Association of severity of organ involvement with mortality and recurrent malignancy in patients with chronic graft-versus-host disease

Inamoto, Yoshihiro; Martin, Paul J.; Storer, Barry E.; Palmer, Jeanne; Weisdorf, Daniel J.; Pidala, Joseph; Flowers, Mary E.D.; Arora, Mukta; Jagasia, Madan; Arai, Sally; Chai, Xiaoyu; Pavletić, Steven Z.; Vogelsang, Georgia B.; Lee, Stephanie J.

doi:10.3324/haematol.2014.109611

Cited by 29 publications

(14 citation statements)

References 26 publications

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“…Complete response and successful withdrawal of systemic treatment after resolution of the disease were considered most appropriate as primary endpoints in phase 3 studies, while non-relapse mortality, survival without recurrent malignancy and overall survival were considered appropriate as secondary endpoints. Notably, in certain subsets of patients who have chronic GVHD with a moderately severe global NIH rating, mortality rates are very low (see on-line Supplement) [23, 26], leaving little opportunity to demonstrate improvement in overall survival.…”

Section: Introductionmentioning

confidence: 99%

National Institutes of Health Consensus Development Project on Criteria for Clinical Trials in Chronic Graft-versus-Host Disease: VI. The 2014 Clinical Trial Design Working Group Report

Martin

Lee

Przepiorka

et al. 2015

Biology of Blood and Marrow Transplantation

105

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Treatment of chronic GVHD is intended to produce a sustainable benefit by reducing symptom burden, controlling objective manifestations of disease activity, preventing damage and impairment, and improving overall survival without causing disproportionate harms related to the treatment itself. Successful management can control the disease until systemic treatment is no longer needed. The complexity of the disease, the extended duration of follow-up needed to observe disease resolution and withdrawal of immunosuppressive treatment, and the lack of fully developed shorter-term endpoints impede progress in the field. Identification and characterization of primary endpoints demonstrating clinical benefit without the need for years of follow-up is an urgent need, with the understanding that clinical benefit encompasses not only the self-evident benefit of the primary endpoint but also any other associated benefits. This report discusses regulatory considerations, eligibility criteria, the value of controlled trial designs, the merits of proposed primary endpoints, and key considerations elaborated from experience and progress during the past decade. The report concludes by mapping an overall approach that could support and lead to maximally informative clinical trials, especially those that seek to demonstrate clinical benefit along a pathway to regulatory review and approval.

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Section: Introductionmentioning

confidence: 99%

National Institutes of Health Consensus Development Project on Criteria for Clinical Trials in Chronic Graft-versus-Host Disease: VI. The 2014 Clinical Trial Design Working Group Report

Martin

Lee

Przepiorka

et al. 2015

Biology of Blood and Marrow Transplantation

105

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“…For the lung, pulmonary function test results are used if available, otherwise the lung score is based on symptoms. Higher organ scores for the skin, 57,58 lung, 59 GI tract, 60 and liver 61 have been associated with worse survival.…”

mentioning

confidence: 99%

Classification systems for chronic graft-versus-host disease

Lee

2017

Blood

237

184

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Chronic graft versus host disease (GVHD) is a major cause of morbidity and mortality after allogeneic hematopoietic cell transplantation. Clinically, chronic GVHD is a pleiotropic, multiorgan syndrome involving tissue inflammation and fibrosis that often results in permanent organ dysfunction. Chronic GVHD is fundamentally caused by replacement of the host’s immune system with donor cells, although the heterogeneity of clinical manifestations suggests that patient, donor, and transplant factors modulate the phenotype. The diagnosis of chronic GVHD and determination of treatment response largely rely on clinical examination and patient interview. The 2005 and 2014 National Institutes of Health Consensus Development Projects on Criteria for Clinical Trials in Chronic GVHD standardized the terminology around chronic GVHD classification systems to ensure that a common language and procedures are being used in clinical research. This review provides a summary of these recommendations and illustrates how they are being used in clinical research and the potential for their use in clinical care.

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“…[11][12][13] In 2014, the NIH Conference was reconvened, and revisions are under consideration to update the recommendations based on available evidence and insights from clinical application of the original recommendations. [14][15][16][17][18][19][20][21][22][23][24][25][26][27][28][29][30][31][32][33] Chronic GVHD has a wide range of pleomorphic manifestations, and many complications can emerge from both the disease and its treatment. A dedicated multidisciplinary team approach with relevant expertise is necessary in order to provide the best care for patients with a chronic illness that can have devastating effects on quality of life.…”

Section: Introductionmentioning

confidence: 99%

How we treat chronic graft-versus-host disease

Flowers

Martin

2015

Blood

305

231

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Chronic graft-versus-host disease (GVHD) remains a common and potentially life-threatening complication of allogeneic hematopoietic stem cell transplantation (HCT). The 2-year cumulative incidence of chronic GVHD requiring systemic treatment is ∼30% to 40% by National Institutes of Health criteria. The risk of chronic GVHD is higher and the duration of treatment is longer after HCT with mobilized blood cells than with marrow cells. Clinical manifestations can impair activities of daily living and often linger for years. Hematology and oncology specialists who refer patients to centers for HCT are often subsequently involved in the management of chronic GVHD when patients return to their care after HCT. Treatment of these patients can be optimized under shared care arrangements that enable referring physicians to manage long-term administration of immunosuppressive medications and supportive care with guidance from transplant center experts. Keys to successful collaborative management include early recognition in making the diagnosis of chronic GVHD, comprehensive evaluation at the onset and periodically during the course of the disease, prompt institution of systemic and topical treatment, appropriate monitoring of the response, calibration of treatment intensity over time in order to avoid overtreatment or undertreatment, and the use of supportive care to prevent complications and disability.

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Association of severity of organ involvement with mortality and recurrent malignancy in patients with chronic graft-versus-host disease

Cited by 29 publications

References 26 publications

National Institutes of Health Consensus Development Project on Criteria for Clinical Trials in Chronic Graft-versus-Host Disease: VI. The 2014 Clinical Trial Design Working Group Report

National Institutes of Health Consensus Development Project on Criteria for Clinical Trials in Chronic Graft-versus-Host Disease: VI. The 2014 Clinical Trial Design Working Group Report

Classification systems for chronic graft-versus-host disease

How we treat chronic graft-versus-host disease

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