"OPTI-CLOT" goes United Kingdom (UK)
Research type
Research Study
Full title
“OPTI-CLOT” goes United Kingdom (UK): A retrospective multi-centre study on perioperative clotting factor IX consumption in Haemophilia: Exploring the effects of pharmacokinetic profiling
IRAS ID
154208
Contact name
R Liesner
Contact email
Sponsor organisation
Erasmus University Medical Center - Sophia Children's Hospital Rotterdam
Research summary
Haemophilia is a bleeding disorder caused by a deficiency of clotting factor VIII (haemophilia A) or clotting factor IX (haemophilia B) in plasma. The overall aim of the “OPTI-CLOT” research programme is to implement pharmacokinetic (PK)-guided dosing of clotting factor replacement therapy to treat people with clotting disorders such as haemophilia. Both in the context of prophylaxis and perioperative replacement therapy to prevent bleeding, as well as on demand treatment of bleeding. Data on clotting factor consumption and corresponding clotting factor levels are currently being collected retrospectively from large, multicentre cohorts of patients with various clotting disorders, such as haemophilia and used to build relevant population PK models. This information and the constructed PK population models will then be used to design prospective, hypothesis driven studies.
In this study, the aim is to strengthen a recently constructed, preliminary, perioperative clotting factor IX (FIX) population PK model based on Dutch data (ASPIRE award; 2012) by collecting historical perioperative data from haemophilia B patients from the United Kingdom (UK). These data will refine the current model, constructed by Bayesian analysis (NONMEM®), making it fit for clinical applications. Differences in PK between plasma derived FIX (pdFIX) and recombinant FIX (rFIX) will be taken into account.
In this multi-centre, retrospective cohort study in peri-operative haemophilia B patients we aim to:
1. To evaluate perioperative clotting factor management in haemophilia B patients.
2. To further develop and strengthen the current peri-operative FIX population PK model by adding UK patient data.Multi-centre studies focusing on PK-guided dosing in haemophilia in a variety of real life clinical situations, in combination with future prospective randomized controlled trials, are essential to implement future PK-guided dosing based on PK population models with patient-tailored, optimized dosing for all haemophilia patients as a consequence.
REC name
South Central - Berkshire Research Ethics Committee
REC reference
15/SC/0367
Date of REC Opinion
17 Jun 2015
REC opinion
Further Information Favourable Opinion