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.
This is a preview of subscription content, access via your institution
Access options
Subscribe to this journal
Receive 12 print issues and online access
$259.00 per year
only $21.58 per issue
Buy this article
- Purchase on Springer Link
- Instant access to full article PDF
Prices may be subject to local taxes which are calculated during checkout
Similar content being viewed by others
References
Lee SJ, Vogelsang G, Flowers ME . Chronic graft-versus-host disease. Biol Blood Marrow Transplant 2003; 9: 215–233.
Goldman JM, Majhail NS, Klein JP, Wang Z, Sobocinski KA, Arora M et al. Relapse and late mortality in 5-year survivors of myeloablative allogeneic hematopoietic cell transplantation for chronic myeloid leukemia in first chronic phase. J Clin Oncol 2010; 28: 1888–1895.
Lee SJ . Have we made progress in the management of chronic graft-vs-host disease? Best Pract Res Clin Haematol 2010; 23: 529–535.
Flowers ME, Storer B, Carpenter P, Rezvani AR, Vigorito AC, Campregher PV et al. Treatment change as a predictor of outcome among patients with classic chronic graft-versus-host disease. Biol Blood Marrow Transplant 2008; 14: 1380–1384.
Wolff D, Schleuning M, von Harsdorf S, Bacher U, Gerbitz A, Stadler M et al. Consensus conference on clinical practice in chronic GVHD: second-line treatment of chronic graft-versus-host disease. Biol Blood Marrow Transplant 2011; 17: 1–17.
Pavletic SZ, Martin P, Lee SJ, Mitchell S, Jacobsohn D, Cowen EW et al. Measuring therapeutic response in chronic graft-versus-host disease: National Institutes of Health Consensus Development Project on Criteria for Clinical Trials in Chronic Graft-versus-Host Disease: IV. Response Criteria Working Group report. Biol Blood Marrow Transplant 2006; 12: 252–266.
Olivieri A, Cimminiello M, Corradini P, Mordini N, Fedele R, Selleri C et al. Long-term outcome and prospective validation of NIH response criteria in 39 patients receiving imatinib for steroid-refractory chronic GVHD. Blood 2013; 122: 4111–4118.
Baird K, Steinberg SM, Grkovic L, Pulanic D, Cowen EW, Mitchell SA et al. National Institutes of Health chronic graft-versus-host disease staging in severely affected patients: organ and global scoring correlate with established indicators of disease severity and prognosis. Biol Blood Marrow Transplant 2013; 19: 632–639.
Filipovich AH, Weisdorf D, Pavletic S, Socie G, Wingard JR, Lee SJ et al. National Institutes of Health consensus development project on criteria for clinical trials in chronic graft-versus-host disease: I. Diagnosis and staging working group report. Biol Blood Marrow Transplant 2005; 11: 945–956.
Grkovic L, Baird K, Steinberg SM, Williams KM, Pulanic D, Cowen EW et al. Clinical laboratory markers of inflammation as determinants of chronic graft-versus-host disease activity and NIH global severity. Leukemia 2012; 26: 633–643.
Lee S, Cook EF, Soiffer R, Antin JH . Development and validation of a scale to measure symptoms of chronic graft-versus-host disease. Biol Blood Marrow Transplant 2002; 8: 444–452.
Hollander M, Wolfe DA . Nonparametric statistical methods. 2nd ednWiley: New York, 1999.
Agresti A . Categorical data analysis. 1st edn. Wiley: New York, 1990.
Felson DT, Anderson JJ, Boers M, Bombardier C, Furst D, Goldsmith C et al. American College of Rheumatology. Preliminary definition of improvement in rheumatoid arthritis. Arthritis Rheum 1995; 38: 727–735.
Best WR, Becktel JM, Singleton JW, Kern F Jr . Development of a Crohn's disease activity index. National Cooperative Crohn's Disease Study. Gastroenterology 1976; 70: 439–444.
Preston CC, Colman AM . Optimal number of response categories in rating scales: reliability, validity, discriminating power, and respondent preferences. Acta Psychol (Amst) 2000; 104: 1–15.
Inamoto Y, Chai X, Kurland BF, Cutler C, Flowers ME, Palmer JM et al. Validation of measurement scales in ocular graft-versus-host disease. Ophthalmology 2012; 119: 487–493.
Akpek G, Zahurak ML, Piantadosi S, Margolis J, Doherty J, Davidson R et al. Development of a prognostic model for grading chronic graft-versus-host disease. Blood 2001; 97: 1219–1226.
Jacobsohn DA, Kurland BF, Pidala J, Inamoto Y, Chai X, Palmer JM et al. Correlation between NIH composite skin score, patient-reported skin score, and outcome: results from the Chronic GVHD Consortium. Blood 2012; 120: 2545–2552 quiz 2774.
Wiener L, Baird K, Crum C, Powers K, Carpenter P, Baker KS et al. Child and parent perspectives of the chronic graft-versus-host disease (cGVHD) symptom experience: a concept elicitation study. Support Care Cancer 2014; 22: 295–305.
Mitchell SA, Leidy NK, Mooney KH, Dudley WN, Beck SL, LaStayo PC et al. Determinants of functional performance in long-term survivors of allogeneic hematopoietic stem cell transplantation with chronic graft-versus-host disease (cGVHD). Bone Marrow Transplant 2010; 45: 762–769.
Acknowledgements
This research was also supported also by National Institutes of Health, Clinical Center, Nursing and Patient Care Services. We thank all patients and their families who participated in the natural history of cGVHD protocol. This research was supported by the Intramural Research Program of the National Institutes of Health (NIH), National Cancer Institute, Center for Cancer Research. The authors are employees of the United States Government, and, as such, this work was carried out in that capacity. The views expressed do not necessarily represent the views of the National Institutes of Health or the United States Government.
Author information
Authors and Affiliations
Corresponding author
Ethics declarations
Competing interests
The authors declare no conflict of interest.
Additional information
Supplementary Information accompanies this paper on Bone Marrow Transplantation website
Supplementary information
Rights and permissions
About this article
Cite this article
Curtis, L., Grkovic, L., Mitchell, S. et al. NIH response criteria measures are associated with important parameters of disease severity in patients with chronic GVHD. Bone Marrow Transplant 49, 1513–1520 (2014). https://doi.org/10.1038/bmt.2014.188
Received:
Revised:
Accepted:
Published:
Issue Date:
DOI: https://doi.org/10.1038/bmt.2014.188
This article is cited by
-
Predictors and significance of kidney dysfunction in patients with chronic graft-versus-host disease
Bone Marrow Transplantation (2023)
-
Non-Expert Markings of Active Chronic Graft-Versus-Host Disease Photographs: Optimal Metrics of Training Effects
Journal of Digital Imaging (2022)
-
Rationale for the clinical use of adipose-derived mesenchymal stem cells for COVID-19 patients
Journal of Translational Medicine (2020)
-
Motor ability, function, and health-related quality of life as correlates of symptom burden in patients with sclerotic chronic graft-versus-host disease receiving imatinib mesylate
Supportive Care in Cancer (2020)
-
Overcoming human disagreement assessing erythematous lesion severity on 3D photos of chronic graft-versus-host disease
Bone Marrow Transplantation (2018)