Efficacy and Safety of Eltrombopag for Refractory Thrombocytopenia Associated With Connective Tissue Disease

NCT06458803 | Completed

• 1 other identifier: TJ-IRB202404119
• Study Type: observational
• Target: 52
• Locations: 0 countries
• Sites: N/A

Brief 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.

Conditions

Refractory Thrombocytopenia; Connective Tissue Diseases

Outcome Measures

Primary Outcomes (1)

• platelet counts of 52 patients from baseline after taking eltrombopag (Therapeutic effects-for refractory CTD-related ITP)

  Platelet counts are measured to determine remission status (CTD-RITP). Partial remission is defined as platelet counts ≥ 50 × 10\^9/L, and complete remission is defined as platelet counts ≥ 100 × 10\^9/L.

  Baseline, post-intervention week 4, post-intervention week 8, post-intervention week 12, post-intervention week 24

Secondary Outcomes (1)

• changes in disease activity index (Systemic Lupus Erythematosus Disease Activity Index 2000 for SLE patients and EULAR Sjögren's Syndrome Disease Activity Index 2000 for pSS patients) from baseline after taking Eltrombopag

  Baseline, post-intervention week 4, post-intervention week 8, post-intervention week 12, post-intervention week 24

Study Arms (1)

El-treatment

patients receive oral eltrombopag at 25 to 75 mg once daily during the course.

Drug: Eltrombopag

Interventions

Eltrombopag DRUG

patients receive oral eltrombopag at 25 to 75 mg once daily according to disease severity，with the treatment of DMARDs at minimal or routine dosages.

Also known as: Promacta

El-treatment

Eligibility Criteria

• Age: 18 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

In this study, a total of 52 CTD patients fulfilling refractory ITP criteria were enrolled, and all received oral eltrombopag at 25 to 75 mg once daily after failing to respond to conventional treatment of corticosteroids for more than one year. The time of follow-up ranged from 1 month to 15 months. 1. Age at diagnosis, median (IQR) 45(34-60) 2. Female, n (%) 43(82.7) 3. SLE, n (%) 28(53.9) 4. SS, n (%) 15(28.9)

You may qualify if:

• 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.

You may not qualify if:

• The patient has a history of lymphoproliferative disease or malignancy of any organ system within the last 5 years;
• The patient has a combination of other autoimmune diseases or malignant haematological diseases;
• Patient's intermittent use of medication and failure to take medication as prescribed;
• Patient refuses to participate in this study.

Sponsors & Collaborators

• Tongji Hospital: lead

MeSH Terms

Conditions

Thrombocytopenia; Connective Tissue Diseases

Interventions

eltrombopag

Condition Hierarchy (Ancestors)

Blood Platelet Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Cytopenia; Skin and Connective Tissue Diseases

Study Officials

• Lingli Dong, MD

  Tongji Hospital

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: observational
• Observational Model: CASE ONLY
• Time Perspective: PROSPECTIVE
• Target Duration: 6 Months
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Professor

Study Record Dates

• First Submitted: June 4, 2024
• First Posted: June 14, 2024
• Study Start: May 6, 2013
• Primary Completion: May 11, 2023
• Study Completion: November 19, 2023
• Last Updated: June 17, 2024

Record last verified: 2024-06