Advertisement

FranceCoag: a 22-year prospective follow-up of the national French cohort of patients with inherited bleeding disorders

  • Alexandra Doncarli
  • Virginie Demiguel
  • Irina Guseva Canu
  • Véronique Goulet
  • Sophie Bayart
  • Thierry Calvez
  • Sabine Castet
  • Vincent Dalibard
  • Yohan Demay
  • Birgit Frotscher
  • Jenny Goudemand
  • Thierry Lambert
  • Vanessa Milien
  • Caroline Oudot
  • Thomas Sannié
  • Hervé Chambost
  • for the FranceCoag Network
DATA RESOURCES
  • 68 Downloads

Abstract

FranceCoag is an ongoing open prospective multicentre cohort project aimed at improving epidemiological knowledge about inherited bleeding disorders in France. The main objective of this article was to evaluate the project’s progress as of the 30th December 2016. Between 1994 and this date, of the 10,047 patients included in the study, 384 (3.8%) were reported by clinicians to have died and 159 (1.6%) to be lost to follow-up. Among the remaining 9504 patients still being followed up, 5748 (60.5%) had haemophilia A, 1300 (13.7%) haemophilia B, 1980 (20.8%) von Willebrand Disease while 476 (5.0%) had another clotting factor deficiency (Factor I, II, V, combined V and VIII, VII, X, XI and XIII). The median age of the population was 32 years (Inter-quartile range (IQR) 18–50 years) at data extraction on December 30th, 2016. The subgroup of children (i.e., < 18 years old) with severe haemophilia and comprehensive information available since the first exposure to treatment was identified as the PUPs (Previously Untreated Patients) cohort. Data for the 643 children included in the PUPs’ cohort had been collected since their birth. Follow-up data were collected by the clinicians in haemophilia treatment centres (HTC) every 12.9 months on median (IQR 11.4–21.3). In the PUPS cohort, data were updated every 6.2 months on median (IQR 3.7–11.7). A unique patient number assigned at study inclusion was kept at individual HTC by participating clinicians. The data collected included demographic, clinical, therapeutic and biological items on standard electronic forms. As of December 30th 2016, a plasma and serum samples was available for 2581 patients (27.1%).

Keywords

Haemophilia Rare inherited disease Risk factor Inhibitor Prophylaxis 

Notes

HTC collaborators (clinicians and non-clinicians)

Abgraal JF, Adjaoud D, Albinni S*, Ancelet D, Aouba A*, Arab B, Ardillon L, Barbay V, Bariller E, Barro C, Bastenaire B, Bayart S, Beaussant-Cohen S, Behar C*, Belkaïd I, Benz-Lemoine E*, Berger C, Berny K, Bertrand MA, Beurrier P*, Bianchin M*, Biernat J*, Biron-Andreani C, Blanc M*, Bodet L, Borg JY*, Boulfroy E, Bovet J*, Briquel ME*, Brouk Z, Brunot A, Castet S, Chambost H, Chaminade A, Chamouni P, Charbonneau S, Chenuel C, Coatmelec B*, Codine P, Collet B*, Combe S, Dalibard V, De Lumley L*, De Raucourt E, Demay Y, Derlon A, Desprez D, Deville A, Dieval J*, d’Oiron R, Donadel Claeyssens S, Donadio D, Douay J, Drugmanne G, Dumesnil C, Dupont de Romemont C*, Durin-Assollant A*, Dutrillaux F*, Falaise C, Faradji A*, Ferré E, Ferrer AM*, Feugeas O, Fiks Sigaud M, Fimbel B*, Fonlupt J*, Fouassier M, Frenzel L, Fressinaud E*, Frotscher B, Gaboulaud V*, Gaillard S*, Gautier P, Gay V, Gembara P, Girault S, Gleizes E, Goesin I, Gorde S*, Goudemand J, Gourou K*, Grenetier S*, Gruel Y, Guérin V*, Guérois C*, Guezet-Souibri M*, Guillet B, Harroche A, Hassenboehler J, Hassoun A, Haya-Baviera G, Henni T, Henrio C*, Hézard N, Huguenin Y, Lambert E, Lambert T, Lartigue B, Laurian Y*, Lauroua P*, Le Cam Duchez V, Leclere A, Le Guyader M, Le Niger C*, Le François A*, Legrand F*, Lienhart A, Li-Thiao-Te V, Lutz P, Macchi L, Maire C, Marichez C, Marie-Cardine A, Marlu R, Marquès-Verdier A*, Martin M, Matingou M, Mahi Y*, Mercy J, Meunier S, Micheau M*, Milien V, Molho P*, Monlibert B, Monpoux F, Monmartin A, Moreau P, Munzer M*, Navarro R, Négrier C, Nemausat N*, Nguyen P, Normand C, Nyombe P, Oudot C, Ounnoughene N, Palamaringue P, Pan Petesch B, Paris C, Parquet A*, Paugy P*, Pautard B*, Pernod G*, Pertuiset N*, Peter O*, Peynet J, Pierre-Louis S, Pignon* B, Pincemaille O*, Pineau-Vincent F, Play B*, Polack B, Pouille Lievin O*, Pouymayou K, Pouzol P*, Rafowicz A, Ramassamy A, Rauch A, Regina S*, Renom P*, Reynaud J*, Ricard C, Risch J, Robert V, Roche M, Rosay A*,Rospide P*, Rothschild C, Rousseau F, Rugeri L, Sanderson F*, Savary I, Schneider P, Schved JF, Selva J, Sénéchal P, Sicardi F*, Sie P, Soler C*, Stieltjes N, Stoven C, Sultan Y*, Susen S, Tahiri C*, Tamburro M, Tardy B, Tarral E*, Ternisien C, Thiercelin Legrand MF, Thouvenin-Doulet S, Tintillier V*, Torchet MF*, Trillot* N, Tron P*, Trossaërt M, Uettwiller F, Valentin JB, Vanderbecken S, Vannier JP*, Vassilieff D*, Vicariot M*, Voisin S, Volot F, Voyer A, Wibaut B, * Collaborators no longer involved in FranceCoag cohort.

Funding

FranceCoag is supported by French public health authorities.

Compliance with ethical standards

Conflict of interest

The authors declare no potential conflicts of interest related to this description of the FranceCoag cohort.

References

  1. 1.
    Calvez T, Biou M, Costagliola D, Jullien AM, Laurian Y, Rossi F, et al. The French haemophilia cohort: rationale and organization of a long-term national pharmacosurveillance system. Haemophilia. 2001;7(1):82–8.CrossRefGoogle Scholar
  2. 2.
    Gouw SC, van der Bom JG, Ljung R, Escuriola C, Cid AR, Claeyssens-Donadel S, et al. Factor VIII products and inhibitor development in severe hemophilia A. N Engl J Med. 2013;368(3):231–9.  https://doi.org/10.1056/NEJMoa1208024.CrossRefPubMedGoogle Scholar
  3. 3.
    Donadieu J, Rothschild C, Laurian Y, Dieval J, Fressinaud E. Cohorte française des patients atteints de maladies hémorragiques par déficits héréditaires en protéines de la coagulation. Réseau FranceCoag. Données descriptives 2005. Enquête. Saint-Maurice: Institut de Veille Sanitaire; 2006.Google Scholar
  4. 4.
    Doncarli A, Demiguel V, Ghez M, Doussin A, Gautier P, Faradji A, et al. Premier état des lieux du suivi de la population hémophile en France (Cohorte FranceCoag), 1994–2005. Bull Epidemiol Hebd. 2006;39:291–4.Google Scholar
  5. 5.
    Chambost H, Suzan F, for FranceCoag Network g. Epidemiology of hereditary bleeding disorders: input of FranceCoag Network. Arch Pediatr. 2010;17(6):618–9.  https://doi.org/10.1016/s0929-693x(10)70026-9.CrossRefPubMedGoogle Scholar
  6. 6.
    Goudemand J, Pouymayou K, Faradji A, Donadel-Claeyssens S, Rothschild C, Peynet J et al. FranceCoag Network is also the French registry of very rare coagulation bleeding disorders. Abstracts of the 29th International Congress of the World Federation of Hemophilia. Buenos Aires, Argentina. July 10–14, 2010. Haemophilia. 2010;16(Suppl 4):1–158.  https://doi.org/10.1111/j.1365-2516.2010.02283.x.
  7. 7.
    Biron-Andréani C, Milien V, Gay V, Stieltjes N, Tintillier V, Falaise C. Rare bleeding disorders (RBD) in the FranceCoag Network: Diagnosis circumstances and severe events in patients with severe FVII deficiency. Abstracts of the 30th International Congress of the World Federation of Hemophilia. July 8-12, 2012. Paris, France. Haemophilia. 2012;18(Suppl 3):1–226.  https://doi.org/10.1111/j.1365-2516.2012.02820.x.
  8. 8.
    Meunier S, Milien V, Pan Petesch B, Biernat J, Girault S, Cohen W et al. Rare bleeding disorders in the FranceCoag Network: Diagnosis circumstances and severe events in patients with severe FXIII deficiency under 10 per cent. Abstracts of the 30th International Congress of the World Federation of Hemophilia. July 8-12, 2012. Paris, France. Haemophilia. 2012;18(Suppl 3):1–226.  https://doi.org/10.1111/j.1365-2516.2012.02820.x.
  9. 9.
    Volot F, Doncarli A, Bertrand MA, Guerin V, Roussel-Robert V, Pouymayou K et al. Rare bleeding disorders in the FranceCoag Network: Circumstances of diagnosis and clinical manifestations in the French population of patients with FXI deficiency under twenty per cent. Abstracts of the 30th International Congress of the World Federation of Hemophilia. July 8-12, 2012. Paris, France. Haemophilia. 2012;18(Suppl 3):1-226.  https://doi.org/10.1111/j.1365-2516.2012.02820.x.
  10. 10.
    Calvez T, Chambost H, Claeyssens-Donadel S, d’Oiron R, Goulet V, Guillet B, et al. Recombinant factor VIII products and inhibitor development in previously untreated boys with severe hemophilia A. Blood. 2014;124(23):3398–408.  https://doi.org/10.1182/blood-2014-07-586347.CrossRefPubMedGoogle Scholar
  11. 11.
    Stonebraker JS, Bolton-Maggs PH, Soucie JM, Walker I, Brooker M. A study of variations in the reported haemophilia A prevalence around the world. Haemophilia. 2010;16(1):20–32.  https://doi.org/10.1111/j.1365-2516.2009.02127.x.CrossRefPubMedGoogle Scholar
  12. 12.
    Collins PW, Palmer BP, Chalmers EA, Hart DP, Liesner R, Rangarajan S, et al. Factor VIII brand and the incidence of factor VIII inhibitors in previously untreated UK children with severe hemophilia A, 2000-2011. Blood. 2014;124(23):3389–97.  https://doi.org/10.1182/blood-2014-07-580498.CrossRefPubMedPubMedCentralGoogle Scholar
  13. 13.
    Messiaen C, Le Mignot L, Rath A, Richard JB, Dufour E, Ben Said M, et al. CEMARA: a Web dynamic application within a N-tier architecture for rare diseases. Stud Health Technol Inform. 2008;136:51–6.PubMedGoogle Scholar
  14. 14.
    Chambost H, Gaboulaud V, Coatmelec B, Rafowicz A, Schneider P, Calvez T, et al. What factors influence the age at diagnosis of hemophilia? Results of the French hemophilia cohort. J Pediatr. 2002;141(4):548–52.  https://doi.org/10.1067/mpd.2002.128115.CrossRefPubMedGoogle Scholar
  15. 15.
    Jousselme C, Demiguel V, Rafowicz A, Torchet MF, Wibaut B, Meunier S et al. Age at diagnosis of hemophilia: update from the French registry “FranceCoag Network”. Abstracts of the 29th International Congress of the World Federation of Hemophilia. Buenos Aires, Argentina. July 10-14, 2010. Haemophilia. 2010;16(Suppl 4):1–158.  https://doi.org/10.1111/j.1365-2516.2010.02283.x.
  16. 16.
    Réseau FranceCoag. Réseau FranceCoag: la prise en charge des patients atteints d’une maladie hémorragique héréditaire. Le point en 2014. Institut de veille sanitaire, Saint-Maurice. 2015. https://www.francecoag.org/SiteWebPublic/pdfs/Plaquette_03-2015.pdf.
  17. 17.
    Meunier S, Chambost H, Demiguel V, Doncarli A, Suzan F, Trossaert M. Use of clinical practice guidelines on long-term prophylaxis in severe hemophilia in France: a retrospective audit. J Pediatr. 2013;162(6):1241–4.  https://doi.org/10.1016/j.jpeds.2012.11.080.CrossRefPubMedGoogle Scholar
  18. 18.
    Calvez T, Chambost H, d’Oiron R, Dalibard V, Demiguel V, Doncarli A, et al. Analyses of the FranceCoag cohort support differences in immunogenicity among one plasma-derived and two recombinant factor VIII brands in boys with severe hemophilia A. Haematologica. 2018;103(1):179–89.  https://doi.org/10.3324/haematol.2017.174706.CrossRefPubMedPubMedCentralGoogle Scholar
  19. 19.
    Gaboulaud V, Parquet A, Tahiri C, Claeyssens S, Potard V, Faradji A, et al. Prevalence of IgG antibodies to human parvovirus B19 in haemophilia children treated with recombinant factor (F)VIII only or with at least one plasma-derived FVIII or FIX concentrate: results from the French haemophilia cohort. Br J Haematol. 2002;116(2):383–9.CrossRefGoogle Scholar

Copyright information

© Springer Nature B.V. 2018

Authors and Affiliations

  • Alexandra Doncarli
    • 1
  • Virginie Demiguel
    • 1
  • Irina Guseva Canu
    • 1
    • 2
  • Véronique Goulet
    • 1
  • Sophie Bayart
    • 3
  • Thierry Calvez
    • 4
  • Sabine Castet
    • 5
  • Vincent Dalibard
    • 6
  • Yohan Demay
    • 7
  • Birgit Frotscher
    • 8
  • Jenny Goudemand
    • 9
  • Thierry Lambert
    • 10
  • Vanessa Milien
    • 11
  • Caroline Oudot
    • 12
  • Thomas Sannié
    • 13
  • Hervé Chambost
    • 14
  • for the FranceCoag Network
  1. 1.Santé publique France, French national public health agencySaint-MauriceFrance
  2. 2.Institute for Work and Health, University of Lausanne, University of GenevaEpalinges-LausanneSwitzerland
  3. 3.Haemophilia Treatment CentreUniversity Hospital of RennesRennesFrance
  4. 4.Inserm, Sorbonne University, Pierre Louis Institute of Epidemiology and Public HealthParisFrance
  5. 5.Haemophilia Treatment CentrePellegrin HospitalBordeauxFrance
  6. 6.Haematology and Transfusion, School of MedecineLille University HospitalLilleFrance
  7. 7.Haemophilia Treatment Centre, APHPNecker HospitalParisFrance
  8. 8.Haemophilia Treatment CentreBrabois HospitalNancyFrance
  9. 9.Haematology and Transfusion, School of MedicineLille University Hospital, Institut Pasteur de Lille, EGID, Inserm, UMR 1011, Lille 2 UniversityLilleFrance
  10. 10.Haemophilia Treatment Centre, APHPBicêtre HospitalLe Kremlin BicêtreFrance
  11. 11.Haemophilia Treatment Centre, APHMChildren Hospital La TimoneMarseilleFrance
  12. 12.Haemophilia Treatment CentreDupuytren HospitalLimogesFrance
  13. 13.French Haemophilia Association (AFH)ParisFrance
  14. 14.Haemophilia Treatment Centre, APHMChildren Hospital La Timone, Aix Marseille University, INSERM, INRA, C2VNMarseilleFrance

Personalised recommendations