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Review
. 2025 Feb 7;14(4):238.
doi: 10.3390/cells14040238.

Treatment Response in Individual Organs Affected by Chronic Graft-Versus-Host Disease

Takanobu Morishita  1 , Paul J Martin  2 , Yoshihiro Inamoto  1
Affiliations
Review

Treatment Response in Individual Organs Affected by Chronic Graft-Versus-Host Disease

Takanobu Morishita et al. Cells. .

Abstract

Chronic graft-versus-host disease (GVHD) occurs in 30-70% of patients after allogeneic hematopoietic cell transplantation (HCT) and increases the risks of morbidity and mortality. Systemic corticosteroids are the standard initial treatment, but one-third of patients require subsequent treatment with other systemic agents. Treatment decisions are often based on physicians' experience. The expected treatment response rates in specific organs affected by chronic GVHD may inform such decisions. In this review, we identify 20 studies reporting treatment response rates in individual organs according to objective criteria, summarize the results, discuss the caveats in data interpretation, identify the unmet needs, and suggest future directions in the field. For cutaneous sclerosis, we observed large discrepancies in organ response rates according to the current NIH criteria and patient-reported improvement, highlighting the need for better measurement tools. High response rates for lung involvement with certain novel drugs deserve further investigation.

Keywords: axatilimab; belumosudil; chronic graft-versus-host disease; extracorporeal photopheresis; ibrutinib; keyword allogeneic hematopoietic cell transplantation; organ response; ruxolitinib.

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Conflict of interest statement

T.M. declares no conflicts of interest. Y.I. received honoraria for speaking from Meiji Seika Pharma Co., Ltd.; Novartis Pharma K.K.; and Janssen Pharmaceutical K.K., and research grants from Meiji Seika Pharma Co., Ltd.; and Incyte. P.J.M. has received honoraria from Pfizer for service on data and safety monitoring committees.

Figures

Figure 1
Figure 1
PRISMA diagram. This flow diagram illustrates the review process for study identification, screening, eligibility assessment, and inclusion. Each box provides the number of studies at that stage, and arrows indicate the flow between stages. * Initial search terms: Search: “Graft versus host disease” AND “Date-Publication from 1 January 2005 to 24 October 2024” NOT “review” NOT “case report” NOT “systemic review” NOT “meta-analysis”. Filters: Full text, English.
Figure 2
Figure 2
Quality assessment for individual reports. This figure shows the total number of quality criteria met by each of the 20 reports selected from the literature review. Y indicates the criteria met by each report, and %Meet indicates the percent of reports that met each criterion. ECP = extracorporeal photopheresis; P = prospective; R = retrospective.
Figure 3
Figure 3
Pooled response rates at 24 weeks for individual drugs according to organs. Best response rates are shown in black and response rates at a fixed point of 24 weeks are shown in red. Diamonds and squares represent mean values with bars showing 95% confidence intervals. The vertical grey line indicates overall means of all drugs regardless of which endpoint was used. ECP = extracorporeal photopheresis, GI = gastrointestinal. These met each criterion.
See this image and copyright information in PMC

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