Immune Tolerance Induction in Haemophilia A Patients Using Wilate or Nuwiq

Octapharma14 enrolled

Overview

Uncontrolled, multi-centre, non-interventional study with a prospective and a retrospective cohort, to evaluate the efficacy of Wilate or Nuwiq in achieving complete or partial immune tolerance induction (ITI) success in severe and moderate haemophilia A patients with inhibitors

Study Type

OBSERVATIONAL

Enrollment

Conditions

Hemophilia A

Interventions

Wilate or NuwiqDRUG

Wilate or Nuwiq administered via intravenous injection

Eligibility

Sex: MALE

Medical Language ↔ Plain English

Inclusion Criteria: * Male patients of any age with moderate or severe haemophilia A. * Patients with a first occurrence of inhibitors, inhibitors refractory to previous ITI attempt(s), or relapsed inhibitors to FVIII, with an inhibitor titre of ≥0.6 BU measured on 2 separate occasions at least 2 weeks apart. * Informed written consent from the patient and/or the patient's parent(s) or legal guardian(s) For patients in the prospective cohort: * Patients who are currently on Wilate or Nuwiq ITI, have just initiated ITI, or are planned to initiate ITI treatment with Wilate or Nuwiq. For patients in the retrospective cohort: * Patients having received Wilate or Nuwiq ITI before entry into this study. Retrospective data will be collected for a maximum of 3 years before enrolment into the study. To be eligible, the following information is needed: * Wilate or Nuwiq treatment details (start date, dose, treatment frequency, and dose change). * Reliably documented bleeding frequency. * FVIII inhibitor titres. * FVIII half-life. * FVIII IVR. Exclusion Criteria: Patients who meet any of the following criteria are not eligible for the study: * Congenital or acquired bleeding disorders other than haemophilia A. * A history of hypersensitivity to blood products and/or plasma-derived FVIII concentrates. * Inability to speak/read English or French well enough to provide consent and adhere to the study. * People who are receiving other non-factor therapies, e.g. concizumab

Locations (3)

Stollery children's hospital, University of Alberta

Edmonton, Alberta, Canada

Children's Hospital of Eastern Ontario

Ottawa, Ontario, Canada

Hamilton Health Science center

Toronto, Ontario, Canada

Outcomes

Primary Outcomes

Number of Moderate and Severe Haemophilia A Patients With Inhibitors Achieving Complete or Partial Immune Tolerance Induction (ITI) Success

ITI success will be determined using predefined success criteria to analyze the proportion of patients achieving complete or partial ITI success. Complete success is defined by achieving all of the following variables: 1) Inhibitor titre \<0.6BU (at least 2 separate blood samplings) assessed using the modified Bethesda assay; 2) Incremental in vivo recovery (IVR) of FVIII in the normal range (≥66% of normal); 3) FVIII half-life ≥6 hours Wilate infusion. Partial success is defined as two of the three criteria being met, whilst partial response is defined as one of the three criteria being met. Partial failure is defined as none of the three criteria are met, but the inhibitor titre has decreased to \<5 BU; complete failure is defined as none of the three criteria are met, and the inhibitor titre is still ≥5 BU.

Time frame: From ITI start until termination of study, a maximum of 2 years

Secondary Outcomes

Time Necessary to Achieve Complete or Partial ITI Success

Time to achieve complete or partial ITI success will be determined using predefined success criteria. Complete success is defined by achieving all of the following variables: 1) Inhibitor titre \<0.6 BU (at least 2 separate blood samplings) assessed using the modified Bethesda assay; 2) Incremental IVR of FVIII in the normal range (≥66% of normal) ; 3) FVIII half-life ≥6 hours. Wilate infusion. Partial success is defined as two of the three criteria being met, whilst partial response is defined as one of the three criteria being met. Partial failure is defined as none of the three criteria are met, but the inhibitor titre has decreased to \<5 BU; complete failure is defined as none of the three criteria are met, and the inhibitor titre is still ≥5 BU. 0 participants analysed for this outcome due to termination of study.