Long-term efficacy and safety of prophylaxis with recombinant factor VIII in Chinese pediatric patients with hemophilia A: a multi-center, retrospective, non-interventional, phase IV (ReCARE) study

Abstract

Background: The first recombinant factor VIII (rFVIII) product was launched in China in 2007. However, until now, no study has been conducted to describe the efficacy and safety of prophylaxis with rFVIII in Chinese pediatric patients with hemophilia A (HA).

Objective: To summarize the efficacy and safety data on prophylaxis with rFVIII 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 (HTCs) across China. The primary endpoints included reduction in annualized bleeding rate (ABR); the secondary endpoints included evaluation of joint function (number and sites of target joints) using Gilbert score and Hemophilia Joint Health Score (HJHS), quality of life (QoL) and factors affecting treatment choices. Safety assessment of rFVIII was also conducted.

Results: We analyzed a total of 183 male pediatric patients (mean age, 7.1 ± 4.23 years) who received prophylaxis between 1 November 2007 and 31 May 2013. Compared with baseline, prophylaxis with rFVIII significantly reduced overall annualized joint bleed rate (AJBR) (p < .001) and ABR (p < .001). Inhibitor formation was reported in 5 (2.7%) patients and hemarthrosis was reported in 1 patient. The mean number of target joints was positively related to age (p < .001) and weight (p = .003) at baseline. Responses from survey questionnaires reported that effective bleeding control, joint protection, improvement in quality of life, favorable medical insurance policies, and economic capability were reasons for choosing prophylaxis.

Conclusion: Prophylaxis with rFVIII reduced bleeding and number of target joints, even with a low-dose regimen, in Chinese pediatric patients with HA. Other than the efficacy and safety, factors such as poor disease control, improved economic stability and stable financial support made prophylaxis as an attractive treatment option.

ClinicalTrials.gov ID: NCT02263066.

Keywords:

• Hemophilia A
• annualized bleeding rate
• Kogenate
• Pediatric
• Prophylaxis
• ReCARE
• rFVIII

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., W.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.

Acknowledgments

The authors thank Karan Sharma MPharm and Dr. Amit Bhat PhD from Indegene Pvt Ltd, Bangalore, India, for providing medical writing support and critically evaluating the literature, and for 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.