Real-world evidence on Kovaltry (81-8973) in children with moderate or severe hemophilia A in Europe: a nested cohort analysis
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...
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| Format: | Article |
| Language: | English |
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BMC
2021-01-01
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| Series: | Orphanet Journal of Rare Diseases |
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| Online Access: | https://doi.org/10.1186/s13023-021-01676-w |
| _version_ | 1819052181100167168 |
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| author | Jamie O’Hara Ceri Hirst Jose Francisco Cabre Marquez Tom Burke |
| author_facet | Jamie O’Hara Ceri Hirst Jose Francisco Cabre Marquez Tom Burke |
| author_sort | Jamie O’Hara |
| collection | DOAJ |
| description | 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 < 18 years with moderate or severe hemophilia A, residing in five European countries and treated with FVIII were studied. The co-primary endpoints were the annualized bleeding rate (ABR) and the annual FVIII utilization rate. Twenty nine patients treated with Kovaltry were included, of whom 93% had severe disease and 75% were on continuous prophylactic treatment. The mean ABR was 2.66 ± 2.06, with rates decreasing with age. The children received on average 2.45 infusions per week, consistent across age groups (median 3; range 1–3). There were no reports of inhibitor development or adverse events in the study (AEs), and all patients were satisfied or very satisfied with the treatment. An exploratory multivariate analysis suggests no significant difference in ABR or units utilized between Kovaltry and some extended half life products in children with severe hemophilia A, though characteristics of these patient cohorts were markedly different. Conclusion This analysis demonstrates the effectiveness and safety of Kovaltry in a pan-European pediatric population with severe hemophilia A. |
| first_indexed | 2024-12-21T12:15:45Z |
| format | Article |
| id | doaj.art-c7b4c3fd50ce4146ac065c52c6d5ff6c |
| institution | Directory Open Access Journal |
| issn | 1750-1172 |
| language | English |
| last_indexed | 2024-12-21T12:15:45Z |
| publishDate | 2021-01-01 |
| publisher | BMC |
| record_format | Article |
| series | Orphanet Journal of Rare Diseases |
| spelling | doaj.art-c7b4c3fd50ce4146ac065c52c6d5ff6c2022-12-21T19:04:28ZengBMCOrphanet Journal of Rare Diseases1750-11722021-01-011611510.1186/s13023-021-01676-wReal-world evidence on Kovaltry (81-8973) in children with moderate or severe hemophilia A in Europe: a nested cohort analysisJamie O’Hara0Ceri Hirst1Jose Francisco Cabre Marquez2Tom Burke3Department of Health and Social Care, University of ChesterReal World Evidence, BayerGlobal Medical Affairs, BayerHCD EconomicsAbstract 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 < 18 years with moderate or severe hemophilia A, residing in five European countries and treated with FVIII were studied. The co-primary endpoints were the annualized bleeding rate (ABR) and the annual FVIII utilization rate. Twenty nine patients treated with Kovaltry were included, of whom 93% had severe disease and 75% were on continuous prophylactic treatment. The mean ABR was 2.66 ± 2.06, with rates decreasing with age. The children received on average 2.45 infusions per week, consistent across age groups (median 3; range 1–3). There were no reports of inhibitor development or adverse events in the study (AEs), and all patients were satisfied or very satisfied with the treatment. An exploratory multivariate analysis suggests no significant difference in ABR or units utilized between Kovaltry and some extended half life products in children with severe hemophilia A, though characteristics of these patient cohorts were markedly different. Conclusion This analysis demonstrates the effectiveness and safety of Kovaltry in a pan-European pediatric population with severe hemophilia A.https://doi.org/10.1186/s13023-021-01676-wHemophilia AKovaltryReal world evidenceFactor utilizationClinical burden |
| spellingShingle | Jamie O’Hara Ceri Hirst Jose Francisco Cabre Marquez Tom Burke Real-world evidence on Kovaltry (81-8973) in children with moderate or severe hemophilia A in Europe: a nested cohort analysis Orphanet Journal of Rare Diseases Hemophilia A Kovaltry Real world evidence Factor utilization Clinical burden |
| title | Real-world evidence on Kovaltry (81-8973) in children with moderate or severe hemophilia A in Europe: a nested cohort analysis |
| title_full | Real-world evidence on Kovaltry (81-8973) in children with moderate or severe hemophilia A in Europe: a nested cohort analysis |
| title_fullStr | Real-world evidence on Kovaltry (81-8973) in children with moderate or severe hemophilia A in Europe: a nested cohort analysis |
| title_full_unstemmed | Real-world evidence on Kovaltry (81-8973) in children with moderate or severe hemophilia A in Europe: a nested cohort analysis |
| title_short | Real-world evidence on Kovaltry (81-8973) in children with moderate or severe hemophilia A in Europe: a nested cohort analysis |
| title_sort | real world evidence on kovaltry 81 8973 in children with moderate or severe hemophilia a in europe a nested cohort analysis |
| topic | Hemophilia A Kovaltry Real world evidence Factor utilization Clinical burden |
| url | https://doi.org/10.1186/s13023-021-01676-w |
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