Phase 2
Completed N=21
NMA Haplo or MUD BMT for Newly Diagnosed Severe Aplastic Anemia
severe aplastic anemia · Bone Marrow Failure · Immunosuppression
Source: ClinicalTrials.gov NCT02833805 ↗
Enrolled (actual)
21
Serious AEs
9.5%
Results posted
Jul 2022
Primary outcomePrimary: Overall Survival and Engraftment at One Year — 19 Participants
Summary
Our primary objective is to determine if it is feasible for previously untreated severe aplastic anemia (SAA) patients to be transplanted using non-myeloablative conditioning and post transplantation cyclophosphamide.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Overall Survival and Engraftment at One Year |
19 | — |
| SECONDARY Overall Survival at One Year |
19 | — |
| SECONDARY Probability of Neutrophil Recovery as Assessed by the Number of Participants Who Have Recovered Neutrophil Counts |
21 | — |
| SECONDARY Probability of Platelet Recovery as Assessed by Number of Participants Who Have Recovered Platelet Counts |
20 | — |
| SECONDARY Number of Participants Who Experience Primary Graft Failure |
21 | — |
| SECONDARY Number of Participants Who Experience Secondary Graft Failure |
19 | — |
| SECONDARY Number of Participants Who Experience Grades II-IV Acute GVHD |
4 | — |
| SECONDARY Number of Participants Who Experience Grades III-IV Acute GVHD |
2 | — |
| SECONDARY Number of Participants Who Experience Chronic GVHD |
1 | — |
| SECONDARY Number of Participants With Full Donor Chimerism |
21 | — |
| SECONDARY GVHD-free Relapse-free Survival (GRFS) |
19 | — |
| SECONDARY Transplant-related Mortality |
2 | — |
Eligibility Criteria
Inclusion Criteria
- Confirmed diagnosis of inherited or acquired severe aplastic anemia (SAA)
- One of the following available donors:
- HLA-haploidentical relative
- If recipient is >= 40 years old, may use HLA-matched related donor
- For recipients with inherited bone marrow failure syndromes (IBMFS) with clear evidence of same disorder in potential related donors, may use 10/10 matched unrelated donor
- Recipient and/or legal guardian must sign protocol informed consent
- Donor must be willing to donate bone marrow
- Left ventricular ejection fraction (LVEF) >= 40%. For recipients = 26% may be used instead.
- Bilirubin = 13 years old: estimated creatinine clearance > 50 mL/min using Cockcroft-Gault formula and actual body weight
- For patients >= 1 but = 90 mL/min/1.73 m^2. If estimated GFR is 50 mL/min/1.73 m^2.
- For patients >= 8 years old, diffusing capacity of the lung for carbon monoxide (DLCO) (corrected for hemoglobin) > 40%; forced expiratory volume at one second (FEV1) > 50%; forced vital capacity (FVC) > 50%
- For patients 92% on room air
- Karnofsky/Lansky status (depending on age) >= 70%
- Females and males of childbearing potential must agree to practice 2 effective methods of contraception at the same time. If unwilling, they must agree to complete abstinence.
Exclusion Criteria
- Previous administration of immunosuppressive therapy for SAA.
- Fanconi anemia. At minimum, this diagnosis must be excluded by diepoxybutane (DEB) or equivalent testing on peripheral blood or marrow in patients 5 years previously. Other prior cancers will not be allowed unless approved by the PI.
Data sourced from ClinicalTrials.gov (NCT02833805). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.