Twelve months of emicizumab prophylaxis in a severe hemophilia A man with inhibitor who failed immune tolerance induction: effectiveness, economic, and safety outcomes

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dc.creatorRicardo Mesquita Camelo
dc.creatorTenille Capuxú de Medeiros
dc.creatorDaniela Gonçalves Braz de Albuquerque
dc.creatorJuliana Alvares Teodoro
dc.date.accessioned2023-07-18T20:30:33Z
dc.date.accessioned2025-09-09T01:01:57Z
dc.date.available2023-07-18T20:30:33Z
dc.date.issued2021
dc.identifier.doihttps://doi.org/10.1016/j.htct.2021.02.010
dc.identifier.issn2531-1387
dc.identifier.urihttps://hdl.handle.net/1843/56640
dc.languagepor
dc.publisherUniversidade Federal de Minas Gerais
dc.relation.ispartofHematology, Transfusion and Cell Therapy
dc.rightsAcesso Aberto
dc.subjectCasos, relatorios clinicos, estatisticas
dc.subjectCase Report
dc.subjectHemofilia
dc.subject.otherHemophilia A (HA)
dc.subject.otherImmune tolerance induction
dc.titleTwelve months of emicizumab prophylaxis in a severe hemophilia A man with inhibitor who failed immune tolerance induction: effectiveness, economic, and safety outcomes
dc.typeArtigo de periódico
local.citation.epage101
local.citation.issue1
local.citation.spage98
local.citation.volume45
local.description.resumoHemophilia A (HA) is a rare inherited bleeding disorder characterized by reduced/absent activity of the clotting factor VIII (FVIII).1 Until recently, treatment consisted mainly of FVIII concentrate intravenous infusions exclusively during hemorrhages (on demand) or regularly to avoid bleedings (prophylaxis).1 Unfortunately, about 30% of previously untreated PwHA may develop antibodies which inactivate FVIII, called inhibitors.1 PwHA and inhibitors (PwHAi) have worse outcome, higher frequencies of bleeding episodes, with lower quality of life and enhanced mortality risk.1 Moreover, since FVIII is ineffective for hemostasis in inhibitor individuals, intravenous bypassing agents (activated prothrombin complex concentrate [aPCC] or recombinant activated factor VII [rFVIIa]) used to be recommended for both on demand and prophylaxis treatments, and treatment cost can be considerably increased.1 Therefore, the best treatment for PwHAi is eradicating the antibodies by regular intravenous infusions of FVIII, called immune tolerance induction (ITI).2 The success rate of ITI is about 70%−80% worldwide, after which FVIII replacement as episodic treatment or prophylaxis can be resumed.2 PwHAi who fail treatment had only the option to receive bypassing agents.1,2 In 2017, the first phase III trial with the humanized bispecific antibody emicizumab was published,3 adding renewed options for treatment of PwHAi. Emicizumab binds to factors IX-activated and X, speeding up the activation of factor X.4 It solved some unmet needs of HA treatment, such as regimen (once weekly up to once monthly infusion) and route of administration (subcutaneous). Most importantly, emicizumab reduced bleeding episodes, and improved the quality of life of PwHAi.3 Although it is an effective non-replacement alternative in the prophylaxis of PwHA with or without inhibitors, its safety has not been clarified yet, and a few – although unprecedent – cases of thrombosis have been described.5,6 The “Brazilian registry of persons with hemophilia A receiving emicizumab” (EMCase Study) is a multicenter observational study and any PwHA receiving emicizumab can be included (e.g., sex, age, inhibitor status etc. are not inclusion nor exclusion criteria). It was approved centrally by the Committee on Ethics in Research of the Universidade Federal de Minas Gerais (CAAE 10,664,919.6.0000.5149), locally by each Hemophilia Treatment Center's Committee on Ethics in Research, and registered in the Brazilian Registry of Clinical Trials (RBR-57rnpz). All the included patients signed the Informed Consent Form, according to the Helsink Declaration. The treatment will be decided among the patient, the physician, and the interdisciplinary team of the hemophilia treatment center. Outcome data, laboratory tests and therapeutic progression will be compiled yearly over a maximum of 10 years. Economic analyses and pharmacovigilance will also be evaluated. Herein we described the one-year experience of emicizumab treatment in the first patient included in the EMCase Study.
local.identifier.orcidhttps://orcid.org/0000-0001-9025-0289
local.identifier.orcidhttps://orcid.org/0000-0001-6346-0897
local.identifier.orcidhttps://orcid.org/0000-0002-1911-6420
local.identifier.orcidhttps://orcid.org/0000-0002-0210-0721
local.publisher.countryBrasil
local.publisher.departmentFAR - DEPARTAMENTO DE FARMÁCIA SOCIAL
local.publisher.initialsUFMG
local.url.externahttps://www.sciencedirect.com/science/article/pii/S2531137921000511?via%3Dihub

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