Azacitidine Plus Luspatercept Versus Azacitidine Monotherapy in Higher-risk Myelodysplastic Neoplasms: a Randomized Prospective Study
试验速览
- 阶段
- 3 期
- 状态
- 尚未招募
- 入组人数
- 86
- 主要终点
- overall response rate
研究概览
简要总结
This study is a randomized, prospective, single-center, open-label cohort study involving untreated HR-MDS patients. The patients were divided randomized into AZA+Lus cohort and AZA monotherapy cohort.
详细描述
The hypomethylating agents (HMA) azacitidine (AZA) and decitabine (DEC) have been shown to improve survival and delay disease progression in patients with high-risk MDS. They are recommended by the NCCN as first-line treatments for patients with high-risk MDS. Clinical trials have demonstrated an OR rate of approximately 40-50% with AZA in patients with high-risk MDS. Despite the efficacy of HMA therapy, the rate of transfusion independence remains low. Anemia remains the most prominent symptom in refractory patients, with very limited options for subsequent treatment. Prolonged anemia affects every organ function and seriously affects the prognosis of patients.
Luspatercept is currently approved for the treatment of patients with both erythropoiesis receptor agonist ( ESA) treatment failures in transfusion-dependent low-risk MDS-RS patients. In a randomized controlled phase III clinical trial, compared to a placebo group, luspatercept significantly improved transfusion dependence and improved hemoglobin and quality of life in refractory MDS-RS patients. A recent conference report suggested that there was no significant difference in efficacy between low-risk and high-risk patients treated with luspatercept and that the HI rate for high-risk patients treated with luspatercept monotherapy was approximately 50%.
Thus this study aimed to compare the efficacy of AZA+luspatercept and AZA monotherapy.
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 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Age ≥18 years old
- •Diagnosed as higher-risk MDS (IPSS intermediate-2/high-risk, or IPSS-R >3.5, or IPSS-M moderate high-, high-, very high-risk)
- •Untreated patients
- •Liver and kidney function less than 2 times of upper limit of normal
- •ECOG≤2 and expected survival more than 6 months
- •Informed consent signed
排除标准
- •With active infection
- •Other malignant tumors
- •Obvious abnormal liver and kidney function, or abnormal function of other organs
- •Combined with myelofibrosis
- •Have undergone bone marrow transplantation
- •Pregnant or lactating women, or men who have recent reproductive needs
- •Allergic to azacytidine, Rotercept or excipients
- •History of polysorbate 80 allergy
- •Refuse to sign informed consent
- •Researchers consider it inappropriate to participate in the experiment
研究组 & 干预措施
Azacitidine+Luspatercept
干预措施: Azacitidine (AZA) (Drug)
Azacitidine+Luspatercept
干预措施: Luspatercept (Drug)
Azacitidine
干预措施: Azacitidine (AZA) (Drug)
结局指标
主要结局
overall response rate
时间窗: 3 months, 6 months
次要结局
- complete response rate(3 months, 6 months)
- rate of transfusion independence(3 months, 6 months)
- adverse event rate(through study completion, an average of 1 year)
- relapse rate(through study completion, an average of 1 year)
- progress-free survival(through study completion, an average of 1 year)
- overall survival(through study completion, an average of 1 year)
研究者
Bing Han
Professor
Peking Union Medical College Hospital