Opinion
Video
Steroid-Refractory cGVHD: A Look at FDA-Approved Options
Panelists discuss how recent FDA approvals and evolving evidence for second-line treatments, including ruxolitinib, ibrutinib, and belumosudil, are reshaping chronic graft-vs-host disease (cGVHD) management by offering tailored, evidence-based options for steroid-refractory patients.
Optimizing Second-Line Treatment Selection in Chronic Graft-Vs-Host Disease: Recent Approvals and Evolving Evidence
This segment of the Pharmacy Times peer exchange focuses on second-line treatment options for steroid-refractory chronic graft-vs-host disease (cGVHD), highlighting the rapid expansion of FDA-approved therapies. The discussion begins with an overview of newly available agents, illustrating the shift from a steroid-only era to a broader, evidence-based treatment landscape. Key therapies include ibrutinib, a BTK inhibitor; ruxolitinib, a JAK1/2 inhibitor; belumosudil, a ROCK2 inhibitor; and the recently approved anti-CSF1R antibody, axatilimab. These agents, each with their own mechanisms of action and clinical profiles, offer new hope for patients who have not responded to steroids or have become steroid-dependent.
Ruxolitinib has emerged as a particularly impactful option, now holding a category 1 recommendation from NCCN guidelines. Backed by robust data from the REACH3 trial and its follow-up analyses, ruxolitinib has shown durable responses, with recent updates reporting an 81% best overall response rate. This durability, supported by both clinical trial data and real-world evidence, has reinforced its role as a first choice in many treatment protocols. Additional data has also shown improved response rates in lung involvement—a historically difficult organ to treat in cGVHD—prompting broader confidence in its use.
As treatment options expand, clinicians are increasingly tailoring second-line therapy to individual patient needs, factoring in organ involvement, prior therapies, and the overall clinical picture. The growing evidence base for agents like ruxolitinib and belumosudil has not only improved outcomes but also enhanced confidence in decision-making. The panel emphasizes the value of familiarity and experience with these drugs, noting that increased use has helped define where each agent performs best and when a combination or escalation strategy may be needed for optimal patient care.
