ± 18.4 | 89.1 ± 8.9 |
| FEV1/FVC Percent Predicted at Two Years Post-Transplant | 87.6 ± 11.9 | 89.4 ± 9.9 |
Collected over Deaths were assessed for all enrolled participants. Adverse Events were assessed for all participants through the follow-up of the study, an expected 2 years post-transplant.. Non-serious events are listed at a 0% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Adult Stratum | 4/54 (7.4%) | 13/54 (24.1%) | 8/54 (14.8%) |
| Pediatric Stratum | 2/41 (4.9%) | 12/41 (29.3%) | 10/41 (24.4%) |
| Event | Adult Stratum | Pediatric Stratum |
|---|---|---|
| COVID-19Infections and infestations | 0/54 | 2/41 |
| Septic shockInfections and infestations | 1/54 | 2/41 |
| SepsisInfections and infestations | 2/54 | 0/41 |
| Adenovirus infectionInfections and infestations | 0/54 | 1/41 |
| Anaphylactic reactionImmune system disorders | 0/54 | 1/41 |
| Fungal infectionInfections and infestations | 0/54 | 1/41 |
| Haemophagocytic lymphohistiocytosisImmune system disorders | 0/54 | 1/41 |
| HaemothoraxRespiratory, thoracic and mediastinal disorders | 0/54 | 1/41 |
| Mental status changesPsychiatric disorders | 0/54 | 1/41 |
| MucormycosisInfections and infestations | 0/54 | 1/41 |
| Event | Adult Stratum | Pediatric Stratum |
|---|---|---|
| Alanine aminotransferaseInvestigations | 6/54 | 7/41 |
| Aspartate aminotransferaseInvestigations | 6/54 | 6/41 |
| Blood bilirubinInvestigations | 0/54 | 5/41 |
| Blood alkaline phosphotaseInvestigations | 1/54 | 2/41 |
| Chronic kidney diseaseRenal and urinary disorders | 2/54 | 0/41 |
| Thrombotic microangiopathyBlood and lymphatic system disorders | 1/54 | 0/41 |
Participants who enrolled and received a haploidentical bone marrow transplant (BMT).
| Age, Continuous(Years) | Adult Stratum | Pediatric Stratum | Total |
|---|---|---|---|
| Mean | 24.1 ± 6.9 | 11.4 ± 2.9 | 18.0 ± 8.3 |
| Sex: Female, Male(Participants) | Adult Stratum | Pediatric Stratum | Total |
|---|---|---|---|
| Female | 17 | 15 | 32 |
| Male | 25 | 24 | 49 |
| Ethnicity (NIH/OMB)(Participants) | Adult Stratum | Pediatric Stratum | Total |
|---|---|---|---|
| Hispanic or Latino | 1 | 1 | 2 |
| Not Hispanic or Latino | 41 | 38 | 79 |
| Unknown or Not Reported | 0 | 0 | 0 |
| Race (NIH/OMB)(Participants) | Adult Stratum | Pediatric Stratum | Total |
|---|---|---|---|
| American Indian or Alaska Native | 0 | 0 | 0 |
| Asian | 0 | 0 | 0 |
| Native Hawaiian or Other Pacific Islander | 0 | 0 | 0 |
| Black or African American | 40 | 36 | 76 |
| White | 2 | 1 | 3 |
| More than one race | 0 | 0 | 0 |
| Unknown or Not Reported | 0 | 2 | 2 |
| Disease Type(Participants) | Adult Stratum | Pediatric Stratum | Total |
|---|---|---|---|
| Hemoglobin SS Disease | 37 | 38 | 75 |
| Hemoglobin SBeta0 Thalassemia | 2 | 1 | 3 |
| Hemoglobin SC | 1 | 0 | 1 |
| Hemoglobin SBeta+ Thalassemia | 2 | 0 | 2 |
| Karnofsky/Lansky Performance Score(Participants) | Adult Stratum | Pediatric Stratum | Total |
|---|---|---|---|
| 60-80 | 12 | 7 | 19 |
| 90-100 | 30 | 32 | 62 |
| Calculated HLA Match Score(Participants) | Adult Stratum | Pediatric Stratum | Total |
|---|---|---|---|
| 4/8 | 31 | 32 | 63 |
| 5/8 | 6 | 5 | 11 |
| 6/8 | 5 | 2 | 7 |
| Donor Gender(Participants) | Adult Stratum | Pediatric Stratum | Total |
|---|---|---|---|
| Female | 23 | 17 | 40 |
| Male | 19 | 22 | 41 |
PDFs hosted by the registry, ClinicalTrials.gov.
Plan to share: Yes — Results will be published in a manuscript and supporting information submitted to NIH BioLINCC (including data dictionaries, case report forms, data submission documentation, documentation for outcomes dataset, etc where indicated).
Supporting information: Study protocol, Informed consent form
Time frame: Within 6 months of official study closure at participating sites.
Access criteria: Available to the public
Data sharing details ↗ · biolincc.nhlbi.nih.gov
This study is completed, as verified in Jun 2026. You cannot join it, but the record below documents what was studied.
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Studies of the drugs tested here: Hydroxyurea · Thymoglobulin · Thiotepa · Fludarabine · Cyclophosphamide · Mesna
Medical College of Wisconsin →