ITP0207Randomized 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.
dexamethasone
+ quality-of-life assessment
+ prednisone
Cytopenia+15
+ Autoimmune Diseases
+ Blood Coagulation Disorders
Treatment Study
Summary
Study start date: April 1, 2008
Actual date on which the first participant was enrolled.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.
Protocol
This section provides details of the study plan, including how the study is designed and what the study is measuring.150 patients to be enrolled
Total number of participants that the clinical trial aims to recruit.Treatment Study
Eligibility
Researchers look for people who fit a certain description, called eligibility criteria: person's general health condition or prior treatments.Any sex
Biological sex of participants that are eligible to enroll.From 18 to 80 Years
Range of ages for which participants are eligible to join.Healthy volunteers not allowed
If individuals who are healthy and do not have the condition being studied can participate.Conditions
Pathology
Criteria
Inclusion criteria * Signed written informed consent according to IGH/EU/GCP and national local laws * Newly diagnosed untreated ITP adult patients * Age \> 18 \< 80 years * Platelet count \<20x109/L * Platelet count \> 20 x109/L and \<50x109/L plus bleeding with score \> 8 (according to grading scale at paragraph 7.1) * Baseline Quality of Life evaluation questionnaire filled in Newly diagnosed untreated ITP adult patients * Age \> 18 \< 80 years * Platelet count \<20x109/L * Platelet count \> 20 x109/L and \<50x109/L plus bleeding with score \> 8 (according to grading scale at paragraph 7.1) * Baseline Quality of Life evaluation questionnaire filled in Exclusion criteria * Active malignancy at time of study entry * Steroids administration (PDN \<1mg/Kg/day) for more than 5 days before randomization * Concomitant treatment with anti-platelet and or anti-coagulant drugs * Concomitant severe psychiatric disorders * Not confirmed diagnosis of ITP for * \*Positivity of autoimmunity markers: antinucleus (≥1:80), anti-tireoglobulin, anti-tireoperoxidase, anti-cardiolipin (≥ 40 GPL UmL), anti-b2glycoprotein (≥ 40 IgG U/mL) antibodies, Lupus Anticoagulant (KCT ratio, dRVVT ratios ≥1.5 times the upper normal limit ), direct antiglobulin test (DAT ). * Presence of autoimmune hemolytic anemia * Presence of connective tissue disease * Women who are pregnant or breastfeeding * Cardiovascular diseases requiring treatment * Severe non-controlled, despite therapy, hypertension and diabetes * Liver and kidney function impairment (creatinine, ALT, AST \>2 times upper normal limit) * HCVAb, HIVAb, HBsAg, HBcAb seropositive status * Chronic liver disease * Documented viral illness by the positivity of IgM, or vaccination both occurred one month before diagnosis * Intake of drugs not previously taken within one week before diagnosis * Bleeding score 15 due to ICH or to GI bleeding (according to grading scale at paragraph 7.1, Tab. 3) * Active gastric ulcer.
Study Plan
Find out more about all the medication administered in this study, their detailed description and what they involve.2 intervention groups are designated in this study
This study does not include a placebo group
Treatment Groups
Group I
ExperimentalGroup II
ExperimentalStudy Objectives
Primary Objectives
Secondary Objectives
Study Centers
These are the hospitals, clinics, or research facilities where the trial is being conducted. You can find the location closest to you and its status.This study has 42 locations
U.O. di Ematologia - Azienda Ospedaliera - Pia Fondazione di Culto e di Religione Card. G.Panico
Tricase, ItalyOpen U.O. di Ematologia - Azienda Ospedaliera - Pia Fondazione di Culto e di Religione Card. G.Panico in Google MapsS.O.C. di Ematologia - Azienda Ospedaliera - SS. Antonio e Biagio e Cesare Arrigo
Alessandria, ItalyAzienda ospedaliera Nuovo Ospedale "Torrette"
Ancona, ItalyUSL 8 - Ospedale S.Donato
Arezzo, Italy