Prednisone or Dexamethasone in Treating Patients With Newly Diagnosed, Previously Untreated Primary Immune Thrombocytopenic Purpura
Information source: National Cancer Institute (NCI)
Information obtained from ClinicalTrials.gov on November 03, 2008
Link to the current ClinicalTrials.gov record.
Condition(s) targeted: Precancerous/Nonmalignant Condition
Intervention: dexamethasone (Drug); prednisone (Drug); quality-of-life assessment (Procedure)
Phase: Phase 3
Status: Recruiting
Sponsored by: Gruppo Italiano Malattie EMatologiche dell'Adulto Official(s) and/or principal investigator(s):
Maria Gabriella Mazzucconi, MD, Principal Investigator, Affiliation: Gruppo Italiano Malattie EMatologiche dell'Adulto
Summary
RATIONALE: Drugs, such as prednisone and dexamethasone, may change the immune system and be
an effective treatment for primary immune thrombocytopenic purpura. It is not yet known which
drug is more effective in treating primary immune thrombocytopenic purpura.
PURPOSE: This randomized phase III trial is studying high-dose dexamethasone to see how well
it works compared to standard-dose prednisone in treating patients with newly diagnosed,
previously untreated primary immune thrombocytopenic purpura.
Clinical Details
Official title: Randomized Study of the Treatment of Primary Immune Thrombocytopenic Purpura (ITP) in Newly Diagnosed Untreated Adult Patients. Comparison of Standard Dose Prednisone Versus High-Dose Dexamethasone.
Study design: Treatment, Randomized, Open Label
Primary outcome: Final response (complete, partial, and minimal response) rate at day 180
from evaluation of initial response
Secondary outcome: Initial response rate at day 42 (arm I), at day 46 (arm II)Quality of response per arm at initial evaluation and at final evaluation Final response rate at day 180 from the statement of initial response Rate of bleeding events Resumed response rate in non-responder patients (at day 42) or
patients who have lost response before day 180 from the first evaluation (arm I only) Time to platelet number increase until a hemostatically effective level is reached and/or
disappearance of bleeding symptoms Rate of persistent response at 12 months from the statement of initial response Association of type of initial response with final and persistent response (in patients with final and persistent response) Rate of rescue interventions after day 180 from evaluation of initial response Rate of splenectomy eligible patients at 12 months from enrollment Rate of patients who have undergone splenectomy during follow-up Rate of patients who develop connective tissue diseases or underlying hematological
diseases (myelodysplastic syndromes, chronic lymphoproliferative diseases, others) during
follow-up
Detailed description:
OBJECTIVES:
Primary
- To evaluate the role of therapy intensification in adult patients with newly diagnosed,
previously untreated primary immune thrombocytopenic purpura with high-dose
dexamethasone (HD-DXM), in terms of improvement of response at 6 months after initial
response, in comparison with standard-doses of prednisone.
Secondary
- Compare rate of initial response.
- Compare quality of response.
- Compare rate of final responses and rate of persistent response.
- Compare rate of bleeding events.
- Determine rate of resumed response with HD-DXM in non-responder patients or patients who
have lost response (arm I only).
- Compare time to platelet number increase until a hemostatically effective level is
reached and/or disappearance of bleeding symptoms.
- Compare rate of rescue interventions.
- Compare rate of eligible patients for splenectomy.
- Compare rate of patients who underwent splenectomy.
- Compare rate of patients who develop connective tissue diseases or underlying
hematological diseases (myelodysplastic syndromes, chronic lymphoproliferative diseases,
others).
- Compare patient's self reported quality of life.
OUTLINE: This is a multicenter study. Patients are stratified by treating center. Patients
are randomized to 1 of 2 treatment arms.
- Arm I (Standard-dose prednisone): Patients receive oral prednisone at a standard dose (1
mg/Kg) once daily on days 1-28 followed by a 14-day taper.
Patients considered non-responders at day 42 or who have lost response before evaluation of
final response (day 180) are crossed to arm II.
- Arm II (High-dose dexamethasone): Patients receive oral dexamethasone at a high dose (40
mg/day) once daily on days 1-4. Treatment repeats every 14 days for 3 courses.
Quality of life is assessed at baseline, on day 42 (arm I) or 46 (arm II) (initial response
evaluation day), 180 days after initial response evaluation, and at 3, 9, 12 months after
randomization.
After completion of study treatment, patients are followed monthly until 1 year after
randomization, every 2 months for 1 year, and then every 3 months for 1 year.
Eligibility
Minimum age: 18 Years.
Maximum age: 80 Years.
Gender(s): Both.
Criteria:
DISEASE CHARACTERISTICS:
- Diagnosis of primary immune thrombocytopenic purpura (ITP)
- Newly diagnosed, previously untreated disease
- Meets 1 of the following criteria:
- Platelet count ≤ 20,000/mm³
- Platelet count > 20,000/mm³ and < 50,000/mm³ plus bleeding symptom score ≥ 8
- Patients with bleeding score 15 due to intracranial hemorrhage or GI
bleeding are not eligible
- Unconfirmed diagnosis of primary ITP not allowed if any of the following criteria are
met:
- Positivity of any of the following autoimmunity markers:
- Antinuclear antibody ≥ 1: 80
- Antithyroglobulin, antitireoperoxidase, anticardiolipin antibodies (GP-I ≥
40 U/mL)
- Anti-β2glycoprotein antibodies (IgG ≥ 40 U/mL)
- Lupus anticoagulant (Kaolin clotting time [KCT] ratio, dilute Russell's
viper venom time [dRVVT] ratio ≥ 1. 5 times the upper limit of normal [ULN])
- Direct antiglobulin test
- Presence of autoimmune hemolytic anemia
- Presence of connective tissue disease
PATIENT CHARACTERISTICS:
- Not pregnant or nursing
- No active malignancy at time of study entry
- No hypertension, cardiovascular diseases, or diabetes requiring treatment
- No active gastric ulcer
- Creatinine ≤ 2 times ULN
- ALT and AST ≤ 2 times ULN
- No hepatitis C virus antibody, HIV antibody, hepatitis B surface antigen, or hepatitis
B core antibody seropositive status
- No chronic liver disease
- No concomitant severe psychiatric disorders
- No documented viral illness as evidenced by positivity of IgM
- No IgM positivity for patients who had vaccination 1 months before diagnosis
PRIOR CONCURRENT THERAPY:
- At least 2 days since prior steroids
- Steroid therapy (equivalent doses of prednisone ≤ 1 mg/kg/day) < 2 days before
randomization allowed
- No initiation of new drug within one week before diagnosis
- No concurrent anti-platelet and/or anticoagulant drugs
Locations and Contacts
Gruppo Italiano Malattie Ematologiche dell'Adulto, Rome 00161, Italy; Recruiting
Contact Person, Phone: 39-06-4424-1984, Email: mazzucconi@bce.uniroma.it
Additional Information
Clinical trial summary from the National Cancer Institute's PDQ® database
Starting date: April 2008
Last updated: October 8, 2008
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