Single-arm, Single-center Clinical Study of Mesenchymal Stem Cells in the Treatment of Refractory Primary Immune Thrombocytopenia
This study is a clinical study initiated by single-arm, single-center, multiple administration researchers. The main purpose of this study is to evaluate the safety and efficacy of mesenchymal stem cells in refractory ITP subjects. The subjects voluntarily signed the informed consent form, and after the evaluation of the screening period and the baseline period, they received 5 times of mesenchymal stem cell infusion in the treatment period, with a dose of 1 × 108/kg mesenchymal stem cells per time, with a frequency of 1 week between the first infusion and the second infusion (a time window of ±1 day), and the 3rd, 4th and 5th infusion were all 2 weeks apart from the last infusion (a time window of ±3 days). After all treatments were completed, the safety and effectiveness of D14, M1, M2, M3, M6, M9 and M12 were evaluated during the follow-up period. The subjects were followed up to M12 or those who met the withdrawal criteria withdrew early, whichever occurred first.
• The subjects voluntarily signed the informed consent form;
• The age is from 4 to 75 years old (including the critical value), male or female;
• Patients clinically diagnosed as ITP (meeting the diagnostic criteria of the Chinese guidelines for the diagnosis and treatment of Primary immune Thrombocytopenia in Adults (2020 Edition)) had persistent thrombocytopenia for more than 3 months, but were ineffective to first-line drugs, second-line drugs for platelet production and rituximab, or ineffective to splenectomy / recurrence after operation.
• Patients who received other maintenance regimens (including but not limited to corticosteroids, azathioprine, danazol or mycophenolate mofetil), but the stable dose had been maintained for at least 4 weeks, and the dose should remain unchanged during the trial period;
• During screening, the liver and kidney function of the subjects met the following criteria: alanine aminotransferase (ALT) and / or aspartate aminotransferase (AST) ≤ 3 times of the upper limit of the normal value; total bilirubin ≤ 1.5 times of the upper limit of the normal value; creatinine ≤ 1.5 times of the upper limit of the normal value or creatinine clearance rate \& gt; 75ml / min;
• Women of childbearing age must have negative blood or urine pregnancy tests and are not breast-feeding, and agree to use medically approved contraceptive measures (such as intrauterine devices, condoms or contraceptive) during the study period.
• Fertile male patients must agree to use barrier contraception or physical abstinence during the study;
• The subjects were able to understand the nature and purpose of the trial, including possible risks and side effects, and to understand the researchers' oral and written medical orders and comply with the requirements of the trial.