ArticleBlood cell therapy2024
Sirolimus as add-on therapy to corticosteroids for moderate-severe chronic graft versus host disease.
Article in Blood cell therapy, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 1 paper, 1 of them a synthesis that pooled it.
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
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Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
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Who cites it
1 citing paper in PubMed, 1 synthesis or guideline pooled it.
- Evolving Paradigms in Chronic Graft-Versus-Host Disease: From Global Immunosuppression to Precision Targeted and Cell-Based Therapies.Stem cell reviews and reports · 2026Pooled it
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Authors and funding
12 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Introduction: The first-line treatment of moderate-severe chronic graft versus host disease (cGVHD) involves systemic corticosteroids ± calcineurin inhibitors. Around half of the patients will need second-line agents for corticosteroid-refractory/dependent (SR/SD) cGVHD. Herein, we report our experience using sirolimus as an add-on agent to corticosteroids in moderate-severe cGVHD. Methods: This was a single-center study of allogeneic cell transplant recipients aged ≥ 12 during 2016-2022. The diagnosis and severity of cGVHD were as per the NIH-2014 criteria. At the physician's discretion, sirolimus was added to corticosteroids for moderate to severe cGVHD. The GVHD response was classified based on the EBMT-NIH-CIBMTR criteria. Results: cGVHD occurred in 66 (49%) out of 134 recipients. It was mild in 13 (10%) and moderate-severe in 53 (39%) recipients. Sirolimus was used in 38 out of 53 (71.6%) patients with moderate-severe cGVHD, with equal proportions of matched-related (53%) and haploidentical HCT (47%) recipients. The median time to onset of cGVHD was 140 days (IQR 108-182). The onset was de novo in 14 (37%), quiescent in 15 (39%), and progressive in 9 (24%) patients. The median duration on sirolimus was 283 days (134-640). cGVHD was controlled in 30 (79%) and active in 8 (21%) recipients at 6 months. Dyslipidemia was the most common (73%) adverse event. Failure-free survival at two years was 61% (95% CI 38-78%). Discussion: This study demonstrates the safety and efficacy of sirolimus as an add-on agent to systemic corticosteroids in managing moderate-severe cGVHD. This strategy can reduce the burden of SR/SD cGVHD.
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