Pomalidomide May Improve Motor and Function in Steroid-Refractory cGVHD
A clinical study published in Transplantation and Cellular Therapy suggests that pomalidomide, an immunomodulatory agent, may help improve motor performance and functional abilities in patients suffering from steroid-refractory chronic graft-versus-host disease (cGVHD), a condition associated with high morbidity and decreased survival.
The trial enrolled 34 adult patients with cGVHD in an unblinded, randomized design to assess the effects of two oral pomalidomide dosing regimens: a low dose (0.5 mg/day) and a high dose (escalating from 0.5 mg/day to a maximum of 2 mg/day). Investigators primarily measured efficacy using the Activity Card Sort (ACS), 2-Minute Walk Test (2MWT), Disabilities of the Arm, Shoulder, and Hand (DASH), Manual Abilities Measure 36 (MAM), and other validated tools of physical and quality-of-life assessment.
After 6 months, pooled data from both dosing groups showed statistically significant improvements in upper extremity function (DASH, P = .01), hand skills (MAM, P = .01), and physical quality of life (SF-36 PCS, P = .02). While no statistically significant difference was observed between the dosing cohorts, the low-dose group demonstrated greater functional gains, including improved walk distance, active range of motion, and participation in high-demand leisure and social activities.
Responders to pomalidomide consistently outperformed nonresponders across multiple domains, underscoring the drug's potential as a salvage therapy in this patient population.
The study also highlights the value of functional assessments like the ACS, DASH, and MAM in evaluating treatment response in cGVHD. Researchers emphasize the need for further studies with larger and more diverse populations to validate these findings and clarify optimal dosing strategies.
Reference
Jorgensen JJ, Joe GO, Jiménez-Silva R. Efficacy of pomalidomide on motor performance and functional abilities in patients with steroid refractory chronic graft versus host disease: a randomized clinical study. Transplan Cell Ther. 2025;31(6):361.e1-361.e15. doi:10.1016/j.jtct.2025.02.023.
