Compliance with Early Long-Term Prophylaxis Guidelines for Severe Hemophilia A

Paul Saultier (1, 2) , Yves Guillaume (3) , Virginie Demiguel (4) , Claire Berger (5) , Annie Borel-Derlon (6) , Ségolène Claeyssens (7) , Annie Harroche (8) , Caroline Oudot (9) , Anne Rafowicz (10) , Marc Trossaert (11) , Bénédicte Wibaut (12) , Christine Vinciguerra (13) , Mohamed Boucekine (14) , Karine Baumstarck (14) , Sandrine Meunier (13) , Thierry Calvez (15) , Hervé Chambost (1) , Achille Aouba , Faezeh Legrand , Chantal Rothschild , Marie-Françoise Torchet , Roseline D’oiron , Thierry Lambert , Yves Laurian , Jennifer Biernat , Jenny Goudemand , Armelle Parquet , Véronique Tintillier , Anne Durin Assollant , Claude Négrier , Sabine Castet , Viviane Guérin , Yohann Huguenin , Marguerite Micheau , Anne Ryman , Céline Falaise , Marie Françoise Thiercelin-Legrand , Marianne Fiks-Sigaud , Marc Fouassier , Edith Fressinaud , Sophie Voisin , Albert Faradji , Patrick Lutz , Marie Elisabeth Briquel , Birgit Frotscher , Béatrice Fimbel , Yves Gruel , Claude Guerois , Sandra Regina , Jean Baptiste Valentin , Christine Biron Andreani , Philippe Codine , Daniel Donadio , Robert Navarro , Paola Rospide , Jean-François Schved (16, 17) , Bénédicte Collet , Annie Borel Derlon , Philippe Gautier , Sophie Bayart , Ben⊚it Guillet , Jeanneyvonne Borg , Cécile Dumesnil , Charline Normand , Pascale Schneider , Philippe Tron , Jean-Pierre Vannier , Fabienne Dutrillaux , Fabienne Volot , Piotr Gembara , Alain Marques-Verdier , Dalila Adjaoud , Claire Barro , Gilles Pernod , Ben⊚it Polack , Patricia Pouzol , Nadra Ounnoughene , Patricia Paugy , Valérie Robert , Natalie Stieltjes , Brigitte Bastenaire , Emmanuelle de Raucourt , Jocelyne Peynet , Valérie Li-Thiao-Te , Brigitte Pautard , Catherine Behar , Stéphanie Gorde , Martine Munzer , Valérie Gay , Elisabeth Benz Lemoine , Laurent Macchi , Lionel de Lumley , Solange Gaillard , Anne Deville , Fabrice Monpoux , Marie Anne Bertrand , Brigitte Pan-Petesch , Abel Hassoun , Brigitte Coatmelec , Philippe Beurrier , Michèle Damay , Philippe Moreau , Odile Pouille-Lievin , Caroline Schoepfer , Eliane Tarral , Monique Bianchin , Joël Nguyen , Olivier Pincemaille , Marie-Odile Peter

1 C2VN - Centre recherche en CardioVasculaire et Nutrition = Center for CardioVascular and Nutrition research
2 TIMONE - Hôpital de la Timone [CHU - APHM]
3 APHM - Assistance Publique - Hôpitaux de Marseille
4 HNSM - Hôpitaux de Saint Maurice
5 CHU ST-E - Centre Hospitalier Universitaire de Saint-Etienne [CHU Saint-Etienne]
6 CUMR Caen - Centre Universitaire des Maladies Rénales [CHU Caen]
7 CHU Toulouse - Centre Hospitalier Universitaire de Toulouse
8 Hôpital Necker - Enfants Malades [AP-HP]
9 CHU Limoges
10 Hôpital Bicêtre [AP-HP, Le Kremlin-Bicêtre]
11 CHU Nantes - Centre Hospitalier Universitaire de Nantes
12 CHRU Lille - Centre Hospitalier Régional Universitaire [CHU Lille]
13 HCL - Hospices Civils de Lyon
14 CEReSS - Centre d'études et de recherche sur les services de santé et la qualité de vie
15 iPLESP - Institut Pierre Louis d'Epidémiologie et de Santé Publique
16 CHRU Montpellier - Centre Hospitalier Régional Universitaire [Montpellier]
17 UM - Université de Montpellier

Objectives: To evaluate the applicability and compliance with guidelines for early initiation of long-term prophylaxis in infants with severe hemophilia A and to identify factors associated with guideline compliance. Study design: This real-world, prospective, multicenter, population-based FranceCoag study included almost all French boys with severe hemophilia A, born between 2000 and 2009 (ie, after guideline implementation). Results: We included 333 boys in the study cohort. The cumulative incidence of long-term prophylaxis use was 61.2% at 3 years of age vs 9.5% in a historical cohort of 39 boys born in 1996 (ie, before guideline implementation). The guidelines were not applicable in 23.1% of patients due to an early intracranial bleeding or inhibitor development. Long-term prophylaxis was delayed in 10.8% of patients. In the multivariate analysis, 2 variables were significantly associated with "timely long-term prophylaxis" as compared with "delayed long-term prophylaxis": hemophilia treating center location in the southern regions of France (OR 23.6, 95% CI 1.9-286.7, P = .013 vs Paris area) and older age at long-term prophylaxis indication (OR 7.2 for each additional year, 95% CI 1.2-43.2, P = .031). Long-term prophylaxis anticipation was observed in 39.0% of patients. Earlier birth year (OR 0.5, 95% CI 0.3-0.8, P = .010 for birth years 2005-2009 vs 2000-2004) and age at first factor replacement (OR 1.9 for each additional year, 95% CI 1.2-3.0, P = .005) were significantly associated with "long-term prophylaxis guideline compliance" vs "long-term prophylaxis anticipation." Conclusions: This study suggests that long-term prophylaxis guidelines are associated with increased long-term prophylaxis use. However, early initiation of long-term prophylaxis remains a challenge.

Domaines: Sciences du Vivant [q-bio]; Pédiatrie

Paul Saultier, Yves Guillaume, Virginie Demiguel, Claire Berger, Annie Borel-Derlon, et al.. Compliance with Early Long-Term Prophylaxis Guidelines for Severe Hemophilia A. The Journal of Pediatrics, 2021, 234, pp.212-219.e3. ⟨10.1016/j.jpeds.2021.02.071⟩. ⟨hal-03653434⟩