Ruxolitinib in Pediatric Steroid-Refractory Graft-Versus-Host Disease: An Experience From a Tertiary Center in India

Steroid-refractory graft-versus-host disease (SR-GVHD) remains a challenging and life-threatening complication following allogeneic hematopoietic stem cell transplantation (HSCT) in children. Ruxolitinib has become the standard for management of SR-GVHD; however, data on its efficacy and safety in the pediatric population are limited, particularly from Indian centers.

This retrospective review included pediatric patients with SR-GVHD treated with ruxolitinib between 2019 and 2024 at a tertiary care center in India. Data on demographics, GVHD details, ruxolitinib dosing, treatment response, and adverse effects were collected.

Of 38 pediatric allogeneic HSCTs performed during the study period, 14 children developed GVHD. Nine had steroid-refractory GVHD, of whom five were treated with ruxolitinib. Three patients had acute GVHD (aGVHD), one had chronic GVHD (cGVHD), and one presented with an overlap syndrome. The organs involved included skin, gastrointestinal tract, or both. Ruxolitinib was administered at doses ranging from 5 to 10 mg/day. It was used as the second line agent after steroids in four children, and as a third line therapy in one child. Three out of four patients with aGVHD/overlap syndrome had overall response (two partial and one complete) at day 28, and the cGVHD patient achieved partial response at week 24. One patient succumbed due to infectious complications.

This early pediatric Indian experience demonstrates promising efficacy of ruxolitinib in SR-GVHD, albeit with a risk of cytopenia and infections. It underscores the need for broader accessibility and prospective studies.