Immune Thrombocytopenia Purpura
Conditions
Keywords
ITP, MMF
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
Interventions
The first-line treatment of immune thrombocytopenia purpura with Mycophenolate Mofetil produced a significant response, a lowered likelihood of refractory ITP, a better outcome, and less financial burden.
Sponsors
National Institute of Blood and Marrow Transplant (NIBMT), Pakistan
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
NONE
Intervention model description
The study will comprise the newly diagnosed patients of Immune thrombocytopenia purpura aged 5-60 year. Patients will with a 1:1 ratio among the two group.
Eligibility
Sex/Gender
ALL
Age
5 Years to 60 Years
Healthy volunteers
No
Inclusion criteria
Inclusion Requirements: • Age: 5-60 years • Number of plates: \<30x109 Exclusion Standards: Pregnancy; lactation; hepatitis B and C; HIV-reactive individuals; and medication allergies
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| 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
| Measure | Time frame | Description |
|---|---|---|
| Assessment of Health-Related Quality of Life questionnaire | 12 months | Health-related quality of life questionnaire |
| Incidence of adverse events | 12 months | — |
Countries
Pakistan
Outcome results
None listed