Disability Related to Chronic Graft-Versus-Host Disease

Abstract Chronic GVHD is a heterogenous syndrome whose symptoms and treatment are often associated with decrease in functional status and quality of life among survivors of transplantation. We explored definitions of chronic GVHD-related disability and factors associated with disability in chronic GVHD. We analyzed 371 patients with chronic GVHD requiring a new systemic therapy with enrollment and 18 month assessments through the Chronic GVHD Consortium, evaluating disability as a composite endpoint including any one of five impairments previously defined by Flowers et al [1] (score 2 or 3 keratoconjunctivitis sicca, score 2 or 3 scleroderma, any diagnosis of bronchiolitis obliterans, score 2 or 3 joint/fasciae involvement, or score 3 esophageal stricture requiring dilation). We also evaluated disability defined as decline in a human activity profile (HAP) score ≥8 or Karnofsky Performance Status (KPS) decline ≥ 20 from enrollment to 18 months. At enrollment, 47% of patients had at least one of the 5 Flowers disability features, with 50% of this group acquiring additional impairments at 18 months. Of the 197 (53%) patients with no Flowers disability at enrollment, 50% of this group progressed with disability features at 18 months. We found that any progressive Flowers impairment was associated with decline in HAP/KPS as well as increased NIH severity scores at 18 months. Enrollment mouth scores, and patient reported eye and skin scores were significantly associated with progressive impairment at 18 months. Progressive disability at 18 months did not predict subsequent non-relapse mortality. Additional studies to define chronic GVHD related-disability and risk factors are needed to develop this important patient-centered outcome.