Registry for haemophiliacs in France : FranceCoag Network Thierry Lambert, MD Haemophilia Treatment Center,, Bicêtre
History Before 1994: nothing 1994: set up of "Suivi" thérapeutique National des Hémophiles" " (SNH) National prospective multicenter cohort study Anonymous Limited to haemophilia A or B patients Mains objectives : Pharmacovigilance of clotting factor concentrates for haemophiliacs, inhibitor s s research projects. Yearly or visits (definited( calendar), specific blood sampling for lab tests, plasma, serum and cells preservation. Intermediate quarterly visits for PUP'S. Patients or parents signature Supported by French health authorities and coordinated by Inserm (research organism) 100% monitoring of data
Assessment of SNH (Y2000) (1) T Calvez et al, Haemophilia, 2001
Assessment of SNH (Y2000) (2) "Only" 1240 patients enrolled (estimated population: > 4000) Unequal representation of regions Difficulty to proper follow-up Tough data collection and paperwork No use of frozen samples Increasing cost of blood sample processing Other congenital coagulation proteins defects? Need to evolve
Birth of FranceCoag Network Initiated 28 Jan 2003 Two main objectives : 1. Exhaustive knowledge of haemophiliac's population in France (namely: geographic breakdown, associated pathologies, clotting factor concentrates consumption, healthcare system requirements ) and follow-up of patients with congenital bleeding disorders due to clotting factor deficiency 2. Tool for sanitary surveillance of these patients receiving clotting factor concentrates. Research objectives (notably Pups Protocol)
National, prospective multicenter cohort study. Observational study: no specific scheduled visits or blood sampling. No signed consent. Anonymous. Alleviated data collection Optional signed informed consent for serum, plasma and cell preservation every 3 years. Every severe congenital deficiency of clotting factor may be included (except moderate and mild form for haemophilia) Opens a specific cohort for pup's haemophiliacs. Alleviated data monitoring More patients for the same cost Supported by French health authorities and initially coordinated by Inserm,, now by National Institute for Public Health Surveillance (InVS)
Organization Steering committee: InVS members (coordinating( center) Authorities of health representative Head of Regional Treatment Centres physicians COMETH physicians INSERM delegate Agency of sanitary safety representative Haemophilia patient's association representatives Scientific experts Several missions : Propose and decide on evolutions of the project, animate the Network, estimate the research projects, etc. 5 meetings a year Report of each steering committee meeting sent to all participant physicians.
Scientific Aspects Any physician can submit a research project to the steering committee, using data and eventually biological samples of the FranceCoag cohort Project is reviewed by steering committee and other experts if necessary, prior to acceptation 3 international publications at now 3 running studies
Rythm of inclusions and follow-up 3300 update to nov.. 2004 3000 2700 Inclusions ou followed 2400 2100 1800 1500 1200 900 600 Patients included Followed patients 300 0 0 2 4 6 8 10 12 14 16 18 20 22 Time since the starting of project (months)
Inclusions and follow-up up, per year 15 dec 2004 update Evolution of inclusions and follow-up visits 2003 2004 Total Inclusions Hemophilia * Von Willebrand disease Others 2013 157 69 587 82 28 2600 (89 %) 239 (8 %) 97 (3 %) Total 2239 697 2936 Follow-up Hemophilia Von Willebrand disease Others 86 0 1 936 48 30 1022 (93 %) 48 (4 %) 31 (3 %) Total 87 1014 1101
Repartition by pathologies Maladie Nombre de patients inclus (n) (%) Afibrinogénémie 15 0.5 Déficit en Facteur II 0 - Déficit en Facteur V 11 0.4 Déficit en Facteur VII 27 0.9 Déficit en Facteur VIII 2150 73.2 Déficit en Facteur IX 450 15.3 Déficit en Facteur X 6 0.2 Déficit en Facteur XI 24 0.8 Déficit en Facteur XIII 14 0.5 Déficit en Facteur Willebrand 239 8.1 Total Inclusions 2936 100.0
Assessment Good participation of French HTC's (36 centres participating, 90 physicians) Adhesion of patients 194 Pup's included in Pups Protocol Useful tool of communication and information for haemophiliac's physicians Data collection is moving to electronic means We have faith in development of new research projects issued from this registry
How to know more about FranceCoag Network? Visit Web Site : http://www.francecoag.org www.francecoag.org/