A multicentre real-world data study to assess the bleeding rate and management of patients with haemophilia A and factor VIII inhibitors in Argentina
| Autor: | Isabel Ramos Mejía, Patricia Do Nascimento, Ludmila Elelhou, Victoria Saenz, Maria E. Arrieta, Mauro Davoli, Andrea Cocca, Daniela Neme |
|---|---|
| Rok vydání: | 2021 |
| Předmět: |
Adult
medicine.medical_specialty Factor VIII business.industry Haemophilia A Argentina Hemorrhage Hematology General Medicine Hemophilia A Rate ratio medicine.disease Haemophilia Confidence interval Clinical trial Internal medicine Ambulatory Immune Tolerance Humans Medicine Child business Real world data Retrospective Studies Cohort study |
| Zdroj: | Blood Coagulation & Fibrinolysis. 32:564-571 |
| ISSN: | 1473-5733 0957-5235 |
| Popis: | The development of inhibitors against factor VIII (FVIII) concentrates is a severe complication of treatment for patients with haemophilia. We investigated annualized bleeding rates (ABRs) in patients in Argentina with haemophilia A with inhibitors and analysed potential differences between treatment strategies. This multicentre, retrospective, real-world data, cohort design study comprised ambulatory paediatric and adult patients with congenital haemophilia A and FVIII inhibitors treated according to standard clinical practice, with 12-months follow-up. Of 69 included patients, 39 (56.5%) received on-demand treatment, 13 (18.8%) received prophylactic treatment, and 17 (24.6%) received immune tolerance induction (ITI) therapy. The mean overall ABR was 7.68 ± 8.18, with similar rates for on-demand (8.59 ± 9.69), prophylaxis (5.54 ± 4.71), and ITI (7.24 ± 6.23) subgroups. In the negative binomial regression model, prophylactic treatment [incidence rate ratio (IRR) 0.41, 95% confidence interval (CI): 0.21-0.79, P |
| Databáze: | OpenAIRE |
| Externí odkaz: |
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