Congenital & Immune Thrombotic Thrombocytopenic Purpura Outcomes
Research type
Research Study
Full title
Natural History and Treatment Outcomes of Congenital and Immune Thrombotic Thrombocytopenic Purpura: a Retrospective Chart Review Study
IRAS ID
294709
Contact name
Ping Du
Contact email
Sponsor organisation
Millennium Pharmaceuticals, Inc. (a Takeda company)
Duration of Study in the UK
1 years, 4 months, 0 days
Research summary
This study looks back over the medical records of patients who have been diagnosed with the bleeding disorder Thrombotic Thromocytopenic Purpura (TTP) both immune (acquired/non-inherited) and congenital (inherited) in order to better understand the natural history of the disease in adults and children. Patients with TTP have difficulty forming blood clots because an important chemical in the blood called ADAMTS13 does not work properly, leading to small blood clots in the blood vessels, low platelet count and destruction of red blood cells. Patients with TTP may experience a wide variety of symptoms including fever, fatigue, headache, confusion and bruises or dots on the skin. TTP episodes are serious and life-threatening, approx. 10-20% of acute patients will die despite current treatments.
This study will be carried out in four UK sites, all of which are considered to be highly experienced in treating patients with bleeding disorders; we aim to collect data from the medical records of approximately 22 patients with TTP (7 congenital and 15 immune) who have had a confirmed diagnosis of c/iTTP (in the absence of other bleeding disorders), received their first treatment for an acute episode or they experienced a TTP related event e.g. a stroke within the index period which has been defined as Jan 1st 2009 to Dec 31st 2017. Eligible patients won’t be asked to do anything other than give informed consent for their medical records to be reviewed by their doctor who will then enter information into a secure electronic data capture system; the decision whether or not to participate will have no impact on their treatment.
Data from eligible patient’s medical records will be used to answer a number of research questions e.g. what are the disease related outcomes, how is the disease managed in a real world care setting, what is the burden associated with long term complications?
REC name
London - City & East Research Ethics Committee
REC reference
21/PR/1247
Date of REC Opinion
22 Sep 2021
REC opinion
Favourable Opinion