Implementation of the French primary long-term prophylaxis guidelines: A real-world prospective study of the FranceCoag PUPs cohort
P. Saultier1,2,*, Y. Guillaume1, V. Demiguel3, C. Berger4, A. Borel-Derlon5, S. Claeyssens6, A. Harroche7, C. Oudot8, A. Rafowicz9, M. Trossaert10, B. Wibaut11, M. Boucekine12, K. Baumstarck12, S. Meunier13, T. Calvez14, H. Chambost1,2, on behalf of FranceCoag PUPs / CoMETH Prophylaxis Study Group
1CHU Timone Enfants, Department of pediatric hematology and oncology, APHM, Marseille, France; 2NORT, Aix Marseille Univ, INSERM, INRA, Marseille, France; 3Santé Publique France, French national public health agency, Saint-Maurice, France; 4Hematology and Oncology Pediatric Unit, Saint-Etienne University Hospital, Saint Etienne, France; 5Center for bleeding disorders, La Côte de Nacre University Hospital, Caen, France; 6Center for bleeding disorders, Purpan University Hospital, Toulouse, France; 7Necker University Hospital, Center for bleeding disorders, Assistance Publique - Hôpitaux de Paris, Paris, France; 8Hematology and Oncology Pediatric Unit, Limoges University Hospital, Limoges, France; 9Bicêtre University Hospital, Center for bleeding disorders, Assistance Publique - Hôpitaux de Paris, Le Kremlin Bicêtre, France; 10Center for bleeding disorders, Nantes University Hospital, Nantes, France; 11Center for bleeding disorders, Lille 2 University Hospital, Lille, France; 12EA 3279, Self-perceived Health Assessment Research Unit, School of Medicine, Aix-Marseille Univ, Marseille, France; 13Lyon University Hospital, Center for bleeding disorders, HCL, Lyon, France; 14Institut Pierre Louis d’Épidémiologie et de Santé publique (IPLESP UMRS 1136), Sorbonne Universités, UPMC Univ Paris 06, Inserm, Paris, France
Introduction: Primary long-term prophylaxis (LTP) efficiently prevents arthropathy in children with severe hemophilia. In early 2000s, the French group of hemophilia treaters (CoMETH) published their first LTP guidelines. Several issues may impair the actual LTP use in young patients. However, real-world evaluations of the implementation of such guidelines are scarce. This study aimed at prospectively analyzing the LTP guidelines implementation in France and at identifying factors modulating the LTP initiation.
Methods: The study group consisted of 395 boys from the FranceCoag PUPs cohort. All included patients had severe hemophilia A or B (FVIII/FIX <1%) and were born in 2000-2009 (i.e. after the French LTP guidelines dissemination). Sociodemographic, clinical, biological, genetic, and therapeutic data were prospectively recorded during medical visits in the hemophilia treatment center (HTC). The study group was compared to a historical group of 44 registered boys with severe hemophilia born in 1996 (retrospective data collection).
Results: In the study group, the median age at first joint bleed was 1.9 years (IQR 1.1-3.0). The cumulative incidence of primary LTP use according to age was dramatically higher in the study group compared to the historical group (e.g. cumulative incidence of LTP use 59.2% versus 8.5% at three years of age, respectively). The prevalence of ineligibility to the guidelines, delay or absence of LTP initiation, anticipated LTP initiation, and timely LTP initiation according to guidelines was 20.8%, 9.9%, 38.5% and 30.9%, respectively. A multivariate analysis showed that two factors were significantly associated with anticipated or timely versus delayed or absent LTP initiation: the year of birth (OR 1.2 per each additional year, 95% CI 1.1-1.3, P < .001) and the HTC location (OR 6.4, 95% CI 1.4-29.2, P = .02 and OR 6.2, 95% CI 2.0-19.4, P = .002 for Northwest and Southeast part of France, respectively).
Discussion/Conclusion: This analysis provides pivotal data to evaluate the adherence to early LTP guidelines and to identify barriers for their implementation. CoMETH guidelines dissemination was associated with a dramatic increase in early LTP use. The study highlights the capacity of FranceCoag device to assess critical variables and evaluate therapeutic practice in one of the largest national PUPs cohort worldwide.
Disclosure of interest: None declared.