Letermovir Prophylaxis in Pediatric Allogeneic Hematopoietic Stem Cell Transplantation: Clinical Effectiveness and...

Cytomegalovirus (CMV) reactivation is a major infectious complication following pediatric allogeneic hematopoietic stem cell transplantation (HSCT), particularly in low- and middle-income countries (LMICs) with high CMV seroprevalence. Data on the clinical and economic impact of letermovir prophylaxis in pediatric HSCT recipients from resource-constrained settings remain limited.

We conducted a retrospective cost-effectiveness analysis of pediatric allogeneic HSCT recipients at a university-affiliated tertiary-care transplant center in India. Clinical outcomes and direct medical costs were compared between patients who received letermovir prophylaxis and those managed with a standard pre-emptive CMV strategy. The primary outcome was CMV reactivation by Day +100 post-transplant. Cost-effectiveness was evaluated using the incremental cost-effectiveness ratio (ICER).

Fifty patients were included, of whom 15 received letermovir prophylaxis and 35 received standard pre-emptive management. By Day +100 post-transplant, CMV reactivation occurred in 26.7% of patients receiving letermovir compared with 91.4% in the non-letermovir group (p < 0.001). Median time to CMV reactivation was longer in the letermovir group at 28.5 days versus 18.0 days. No patient in the letermovir group required additional CMV-related hospitalization, whereas 50% of patients in the non-letermovir group required prolonged hospitalization. Mean total treatment costs were higher in the letermovir group at $1868.5 compared with $1698.6 in the non-letermovir group; however, the incremental cost-effectiveness ratio was $262.6 per additional patient without CMV reactivation.

Despite higher upfront costs, letermovir demonstrated favorable cost-effectiveness in pediatric allogeneic HSCT recipients in resource-limited settings, while providing significant clinical benefits through reduced CMV reactivation, delayed breakthrough infection, and decreased CMV-related hospitalization.