Immune Thrombocytopenia Purpura Clinical Trial
Official title:
An Outcome Study of Conventional Steroids vs. Steroids Combined With Mycophenolate for the Management of Newly Diagnosed Immune Thrombocytopenia Purpura: A Cost Effective Measure in Developing Nations.
Immune thrombocytopenia Purpura (ITP) is an autoimmune condition delineated by humoral as well as cell mediated immune response against thrombocyte surface proteins GPIIb/IIIa receptors, affecting primary homeostasis leading to mucocutaneous bleeding.ITP is characterized by platelet count <100 x 109/L. The conventional line of treatment for newly diagnosed ITP is steroids but significant disadvantages have been associated with long term use and a high risk of relapse when reducing the dose. The addition of MMF to the first line treatment of ITP resulted in substantial response and a lower risk of refractory ITP with decreased financial burden and improved outcome.
Immune thrombocytopenia Purpura is an acquired immune mediated state characterized by an extreme reduction in peripheral thrombocyte count <100 x 109/L causing mucocutaneous bleeding.Our main objective is to analyze and assess the addition of MMF along with steroids to the first line treatment of ITP resulted in greater response and a lower risk of refractory and chronic ITP, with decreased financial burden and improved outcome. This prospective single-center ,Randomized Interventional study is scheduled to take place at NIBD and BMT Hospital in Karachi. The study has received approval from the institute's ethical review board. The study will comprise the newly diagnosed patients of Immune thrombocytopenia purpura aged 5-60years after detailed counseling regarding the study and explaining the adverse effects of use drug. 104 patients will be included in this trial, with a 1:1 ratio among the two groups 52 patients will be in each group. Based on the findings of a prior study, there was a notable difference of 22% in prevalence between the treatment and control groups. This calculated difference informed the determination of the sample size, which is set at 104. The study is designed with a statistical power of 80% and a margin of error of 0.05. The sample size calculations were performed using open epi version 3. One group will receive steroids alone and the other will receive steroids and MMF. Exclusions criteria includes patients having a history of hepatitis B and C infection, HIV infection, lactating mothers, pregnancy and allergic to drug. Patients will be assessed for confirmatory clinical diagnosis for ITP by monitoring parameters comprising of immature platelet fraction (IPF), platelet count <100x109/L, clinical significant indication to start therapy. The other possibilities of thrombocytopenia will be ruled out such as autoimmune profile, H-pylori antigen and viral markers. Patients will be followed up for a period of 12 months. Detailed history and examination will be done at baseline and during each follow-up visit initially at 2weeks of interval followed by 4weeks. At follow up visit, enquiry will be done regarding adverse events related to MMF. Before adding the experimental drug (MMF) biochemical markers will also be assessed at baseline such as CBC, LFTs, Urea and creatinine.CBC will be repeated monthly and LFTs, urea, creatinine after every 3months to ensure the drug safety profile. The drug pharmacovigilance will also be checked by adverse drug events Performa on every follow-up visit. The prednisolone dose will be given at 2mg/kg with PPI or H2 antagonist. MMF dose will be start from 500mg twice daily, if the drug does not achieve complete or partial response then increased to 750mg twice daily after 2 weeks of treatment. If the drug is well tolerated and does not achieved response then increase the dose to 1gtwice daily after 4weeks after starting the treatment. The pediatric dose for MMF is 15mg/kg twice daily. Attending physician will be allowed to reduce the dose if the patient suffered from any adverse event. The data collection procedure will be Performa based. The relationships between the groups were assessed using different statistical methods based on the type of data under investigation. The associations between the groups were measured by the Chi-square for categorical variables and for quantitative variables ANOVA will be applied for the determination of significance difference between the treatments. Kaplan-Meier curves will be applied to measure the proportion of survival. The difference between the treatment groups will be measured by log-rank test. Cox-regression methods will be applied for the hazard ratios with different variables. SPSS version 25 and STATA version 15 will be used for the analysis. The p-value of 0.05 will be considered statistically significant. The purpose of our study is to ensure the efficacy and pharmacovigilance of mycophenolate mofetil by introducing it as a first line treatment preference as well as reducing the economical burden and risk of relapse ITP. ;
| Status | Clinical Trial | Phase | |
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