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Article Abstract
Introduction: Treatment options for patients with hemophilia (PWH) have changed substantially in the last years. This study aimed to compare hemophilia treatment in the eastern part of Germany in 2021 with data from 2015.
Methods: Substitution diaries and patient records of PWH from 2021 were collected in 13 hemophilia centers from the "Kompetenznetz Hämorrhagische Diathese Ost" (KHDO) and compared with 2015.
Results: A total of 130 children and 357 adults, 411 hemophilia A (HA) and 76 hemophilia B (HB), were included in 2021, and 359 were already analyzed in 2015. In 2021, 97.8% of children and 95.7% of adults with severe hemophilia had prophylaxis compared with 98.8 and 80.2% in 2015. Plasma-derived concentrates were used in 25.6%, recombinant in 30.2%, extended half-life (EHL) factor concentrates in 24.4%, and emicizumab in 19.8% of the children with severe HA (sHA). In adults with sHA, plasma-derived, recombinant concentrates, EHL, and emicizumab were used in 21.0, 33.2, 31.2, and 14.2%, respectively. All children and 93.3% of the adults with severe HB (sHB) were on EHL. Median annual factor consumption per body weight increased in adults with sHA, remained stable in children with sHA and adults with sHB, and decreased in children with sHB between 2015 and 2021. Annualized bleeding rate (ABR) decreased in children with sHB and sHA.
Conclusion: The use of EHL and emicizumab has changed hemophilia treatment. About 50% of the sHA patients switched to EHL or emicizumab and almost all sHB patients to EHL. More adults with sHA received prophylaxis and ABR decreased in children.
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| http://dx.doi.org/10.1055/a-2499-2912 | DOI Listing |
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