BAY 81‐8973 demonstrated efficacy, safety and joint status improvement in patients with severe haemophilia A in the LEOPOLD I extension for ≤2 years
| Autor: | Nikki Church, Despina Tseneklidou-Stoeter, Elena Santagostino, Shadan Lalezari, Johnny Mahlangu, Maria Fernanda Lopez Fernandez, Horst Beckmann |
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| Rok vydání: | 2020 |
| Předmět: |
Adult
Male medicine.medical_specialty Adolescent Haemophilia A haemophilia A Hemophilia A recombinant proteins Severity of Illness Index Young Adult 03 medical and health sciences 0302 clinical medicine Internal medicine Hemarthrosis medicine Humans In patient Myocardial infarction Child Adverse effect Aged recombinant factor VIII Factor VIII business.industry clinical trial Original Articles Hematology General Medicine Middle Aged medicine.disease Clinical trial Treatment Outcome Tolerability 030220 oncology & carcinogenesis Quality of Life Original Article intravenous infusions Severe haemophilia A business Bay 030215 immunology |
| Zdroj: | European Journal of Haematology |
| ISSN: | 1600-0609 0902-4441 |
| DOI: | 10.1111/ejh.13402 |
| Popis: | Objectives BAY 81‐8973 (Kovaltry®), 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. |
| Databáze: | OpenAIRE |
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