Excellent outcomes of HLA identical related donor hematopoietic stem cell transplant (HSCT) in patients with sickle cell disease (SCD) have put it as a standard of care in symptomatic patients especially after a failed hydroxyurea therapy, however a large part of this data comes from resourcerich settings. These encouraging outcomes are in-turn due to patients being considered for HSCT in relatively well-preserved condition, safer transplant conditioning, better GvHD prophylaxis strategies and improved overall supportive care. Large-scale real-world data from resource-constraint settings is however missing. We retrospectively analysed baseline characteristics and longitudinal data of 85 paediatric and young adults who underwent matched sibling donor (MSD) HSCT for SCD over a decade. Patients received conditioning as per APOLLO PROTOCOL from May 2019. Median age was 8 years (range 10 months - 32 years). Median time to neutrophil and platelet engraftment was 13 days (range, 10-19) and 14 days (range, 6-48). None experienced primary graft failure and one had secondary graft failure. Acute GvHD was seen in 5 patients (4- acute skin GvHD, grade I/II; 1- gut GvHD, grade IV). At a median follow up of 1191 days (range, 23-4226), Kaplan Meier estimated EFS and OS is 94.11% and 96.47% respectively. OS in APOLLO protocol cohort was 100% and 91.4% using conventional (BU-CY or TTF based conditioning). Age > 10 years turned out to be the only significant risk factor affecting outcome (100% vs 89.28%, P=0.007). This emphasizes the need of early intervention < 10 years of age for best clinical outcomes.
Kharya et al. (Thu,) studied this question.