A Phase 2 interventional study of e-ATG and Immunosuppression Only Conditioning in Lymphoproliferative Disorders, Autoimmune Lymphoproliferative and Primary T-cell Immunodeficiency Disorders, sponsored by National Cancer Institute (NCI). Active, not recruiting at 2 sites in United States. Open to participants aged 4 Years and older, including healthy volunteers. Per ClinicalTrials.gov, last updated 2026-03-17.
Sponsored by National Cancer Institute (NCI) · Phase 2, Interventional, and Treatment
Background:
Blood stem cells in the bone marrow make all the cells to normally defend a body against disease. Allogeneic blood or marrow transplant is when these stem cells are transferred from one person to another. Researchers think this treatment can provide a new, healthy immune system to correct T-cell problems in some people.
Objective:
To see if allogeneic blood or bone marrow transplant is safe and effective in treating people with T-cell problems.
Eligibility:
Donors: Healthy people ages 4 and older
Recipients: People the same age with abnormal T-cell function causing health problems
Design:
All participants will be screened with:
Donors will also have an electrocardiogram and chest x-ray. They may have veins tested or a pre-anesthesia test.
Recipients will also have lung tests.
Some participants will have scans and/or bone marrow collected by needle in the hip bones.
Donors will learn about medicines and activities to avoid and repeat some screening tests.
Some donors will stay in the hospital overnight and have bone marrow collected with anesthesia.
Other donors will get shots for several days to stimulate cells. They will have blood removed by plastic tube (IV) in an arm vein. A machine will remove stem cells and return the rest of the blood to the other arm.
Recipients will have:
Background:
Primary Objective:
- Separately by arm: To estimate the percentage of recipients with >50% donor T cell chimerism and graft-failure free survival at day +180 post-HCT
Eligibility:
TCP/D deemed to be of sufficient past severity to warrant HCT that meets at least one of the criteria below:
Design:
RIC arm: e-ATG 40 mg/kg/day IV on days -14 and -13, pentostatin 4 mg/m\^2/day IV on days -11 and -7, low-dose cyclophosphamide orally daily on days -11 through -4; busulfan IV, pharmacokinetically dosed, on days -3 and -2.
-- Subjects will be assigned to the IOC arm if there is significant end-organ dysfunction present and it is felt that a conditioning regimen that includes busulfan would likely be associated with intolerable or life-threatening toxicities for the subject. Subjects will also be assigned to the IOC arm if they possess a deoxyribonucleic acid (DNA) repair defect, telomere maintenance defect, or familial cancer predisposition syndrome that necessitates limiting chemotherapy as much as possible to prevent future cancer risk.
Graft-versus-host disease (GVHD) prophylaxis:
T-cell proliferation and/or dysregulation (TCP/D) deemed to be of sufficient past severity to warrant hematopoietic cell transplantation (HCT) that meets at least one of the criteria below:
Adequate end-organ function, as measured by:
EXCLUSION CRITERIA - RECIPIENT:
INCLUSION CRITERIA RELATED DONOR
EXCLUSION CRITERIA - RELATED DONOR:
-None
INCLUSION CRITERIA - UNRELATED DONOR:
-Unrelated donors will be evaluated in accordance with existing National Marrow Donor Program (NMDP) Standard Policies and Procedures, available at: http://bethematch.org/About-Us/Global- transplant-network/Standards/, except for the additional requirement of EBV serostatus testing for clinical purposes of donor selection. Note that participation in this study is offered to all unrelated donors but not required for clinical donation, so it is possible that not all unrelated donors will enroll on this study. Unrelated donors only enroll if they contribute research specimens, which is optional.
EXCLUSION CRITERIA - UNRELATED DONOR:
-Unrelated donors: failure to qualify as a National Marrow Donor Program (NMDP) donor per current NMDP Standards, available at: http://bethematch.org/About-Us/Global-transplant-network/Standards/. Exceptions to donor eligibility (e.g. foreign travel, tattoos) do not automatically exclude the donor and will be reviewed by the PI.
Reduced Intensity Conditioning Arm.
Drug: e-ATG · Procedure: Immunosuppression Only Conditioning · Procedure: Reduced Intensity Conditioning · Drug: GVHD Prophylaxis · Procedure: Allogeneic HSC · Drug: Bisulfan · Drug: Prednisone · Drug: Cyclophosphamide · Drug: MMF · Drug: Mesna · Drug: Tacrolimus · Drug: Pentostatin · Diagnostic Test: PFTs · Diagnostic Test: DEXA · Procedure: Bone Marrow Aspirate & Biopsy · Diagnostic Test: EKG · Diagnostic Test: 2D ECHO
Immunosuppression Only Conditioning Arm.
Drug: e-ATG · Procedure: Immunosuppression Only Conditioning · Drug: GVHD Prophylaxis · Procedure: Allogeneic HSC · Drug: Bisulfan · Drug: Prednisone · Drug: Cyclophosphamide · Drug: MMF · Drug: Mesna · Drug: Tacrolimus · Drug: Pentostatin · Diagnostic Test: PFTs · Diagnostic Test: DEXA · Procedure: Bone Marrow Aspirate & Biopsy · Diagnostic Test: EKG · Diagnostic Test: 2D ECHO
Healthy Donor- Donors for recipients in arm 1 or arm 2.
During Immunosuppression Only Conditioning (IOC) and Reduced Intensity Conditioning (RIC).
Also known as: Antithymocyte Globulin (Equine), Atgam
Equine anti-thymocyte globulin (e-ATG) 40 mg/kg intravenous (IV) once daily for days -14 and -13. Prednisone: Tapering doses, given orally daily, and given prior to each daily dose of e-ATG on days -14 and -13, Pentostatin:4 mg/m\^2/day IV on days -9 and -5, cyclophosphamide:5 mg/kg orally daily on days -9 through -2.
Also known as: IOC
Equine anti-thymocyte globulin (e-ATG) 40 mg/kg intravenous (IV) once daily for days -14 and -13. Prednisone: Tapering doses, given orally daily, and given prior to each daily dose of e-ATG on days -14 and -13, Pentostatin:4 mg/m\^2/day IV on days -11 and -7, cyclophosphamide: 5 mg/kg orally daily on days -11 through -4, Busulfan IV, pharmacokinetically dosed, on days -3 and -2.
Also known as: RIC
High-dose, post-transplantation cyclophosphamide (PTCy) 25-50 mg/kg on days +3 and +4, Mesna: 25-50 mg/kg weight-based dosing, Tacrolimus 0.02 mg/kg on days +5 through +90, and mycophenolate mofetil (MMF) 15 mg/kg on days +5 through +25.
Also known as: Graft-versus-host disease prophylaxis
Stem cell transplant
Also known as: Allogeneic hematopoietic stem cell (HSC)
During Reduced Intensity Conditioning (RIC).
Also known as: Busulfex, Myleran
During Immunosuppression Only Conditioning (IOC) and Reduced Intensity Conditioning (RIC).
Also known as: Deltasone
During Immunosuppression Only Conditioning (IOC), Reduced Intensity Conditioning (RIC) and Graft-versus-host disease prophylaxis (GVHD).
Also known as: Cytoxan, Neosar
During Graft-versus-host disease prophylaxis (GVHD).
Also known as: Mycophenolate mofetil, CellCept
During Graft-versus-host disease prophylaxis (GVHD).
Also known as: Mesnex, Uromitexan
During Graft-versus-host disease prophylaxis (GVHD).
Also known as: Prograf
During Immunosuppression Only Conditioning (IOC) and Reduced Intensity Conditioning (RIC).
Also known as: Nipent, Deoxycoformycin
Screening ≤4 weeks pretreatment (rx), Day +180 (≤ 14 days), Day +36 (± 21 days), Day +548 (18 months) (± 28 days), and at 2 years and yearly thereafter through +5 years (± 56 days).
Also known as: Pulmonary function tests
Baseline, Day +365 (± 21 days), at 2 years and yearly thereafter through +5 years (± 56 days), and as clinically indicated after hematopoietic cell transplant (HCT).
Also known as: Dual-energy x-ray absorptiometry
Baseline, Day +60 (± 3 days) and Day +365 (±21 days).
Also known as: BM aspirate & bx
Baseline
Also known as: Electrocardiogram
Screening ≤4 weeks pretreatment (rx), Day +180 (≤ 14 days), Day +36 (± 21 days), Day +548 (18 months) (± 28 days), and at 2 years and yearly thereafter through +5 years (± 56 days).
Also known as: 2-dimensional echocardiogram
Percentage of Recipients Who Are Alive With >50% Donor T Cell Chimerism and Graft-failure Free at 180 Days Post Hematopoietic Cell Transplant (HCT) Reported With an 80% Confidence Interval
Percentage of recipients with \> 50% donor T cell chimerism and without death or graft failure. A graft failure event is defined as either primary or secondary graft failure, in the absence of a recurrent marrow malignancy.
Time frame: Day +180 post-HCT
Percentage of Recipients Who Are Alive With >50% Donor T Cell Chimerism and Graft-failure Free at 180 Days Post Hematopoietic Cell Transplant (HCT) Reported With a 95% Confidence Interval
Percentage of recipients with \> 50% donor T cell chimerism and without death or graft failure. A graft failure event is defined as either primary or secondary graft failure, in the absence of a recurrent marrow malignancy.
Time frame: Day +180 post -HCT
Cumulative Incidence of Transplant-related Mortality
Cumulative incidence of transplant-related mortality at 180 days and 1-year post-transplant. Transplant related mortality is defined as any death that occurs outside the setting of the hematopoietic cell transplant (HCT) post-allogeneic relapse of a pre-transplant malignancy or lymphoid disorder.
Time frame: Day +180, and 1-year post-transplant
Cumulative Incidence of Secondary Graft Failure
Cumulative incidence of secondary graft failure at 1-year post-transplant. Secondary graft failure is defined as initial blood or marrow donor myeloid chimerism ≥5%, declining to \<5% on subsequent measurements. \<5% indicates graft failure (undesirable outcome).
Time frame: 1-, 3-, and 5-years post-transplant
Percent Probability of Overall Survival (OS)
OS is defined as the time in whole days from hematopoietic cell transplantation (HCT) to death from any cause, with surviving recipients censored at the time of last contact.
Time frame: 1-, 3-, and 5-years post-transplant
Percentage of Participants Who Achieve Chimerism at Stated Days Between Those Who Have Failed by Day 60 or Have Not
Percentage of participants who achieve early chimerism (\>50% T cell chimerism) at stated days between those who have failed by day 60 or have not. Comparison to be performed using Fisher's exact test. Chimerism is the percentage of donor cells in the peripheral blood.
Time frame: Day +21, +28, +35, +42, and +60 after hematopoietic cell transplant (HCT)
Percentage of Donor T-cell Populations at Days +28, +42, +60, +100, +180, and 1-year Post Hematopoietic Cell Transplant (HCT)
The percentage of donor T-cell populations at days +28, +42, +60, +100, +180, and 1-year post hematopoietic cell transplant.
Time frame: Days +28, +42, +60, +100, +180, and 1-year post hematopoietic cell transplant
Cumulative Incidence of Chronic Graft-versus-host Disease (cGVHD)
Cumulative incidence curves of chronic graft versus host disease and two-sided 95% confidence intervals at 1 and 2-years post -transplant. cGVHD was scored according to the 2014 National Institutes of Health (NIH) Consensus Criteria for Clinical Trials in Chronic GVHD. Eight organs will be scored on a 0-3 scale.
Time frame: 1 and 2-years post-transplant
Cumulative Incidence of Acute Graft-versus-host Disease (aGVHD) at 1 Year
Cumulative incidence curves of acute graft versus host disease and two-sided 95% confidence intervals at 1-year post transplant according to Keystone Criteria of the 1994 Consensus Conference on Acute GVHD Grading. Acute GVHD is defined as any grade, grade 2, 3, or 4 and grade 3-4 acute GVHD. The Keystone criteria provide the basis for grading acute GVHD as follows: Organ-Specific Staging: Each affected organ (skin, liver, gut) is staged 0 (absent) to 4 (severe). Overall Grading (I-IV): Based on the most severe organ involvement. Skin (Grade 0-4): Based on % body surface area (BSA) involvement (e.g., \<25% for Grade 1, \>50% for Grade 3, bullae for Grade 4). Liver (Grade 0-4): Based on total serum bilirubin levels (e.g., 2-2.9 mg/dL for Grade 1, \>15 mg/dL for Grade 4). Gut (Grade 0-4): Based on diarrhea volume and severity (e.g., \>500 mL/day for Grade 1, \>2000 mL/day or ileus/severe pain for Grade 4). Upper GI: Included for classification, with specific criteria for staging.
Time frame: 1-year post-transplant
Percent Probability Event-free Survival (EFS)
EFS is defined as the time from transplant to death of any cause or other event, including disease relapse, graft failure, grade 3-4 acute graft versus host disease (GVHD), chronic GVHD requiring systemic therapy, or receipt of post-transplant donor cell infusion.
Time frame: 1, 3, and 5-years post-transplant
Cumulative Incidence of Primary Graft Failure at Day +60
Primary graft failure at day +60 estimated using cumulative incidence curves and 95% two-sided confidence intervals. Primary graft failure is defined as \< 5% donor myeloid chimerism in blood and/or bone marrow on all evaluations up to and including day +60, in the absence of a recurrent marrow malignancy.
Time frame: Day +60
Percentage of Participants With Lymphoproliferative Disease/Lymphoma Relapse at 1, 3, and 5-years Post-hematopoietic Cell Transplant (HCT)
Lymphoproliferative disease/lymphoma relapse at 1, 3, and 5-years post-HCT estimated using cumulative incidence curves and two-sided 95% confidence intervals at each timepoint.
Time frame: 1, 3, and 5 years post-HCT
Percent Probability Graft Versus Host Disease (GVHD)-Free Graft Failure-free Survival (GGFS)
Probabilities of GGFS were estimated using the Kaplan-Meier method. GGFS is
Time frame: 1, 3, and 5 years post-hematopoietic cell transplant (HCT)
Percent Probability of Graft Versus Host Disease (GVHD)-Free Relapse-free Survival (GRFS)
GRFS was estimated using the Kaplan-Meier method. Relapse free survival is
Time frame: 1, 3 and 5-years post-hematopoietic cell transplant (HCT)
Cumulative Incidences of Cytomegalovirus (CMV), BK Virus (BK), Adenovirus, Human Herpes Virus 6 (HHV6), JC Virus (JCV), and Epstein-Barr Virus (EBV) Detection in Blood at Day +100 Post-HCT
Cumulative incidences of CMV, BK, adenovirus, HHV6, JCV, and EBV detection in blood at day +100 post-HCT estimated using cumulative incidence curves along with two-sided 95% confidence intervals.
Time frame: day +100 post-HCT
Percentage of Donor B-cell Populations at Days +28, +42, +60, +100, +180, and 1-year Post-transplant
The percentage of donor B-cell populations at days +28, +42, +60, +100, +180, and 1-year post-transplant.
Time frame: Days +28, +42, +60, +100, +180, and 1-year post-transplant
Percentage of Donor Natural Killer (NK-) Cell Populations at Days +28, +42, +60, +100, +180, and 1-year Post Transplant
Percentage of donor natural killer (NK-) cell populations at days +28, +42, +60, +100, +180, and 1-year post transplant.
Time frame: Days +28, +42, +60, +100, +180, and 1-year post transplant
Percentage of Donor Myeloid Cell Populations at Days +28, +42, +60, +100, +180, and 1-year Post Transplant
Percentage of donor myeloid cell populations at days +28, +42, +60, +100, +180, and 1-year post transplant.
Time frame: Days +28, +42, +60, +100, +180, and 1-year post transplant
Number of Participants With Serious and/or Non-serious Adverse Events Assessed by the Common Terminology Criteria for Adverse Events (CTCAE v5.0)
Here is the number of participants with serious and/or non-serious adverse events assessed by the Common Terminology Criteria for Adverse Events (CTCAE v5.0). A non-serious adverse event is any untoward medical occurrence. A serious adverse event is an adverse event or suspected adverse reaction that results in death, a life-threatening adverse drug experience, hospitalization, disruption of the ability to conduct normal life functions, congenital anomaly/birth defect or important medical events that jeopardize the patient or subject and may require medical or surgical intervention to prevent one of the previous outcomes mentioned.
Time frame: Conditioning start until return to baseline/stabilization, 30 days post-therapy end, removal from therapy, or off study, whichever comes first for all AEs, followed by AE collection per principal investigator discretion, on average 2 years.
| Milestone | Reduced Intensity Conditioning+Allogeneic Hematopoietic Cell Transplant + Graft-versus-Host Disease | Immunosuppression-Only Conditioning + Allo Hematopoietic Cell Transplant +Graft-versus-Host Disease | 7-8/8 Human Leukocyte Antigen (HLA)-Matched Related or Unrelated, or HLA-haploidentical Donor | Participants Enrolled But Not Treated | National Marrow Donor Program (NMDP) Donors |
|---|---|---|---|---|---|
| Started | 25 | 4 | 10 | 20 | 12 |
| Completed follow-up period | 9 | 1 | 0 | 0 | 6 |
| Completed | 9 | 1 | 3 | 0 | 6 |
| Not completed | 16 | 3 | 7 | 20 | 6 |
| Withdrew: Screen failures | 0 | 0 | 0 | 20 | 0 |
| Withdrew: Death | 4 | 2 | 0 | 0 | 0 |
| Withdrew: Donor is not removed until recipient completes follow up | 0 | 0 | 7 | 0 | 6 |
| Withdrew: Graft failure | 0 | 1 | 0 | 0 | 0 |
| Withdrew: Less than 5 years post-transplant | 12 | 0 | 0 | 0 | 0 |
Percentage of recipients with \> 50% donor T cell chimerism and without death or graft failure. A graft failure event is defined as either primary or secondary graft failure, in the absence of a recurrent marrow malignancy.
| Percentage of participants | Reduced Intensity Conditioning+Allogeneic Hematopoietic Cell Transplant + Graft-versus-Host Disease | Immunosuppression-Only Conditioning + Allo Hematopoietic Cell Transplant +Graft-versus-Host Disease |
|---|---|---|
| Percentage of Recipients Who Are Alive With >50% Donor T Cell Chimerism and Graft-failure Free at 180 Days Post Hematopoietic Cell Transplant (HCT) Reported With an 80% Confidence Interval | 80 (69.7 to 90.3) | 25 (-2.7 to 52.7) |
Percentage of recipients with \> 50% donor T cell chimerism and without death or graft failure. A graft failure event is defined as either primary or secondary graft failure, in the absence of a recurrent marrow malignancy.
| Percentage of participants | Reduced Intensity Conditioning+Allogeneic Hematopoietic Cell Transplant + Graft-versus-Host Disease | Immunosuppression-Only Conditioning + Allo Hematopoietic Cell Transplant +Graft-versus-Host Disease |
|---|---|---|
| Percentage of Recipients Who Are Alive With >50% Donor T Cell Chimerism and Graft-failure Free at 180 Days Post Hematopoietic Cell Transplant (HCT) Reported With a 95% Confidence Interval | 80 (64.3 to 95.7) | 25 (-17.4 to 67.4) |
Cumulative incidence of transplant-related mortality at 180 days and 1-year post-transplant. Transplant related mortality is defined as any death that occurs outside the setting of the hematopoietic cell transplant (HCT) post-allogeneic relapse of a pre-transplant malignancy or lymphoid disorder.
| percent of participants | Reduced Intensity Conditioning+Allogeneic Hematopoietic Cell Transplant + Graft-versus-Host Disease | Immunosuppression-Only Conditioning + Allo Hematopoietic Cell Transplant +Graft-versus-Host Disease |
|---|---|---|
| Day +180 post-transplant | 12 | 50 |
| 1-year post-transplant | 12 | 50 |
Cumulative incidence of secondary graft failure at 1-year post-transplant. Secondary graft failure is defined as initial blood or marrow donor myeloid chimerism ≥5%, declining to \<5% on subsequent measurements. \<5% indicates graft failure (undesirable outcome).
Results for this outcome have not been posted.
OS is defined as the time in whole days from hematopoietic cell transplantation (HCT) to death from any cause, with surviving recipients censored at the time of last contact.
Results for this outcome have not been posted.
Percentage of participants who achieve early chimerism (\>50% T cell chimerism) at stated days between those who have failed by day 60 or have not. Comparison to be performed using Fisher's exact test. Chimerism is the percentage of donor cells in the peripheral blood.
| Percentage of participants | Arm 1: Participants Who Achieved >50% T Cell Chimerism by Day +60 | Arm 1: Participants Who Failed to Achieve >50% T Cell Chimerism by Day +60 | Arm 2: Participants Who Achieved >50% T Cell Chimerism by Day +60 | Arm 2: Participants Who Failed to Achieve >50% T Cell Chimerism by Day +60 |
|---|---|---|---|---|
| (>50% T cell chimerism at +21 days post-HCT | 69 | — | 100 | NA |
| (>50% T cell chimerism at +28 days post-HCT | 85 | — | 100 | — |
| (>50% T cell chimerism at +35 days post-HCT | 83 | — | 100 | NA |
| (>50% T cell chimerism at +42 days post-HCT | 84 | — | 100 | — |
The percentage of donor T-cell populations at days +28, +42, +60, +100, +180, and 1-year post hematopoietic cell transplant.
| percentage T-cells | Reduced Intensity Conditioning+Allogeneic Hematopoietic Cell Transplant + Graft-versus-Host Disease | Immunosuppression-Only Conditioning + Allo Hematopoietic Cell Transplant +Graft-versus-Host Disease |
|---|---|---|
| Day +28 after HCT | 91 (9 to 100) | 95 (95 to 95) |
| Day +42 after HCT | 97 (0 to 100) | 99.5 (99 to 100) |
| Day +60 after HCT | 97.5 (0 to 100) | 98 (0 to 100) |
| Day +100 after HCT | 98 (56 to 100) | 98 (98 to 100) |
| Day +180 after HCT | 100 (67 to 100) | 100 (100 to 100) |
| Day +365 after HCT | 100 (79 to 100) | 90 (90 to 90) |
Cumulative incidence curves of chronic graft versus host disease and two-sided 95% confidence intervals at 1 and 2-years post -transplant. cGVHD was scored according to the 2014 National Institutes of Health (NIH) Consensus Criteria for Clinical Trials in Chronic GVHD. Eight organs will be scored on a 0-3 scale.
Results for this outcome have not been posted.
Cumulative incidence curves of acute graft versus host disease and two-sided 95% confidence intervals at 1-year post transplant according to Keystone Criteria of the 1994 Consensus Conference on Acute GVHD Grading. Acute GVHD is defined as any grade, grade 2, 3, or 4 and grade 3-4 acute GVHD. The Keystone criteria provide the basis for grading acute GVHD as follows: Organ-Specific Staging: Each affected organ (skin, liver, gut) is staged 0 (absent) to 4 (severe). Overall Grading (I-IV): Based on the most severe organ involvement. Skin (Grade 0-4): Based on % body surface area (BSA) involvement (e.g., \<25% for Grade 1, \>50% for Grade 3, bullae for Grade 4). Liver (Grade 0-4): Based on total serum bilirubin levels (e.g., 2-2.9 mg/dL for Grade 1, \>15 mg/dL for Grade 4). Gut (Grade 0-4): Based on diarrhea volume and severity (e.g., \>500 mL/day for Grade 1, \>2000 mL/day or ileus/severe pain for Grade 4). Upper GI: Included for classification, with specific criteria for staging.
| percentage | Reduced Intensity Conditioning+Allogeneic Hematopoietic Cell Transplant + Graft-versus-Host Disease | Immunosuppression-Only Conditioning + Allo Hematopoietic Cell Transplant +Graft-versus-Host Disease |
|---|---|---|
| Cumulative Incidence of Acute Graft-versus-host Disease (aGVHD) at 1 Year | 32 (15.2 to 50.2) | 50 (5.8 to 84.5) |
EFS is defined as the time from transplant to death of any cause or other event, including disease relapse, graft failure, grade 3-4 acute graft versus host disease (GVHD), chronic GVHD requiring systemic therapy, or receipt of post-transplant donor cell infusion.
Results for this outcome have not been posted.
Primary graft failure at day +60 estimated using cumulative incidence curves and 95% two-sided confidence intervals. Primary graft failure is defined as \< 5% donor myeloid chimerism in blood and/or bone marrow on all evaluations up to and including day +60, in the absence of a recurrent marrow malignancy.
| percentage | Reduced Intensity Conditioning+Allogeneic Hematopoietic Cell Transplant + Graft-versus-Host Disease | Immunosuppression-Only Conditioning + Allo Hematopoietic Cell Transplant +Graft-versus-Host Disease |
|---|---|---|
| Cumulative Incidence of Primary Graft Failure at Day +60 | 0 (0 to 0) | 50 (15.5 to 94.2) |
Lymphoproliferative disease/lymphoma relapse at 1, 3, and 5-years post-HCT estimated using cumulative incidence curves and two-sided 95% confidence intervals at each timepoint.
Results for this outcome have not been posted.
Probabilities of GGFS were estimated using the Kaplan-Meier method. GGFS is
Results for this outcome have not been posted.
GRFS was estimated using the Kaplan-Meier method. Relapse free survival is
Results for this outcome have not been posted.
Cumulative incidences of CMV, BK, adenovirus, HHV6, JCV, and EBV detection in blood at day +100 post-HCT estimated using cumulative incidence curves along with two-sided 95% confidence intervals.
| percent | Reduced Intensity Conditioning+Allogeneic Hematopoietic Cell Transplant + Graft-versus-Host Disease | Immunosuppression-Only Conditioning + Allo Hematopoietic Cell Transplant +Graft-versus-Host Disease |
|---|---|---|
| CMV | 44 (27.3 to 65.2) | 25 (3.95 to 87.2) |
| BK | 64 (45.8 to 81.8) | 100 (0 to 100) |
| Adenovirus | 8 (2.06 to 28.4) | 75 (33.5 to 99.1) |
| HHV6 | 57.5 (39.2 to 77.1) | 75 (33.5 to 99.1) |
| JCV | 32 (17.5 to 53.9) | 75 (33.5 to 99.1) |
| EBV | 60 (41.9 to 78.7) | 25 (3.95 to 87.2) |
The percentage of donor B-cell populations at days +28, +42, +60, +100, +180, and 1-year post-transplant.
Results for this outcome have not been posted.
Percentage of donor natural killer (NK-) cell populations at days +28, +42, +60, +100, +180, and 1-year post transplant.
Results for this outcome have not been posted.
Percentage of donor myeloid cell populations at days +28, +42, +60, +100, +180, and 1-year post transplant.
| percentage cells | Reduced Intensity Conditioning+Allogeneic Hematopoietic Cell Transplant + Graft-versus-Host Disease | Immunosuppression-Only Conditioning + Allo Hematopoietic Cell Transplant +Graft-versus-Host Disease |
|---|---|---|
| Day +28 post-transplant | 100 (78 to 100) | 23 (4 to 42) |
| Day +42 post-transplant | 100 (61 to 100) | 4 (0 to 33) |
| Day +60 post-transplant | 100 (34 to 100) | 3 (0 to 29) |
| Day +100 post-transplant | 100 (56 to 100) | 31 (12 to 50) |
| Day 180 post-transplant | 100 (67 to 100) | 22 (22 to 22) |
| 1-year post-transplant | 100 (92 to 100) | 33 (33 to 33) |
Here is the number of participants with serious and/or non-serious adverse events assessed by the Common Terminology Criteria for Adverse Events (CTCAE v5.0). A non-serious adverse event is any untoward medical occurrence. A serious adverse event is an adverse event or suspected adverse reaction that results in death, a life-threatening adverse drug experience, hospitalization, disruption of the ability to conduct normal life functions, congenital anomaly/birth defect or important medical events that jeopardize the patient or subject and may require medical or surgical intervention to prevent one of the previous outcomes mentioned.
| Participants | Reduced Intensity Conditioning+Allogeneic Hematopoietic Cell Transplant + Graft-versus-Host Disease | Immunosuppression-Only Conditioning + Allo Hematopoietic Cell Transplant +Graft-versus-Host Disease | 7-8/8 Human Leukocyte Antigen (HLA)-Matched Related or Unrelated, or HLA-haploidentical Donor |
|---|---|---|---|
| Number of Participants With Serious and/or Non-serious Adverse Events Assessed by the Common Terminology Criteria for Adverse Events (CTCAE v5.0) | 25 | 4 | 1 |
Collected over For Arms 1 and 2, recipients, All-Cause Mortality was monitored/assessed, an average of 2 years. All Adverse Events (AEs) were monitored/assessed from the start of conditioning (hematopoietic cell transplant day -14) until return to baseline or stabilization of event, through 30 days after end of therapy, removal from protocol therapy, or until off study, whichever comes first, followed by collection of AEs per principal investigator discretion, an average of 2 years.. Non-serious events are listed at a 0% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Reduced Intensity Conditioning+Allogeneic Hematopoietic Cell Transplant + Graft-versus-Host Disease | 4/25 (16%) | 18/25 (72%) | 25/25 (100%) |
| Immunosuppression-Only Conditioning + Allo Hematopoietic Cell Transplant +Graft-versus-Host Disease | 2/4 (50%) | 4/4 (100%) | 4/4 (100%) |
| 7-8/8 Human Leukocyte Antigen (HLA)-Matched Related or Unrelated, or HLA-haploidentical Donor | 1/10 (10%) | 0/10 (0%) | 1/10 (10%) |
| Event | Reduced Intensity Conditioning+Allogeneic Hematopoietic Cell Transplant + Graft-versus-Host Disease | Immunosuppression-Only Conditioning + Allo Hematopoietic Cell Transplant +Graft-versus-Host Disease | 7-8/8 Human Leukocyte Antigen (HLA)-Matched Related or Unrelated, or HLA-haploidentical Donor |
|---|---|---|---|
| HypotensionVascular disorders | 1/25 | 2/4 | 0/10 |
| HypoxiaRespiratory, thoracic and mediastinal disorders | 1/25 | 2/4 | 0/10 |
| Platelet count decreasedInvestigations | 11/25 | 0/4 | 0/10 |
| Neutrophil count decreasedInvestigations | 10/25 | 1/4 | 0/10 |
| White blood cell decreasedInvestigations | 7/25 | 1/4 | 0/10 |
| Abdominal painGastrointestinal disorders | 0/25 | 1/4 | 0/10 |
| Acute kidney injuryRenal and urinary disorders | 1/25 | 1/4 | 0/10 |
| AscitesGastrointestinal disorders | 0/25 | 1/4 | 0/10 |
| BacteremiaInfections and infestations | 5/25 | 1/4 | 0/10 |
| Creatinine increasedInvestigations | 1/25 | 1/4 | 0/10 |
| Event | Reduced Intensity Conditioning+Allogeneic Hematopoietic Cell Transplant + Graft-versus-Host Disease | Immunosuppression-Only Conditioning + Allo Hematopoietic Cell Transplant +Graft-versus-Host Disease | 7-8/8 Human Leukocyte Antigen (HLA)-Matched Related or Unrelated, or HLA-haploidentical Donor |
|---|---|---|---|
| White blood cell decreasedInvestigations | 19/25 | 4/4 | 0/10 |
| Neutrophil count decreasedInvestigations | 19/25 | 2/4 | 0/10 |
| Infusion related reactionInjury, poisoning and procedural complications | 9/25 | 3/4 | 0/10 |
| AnemiaBlood and lymphatic system disorders | 18/25 | 2/4 | 0/10 |
| Platelet count decreasedInvestigations | 18/25 | 1/4 | 0/10 |
| HypokalemiaMetabolism and nutrition disorders | 15/25 | 1/4 | 0/10 |
| NauseaGastrointestinal disorders | 15/25 | 1/4 | 0/10 |
| Mucositis oralGastrointestinal disorders | 13/25 | 0/4 | 0/10 |
| AnorexiaMetabolism and nutrition disorders | 4/25 | 2/4 | 0/10 |
| HypoalbuminemiaMetabolism and nutrition disorders | 4/25 | 2/4 | 0/10 |
| Age, Categorical(Participants) | Reduced Intensity Conditioning+Allogeneic Hematopoietic Cell Transplant + Graft-versus-Host Disease | Immunosuppression-Only Conditioning + Allo Hematopoietic Cell Transplant +Graft-versus-Host Disease | 7-8/8 Human Leukocyte Antigen (HLA)-Matched Related or Unrelated, or HLA-haploidentical Donor | Participants Enrolled But Not Treated | National Marrow Donor Program (NMDP) Donors | Total |
|---|---|---|---|---|---|---|
| <=18 years | 7 | 0 | 0 | 8 | — | 15 |
| Between 18 and 65 years | 18 | 4 | 9 | 12 | — | 43 |
| >=65 years | 0 | 0 | 1 | 0 | — | 1 |
| Age, Continuous(years) | Reduced Intensity Conditioning+Allogeneic Hematopoietic Cell Transplant + Graft-versus-Host Disease | Immunosuppression-Only Conditioning + Allo Hematopoietic Cell Transplant +Graft-versus-Host Disease | 7-8/8 Human Leukocyte Antigen (HLA)-Matched Related or Unrelated, or HLA-haploidentical Donor | Participants Enrolled But Not Treated | National Marrow Donor Program (NMDP) Donors | Total |
|---|---|---|---|---|---|---|
| Mean | 26.04 ± 15.1 | 32 ± 12.11 | 35.5 ± 16.16 | 26.35 ± 14.45 | — | 28.15 ± 14.97 |
| Sex: Female, Male(Participants) | Reduced Intensity Conditioning+Allogeneic Hematopoietic Cell Transplant + Graft-versus-Host Disease | Immunosuppression-Only Conditioning + Allo Hematopoietic Cell Transplant +Graft-versus-Host Disease | 7-8/8 Human Leukocyte Antigen (HLA)-Matched Related or Unrelated, or HLA-haploidentical Donor | Participants Enrolled But Not Treated | National Marrow Donor Program (NMDP) Donors | Total |
|---|---|---|---|---|---|---|
| Female | 11 | 1 | 3 | 7 | 4 | 26 |
| Male | 14 | 3 | 7 | 13 | 8 | 45 |
| Race/Ethnicity, Customized(Participants) | Reduced Intensity Conditioning+Allogeneic Hematopoietic Cell Transplant + Graft-versus-Host Disease | Immunosuppression-Only Conditioning + Allo Hematopoietic Cell Transplant +Graft-versus-Host Disease | 7-8/8 Human Leukocyte Antigen (HLA)-Matched Related or Unrelated, or HLA-haploidentical Donor | Participants Enrolled But Not Treated | National Marrow Donor Program (NMDP) Donors | Total |
|---|---|---|---|---|---|---|
| Ethnicity - Hispanic or Latino | 7 | 0 | 3 | 2 | — | 12 |
| Ethnicity - Not Hispanic or Latino | 17 | 4 | 5 | 17 | — | 43 |
| Ethnicity - Unknown or Not Reported | 1 | 0 | 2 | 1 | — | 4 |
| Race - American Indian or Alaska Native | 0 | 0 | 0 | 0 | — | 0 |
| Race - Asian | 1 | 0 | 1 | 2 | — | 4 |
| Race - Native Hawaiian or Other Pacific Islander | 0 | 0 | 0 | 0 | — | 0 |
| Race - Black or African American | 1 | 1 | 0 | 3 | — | 5 |
| Race - White | 20 | 3 | 6 | 13 | — | 42 |
| Race - More Than One Race | 1 | 0 | 0 | 0 | — | 1 |
| Race - Unknown or Not Reported | 2 | 0 | 2 | 1 | — | 5 |
| Race - Other | 0 | 0 | 1 | 0 | — | 1 |
| Race - Asian White | 0 | 0 | 0 | 1 | — | 1 |
| Region of Enrollment(participants) | Reduced Intensity Conditioning+Allogeneic Hematopoietic Cell Transplant + Graft-versus-Host Disease | Immunosuppression-Only Conditioning + Allo Hematopoietic Cell Transplant +Graft-versus-Host Disease | 7-8/8 Human Leukocyte Antigen (HLA)-Matched Related or Unrelated, or HLA-haploidentical Donor | Participants Enrolled But Not Treated | National Marrow Donor Program (NMDP) Donors | Total |
|---|---|---|---|---|---|---|
| United States | 25 | 4 | 10 | 20 | 12 | 71 |
Documents are hosted by the registry — open the source record to download them.
Plan to share: Yes — All individual participant data (IPD) recorded in the medical record will be shared with intramural investigators upon request. In addition, all large-scale genomic sequencing data will be shared with subscribers to the database of Genotypes and Phenotypes (dbGaP).
Supporting information: Study protocol, Sap, Icf
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Lymphoproliferative Disorders→
National Cancer Institute (NCI)