The Study of Different Cycles of High-dose Dexamethasone in the Treatment of ITP

Dicycle Versus Tri-cycle High-dose Dexamethasone in Adult ITP: a Multicenter Randomized Controlled Trial

Primary immune thrombocytopenia is an autoimmune disorder characterised by decreased platelet counts and increased bleeding risk. Corticosteroids have been the standard initial treatment of primary immune thrombocytopenia for more than 30 years. The aim of this randomized controlled trial is to compare the efficacy and safety of high-dose dexamethasone in treating new-diagnosed primary immune thrombocytopenia (ITP) in di-cycle and tri-cycle.

Study Overview

• Status: Not yet recruiting
• Conditions: Thrombocytopenia
• Intervention / Treatment: Drug: Dexamethasone; Drug: Dexamethasone

Detailed Description

In this multicentre, open-label, randomized controlled trial, about 118 new-diagnosed ITP patients will be enrolled from five tertiary medical centres in China. Eligible participants are randomly assigned (1:1) to 2 groups: group DEX2 and group DEX3. In group DEX2, dexamethasone was administered orally at 40 mg per day for two cycles (days1-4, and days 11-14). In group DEX3, dexamethasone was administered orally at 40 mg per day for three cycles (days1-4, days 11-14, and days 21-24). The clinical effect, onset time, duration of efficacy and adverse reactions were observed to compare the efficacy and safety of two different plans.

• Study Type: Interventional
• Enrollment (Estimated): 118
• Phase: Phase 2; Phase 3

Contacts and Locations

• Study Contact: Name: Ming Hou, Pro.; Phone Number: 86-531-82169896; Email: houming@medmail.com.cn
• Study Contact Backup: Name: Yu Hou, Pro.; Email: houyu2009@sina.com

Study Locations

• China
  • Shandong
    • Jinan, Shandong, China, 273300
      • Qilu Hospital of Shandong University

Participation Criteria

Eligibility Criteria

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

Description

Inclusion Criteria:

• Participant must be at least 18 years of age at the time of the screening.
• Participant may be male or female.
• Participant has a confirmed diagnosis of newly diagnosed ITP according to the 2019 International Working Group assessment at screening, and has a baseline platelet count of less than 30 × 10^9 cells per L or had bleeding manifestations, or both.

Exclusion Criteria:

• Participant has evidence of a secondary cause of immune thrombocytopenia (e.g. leukemia, lymphoma, common variable immune- deficiency, systemic lupus erythematosus, autoimmune thyroid disease, past medical history of untreated H. pylori infection) or to drug treatments (e.g. heparin, quinine, antimicrobials, anticonvulsants) or participant has a multiple immune cytopenia, e.g. Evan's syndrome.
• Participant has clinically life-threatening bleeding (e.g. central nervous system bleeding, menorrhagia with significant drop in hemoglobin).
• Participant has a history of coagulopathy disorders other than ITP.
• Participant has a history of arterial or venous thromboembolism (e.g. stroke, transient ischemic attach, myocardial infarction, deep vein thrombosis or pulmonary embolism) within the 6 months prior to randomization or requires anticoagulant treatment.
• Participant has 12-lead ECG with changes considered to be clinically significant upon medical review at baseline.
• Participant has severe renal impairment (glomerular filtration rate less than 45ml/min/1.73 m2).
• Participant has 3 × upper limit of normal of any of the following: alanine aminotransferase, aspartate aminotransferase, or alkaline phosphatase.
• Participant with any of the following conditions: severe immunodeficiency, active or previous malignancy, human immunodeficiency virus (HIV), hepatitis B or C virus infection, pregnancy or lactation.

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: group DEX2; In group DEX2, dexamethasone was administered orally at 40 mg per day for two cycles (days1-4, and days 11-14).	Drug: Dexamethasone; given orally at 40 mg per day for two cycles (days1-4, and days 11-14).; Other Names: DEX; Drug: Dexamethasone; given orally at 40 mg per day for three cycles (days1-4, days 11-14, and days 21-24).; Other Names: DEX
Experimental: group DEX3; In group DEX3, dexamethasone was administered orally at 40 mg per day for three cycles (days1-4, days 11-14, and days 21-24).	Drug: Dexamethasone; given orally at 40 mg per day for two cycles (days1-4, and days 11-14).; Other Names: DEX; Drug: Dexamethasone; given orally at 40 mg per day for three cycles (days1-4, days 11-14, and days 21-24).; Other Names: DEX

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Sustained response	Sustained response was defined as platelet count maintained above 30 × 10^9/L with an absence of bleeding symptoms or no requirement for additional ITP treatment for six consecutive months following achievement of initial response. Complete response was defined as a platelet count of 100 × 10^9 cells per L or higher and an absence of bleeding. Partial response was defined as a platelet count of 30×10^9 cells per L or higher, but less than 100×10^9 cells per L, and at least a doubling of the baseline platelet count and an absence of bleeding.	Month 6

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Health-related quality of life assessment	Health-related quality of life was assessed using a self-administered immune thrombocytopenia Patient Assessment Questionnaire (ITP-PAQ) at baseline and at week 12. Scores ranged from 0 to 100, with higher values indicating better quality of life.	12 weeks
Initial response	Initial responses were assessed by day 24. Complete response was defined as a platelet count of 100 × 10^9 cells per L or higher and an absence of bleeding. Partial response was defined as a platelet count of 30×10^9 cells per L or higher, but less than 100×10^9 cells per L, and at least a doubling of the baseline platelet count and an absence of bleeding.	Day 24
Time to response	Time to response was defined as the time from treatment initiation to achieve a complete response or a partial response, whichever came first, assessed up to day 24.	24 days
Duration of response	Duration of response was defined as the time from achievement of a complete response or a partial response to the loss of response (platelet count <30 × 10^9 cells per L; measured on two occasions more than 1 day apart or the presence of bleeding).	12 months
The number of participants with Adverse events	Adverse events were graded according to the Common Terminology Criteria for Adverse Events (version 4.0). At each visit, we recorded adverse events.	12 months
Bleeding scores	Bleeding symptoms were graded according a standardized bleeding scale specific to primary immune thrombocytopenia on the basis of site and severity of bleeding by Khellaf et al (PMID: 15951296). A modification was made to exclude age from the original scale so that only bleeding symptoms were described. At each visit, we recorded bleeding scores.Scores ranged from 0 to 59, with higher values indicating higher bleeding risk.	12 months

Collaborators and Investigators

• Sponsor: Shandong University

Study record dates

Study Major Dates

• Study Start (Estimated): May 1, 2025
• Primary Completion (Estimated): May 30, 2026
• Study Completion (Estimated): May 30, 2027

Study Registration Dates

• First Submitted: March 30, 2025
• First Submitted That Met QC Criteria: March 30, 2025
• First Posted (Actual): April 6, 2025

Study Record Updates

• Last Update Posted (Actual): April 8, 2025
• Last Update Submitted That Met QC Criteria: April 6, 2025
• Last Verified: April 1, 2025

More Information

Terms related to this study

• Keywords: Dexamethasone; ITP
• Additional Relevant MeSH Terms: Cytopenia; Hematologic Diseases; Blood Platelet Disorders; Thrombocytopenia; Antineoplastic Agents; Physiological Effects of Drugs; Molecular Mechanisms of Pharmacological Action; Anti-Inflammatory Agents; Antiemetics; Autonomic Agents; Peripheral Nervous System Agents; Gastrointestinal Agents; Glucocorticoids; Hormones; Hormones, Hormone Substitutes, and Hormone Antagonists; Antineoplastic Agents, Hormonal; Protease Inhibitors; Enzyme Inhibitors; Dexamethasone; Dexamethasone acetate; BB 1101
• Other Study ID Numbers: DEX 2v3 in ITP

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No