Survival without Progressive Impairment As a Novel Endpoint in Chronic Graft-Versus-Host Disease

response rates following second-line therapy for SR-aGVHD in this modern era. Methods: We retrospectively analyzed day 28 response rates and clinical outcomes of 113 HCT patients (median age 39 years, range 1-75) who received second-line therapy for SR-aGVHD from 1998-2012 at the University of Minnesota. Results: Patients received grafts from 43 related (38 matched, 5 mismatched), 31 unrelated (23 HLA 6-8/8, 8 HLA 25/8) and 39 cord blood donors (23 HLA 6-8/8, 16 HLA 25/8); 76 patients (67.3%) received myeloablative regimens. Second-line therapy consisted of ATG (44.2%), MMF (24.8%), steroid boost (22.1%) or other (8.8%). Patients receiving ATG had higher-risk GVHD Risk Scores (MacMillan et al, Br J Haem, 2012) at second-line initiation compared with patients receiving MMF or steroids. At day 28, overall response (complete response [CR]/partial response [PR]) was observed in 32 patients (28%). All therapies had similar responses. In multivariate analysis, factors associated with day 28 CR/PR included GVHD Risk Score and days from onset of aGVHD to second-line therapy (Table); Six-month non-relapse mortality (NRM) was lower among patients receiving steroid boosts (24% [7-41%, 95% CI]) vs. ATG (64% [48-90%, 95% CI]) with no differences vs. MMF or other therapies (P1⁄40.01, Figure 1).