Patient perspective on living with mild hemophilia in Germany: results from a nationwide survey

Rosa Sonja Alesci; Georg Goldmann; Susan Halimeh; Katharina Holstein; Christoph Königs; Wolfgang Miesbach; Christian Pfrepper; Martin Olivieri

doi:10.3389/fmed.2024.1347024

Patient perspective on living with mild hemophilia in Germany: results from a nationwide survey

Front Med (Lausanne). 2024 Feb 5:11:1347024. doi: 10.3389/fmed.2024.1347024. eCollection 2024.

Authors

Rosa Sonja Alesci¹, Georg Goldmann², Susan Halimeh³, Katharina Holstein⁴, Christoph Königs⁵, Wolfgang Miesbach⁶, Christian Pfrepper⁷, Martin Olivieri⁸

Affiliations

¹ IMD Blood Coagulation Center Hochtaunus, Bad Homburg, Germany.
² Institute of Experimental Hematology and Transfusion Medicine, University of Bonn, Bonn, Germany.
³ Blood Coagulation Center Rhein-Ruhr, Duisburg-Altstadt, Germany.
⁴ II. Medical Department, Coagulation and Hemophilia Center, University Medical Center Hamburg-Eppendorf, Hamburg, Germany.
⁵ Department of Paediatrics and Adolescent Medicine, Clinical and Molecular Haemostasis, Goethe University, University Hospital Frankfurt, Frankfurt, Germany.
⁶ Department of Hemostaseology and Hemophilia Center, Medical Clinic 2, University Hospital Frankfurt, Frankfurt, Germany.
⁷ Division of Hemostaseology, Department of Hematology, Cellular Therapy, Hemostaseology and Infectiology, University of Leipzig Medical Center, Leipzig, Germany.
⁸ Pediatric Thrombosis and Hemostasis Unit, Pediatric Hemophilia Center, Dr. von Hauner Children's Hospital, LMU München, Munich, Germany.

Abstract

Introduction: The disease burden and bleeding risk of patients with mild hemophilia may be underestimated. Their health-related quality of life (QoL) may be negatively impacted by insufficient treatment and bleed-related joint damage connected to a potentially delayed diagnosis.

Aim: This study aims to gain information on the care reality and QoL of patients aged ≥12 years with mild hemophilia in Germany.

Methods: An anonymous cross-sectional patient survey using standardized questionnaires was conducted in a validated electronic patient-reported outcome system. Medical specialists, hemophilia centers, patient organizations, and support groups across Germany invited the patients.

Results: A total of 43 patients (35 patients with hemophilia A, 5 patients with hemophilia B, and 3 patients for whom the information was missing) with a median age of 33 years were analyzed. The median age at diagnosis was 6.0 years (interquartile range [IQR] 2.0-15.0), and the median factor activity was 14.0% (IQR 12.0-25.0). Nearly 85% of the patients received factor concentrates in the past, and the most common reasons for the treatment were surgery or joint bleeding (each 65.6%). Half of the patients who provided feedback experienced complications during bleeding episodes. Prophylactic treatment with factor concentrates was rare (10.3%). The patients had minor problems regarding their health status.

Conclusion: Bleeding complications and joint bleeding, in particular, may be highly underestimated in patients with mild hemophilia, highlighting a medical need in this population. Patients with a potential benefit from prophylaxis need to be identified. Mild hemophilia has a negative impact on patients' QoL. Hemophilia centers satisfied the patients' needs. Further research is needed to address the current lack of awareness and improve adequate treatment in the future.

Keywords: care reality; hemophilia A; hemophilia B; joint bleeding; mild hemophilia; quality of life.

Grants and funding

The author(s) declare financial support was received for the research, authorship, and/or publication of this article.