Ruxolitinib as GVHD Prophylaxis in AA Patients

Phase 2 Unknown 46 enrolled Institute of Hematology & Blood Diseases Hospital, China

NaSingle-groupOpen-labelTreatment

Summary

Our study aimed to determine the prophylactic value of ruxolitinib for aGVHD in HSCT recipients of AA.Ruxolitinib was initiated at the beginning of conditioning regimen at 5 twice daily until 3 months post-transplantation. The cumulative incidence of aGVHD within 6 months after HSCT will be the primary observation item.

Timeline

Start: 2023-07-01
Primary completion: 2025-05-01
Completion: 2025-05-01

Outcome

Met primary endpoint

paper Comparative analysis showed that ruxolitinib significantly reduced the cumulative incidence of grade II-IV aGVHD (HR 0.24; p = 0.004) PMID 41796372 ↗

Publications

Zhang X, Pan L, Zhao Y, Ma R, Zhang L, Zhang Y, Li G, Zhai W, Ma Q, Pang A, Yang D, Feng S, Zhang P, He Y, Qin G, Jiang E, Han M. Efficacy and safety of ruxolitinib for graft-versus-host disease prophylaxis in patients with aplastic anemia undergoing PBSC-only allogeneic stem cell transplantation: a prospective phase II study. Exp Hematol Oncol. 2026 Mar 8;15(1):31. doi: 10.1186/s40164-026-00753-9.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	Ruxolitinib	Small molecule	5 mg	Oral

Indications

No indication recorded.