{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,6,15]],"date-time":"2026-06-15T09:55:15Z","timestamp":1781517315256,"version":"3.54.1"},"reference-count":37,"publisher":"SAGE Publications","issue":"7","license":[{"start":{"date-parts":[[2025,12,26]],"date-time":"2025-12-26T00:00:00Z","timestamp":1766707200000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/journals.sagepub.com\/page\/policies\/text-and-data-mining-license"}],"content-domain":{"domain":["journals.sagepub.com"],"crossmark-restriction":true},"short-container-title":["Ann Pharmacother"],"published-print":{"date-parts":[[2026,7]]},"abstract":"<jats:sec>\n                    <jats:title>Objective:<\/jats:title>\n                    <jats:p>The objective of the study is to critically review the literature about clinical evidence on the efficacy and safety of fitusiran among patients with hemophilia A or B.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Data sources:<\/jats:title>\n                    <jats:p>A targeted search of PubMed, Embase, the Cochrane Library, and ClinicalTrials.gov was performed from inception through November 2025 using the terms fitusiran, antithrombin, RNA interference, siRNA, and hemophilia A or B.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Study selection and data extraction:<\/jats:title>\n                    <jats:p>Eligible studies were randomized controlled trials across all phases enrolling patients with hemophilia A or B. The review incorporated 2 phase 1 studies, 1 phase 2 study, and 4 phase 3 trials.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Data synthesis:<\/jats:title>\n                    <jats:p>Fitusiran prophylaxis reduced annualized bleeding rates by about 70% to 90% compared with on-demand therapy or previous prophylaxis using bypassing agents, and by up to 50% compared with prior prophylaxis with clotting factor concentrates. Between 50% and 65% of participants experienced no bleeds requiring treatment. Health-related quality of life improved, with mean reductions in Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) scores ranging from \u22124.6 to \u221212.3. Safety evaluations showed aminotransferase elevations in 19% to 25%, gallbladder events in 13% to 15%, and thrombotic events in \u22645% of patients, primarily associated with low antithrombin levels or excessive factor use. Long-term extension studies confirmed sustained bleed protection and safety with antithrombin-guided dosing.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Relevance to patient care and clinical practice in comparison to existing drugs:<\/jats:title>\n                    <jats:p>Fitusiran is a subcutaneous nonfactor prophylactic treatment that lowers antithrombin to rebalance coagulation with or without inhibitors in patients with hemophilia A and B. It is administered every 2 months, and associated with fewer bleeds, and improved quality of life compared with standard of care.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Conclusions:<\/jats:title>\n                    <jats:p>Fitusiran expands prophylactic options for hemophilia A and B, providing sustained bleed protection with infrequent dosing. Integration into practice requires monitoring of antithrombin activity to mitigate thrombotic or bleeding risk, and liver and gallbladder functions for safety.<\/jats:p>\n                  <\/jats:sec>","DOI":"10.1177\/10600280251403512","type":"journal-article","created":{"date-parts":[[2025,12,26]],"date-time":"2025-12-26T08:43:36Z","timestamp":1766738616000},"page":"700-709","update-policy":"https:\/\/doi.org\/10.1177\/sage-journals-update-policy","source":"Crossref","is-referenced-by-count":0,"title":["Fitusiran: A Novel Antithrombin-Targeting Therapy for Hemophilia A and B, With or Without Inhibitors"],"prefix":"10.1177","volume":"60","author":[{"ORCID":"https:\/\/orcid.org\/0009-0004-7635-8567","authenticated-orcid":false,"given":"Mohammad T.","family":"Alashqar","sequence":"first","affiliation":[{"name":"Institute of Therapeutic Innovations and Outcomes, College of Pharmacy, The Ohio State University, Columbus, OH, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-1936-0666","authenticated-orcid":false,"given":"Milap C.","family":"Nahata","sequence":"additional","affiliation":[{"name":"Institute of Therapeutic Innovations and Outcomes, College of Pharmacy, The Ohio State University, Columbus, OH, USA"},{"name":"College of Medicine, The Ohio State University, Columbus, OH, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"179","published-online":{"date-parts":[[2025,12,26]]},"reference":[{"key":"e_1_3_2_2_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.blre.2019.100618"},{"key":"e_1_3_2_3_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.hoc.2019.01.007"},{"key":"e_1_3_2_4_2","doi-asserted-by":"publisher","DOI":"10.1080\/17474086.2020.1739518"},{"key":"e_1_3_2_5_2","doi-asserted-by":"publisher","DOI":"10.2450\/2018.0272-17"},{"key":"e_1_3_2_6_2","doi-asserted-by":"publisher","DOI":"10.1080\/14712598.2019.1614163"},{"key":"e_1_3_2_7_2","doi-asserted-by":"publisher","DOI":"10.1111\/bjh.18379"},{"key":"e_1_3_2_8_2","doi-asserted-by":"publisher","DOI":"10.1111\/hae.14167"},{"key":"e_1_3_2_9_2","doi-asserted-by":"publisher","DOI":"10.1111\/hae.14976"},{"key":"e_1_3_2_10_2","doi-asserted-by":"publisher","DOI":"10.1182\/blood-2018-07-820712"},{"key":"e_1_3_2_11_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.rpth.2022.100018"},{"key":"e_1_3_2_12_2","doi-asserted-by":"publisher","DOI":"10.5582\/ddt.2025.01031"},{"key":"e_1_3_2_13_2","doi-asserted-by":"publisher","DOI":"10.2147\/JBM.S159297"},{"key":"e_1_3_2_14_2","doi-asserted-by":"publisher","DOI":"10.2147\/JBM.S390521"},{"key":"e_1_3_2_15_2","doi-asserted-by":"publisher","DOI":"10.1007\/s40265-025-02203-y"},{"key":"e_1_3_2_16_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.rpth.2023.100179"},{"key":"e_1_3_2_17_2","unstructured":"Sanofi. QFITLIA (Fitusiran) Prescribing Information. Genzyme Corporation; 2025. Accessed September 22 2025. https:\/\/products.sanofi.us\/qfitlia\/qfitlia.pdf."},{"key":"e_1_3_2_18_2","doi-asserted-by":"publisher","DOI":"10.1016\/S2352-3026(24)00307-7"},{"key":"e_1_3_2_19_2","doi-asserted-by":"publisher","DOI":"10.1016\/S2352-3026(23)00037-6"},{"key":"e_1_3_2_20_2","doi-asserted-by":"publisher","DOI":"10.1016\/S0140-6736(23)00284-2"},{"key":"e_1_3_2_21_2","doi-asserted-by":"publisher","DOI":"10.1182\/blood.2023021864"},{"key":"e_1_3_2_22_2","doi-asserted-by":"publisher","DOI":"10.1182\/blood.2024027008"},{"key":"e_1_3_2_23_2","unstructured":"ClinicalTrials.gov. Fitusiran prophylaxis in male pediatric subjects aged 1 to less than 12 years with hemophilia A or B (ATLAS-PEDS). Accessed September 15 2025. https:\/\/clinicaltrials.gov\/ct2\/show\/NCT03974113"},{"key":"e_1_3_2_24_2","unstructured":"ClinicalTrials.gov. A study to test a medicine (fitusiran) injected under the skin for preventing bleeding episodes in male adolescent or adult participants with severe hemophilia (ATLAS-NEO). Accessed September 15 2025. https:\/\/clinicaltrials.gov\/ct2\/show\/NCT05662319"},{"key":"e_1_3_2_25_2","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMoa1616569"},{"key":"e_1_3_2_26_2","doi-asserted-by":"publisher","DOI":"10.1111\/jth.15270"},{"key":"e_1_3_2_27_2","doi-asserted-by":"publisher","DOI":"10.1182\/bloodadvances.2024013900"},{"key":"e_1_3_2_28_2","unstructured":"FDA. FDA Approves Novel Treatment for Hemophilia A or B With or Without Factor VIII or IX Inhibitors. US Food and Drug Administration; 2025. 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