Application of a hemophilia mortality framework to the Emicizumab Global Safety Database
| Autor: | Glenn F. Pierce, Peter J. Kuebler, Midori Shima, Rebecca Kruse-Jarres, Johnny Mahlangu, Charles R. M. Hay, Steven W. Pipe, Flora Peyvandi |
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| Jazyk: | angličtina |
| Rok vydání: | 2020 |
| Předmět: |
safety
Male Hepatitis C virus 030204 cardiovascular system & hematology computer.software_genre Malignancy medicine.disease_cause Antibodies Monoclonal Humanized Hemophilia A Sepsis 03 medical and health sciences cause of death 0302 clinical medicine Life Expectancy Antibodies Bispecific medicine Humans benchmarking Cause of death Emicizumab Factor VIII Database business.industry Hematology Original Articles medicine.disease mortality Clinical trial Safety profile Original Article Female Risk of death business computer |
| Zdroj: | Journal of Thrombosis and Haemostasis |
| ISSN: | 1538-7836 1538-7933 |
| Popis: | Background As the first non-factor replacement therapy for persons with congenital hemophilia A (PwcHA), emicizumab's safety profile is of particular interest to the community. Objectives We applied an algorithm for categorization of fatal events contemporaneous to emicizumab using reporter-assessed causality documented in the Roche Emicizumab Global Safety Database. Patients/methods All fatalities in PwcHA reported to the database (from clinical trials, pre-market access, and spontaneous post-marketing reports) were categorized into: associated with hemophilia A-hemorrhagic, thrombotic, human immunodeficiency virus (HIV)/hepatitis C virus (HCV), hepatic (non-HCV); associated with general population-trauma/suicide, non-HA-associated conditions; or, unspecified. Reported cause of death was not reassessed. Results As of cut-off May 15, 2020, 31 fatalities in PwcHA taking emicizumab were reported. Median age at death was 58 years; 51% had factor VIII inhibitors. Fifteen fatalities were considered associated with HA; overall, the most frequent category was hemorrhage (11/31). Of these, six had a history of life-threatening bleeds, and four had a history of intracranial hemorrhage. The remaining HA-associated fatalities were related to HIV/HCV (3/31) and other hepatic causes (1/31). No cases were categorized as thrombotic. Of 10 cases considered not associated with HA, two were categorized as cardiovascular (non-thrombotic), five as infection/sepsis, and one each of trauma/suicide, pulmonary, and malignancy. Six cases were unspecified. Conclusions No unique risk of death was associated with emicizumab prophylaxis in PwcHA. The data reveal that mortality in PwcHA receiving emicizumab was primarily associated with hemorrhage or non-HA-associated conditions, and was not reported by treaters to be related to emicizumab treatment. |
| Databáze: | OpenAIRE |
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