Aplastic Anaemia, Hematopoietic Cell Transplant, Severe Aplastic Anemia
Conditions
Brief summary
This prospective, single-center, single-arm interventional study evaluates the safety and preliminary efficacy of a thiotepa-based conditioning regimen in patients aged 50 years or older with severe or very severe aplastic anemia undergoing haploidentical hematopoietic stem cell transplantation.
Interventions
DRUGThiotepa
Thiotepa 10 mg/kg is administered on Day -6.
DRUGBusulfan (BU)
3.2 mg/kg/day intravenously on Days -8 to -7.
DRUGFludarabine
Total dose 150 mg/m² intravenously on Days -6 to -2.
Total dose 7.5 mg/kg intravenously on Days -5 to -2.
Basiliximab 20 mg is administered on Day -1 and Day +4.
Sponsors
Peking University People's Hospital
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
50 Years to No maximum
Healthy volunteers
No
Inclusion criteria
1. Age 50 years or older. 2. Diagnosis of severe aplastic anemia or very severe aplastic anemia according to institutional diagnostic criteria. 3. Planned to undergo haploidentical hematopoietic stem cell transplantation with an eligible haploidentical donor. 4. Meets institutional performance status and cardiac, hepatic, renal, and pulmonary function requirements for transplantation. 5. Provides written informed consent.
Exclusion criteria
1. Previous allogeneic hematopoietic stem cell transplantation. 2. Uncontrolled active infection at the time of enrollment or transplantation. 3. Known hypersensitivity to busulfan, fludarabine, thiotepa, basiliximab, or any relevant excipient. 4. Concurrent hematologic malignancy or another condition that, in the investigator's judgment, makes transplantation unsafe. 5. Pregnancy or breastfeeding. 6. Any serious uncontrolled medical, psychiatric, or social condition that could compromise protocol compliance or participant safety. \-
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| 1-year transplant-related mortality (TRM) | From Day 0 through 1 year after transplantation | 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. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Neutrophil Engraftment | From Day 0 through Day 28 | Time to neutrophil engraftment, defined as the first of 3 consecutive days with an absolute neutrophil count ≥0.5 × 10\^9/L； |
| Platelet Engraftment | From Day 0 through Day 100 | 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 | From Day 0 through Day 100 | Proportion of participants without neutrophil engraftment or with graft failure, according to the study protocol. |
| Overall Survival | From Day 0 through 1 year after transplantation | Proportion of participants alive, irrespective of cause of death. |
| Grade II-IV Acute GVHD | From Day 0 through Day 100 | Incidence of grade II-IV acute graft-versus-host disease. |
| Moderate or Severe Chronic GVHD | From Day 0 through 1 year after transplantation | Incidence of moderate or severe chronic graft-versus-host disease. |
Contacts
CONTACTXu zheng li
Outcome results
None listed