Post-transplant Cyclophosphamide (PTCy) for HLA-Matched Hematopoietic Stem Cell Transplant (HCT) in Pediatric and Young Adult Patients with Hematologic Malignancies - PubMed
4 hours ago
- #GVHD Prophylaxis
- #Pediatric Oncology
- #Hematopoietic Stem Cell Transplant
- Post-transplant Cyclophosphamide (PTCy) is used for GVHD prophylaxis in HLA-matched HCT for pediatric and young adult patients.
- Study shows PTCy leads to successful engraftment, survival, and low GVHD rates in pediatric patients.
- Retrospective analysis of 22 patients (≤21 years) at Johns Hopkins (2013-2023) with HLA-matched related or unrelated HCT.
- GVHD prophylaxis included PTCy alone or combined with tacrolimus and MMF for certain grafts or conditioning regimens.
- Median age was 16.5 years; diagnoses included AML, ALL, and MDS with full donor chimerism at day +60.
- Median time to neutrophil and platelet recovery was 18 and 19.5 days, respectively.
- Two cases of severe acute GVHD (Gr IV) resulted in death; no chronic GVHD observed.
- 3-year cumulative incidence of relapse was 48% (38% for MRD-negative pre-HCT).
- Actuarial overall survival (OS) was 67%, event-free survival (EFS) 43%, and GVHD-free relapse-free survival (GRFS) 43% at 3 years.
- PTCy is feasible for HLA-matched HCT in pediatrics with 100% engraftment and no GVHD in matched sibling donors.