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 de Saint-Etienne - Centre Hospitalier Universitaire de Saint-Etienne
6 CUMR Caen - Centre Universitaire des Maladies Rénales [CHU Caen]
7 CHU Toulouse - Centre Hospitalier Universitaire de 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
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 - Centre Hospitalier Universitaire de 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
Paul Saultier
- Function : Author
- PersonId : 780499
- ORCID : 0000-0002-6327-0898
Virginie Demiguel
- Function : Author
- PersonId : 794315
- ORCID : 0000-0002-2677-6259
Claire Berger
- Function : Author
- PersonId : 757697
- ORCID : 0000-0001-9875-5150
Sandrine Meunier
- Function : Author
- PersonId : 800777
- ORCID : 0000-0001-6785-4332
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
Marie Françoise Thiercelin-Legrand
- Function : Author
Marianne Fiks-Sigaud
- Function : Author
Marc Fouassier
- Function : Author
Edith Fressinaud
- Function : Author
Sophie Voisin
- Function : Author
- PersonId : 801291
- ORCID : 0000-0002-2871-166X
- IdRef : 114514712
Albert Faradji
- Function : Author
Patrick Lutz
- Function : Author
Marie Elisabeth Briquel
- Function : Author
Birgit Frotscher
- Function : Author
Béatrice Fimbel
- Function : Author
Yves Gruel
- Function : Author
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
- Function : Author
- PersonId : 759214
- ORCID : 0000-0003-1780-8746
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.