Wolff, Daniel, Radojcic, Vedran, Lafyatis, Robert, Cinar, Resat, Rosenstein, Rachel K., Cowen, Edward W., Cheng, Guang-Shing, Sheshadri, Ajay, Bergeron, Anne, Williams, Kirsten M., Todd, Jamie L., Teshima, Takanori, Cuvelier, Geoffrey D. E., Holler, Ernst, McCurdy, Shannon R., Jenq, Robert R., Hanash, Alan M., Jacobsohn, David, Santomasso, Bianca D., Jain, Sandeep, Ogawa, Yoko, Steven, Philipp ORCID: 0000-0001-6892-3619, Luo, Zhonghui Katie, Dietrich-Ntoukas, Tina, Saban, Daniel, Bilic, Ervina, Penack, Olaf, Griffith, Linda M., Cowden, Meredith, Martin, Paul J., Greinix, Hildegard T., Sarantopoulos, Stefanie, Socie, Gerard, Blazar, Bruce R., Pidala, Joseph, Kitko, Carrie L., Couriel, Daniel R., Cutler, Corey, Schultz, Kirk R., Pavletic, Steven Z., Lee, Stephanie J. and Paczesny, Sophie (2021). National Institutes of Health Consensus Development Project on Criteria for Clinical Trials in Chronic Graft-versus-Host Disease: IV. The 2020 Highly morbid forms report. Transpl. Cell. Ther., 27 (10). S. 817 - 836. NEW YORK: ELSEVIER SCIENCE INC. ISSN 2666-6367

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Abstract

Chronic graft-versus-host disease (GVHD) can be associated with significant morbidity, in part because of nonreversible fibrosis, which impacts physical functioning (eye, skin, lung manifestations) and mortality (lung, gastrointestinal manifestations). Progress in preventing severe morbidity and mortality associated with chronic GVHD is limited by a complex and incompletely understood disease biology and a lack of prognostic biomarkers. Likewise, treatment advances for highly morbid manifestations remain hindered by the absence of effective organ-specific approaches targeting irreversible fibrotic sequelae and difficulties in conducting clinical trials in a heterogeneous disease with small patient numbers. The purpose of this document is to identify current gaps, to outline a roadmap of research goals for highly morbid forms of chronic GVHD including advanced skin sclerosis, fasciitis, lung, ocular and gastrointestinal involvement, and to propose strategies for effective trial design. The working group made the following recommendations: (1) Phenotype chronic GVHD clinically and biologically in future cohorts, to describe the incidence, prognostic factors, mechanisms of organ damage, and clinical evolution of highly morbid conditions including long-term effects in children; (2) Conduct longitudinal multicenter studies with common definitions and research sample collections; (3) Develop new approaches for early identification and treatment of highly morbid forms of chronic GVHD, especially biologically targeted treatments, with a special focus on fibrotic changes; and (4) Establish primary endpoints for clinical trials addressing each highly morbid manifestation in relationship to the time point of intervention (early versus late). Alternative endpoints, such as lack of progression and improvement in physical functioning or quality of life, may be suitable for clinical trials in patients with highly morbid manifestations. Finally, new approaches for objective response assessment and exploration of novel trial designs for small populations are required. (C) 2021 The American Society for Transplantation and Cellular Therapy. Published by Elsevier Inc. All rights reserved.

Item Type: Journal Article
Creators:
CreatorsEmailORCIDORCID Put Code
Wolff, DanielUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Radojcic, VedranUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Lafyatis, RobertUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Cinar, ResatUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Rosenstein, Rachel K.UNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Cowen, Edward W.UNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Cheng, Guang-ShingUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Sheshadri, AjayUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Bergeron, AnneUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Williams, Kirsten M.UNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Todd, Jamie L.UNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Teshima, TakanoriUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Cuvelier, Geoffrey D. E.UNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Holler, ErnstUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
McCurdy, Shannon R.UNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Jenq, Robert R.UNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Hanash, Alan M.UNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Jacobsohn, DavidUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Santomasso, Bianca D.UNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Jain, SandeepUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Ogawa, YokoUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Steven, PhilippUNSPECIFIEDorcid.org/0000-0001-6892-3619UNSPECIFIED
Luo, Zhonghui KatieUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Dietrich-Ntoukas, TinaUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Saban, DanielUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Bilic, ErvinaUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Penack, OlafUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Griffith, Linda M.UNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Cowden, MeredithUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Martin, Paul J.UNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Greinix, Hildegard T.UNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Sarantopoulos, StefanieUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Socie, GerardUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Blazar, Bruce R.UNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Pidala, JosephUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Kitko, Carrie L.UNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Couriel, Daniel R.UNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Cutler, CoreyUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Schultz, Kirk R.UNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Pavletic, Steven Z.UNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Lee, Stephanie J.UNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Paczesny, SophieUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
URN: urn:nbn:de:hbz:38-608000
DOI: 10.1016/j.jtct.2021.06.001
Journal or Publication Title: Transpl. Cell. Ther.
Volume: 27
Number: 10
Page Range: S. 817 - 836
Date: 2021
Publisher: ELSEVIER SCIENCE INC
Place of Publication: NEW YORK
ISSN: 2666-6367
Language: English
Faculty: Unspecified
Divisions: Unspecified
Subjects: no entry
Uncontrolled Keywords:
KeywordsLanguage
STEM-CELL TRANSPLANTATION; BRONCHIOLITIS OBLITERANS SYNDROME; MURINE CHRONIC GVHD; MACHINE LEARNING ALGORITHMS; CENTRAL-NERVOUS-SYSTEM; A-COUPLED LIPOSOMES; AIR-FLOW DECLINE; RISK-FACTORS; DRY EYE; OCULAR SURFACEMultiple languages
Hematology; Immunology; TransplantationMultiple languages
URI: http://kups.ub.uni-koeln.de/id/eprint/60800

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