Validation of the Day 28 Refined Response for Acute GVHD in Clinical Trials

Validation of the Day 28 Refined Response for Acute GVHD in Clinical Trials

Acute graft-versus-host disease (GVHD) is a common complication after allogeneic hematopoietic cell transplantation (HCT), and the development of new therapeutic agents is needed. Accurate evaluation of treatment response is a key component in developing new treatments. Overall response, which combines complete and partial responses at day 28, is the conventional endpoint used in trials. We recently proposed a new surrogate endpoint, “Day 28 Refined Response,” based on a large real-world registry dataset from Japan and the US/Germany, and published it in Blood Advances (PMID: 40590886). This new criterion more precisely reclassifies patients within the conventional partial response category. Since the proportion of patients with partial response is generally higher in clinical trials than in real-world practice—due to the lower threshold for initiating additional treatment in daily practice—we hypothesize that the advantage of the refined response will be even greater in the trial setting. Therefore, we aim to compare the new and conventional surrogate endpoints using clinical trial datasets. The development and validation of improved response criteria will facilitate GVHD treatment trials and ultimately help improve HCT outcomes.

[{ "PostingID": 21087, "Title": "NOVARTIS-CINC424C2301", "Description": "Safety and Efficacy of Ruxolitinib Versus Best Available Therapy in Patients With Corticosteroid-refractory Acute Graft vs. Host Disease After Allogeneic Stem Cell Transplantation (REACH2)" }]