Carfilzomib for Treatment of Chronic Graft vs. Host Disease
The primary endpoint is overall treatment failure at 6 months from study enrollment. Treatment failure represents the composite of addition of a new line of systemic immune-suppressive therapy beyond carfilzomib (objective signal of failure to control GVHD with carfilzomib), non-relapse death, and primary malignancy relapse.6 The historical benchmark for this outcome based on the analysis of 312 chronic GVHD patients is a 6 month treatment failure rate of 44%. With 20 patients, the standard error of the estimated failure rate will be approximately 10 percentage points. In the analysis of secondary objectives, we will study the 3 and 6 month overall (complete + partial) response and complete response rates, other efficacy measures listed above, patient reported outcomes, and biologic outcome measures. We will study association between biologic outcome measures and clinical parameters (response, treatment failure, mortality).