Randomized, open-label, multicenter study to compare the efficacy and safety of combination of Sitagliptin and danazol versus danazol for the treatment of adults with steroid-resistant/relapse immune thrombocytopenia (ITP).
The investigators are undertaking a parallel group, multicenter, randomized controlled trial of 120 adults with steroid-resistant/relapse ITP in China. Patients were randomized to Sitagliptin plus danazol and danazol monotherapy group. Platelet count, bleeding and other symptoms were evaluated before and after treatment. Adverse events are also recorded throughout the study.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
120
Oral Sitagliptin (100mg/m2 daily) for 12 weeks. DPP4 inhibitors have anti-inflammatory, immunomodulatory, and hematopoietic effects in addition to their glycemic regulatory effects. In vivo experiments found that endogenous DPP-4 inhibits megakaryopoiesis, and knockout or inhibition of DPP4 in vivo can enhance the recovery of hematopoietic function after stress. The loss of DPP-4 activity in DPP-4-/-mice mice resulted in an expansion of the megakaryocyte progenitor population in vivo, the recovery time of platelets was shorter than in normal mice after platelet depletion with anti-mouse CD41 antibody. Compared with ordinary mice, the number of platelets increased, which provides a theoretical basis for the use of DPP-4 inhibitors in patients with ITP, and has potential clinical significance.
Oral danazol (200 mg twice daily) 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.
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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