Study of Surgical Practices in Patients With Haemophilia A or B Treated With an Extended Half-life Recombinant Factor VIII-Fc or IX-Fc (ELOCTA®, ALPROLIX®)

NCT06158334 | Unknown

• 1 other identifier: 868
• Study Type: observational
• Target: 400
• Locations: 1 country
• Sites: 1

Brief Summary

Haemophilia A and haemophilia B are inherited bleeding disorders resulting from the absence or deficiency of coagulation factors VIII and IX, respectively. The peri-operative period of people with haemophilia is commonly managed with replacement therapy. In phase 3 studies of Elocta® (extended half-life recombinant factor VIII-Fc) and Alprolix® (extended half-life recombinant factor IX-Fc), haemostatic efficacy was demonstrated to be good or excellent, close to the haemostatic efficacy usually seen in people without haemophilia, with maintenance and stability of circulating FVIII and FIX levels compatible with the surgical procedure, while reducing the frequency of infusions and consumption of therapeutic units. In 2019, the National Protocol for Diagnosis and Care in haemophilia recommended 2 methods for managing patients with haemophilia in the peri-operative period, either discontinuous injections of standard or extended half-life factor VIII/IX or a continuous infusion of FVIII/IX. Many countries, including France, have adopted these rFVIII/IXFc therapeuitic products and recommended their use in the surgical management of patients. However, the use of these two products in real life during surgery in haemophilic A and B patients has not been described in detail. It seems therefore relevant to better document their use in order to progressively specify their use during surgeries with varied bleeding risks.

Conditions

Hemophilia A; Hemophilia B

Keywords

surgery; Haemophilia A; Haemophilia B; Elocta®; Alprolix®

Outcome Measures

Primary Outcomes (1)

• Perioperative haemostatic efficacy described in real life with ELOCTA® and ALPROLIX® in haemophilia A and B patients. Haemostatic efficacy is defined by excellent, good, fair, poor.

  changes in hemostatic efficacy between surgery and 15 days post-surgery

  15 days post-surgery

Study Arms (2)

Minor and major surgeries of patients with haemophilia A treated with Elocta®

Description of all surgical data (minor and major) on Elocta® (all dosage and treatment regimens)

Drug: Describe the respective haemostatic efficacy of Elocta® for haemopilia A during surgical procedures

Minor and major surgeries of patients with haemophilia B treated with Alprolix®

Description of all surgical data (minor and major) on Alprolix® (all dosage and treatment regimens)

Drug: Describe the respective haemostatic efficacy of Alprolix® for haemopilia B during surgical procedures

Interventions

Describe the respective haemostatic efficacy of Elocta® for haemopilia A during surgical procedures DRUG

All surgical data will be collected: surgery, biology, administration of Elocta®, bleeding and transfusion, hemostatic efficacity, surgery complication and bleeding

Minor and major surgeries of patients with haemophilia A treated with Elocta®

Describe the respective haemostatic efficacy of Alprolix® for haemopilia B during surgical procedures DRUG

All surgical data will be collected: surgery, biology, administration of Alprolix®, bleeding and transfusion, hemostatic efficacity, surgery complication and bleeding…

Minor and major surgeries of patients with haemophilia B treated with Alprolix®

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Probability Sample

Study Population

Patients (minor and adults) with Haemophilia A or B for whom minor and major surgeries is performed with Elocta® or Alprolix®.

You may qualify if:

• patient with Haemophilia A or B
• surgery performed with Elocta® or Alprolix®
• Information leaflet given to the patient who as reached the age of majority or to the parents or legal guardian for minors

You may not qualify if:

• Any blood coagulation disorder other than Haemophilia A or B
• Patient with factor VIII or IX inhibitor
• Severe liver disease (serum ALAT/ASAT levels\> 5 x ULN)
• Severe renal disease (serum creatinine \> 2x ULN)
• Known hypersensitivity to the substances or its excipients

Sponsors & Collaborators

• Hospices Civils de Lyon: lead

Study Sites (1)

Centre de Référence en Hémophilie, hôpityal Louis Pradel, GHE- Hospices Civils de Lyon

Bron, France

RECRUITING

MeSH Terms

Conditions

Hemophilia A; Hemophilia B

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, Inherited; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Genetic Diseases, X-Linked

Central Study Contacts

Anne LIEHNART, MD

CONTACT

+33 4 72 11 88 10; anne.lienhart@chu-lyon.fr

Emilie PROME COMBEL, RCA

CONTACT

+33 4 72 11 66 99; emilie.prome@chu-lyon.fr

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: CROSS SECTIONAL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 19, 2023
• First Posted: December 6, 2023
• Study Start: June 1, 2022
• Primary Completion: June 1, 2024
• Study Completion: December 1, 2024
• Last Updated: December 6, 2023

Record last verified: 2023-11

Locations

FR (1)