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®) (CHALE)

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

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.

Study Overview

• Status: Recruiting
• Conditions: Hemophilia A; Hemophilia B
• Intervention / Treatment: Drug: Describe the respective haemostatic efficacy of Elocta® for haemopilia A during surgical procedures; Drug: Describe the respective haemostatic efficacy of Alprolix® for haemopilia B during surgical procedures

• Study Type: Observational
• Enrollment (Estimated): 400

Contacts and Locations

• Study Contact: Name: Anne LIEHNART, MD; Phone Number: +33 4 72 11 88 10; Email: anne.lienhart@chu-lyon.fr
• Study Contact Backup: Name: Emilie PROME COMBEL, RCA; Phone Number: +33 4 72 11 66 99; Email: emilie.prome@chu-lyon.fr

Study Locations

• France
  • Bron, France
    • Recruiting
    • Centre de Référence en Hémophilie, hôpityal Louis Pradel, GHE- Hospices Civils de Lyon
    • Contact: Anne LIENHART, MD

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult; Older Adult
• Accepts Healthy Volunteers: No

• 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®.

Description

Inclusion Criteria:

• 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

Exclusion Criteria:

• 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
• patient participating in another clinical trial or having participated in another clinical trial within the previous 30 days (non-interventional studies are not a criterion for non-inclusion)

Study Plan

How is the study designed?

Number of groups / cohorts

2

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
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; 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 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; All surgical data will be collected: surgery, biology, administration of Alprolix®, bleeding and transfusion, hemostatic efficacity, surgery complication and bleeding…

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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

Collaborators and Investigators

• Sponsor: Hospices Civils de Lyon

Study record dates

Study Major Dates

• Study Start (Actual): June 1, 2022
• Primary Completion (Estimated): June 1, 2024
• Study Completion (Estimated): December 1, 2024

Study Registration Dates

• First Submitted: June 19, 2023
• First Submitted That Met QC Criteria: November 28, 2023
• First Posted (Actual): December 6, 2023

Study Record Updates

• Last Update Posted (Actual): December 6, 2023
• Last Update Submitted That Met QC Criteria: November 28, 2023
• Last Verified: November 1, 2023

More Information

Terms related to this study

• Keywords: surgery; Haemophilia A; Haemophilia B; Elocta®; Alprolix®
• Additional Relevant MeSH Terms: Hematologic Diseases; Blood Coagulation Disorders, Inherited; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Genetic Diseases, X-Linked; Blood Coagulation Disorders; Hemophilia A; Hemophilia B; Coagulants; Hemostatics
• Other Study ID Numbers: 868