title: NIH response criteria measures are associated with important parameters of disease severity in patients with chronic GVHD.
abstract: Lack of standardized criteria measuring therapeutic response remains an obstacle to the development of better treatments for chronic GVHD (cGVHD). This cross-sectional prospective study examined the concurrent and predictive validity of 18 clinician-reported ('Form A') and 8 patient-reported ('Form B') response measures proposed by NIH criteria. Concurrent parameters of interest were NIH global score, cGVHD activity, Lee symptom score and SF36 PCS. Patient cohort included 193 adults with moderate-to-severe cGVHD. Measures associated with the highest number of outcomes were lung function score (LFS), 2-min walk, grip strength, 4-point health-care provider (HCP) and patient global scores, 11-point clinician- and patient-reported global symptom severity scores, and Karnofsky performance score (KPS). Measures associated with survival in univariate analyses led to a Cox model containing skin erythema, LFS, KPS, eosinophil count and interval from cGVHD diagnosis to enrollment as jointly associated with survival. In conclusion, 4-point HCP and patient global scores and 11-point clinician- and patient-reported global symptom severity scores are associated with the majority of concurrent outcomes. Skin erythema is a potentially reversible sign of cGVHD that is associated with survival. These results define a subset of measures that should be prioritized for evaluation in future studies.
authors.affiliation: 1] Medical Oncology Service, National Institutes of Health, National Cancer Institute, Bethesda, MD, USA [2] Experimental Transplantation and Immunology Branch, National Institutes of Health, National Cancer Institute, Bethesda, MD, USA.
authors.forename: L M
authors.initials: LM
authors.lastname: Curtis
authors.affiliation: 1] Experimental Transplantation and Immunology Branch, National Institutes of Health, National Cancer Institute, Bethesda, MD, USA [2] Department of Hematology, Clinical Hospital Center Zagreb, University of Zagreb, Zagreb, Croatia.
authors.forename: L
authors.initials: L
authors.lastname: Grkovic
authors.affiliation: Outcomes Research Branch; Division of Cancer Control and Population Sciences, National Institutes of Health, National Cancer Institute, Bethesda, MD, USA.
authors.forename: S A
authors.initials: SA
authors.lastname: Mitchell
authors.affiliation: Biostatistics and Data Management Section, National Institutes of Health, National Cancer Institute, Rockville, MD, USA.
authors.forename: S M
authors.initials: SM
authors.lastname: Steinberg
authors.affiliation: Dermatology Branch, National Institutes of Health, National Cancer Institute, Bethesda, MD, USA.
authors.forename: E W
authors.initials: EW
authors.lastname: Cowen
authors.affiliation: National Institutes of Health, National Eye Institute, Bethesda, MD, USA.
authors.forename: M B
authors.initials: MB
authors.lastname: Datiles
authors.affiliation: National Institute of Dental and Craniofacial Research, National Institutes of Health, Bethesda, MD, USA.
authors.forename: J
authors.initials: J
authors.lastname: Mays
authors.affiliation: National Institute of Dental and Craniofacial Research, National Institutes of Health, Bethesda, MD, USA.
authors.forename: C
authors.initials: C
authors.lastname: Bassim
authors.affiliation: Rehabilitation Medicine Department, Clinical Center, National Institutes of Health, Bethesda, MD, USA.
authors.forename: G
authors.initials: G
authors.lastname: Joe
authors.affiliation: Rehabilitation Medicine Department, Clinical Center, National Institutes of Health, Bethesda, MD, USA.
authors.forename: L E
authors.initials: LE
authors.lastname: Comis
authors.affiliation: Pain and Palliative Care Medicine Department, Clinical Center, National Institutes of Health, Bethesda, MD, USA.
authors.forename: A
authors.initials: A
authors.lastname: Berger
authors.affiliation: Experimental Transplantation and Immunology Branch, National Institutes of Health, National Cancer Institute, Bethesda, MD, USA.
authors.forename: D
authors.initials: D
authors.lastname: Avila
authors.affiliation: Experimental Transplantation and Immunology Branch, National Institutes of Health, National Cancer Institute, Bethesda, MD, USA.
authors.forename: T
authors.initials: T
authors.lastname: Taylor
authors.affiliation: Department of Hematology, Clinical Hospital Center Zagreb, University of Zagreb, Zagreb, Croatia.
authors.forename: D
authors.initials: D
authors.lastname: Pulanic
authors.affiliation: Experimental Transplantation and Immunology Branch, National Institutes of Health, National Cancer Institute, Bethesda, MD, USA.
authors.forename: K
authors.initials: K
authors.lastname: Cole
authors.affiliation: Experimental Transplantation and Immunology Branch, National Institutes of Health, National Cancer Institute, Bethesda, MD, USA.
authors.forename: J
authors.initials: J
authors.lastname: Baruffaldi
authors.affiliation: Experimental Transplantation and Immunology Branch, National Institutes of Health, National Cancer Institute, Bethesda, MD, USA.
authors.forename: D H
authors.initials: DH
authors.lastname: Fowler
authors.affiliation: Experimental Transplantation and Immunology Branch, National Institutes of Health, National Cancer Institute, Bethesda, MD, USA.
authors.forename: R E
authors.initials: RE
authors.lastname: Gress
authors.affiliation: Experimental Transplantation and Immunology Branch, National Institutes of Health, National Cancer Institute, Bethesda, MD, USA.
authors.forename: S Z
authors.initials: SZ
authors.lastname: Pavletic
mesh_terms: D000293:Adolescent; D000328:Adult; D000368:Aged; D003430:Cross-Sectional Studies; D005260:Female; D006086:Graft vs Host Disease / Q000276:immunology*; D019337:Hematologic Neoplasms / Q000628:therapy*; D018380:Hematopoietic Stem Cell Transplantation; D006801:Humans; D017567:Karnofsky Performance Status; D008297:Male; D008875:Middle Aged; D009316:National Institutes of Health (U.S.); D017063:Outcome Assessment, Health Care; D011237:Predictive Value of Tests; D016016:Proportional Hazards Models; D011446:Prospective Studies; D012129:Respiratory Function Tests; D019172:Transplantation Conditioning / Q000379:methods* / Q000592:standards; D016896:Treatment Outcome; D014481:United States; D055815:Young Adult