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

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.

Study Overview

• Status: Completed
• Conditions: Immune Thrombocytopenia Purpura
• Intervention / Treatment: Drug: Mycophenolate Mofetil (MMF) Treatment

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

• Study Type: Interventional
• Enrollment (Actual): 118
• Phase: Phase 3

Contacts and Locations

Study Locations

• Pakistan
  • Sindh
    • Karachi, Sindh, Pakistan, 75300
      • National Institute of Blood Diseases and Bone Marrow Transplantation

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult
• Accepts Healthy Volunteers: No

Description

Inclusion Requirements:

• Age: 5-60 years • Number of plates: <30x109

Exclusion Standards:

Pregnancy; lactation; hepatitis B and C; HIV-reactive individuals; and medication allergies

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: None (Open Label)

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: 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.	Drug: Mycophenolate Mofetil (MMF) Treatment; 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.; Other Names: Suprimun
No Intervention: Standard Treatment Control; 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.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Platelet count monitoring	Platelet count improvement, as assessed through regular monitoring and according to response criteria: The response criteria to the treatment described as follow: Complete response: Platelet count ≥ 100 x 10^ 9 /L; Partial response: Platelet count 30≥ 100 x 10 ^9 /L; No response: Platelet count 30 x 10^9 /	assessed after 3 months and 6 months of treatment.

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Incidence of adverse events		12 months
Assessment of Health-Related Quality of Life questionnaire	Health-related quality of life questionnaire	12 months

Collaborators and Investigators

• Sponsor: National Institute of Blood and Marrow Transplant (NIBMT), Pakistan
• Investigators: Principal Investigator: Rukh-e- Zainub, Pharm-D,Mphil(enrolled), National Institute of Blood Diseases and Bone Marrow Transplantation; Study Director: Shafaq Abdul Samad, MBBS,FCPS, National Institute of Blood Diseases and Bone Marrow Transplantation; Study Chair: Dr.Iyad Naeem, Ph.D, KARACHI UNIVERSITY

Study record dates

Study Major Dates

• Study Start (Actual): August 18, 2023
• Primary Completion (Actual): February 18, 2024
• Study Completion (Actual): August 18, 2024

Study Registration Dates

• First Submitted: February 16, 2024
• First Submitted That Met QC Criteria: February 29, 2024
• First Posted (Actual): March 1, 2024

Study Record Updates

• Last Update Posted (Actual): November 6, 2024
• Last Update Submitted That Met QC Criteria: November 4, 2024
• Last Verified: October 1, 2024

More Information

Terms related to this study

• Keywords: MMF; ITP
• Additional Relevant MeSH Terms: Cytopenia; Pathologic Processes; Autoimmune Diseases; Immune System Diseases; Hemorrhage; Skin Manifestations; Hematologic Diseases; Blood Coagulation Disorders; Hemorrhagic Disorders; Blood Platelet Disorders; Thrombotic Microangiopathies; Thrombocytopenia; Purpura; Purpura, Thrombocytopenic, Idiopathic; Purpura, Thrombocytopenic; Anti-Bacterial Agents; Anti-Infective Agents; Antibiotics, Antineoplastic; Antineoplastic Agents; Molecular Mechanisms of Pharmacological Action; Enzyme Inhibitors; Antibiotics, Antitubercular; Antitubercular Agents; Mycophenolic Acid
• Other Study ID Numbers: NIBD/IRB-263/08-2023

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No