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).
This pilot phase II trial studies how well carfilzomib works in treating patients with chronic graft-versus-host disease. Chronic graft-versus-host disease is a complication of a donor bone marrow or blood cell transplant, usually occurring more than three months after transplant, in which donor cells damage the host tissue. Carfilzomib may be an effective treatment for chronic graft-versus-host disease.