SINGLE-CENTRE PERSPECTIVE ON ALLOGENEIC HEMATOPOIETIC STEM CELL TRANSPLANTATION FOR PAEDIATRIC MYELODYSPLASTIC SYNDROME IN THE DEVELOPING WORLD

Abstract

Introduction: Pediatric Myelodysplastic Syndrome (MDS) presents complex challenges, often requiring Allogeneic Hematopoietic Stem Cell Transplantation (HSCT). This study explores the clinical profile and demographics of seven pediatric MDS patients post-HSCT, shedding light on causes, molecular abnormalities, and patient diversity. Methods: Retrospective data from 2014 to 2023 were gathered from the Pediatric Hematology Oncology & Bone Marrow Transplantation Unit at the Cancer Institute in North India. Results: Patient Demographics: Seven pediatric patients (4 males, 3 females), median age 12, underwent HSCT (Five matched related donor HSCT and two haploidentical). Underlying Causes of MDS: Varied etiological factors identified, including Fanconi anemia (n=2), Emberger syndrome (n=1), therapy-related (n=1), GATA-2 insufficiency (n=1), and de novo cases in 2 patients, highlighting population heterogeneity. Molecular Abnormalities: Analysis revealed Monosomy-7 in two patients, NPM-1 in one, and GATA-2 mutation in two cases, providing crucial insights into the genetic landscape of MDS in this specific patient group. Post-transplant Outcome: Successful engraftment for all, with median neutrophil engraftment at 15.5 days and platelet engraftment at 13.5 days. GVHD and viral reactivation observed, yet 85.7% survived with complete donor chimerism. Unfortunately, one FA patient succumbed on day +72 due to E.coli sepsis with grade-IV GVHD. Mean follow-up is 33.5 months (2.7 years), ranging from 2.5 to 60 months. Conclusions: HSCT emerges as an effective therapy for pediatric MDS patients, yielding promising results in this developing world context.