{"doi":"10.1182/blood.2020005338","title":"Sirolimus with CSP and MMF as GVHD prophylaxis for allogeneic transplantation with HLA antigen–mismatched donors","abstract":"This trial aimed to evaluate the efficacy of sirolimus in addition to cyclosporine (CSP) and mycophenolate mofetil (MMF) for graft-versus-host disease (GVHD) prophylaxis after nonmyeloablative conditioning for HLA class I or II mismatched hematopoietic cell transplantation (HCT). Eligible patients had hematologic malignancies treatable by allogeneic HCT. Conditioning consisted of fludarabine (90 mg/m2) and 2 to 3 Gy total body irradiation. GVHD prophylaxis comprised cyclosporine, mycophenolate mofetil, and sirolimus. The primary objective was to determine whether the cumulative incidence of grade 2 to 4 acute GVHD could be reduced to <70% in HLA class I or II mismatched HCT. The study was closed on December 20, 2018. Seventy-seven participants were recruited between April 14, 2011, and December 12, 2018, of whom 76 completed the study intervention. Median follow-up was 47 months (range, 4-94 months). The cumulative incidence of grade 2 to 4 acute GVHD at day 100 was 36% (95% confidence interval [CI], 25-46), meeting the primary end point. The cumulative incidence of nonrelapse morality, relapse/progression, and overall survival was 18% (95% CI, 9-27), 30% (interquartile range, 19-40), and 62% (95% CI, 50-73) after 4 years. In conclusion, the addition of sirolimus to cyclosporine and mycophenolate mofetil resulted in a lower incidence of acute GVHD, thus translating into superior overall survival compared with historical results. This trial was registered at www.clinicaltrials.gov as #NCT01251575.","journal":"Blood","year":2020,"id":75376,"datarank":1.3816278648859939,"base_score":3.258096538021482,"endowment":3.258096538021482,"self_citation_contribution":0.4887144807032224,"citation_network_contribution":0.8929133841827716,"self_endowment_contribution":0.4887144807032224,"citer_contribution":0.8929133841827716,"corpus_percentile":null,"corpus_rank":null,"citation_count":25,"citer_count":20,"citers_with_citation_signal":15,"citers_with_endowment":15,"datacite_reuse_total":0,"is_dataset":false,"is_dataset_confidence":0.9563,"is_data_producer":true,"deposit_databanks":{"ClinicalTrials.gov":["NCT01251575"]},"is_oa":true,"file_count":0,"downloads":0,"has_version_chain":false,"published_date":"2020-01-01","fair_score":null,"fair_percentile":null,"algorithm_id":"datarank_citation_only_1hop_v6","ranking_scope":"data_only","authors":[{"id":393373,"name":"Barry E. Storer","orcid":"0000-0003-1565-435X","position":1,"is_corresponding":false},{"id":394292,"name":"Niels Smedegaard Andersen","orcid":null,"position":2,"is_corresponding":false},{"id":394293,"name":"Michael B. Maris","orcid":null,"position":3,"is_corresponding":false},{"id":393374,"name":"Thomas R. Chauncey","orcid":"0000-0003-2815-7301","position":4,"is_corresponding":false},{"id":312327,"name":"Effie W. Petersdorf","orcid":"0000-0002-6454-3683","position":5,"is_corresponding":false},{"id":348678,"name":"Ann E. Woolfrey","orcid":"0000-0001-7222-3607","position":6,"is_corresponding":false},{"id":326533,"name":"Mary E.D. Flowers","orcid":"0000-0003-1631-0911","position":7,"is_corresponding":false},{"id":236734,"name":"Rainer Storb","orcid":"0000-0002-9343-4099","position":8,"is_corresponding":false},{"id":232698,"name":"David G. Maloney","orcid":"0000-0002-8477-6919","position":9,"is_corresponding":false},{"id":236728,"name":"Brenda M. Sandmaier","orcid":"0000-0002-9767-9739","position":10,"is_corresponding":false},{"id":394291,"name":"Brian Kornblit","orcid":null,"position":0,"is_corresponding":true}],"reference_count":35,"raw_metadata":null,"created_at":"2026-07-18T21:46:33.313355Z","pmid":"32603426","pmcid":null,"fwci":null,"citation_percentile":null,"influential_citations":0,"oa_status":null,"license":null,"views":0,"total_file_size_bytes":0,"version_count":0,"fair_f":null,"fair_a":null,"fair_i":null,"fair_r":null,"fair_zscore":null,"fair_rationale":null,"fair_model":null,"fair_agent_version":null,"fair_fulltext_source":null,"fair_has_llm":null,"fair_computed_at":null,"clinical_trials":[],"software_tools":[],"db_accessions":[],"linked_datasets":[],"topics":[]}