Sistema de Salut de Catalunya
Institut Català de la Salut
[Calvo-Villas JM] Hematology Department, University Hospital Miguel Servet, Zaragoza, Spain. [Núñez-Vázquez R] Hemophilia Unit, University Hospital Virgen del Rocío, Sevilla, Spain. [Benítez-Hidalgo O] Servei d’Hematologia, Vall d’Hebron Hospital Universitari, Barcelona, Spain. [García-Díaz C] Hematology Department, University Hospital of Burgos, Spain. [Galmés B] Hematology Department, University Hospital Son Espases, Palma de Mallorca, Spain. [Carvalho M] Congenital Coagulopathies Reference Centre, São João University Hospital Centre, Porto, Portugal
Vall d'Hebron Barcelona Hospital Campus
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.
Article
Published version
English
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
Wolters Kluwer Health
Medicine;103(36)
https://doi.org/10.1097/MD.0000000000039255
Attribution 4.0 International
http://creativecommons.org/licenses/by/4.0/
Articles científics - HVH [3439]
