Prednisone or Dexamethasone in Treating Patients With Newly Diagnosed, Previously Untreated Primary Immune Thrombocytopenic Purpura (ITP0207)

• Final response (complete, partial, and minimal response) rate from evaluation of initial response [ Time Frame: At day +180 from evaluation of initial response ] [ Designated as safety issue: No ]
  

• Initial response rate [ Time Frame: At day 42 (arm I), at day 46 (arm II) ] [ Designated as safety issue: No ]
  
• Quality of response per arm [ Time Frame: At initial evaluation and at final evaluation ] [ Designated as safety issue: No ]
  
• Final response rate [ Time Frame: At day 180 from the statement of initial response ] [ Designated as safety issue: No ]
  
• Rate of bleeding events [ Time Frame: At 3 years from study entry ] [ Designated as safety issue: Yes ]
  
• 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 Frame: At day 42 or before day 180 from the first evaluation ] [ Designated as safety issue: No ]
  
• Time to platelet number increase until a hemostatically effective level is reached and/or disappearance of bleeding symptoms [ Time Frame: At 3 years from study entry ] [ Designated as safety issue: No ]
  
• Rate of persistent response [ Time Frame: At 12 months from the statement of initial response ] [ Designated as safety issue: No ]
  
• Association of type of initial response with final and persistent response (in patients with final and persistent response) [ Time Frame: At 3 years from study entry ] [ Designated as safety issue: No ]
  
• Rate of rescue interventions [ Time Frame: After day 180 from evaluation of initial response ] [ Designated as safety issue: No ]
  
• Rate of splenectomy eligible patients [ Time Frame: At 12 months from enrollment ] [ Designated as safety issue: No ]
  
• Rate of patients who have undergone splenectomy during follow-up [ Time Frame: At 3 years from study entry ] [ Designated as safety issue: No ]
  
• Rate of patients who develop connective tissue diseases or underlying hematological diseases (myelodysplastic syndromes, chronic lymphoproliferative diseases, others) during follow-up [ Time Frame: At 3 years from study entry ] [ Designated as safety issue: No ]
  

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.