Single-arm, open-label, single center study to assess the efficacy and safety of terbutaline plus danazol in patients with corticosteroid resistant/relapsed ITP.
Immune thrombocytopenia (ITP) is a severe bleeding disorder. Approximately 2/3 of patients achieve remission from first-line therapies. However, the underlying mechanism of corticosteroid-resistant or relapsed ITP is not well understood; thus, treatment remains a great challenge. β2-AR agonist terbutaline modulates T cell differentiation and effector cell function. A single center prospective study was performed in non-splenectomized ITP patients who were either resistant to a standard dose of corticosteroids or had relapsed. Patients were assigned to terbutaline plus danazol group. Platelet count, bleeding and other symptoms were evaluated before and after treatment. Adverse events are also recorded throughout the study, in order to assess the efficacy and safety of terbutaline plus danazol in patients with corticosteroid-resistant/relapsed ITP.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
40
2.5mg po tid for 12 weeks
200mg po bid for 12 weeks
Peking University Insititute of Hematology, Peking University People's Hospital
Beijing, Beijing Municipality, China
RECRUITINGSustained response
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 6-month follow-up. Interim analysis was scheduled at 50% through recruitment.
Time frame: 6 months
complete remission
The number of participants (responders) with platelet count\>=100x10\^9/L (CR) and the absence of bleeding.
Time frame: 6 months
partial remission
The number of participants (responders) with platelet count \>=30x10\^9/L and at least a 2-fold increase in the baseline count (PR) without the administration of any other platelet increasing therapy.
Time frame: 6 months
time to response
Time to response was defined as the time from starting treatment to the time to achieve the response. Interim analysis
Time frame: 6 months
duration of response
Duration of response was measured from the achievement of response to the loss of response.
Time frame: 6 months
incidence of treatment-emergent adverse events
Adverse events were scaled according to Common Terminology Criteria for Adverse Events (CTCAE) version 5.0.
Time frame: 6 months
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