Using Functional Performance Measures to Assess Clinical Response in Patients with Sclerotic Chronic Graft Versus Host Disease: An Exploratory Study

Abstract

Introduction In allogeneic hematopoietic stem cell transplant survivors, sclerotic chronic graft-versus-host disease (ScGVHD) is associated with poor quality of life (QOL) and range of motion restrictions. The latter can limit patients' ability to perform important activities of daily living (ADLs). The NIH Consensus Development Project established a standard set of measures to monitor chronic GVHD (cGVHD) progression. This includes indicators of patient-reported QOL and symptom burden, which is defined as the subjective severity and impact of physiological symptoms. Adding measures of functional performance may allow clinicians to more fully characterize the impact of ScGVHD on daily life. Objective To determine whether change in functional capacity and QOL relate to changes in symptom burden and clinician-rated cGVHD severity. Methods Between December 2008 and February 2011, patients with ScGVHD enrolled in a single-arm prospective clinical trial assessing the efficacy of imatinib mesylate. At baseline and 6 months, patients completed measures of functioning [Disabilities of the Arm, Shoulder, and Hand (DASH); Human Activity Profile (HAP); Manual Ability Measure (MAM-36)] and QOL [Short Form 36 version 2 (SF 36); Lee Symptom Scale (Lee)]. Clinicians rated patients' ADL ability [Assessment of Motor and Process Skills version 7 (AMPS)] and cGVHD symptom severity [Provider Global Score (PGS)]. Spearman's rank correlation tests evaluated the association among measures. The alpha level was set to 0.01. Results Twenty patients with ScGVHD enrolled in the trial; 13 patients were assessable for primary endpoint analysis. At study entry, patients were a median of 53 years old, a median of 53 months post-transplant, and had a median of 5 cGVHD-affected organs. Change in PGS was not correlated with change of any measures. Reduced symptom burden (Lee) correlated with improved SF 36 physical component scores (r = -0.72, p = 0.008), AMPS ADL motor skill scores (r = -0.84, p Conclusion Our results suggest the value of supplementing the Lee scale and the SF 36 with functional measures to assess clinical response to ScGVHD treatment. The association between changes in symptom burden and changes in ADL ability (AMPS and DASH scores) indicate the potential utility of these measures in this clinical population. Such scales may allow for a better understanding of patients' functional limitations, thereby promoting individualized clinical care and rehabilitative efforts. Clinician-reported cGVHD severity may not have been related to functional changes due to insufficient sensitivity of the measure or the small size of this sample. Our findings highlight the potential relevance of the AMPS and DASH in patients with cGVHD, though further exploration in larger and more diverse samples is necessary.