Outcomes of hematopoietic stem cell transplantation in adult sickle cell disease patients: A Single-Center experience from India

Sickle cell disease (SCD) in adults remains a significant clinical challenge, with limited curative options and high morbidity and mortality. Hematopoietic stem cell transplantation (HSCT) offers a potential cure, yet data on its feasibility and outcomes in adults, particularly from low- and middle-income countries, remain scarce. This retrospective single-centre study reports outcomes of HSCT in 14 adult SCD patients treated between 2018 and 2024 in India—the largest such series from South-East Asia. Patients underwent either matched sibling donor (MSD, n = 9) or haploidentical (Haplo, n = 5) transplants following myeloablative conditioning. All patients had high symptom burden, including ≥ 2 vaso-occlusive crises annually. Median follow-up was 22.4 months. Event-free survival (EFS) and overall survival (OS) were 71.4% and 78.6%, respectively. Full or mixed donor chimerism was achieved in 84.6% of patients. Notably, patients achieving chimerism reported no sickle-related events post-transplant. Acute GVHD occurred in 6 patients, all grade ≤ 2; no grade 3/4 GVHD or GVHD-related mortality was observed. Cytomegalovirus reactivation and posterior reversible encephalopathy syndrome (PRES) were manageable with standard interventions. This study demonstrates that HSCT in symptomatic adult SCD patients is both feasible and effective in resource-constrained settings when conducted in specialized centers with multidisciplinary support. These findings challenge the historical reluctance to offer HSCT to adults with SCD and underscore the urgent need for accessible curative options in this population. Policy reforms, expanded transplant access, and early referral pathways are vital to improving outcomes in adult SCD across similar healthcare settings.