{"doi":"10.1111/bjh.16385","title":"A pilot trial of complement inhibition using eculizumab to overcome platelet transfusion refractoriness in human leukocyte antigen allo‐immunized patients","abstract":"Heavily transfused patients frequently develop human leukocyte antigen (HLA) allo-immunization resulting in platelet transfusion refractoriness and a high risk for life-threatening thrombocytopenia. Data suggest complement activation leading to the destruction of platelets bound by HLA allo-antibodies may play a pathophysiologic role in platelet refractoriness. Here we conducted a pilot trial to investigate the use of eculizumab, a monoclonal antibody that binds and inhibits C5 complement, to treat platelet transfusion refractoriness in allo-immunized patients with severe thrombocytopenia. A single eculizumab infusion was administered to 10 eligible patients, with four (40%) patients overcoming platelet refractories assessed measuring the corrected platelet count increment (CCI) 10-60 min and 18-24 h post transfusion. Responding patients had a reduction in the requirement for subsequent platelet transfusions and had higher post-transfusion platelet increments for 14 days following eculizumab administration. Remarkably, three of the four responders met CCI criteria for response despite receiving HLA-incompatible platelets. Our results suggest that eculizumab has the ability to overcome platelet transfusion refractoriness in patients with broad HLA allo-immunization. This study establishes proof of principle that complement inhibition can treat platelet transfusion refractoriness, laying the foundation for a large multicentre trial to assess the overall efficacy of this approach (ClinicalTrials.gov, identifier: NCT02298933).","journal":"British Journal of Haematology","year":2020,"id":98997,"datarank":1.2567789791114015,"base_score":3.4339872044851463,"endowment":3.4339872044851463,"self_citation_contribution":0.515098080672772,"citation_network_contribution":0.7416808984386296,"self_endowment_contribution":0.515098080672772,"citer_contribution":0.7416808984386296,"corpus_percentile":null,"corpus_rank":null,"citation_count":30,"citer_count":30,"citers_with_citation_signal":21,"citers_with_endowment":21,"datacite_reuse_total":0,"is_dataset":false,"is_dataset_confidence":0.9565,"is_data_producer":true,"deposit_databanks":{"ClinicalTrials.gov":["NCT02298933"]},"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":489303,"name":"Enkhtsetseg Purev","orcid":null,"position":1,"is_corresponding":false},{"id":320681,"name":"Kamille A. West","orcid":"0000-0001-8152-804X","position":2,"is_corresponding":false},{"id":489304,"name":"Emily McDuffee","orcid":null,"position":3,"is_corresponding":false},{"id":489305,"name":"Tatyana Worthy","orcid":null,"position":4,"is_corresponding":false},{"id":489306,"name":"Lisa Cook","orcid":null,"position":5,"is_corresponding":false},{"id":489307,"name":"Geri Hawks","orcid":null,"position":6,"is_corresponding":false},{"id":184265,"name":"BRIAN WELLS","orcid":null,"position":7,"is_corresponding":false},{"id":488008,"name":"Reem Shalabi","orcid":"0000-0002-4495-5445","position":8,"is_corresponding":false},{"id":347479,"name":"Willy A. Flegel","orcid":"0000-0002-1631-7198","position":9,"is_corresponding":false},{"id":370507,"name":"Sharon Adams","orcid":null,"position":10,"is_corresponding":false},{"id":343640,"name":"Robert Reger","orcid":null,"position":11,"is_corresponding":false},{"id":488009,"name":"Georg Aue","orcid":"0000-0003-2497-737X","position":12,"is_corresponding":false},{"id":262681,"name":"Xin Tian","orcid":"0000-0003-1896-2462","position":13,"is_corresponding":false},{"id":276347,"name":"Richard Childs","orcid":null,"position":14,"is_corresponding":false},{"id":488007,"name":"Phuong Vo","orcid":"0000-0002-0823-0244","position":0,"is_corresponding":true}],"reference_count":25,"raw_metadata":null,"created_at":"2026-07-18T22:37:35.547042Z","pmid":"32086819","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":[]}