{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,7,21]],"date-time":"2026-07-21T05:03:04Z","timestamp":1784610184779,"version":"3.55.0"},"reference-count":44,"publisher":"MDPI AG","issue":"7","license":[{"start":{"date-parts":[[2026,7,17]],"date-time":"2026-07-17T00:00:00Z","timestamp":1784246400000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["Diseases"],"abstract":"<jats:p>Hemophilia is an X-linked inherited bleeding disorder, classified as type A or type B. Therapeutic advances offer new treatment options that improve disease control and reduce associated complications, including inhibitor development and hemophilic arthropathy. This document aims to update the Mexican hemophilia consensus, reviewing current evidence on diagnosis and management, and addressing gaps in the treatment and follow-up of patients in Mexico, aligning local needs with international recommendations. A PubMed literature search covering the last five years (up to September 2025) was conducted, prioritizing consensus statements, guidelines, and systematic reviews. Using the Delphi methodology, a structured questionnaire was submitted electronically to forty-four experts. Aspects without initial agreement were discussed at an in-person meeting. Consensus was defined as at least 80% of votes in favor. Recommendations were issued across six domains: laboratory diagnosis, genetic testing, management of hemophilia A and B without and with inhibitors, adjuvant treatments, and gene therapy. The recommendations address prophylaxis with coagulation factor concentrates, non-factor therapies, immune tolerance induction, perioperative management, pain management, and eligibility criteria and follow-up protocols for gene therapy with adeno-associated viral vectors. This consensus provides updated, evidence-based recommendations adapted to the Mexican healthcare context, identifying priority areas, including timely access to non-factor therapies and gene therapy, development of a national referral network for complex cases, and inclusion of novel therapeutic agents in the institutional essential medicines list, with the aim of improving the quality of life of people with hemophilia in Mexico.<\/jats:p>","DOI":"10.3390\/diseases14070259","type":"journal-article","created":{"date-parts":[[2026,7,20]],"date-time":"2026-07-20T02:32:08Z","timestamp":1784514728000},"page":"259","update-policy":"https:\/\/doi.org\/10.3390\/mdpi_crossmark_policy","source":"Crossref","is-referenced-by-count":0,"title":["Hemophilia in Mexico: Updated Consensus Recommendations on Diagnosis, Treatment and Gene Therapy"],"prefix":"10.3390","volume":"14","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-5208-5558","authenticated-orcid":false,"given":"Martha","family":"Alvarado 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Thromb. Haemost."},{"key":"ref_2","first-page":"S1","article-title":"Consenso de hemofilia en M\u00e9xico","volume":"157","year":"2021","journal-title":"Gac. Med. Mex."},{"key":"ref_3","first-page":"91","article-title":"Consenso mexicano para el diagn\u00f3stico por laboratorio de las coagulopat\u00edas","volume":"6","year":"2024","journal-title":"Lat. Am. J. Clin. Sci. Med. Technol."},{"key":"ref_4","doi-asserted-by":"crossref","first-page":"1","DOI":"10.1111\/hae.14046","article-title":"WFH Guidelines for the Management of Hemophilia panelists and co-authors. WFH Guidelines for the Management of Hemophilia, 3rd edition","volume":"26","author":"Srivastava","year":"2020","journal-title":"Hemophilia"},{"key":"ref_5","first-page":"3","article-title":"Consenso latinoamericano de hemofilia","volume":"2","author":"Berro","year":"2025","journal-title":"Hemost. Trombos."},{"key":"ref_6","doi-asserted-by":"crossref","first-page":"413","DOI":"10.1111\/ijlh.14109","article-title":"International Council for Standardization in Haematology recommendations for laboratory measurement of factor VIII and FIX type I inhibitors","volume":"45","author":"Meijer","year":"2023","journal-title":"Int. J. Lab. Hematol."},{"key":"ref_7","doi-asserted-by":"crossref","unstructured":"Carcao, M., and Goudemand, J. (2018). Inhibitors in Hemophilia: A Primer, World Federation of Hemophilia. [5th ed.].","DOI":"10.1016\/B978-0-323-35762-3.00135-9"},{"key":"ref_8","doi-asserted-by":"crossref","first-page":"56","DOI":"10.15406\/htij.2024.12.00333","article-title":"Hemophilia: Review of the past and present","volume":"12","year":"2024","journal-title":"Hematol. Transfus. Int."},{"key":"ref_9","doi-asserted-by":"crossref","first-page":"390","DOI":"10.1055\/a-1945-9429","article-title":"Insights into the Molecular Genetic of Hemophilia A and Hemophilia B: The Relevance of Genetic Testing in Routine Clinical Practice","volume":"42","author":"Pezeshkpoor","year":"2022","journal-title":"Hamostaseologie"},{"key":"ref_10","doi-asserted-by":"crossref","first-page":"411","DOI":"10.1111\/jth.13597","article-title":"Safety and efficacy of BAY 94-9027, a prolonged-half-life factor VIII","volume":"15","author":"Reding","year":"2017","journal-title":"J. Thromb. Haemost."},{"key":"ref_11","doi-asserted-by":"crossref","first-page":"1761","DOI":"10.1182\/blood-2015-09-669234","article-title":"PROLONG-9FP Investigators Study Group. Long-acting recombinant coagulation factor IX albumin fusion protein (rIX-FP) in hemophilia B: Results of a phase 3 trial","volume":"127","author":"Santagostino","year":"2016","journal-title":"Blood"},{"key":"ref_12","doi-asserted-by":"crossref","first-page":"809","DOI":"10.1056\/NEJMoa1703068","article-title":"Emicizumab Prophylaxis in Hemophilia A with Inhibitors","volume":"377","author":"Oldenburg","year":"2017","journal-title":"N. Engl. J. Med."},{"key":"ref_13","doi-asserted-by":"crossref","first-page":"783","DOI":"10.1056\/NEJMoa2216455","article-title":"Phase 3 Trial of Concizumab in Hemophilia with Inhibitors","volume":"389","author":"Matsushita","year":"2023","journal-title":"N. Engl. J. Med."},{"key":"ref_14","doi-asserted-by":"crossref","first-page":"702","DOI":"10.1111\/hae.14595","article-title":"Befovacimab, an anti-tissue factor pathway inhibitor antibody: Early termination of the multiple-dose, dose-escalating Phase 2 study due to thrombosis","volume":"28","author":"Mancuso","year":"2022","journal-title":"Haemophilia"},{"key":"ref_15","doi-asserted-by":"crossref","first-page":"1147","DOI":"10.1182\/bloodadvances.2024013900","article-title":"Long-term safety and efficacy of fitusiran prophylaxis, and perioperative management, in people with hemophilia A or B","volume":"9","author":"Pipe","year":"2025","journal-title":"Blood Adv."},{"key":"ref_16","doi-asserted-by":"crossref","first-page":"1654","DOI":"10.1182\/blood.2024027468","article-title":"Marstacimab Prophylaxis in Hemophilia A\/B Without Inhibitors: Results from the Phase 3 BASIS Trial","volume":"146","author":"Matino","year":"2025","journal-title":"Blood"},{"key":"ref_17","doi-asserted-by":"crossref","first-page":"1483","DOI":"10.1016\/j.jtha.2024.11.015","article-title":"Deconstructing the ISTH hemophilia guidelines for the clinician","volume":"23","author":"Young","year":"2025","journal-title":"J. Thromb. Haemost."},{"key":"ref_18","doi-asserted-by":"crossref","first-page":"723","DOI":"10.1055\/s-0033-1354422","article-title":"Arthropathy in patients with moderate hemophilia A: A systematic review of the literature","volume":"39","author":"Ambrosino","year":"2013","journal-title":"Semin. Thromb. Hemost."},{"key":"ref_19","doi-asserted-by":"crossref","unstructured":"Castaman, G., Peyvandi, F., De Cristofaro, R., Pollio, B., and Di Minno, D.M.N. (2023). Mild and Moderate Hemophilia A: Neglected Conditions, Still with Unmet Needs. J. Clin. Med., 12.","DOI":"10.3390\/jcm12041368"},{"key":"ref_20","doi-asserted-by":"crossref","first-page":"1299","DOI":"10.1111\/hae.14851","article-title":"Psychometrics and applications of a novel self-report measure of emicizumab adherence: VERITASNexGen","volume":"29","author":"Okolo","year":"2023","journal-title":"Haemophilia"},{"key":"ref_21","doi-asserted-by":"crossref","first-page":"649","DOI":"10.1080\/17474086.2025.2522296","article-title":"Safety and efficacy of marstacimab in patients with hemophilia A and B: A systematic review and meta-analysis","volume":"18","author":"Mokresh","year":"2025","journal-title":"Expert Rev. Hematol."},{"key":"ref_22","doi-asserted-by":"crossref","first-page":"1355","DOI":"10.1182\/blood.2023021832","article-title":"Emicizumab prophylaxis in infants with hemophilia A (HAVEN 7): Primary analysis of a phase 3b, open-label trial","volume":"143","author":"Pipe","year":"2024","journal-title":"Blood"},{"key":"ref_23","doi-asserted-by":"crossref","first-page":"109306","DOI":"10.1016\/j.thromres.2025.109306","article-title":"FVIII half-life products: A real-world experience","volume":"249","author":"Zanon","year":"2025","journal-title":"Thromb. Res."},{"key":"ref_24","doi-asserted-by":"crossref","first-page":"310","DOI":"10.1056\/NEJMoa2209226","article-title":"Efanesoctocog Alfa Prophylaxis for Patients with Severe Hemophilia A","volume":"388","author":"Chowdary","year":"2023","journal-title":"N. Engl. J. Med."},{"key":"ref_25","doi-asserted-by":"crossref","first-page":"379","DOI":"10.2147\/BTT.S500480","article-title":"Marstacimab for the Treatment of Hemophilia A or B","volume":"19","author":"Mahlangu","year":"2025","journal-title":"Biol. Targets Ther."},{"key":"ref_26","doi-asserted-by":"crossref","first-page":"e891","DOI":"10.1016\/S2352-3026(24)00307-7","article-title":"Concizumab prophylaxis in people with hemophilia A or hemophilia B without inhibitors (explorer8): A prospective, multicentre, open-label, randomised, phase 3a trial","volume":"11","author":"Chowdary","year":"2024","journal-title":"Lancet Haematol."},{"key":"ref_27","doi-asserted-by":"crossref","first-page":"1427","DOI":"10.1016\/S0140-6736(23)00284-2","article-title":"Efficacy and safety of fitusiran prophylaxis in people with hemophilia A or hemophilia B with inhibitors (ATLAS-INH): A multicentre, open-label, randomised phase 3 trial","volume":"401","author":"Young","year":"2023","journal-title":"Lancet"},{"key":"ref_28","doi-asserted-by":"crossref","first-page":"e322","DOI":"10.1016\/S2352-3026(23)00037-6","article-title":"Fitusiran prophylaxis in people with severe hemophilia A or hemophilia B without inhibitors (ATLAS-A\/B): A multicentre, open-label, randomised, phase 3 trial","volume":"10","author":"Srivastava","year":"2023","journal-title":"Lancet Haematol."},{"key":"ref_29","doi-asserted-by":"crossref","first-page":"221","DOI":"10.1111\/hae.14222","article-title":"Development and testing of the Satisfaction Questionnaire with Intravenous or Subcutaneous Hemophilia Injection and results from the Phase 3 HAVEN 3 study of emicizumab prophylaxis in persons with hemophilia A without FVIII inhibitors","volume":"27","author":"Kempton","year":"2021","journal-title":"Haemophilia"},{"key":"ref_30","doi-asserted-by":"crossref","first-page":"100020","DOI":"10.1016\/j.rpth.2022.100020","article-title":"Determinants of successful immune tolerance induction in hemophilia A: Systematic review and meta-analysis","volume":"7","author":"Oomen","year":"2022","journal-title":"Res. Pract. Thromb. Haemost."},{"key":"ref_31","doi-asserted-by":"crossref","first-page":"789","DOI":"10.1111\/hae.13819","article-title":"Immune tolerance induction in pediatric patients with hemophilia A and inhibitors receiving emicizumab prophylaxis","volume":"25","author":"Batsuli","year":"2019","journal-title":"Hemophilia"},{"key":"ref_32","doi-asserted-by":"crossref","first-page":"1787","DOI":"10.1111\/trf.13016","article-title":"EQOFIX: A combined economic and quality-of-life study of hemophilia B treatments in France","volume":"55","author":"Polack","year":"2015","journal-title":"Transfusion"},{"key":"ref_33","doi-asserted-by":"crossref","first-page":"100885","DOI":"10.1016\/j.blre.2021.100885","article-title":"Improving assessment and management of pain in hemophilia: An Italian Delphi consensus statement","volume":"51","author":"Santoro","year":"2022","journal-title":"Blood Rev."},{"key":"ref_34","doi-asserted-by":"crossref","first-page":"1013","DOI":"10.1056\/NEJMoa2113708","article-title":"Valoctocogene Roxaparvovec Gene Therapy for Hemophilia A","volume":"386","author":"Ozelo","year":"2022","journal-title":"N. Engl. J. Med."},{"key":"ref_35","doi-asserted-by":"crossref","first-page":"706","DOI":"10.1056\/NEJMoa2211644","article-title":"Gene Therapy with Etranacogene Dezaparvovec for Hemophilia B","volume":"388","author":"Pipe","year":"2023","journal-title":"N. Engl. J. Med."},{"key":"ref_36","doi-asserted-by":"crossref","first-page":"88","DOI":"10.1007\/s12038-020-00057-y","article-title":"Advances in gene therapy for hemophilia","volume":"45","year":"2020","journal-title":"J. Biosci."},{"key":"ref_37","doi-asserted-by":"crossref","first-page":"95","DOI":"10.2147\/JBM.S490588","article-title":"Emerging Therapies in Hemophilia: Improving Equitable Access to Care","volume":"16","author":"Lewandowska","year":"2025","journal-title":"J. Blood Med."},{"key":"ref_38","doi-asserted-by":"crossref","first-page":"1108","DOI":"10.1056\/NEJMoa2302982","article-title":"Gene Therapy with Fidanacogene Elaparvovec in Adults with Hemophilia B","volume":"391","author":"Cuker","year":"2024","journal-title":"N. Engl. J. Med."},{"key":"ref_39","doi-asserted-by":"crossref","first-page":"1510","DOI":"10.1016\/j.jtha.2023.12.039","article-title":"Recommendations for a minimum data set for monitoring gene therapy in hemophilia: Communication from the ISTH SSC Working Group on Gene Therapy","volume":"22","author":"Miesbach","year":"2024","journal-title":"J. Thromb. Haemost."},{"key":"ref_40","unstructured":"Federaci\u00f3n de Hemofilia de la Rep\u00fablica Mexicana (FHRM) (2026, June 15). Viviendo Juntos con Hemofilia y Otras Coagulopat\u00edas. Available online: https:\/\/hemofiliamx.org\/."},{"key":"ref_41","unstructured":"Bobierno de Mexico (2026, June 15). Programa de Acci\u00f3n Espec\u00edfico de Enfermedades Raras y Hemofilia, Secretar\u00eda de Salud, M\u00e9xico. Available online: https:\/\/www.gob.mx\/salud\/documentos\/programa-de-accion-especificao."},{"key":"ref_42","doi-asserted-by":"crossref","first-page":"591","DOI":"10.1111\/hae.14012","article-title":"The World Federation of Hemophilia Annual Global Survey 1999\u20132018","volume":"26","author":"Stonebraker","year":"2020","journal-title":"Haemophilia"},{"key":"ref_43","doi-asserted-by":"crossref","first-page":"20","DOI":"10.1111\/j.1365-2516.2009.02127.x","article-title":"A study of variations in the reported haemophilia A prevalence around the world","volume":"16","author":"Stonebraker","year":"2010","journal-title":"Haemophilia"},{"key":"ref_44","doi-asserted-by":"crossref","first-page":"1287","DOI":"10.1002\/jha2.503","article-title":"Prophylaxis and hemophilia care in LATAM: Baring it all-Highlights from the CLAHT 2021 symposium","volume":"3","author":"DiMinno","year":"2022","journal-title":"EJHaem"}],"container-title":["Diseases"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/www.mdpi.com\/2079-9721\/14\/7\/259\/pdf","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2026,7,21]],"date-time":"2026-07-21T04:15:29Z","timestamp":1784607329000},"score":1,"resource":{"primary":{"URL":"https:\/\/www.mdpi.com\/2079-9721\/14\/7\/259"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2026,7,17]]},"references-count":44,"journal-issue":{"issue":"7","published-online":{"date-parts":[[2026,7]]}},"alternative-id":["diseases14070259"],"URL":"https:\/\/doi.org\/10.3390\/diseases14070259","relation":{},"ISSN":["2079-9721"],"issn-type":[{"value":"2079-9721","type":"electronic"}],"subject":[],"published":{"date-parts":[[2026,7,17]]}}}