ArticleHaematologica2011
Sirolimus demonstrates activity in the primary therapy of acute graft-versus-host disease without systemic glucocorticoids.
Article in Haematologica, 2011. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 12 papers.
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Who cites it
12 citing papers in PubMed, 21 citations in OpenAlex.
- Trial
- Current approaches to prevent and treat GVHD after allogeneic stem cell transplantation.Hematology. American Society of Hematology. Education Program · 2018Review
- New and emerging therapies for acute and chronic graftTherapeutic advances in hematology · 2018Review
- Everolimus for pediatric patients with acute graft-versus-host disease after hematopoietic stem cell transplantation: A pilot study.Medicine · 2017Article
- Outcomes of high-grade gastrointestinal graft-versus-host disease posthematopoietic stem cell transplantation in children.Medicine · 2016Article
- New perspectives on the use of mTOR inhibitors in allogeneic haematopoietic stem cell transplantation and graft-versus-host disease.British journal of clinical pharmacology · 2016Review
- Therapeutic targets and emerging treatment options in gastrointestinal acute graft-versus-host disease.Expert opinion on orphan drugs · 2016Article
- 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.Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation · 2015Review
- Blood and marrow transplant clinical trials network state of the Science Symposium 2014.Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation · 2015Article
- Thinking out of the box--new approaches to controlling GVHD.Current hematologic malignancy reports · 2014Review
- Have we improved in preventing and treating acute graft-versus-host disease?Current opinion in hematology · 2011Review
- Graft-versus-host disease therapy: something else beyond glucocorticoids?Haematologica · 2011Article
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Authors and funding
12 authors at 2 institutions in 1 country.
Funding
Abstract
backgroundAdvances in acute graft-versus-host disease therapy are needed. DESIGN AND
methodsWe examined the efficacy of sirolimus as primary therapy for acute graft-versus-host disease in 32 patients.
resultsAcute graft-versus-host disease involved the skin in 53% of cases, gastrointestinal tract in 66%, liver in 16%. The syndrome was overall grade 1 in 12% cases, grade 2 in 75%, and grade 3 in 13%. Sirolimus was targeted to achieve serum trough levels of 5-14 ng/mL. Sixteen (50%) patients achieved sustained, complete resolution of acute graft-versus-host disease with sirolimus alone. In contrast, 19 of 32 (59%) matched historical controls treated with standard 1 mg/kg steroids achieved complete response (P=0.47). With median follow-up time for surviving patients of 16 (range 6-26) months, one year overall survival was 56% (95% CI 38-74%). The cumulative incidence of relapse at one year was 37% (95% CI 23-60%), and mortality in remission was 20% (95% CI 10-42%). The cumulative incidence of chronic graft-versus-host disease was 55% (95% CI 39-79%). Thrombotic microangiopathy occurred in 3 cases (grade 1 n=1; grade 2 n=2), and responded to dose reduction of calcineurin inhibitor.
conclusionsIn this retrospective series, sirolimus demonstrates activity comparable to that of high-dose glucocorticoids in the primary therapy of acute graft-versus-host disease. Confirmation of this activity requires prospective clinical trials.
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Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.