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Journal articles

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⊚ıt 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⊚ıt 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 de Saint-Etienne - Centre Hospitalier Universitaire de Saint-Etienne
6 CUMR Caen - Centre Universitaire des Maladies Rénales [CHU Caen]
7 CHU Toulouse [Toulouse]
8 CHU Necker - Enfants Malades [AP-HP]
9 CHU Limoges
10 AP-HP Hôpital Bicêtre (Le Kremlin-Bicêtre)
11 CHU Nantes - Centre hospitalier universitaire de Nantes
12 CHRU Lille - Centre Hospitalier Régional Universitaire [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
Abstract : 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.
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https://hal-amu.archives-ouvertes.fr/hal-03653434
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Submitted on : Wednesday, April 27, 2022 - 5:29:44 PM
Last modification on : Saturday, May 21, 2022 - 3:58:51 AM

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UNIV-AMU | INCIAM | INRAE | UNIV-MONTPELLIER | HCL | IPLESP | SORBONNE-UNIVERSITE | SU-MEDECINE | COMUE-NORMANDIE | UNICAEN

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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, Elsevier, 2021, 234, pp.212-219.e3. ⟨10.1016/j.jpeds.2021.02.071⟩. ⟨hal-03653434⟩

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