2026-04-17T16:14:30Zhttps://recercat.cat/oai/request

oai:recercat.cat:11351/125702025-10-24T10:31:11Zcom_2072_378070com_2072_378040col_2072_378092

00925njm 22002777a 4500 dc CALVO VILLAS, JOSE MANUEL author Garcia Diaz, Covadonga author Galmés, Bernat author Carvalho, Manuela author Nuñez, Ramiro author Benítez Hidalgo, Olga author 2025-02-10T07:30:48Z 2025-02-10T07:30:48Z 2024-09-06 Hemophilia A; Factor VIII Hemofília A; Factor VIII Hemofilia A; Factor VIII The real-world outcomes of lonoctocog alfa (rVIII-SingleChain), a long-acting factor VIII (FVIII) with a favorable safety and efficacy profile in trials, were assessed in patients with hemophilia A in Iberian (Spain and Portugal). This was a retrospective study involving patients switching to rVIII-SingleChain from other FVIIIs in 7 Spanish and Portuguese hospitals. The efficacy and safety of replacement therapies were compared between 12 months before switching and the period from switching to the end of the study. Twenty-nine patients (median age 25 years; severe hemophilia A, 37.9%) were recruited. Before switching, 12 were on prophylaxis and were followed-up for a median of 12 months. After switching, 17 received prophylaxis with rVIII-SingleChain and were followed-up for a median of 41 months. Those with ≤2 weekly infusions increased from 37.5% before switching to 60.7% after switching to rVIII-SingleChain. The median monthly consumption was 312 IU/kg with prior FVIIIs and 273 IU/kg with rVII-SingleChain. Six spontaneous bleeds were reported in each period in the prophylaxis patients. In the entire cohort, 50 bleeds were reported with prior FVIIIs and 33 were reported after switching to rVIII-SingleChain. Patients requiring ≤1 dose for hemostasis increased from 44.0% with prior FVIIIs to 60.6% with rVIII-SingleChain. Responses were rated good/excellent in 95.4% of cases. No safety concerns were reported. Patients who switched to rVIII-SingleChain prophylaxis had excellent bleeding control and reduced infusion frequency in regular clinical practice, with the subsequent increase in quality-of-life. http://hdl.handle.net/11351/12570 Avaluació de resultats (Assistència sanitària) Hemofília - Tractament Sang - Coagulació - Factor VIII - Ús terapèutic DISEASES::Hemic and Lymphatic Diseases::Hematologic Diseases::Blood Coagulation Disorders::Blood Coagulation Disorders, Inherited::Hemophilia A Other subheadings::Other subheadings::Other subheadings::/drug therapy CHEMICALS AND DRUGS::Amino Acids, Peptides, and Proteins::Proteins::Blood Proteins::Blood Coagulation Factors::Factor VIII Other subheadings::Other subheadings::/therapeutic use ANALYTICAL, DIAGNOSTIC AND THERAPEUTIC TECHNIQUES, AND EQUIPMENT::Diagnosis::Prognosis::Treatment Outcome ENFERMEDADES::enfermedades hematológicas y linfáticas::enfermedades hematológicas::trastornos de la coagulación sanguínea::trastornos de la coagulación sanguínea hereditarios::hemofilia A Otros calificadores::Otros calificadores::Otros calificadores::/farmacoterapia COMPUESTOS QUÍMICOS Y DROGAS::aminoácidos, péptidos y proteínas::proteínas::proteínas sanguíneas::factores de coagulación de la sangre::factor VIII Otros calificadores::Otros calificadores::/uso terapéutico TÉCNICAS Y EQUIPOS ANALÍTICOS, DIAGNÓSTICOS Y TERAPÉUTICOS::diagnóstico::pronóstico::resultado del tratamiento Switching hemophilia A patients to rVIII-SingleChain: The Iberian experience