Efficacy and Safety of Bortezomib as First-line Treatment of Acquired TTP

Peking Union Medical College Hospital30 enrolled

Overview

To evaluate the efficacy and safety of bortezomib in the first-line treatment of patients with acquired TTP，we design this prospective, multi-center, single-arm interventional study.All enrolled TTP patients were given bortezomib 1.3 mg/m2 intravenous injection d1, 4, 8, on the basis of standard single membrane plasma exchange (2L/d) and hormone therapy (1mg/kg prednisone or equivalent methylprednisolone). 11 (4 doses in total). Bortezomib should be administered immediately after each plasma exchange, and the interval between the next plasma exchange is\> 24h. Plasma exchange continued until the patient's platelet count was \>100×109/L for 2 consecutive days, and then changed to once every other day for a total of two times and then stopped.

Study Type

INTERVENTIONAL

Allocation

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Thrombotic Thrombocytopenic Purpura, Acquired

Eligibility

Sex: ALLMin age: 18 YearsMax age: 80 Years

Medical Language ↔ Plain English

Inclusion Criteria: * clinically diagnosed as TTP (thrombocytopenia + MAHA± clinical evidence of related organ damage， a significant reduction in ADAMTS13 activity level and/or positive antibody screening） * elder than 18 years old; * informed consent is required; Exclusion Criteria: * Uncontrollable systemic infection; * Known allergy to bortezomib; * Expected survival time \<1 week; * Pregnant or lactating women (women of childbearing age have a positive pregnancy test at baseline or have not received a pregnancy test. Postmenopausal women must be at least 12 months after menopause); * If the creatinine level is ≥200μmol/l (1.5mg/dl), the levels of transaminase and bilirubin are 2 times higher than the upper limit of normal (except due to the primary disease); * Known congenital TTP or a clear family history of TTP; * Other diseases that cause microangiopathic hemolysis and thrombocytopenia, such as DIC, APS, HUS, malignant hypertension, transplantation-related microangiopathy; * active malignant tumors (except skin basal cell carcinoma or cervical carcinoma in situ) ( have not been treated or recurred within 5 years before signing the informed consent); * peripheral neuropathy; * Patients or family members cannot understand the conditions and goals of this study; * The investigator believes that the patient should not participate in any other situations in this trial.

Outcomes

Primary Outcomes

Time to clinical remission and the number of plasma exchanges required

clinical remission means clinical symptom remission and/or adamts13 antibody negative，time to clinical remission means the days from first plasma exchange to clinical remission. the number of plasma exchanges required means the whole number of plasma exchanges in this course of TTP.

Time frame: one month

Secondary Outcomes

Mortality rate

The ratio of the number of deaths to the total number of cases

Time frame: 6 months

ICU hospitalization time and total hospitalization time;

ICU hospitalization time means Duration of treatment in the ICU during the course of the disease(the number of days), total time in hospital(the number of days)