Projects - McMaster

Assessment of Inhibitor Risk and Relevance in Various Risk
Populations

The development of inhibitors has remained as the most dreadful complication of hemophilia treatment, with huge consequences both on patients’ quality of life and on societal costs. We have performed several projects in trying to shed new light on the mechanisms behind inhibitor development and course.

A) Meta-analysis of the evidence about inhibitor development in previously treated patients (PTPs)

This was a project funded within the framework of the European Hemophilia Surveillance Scheme, reported in the Journal of Thrombosis and Haemostasis.
http://www.ncbi.nlm.nih.gov/pubmed/23802542

B) Individual Patient Data Meta-Analysis on the Determinant of Risk Inhibitor Development in Previously Untreated Patients (sumbitted)

This was a collaborative work, sponsored as a working party of the European Association for Hemophilia and Allied Disorders (EAHAD).

C) Investigations of the Determinant of Inhibitor Development in PTPs and Study of Their Clinical Course

This is an ongoing project with two components. The first is a reassessment of all the published cases of inhibitors developed in PTPs (sponsored by CHS within the granting opportunity Care until Cure); the second is the same investigations performed for the inhibitors reported to EUHASS (funded as IIT by Pfizer Canada).

The study is ongoing.

Decision Box (D-Box)

The decision box project aims to create two-sided decision tools - a decision box for the physician and a corresponding patient decision aid - to facilitate shared decision making for treatment decisions in haemophilia. These tools are designed to be used contemporaneously to prepare both parties for a clinical encounter where both parties can come together to make an informed, evidence-based, value-driven decision about a hemophilia treatment decision. Each tool contains information about the decision to be considered, treatment options, and Scientific evidence about the benefits and harms of each option.

Currently, the three areas for which these decision tools have been completed are:
1) Starting prophylaxis treatment in previously untreated patients: when and what dose,
2) Choice of factor concentrate: either recombinant or plasma-derived, and
3) Starting immune tolerance induction for factor inhibitory antibodies: when and what dose.      Decision tools for a fourth area- Starting secondary prophylaxis in previously treated adults-      are being completed.

Patients

Decision Box 1:  
Decision Box 2 :  
Decision Box 3:  

Clinicians

Decision Box 1:  
Decision Box 2:  
Decision Box 3:  

Meta-analysis of Post-Authorization Surveillance Data (PASS)

This is a project aiming at exploiting all the evidence collected with this peculiar study design, with focus on both the specific factor VIII for which we gathered data (ADVATE) and for the analysis method. This project is supported by the manufacturer of Advate (Baxter HealthCare) and by MITACS grants for the learners involved.

Phase I has been completed and reported
http://onlinelibrary.wiley.com/doi/10.1111/hae.12480/pdf

Phase II and III are ongoing, dealing respectively with the analysis of a subpopulation of patients with uncertain risk of inhibitors and on methodological challenges in performing these studies

WAPPS-Hemo (Web-based Application for the Population
Pharmacokinetic Service - Hemophilia)

The WAPPS-Hemo service will empower hemophillia treatment by making it easier to perform individual pharmacokinetic (PK) assessment. The WAPPS-Hemo service will allow for the robust estimation of individual PK parameters with a reduced number of plasma samples. It will also enhance knowledge about the PK of factor VIII and IX.

Website: http://www.wapps-hemo.org

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