The Combination of High-dose Dexamethasone and Acetylcysteine as the Treatment of Newly-diagnosed ITP

Study Overview
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Brief Summary
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A single-arm, open-label study to evaluate the efficacy and safety of Acetylcysteine plus high-dose Dexamethasone in adults newly diagnosed with primary immune thrombocytopenia (ITP).

Detailed Description
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This is a prospective, open-label, Phase II, single-arm interventional trial performed in a single center in China. The investigators explore the efficacy and safety of Acetylcysteine plus high-dose Dexamethasone in adults newly diagnosed with primary immune thrombocytopenia.

Official Title
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The Combination of High-dose Dexamethasone and Acetylcysteine as the Treatment of Newly-diagnosed Immune Thrombocytopenia: A Single-arm, Open-label Trial

Conditions
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Immune Thrombocytopenia

Intervention / Treatment
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* Drug: Dexamethasone
* Drug: Acetylcysteine


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Clinically confirmed immune thrombocytopenic purpura (ITP) newly diagnosed Platelet count less than 30×10^9/L on two occasions or Platelets above 30×10^9/L combined with bleeding manifestation (WHO bleeding scale 2 or above) Subject is ≥ 18 years and ≤80years Subject has signed and dated written informed consent. Fertile patients must use effective contraception during treatment and observational period Negative pregnancy test Exclusion Criteria: Have an impaired renal function as indicated by a serum creatinine level > 2.0 mg/dL Have an inadequate liver function as indicated by a total bilirubin level > 2.0 mg/dL and/or an aspartate aminotransaminase or alanine aminotransferase level > 3×upper limit of normal Have a New York Heart Classification III or IV heart disease Have a history of severe psychiatric disorder or are unable to comply with study and follow-up procedures Have active hepatitis B or hepatitis C infection Have a HIV infection Have active infection requiring antibiotic therapy within 7 days prior to study entry Are pregnant or lactating women, or plan to become pregnant or impregnated within 12 months of receiving study drug Previous treatment with rituximab Previous splenectomy Had previous or concomitant malignant disease Not willing to participate in the study. Expected survival of < 2 years Intolerant to murine antibodies Immunosuppressive treatment within the last month Connective tissue disease Autoimmune hemolytic anemia Patients currently involved in another clinical trial with evaluation of drug treatment

Ages Eligible for Study
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Minimum Age: 18 Years
Maximum Age: 80 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: High-dose Dexamethasone plus Acetylcysteine<br>For all patients in this arm, DXM was administered intravenously at 40 mg daily for 4 consecutive days and then stopped. If platelet count remained below 30×10^9/L or there were bleeding symptoms by day 10 to 14, an additional 4-day course of DXM (40 mg daily) was given. Besides, all patients received Acetylcysteine orally at 0.4g three times a day for 4 consecutive weeks in the first month. | Drug: Dexamethasone<br>* Dexamethasone, iv, 40 mg/d, for 4 days (The 4-day course of dexamethasone was repeated on days 11 to 14 in the case of lack of response by day 10)<br>* Other names: High-dose Dexamethasone;Drug: Acetylcysteine<br>* Acetylcysteine, po, 400mg tid, for 4 weeks<br>* Other names: YiWeiShi;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Number of patients with durable response after 6 months | Number of patients with durable response after 6 months | 6 months | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Early response | Number of patients with early response on day 7 | 7 days | 
| Initial response | Number of patients with initial response at 1 month | 1 month | 
| Bleeding | Number of patients with bleeding complication therapy; bleeding score | 1 year | 
| DOR | duration of response | 1 year | 
| Relapse | Number of patients relapse during the observation | 1 year | 
| Adverse events | Number of patients with adverse events | 1 year | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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immune thrombocytopenia, acetylcysteine