Quality of life (QOL) after hematopoietic cell transplantation (HCT) is compromised by chronic GVHD. In a prospectively assembled multicenter cohort of adults with chronic GVHD (n ‫؍‬ 298), we examined the relationship between chronic GVHD severity defined by Na-

, moderate, severe). The Chronic GVHD Consortium was established to test these new criteria. This report includes the first 298 adult patients enrolled at 5 centers of the Consortium. Patients were assessed every 3-6 months using standardized forms recommended by the Consensus Conference. At the time of study enrollment, global chronic GVHD severity was mild in 10% (n ‫؍‬ 32), moderate in 59% (n ‫؍‬ 175), and severe in 31% (n ‫؍‬ 91). Skin, lung, or eye scores determined the global severity score in the majority of cases, with the other 5 organs determining 16% of the global severity scores. Conventional risk factors predictive for onset of chronic GVHD and nonrelapse mortality in people with chronic GVHD were not associated with NIH global severity scores. Global severity scores at enrollment were associated with nonrelapse mortality (P < .0001) and survival (P < .0001); 2-year overall survival was 62% (severe), 86% (moderate), and 97% (mild

Bronchiolitis obliterans syndrome (BOS) after allogeneic hematopoietic cell transplantation (HCT) is associated with high mortality. Purpose: We hypothesized that FAM (inhaled Fluticasone, Azithromycin, and Montelukast) with a brief steroid pulse could avert progression of new-onset BOS. Experimental design: We tested this in a phase II, single-arm, open label, multicenter study (NCT01307462). Results: Thirty-six patients were enrolled within 6 months of BOS diagnosis. The primary endpoint was treatment failure, defined as 10% or greater FEV1% decline at 3 months. At 3 months, 6% (2/36, 95% CI 1%–19%) had treatment failure (vs. 40% in historical controls, p<0.001). FAM was well tolerated. Steroid dose was reduced by 50% or more at 3 months in 48% of patients who could be evaluated (n=27). Patient-reported outcomes at 3 months were statistically significantly improved for SF-36 social functioning score and mental component score, FACT emotional well-being, and Lee symptom scores in lung, skin, mouth, and the overall summary score compared to enrollment (n=24). At 6 months, 36% had treatment failure (95% CI 21%–54%, n=13/36, with 6 documented failures, 7 missing pulmonary function tests). Overall survival was 97% (95% CI 84%–100%) at 6 months. These data suggest that FAM was well tolerated and that treatment with FAM and steroid pulse may halt pulmonary decline in new-onset BOS in the majority of patients and permit reductions in systemic steroid exposure, which collectively may improve quality of life. However, additional treatments are needed for progressive BOS despite FAM.

Several distinct graft-versus-host disease (GVHD)-related syndromes have been defined by the NIH Consensus Conference. We enrolled a prospective cohort of 911 hematopoietic cell transplantation (HCT) recipients at 13 centers between March 2011 and May 2014 to evaluate four GVHD syndromes: late acute GVHD, chronic GVHD, bronchiolitis obliterans syndrome, and cutaneous sclerosis. The median age at HCT was 53.7 years. Most patients received peripheral blood stem cell transplant (81%) using a non myeloablative or reduced intensity conditioning (55%). Pediatric age group and use of bone marrow and umbilical cord blood were underrepresented in our cohort (<=11%). The cumulative incidence of late acute GVHD (late onset and recurrent) was 10% at a median of 5.5 months, chronic GVHD was 47% at a median of 7.4 months, bronchiolitis obliterans was 3% at a median of 12.2 months, and cutaneous sclerosis was 8% at a median onset of 14.0 months after HCT. Late acute GVHD and bronchiolitis obliterans had particularly high non-relapse mortality of 23% and 32% by 2 years after diagnosis. The probability of late acute- and chronic-GVHD-free, relapse-free survival at one and two years after HCT was 38% and 26%. This multi-center, prospective study confirms the high rate of late acute and chronic GVHD syndromes and supports the need for continuous close monitoring and development of more effective GVHD treatment strategies to improve HCT success.

Erlotinib did not increase the toxicity of cisplatin and radiotherapy in patients with locally advanced HNSCC but failed to significantly increase CRR or PFS.