Improving outcomes of hematopoietic stem cell transplant for leukemia in children and young adults in resource-limited setting

Abstract

IntroductionAllogeneic hematopoietic stem cell transplantation (AHSCT) is a curative approach in patients with relapsed and high-risk leukemia. This retrospective analysis aimed to investigate outcomes and risk factors associated with HSCT in children and young adults with hematological malignancies in a resource-limited setting. MethodsWe retrospectively evaluated 48 pediatric and young adult patients diagnosed with hematological malignancies who underwent HSCT at our institution between April 2017 and Jan 2023. ResultThe median age of the cohort was 10 years (6 months - 21 years), and the majority of patients underwent transplants for ALL (n = 24; 50 %) and AML (n = 18; 37.5%). Median time to neutrophil and platelet engraftment were 15 days (range 9–28) and 18 days (range 9–40), respectively. One patient had primary graft failure. Cytomegalovirus (CMV) reactivation occurred in 21 patients (43.8%). Seventeen patients received escalated DLI. With a median follow-up of 902 days (52–2490 days), the probability of event free survival and overall survival (OS) was 65.9% and 69.4%. OS for lymphoid and myeloid malignancies were 64.7% and 75.2%, respectively. Kaplan-Meier probabilities of OS for patients in first complete remission (CR1), CR2, CR3, or with refractory disease were 80%, 76.2%, 0%, and 40%, respectively (p = 0.02). OS of the patients undergoing matched sibling donor (n = 17) or haploidentical HSCT (n = 31) was 70.6% and 69%, respectively (p = 0.989). ConclusionsOur study demonstrates excellent outcomes of patients with hematological malignancies undergoing allogeneic HSCT with similar outcomes in HLA-identical sibling donors and haploidentical first-degree family donors. Prophylactic donor lymphocyte infusion might be considered in patients with high-risk leukemia to prevent relapse, including ALL.