Clinical trial NCT03033199: Clinical Trial of Gut Microbiota in the Management of Immune Thrombocytopenia
Official title: A Multi-center Randomized Clinical Trial of Regulating Gut Microbiota by Probiotic Agents in Management of Immune Thrombocytopenia
Study type: INTERVENTIONAL
Overall status: UNKNOWN
Phase: PHASE2
Conditions: Immune Thrombocytopenia
Keywords: Gut microbiota
Immune thrombocytopenia
Lead sponsor: Shandong University
Sponsor class: OTHER
Collaborators: name: Jinan Central Hospital
name: The Second Hospital of Shandong University
name: Qianfoshan Hospital
name: Qingdao University
name: Yantai Yuhuangding Hospital
First posted: 2017-01-26
Enrollment: 60
Sex: ALL
Minimum age (years): 18
Maximum age (years): 70
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: Primary immune thrombocytopenia (ITP) is an acquired autoimmune bleeding disorder, accounting for about 1/3 of clinical hemorrhagic diseases. Loss of immune tolerance leading to increased platelet destruction and decreased platelet production is the main pathogenesis of ITP. Dysbiosis of the gut microbiota was found in many autoimmune diseases like rheumatic arthritis(RA),inflammatory bowel disease(IBD),multiple sclerosis and probiotic treatment or fecal microbiota transplantation(FMT) which can regulate the gut microbiota has good clinical efficacy in those disorders. One ITP patient with ulcerative colitis(UC) was treated with FMT and got progressive but significant increase in platelet level and lasted for several years.

Detailed description: The investigators are undertaking a multicenter, single-arm study of 60 primary ITP adult patients from 5 medical centers in China. All the participants are randomly divided into two groups to orally intake either probiotic supplements (n = 30) or placebo (n = 30) for 4 weeks in addition with high dose dexamethasone(40 mg/d for 4 days). Platelet count, bleeding and other symptoms were evaluated before and after treatment. Adverse events are also recorded throughout the study.

Eligibility criteria: Inclusion Criteria:

* 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 adjunctive therapy alone does not qualify the patient as refractory
* primary ITP confirmed by excluding other supervened causes of thrombocytopenia

Exclusion Criteria:

* pregnancy
* hypertension
* cardiovascular disease
* diabetes
* liver and kidney function impairment
* HCV, HIV, HBsAg seropositive status
* patients with systemic lupus erythematosus and/or antiphospholipid syndrome
* patients with known gastro-intestinal bleeding.
* use of antibiotics, prebiotics or probiotics in the past 4 weeks;

Primary outcomes:
measure: Platelet count
timeFrame: From date of randomization until the date of first documented progression,up to 12 months
Secondary outcomes:
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: Kang Y, Cai Y, Zhang X, Kong X, Su J. Altered gut microbiota in RA: implications for treatment. Z Rheumatol. 2017 Jun;76(5):451-457. doi: 10.1007/s00393-016-0237-5.
citation: Vaghef-Mehrabany E, Alipour B, Homayouni-Rad A, Sharif SK, Asghari-Jafarabadi M, Zavvari S. Probiotic supplementation improves inflammatory status in patients with rheumatoid arthritis. Nutrition. 2014 Apr;30(4):430-5. doi: 10.1016/j.nut.2013.09.007. Epub 2013 Dec 17.
citation: Smits LP, Bouter KE, de Vos WM, Borody TJ, Nieuwdorp M. Therapeutic potential of fecal microbiota transplantation. Gastroenterology. 2013 Nov;145(5):946-53. doi: 10.1053/j.gastro.2013.08.058. Epub 2013 Sep 7.
citation: Zamani B, Golkar HR, Farshbaf S, Emadi-Baygi M, Tajabadi-Ebrahimi M, Jafari P, Akhavan R, Taghizadeh M, Memarzadeh MR, Asemi Z. Clinical and metabolic response to probiotic supplementation in patients with rheumatoid arthritis: a randomized, double-blind, placebo-controlled trial. Int J Rheum Dis. 2016 Sep;19(9):869-79. doi: 10.1111/1756-185X.12888. Epub 2016 May 2.