This is a prospective, multicenter, single-arm, pilot study. The aim of this study is to evaluate the efficacy and safety of Linperlisib, the PI3K delta inhibitor for autoimmune hemolytic anemia patients who failed the second line therapy.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
4
The phosphatidylinositol 3-kinase delta (PI3Kδ) signaling pathway plays a critical role in the activation, proliferation, and tissue homing of self-reactive B cells that contribute to autoimmune diseases. B cells play an essential role in immune system function and dysfunction (e.g., autoimmunity) by producing antibodies and by acting as antigen-presenting cells (APCs) for T cells. Signaling via PI3K controls many essential B cell functions and is therefore a promising target for preventing aberrant B cell activation.
Zhoukou Central Hospital
Zhoukou, Henan, China
Regenerative Medicine Center
Tianjin, Tianjin Municipality, China
Overall response rate
Percentage of patients with hematological response. Hematological response is evaluated by hemoglobin and other hemolysis-related laboratory indicators.
Time frame: 6-12 weeks
Incidence of the adverse event
Use Common Terminology Criteria for Adverse Events (CTCAE) Version 5 to assess the adverse event.
Time frame: 12 weeks
Complete response rate
Percentage of patients with hematological complete response. Hematological response is evaluated by hemoglobin and other hemolysis-related laboratory indicators.
Time frame: 6-12 weeks
Complete response with incomplete hemolysis recovery, CRi
Percentage of patients with CRi which is evaluated by hemoglobin and other hemolysis-related laboratory indicators.
Time frame: 6-12 weeks
Mean change from baseline in hemoglobin (Hgb) levels
Time frame: 6-12 weeks
Time to achieve partial response (PR)
Duration time was calculated from enrollment to PR. PR is assessed by hemoglobin and blood transfusion.
Time frame: 6-12 weeks
Time to achieve complete response (CR)
Duration time was calculated from enrollment to CR.
Time frame: 6-12 weeks
Change of the health-related quality of life
Medical Outcomes Study Questionnaire Short Form 36 Health Survey (SF-36) is used to assess the health-related quality of life of patients. The SF-36 has eight scaled scores; the scores are weighted sums of the questions in each section. Scores range from 0 - 100. Lower scores = more disability, higher scores = less disability
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Time frame: Baseline and 12 weeks