Outcome Study of Conventional Steroids Vs. Steroids Combined with Mycopehnolate in Newly Diagnosed Immune Thrombocytopenia Purpura.

National Institute of Blood and Marrow Transplant (NIBMT), Pakistan

Status and phase: Completed; Phase 3

Conditions: Immune Thrombocytopenia Purpura

Treatments: Drug: Mycophenolate Mofetil (MMF) Treatment

Study type: Interventional

Funder types: Other

Identifiers

NCT06288932

NIBD/IRB-263/08-2023

Details and patient eligibility

About

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.

Full 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

Enrollment: 118 patients

Sex: All

Ages: 5 to 60 years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion 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

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: None (Open label)

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118 participants in 2 patient groups

Mycophenolate Mofetil (MMF) Treatment

Experimental group

Description:

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.

Treatment:

Drug: Mycophenolate Mofetil (MMF) Treatment

Standard Treatment Control

No Intervention group

Description:

Patients in this arm will receive the standard treatment for ITP, which may include corticosteroids (prednisolone) commonly used for ITP management.The treatment regimen will be determined based on current standard guidelines and clinical practice.