Purpura, Thrombocytopenic, Idiopathic
Conditions
Keywords
Resistant/recurrent ITP, MSC-C5b-9 marker, all-trans retinoic acid, Eltrombopag, prospective clinical study
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.
Interventions
DRUGall-trans retinoic acid
ATRA 10 mg, 2 times a day, orally.The treatment course is 12 weeks.
DRUGEltrombopag
The initial dose of eltrombopag is 50 mg/time, once a day, and the dose is increased when the platelet count is lower than 5×109/L, the maximum is 75 mg/d, and the dose is higher than 200×109/L. When the drug is temporarily discontinued, the drug is re-administered according to the platelet count.The treatment course is 12 weeks.
Sponsors
Beijing Friendship Hospital
Beijing Tongren Hospital
Beijing Hospital
Navy General Hospital, Beijing
Peking University People's Hospital
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* 1\. 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.
Exclusion criteria
* 1\. 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.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| 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
| Measure | Time frame | Description |
|---|---|---|
| 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 |
| Inefficiency (NR) | 18 months | Platelet count \<30×10\^9/L, or less than 2-fold increase from baseline or associated with bleeding |
| 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 |
| 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. |
| 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. |
| Time to response (TTR) | 18 months | The time from starting treatment to time of achievement of CR or R |
| 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. |
Countries
China
Outcome results
None listed