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Real-world evidence on Kovaltry (81-8973) in children with moderate or severe hemophilia A in Europe: a nested cohort analysis
- Source :
- Orphanet Journal of Rare Diseases, Vol 16, Iss 1, Pp 1-5 (2021), Orphanet Journal of Rare Diseases
- Publication Year :
- 2021
- Publisher :
- BMC, 2021.
-
Abstract
- Background Untreated hemophilia A patients may experience recurrent bleeding events leading to debilitating joint damages. While RCT and pharmacokinetic data support the value of Kovaltry [an unmodified full-length recombinant factor VIII (FVIII) product], real world evidence in children is lacking. This report describes a descriptive and multivariate analysis of the effectiveness of Kovaltry in children with hemophilia A in the real-world setting, using data from medical chart abstraction and cross-sectional surveys of physicians, patients, and caregivers. Results Male patients aged Conclusion This analysis demonstrates the effectiveness and safety of Kovaltry in a pan-European pediatric population with severe hemophilia A.
- Subjects :
- Male
medicine.medical_specialty
Pediatrics
Letter to the editor
Multivariate analysis
lcsh:Medicine
030204 cardiovascular system & hematology
Real world evidence
Hemophilia A
law.invention
Cohort Studies
03 medical and health sciences
0302 clinical medicine
Randomized controlled trial
law
Internal medicine
hemic and lymphatic diseases
medicine
Humans
Clinical burden
Pharmacology (medical)
Adverse effect
Child
Letter to the Editor
Genetics (clinical)
Hematology
Factor VIII
business.industry
Medical record
Kovaltry
lcsh:R
General Medicine
Factor utilization
Europe
Cross-Sectional Studies
030220 oncology & carcinogenesis
business
Cohort study
Subjects
Details
- Language :
- English
- ISSN :
- 17501172
- Volume :
- 16
- Issue :
- 1
- Database :
- OpenAIRE
- Journal :
- Orphanet Journal of Rare Diseases
- Accession number :
- edsair.doi.dedup.....f07247c1c027e57f1db70e9d6c380798