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Journal Articles The Journal of Pediatrics Year : 2021

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
Virginie Demiguel
Caroline Oudot
  • Function : Author
Sandrine Meunier
Achille Aouba
  • Function : Author
Faezeh Legrand
  • Function : Author
Chantal Rothschild
  • Function : Author
Marie-Françoise Torchet
  • Function : Author
Roseline D’oiron
  • Function : Author
Thierry Lambert
  • Function : Author
  • PersonId : 756261
  • IdRef : 026962772
Yves Laurian
  • Function : Author
Jennifer Biernat
  • Function : Author
Jenny Goudemand
  • Function : Author
Armelle Parquet
  • Function : Author
Véronique Tintillier
  • Function : Author
Anne Durin Assollant
  • Function : Author
Claude Négrier
  • Function : Author
Sabine Castet
  • Function : Author
Viviane Guérin
  • Function : Author
Yohann Huguenin
  • Function : Author
Marguerite Micheau
  • Function : Author
Anne Ryman
  • Function : Author
Céline Falaise
  • Function : Author
Marianne Fiks-Sigaud
  • Function : Author
Marc Fouassier
  • Function : Author
Edith Fressinaud
  • Function : Author
Sophie Voisin
Albert Faradji
  • Function : Author
Patrick Lutz
  • Function : Author
Marie Elisabeth Briquel
  • Function : Author
Birgit Frotscher
  • Function : Author
Béatrice Fimbel
  • Function : Author
Yves Gruel
Claude Guerois
  • Function : Author
Sandra Regina
  • Function : Author
Jean Baptiste Valentin
  • Function : Author
Christine Biron Andreani
  • Function : Author
Philippe Codine
  • Function : Author
Daniel Donadio
  • Function : Author
Robert Navarro
  • Function : Author
Paola Rospide
  • Function : Author
Bénédicte Collet
  • Function : Author
Annie Borel Derlon
  • Function : Author
Philippe Gautier
  • Function : Author
Sophie Bayart
  • Function : Author
Ben⊚it Guillet
  • Function : Author
Jeanneyvonne Borg
  • Function : Author
Cécile Dumesnil
  • Function : Author
Charline Normand
  • Function : Author
Pascale Schneider
  • Function : Author
Philippe Tron
  • Function : Author
Jean-Pierre Vannier
  • Function : Author
Fabienne Dutrillaux
  • Function : Author
Fabienne Volot
  • Function : Author
Piotr Gembara
  • Function : Author
Alain Marques-Verdier
  • Function : Author
Dalila Adjaoud
  • Function : Author
Claire Barro
  • Function : Author
Gilles Pernod
  • Function : Author
Ben⊚it Polack
  • Function : Author
Patricia Pouzol
  • Function : Author
Nadra Ounnoughene
  • Function : Author
Patricia Paugy
  • Function : Author
Valérie Robert
  • Function : Author
Natalie Stieltjes
  • Function : Author
Brigitte Bastenaire
  • Function : Author
Emmanuelle de Raucourt
  • Function : Author
Jocelyne Peynet
  • Function : Author
Valérie Li-Thiao-Te
  • Function : Author
Brigitte Pautard
  • Function : Author
Catherine Behar
  • Function : Author
Stéphanie Gorde
  • Function : Author
Martine Munzer
  • Function : Author
Valérie Gay
  • Function : Author
Elisabeth Benz Lemoine
  • Function : Author
Laurent Macchi
  • Function : Author
Lionel de Lumley
  • Function : Author
Solange Gaillard
  • Function : Author
Anne Deville
  • Function : Author
Fabrice Monpoux
  • Function : Author
Marie Anne Bertrand
  • Function : Author
Brigitte Pan-Petesch
  • Function : Author
Abel Hassoun
  • Function : Author
Brigitte Coatmelec
  • Function : Author
Philippe Beurrier
  • Function : Author
Michèle Damay
  • Function : Author
Philippe Moreau
Odile Pouille-Lievin
  • Function : Author
Caroline Schoepfer
  • Function : Author
Eliane Tarral
  • Function : Author
Monique Bianchin
  • Function : Author
Joël Nguyen
  • Function : Author
Olivier Pincemaille
  • Function : Author
Marie-Odile Peter
  • Function : Author

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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Dates and versions

hal-03653434 , version 1 (27-04-2022)

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