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.