{"doi":"10.1182/blood.2020004823","title":"Ruxolitinib for the treatment of steroid-refractory acute GVHD (REACH1): a multicenter, open-label phase 2 trial","abstract":"Patients who develop steroid-refractory acute graft-versus-host disease (aGVHD) after allogeneic hematopoietic cell transplantation have poor prognosis, highlighting an unmet therapeutic need. In this open-label phase 2 study (ClinicalTrials.gov identifier: NCT02953678), patients aged at least 12 years with grades II to IV steroid-refractory aGVHD were eligible to receive ruxolitinib orally, starting at 5 mg twice daily plus corticosteroids, until treatment failure, unacceptable toxicity, or death. The primary end point was overall response rate (ORR) at day 28; the key secondary end point was duration of response (DOR) at 6 months. As of 2 July 2018, 71 patients received at least 1 dose of ruxolitinib. Forty-eight of those patients (67.6%) had grade III/IV aGVHD at enrollment. At day 28, 39 patients (54.9%; 95% confidence interval, 42.7%-66.8%) had an overall response, including 19 (26.8%) with complete responses. Best ORR at any time was 73.2% (complete response, 56.3%). Responses were observed across skin (61.1%), upper (45.5%) and lower (46.0%) gastrointestinal tract, and liver (26.7%). Median DOR was 345 days. Overall survival estimate at 6 months was 51.0%. At day 28, 24 (55.8%) of 43 patients receiving ruxolitinib and corticosteroids had a 50% or greater corticosteroid dose reduction from baseline. The most common treatment-emergent adverse events were anemia (64.8%), thrombocytopenia (62.0%), hypokalemia (49.3%), neutropenia (47.9%), and peripheral edema (45.1%). Ruxolitinib produced durable responses and encouraging survival compared with historical data in patients with steroid-refractory aGVHD who otherwise have dismal outcomes. The safety profile was consistent with expectations for ruxolitinib and this patient population.","journal":"Blood","year":2020,"id":49017,"datarank":6.956867076871364,"base_score":5.730099782973575,"endowment":5.730099782973575,"self_citation_contribution":0.8595149674460364,"citation_network_contribution":6.097352109425328,"self_endowment_contribution":0.8595149674460364,"citer_contribution":6.097352109425328,"corpus_percentile":null,"corpus_rank":null,"citation_count":307,"citer_count":100,"citers_with_citation_signal":100,"citers_with_endowment":100,"datacite_reuse_total":0,"is_dataset":false,"is_dataset_confidence":0.9483,"is_data_producer":true,"deposit_databanks":{"ClinicalTrials.gov":["NCT02953678"]},"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":108873,"name":"Miguel‐Angel Perales","orcid":"0000-0002-5910-4571","position":1,"is_corresponding":false},{"id":235013,"name":"Mark A. Schroeder","orcid":"0000-0003-4924-8990","position":2,"is_corresponding":false},{"id":235014,"name":"Haris Ali","orcid":"0000-0002-9728-7292","position":3,"is_corresponding":false},{"id":235015,"name":"Nirav N. Shah","orcid":"0000-0002-4336-1071","position":4,"is_corresponding":false},{"id":91627,"name":"Yi‐Bin Chen","orcid":"0000-0002-9554-1058","position":5,"is_corresponding":false},{"id":238578,"name":"Salman Fazal","orcid":null,"position":6,"is_corresponding":false},{"id":238579,"name":"Fitzroy W. Dawkins","orcid":null,"position":7,"is_corresponding":false},{"id":238580,"name":"Michael Arbushites","orcid":null,"position":8,"is_corresponding":false},{"id":235016,"name":"Chuan Tian","orcid":"0000-0001-5048-1824","position":9,"is_corresponding":false},{"id":235017,"name":"Laura Connelly‐Smith","orcid":"0000-0001-9646-6216","position":10,"is_corresponding":false},{"id":87829,"name":"Michael Howell","orcid":"0000-0002-1767-5157","position":11,"is_corresponding":false},{"id":235018,"name":"H. Jean Khoury","orcid":"0000-0002-8434-3988","position":12,"is_corresponding":false},{"id":235012,"name":"Madan Jagasia","orcid":"0000-0001-5757-5604","position":0,"is_corresponding":true}],"reference_count":28,"raw_metadata":null,"created_at":"2026-07-18T20:36:25.163232Z","pmid":"32160294","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":[]}