Clinical trial NCT06158334: 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®)
Official title: 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®)
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Hemophilia A
Hemophilia B
Keywords: surgery
Haemophilia A
Haemophilia B
Elocta®
Alprolix®
Lead sponsor: Hospices Civils de Lyon
Sponsor class: OTHER
First posted: 2023-12-06
Enrollment: 400
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: CROSS_SECTIONAL
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®.

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.


Eligibility criteria: 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)

Primary outcomes:
measure: 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.
timeFrame: 15 days post-surgery
Secondary outcomes:
Other outcomes:
Locations:
city: Bron
country: France
facility: Centre de Référence en Hémophilie, hôpityal Louis Pradel, GHE- Hospices Civils de Lyon
Study officials:
References: