The goal of this clinical trial is to learn if hydroxychloroquine (HCQ) plus thrombopoietin receptor agonists (TPO-RA) works to treat primary immune thrombocytopenia with positive anti-nuclear antibodies in adults. It will also learn about the safety of HCQ plus TPO-RA. The main questions it aims to answer are: Does HCQ plus TPO-RA raise the response rate in participants, compared to TPO-RA alone? Does HCQ plus TPO-RA prolong the response duration in participants, compared to Pred alone? Does HCQ plus TPO-RA decrease the dose of TPO-RA to maintain response in participants, compared to TPO-RA alone? What medical problems do participants have when taking HCQ plus TPO-RA? Researchers will compare HCQ plus TPO-RA with TPO-RA alone to see if HCQ plus TPO-RA works better to treat primary immune thrombocytopenia with positive anti-nuclear antibodies. Participants will: Take TPO-RA every day for no more than 24 weeks, adjust the dose of TPO-RA according to the platelet level, with or without HCQ twice a day for 1 year; Visit the clinic once every 1 weeks for the first 8 weeks, and once every 2-4 weeks in the following 10 months for checkups and tests; Keep a diary of their symptoms
Primary immune thrombocytopenia (Primary immune thrombocytopenia, ITP) is an acquired autoimmune hemorrhagic disease characterized by a decreased peripheral platelet count and an increased risk of bleeding. It has been reported that 33.3% -39.2% of ITP patients have positive antinuclear antibodies (ANA) in the course of the disease.In the meantime, they do not meet the diagnostic criteria for rheumatic diseases such as lupus erythematosus(SLE). ITP patients with positive ANA are prone to relapse and chronicity. Therefore, it is necessary to explore new clinical treatments to attain long-term remission in these patients. Hydroxychloroquine (HCQ) has immune modulating role on a variety of immune cells.A clinical trial enrolled immune thrombocytopenia secondary to SLE, and ITP with positive anti-nuclear antibodiy (ANA) were treated with HCQ combined with glucocorticoids. The results showed an overall response rate of 60% (24 / 40), including 18 continuous complete response (CR) and 6 continuous response (R), and some patients had continued elevated platelet counts 3 months after treatment initiation. The above studies illustrate that HCQ contributes to the treatment of chronic ITP, especially as a long-term therapeutic agent with low economic burden and good tolerance. In patients who do not response to HCQ plus corticosteroids, TPO-RA, a second line treatment, will be recommended. By now, no study has assess the efficacy and safety of HCQ plus TPO-RA in these patients. In the current study, the question will be answered.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
SINGLE
Enrollment
126
Hydroxychloroquine is taken at the dose of 0.1g / dose, twice a day for 1 year, regardless of food intake.
Participants will take eltrombopag for at least 24 weeks, the dose of eltrombopag is adjusted according to participants' platelet count.
Shanghai Zhongshan Hospital
Shanghai, Shanghai Municipality, China
RECRUITINGWusong Hospital, Zhongshan Hospital, Fudan University
Shanghai, Shanghai Municipality, China
RECRUITINGShanghai Jinshan Hospital
Shanghai, Shanghai Municipality, China
RECRUITINGQingpu Branch of Zhongshan Hospital, Fudan University
Shanghai, Shanghai Municipality, China
RECRUITINGHealth and Humanity Research Centre, Hongkong, China.
Hong Kong, Hong Kong
RECRUITINGDr. Stanley Ho Medical Foundation
Macao, Macau
RECRUITINGUniversity Hospital, Macau University of Science and Technology.
Macao, Macau
RECRUITINGOverall response rate
The percentage of participants with platelet counts higher than 30×10\^9/L and at least twice the baseline platelet count , for at least two consecutive tests (7 days apart).
Time frame: 4 weeks
Complete response rate
The percentage of participants with platelet counts higher than 100×10\^9/L , for at least two consecutive tests (7 days apart).
Time frame: 1 year
Duration of response
Time from response to disease relapse (platelet count ≤ 30×10\^9/L on any test or occurance of bleeding symptoms )
Time frame: 1 year
Durable response rate
Percentage of patients with complete remission lasting at least 6 months without any additional ITP-specific therapy
Time frame: 1 year
Platelet count at each visit
Average platelet count at each visit
Time frame: 1 year
Time to response
Time from starting treatment to response
Time frame: 4 weeks
Time to TPO-RA withdrawal
Time from starting treatment to TPO-RA withdrawal
Time frame: 24 weeks
Rate of TPO-RA withdrawal
Rate of participants who withdraw TPO-RA with platelet count over 30×10\^9/L
Time frame: 24 weeks
The maximum dose of TPO-RA to attain response
The maximum dose of TPO-RA to attain response (platelet count over 30×10\^9/L)
Time frame: 8 weeks
Response rate throughout the trial
The percentage of response participants (platelet counts higher than 30×10\^9/L and at least twice the baseline platelet count ) at each visit
Time frame: 1 year
Once response rate throughout the trial
The percentage of once response participants (platelet counts higher than 30×10\^9/L and over twice the baseline platelet count for at least one visit) throughout 1 year
Time frame: 1 year
WHO bleeding score
WHO bleeding score at each visit
Time frame: 1 year
Adverse reaction
Adverse reaction at each visit
Time frame: 1 year
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