Outcome Study of Conventional Steroids Vs. Steroids Combined with Mycopehnolate in Newly Diagnosed Immune Thrombocytopenia Purpura.
- Conditions
- Immune Thrombocytopenia Purpura
- Interventions
- Registration Number
- NCT06288932
- Lead Sponsor
- National Institute of Blood and Marrow Transplant (NIBMT), Pakistan
- Brief Summary
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.
- Detailed Description
Immune Thrombocytopenia Purpura (ITP) is an acquired, immune-mediated disorder characterized by a severe decrease in peripheral thrombocyte count to less than 100 x 10⁹/L, leading to mucocutaneous bleeding. Our primary objective is to evaluate the efficacy of adding Mycophenolate Mofetil (MMF) alongside steroids in the first-line treatment of ITP, aiming for an enhanced therapeutic response, lower risk of refractory and chronic ITP, reduced financial burden, and improved overall outcomes.
The study aims to validate the efficacy and safety of MMF as a first-line treatment option for ITP while reducing the economic burden and risk of relapse associated with the condition
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 118
Not provided
Not provided
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description Mycophenolate Mofetil (MMF) Treatment Mycophenolate Mofetil (MMF) Treatment Patients in this arm will receive Mycophenolate Mofetil (MMF) at a starting dose of 500mg twice daily along with Prednisolone. The pediatric dose of MMF will be calculated by 30mg/kg/day. Dose adjustment will be based on treatment response, with potential increases to 750mg twice daily or 1g twice daily. MMF will be administered for a specific duration, as determined by the study design.
- Primary Outcome Measures
Name Time Method Platelet count monitoring assessed after 3 months and 6 months of treatment. Platelet count improvement, as assessed through regular monitoring and according to response criteria:
The response criteria to the treatment described as follow:
1. Complete response: Platelet count ≥ 100 x 10\^ 9 /L
2. Partial response: Platelet count 30≥ 100 x 10 \^9 /L
3. No response: Platelet count 30 x 10\^9 /
- Secondary Outcome Measures
Name Time Method Assessment of Health-Related Quality of Life questionnaire 12 months Health-related quality of life questionnaire
Incidence of adverse events 12 months
Trial Locations
- Locations (1)
National Institute of Blood Diseases and Bone Marrow Transplantation
🇵🇰Karachi, Sindh, Pakistan