Single-arm, open-label, single-center study to evaluate the efficacy and safety of baricitinib for the treatment of adults with steroid-resistant/relapse immune thrombocytopenia (ITP).
The investigators are undertaking a prospective trial of 20 adults with ITP in China. Baricitinib is administered as 4 mg po. daily. Safety outcomes and efficacy outcomes are assessed on scheduled study visits (primary endpoint defined as durable response at 6-month follow-up).
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
35
Oral baricitinib was given at a dose of 4 mg daily. The decision to initiate rescue therapy was made after assessment of the extent of bleeding, patient preferences, lifestyle and activity, the complications of specific therapies, comorbidities that predisposed patients to bleeding and the tolerance of side effects. If a platelet count over 300,000/μL was observed for two consecutive tests at least 2 weeks apart, baricitinib treatment was interrupted.
Peking University Insititute of Hematology, Peking University People's Hospital
Beijing, Beijing Municipality, China
RECRUITINGDurable response
The maintenance of a platelet count ≥30,000/μL, 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.
Time frame: 6 months
Complete response (CR)
Complete response (CR) was defined as a platelet count over 100,000/μL and absence of bleeding.
Time frame: 1 month
Response (R)
Response (R) as a platelet count over 30,000/μL and at least 2-fold increase of the baseline count and absence of bleeding.
Time frame: 1 month
Time to response
The time from starting treatment to time of achievement of CR or R.
Time frame: 6 months
Duration of response
Duration of response at 6-month follow up.
Time frame: 6 months
Early response
Achievement of CR or R at day 7
Time frame: 7 days
Initial response
Achievement of CR or R at day 28
Time frame: 28 days
Bleeding events
Clinically significant bleeding as assessed using the world health organization (WHO) bleeding scale.
Time frame: From the start of study treatment (Day 1) to the end of week 24
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Health-related quality of life (HRQoL)
ITP-PAQ is used to assess the Health Related Quality of Life (HRQoL) before and after treatment.
Time frame: From the start of study treatment (Day 1) to the end of week 24
Adverse events
Adverse events (AEs) are reported and graded in accordance with the Common Terminology Criteria for Adverse Events (CTCAE), version 5.0.
Time frame: From the start of study treatment (Day 1) to the end of week 24