Real world treatment pathways in patients with immune thrombocytopenia
Research type
Research Study
Full title
Retrospective observational study of real world treatment pathways for patients with newly diagnosed or persistent primary immune thrombocytopenia in the United Kingdom.
IRAS ID
282590
Contact name
Vickie McDonald
Contact email
Sponsor organisation
Amgen
Clinicaltrials.gov Identifier
NA, NA
Duration of Study in the UK
0 years, 9 months, 31 days
Research summary
This is a multi-centre, retrospective observational study of real-world treatment and management pathways for patients with newly diagnosed or persistent primary immune thrombocytopenia (ITP).
The disorder ITP can lead to easy or excessive bruising and bleeding. The bleeding results from unusually low levels of platelets – the cells that help blood to clot. Three distinct phases of ITP have been previously defined. They are: 1) Newly diagnosed ITP (< 3 months after initial diagnosis); 2) Persistent ITP (3 to 12 months after diagnosis), and 3) Chronic ITP (> 12 months after diagnosis).
Treatment options for newly diagnosed ITP include corticosteroids, intravenous immunoglobulins, rituximab, mycophenolate mofetil and splenectomy. Newer treatment options are thrombopoietin receptor agonists (TPO-RA). These tend to be reserved for chronic ITP patients. A TPO-RA example is the agent romiplostim. Several studies have demonstrated its efficacy in chronic ITP patients.
A recent pooled analysis has shown that romiplostim treatment may be effective in maintaining platelet counts, reducing rescue medications and reducing bleeding episodes in both persistent and chronic ITP patients. While several previous studies have focused on patients with chronic ITP receiving one type of TPO-RA treatment, there is a need for more studies documenting the real-world treatment pathways for patients with newly diagnosed/persistent ITP. This study aims to address this knowledge gap.
Retrospective data will be collected from patients’ medical records for the 24-month period following diagnosis of primary ITP. The study population will comprise of two groups:
Group 1: 25-30 patients who received romiplostim during the first 12 months after ITP diagnosis.
Group 2: 70-75 patients who received other ITP treatments (excluding TPO-RA treatments) during the first 12 months after ITP diagnosis.Treatment pathways, healthcare resources, patient’s demographic and clinical characteristics at the time of diagnosis will be analysed. This study will be conducted in 5-10 UK hospitals.
REC name
Yorkshire & The Humber - Leeds East Research Ethics Committee
REC reference
20/YH/0226
Date of REC Opinion
16 Sep 2020
REC opinion
Further Information Favourable Opinion