N/A
Completed N=61
Hematopoietic Stem Cell Transplant for Dyskeratosis Congenita or Severe Aplastic Anemia
Source: ClinicalTrials.gov NCT02162420 ↗Enrolled (actual)
61
Serious AEs
0.0%
Results posted
May 2025
Primary outcomePrimary: Incidence of Neutrophil Engraftment — 100; 100; 96; 100 Percentage of participants
Summary
Fludarabine-based preparative regimen followed by an allogeneic hematopoietic stem cell transplant using related or unrelated donor in persons 0-70 years of age diagnosed with dyskeratosis congenita or severe aplastic anemia who have bone marrow failure characterized by a requirement for red blood cell and platelet transfusions. Three different preparative regimens are included based on disease and donor type.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Incidence of Neutrophil Engraftment |
100; 100; 96; 100 | — |
| PRIMARY Incidence of Platelet Engraftment |
100; 100; 96; 100 | — |
| SECONDARY Incidence of Regimen Related Mortality |
0; 6; 6; 0 | — |
| SECONDARY Incidence of Acute Graft-versus-host Disease |
0; 11; 9; 0 | — |
| SECONDARY Incidence of Chronic Graft-versus-host Disease |
0; 6; 0; 0 | — |
| SECONDARY Incidence of Secondary Malignancies |
0; 0; 0; 0 | — |
Eligibility Criteria
Inclusion Criteria
- Aged 0 - 70 years
- Acceptable hematopoeitic stem cell donor
- Dyskeratosis Congenita (DC) with evidence of BM failure defined as:
- requirement for red blood cell and/or platelet transfusions or
- requirement for G-CSF or GM-CSF or erythropoietin or
- refractory cytopenias having one of the following three
- platelets 30% blasts
Data sourced from ClinicalTrials.gov (NCT02162420). 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.