An observational study of haemophilia A patients without inhibitors using the French national claims (SNDS) database

ABSTRACT

Objectives:

To describe clinical characteristics, factor consumption, and events of interest in patients with haemophilia A without inhibitors receiving prophylaxis in France, and the clinical impact of switching to Elocta® in this population.

Methods:

This retrospective, observational study using the Système National des Données de Santé database, analysed data from patients with haemophilia A without inhibitors using prophylactic factor VIII (FVIII) replacement therapy during 2016–2019. Clinical characteristics, treatment patterns and switches, factor consumption, and rate of events of interest were determined. In a sub-cohort of patients treated with Elocta®, clinical characteristics, factor consumption, and rate of events of interest before and after switching to Elocta® were compared.

Results:

For 545 patients, with mean age (standard deviation [SD]) 25.4 (17.8) years, Elocta® was the most used treatment. Bleeding events and articular non-bleeding events leading to hospitalization occurred in 15.4% and 13.9% of patients, respectively, and 9.9% of patients had surgeries or procedures related to haemophilic arthropathy. The mean (SD) FVIII product consumption was 344 (93) IU/kg/month for extended half-life treatment, and 331 (98) IU/kg/month for standard half-life products. For the sub-cohort of 146 patients, bleeding events (SD) decreased from 0.32 (2.2) to 0.09 (0.42) events/patient/year (p = 0.227) after switching to Elocta®. There was no statistically significant difference in rates of factor consumption or articular non-bleeding events before and after initiation of Elocta®.

Conclusion:

This study provides real-world insights that advance the understanding of treatment patterns and events of interest in patients with haemophilia A on prophylactic regimens in France.

KEYWORDS:

• Haemophilia
• factor replacement therapy
• real-world data
• observational

Acknowledgements

We acknowledge the contribution of Valérie Bastard, of Sobi, Paris, France, in the preparation of this article. Medical writing support, funded by Sobi, was provided by Helen Bristow, BSc, and Hayley Owen, PhD, Bioscript Group, Macclesfield, UK. Sobi and Sanofi reviewed and provided comments on the manuscript. The authors had full editorial control of the manuscript and provided their final approval of all content.

Disclosure statement

MT declares support for the present manuscript from Sobi and grants and contracts from Takeda, Novo Nordisk and LFB. AF is a Sobi employee and shareholder of Sobi. LG was an employee of Cerner Enviza who received funding from Sobi to conduct the study and is currently an employee of Janssen France. NK is an employee of Sobi and holds Sobi stock. OvH is an employee of Sobi. RV declares support for the present manuscript from Sobi, grants and contracts from LFB, Takeda, Biomarin, Novo Nordisk and Roche, and support for attending meetings and/or travel from Takeda and LFB.

Author contribution

MT and RV contributed to the study concept and study design and interpreted the data. AF interpreted the data. LG performed quality control of data and algorithms, analysed and interpreted the data and performed statistical analysis. NK analysed and interpreted the data and performed statistical analysis. OvH contributed to the study concept and study design, interpreted the data, and performed statistical analysis. All authors critically reviewed and revised the draft manuscript, approved the final version to be published and agreed to be accountable for all aspects of the work.

Data availability

Sobi is committed to responsible and ethical sharing of data on participant level and summary data for medicines and indications approved by EMA and/or FDA, while protecting individual participant integrity and compliance with applicable legislation. Data access will be granted in response to qualified research requests. All requests are evaluated by a cross-functional panel of experts within Sobi and a decision on sharing will be based on the scientific merit and feasibility of the research proposal, maintenance of personal integrity and commitment to publication of the results. To request access to study data, a data sharing request form (available on www.sobi.com) should be sent to [email protected]. Further information on Sobi’s data sharing policy and process for requesting access can be found at: https://www.sobi.com/en/policies. The raw data were part of the SNDS and were available from the Health Data Hub under special authorization.

Additional information

Funding

The study was funded by Sobi.