An observational study in Severe Aplastic Anemia and Hematopoietic Cell Transplant, sponsored by Peking University People's Hospital. Not yet recruiting. Open to participants aged 50 Years and older. Per ClinicalTrials.gov, last updated 2026-09-21.
Sponsored by Peking University People's Hospital · Observational
This prospective, single-center observational cohort study will follow participants aged 50 years or older with severe or very severe aplastic anemia who receive haploidentical hematopoietic stem cell transplantation with a thiotepa-based conditioning regimen as part of routine clinical care. Treatment decisions, including conditioning, prevention of graft-versus-host disease, and supportive care, are made by the clinical care team and are not assigned or changed by this study. The study will collect routine clinical and follow-up information for 1 year after transplantation to describe transplant-related mortality, survival, engraftment, graft failure, graft-versus-host disease, infections, and serious adverse events.
Severe aplastic anemia is a bone marrow failure disorder characterized by reduced blood cell production. For participants who need transplantation and do not have a fully matched sibling donor, haploidentical hematopoietic stem cell transplantation is an important treatment option. Older participants may have reduced organ function, a higher risk of infection, and lower tolerance of intensive treatment.
Thiotepa-based conditioning regimens are used in routine clinical practice before transplantation. In this study, the treating clinical team independently determines the conditioning regimen, medication doses and schedules, prevention of graft-versus-host disease, and supportive care according to each participant's clinical condition. Study participation does not assign, require, or change any treatment.
This is a prospective, single-center observational cohort study. The study will consecutively include participants aged 50 years or older with severe or very severe aplastic anemia who receive haploidentical hematopoietic stem cell transplantation with thiotepa-based conditioning at Peking University People's Hospital. Routine clinical information, transplantation information, and routinely collected follow-up information will be recorded through 1 year after transplantation.
The primary outcome is transplant-related mortality within 1 year after transplantation. Secondary outcomes include overall survival, hematopoietic engraftment, graft failure, acute and chronic graft-versus-host disease, cytomegalovirus and Epstein-Barr virus reactivation, serious infections, and serious adverse events. The study will not add treatment, examinations, blood collection, or follow-up visits beyond routine care.
371 studies on the registry are indexed under Anemia, Aplastic; 98 are open to participants now.
This study's planned enrollment of 20 is below the median of 100 across 64 observational studies indexed under Anemia, Aplastic.
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The study population will consist of consecutively identified participants aged 50 years or older with severe or very severe aplastic anemia who are scheduled to undergo haploidentical hematopoietic stem cell transplantation at Peking University People's Hospital. The treating clinical team will independently select a thiotepa-based conditioning regimen as part of routine clinical care. The study will prospectively observe participants with available baseline, transplantation, and routine follow-up information for 1 year after transplantation. Up to 10 participants are planned for enrollment.
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Exclusion Criteria:
Participants aged 50 years or older with severe aplastic anemia or very severe aplastic anemia who receive haploidentical hematopoietic stem cell transplantation with a thiotepa-based conditioning regimen. The conditioning regimen is selected by the treating clinical team before study enrollment. The study does not assign or alter treatment.
Drug: Thiotepa
The exposure of interest is a conditioning regimen containing thiotepa. Thiotepa and all accompanying treatment are selected by treating clinicians as part of routine care, independently of study participation. The study does not mandate drug dose, schedule, graft-versus-host disease prophylaxis, or supportive care.
1-year transplant-related mortality (TRM)
The proportion of participants who die from transplant-related causes, including regimen-related toxicity, infection, and graft-versus-host disease, as defined in the study protocol.
Time frame: From Day 0 through 1 year after transplantation
Neutrophil Engraftment
Time to neutrophil engraftment, defined as the first of 3 consecutive days with an absolute neutrophil count ≥0.5 × 10\^9/L；
Time frame: From Day 0 through Day 28
Platelet Engraftment
Time to platelet engraftment, defined as the first of 7 consecutive days with platelet count ≥20 × 10\^9/L without platelet transfusion.
Time frame: From Day 0 through Day 100
Primary Graft Failure
Proportion of participants without neutrophil engraftment or with graft failure, according to the study protocol.
Time frame: From Day 0 through Day 100
Overall Survival
Proportion of participants alive, irrespective of cause of death.
Time frame: From Day 0 through 1 year after transplantation
Grade II-IV Acute graft-versus-host disease（ GVHD）
Incidence of grade II-IV acute graft-versus-host disease.
Time frame: From Day 0 through Day 100
Moderate or Severe Chronic graft-versus-host disease（GVHD）
Incidence of moderate or severe chronic graft-versus-host disease.
Time frame: From Day 0 through 1 year after transplantation
No study locations are listed for this record.
Start is estimated for Sep 2026; primary completion for Dec 2027, 15 months later.
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This study is not yet recruiting, as verified in Sep 2026. You cannot join it, but the record below documents what was studied.
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