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3982 Health Care Resource Use, Comorbidities, and Costs of Hemophilia B Patients in France: A Nationwide Claims Database Analysis

Program: Oral and Poster Abstracts
Session: 322. Disorders of Coagulation or Fibrinolysis: Clinical and Epidemiological: Poster III
Hematology Disease Topics & Pathways:
Research
Monday, December 11, 2023, 6:00 PM-8:00 PM

Laurent Frenzel, MD, PhD1*, Véronique Cahoreau2*, Nicolas Giraud3*, Stephanie Delienne4*, Francis Fagnani5*, Stéphane Bouée6*, Juliette Cottin5*, Isabelle Bureau5*, Jeremie Rudant7*, Anais Reynaud8*, Amelie Coumert8*, Herve Lilliu9* and Aurelien Lebreton10*

1Department of Haematology, Institut Necker, Paris, FRA
2CHU de Bordeaux, Bordeaux, France
3Association Française des Hémophiles, Paris, France
4CHU Dijon, Dijon, France
5Cemka, Bourg-la-Reine, France
6CEMKA, Bourg-la-Reine, France
7Pfizer France, Paris, France
8Pfizer, Paris, France
9Inbeeo, London, United Kingdom
10CHU Clermond Ferrand, Clermond Ferrand, France

Background and objectives

Hemophilia B is an inherited bleeding disorder characterized by a deficiency in blood clotting factor IX (FIX). Its severity varies based on the degree of FIX deficiency, with the most severe cases experiencing frequent spontaneous hemorrhages (FIX levels < 1 IU/dL). Treatment options include on-demand administration following a hemorrhage or prophylaxis to prevent bleeding. A complication is the production of inhibitory antibodies against the coagulation factors in some patients, leading to more aggressive and expensive treatments.

The aim of this study was to describe Hemophilia B patients according to severity in France in 2021, including sociodemographic characteristics and comorbidities, current treatments, healthcare resource use and related costs.

Methods

This study used the French health-insurance claims database (SNDS) containing pseudonymized individual data for over 66 million people. Hemophilia B patients are fully covered in France and might be identified by the ICD-10 code D67. Patients alive on January 1st, 2021, were selected after exclusion of those presenting with Willebrand disease and other rare bleeding disorders. Comorbidities were identified through validated algorithms. An age and gender matched control group without Hemophilia B was randomly selected in the general population: for each HB patient a subject of the same age,gender and region of residence was randomly selected among the overall population of French citizens (excluding HB patients). Sub-group analyses were performed according to the treatment pattern (on demand / in prophylaxis) and the presence of inhibitors (defined with the treatment: high dose of factor IX or bypassing agents). Direct costs were estimated in a societal perspective.

Results

1,311 patients with Hemophilia B were identified. Mean age was 36 years and 83.5% were males.

Compared to controls, hemophilia patients had significantly (p<0.05) more frequently an HIV infection, hepatitis B or C, and a chronic cardiopathy (Figure 1). There were no significant differences for coronary artery diseases, chronic heart failure, arrhythmia, and hepatic diseases.

Respectively 996 (76.0%) and 303 (23.1%) patients were treated on demand and in prophylaxis both without inhibitors. Less than 10 patients were treated either on demand with inhibitors or in prophylaxis with inhibitors.

Compared to controls, patients with hemophilia B had more frequent consultations with general practitioners and hospital specialists, visits to emergency unit, with nurses and physiotherapists (Table 1). They also were more frequently treated with analgesics and corticosteroids but less frequently with non-steroid anti-inflammatory drugs. The proportion of hemophilia B patients hospitalized was higher than controls, overall and also for bleeding and for orthopedic surgery.

The mean annual direct medical costs (drugs, consultations, hospitalizations, ...) varied strongly according to treatment modalities:

  • €6,506 for patients treated on demand without inhibitors (versus €1,795 for their matched controls)
  • €162,635 for patients treated in prophylaxis without inhibitors (versus €1,071 for their matched controls).

The majority of the costs was related to antihemophilic drugs: 33% and 92% respectively for these 2 treatment groups.

Conclusion:

These results highlight the burden of hemophilia B in terms of comorbidities, healthcare consumptions and cost. The cost of Hemophilia B varied greatly with disease severity and was mostly due to the antihemophilic drugs

Disclosures: Frenzel: Roche: Consultancy; Pfizer: Consultancy, Other: Grant, Research Funding; CSL Berhing: Consultancy, Research Funding; Biomarin: Consultancy. Cahoreau: Pfizer: Consultancy. Delienne: Pfizer: Consultancy. Rudant: Pfizer: Current Employment. Reynaud: Pfizer: Current Employment. Coumert: Pfizer: Current Employment. Lilliu: Pfizer: Consultancy. Lebreton: Pfizer: Consultancy; Pfizer: Research Funding.

*signifies non-member of ASH