Immune Thrombocytopenia Management in Adults

The Outcomes and Safety of Immunomodulators and Thrombopoietin Receptor Agonists in Primary Immune Thrombocytopenia Egyptian Patients With Hemorrhage Comorbidity

Immune thrombocytopenia treatment has evolved recently. However, none of treatments have only benefits without drawbacks. This study compares the clinical outcomes and adverse drug patterns of different treatment options. Medications which will be assessed during the current study are High Dose-dexamethasone (HD-DXM) (control group), Prednisolone + Azathioprine, Rituximab, Eltrombopag, and Romiplostim.

Study Overview

• Status: Recruiting
• Conditions: Immune Thrombocytopenia Purpura
• Intervention / Treatment: Drug: Dexamethasone; Drug: Prednisolone and Azathioprine; Drug: Rituximab; Drug: Eltrombopag; Drug: Romiplostim

Detailed Description

A prospective controlled randomized study was conducted on primary Immune thrombocytopenia patients. The study's main objective is to evaluate the efficacy and adverse events profile of the different therapeutic approaches during Immune thrombocytopenia. Upon the confirmation of the Immune thrombocytopenia diagnosis, all patients immediately initiated the High Dose-dexamethasone as a frontline therapy for Immune thrombocytopenia with a dose of 40 mg/m2 daily for 4 days/week in the first month for one cycle. Then, the recruited patients who fulfilled the inclusion criteria are randomly assigned into one of five groups. Among these patients, the control group received IV pulse (HD-DXM) therapy with 40 mg/m2 daily for 4 successive days in a 28-day cycle to complete the six cycles. The Prednisolone + Azathioprine group received 20 mg of Prednisolone three times daily and 1 mg/kg of oral Azathioprine once daily for two weeks, then tapering the Prednisolone dose through the subsequent weeks (6 weeks). While continuing treatment with Azathioprine for a total of six months. The Rituximab group received 500 mg/m2 intravenously of Rituximab once weekly for one month. The Eltrombopag group received 50 mg of Eltrombopag four hours before or after meals as oral daily doses for 6 months. The Romiplostim group received 3μg/kg sub-cutaneous injection of Romiplostim once a week for 6 months. The first evaluation date of confirmed ITP diagnosis was well-defined as the first index date (baseline). After that, every patient visited the investigational site as the protocol prescribes once weekly to assess and adjust the doses of study medications. The outcome measures were judged at baseline, at the end of treatment (6 months), and after an additional 6-month free treatment period.

• Study Type: Interventional
• Enrollment (Anticipated): 467
• Phase: Phase 4

Contacts and Locations

• Study Contact: Name: Eman Mostafa Hamed, master; Phone Number: 01019834193; Email: eman.hamed@nub.edu.eg
• Study Contact Backup: Name: Mostafa Hamed; Phone Number: 01286744337; Email: eman.hamed@nub.edu.eg

Study Locations

• Egypt
  • Cairo, Egypt, 52611
    • Recruiting
    • EL-Kasr elineiy
    • Contact: Doaa Mohamed El Demerdash; Phone Number: 01003516382; Email: dr_eldemerdash@kasralainy.edu.eg
    • Contact: Ahmed M. Khalaf; Phone Number: 01204419172; Email: dr.ahmed201176@yahoo.com

Participation Criteria

Eligibility Criteria

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

Description

Inclusion Criteria:

• Inclusion criteria were adult patients aged 18 years or older, diagnosed with primary ITP after excluding secondary causes and with an initial PLTs count of less than 30 ×109/L or with hemorrhage manifestations.

Exclusion Criteria:

• Patients with a confirmed secondary ITP diagnoses such as (chemicals induced, systemic lupus erythematosus, immune thyroid diseases, a lymphoproliferative disease, or chronic infection, such as Helicobacter pylori, human immunodeficiency virus (HIV) or hepatitis C virus (HCV); with cardiac, renal, or liver disease; who had received NSAIDs or anti-platelets within one month before the initiation of the enrollment were excluded from the study.

Study Plan

How is the study designed?

Design Details

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

Number of Arms

5

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Active Comparator: Control group; The first (control )group includes patients with confirmed diagnosed who received IV pulse (High Dose-Dexamethasone) therapy	Drug: Dexamethasone; Patients were initiated with High Dose-Dexamethasone with a dose of 40 mg/m2 daily for four days per week immediately after the diagnosis of ITP for 6 months
Experimental: PSL - AZA group; The second group includes patients with confirmed diagnosed who received Prednisolone -Azathioprine therapy	Drug: Prednisolone and Azathioprine; the second group received 20 mg of Prednisolone three times daily and 1 mg/kg of oral Azathioprine once daily for two weeks, then tapering the Prednisolone dose through the subsequent weeks (6 weeks). While continuing treatment with Azathioprine for a total of six months.
Experimental: The RTX group; The third group includes patients with confirmed diagnosed who received Prednisolone -Azathioprine therapy	Drug: Rituximab; The third group received 500 mg/m2 intravenously of Rituximab once weekly for one month
Experimental: The ELTRO group; The fourth group includes patients with confirmed diagnosed who received E therapy	Drug: Eltrombopag; The fourth group received 50 mg of Eltrombopag four hours before or after meals as oral daily doses for 6 months
Experimental: The ROMP group; The fifth group includes patients with confirmed diagnosed who received Romiplostim therapy	Drug: Romiplostim; The fifth group received 3μg/kg subcutaneous injection of Romiplostim once a week for 6 months

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
total patients who achieved sustained and overall response	The primary outcomes were the total percentage of patients achieving a sustained response (SR) till the end of the study, complete response (CR), and partial response (PR). CR was characterized by the absence of bleeding and an increase in the platelet count to above 100×109/L after one month of the treatment. SR was defined as achieving CR or partial response (PR) until the end of the study with a 2-fold upsurge from starting point [20, 21]. PR was represented as PLTs count ≥ 30×109/L after one month following therapy, and no response (NR) was defined as platelets < 30×109/L or bleeding	18 months

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
number of patients relapsed and adverse events	The secondary outcome measures were a number of patients relapsed and adverse events (AEs). Relapse was pointed out as PLTs count below 30×109/L or bleeding episodes owing to thrombocytopenia afterward achieving the CR	18 months

Collaborators and Investigators

• Sponsor: Nahda University
• Investigators: Study Chair: Mohamed Hussein Meabed, professor, Faculty of medicine Beni-Suef University

Study record dates

Study Major Dates

• Study Start (Actual): May 5, 2020
• Primary Completion (Anticipated): January 18, 2024
• Study Completion (Anticipated): April 2, 2024

Study Registration Dates

• First Submitted: April 27, 2023
• First Submitted That Met QC Criteria: May 6, 2023
• First Posted (Actual): May 16, 2023

Study Record Updates

• Last Update Posted (Actual): May 16, 2023
• Last Update Submitted That Met QC Criteria: May 6, 2023
• Last Verified: May 1, 2023

More Information

Terms related to this study

• Keywords: Rituximab; Dexamethasone; Autoimmune Disorder; Romiplostim; Primary Immune Thrombocytopenia; Etrombopag
• Additional Relevant MeSH Terms: Pathologic Processes; Immune System Diseases; Autoimmune Diseases; Hematologic Diseases; Hemorrhage; Hemorrhagic Disorders; Blood Coagulation Disorders; Skin Manifestations; Blood Platelet Disorders; Thrombotic Microangiopathies; Purpura; Purpura, Thrombocytopenic; Purpura, Thrombocytopenic, Idiopathic; Thrombocytopenia; Physiological Effects of Drugs; Molecular Mechanisms of Pharmacological Action; Autonomic Agents; Peripheral Nervous System Agents; Anti-Inflammatory Agents; Antirheumatic Agents; Antimetabolites, Antineoplastic; Antimetabolites; Antineoplastic Agents; Immunosuppressive Agents; Immunologic Factors; Antiemetics; Gastrointestinal Agents; Glucocorticoids; Hormones; Hormones, Hormone Substitutes, and Hormone Antagonists; Antineoplastic Agents, Hormonal; Antineoplastic Agents, Immunological; Dexamethasone; Prednisolone; Rituximab; Azathioprine
• Other Study ID Numbers: NahdaV

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