Therapeutic Efficacy and Mechanism Study of Retinoid Acid on Immune Thrombocytopenia Patients
Drug: All-trance retinoid acid
|Study Design:||Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Open Label
Primary Purpose: Treatment
|Official Title:||Clinical Study on Therapeutic Efficacy and Mechanism of All-trance-retinoid Acid in Refractory Immune Thrombocytopenia Patients|
- Platelet count [ Time Frame: Every two weeks ] [ Designated as safety issue: Yes ]Platelet count and change in platelet count
- Megakaryocyte count [ Time Frame: Every two weeks ] [ Designated as safety issue: No ]Megakaryocyte count and change in megakaryocyte count
- Megakaryocyte ploidy distribution [ Time Frame: Every two weeks ] [ Designated as safety issue: No ]Megakaryocyte ploidy distribution
- Bleeding episodes [ Time Frame: Every two weeks ] [ Designated as safety issue: Yes ]Number of bleeding episodes and bleeding site
- Number of patients with adverse events [ Time Frame: Every two weeks ] [ Designated as safety issue: No ]
|Study Start Date:||June 2011|
|Estimated Study Completion Date:||June 2013|
|Estimated Primary Completion Date:||June 2013 (Final data collection date for primary outcome measure)|
Experimental: All-trance retinoid acid ＆Danazol
Danazol 200mg tablet by mouth and all-trance retinoid acid 10mg tablet by mouth every 8 hours a day for 12 consecutive weeks.
Drug: All-trance retinoid acid
Other Name: Retinoid acidDrug: Danazol
Active Comparator: Danazol
Danazol 200mg tablet by mouth every 8 hours a day for 12 consecutive weeks.
Refractory immune thrombocytopenia (RITP) is a severe bleeding disorder with a high mortality rate of 10% to 30%, which is resulted from the toxicities associated with therapies, as well as from life-threatening bleedings. Up to now, treatments for RITP patients are still limited and no consensus has been reached about the standard treatment protocol, therefore, it remains a great problem and challenge for hematologists to think out ways to treat RITP, that is to say, new therapies should be explored to maintain a relatively stable and safe platelet counts with minimum toxicities, so as to improve patients' quality of life and reduce mortalities related to severe bleedings and therapies.
Immune thrombocytopenia (ITP) is an autoimmune disorder ascribed not only to increased platelet (PLT) destruction, but also to reduced PLT production, which is appears to be related to impaired megakaryocyte maturation. All-trance retinoid acid (ATRA) belongs to a class of retinoids, which exerts immunomodulatory and differentiation inducing capacities, and has been studied in clinical trails about autoimmune disease and has been successfully applied in clinic to cure APL by inducing the differentiation of cancer cells.
In this study, the investigators retrospectively reviewed the medical chart of 35 RITP patients who take ATRA as part of the combination therapy and have been regularly visiting professor Zhang's clinic, a professional clinic of bleeding and coagulation disorders from February 2008 to August 2012. The investigators made phone calls for some other detailed medically relevant information not recorded, including previous ITP history, bleeding episodes and etc.
Please refer to this study by its ClinicalTrials.gov identifier: NCT01667263
|Peking University People's Hospital, Peking University Insititute of Hematology|
|Beijing, Beijing, China, 100044|
|Principal Investigator:||Xiao-hui Zhang, Professor||Peking University People's Hospital, Peking University Insititute of Hematology|