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.
Age 50 years or older at transplantation.
Diagnosis of severe aplastic anemia or very severe aplastic anemia.
Planned to undergo haploidentical hematopoietic stem cell transplantation at Peking University People's Hospital.
The treating clinical team has independently selected a thiotepa-based conditioning regimen as part of routine clinical care before study enrollment.
Diagnosis or transplantation type does not meet the study eligibility criteria.
The conditioning regimen does not include thiotepa.
Essential baseline information is unavailable, such that study eligibility cannot be determined.
For a participant with more than one transplantation episode, only the first episode meeting all eligibility criteria will be included in the analysis.
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.
Cohort
Prospective
Thiotepa
DrugThe 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.
Neutrophil Engraftment
Time to neutrophil engraftment, defined as the first of 3 consecutive days with an absolute neutrophil count ≥0.5 × 10\^9/L；
Platelet Engraftment
Time to platelet engraftment, defined as the first of 7 consecutive days with platelet count ≥20 × 10\^9/L without platelet transfusion.
Primary Graft Failure
Proportion of participants without neutrophil engraftment or with graft failure, according to the study protocol.
Overall Survival
Proportion of participants alive, irrespective of cause of death.
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