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BAY 81-8973 demonstrated efficacy, safety and joint status improvement in patients with severe haemophilia A in the LEOPOLD I extension for ?2 years
dc.contributor.author | Mahlangu, Johnny | |
dc.contributor.author | López Fernández, María Fernanda | |
dc.contributor.author | Santagostino, Elena | |
dc.contributor.author | Lalezari, Shadan | |
dc.contributor.author | Tseneklidou-Stoeter, Despina | |
dc.contributor.author | Beckmann, Horst | |
dc.contributor.author | Church, Nikki | |
dc.date.accessioned | 2022-03-08T08:50:00Z | |
dc.date.available | 2022-03-08T08:50:00Z | |
dc.date.issued | 2020 | |
dc.identifier.issn | 0902-4441 | |
dc.identifier.other | https://www.ncbi.nlm.nih.gov/pubmed/32112434 | es |
dc.identifier.uri | http://hdl.handle.net/20.500.11940/16205 | |
dc.description.abstract | OBJECTIVES: BAY 81-8973 (Kovaltry((R)) ), a full-length, unmodified, recombinant human factor VIII, provided excellent bleeding control for patients with haemophilia A in the pivotal 1-year LEOPOLD I trial. The LEOPOLD I extension evaluated long-term efficacy and safety of BAY 81-8973 prophylaxis. METHODS: After completing LEOPOLD I, patients continued receiving 20-50 IU/kg BAY 81-8973 two- or three-times weekly in the extension. Outcomes included annualised bleeding rate (ABR) and haemostasis during surgery. RESULTS: Fifty-five patients aged 12-65 years participated in the extension. Median (range) exposure days during the 2-year total study period was 309 (115-355). No patient switched regimens. Median (Q1; Q3) ABR for all bleeds was 2.0 (1.0; 6.1) during the pivotal study, 2.0 (0.0; 5.2) during the extension, and 2.0 (0.5; 5.5) combined. The proportion of joint bleeds affecting target joints decreased (pivotal study: 90.9%, extension: 60.0%). Haemostasis was assessed as excellent/good in all five major surgeries. One serious adverse event (myocardial infarction) occurred in a patient with cardiovascular risk factors. No patients developed inhibitors. CONCLUSIONS: BAY 81-8973 prophylaxis efficacy outcomes in the pivotal study were maintained or, in the case of joint protection, improved during the extension, with a safety and tolerability profile consistent with previous experience. | en |
dc.rights | Atribución-NoComercial 4.0 Internacional | |
dc.rights.uri | http://creativecommons.org/licenses/by-nc/4.0/ | |
dc.subject.mesh | Adult | * |
dc.subject.mesh | Hemophilia A | * |
dc.subject.mesh | Middle Aged | * |
dc.subject.mesh | Adolescent | * |
dc.subject.mesh | Severity of Illness Index | * |
dc.subject.mesh | Hemarthrosis | * |
dc.subject.mesh | Quality of Life | * |
dc.subject.mesh | Clinical Trial | * |
dc.subject.mesh | Humans | * |
dc.subject.mesh | Treatment Outcome | * |
dc.subject.mesh | Factor VIII | * |
dc.subject.mesh | Young Adult | * |
dc.subject.mesh | Recombinant Proteins | * |
dc.subject.mesh | Aged | * |
dc.title | BAY 81-8973 demonstrated efficacy, safety and joint status improvement in patients with severe haemophilia A in the LEOPOLD I extension for ?2 years | en |
dc.type | Journal Article | es |
dc.authorsophos | Mahlangu, Johnny;Lopez Fernandez, Maria Fernanda;Santagostino, Elena;Lalezari, Shadan;Tseneklidou-Stoeter, Despina;Beckmann, Horst;Church, Nikki | |
dc.identifier.doi | 10.1111/ejh.13402 | |
dc.identifier.pmid | 32112434 | |
dc.identifier.sophos | 35852 | |
dc.issue.number | 6 | es |
dc.journal.title | EUROPEAN JOURNAL OF HAEMATOLOGY | es |
dc.organization | Servizo Galego de Saúde::Estrutura de Xestión Integrada (EOXI)::EOXI de A Coruña - Complexo Hospitalario Universitario de A Coruña::Hematoloxía clínica | es |
dc.organization | Servizo Galego de Saúde::Estrutura de Xestión Integrada (EOXI)::Instituto de Investigación Biomédica da Coruña (INIBIC) | es |
dc.relation.publisherversion | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7383510/pdf/EJH-104-594.pdf | es |
dc.rights.accessRights | openAccess | |
dc.subject.decs | proteínas recombinantes | * |
dc.subject.decs | resultado del tratamiento | * |
dc.subject.decs | hemartrosis | * |
dc.subject.decs | factor VIII | * |
dc.subject.decs | mediana edad | * |
dc.subject.decs | adulto | * |
dc.subject.decs | índice de gravedad de la enfermedad | * |
dc.subject.decs | calidad de vida | * |
dc.subject.decs | hemofilia A | * |
dc.subject.decs | anciano | * |
dc.subject.decs | adulto joven | * |
dc.subject.decs | humanos | * |
dc.subject.decs | ensayo clínico | * |
dc.subject.decs | adolescente | * |
dc.subject.keyword | CHUAC | es |
dc.subject.keyword | INIBIC | es |
dc.typefides | Artículo Original | es |
dc.typesophos | Artículo Original | es |
dc.volume.number | 104 | es |
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