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. 2024 Feb 5:11:1347024.
doi: 10.3389/fmed.2024.1347024. eCollection 2024.

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

Rosa Sonja Alesci  1 , Georg Goldmann  2 , Susan Halimeh  3 , Katharina Holstein  4 , Christoph Königs  5 , Wolfgang Miesbach  6 , Christian Pfrepper  7 , Martin Olivieri  8
Affiliations

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

Rosa Sonja Alesci et al. Front Med (Lausanne). .

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.

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Conflict of interest statement

RA has acted as a paid consultant to CSL Behring and has received funding for research carried out in this work. She has received funding from Takeda, Sobi, Octapharma, Grifols, and Novartis and meeting/congress support from Takeda, Sobi, and Bayer. Furthermore, RA acted as a paid consultant to Amgen, Bayer, BFSH, Grifols, LFB Pharma, Medlearning, Octapharma, onkowissen, STREAMED UP (Pfizer, Grifols), Sobi, and Takeda Pharma. GG reports receiving honoraria for lectures from Bayer, Biotest, Novo Nordisk, Octapharma, Roche, Sobi, and Takeda, and reimbursement for travel expenses from Biotest, Novo Nordisk, and Sobi. SH reports grants or contracts from Bayer Healthcare, Baxalta Innovations (now Takeda), Biotest, CSL Behring, Novo Nordisk Pharma, Octapharma, Pfizer Pharma. Further, SH has received honoraria from Bayer Healthcare, Baxalta Innovations (now Takeda), Biotest, CSL Behring, Novartis Pharma, Novo Nordisk Pharma, Octapharma, Pfizer Pharma, Roche Pharma; Swedish Orphan Biovitrum and consulting fees from Bayer Healthcare, Biotest, CSL Behring, Novo Nordisk Pharma, Octapharma, Chugai Pharma Germany, Swedish Orphan Biovitrum. KH reports grants for research or clinical studies (to institution): Bayer, CSL Behring, Novo Nordisk, Pfizer, Sobi and GWT/Roche, and honoraria for lectures or consultancy (to person): Bayer, BioMarin, Biotest, Chugai, CSL Behring, LFB, Novo Nordisk, Pfizer, Roche, Sobi and Takeda. KH has served as a consultant for Bayer, Biotest, CSL Behring, LFB, Novo Nordisk, Pfizer, Roche and Sobi, and in an advisory board for Octapharma. She has received meeting/congress support from Pfizer, Sobi, Takeda, Bayer, CSL Behring, Novo Nordisk and has served as a speaker for the Haemophilia Board of the GTH and is a member of the Ärztlicher Beirat of the Deutsche Hämophiliegesellschaft e.V. (DGH; Medical Advisory Board of the German Haemophilia Society e.V.). CK has received consulting fees or honoraria for lectures and meeting/congress support from BFSH, CSL Behring, MSD, Novo Nordisk, Roche/Chugai, Sobi/Sanofi, and Takeda. CK’s institution has received grants from Bayer Vital GmbH, Biotest, CSL Behring, Intersero, Novo Nordisk, Pfizer, Roche/Chugai, Sanofi/Sobi, Takeda, and the European Union (H2020) and federal funding. He is a member of the German Paediatric AIDS Society and Head of the German Haemophilia PUP Registry. WM has acted as a paid consultant to Bayer, BioMarin, Biotest, CSL Behring, Chugai, Freeline, LFB, Novo Nordisk, Octapharma, Pfizer, Regeneron, Roche, Sanofi, Sigilon, Sobi, Takeda/Shire, uniQure and has received research funding form Bayer, Biotest, CSL Behring, LFB, Novo Nordisk, Octapharma, Pfizer, Sobi, Takeda/Shire. WM has received honoraria for lectures from Bayer, BioMarin, Biotest, CSL Behring, Chugai. LFB, Novo Nordisk, Octapharma, Pfizer, Roche, Sobi, and Takeda/Shire and has received meeting/congress support from Bayer, BioMarin, Biotest, CSL Behring, LFB, Novo Nordisk, Octapharma, Pfizer, Roche, Sobi, Takeda/Shire, uniQure. CP reports grants for studies and research from Chugai, Roche, LeoPharma, Zacros, and Takeda; and personal fees for lectures or consultancy from Alexion, Bayer, BioMarin, Chugai Pharma, Roche, CSL Behring, Leo Pharma, Novo Nordisk, Pfizer, BMS, Sanofi, Sobi, Takeda, and Zarcos. MO has received honoraria or consulting fees from Bayer, BioMarin, Biotest, Chugai, CSL Behring, Novo Nordisk, Octapharma, Pfizer, Roche, Sobi, Stago and Takeda. MO has participated in DSMB/advisory boards from Bayer, Biomarin, Biotest, Chugai, CSL Behring, Novo Nordisk, Octapharma, Pfizer, Roche, Sobi and Takeda and has received meeting/travel support from Bayer, Biotest, CSL Behring, Novo Nordisk, Pfizer, Roche, Sobi, Takeda. This study received funding from CSL Behring, Germany. The funder had the following involvement with the study: study design, the writing of this article (funding of medical writing support), and the decision to submit it for publication.

Figures

Figure 1
Figure 1
A bar chart depicting patient satisfaction with the support and therapy for the treatment of bleeding episodes at the hemophilia center. This chart is based on the information provided by 37 patients; the answers for six patients per topic were missing. The bars show the patient numbers at the inside end of the bars; the corresponding percentages are shown on top of the bars. Percentages relate to the number of patients who provided answers.
Figure 2
Figure 2
A bar chart representing the impact of mild hemophilia on daily life; this chart is based on the information provided by 37 patients; the answers for six patients were missing. The bars show the patient numbers at the inside end of the bars; the corresponding percentages are shown on top of the bars. Percentages relate to the number of patients who provided answers. (A) The majority of patients can always or usually detect bleeding episodes and assess when they need treatment; (B) Hemophilia is sometimes a burden on the patients, e.g., at work, school, or during leisure time; (C) The majority of patients always or usually needs support from the hemophilia center if bleeding occurs, which is sometimes the case for seven patients.
Figure 3
Figure 3
A bar chart representing the health of patients with mild hemophilia at the day of the survey; this chart is based on the information provided by 34, 35, and 37 patients, with missing information for six patients in mobility, eight patients in self-care, and nine patients each in their usual activities, pain/discomfort, and anxiety/depression. The bars show the patient numbers in the center of the respective bar section. Most patients experienced no limitations due to their condition in all five dimensions, except for the pain/discomfort dimension where nearly half of the patients experienced slight impairment.
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