Abstract
Background: No study has reported the status and chronological trend of prophylactic recombinant factor VIII (rFVIII) use in Chinese pediatric patients with hemophilia A (HA).
Objective: We aimed to analyze the status and trend of rFVIII-containing prophylaxis in Chinese pediatric patients with HA.
Methods: ReCARE (Retrospective study in Chinese pediatric hemophilia A patients with rFVIII contained REgular prophylaxis) was a retrospective study conducted in 12 hemophilia treatment centers across China. The trend of prophylaxis was evaluated by determining the mean duration of prophylaxis, mean injection frequency (per week), mean dose of each injection (IU/kg), mean total dose injected/week (IU) and proportion of rFVIII consumption relative to factor VIII (FVIII) consumption over the study period.
Results: We analyzed 183 male pediatric patients with HA (mean age, 7.1 ± 4.23 years), who received intermittent prophylaxis between 1 November 2007 and 31 May 2013. The mean duration of prophylaxis with rFVIII increased from 16.72 weeks in 2008 to 32.77 in 2012. Per injection dose of rFVIII increased significantly from 2008 to 2013 (25.89 to 28.31 IU/kg, p < .001). An increase was also reported in the mean total FVIII consumed (699.97 ± 173.25 IU in 2008 and 891.30 ± 730.341 in 2013) and mean proportion of rFVIII used (33.33 ± 57.73% in 2008 to 85.50 ± 29.077% in 2013).
Conclusion: Our data revealed an overall improvement in treatment dosage and duration with an increase in the number of patients receiving prophylaxis. The total proportion of rFVIII also increased gradually indicating the development of economy and safety awareness.
Trial Registration: The trial is registered at ClinicalTrials.gov (CT.gov identifier: NCT02263066).
Notes
Transparency
Declaration of funding
This study was sponsored by Bayer Healthcare Co. Ltd.
Author contributions: C.G.L., X.Z., Y.Z., R.W., Q.H., W.X., J.S., R.Y., X.L., R.Z., S.L., J.G., J.W. and Q.H. contributed in conceptualizing the study design, data collection, analysis and interpretation. All the authors have approved the final version of the manuscript.
Declaration of financial/other relationships
C.G.L., X.Z., Y.Z., R.W., Q.H., V.X., J.S., R.Y., X.L., R.Z., S.L., J.G., J.W. and Q.H. have disclosed that they have no significant relationships with or financial interests in any commercial companies related to this study or article.
CMRO peer reviewers on this manuscript have no relevant financial or other relationships to disclose.
Acknowledgements
We would like to thank Mr. Karan Sharma (MPharm) and Dr. Amit Bhat (PhD) from Indegene Pvt Ltd, Bangalore, India for providing medical writing support and critical evaluation of literature in the development of this manuscript, as funded by Bayer Healthcare Co. Ltd.
Notes
1 Kogenate is a registered trade name of Bayer Healthcare Co. Ltd
2 Advate is a registered trade name of Baxter Corporation
3 Xyntha is a registered trade name of Pfizer Inc.