The Combination of ATRA and Eltrombopag as the Treatment of Steroid-resistant/Relapse ITP Based on MSC-C5b-9
- Conditions
- Purpura, Thrombocytopenic, Idiopathic
- Interventions
- Registration Number
- NCT05438875
- Lead Sponsor
- Peking University People's Hospital
- Brief Summary
A Prospective, Randomized, Open-Label, Multicenter Clinical Trial study to compare the efficacy and safety of ATRA plus eltrombopag compared to eltrombopag monotherapy in the treatment of steroid-resistant/relapsed immune thrombocytopenia (ITP).
- Detailed Description
The investigators are undertaking a parallel group, multicenter, randomized controlled trial of patients with ITP in China. Patients were tested for MSCs, and they were divided into MSC-C5b-9+ group and MSC-C5b-9- group according to the test results, and the two groups were randomized to ATRA + eltrombopag and eltrombopag monotherapy group. Platelet count, bleeding and other symptoms were evaluated before and after treatment. Adverse events are also recorded throughout the study.
Recruitment & Eligibility
- Status
- RECRUITING
- Sex
- All
- Target Recruitment
- 96
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- Isolated thrombocytopenia (platelet count <30 × 109/L); 2. age > 18 years; 3. normal white blood cells and red blood cells on bone marrow examination; 4. increased number of megakaryocytes (bone marrow examination was performed in all patients except for myelofibrosis or other conditions that can cause thrombocytopenia disease); 5. the spleen was normal in size; 6. Eastern Cooperative Oncology Group status score (ECOG score) ≤ 2; 7. ineffective or relapsed after at least 1 course of full-dose full-course hormone therapy; 8. Failure of prior ITP therapy (eg, hormones, splenectomy, and cyclosporine) and at least 4 weeks from enrollment.
-
- Secondary ITP such as drug-related thrombocytopenia; 2. thrombocytopenia due to viral infection (HIV, hepatitis B virus, or hepatitis C virus); 3. severe cardiac, renal, hepatic, or respiratory insufficiency; 4. severe immunodeficiency; 5. pregnancy or lactation; 6. myelodysplasia or Myelofibrosis; 7. history of malignancy; 8. ongoing immunosuppressive therapy for other diseases; 9. patients previously treated with eltrombopag were excluded from this study.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description Eltrombopag Eltrombopag The initial dose of eltrombopag was 50 mg/time, once a day. According to the clinical standard of eltrombopag dose adjustment in the research protocol set by the research group, the dose was increased when the platelet count was lower than 5×109/L. The highest is 75mg/d, if the dose is higher than 200×109/L, the dose is reduced. When the dose is higher than 400×109/L, the drug is temporarily discontinued, and the drug is repeated according to the platelet count. The treatment course is 12 weeks. ATRA and Eltrombopag all-trans retinoic acid ATRA 10 mg, 2 times a day, orally; The initial dose of eltrombopag was 50 mg/time, once a day. According to the clinical standard of eltrombopag dose adjustment in the research protocol set by the research group, the dose was increased when the platelet count was lower than 5×109/L. The maximum dose is 75 mg/d, the dose is reduced if it is higher than 200×109/L, and the drug is temporarily discontinued when it is higher than 400×109/L, and the drug is repeated according to the platelet count. The treatment course is 12 weeks. ATRA and Eltrombopag Eltrombopag ATRA 10 mg, 2 times a day, orally; The initial dose of eltrombopag was 50 mg/time, once a day. According to the clinical standard of eltrombopag dose adjustment in the research protocol set by the research group, the dose was increased when the platelet count was lower than 5×109/L. The maximum dose is 75 mg/d, the dose is reduced if it is higher than 200×109/L, and the drug is temporarily discontinued when it is higher than 400×109/L, and the drug is repeated according to the platelet count. The treatment course is 12 weeks.
- Primary Outcome Measures
Name Time Method Sustained Response Rate (SR) at 18 months 18 months The maintenance of platelet count ≥ 30 x 10\^9/L, at least 2-fold increase of the baseline count, the absence of bleeding, and no need for rescue medication at the 18-month follow-up
- Secondary Outcome Measures
Name Time Method Recurrence rate (relapse) 18 months After the treatment is effective, the platelet count drops below 30×10\^9/L or drops to less than 2 times the basal value, or bleeding symptoms occur
Initial response 1 month Initial response as platelet count more than 30×10\^9/L and at least 2-fold increase of the baseline count and absence of bleeding.
Complete response rate (CR) 18 months The complete response (CR) was defined as platelet count more than 100×10\^9/L and absence of bleeding.
Inefficiency (NR) 18 months Platelet count \<30×10\^9/L, or less than 2-fold increase from baseline or associated with bleeding
Response rate (R) 18 months The Response rate (R) was defined as platelet count more than 30×10\^9/L and more than 2 times higher than baseline, without bleeding
Time to relapse (duration of efficacy) 18 months The time from achievement of CR or R to time of relapse
Early response 1 week Platelet count ≥30×10\^9/L and at least doubling baseline at 1 wk.
Time to response (TTR) 18 months The time from starting treatment to time of achievement of CR or R
Related Research Topics
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Trial Locations
- Locations (5)
Peking University Insititute of Hematology, Peking University People's Hospital
🇨🇳Beijing, Beijing, China
Department of Hematology, Beijing Friendship Hospital, Capital Medical University
🇨🇳Beijing, China
Department of Hematology, Beijing Hospital
🇨🇳Beijing, China
Department of Hematology, Senior Department of Hematology, the Fifth Medical Center of PLA General Hospital
🇨🇳Beijing, China
Beijing Tongren hospital
🇨🇳Beijing, Beijing, China