Efficacy and Safety of Eltrombopag for Refractory Thrombocytopenia Associated With Connective Tis… (NCT06458803) | Clinical Trial Compass
CompletedNot Applicable
Efficacy and Safety of Eltrombopag for Refractory Thrombocytopenia Associated With Connective Tissue Disease
52 participantsStarted 2013-05-06
Plain-language summary
Immune thrombocytopenia (ITP) is a haemorrhagic disorder often associated with CTD. Corticosteroids are the first-line treatment for CTD-associated thrombocytopenia, but not all patients respond well. Eltrombopag is an oral, small molecule thrombopoietin receptor agonist. It interacts with the transmembrane domain of the thrombopoietin receptor and stimulates platelet production. This study is designed to evaluate the efficacy and safety of eltrombopag in patients with refractory CTD-ITP. It is a single-centre, retrospective, observational study involving a cohort of 52 patients diagnosed with CTD-RITP who received eltrombopag between 2013 and 2023. Follow-up data will be systematically collected and analysed to evaluate the therapeutic efficacy and safety of the drug. The study will provide valuable insight into the benefit of eltrombopag in CTD-RITP by reviewing baseline characteristics and performing subsequent clinical assessments to determine drug response and adverse events.
Who can participate
Age range
18 Years – 75 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion criteria
. The patient is diagnosed with CTD, SLE, SS, APS, MCTD, ANCA or RA according to international diagnostic criteria;
. The patient meets the diagnostic criteria of RITP: (i) glucocorticoid therapy and/or splenectomy is ineffective; (ii) age ≥18 years; (iii) duration of the disease \>6 months; (iv) no other diseases leading to thrombocytopenia; (v) platelet count ≤30×109/L;
. At the time of enrolment, patients have received ineffective conventional first-line treatment for one year or more;
. Enrolled patients need to be aware of the clinical information they are being given.
Exclusion criteria
. The patient has a history of lymphoproliferative disease or malignancy of any organ system within the last 5 years;
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
platelet counts of 52 patients from baseline after taking eltrombopag (Therapeutic effects-for refractory CTD-related ITP)