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.
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 入组人数
- 150
- 试验地点
- 42
- 主要终点
- Final response (complete, partial, and minimal response) rate from evaluation of initial response
研究概览
简要总结
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.
详细描述
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.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 80 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- 未提供
排除标准
- 未提供
研究组 & 干预措施
ARM A - PDN
PDN is administered orally at the daily dose of 1 mg/Kg for 4 consecutive weeks (from day 0 to day 28), then, therapy is tapered within 14 days. The patients considered NOT RESPONDER at day 42 or WHO HAVE LOST THE RESPONSE within the evaluation of final response (see paragraph 8.1), will be crossed to ARM B.
干预措施: prednisone (Drug)
ARM A - PDN
PDN is administered orally at the daily dose of 1 mg/Kg for 4 consecutive weeks (from day 0 to day 28), then, therapy is tapered within 14 days. The patients considered NOT RESPONDER at day 42 or WHO HAVE LOST THE RESPONSE within the evaluation of final response (see paragraph 8.1), will be crossed to ARM B.
干预措施: quality-of-life assessment (Procedure)
ARM B - DXM
DXM is administered orally at single fixed daily doses of 40 mg for 4 consecutive days, every 14 days, for 3 consecutive courses. If platelet count is £ 20x109/L or bleeding symptoms related to thrombocytopenia are present, lowdose DXM (0.035 mg/Kg/day) between courses is given. The patients (either from ARM A+B or from ARM B) considered NOT RESPONDER at day 46 or who HAVE LOST THE RESPONSE within the evaluation of final response (see paragraph 8.1), will be considered OFF TREATMENT.
For these patients a second line therapy will be considered, according to the medical practice of the Centre (splenectomy or other).
干预措施: dexamethasone (Drug)
ARM B - DXM
DXM is administered orally at single fixed daily doses of 40 mg for 4 consecutive days, every 14 days, for 3 consecutive courses. If platelet count is £ 20x109/L or bleeding symptoms related to thrombocytopenia are present, lowdose DXM (0.035 mg/Kg/day) between courses is given. The patients (either from ARM A+B or from ARM B) considered NOT RESPONDER at day 46 or who HAVE LOST THE RESPONSE within the evaluation of final response (see paragraph 8.1), will be considered OFF TREATMENT.
For these patients a second line therapy will be considered, according to the medical practice of the Centre (splenectomy or other).
干预措施: quality-of-life assessment (Procedure)
结局指标
主要结局
Final response (complete, partial, and minimal response) rate from evaluation of initial response
时间窗: At day +180 from evaluation of initial response
次要结局
- 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(At 3 years from study entry)
- Time to platelet number increase until a hemostatically effective level is reached and/or disappearance of bleeding symptoms(At 3 years from study entry)
- 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)(At 3 years from study entry)
- Rate of patients who have undergone splenectomy during follow-up(At 3 years from study entry)
- 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)(At day 42 or before day 180 from the first evaluation)
- 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 develop connective tissue diseases or underlying hematological diseases (myelodysplastic syndromes, chronic lymphoproliferative diseases, others) during follow-up(At 3 years from study entry)