Clinical trial NCT01568333: A Multi-center Study of Low-dose Decitabine for the Treatment of Immune Thrombocytopenia
Official title: A Multi-center Study of Low-dose Decitabine for the Treatment of Immune Thrombocytopenia
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Immune Thrombocytopenia
Keywords: Decitabine
Immune thrombocytopenia
Lead sponsor: Shandong University
Sponsor class: OTHER
Collaborators: name: Qingdao University
name: Shandong Provincial Hospital
First posted: 2012-04-02
Enrollment: 45
Sex: ALL
Minimum age (years): 18
Maximum age (years): 70
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: Decitabine has been reported to have a clinically significant, often long lasting effect on the platelet count in myelodysplastic syndromes(MDS). It is also reported that decitabine could increase platelet counts by enhancing megakaryocyte maturation and platelet release. Immune thrombocytopenia(ITP) is known as an immune-mediated acquired disease characterized by transient or persistent decrease of the platelet count. However, refractory ITP is lacking of effective treatments and the efficacy of decitabine in ITP remains poorly understood. Data from this study may provide some idea of decitabine in the treatment of ITP.

Detailed description: The investigators are undertaking a multicenter, single-arm study of primary ITP adult patients from 9 medical centers in China. All the participants are selected to receive low-dose of decitabine treatment (given intravenously at a dose of 3.5mg/m2 for 3 days per cycle for 3 cycles). Platelet count, bleeding and other symptoms were evaluated before and after treatment. Adverse events are also recorded throughout the study.

Eligibility criteria: Inclusion Criteria:

* adult patients with the diagnosis of ITP according to the International Working Group (IWG) guidelines
* failure to splenectomy or at least four standard ITP-specific treatments, but not necessarily undergone splenectomye
* baseline peripheral platelet count less than 30,000/uL or the presence of bleeding symptoms
* need of treatment(s) (including, but not limited to, low dose of corticosteroids) to minimize the risk of clinically significant bleeding. Need of on-demand or

Exclusion Criteria:

* secondary ITP
* pregnancy
* hypertension
* cardiovascular disease
* diabetes
* liver and kidney function impairment
* HCV, HIV, HBsAg seropositive status
* patients with systemic lupus erythematosus and/or antiphospholipid syndrome

Primary outcomes:
measure: Platelet count
timeFrame: the third month after the first dose of decitabine
Secondary outcomes:
measure: Bleeding score
timeFrame: at enrollment and the third month after the first dose of decitabine
Other outcomes:
Locations:
city: Jinan
country: China
facility: Shandong University Qilu hospital
state: Shandong
Study officials:
affiliation: Qilu Hospital, Shandong University
name: Ming Hou
role: PRINCIPAL_INVESTIGATOR
References:
citation: van den Bosch J, Lubbert M, Verhoef G, Wijermans PW. The effects of 5-aza-2'-deoxycytidine (Decitabine) on the platelet count in patients with intermediate and high-risk myelodysplastic syndromes. Leuk Res. 2004 Aug;28(8):785-90. doi: 10.1016/j.leukres.2003.11.016.
citation: Wang J, Yi Z, Wang S, Li Z. The effect of decitabine on megakaryocyte maturation and platelet release. Thromb Haemost. 2011 Aug;106(2):337-43. doi: 10.1160/TH10-11-0744. Epub 2011 Jun 28.
citation: Zhou H, Qin P, Liu Q, Yuan C, Hao Y, Zhang H, Wang Z, Ran X, Chu X, Yu W, Wang X, Hou Y, Peng J, Hou M. A prospective, multicenter study of low dose decitabine in adult patients with refractory immune thrombocytopenia. Am J Hematol. 2019 Dec;94(12):1374-1381. doi: 10.1002/ajh.25646. Epub 2019 Oct 17.