Biology of Blood and Marrow Transplantation
Volume 12, Issue 3 , Pages 252-266 , March 2006

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

  • Steven Z. Pavletic

      Affiliations

    • National Cancer Institute, National Institutes of Health, Bethesda, Maryland
    • Corresponding Author InformationCorrespondence and reprint requests: Steven Z. Pavletic, MD, Graft-versus-Host and Autoimmunity Unit, Experimental Transplantation and Immunology Branch, National Cancer Institute, 9000 Rockville Pike, Bethesda, MD 20892-1203
  • ,
  • Paul Martin

      Affiliations

    • Fred Hutchinson Cancer Research Center, University of Washington School of Medicine, Seattle, Washington
  • ,
  • Stephanie J. Lee

      Affiliations

    • Dana-Farber Cancer Institute, Boston, Massachusetts
  • ,
  • Sandra Mitchell

      Affiliations

    • National Cancer Institute, National Institutes of Health, Bethesda, Maryland
  • ,
  • David Jacobsohn

      Affiliations

    • Children’s Memorial Hospital, Northwestern University School of Medicine, Chicago, Illinois
  • ,
  • Edward W. Cowen

      Affiliations

    • National Cancer Institute, National Institutes of Health, Bethesda, Maryland
  • ,
  • Maria L. Turner

      Affiliations

    • National Cancer Institute, National Institutes of Health, Bethesda, Maryland
  • ,
  • Gorgun Akpek

      Affiliations

    • University of Maryland School of Medicine, Baltimore, Maryland
  • ,
  • Andrew Gilman

      Affiliations

    • University of North Carolina School of Medicine, Chapel Hill, North Carolina
  • ,
  • George McDonald

      Affiliations

    • Fred Hutchinson Cancer Research Center, University of Washington School of Medicine, Seattle, Washington
  • ,
  • Mark Schubert

      Affiliations

    • Fred Hutchinson Cancer Research Center, University of Washington School of Medicine, Seattle, Washington
  • ,
  • Ann Berger

      Affiliations

    • Warren Grant Magnuson Clinical Center, National Institutes of Health, Bethesda, Maryland
  • ,
  • Peter Bross

      Affiliations

    • US Food and Drug Administration, Rockville, Maryland
  • ,
  • Jason W. Chien

      Affiliations

    • Fred Hutchinson Cancer Research Center, University of Washington School of Medicine, Seattle, Washington
  • ,
  • Daniel Couriel

      Affiliations

    • University of Texas M.D. Anderson Cancer Center, Houston, Texas
  • ,
  • J.P. Dunn

      Affiliations

    • Johns Hopkins University School of Medicine, Baltimore, Maryland
  • ,
  • Jane Fall-Dickson

      Affiliations

    • National Institute of Nursing Research, National Institutes of Health, Bethesda, Maryland
  • ,
  • Ann Farrell

      Affiliations

    • US Food and Drug Administration, Rockville, Maryland
  • ,
  • Mary E.D. Flowers

      Affiliations

    • Fred Hutchinson Cancer Research Center, University of Washington School of Medicine, Seattle, Washington
  • ,
  • Hildegard Greinix

      Affiliations

    • Medical University of Vienna, Vienna, Austria
  • ,
  • Steven Hirschfeld

      Affiliations

    • US Food and Drug Administration, Rockville, Maryland
  • ,
  • Lynn Gerber

      Affiliations

    • Warren Grant Magnuson Clinical Center, National Institutes of Health, Bethesda, Maryland
  • ,
  • Stella Kim

      Affiliations

    • University of Texas M.D. Anderson Cancer Center, Houston, Texas
  • ,
  • Robert Knobler

      Affiliations

    • Medical University of Vienna, Vienna, Austria
  • ,
  • Peter A. Lachenbruch

      Affiliations

    • US Food and Drug Administration, Rockville, Maryland
  • ,
  • Frederick W. Miller

      Affiliations

    • National Institute of Environmental Health Sciences, National Institutes of Health, Bethesda, Maryland
  • ,
  • Barbara Mittleman

      Affiliations

    • National Institute of Arthritis and Musculoskeletal and Skin Diseases, Bethesda, Maryland
  • ,
  • Esperanza Papadopoulos

      Affiliations

    • Memorial Sloan-Kettering Cancer Center, New York, New York
  • ,
  • Susan K. Parsons

      Affiliations

    • Tufts-New England Medical Center, Boston, Massachusetts
  • ,
  • Donna Przepiorka

      Affiliations

    • University of Tennessee, Memphis, Tennessee
  • ,
  • Michael Robinson

      Affiliations

    • National Eye Institute; National Institutes of Health; Bethesda, Maryland
  • ,
  • Michael Ward

      Affiliations

    • National Institute of Arthritis and Musculoskeletal and Skin Diseases, Bethesda, Maryland
  • ,
  • Bryce Reeve

      Affiliations

    • National Cancer Institute, National Institutes of Health, Bethesda, Maryland
  • ,
  • Lisa G. Rider

      Affiliations

    • National Institute of Environmental Health Sciences, National Institutes of Health, Bethesda, Maryland
  • ,
  • Howard Shulman

      Affiliations

    • Fred Hutchinson Cancer Research Center, University of Washington School of Medicine, Seattle, Washington
  • ,
  • Kirk R. Schultz

      Affiliations

    • University of British Columbia, British Columbia Children’s Hospital, Vancouver, British Columbia, Canada
  • ,
  • Daniel Weisdorf

      Affiliations

    • University of Minnesota, Minneapolis, Minnesota
  • ,
  • Georgia B. Vogelsang

      Affiliations

    • Johns Hopkins University School of Medicine, Baltimore, Maryland

Received 17 January 2006 ,Accepted 18 January 2006.

References 

  1. Stewart BL , Storer B , Storek J , et al.   Duration of immunosuppressive treatment for chronic graft-versus-host disease . Blood . 2004;104:3501–3506
  2. Pavletic SZ , Carter SL , Kernan NA , et al.   Influence of T-cell depletion on chronic graft-versus-host disease (results of a multicenter randomized trial in unrelated marrow donor transplantation) . Blood . 2005;106:3308–3313
  3. Koc S , Leisenring W , Flowers ME , et al.   Therapy for chronic graft-versus-host disease (a randomized trial comparing cyclosporine plus prednisone versus prednisone alone) . Blood . 2002;100:48–51
  4. Farag SS . Chronic graft-versus-host disease (where do we go from here?) . Bone Marrow Transplant . 2004;33:569–577
  5. Felson DT , Anderson JJ , Boers M , et al.   The American College of Rheumatology preliminary core set of disease activity measures for rheumatoid arthritis clinical trials (the committee on outcome measures in rheumatoid arthritis clinical trials) . Arthritis Rheum . 1993;36:729–740
  6. Ward MM , Marx AS , Barry NN . Comparison of the validity and sensitivity to change of 5 activity indices in systemic lupus erythematosus . J Rheumatol . 2000;27:664–670
  7. Sandborn WJ , Feagan BG , Hanauer SB , et al.   A review of activity indices and efficacy endpoints for clinical trials of medical therapy in adults with Crohn’s disease . Gastroenterology . 2002;122:512–530
  8. Rider LG , Giannini EH , Harris-Love M , et al.   Defining clinical improvement in adult and juvenile myositis . J Rheumatol . 2003;30:603–617
  9. Filipovich AH , Weisdorf D , Pavletic S , 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
  10. Acquadro C , Berzon R , Dubois D , et al.   Incorporating the patient’s perspective into drug development and communication (an ad hoc task force report of the patient-reported outcomes (PRO) harmonization group meeting at the Food and Drug Administration, February 16, 2001) . Value Health . 2003;6:522–531
  11. Revicki DA , Osoba D , Fairclough D , et al.   Recommendations on health-related quality of life research to support labeling and promotional claims in the United States . Qual Life Res . 2000;9:887–900
  12. Lee S , Cook EF , Soiffer R , et al.   Development and validation of a scale to measure symptoms of chronic graft-versus-host disease . Biol Blood Marrow Transplant . 2002;8:444–452
  13. Cleeland CS , Mendoza TR , Wang XS , et al.   Assessing symptom distress in cancer patients (the MD Anderson symptom inventory) . Cancer . 2000;89:1634–1646
  14. Osoba D , Rodrigues G , Myles J , et al.   Interpreting the significance of changes in health-related quality-of-life scores . J Clin Oncol . 1998;16:139–144
  15. Mathiowetz V , Kashman N , Volland G , et al.   Grip and pinch strength (normative data for adults) . Arch Phys Med Rehabil . 1985;66:69–74
  16. Mathiowetz V , Weber K , Volland G , et al.   Reliability and validity of grip and pinch strength evaluations . J Hand Surg [Am] . 1984;9:222–226
  17. Mathiowetz V , Wiemer DM , Federman SM . Grip and pinch strength (norms for 6- to 19-year-olds) . Am J Occup Ther . 1986;40:705–711
  18. Waters RL , Lunsford BR , Perry J , et al.   Energy-speed relationship of walking (standard tables) . J Orthop Res . 1988;6:215–222
  19. Daughton DM , Fix AJ , Kass I , et al.   Maximum oxygen consumption and the ADAPT quality-of-life scale . Arch Phys Med Rehabil . 1982;63:620–622
  20. Ware JE , Sherbourne CD . The MOS 36-item short-form health survey (SF-36), I (conceptual framework and item selection) . Med Care . 1992;30:473–483
  21. Ware JE . SF-36 health survey update . Spine . 2000;25:3130–3139
  22. McQuellon RP , Russell GB , Cella DF , et al.   Quality of life measurement in bone marrow transplantation (development of the functional assessment of cancer therapy-bone marrow transplant (FACT-BMT) scale) . Bone Marrow Transplant . 1997;19:357–368
  23. Plint AC , Gaboury I , Owen J , et al.   Activities scale for kids (an analysis of normals) . J Pediatr Orthop . 2003;23:788–790
  24. Young NL , Williams JI , Yoshida KK , et al.   Measurement properties of the activities scale for kids . J Clin Epidemiol . 2000;53:125–137
  25. Young NL , Yoshida KK , Williams JI , et al.   The role of children in reporting their physical disability . Arch Phys Med Rehabil . 1995;76:913–918
  26. Lansky SB , List MA , Lansky LL , et al.   The measurement of performance in childhood cancer patients . Cancer . 1987;60:1651–1656
  27. Parsons SK , Barlow SE , Levy SL , et al.   Health-related quality of life in pediatric bone marrow transplant survivors (according to whom?) . Int J Cancer Suppl . 1999;12:46–51
  28. Parsons SK , Shih MC , Duhamel KN , et al.   Original research article (maternal perspectives on children’s health-related quality of life during the first year after pediatric hematopoietic stem cell transplant) . J Pediatr Psychol . 2005;
  29. Parsons SK , Shih MC , Mayer DK , et al.   Preliminary psychometric evaluation of the child health ratings inventory (CHRIs) and disease-specific impairment inventory-hematopoietic stem cell transplantation (DSII-HSCT) in parents and children . Qual Life Res . 2005;14:1613–1625
  30. Seyger MM , van den Hoogen FH , de Boo T , et al.   Reliability of two methods to assess morphea (skin scoring and the use of a durometer) . J Am Acad Dermatol . 1997;37:793–796
  31. Aghassi D , Monoson T , Braverman I . Reproducible measurements to quantify cutaneous involvement in scleroderma . Arch Dermatol . 1995;131:1160–1166
  32. Falanga V , Bucalo B . Use of a durometer to assess skin hardness . J Am Acad Dermatol . 1993;29:47–51
  33. Gottlober P , Leiter U , Friedrich W , et al.   Chronic cutaneous sclerodermoid graft-versus-host disease (evaluation by 20-MHz sonography) . J Eur Acad Dermatol Venereol . 2003;17:402–407
  34. Dumford K , Anderson JC . CT and MRI findings in sclerodermatous chronic graft vs host disease . Clin Imaging . 2001;25:138–140
  35. Bron AJ , Evans VE , Smith JA . Grading of corneal and conjunctival staining in the context of other dry eye tests . Cornea . 2003;22:640–650
  36. Robinson MR , Lee SS , Rubin BI , et al.   Topical corticosteroid therapy for cicatricial conjunctivitis associated with chronic graft-versus-host disease . Bone Marrow Transplant . 2004;33:1031–1035
  37. Schiffman RM , Christianson MD , Jacobsen G , et al.   Reliability and validity of the ocular surface disease index . Arch Ophthalmol . 2000;118:615–621
  38. Schubert MM , Williams BE , Lloid ME , et al.   Clinical assessment scale for the rating of oral mucosal changes associated with bone marrow transplantation (development of an oral mucositis index) . Cancer . 1992;69:2469–2477
  39. Spinelli S , Chiodi S , Costantini S , et al.   Female genital tract graft-versus-host disease following allogeneic bone marrow transplantation . Haematologica . 2003;88:1163–1168
  40. Stratton PTM , Turner M . Vulvar and vaginal graft versus host disease in women after hematopoietic stem cell transplantation . J Soc Gynecol Investig . 2004;11:162A
  41. Hann DM , Denniston MM , Baker F . Measurement of fatigue in cancer patients (further validation of the fatigue symptom inventory) . Qual Life Res . 2000;9:847–854
  42. Buysse DJ , Reynolds CF , Monk TH , et al.   The Pittsburgh sleep quality index (a new instrument for psychiatric practice and research) . Psychiatry Res . 1989;28:193–213
  43. Carpenter JS , Andrykowski MA . Psychometric evaluation of the Pittsburgh sleep quality index . J Psychosom Res . 1998;45:5–13
  44. Mitchell S , Reeve B , Cowen E , et al.   Feasibility and reproducibility of the new NIH consensus criteria to evaluate response in chronic GVHD–a pilot study . [abstract] Blood . 2005;106(suppl 1):873a
  45. Vitali C , Bombardieri S , Jonsson R , et al.   Classification criteria for Sjögren’s syndrome (a revised version of the European criteria proposed by the American-European consensus group) . Ann Rheum Dis . 2002;61:554–558
  46. Lee SJ , Vogelsang G , Flowers ME . Chronic graft-versus-host disease . Biol Blood Marrow Transplant . 2003;9:215–233
  47. Jacobsohn DA , Schechter T , Seshadri R , et al.   Eosinophilia correlates with the presence or development of chronic graft-versus-host disease in children . Transplantation . 2004;77:1096–1100
  48. Jacobsohn DA , Margolis J , Doherty J , et al.   Weight loss and malnutrition in patients with chronic graft-versus-host disease . Bone Marrow Transplant . 2002;29:231–236
  49. Chien JW , Madtes DK , Clark JG . Pulmonary function testing prior to hematopoietic stem cell transplantation . Bone Marrow Transplant . 2005;35:429–435
  50. Parimon T , Madtes DK , Au DH , et al.   Pretransplant lung function, respiratory failure, and mortality after stem cell transplantation . Am J Respir Crit Care Med . 2005;172:384–390
  51. Norkin C , White D . Measurement of Joint Motion . Philadelphia: FA Davis Co; 1995;
  52. Matza LS , Swensen AR , Flood EM , et al.   Assessment of health-related quality of life in children (a review of conceptual, methodological, and regulatory issues) . Value Health . 2004;7:79–92
  53. Goodell TT , Nail LM . Operationalizing symptom distress in adults with cancer (a literature synthesis) . Oncol Nurs Forum . 2005;32:E42–E47
  54. Lee SJ , Klein JP , Barrett AJ , et al.   Severity of chronic graft-versus-host disease (association with treatment-related mortality and relapse) . Blood . 2002;100:406–414
  55. Nagashima M , Shu G , Yamamoto K , et al.   The ability of disease modifying antirheumatic drugs to induce and maintain improvement in patients with rheumatoid arthritis (epidemiology of DMARDs treatment in Japan) . Clin Exp Rheumatol . 2005;23:27–35
  56. Koller WC , Lyons KE , Truly W . Effect of levodopa treatment for parkinsonism in welders (a double-blind study) . Neurology . 2004;62:730–733
  57. Craig J , Young CA , Ennis M , et al.   A randomized controlled trial comparing rehabilitation against standard therapy in multiple sclerosis patients receiving intravenous steroid treatment . J Neurol Neurosurg Psychiatry . 2003;74:1225–1230
  58. Brooks D , Parsons J , Tran D , et al.   The two-minute walk test as a measure of functional capacity in cardiac surgery patients . Arch Phys Med Rehabil . 2004;85:1525–1530
  59. Eiser N , Willsher D , Dore CJ . Reliability, repeatability and sensitivity to change of externally and self-paced walking tests in COPD patients . Respir Med . 2003;97:407–414
  60. Akpek G , Zahurak ML , Piantadosi S , et al.   Development of a prognostic model for grading chronic graft-versus-host disease . Blood . 2001;97:1219–1226
  61. Wiklund I . Assessment of patient-reported outcomes in clinical trials (the example of health-related quality of life) . Fundam Clin Pharmacol . 2004;18:351–363
  62. Walters SJ , Brazier JE . What is the relationship between the minimally important difference and health state utility values? The case of the SF-6D . Health Qual Life Outcomes . 2003;1:4
  63. Socie G , Stone JV , Wingard JR , et al.   Long-term survival and late deaths after allogeneic bone marrow transplantation (late effects working committee of the international bone marrow transplant registry) . N Engl J Med . 1999;341:14–21
  64. Norman GR , Sloan JA , Wyrwich KW . Interpretation of changes in health-related quality of life (the remarkable universality of half a standard deviation) . Med Care . 2003;41:582–592
  65. Norman GR , Sloan JA , Wyrwich KW . Is it simple or simplistic? . Med Care . 2003;41:599–600
  66. Schultz K, Miklos D, Fowler D, et al. Towards biomarkers for chronic graft versus host disease. Biol Blood Marrow Transplant. In press.

PII: S1083-8791(06)00070-X

doi: 10.1016/j.bbmt.2006.01.008

Biology of Blood and Marrow Transplantation
Volume 12, Issue 3 , Pages 252-266 , March 2006