French national reference center for hemophilia and other rare coagulation protein deficiencies
The reference center for hemophilia and other rare coagulation protein deficiencies works to improve care and quality of life for patients with hemophilia or other rare coagulation deficiencies. It is particularly concerned with the management of hemophilia A (factor VIII deficiency) or B (factor IX deficiency), but also other even rarer diseases affecting coagulation.
The Necker center site is staffed by a multidisciplinary team (hematologists, rheumatologist, pediatrician, nurse, physiotherapist, psychologist, social worker) specifically trained in the management of hemorrhagic diseases from birth to adulthood. It provides consultations (pediatric and adult), hospitalization and therapeutic education sessions. It is particularly involved in the management of the frequent joint complications of these hemorrhagic diseases, with a dedicated fundamental research unit at the Imagine Institute. With the clinical research team at Necker’s adult hematology department, it is involved in numerous studies offering patients access to innovative drugs such as gene therapy or mesenchymal stem cells. It also contributes to the training of medical, pharmacy and nursing students at the Université Paris Cité.
Through the Association Française des Hémophiles (AFH), it also runs educational programs for the hemophilia community. It also organizes bi-annual humanitarian missions to Guinea Conakry to bring the expertise of our center and the support of the World Federation of Hemophilia (WFH) to more effectively treat Guinean hemophilia patients.
This reference center is affiliated with the MHEMO rare diseases healthcare network and the EuroBloodNet European reference networks (ERN).
Medical
team
Dr Laurent Frenzel
MD, PhD
Contact us
Talk to someone or make an appointment
Phone. +33 1 44 49 52 73
Recommendations
Provide the reason for the consultation (first time or follow-up), together with a letter from the doctor and the biological tests.
In case of emergency
- Hemophilia A
- Hemophilia B
- Congenital vitamin K-dependent coagulation factors deficiency
- Congenital factor V deficiency
- Congenital factor XI deficiency
- Congenital factor XIII deficiency
- Congenital fibrinogen deficiency
- Combined deficiency of factor V and factor VIII
- Hereditary combined deficiency of vitamin K-dependent clotting factors
- Rare hemorrhagic disorder due to an acquired coagulation factor defect
- Rare hemorrhagic disorder due to a constitutional coagulation factors defect
- Rare thrombotic disorder due to a constitutional coagulation factors defect
- Dr Laurent Frenzel, rheumatologist/hematologist
- Dr Annie Harroche, pediatrician
- Dr Cécile Bally, hematologist
- Malika Makhloufi, coordinating nurse
- Sophie Jacquet, day hospital nurse
- Nadine Dujardin, medical secretary
- Saïda Bouighamdane, medical secretary
- Rafik Bouali, clinical research coordinator
- Alya Bouhaouche, clinical research associate
- Rym Bouhaouche, clinical research associate
- Yohan Demay, clinical research associate
- Nawel Zegrour, clinical research associate
- Ariane Le Guay, psychologist
- Laurence Boismaigre, social worker
- Nathalie Lherisson, health manager
- Pr Olivier Hermine, head of department
- A-Move une étude évaluant l’impact d’un examen articulaire systématique par échographie, exploration fonctionnelle et examen clinique sur la prise en charge de patients souffrant d’hémophilie A
- ATLAS-A/B: A Phase 3 Study to Evaluate the Efficacy and Safety of Fitusiran in Patients With Hemophilia A or B, Without Inhibitory Antibodies to Factor VIII or IX
- ATLAS-OLE: An Open-label, Long-term Safety and Efficacy Study of Fitusiran in Patients with Hemophilia A or B, with or without Inhibitory Antibodies to Factor VIII or IX
- ATLAS-Neo : A Phase 3, single-arm, multicenter, multinational, open-label, one-way crossover study to investigate the efficacy and safety of fitusiran prophylaxis in male participants aged ≥12 years with severe hemophilia A or B, with or without inhibitory antibodies to factor VIII or IX
- AP-0105 : A Global, Non-interventional Study to Prospectively Evaluate Bleeding Episodes and Treatment Use in Patients with Hemophilia
- AP-0102 : A Global, Open-label, Adaptive Design Study to Investigate the Efficacy and Safety of SerpinPC in Subjects With Severe Hemophilia A or Moderately Severe to Severe Hemophilia B
- AP-0103 : A Global, Open-label, Adaptive Design Study to Investigate the Efficacy and Safety of SerpinPC in Subjects With Severe Hemophilia B with inhibitor
- B-Sure : A 24-month prospective, non-interventional, multicentre study to evaluate the real-world effectiveness and usage of Alprolix in patients with haemophilia B in France
- C0371002 HB (Bene-Gene2) : Therapie Génique/ Phase 3, open label, single arm study to evaluate efficacy and safety of FIX gene transfer with PF-06838435 (rAAV-Spark100-hFIX-Padua) in adult male participants with moderately severe to severe hemophilia B (FIX:C£2%) (BeneGene-2)
- C0371004 : Open label, lead-in study to evaluate at least 6 months of prosepective efficacy and selected safety data of current (FIX) prophylaxis replacement therapy in the usual care setting of modertely severe to severe adulte HB (FIX:c <2%
- C3731003/Gene therapy : Phase 3, Open-Label, Single-Arm Study to Evaluate the Efficacy and Safety of PF-07055480 (Recombinant AAV2/6 Human Factor VIII Gene Therapy) in Adult Male Participants with Moderately Severe to Severe Hemophilia A (FVIII:C≤1%)
- EXPLORER10 : Étude en ouvert évaluant l’efficacité, la sécurité et la pharmacocinétique de concizumab en prophylaxie chez des enfants âgés de moins de 12 ans, hémophiles A ou B avec ou sans inhibiteurs
- Explorer 6 : A prospective, multi-national, non-interventional study in haemophilia A and B patients with or without inhibitors treated according to routine clinical treatment practice
- Freeline/FLT01/Eclipse « Protocole de sélection et d’observation préliminaire visant à déterminer l’éligibilité potentielle des patients en vue d’essais cliniques de thérapie génique utilisant un AAV dans l’hémophilie B (ECLIPSE) ».
- Get8-Bayer-19429 A phase 1/2 open-label safety and dose-finding study of BAY 2599023 (DTX201), an adeno-associated virus (AAV) hu37-mediated gene transfer of B-domain deleted human factor VIII, in adults with severe hemophilia A
- Haven3 : A randomized, multicenter, open-label, phase iii clinical trial to evaluate the efficacy, safety, and pharmacokinetics of prophylactic emicizumab versus no prophylaxis in hemophilia a patients without inhibitors
- Haven6 : A multicenter, open-label study to evaluate the safety, efficacy, pharmacokinetics, and pharmacodynamics of emicizumab in patients with mild or moderate hemophilia a without fviii inhibitors
- Haven7 : A Phase IIIb, multicenter, open-label, single-arm study to evaluate the efficacy, safety, pharmacokinetics, and pharmacodynamics of subcutaneous emicizumab in patients from birth to 12 months of age with hemophilia a without inhibitors ».
- B7841005/Basis : An Open-Label Study in Adolescent and Adult Severe (Coagulation Factor Activity <1%) Hemophilia A or B Patients With or Without Inhibitors Comparing Standard Treatment to PF-06741086 Prophylaxis
- B7841007/Basis-OLE : An open-label extension study to evaluate the long-term safety, tolerability, and efficacy of marstacimab prophylaxis in severe (coagulation factor activity <1%) hemophilia a participants with or without inhibitors or moderately severe to severe hemophilia b participants (coagulation factor activity ≤2%) with or without inhibitors
- B7841008/Basis Kids : An open-label study in pediatric (<18 years of age), severe hemophilia a participants (coagulation factor activity <1%) with or without inhibitors or moderately severe to severe hemophilia b participants (coagulation factor activity =2%) with or without inhibitors comparing 12 months of historical standard treatment to marstacimab prophylaxis
- SPK8011-301 : An Open-label, Non-investigational Product, Multi-center, Lead-in Study to Evaluate Prospective Bleeding and Infusion Data of Current FVIII Replacement Therapy in Adult Males with Hemophilia A
- PHILEOS : Hémophilie et perte osseuse
- FranceCoag : Cohorte prospective multi-centrique des patients vivant avec une maladie hemorragique constitutionnelle (mhc)
- SENIORHEMO : État de santé objectif et perçu des séniors atteints d’hémophilie modérée ou sévère en France Une étude ancillaire de la cohorte FranceCoag (SENIORHEMO)
- Gena25 : Utilisation dans la pratique des concentrés de FVIII d’Octapharma chez des patients atteints d’hémophilie A naïfs de traitement ou ayant reçu un traitement minimum et qui commencent un traitement clinique de routine (par Nuwiq, Octanate ou Wilat
- EFC16295 : Etude de Phase 3 en ouvert, multicentrique, portant sur la sécurité d’emploi, l’efficacité et la pharmacocinétique du Facteur VIII de coagulation recombinant fusionné au fragment Fc – Facteur von Willebrand – Protéine de fusion XTEN (rFVIIIFc-VWF-XTEN ; BIVV001) par voie intraveineuse chez des patients pédiatriques âgés de moins de 12 ans atteints d’hémophilie A sévère et ayant déjà été traités
- R131L1265-HEMB-2318 BEYOND-9 : A two-part open-label study of regv131-lnp1265, a crispr/cas9-based coagulation factor ix gene insertion therapy in participants with hemophilia b
- CSL222_4001 : An Observational Post-authorization Long-term Follow-up Study to Characterize the Effectiveness and Safety of HEMGENIX® (Etranacogene Dezaparvovec) in Patients with Hemophilia B
- HMB-001-CL101 : A Phase 1/2, First-in-Human, Single and Multiple Ascending Dose Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of HMB-001 in Participants with Glanzmann Thrombasthenia
- Orphée : Etude non interventionnelle, prospective et nationale dont l’objectif est d’enregistrer des données en vie réelle chez des patients français hémophiles B traités par Idelvion® afin de confirmer l’efficacité et la sécurité de ce produit établies dans des études de développement clinique
- OPALE : Description de l’évolution à long terme des patients atteints de la maladie de von Willebrand et traités par Voncento® et de l’efficacité hémostatique dans la prévention et le traitement des épisodes hémorragiques non chirurgicaux et la prévention des saignements chirurgicaux pendant 2 ans après l’inclusion des patients.
- ONE-TO-ONE : MOdalities of use, effectiveNEss and TOlerability of Eqwilate® a balanced combInatiON of VWF and FVIII in von WillEbrand patients in real-life conditions: the ONE-TO-ONE study
- Clinical hemostasis IUD
Lyon university - Pediatric rheumatism IUD
University of Paris - Odontology IUD
University of Paris
2024
– Emicizumab and asparaginase, A first experience to share.
Goubeau L, Bally C, Borgel D, Alby-Laurent F, Petit A, Frenzel L, Lasne D, Harroche A.,
Haemophilia. 2024 Mar
– Emicizumab use in females with moderate or mild hemophilia A without factor VIII inhibitors who warrant prophylaxis.
Hermans C, Ventriglia G, Obaji S, Beckermann BM, Lehle M, Catalani O, d’Oiron R, Frenzel L.
Res Pract Thromb Haemost. 2023 Oct 21
– The effects of time and temperature on the in vitro stability of emicizumab in blood samples: Implications for laboratory and clinical practice.
Bentounes NK, Lasne D, Habay C, Bally C, Frenzel L, Harroche A, Borgel D, Auditeau C.
Haemophilia. 2024 Mar
– Gene therapy for haemophilia A and B, from basic principles to clinical implementation: An illustrated review.
Ay C, Frenzel L, Pinachyan K, Le Quellec S.
Haemophilia. 2024 Jan
– Venous thromboembolism prophylaxis and multiple myeloma patients in real-life: Results of a large survey and clinical guidance recommendations from the IFM group.
Frenzel L, Decaux O, Macro M, Belhadj-Merzoug K, Manier S, Touzeau C, Leleu X, Frère C, Lecompte T, Perrot A, Avet-Loiseau H, Moreau P, Chalayer E.
Thromb Res. 2024 Jan
2023
– Interferences by factor VIII and lupus anticoagulant in the modified one-stage assay for emicizumab.
Habay C, Auditeau C, Blandinières A, Bentounes NK, Lavenu-Bombled C, Harroche A, Bally C, Frenzel L, Borgel D, Lasne D.
Haemophilia. 2023 Nov
– Matching-adjusted indirect comparison of bleeding outcomes in severe haemophilia A: Comparing valoctocogene roxaparvovec gene therapy, emicizumab prophylaxis, and FVIII replacement prophylaxis.
Astermark J, Buckner TW, Frenzel L, Hatswell AJ, You X, Liu H, Goodman E, Santos S, Hawes C, Hinds D, Klamroth R.
Haemophilia. 2023 Jul
– Fitusiran prophylaxis in people with severe haemophilia A or haemophilia B without inhibitors (ATLAS-A/B): a multicentre, open-label, randomised, phase 3 trial.
Srivastava A, Rangarajan S, Kavakli K, Klamroth R, Kenet G, Khoo L, You CW, Xu W, Malan N, Frenzel L, Bagot CN, Stasyshyn O, Chang CY, Poloskey S, Qiu Z, Andersson S, Mei B, Pipe SW.
Lancet Haematol. 2023 May
– How to translate and implement the current science of gene therapy into haemophilia care ?
Hermans C, Gruel Y, Frenzel L, Krumb E.
Ther Adv Hematol. 2023 Jan 12
2022
– Emicizumab does not interfere with the activated clotting time.
Capdevila L, Frère C, Desvages M, Harroche A, Bally C, Abbes A, d’Oiron R, Frenzel L, Borgel D, Lasne D.
Haemophilia. 2022 Mar
2021
– Non-inhibitory antibodies inducing increased emicizumab clearance in a severe haemophilia A inhibitor patient.
Harroche A, Sefiane T, Desvages M, Borgel D, Lasne D, Casari C, Peyron I, Frenzel L, Chhun S, Lenting PJ, Bally C.
Haematologica. 2021 Aug 1
– Intraperitoneal bleeding as the initial presentation of Randall-type monoclonal immunoglobulin deposition disease with hepatic involvement.
Gkalea V, Amiot X, Jo Molina T, Frenzel L, Bachmeyer C.
Leuk Lymphoma. 2021 May
2020
– Unique inflammatory signature in haemophilic arthropathy: miRNA changes due to interaction between blood and fibroblast-like synoviocytes.
Mignot S, Cagnard N, Albaud B, Bally C, Siavellis J, Hermine O, Frenzel L.
J Cell Mol Med. 2020 Dec
Living with hemophilia
Hemophilia in Africa
Hemophilia and joint complications
Contact information
Necker-Enfants malades university hospital
> Adult hematology department
Hemophilia and hemorrhagic diseases treatment center
Adults and children
149 rue de Sèvres
75743 PARIS Cedex 15
> Welcome booklet – Adult patients
In Necker, the reference center for hemophilia and other rare coagulation protein deficiencies site in brief …
* data valid for 2022