Outcome of pediatric allogeneic stem cell transplantation in non-malignant diseases at National Stem Cell Center (HAYAT) in Syria.

Abstract

Background: Allogeneic stem cell transplantation (allo-HSCT) is a potentially curative treatment for a variety of non-malignant pediatric diseases. Notably, pediatric HSCT commenced in Syria in 2021 amidst the prevailing crisis. This study aims to evaluate the outcomes, demographic and clinical profiles of pediatric patients undergoing allo-HSCT for non-malignant conditions at the National Stem Cell Center (HAYAT) Syria's first pediatric HSCT center. Numbers of transplants have gradually increased. So far, we have conducted 36 transplants despite the challenges and war in our country. 17 of them are non-malignant patients.

Method: A retrospective analysis was conducted on pediatric patients who underwent allo-HSCT for non-malignant diseases at HAYAT center between 2021 and 2024. Data on patient demographics, underlying diseases, conditioning regimens, donor types, graft-versus-host disease (GVHD), engraftment, and survival outcomes were collected and analyzed.

Result: 17 children underwent the transplant, distributed among 12 patients with thalassemia major, 4 patients with severe aplastic anemia, and 1 patient with severe immunodeficiency. Transplant-related mortality was recorded in one out of seventeen cases, resulting in a mortality rate of 6%. Overall survival and disease-free survival rates were favorable, with 94% of patients (N=16) achieving successful outcomes. The median duration for neutrophil engraftment was 14 days, while platelet engraftment occurred at a median of 13 days, except for one patient who experienced delayed engraftment due to graft failure. Acute graft-versus-host disease (GVHD) occurred in 47% of cases (N=8), with gastrointestinal involvement in five cases and cutaneous involvement in three cases. Among these, six cases were classified as grade I-II, while two cases were classified as grade IV-III.

Conclusion: Our study provides insights into the outcomes of pediatric allo-HSCT for non-malignant diseases at HAYAT. Despite the inherent challenges, allo-HSCT remains a viable therapeutic option with the potential for long-term disease control and improved quality of life in pediatric patients with non-malignant conditions. Further research and advancements in transplant techniques are warranted to optimize outcomes and minimize complications in this patient population.

Keywords: Hematopoietic Stem Cell Transplantation, Pediatric, Allogeneic Bone Marrow Transplantation, Non-Malignant Diseases.