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Severity and burden of sickle cell disease in France: a nationwide real-world study.
Brousse, Valentine; Bernaudin, Francoise; Melaine, Asma; Goguillot, Melanie; Gallagher, Meghan; Benard, Steve; Habibi, Anoosha.
Afiliação
  • Brousse V; Centre de Référence MCGRE, Service d'Hématologie-Immunologie, AP-HP, Hôpital Robert Debré, F-75019 Paris, France 2. Université Paris Cité and Université des Antilles, Inserm, BIGR, F-75015 Paris.
  • Bernaudin F; French Referral Center for Sickle Cell Disease; SFGM-TC (Société Française de Greffe de Moelle et de Thérapie Cellulaire); DrepaGreffe Association 20 rue de Coulmiers, 94130 Nogent sur Marne.
  • Melaine A; Steve Consultants, Oullins.
  • Goguillot M; Steve Consultants, Oullins.
  • Gallagher M; bluebird bio, Inc., address 455 Grand Union Blvd Somerville, MA, 02145-1446 United States of America.
  • Benard S; Steve Consultants, Oullins. sbenard@steve-consultants.com.
  • Habibi A; Sickle Cell Referral Center, Henri Mondor Hospital, AP-HP, UPEC, Laboratory of Excellence GR-Ex, INSERM Unit 955, Mondor Institute of Biomedical Research, Paris-Est Creteil University, Creteil.
Haematologica ; 108(9): 2476-2486, 2023 09 01.
Article em En | MEDLINE | ID: mdl-36924235
The burden of sickle cell disease (SCD) in France has been difficult to apprehend due to the paucity of reliable nationwide epidemiological data. We aimed to describe the epidemiology of SCD and evaluate its burden and costs. Patients with SCD and most severely affected patients were identified between 2012 and 2018 from the French National Health Data System database (SNDS, Système national des données de santé). Outcomes of interest included rates of acute and chronic complications, healthcare resource utilization and associated costs, and were compared in subpopulations of patients before and after hematopoietic stem cell transplantation, initiating hydroxyurea or a chronic transfusion program. Between 2012 and 2018, 22,619 patients with SCD were identified, among which 4,270 patients were defined as most severely affected. Rates of vaso-occlusion episodes and acute chest syndrome were 86.29 (95% confidence interval [CI]: 85.75-86.83] and 12.90 (95% CI: 12.69-13.11) per 100 person years in the study population and 166.9 (95% CI: 165.4- 168.4) and 22.71 (95% CI: 22.16-23.27) per 100 person years in most severely affected patients. Median (Q1-Q3) annualized total costs were €5,073.63 (range, €1,633.74-14,000.94) and €13,295.67 (range, €5,754.67-26,385.23) in the study population and most severely affected patients. Median annualized costs were ten times lower after treatment intensification for hematopoietic stem cell transplantation (€29,011.75 vs. €2,465.98; P<0.001), they slightly decreased after hydroxyurea initiation (€13,057.79 vs. €12,752.44; P=0.003) and were five times higher after chronic transfusion program initiation (€4,643.11 vs. €22,715.85; P<0.001). SCD still places a significant demand on health resources, even after therapeutic intensification.
Assuntos

Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Transplante de Células-Tronco Hematopoéticas / Anemia Falciforme Limite: Humans País/Região como assunto: Europa Idioma: En Ano de publicação: 2023 Tipo de documento: Article

Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Transplante de Células-Tronco Hematopoéticas / Anemia Falciforme Limite: Humans País/Região como assunto: Europa Idioma: En Ano de publicação: 2023 Tipo de documento: Article