Current first line treatments for immune thrombocytopenia (ITP) usually have transient effects and prolonged platelet response rate off therapy remains low. The aim is to evaluate whether a 12-week course of eltrombopag plus pulsed dexamethasone as first line therapy can increase the proportion of patients with prolonged response. Diagnosis of ITP is established according to the American Society of Hematology guidelines. Eligible ITP subjects have platelet counts \<30×109/L or counts \<50×109/L and significant bleeding symptoms (WHO bleeding scale 2 or above). Subjects must have no prior ITP treatment except platelet transfusions. Treatment consists of eltrombopag 25-75 mg daily according to platelet response for 12 weeks plus pulsed dexamethasone, 40 mg daily for 4 consecutive days every 4 weeks for 1-3 courses. The primary endpoint is prolonged response rate which was defined as the proportion of enrolled subjects maintaining platelet counts \>50×109/L for more than 6 months without any ITP therapy after completion of 12-week therapy.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
53
Eltrombopag Oral Tablet 25-75 mg daily for 12 weeks plus pulsed dexamethasone
Humanity & Health Research Centre
Hong Kong, Hong Kong SAR, Hong Kong
The prolonged response rate
Defined as proportion of patients with platelet counts \> 50,000/μl at 6 months after completion of therapy and free of ITP rescue therapy
Time frame: 6 months after completion of therapy
Time to relapse
Defined as the interval from completion of 3 months treatment to platelet count\<30,000/ul and restarting of ITP therapy including but not limited to platelet transfusion, IVIG, corticosteroids, immunosuppressive drugs, rituximab
Time frame: 6 months after completion of therapy
Early response rate
Defined as proportion of patients requiring less than 3 courses of pulsed dexamethasone to maintain platelet counts \> 150,000/ul
Time frame: 6 months after completion of therapy
Health related quality of life
Assessed by SV36 HRQoL questionnaires
Time frame: 6 months after completion of therapy
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