{"doi":"10.1002/ajh.26924","title":"Alemtuzumab in relapsed immune severe aplastic anemia: Long‐term results of a phase II study","abstract":"Immune severe aplastic anemia (SAA) is characterized by pancytopenia and immune-mediated bone marrow destruction. SAA may be treated with hematopoietic stem cell transplantation (HSCT) or immunosuppressive therapy (IST). However, 30% of patients treated with IST relapse. We previously reported a clinical trial of alemtuzumab in which more than half of 25 relapsed SAA patients (56%) responded hematologically. Here, we present long-term results of a total of 42 patients. Participants with SAA who had previously completed antithymocyte globulin (ATG)-based IST, but had relapsed, were enrolled on this study. Alemtuzumab was administered intravenously (IV) (n = 28) or subcutaneously (SC) (n = 14). The primary endpoint was hematologic response at 6 months. Secondary endpoints included relapse, clonal evolution, and survival. This trial was registered at clinicaltrials.gov (NCT00195624). Patients were enrolled over 9 years, with median follow-up of 6 years. Median age was 32 years, with 57% being female. At 6 months, 18 patients (43%) achieved response; 15 (54%) of those who received IV compared with 3 (21%) who received SC therapy. Six patients (14%) had durable long-term response without need for subsequent AA-directed therapy or HSCT at last follow-up. Nine patients had clonal evolution, with high-risk evolution occurring in 6. Overall survival was 67% at median follow-up of 6 years. Prolonged iatrogenic immunosuppression was observed as long as 2 years after alemtuzumab administration. Alemtuzumab induces responses in relapsed SAA, some of which are durable long-term. However, immunosuppression can persist for years, requiring long-term monitoring.","journal":"American Journal of Hematology","year":2023,"id":361985,"datarank":0.48977657647120587,"base_score":2.1972245773362196,"endowment":2.1972245773362196,"self_citation_contribution":0.32958368660043297,"citation_network_contribution":0.1601928898707729,"self_endowment_contribution":0.32958368660043297,"citer_contribution":0.1601928898707729,"corpus_percentile":null,"corpus_rank":null,"citation_count":8,"citer_count":8,"citers_with_citation_signal":5,"citers_with_endowment":5,"datacite_reuse_total":0,"is_dataset":false,"is_dataset_confidence":0.9562,"is_data_producer":true,"deposit_databanks":{"ClinicalTrials.gov":["NCT00195624"]},"is_oa":true,"file_count":0,"downloads":0,"has_version_chain":false,"published_date":"2023-01-01","fair_score":null,"fair_percentile":null,"algorithm_id":"datarank_citation_only_1hop_v6","ranking_scope":"data_only","authors":[{"id":1026154,"name":"Ash Lee Manley","orcid":null,"position":1,"is_corresponding":false},{"id":331116,"name":"Ruba Shalhoub","orcid":"0000-0001-7349-6199","position":2,"is_corresponding":false},{"id":411629,"name":"Jibran Durrani","orcid":"0000-0001-9172-4706","position":3,"is_corresponding":false},{"id":417532,"name":"Olga Rios","orcid":"0000-0001-6790-9313","position":4,"is_corresponding":false},{"id":265130,"name":"Jennifer Lotter","orcid":null,"position":5,"is_corresponding":false},{"id":218228,"name":"Bhavisha A. Patel","orcid":"0000-0002-2974-7701","position":6,"is_corresponding":false},{"id":85436,"name":"Colin O. Wu","orcid":"0000-0002-4514-0926","position":7,"is_corresponding":false},{"id":218269,"name":"Neal S. Young","orcid":"0009-0007-0999-2519","position":8,"is_corresponding":false},{"id":218230,"name":"Emma M. Groarke","orcid":"0000-0002-4648-5926","position":9,"is_corresponding":false},{"id":1112755,"name":"Nidhi Aggarwal","orcid":"0000-0002-2906-9920","position":0,"is_corresponding":true}],"reference_count":28,"raw_metadata":null,"created_at":"2026-07-19T01:14:15.063476Z","pmid":"37021397","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":[]}