Evaluation of the Incidence and Prognostic Impact of Molecular and Genetic Abnormalities in Patients With Acute Myeloid Leukemia, Myelodysplastic Syndrome and Myeloproliferative Neoplasms
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 455
- 试验地点
- 1
- 主要终点
- Prevalence of molecular and cytogenetic abnormalities
研究概览
简要总结
The objective of this study is to describe the prevalence and prognostic impact of the most common genetic abnormalities in patients with Myeloid Neoplasms, including Acute Myeloid Leukemia (AML), Myeloproliferative Neoplasms (MPN), Myelodysplastic Syndromes (MDS) and Myeloproliferative/Myelodysplastic Neoplasms. Patients will have samples of blood and/or bone marrow collected and sent to Hospital Israelita Albert Einstein for analysis and storage.
Patients with a diagnosis of Acute Myeloid Leukemia will be treated according to an uniform protocol.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Parallel
- 主要目的
- Basic Science
- 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
- •Diagnosis of AML according to WHO criteria
- •Age greater than 18 years
- •Performance status (ECOG) between 0-2
- •Adequate liver and kidney function
- •Signed Informed Consent form
- •No prior therapy for AML, except use of hydroxyurea for control of elevated white blood cell counts
- •Adequate contraception for fertile men and women
- •Eligible for intensive chemotherapy (as judged by the treating physician)
排除标准
- •Acute myeloid leukemia with retinoic acid receptor alpha (RARA) translocations (APL, acute promyelocytic leukemia)
- •Pregnant women
- •HIV-positivity
- •New York Heart Association class III and IV congestive heart failure
- •Patient refuses to use adequate contraception
- •History of hypersensibility to any of the used chemotherapy drugs
- •Patient refuses to sign informed consent form
- •Acute Myeloid Leukemia-Non-Intensive Chemotherapy
- •Inclusion Criteria:
- •Diagnosis of AML according to WHO criteria
- •Age greater than 18 years
- •Signed Informed Consent form
- •No prior therapy for AML, except use of hydroxyurea for control of elevated white blood cell counts
- •Adequate contraception for fertile men and women
- •Non-eligible for intensive chemotherapy (as judged by the treating physician)
- •Exclusion Criteria:
- •Acute myeloid leukemia with RARA translocations (APL, acute promyelocytic leukemia)
- •Pregnant women
- •HIV-positivity
- •Patient refuses to use adequate contraception
- •History of hypersensibility to any of the used chemotherapy drugs
- •Patient refuses to sign informed consent form
- •Chronic Myeloid Disorders:
- •Inclusion Criteria:
- •Diagnosis of Myeloproliferative Neoplasm or Myelodysplastic Syndrome or Myeloproliferative/Myelodysplastic Neoplasm according to WHO criteria
- •Age greater than 18 years
- •Signed Informed Consent form
- •Exclusion Criteria:
- •Patient refuses to sign informed consent form
研究组 & 干预措施
AML-Intensive Chemotherapy
Patients with Acute Myeloid Leukemia fit for intensive chemotherapy Patients will receive Induction Chemotherapy, and CR will be evaluated after 28 days.
Patients who achieve CR post-induction chemotherapy will receive post-remission therapy according to risk:
- Low risk patients: Consolidation chemotherapy or Autologous stem cell transplantation
- Intermediate and high-risk patients: Allogeneic stem cell transplantation Patients who do not achieve CR may receive one second induction cycle, and if CR is achieved may proceed to post-remission therapy as per above. Patients who do not achieve CR after two cycles of induction will be deemed refractory and removed from the study.
干预措施: Induction Chemotherapy (Drug)
AML-Intensive Chemotherapy
Patients with Acute Myeloid Leukemia fit for intensive chemotherapy Patients will receive Induction Chemotherapy, and CR will be evaluated after 28 days.
Patients who achieve CR post-induction chemotherapy will receive post-remission therapy according to risk:
- Low risk patients: Consolidation chemotherapy or Autologous stem cell transplantation
- Intermediate and high-risk patients: Allogeneic stem cell transplantation Patients who do not achieve CR may receive one second induction cycle, and if CR is achieved may proceed to post-remission therapy as per above. Patients who do not achieve CR after two cycles of induction will be deemed refractory and removed from the study.
干预措施: Consolidation Chemotherapy (Drug)
AML-Intensive Chemotherapy
Patients with Acute Myeloid Leukemia fit for intensive chemotherapy Patients will receive Induction Chemotherapy, and CR will be evaluated after 28 days.
Patients who achieve CR post-induction chemotherapy will receive post-remission therapy according to risk:
- Low risk patients: Consolidation chemotherapy or Autologous stem cell transplantation
- Intermediate and high-risk patients: Allogeneic stem cell transplantation Patients who do not achieve CR may receive one second induction cycle, and if CR is achieved may proceed to post-remission therapy as per above. Patients who do not achieve CR after two cycles of induction will be deemed refractory and removed from the study.
干预措施: Autologous Stem Cell Transplantation (Drug)
AML-Intensive Chemotherapy
Patients with Acute Myeloid Leukemia fit for intensive chemotherapy Patients will receive Induction Chemotherapy, and CR will be evaluated after 28 days.
Patients who achieve CR post-induction chemotherapy will receive post-remission therapy according to risk:
- Low risk patients: Consolidation chemotherapy or Autologous stem cell transplantation
- Intermediate and high-risk patients: Allogeneic stem cell transplantation Patients who do not achieve CR may receive one second induction cycle, and if CR is achieved may proceed to post-remission therapy as per above. Patients who do not achieve CR after two cycles of induction will be deemed refractory and removed from the study.
干预措施: Allogeneic Stem Cell Transplantation (Drug)
AML-Non-intensive chemotherapy
Patients with acute myeloid leukemia not fit for intensive chemotherapy Patients will receive induction chemotherapy with either low dose cytarabine or decitabine. Assignment to each drug will depend on drug availability and physician discretion. No randomization will be done between the drugs.
Cycles will be repeated every 28 days. Patients who achieve CR will continue to post-consolidation therapy with either cytarabine or decitabine, based on the induction therapy received. Patients will receive a maximum of 4 cycles until achieving CR, if no response is seen after 4 cycles patients will be deemed refractory.
干预措施: Low Dose Cytarabine (Drug)
AML-Non-intensive chemotherapy
Patients with acute myeloid leukemia not fit for intensive chemotherapy Patients will receive induction chemotherapy with either low dose cytarabine or decitabine. Assignment to each drug will depend on drug availability and physician discretion. No randomization will be done between the drugs.
Cycles will be repeated every 28 days. Patients who achieve CR will continue to post-consolidation therapy with either cytarabine or decitabine, based on the induction therapy received. Patients will receive a maximum of 4 cycles until achieving CR, if no response is seen after 4 cycles patients will be deemed refractory.
干预措施: Decitabine (Drug)
结局指标
主要结局
Prevalence of molecular and cytogenetic abnormalities
时间窗: 2 years
As assessed by results of molecular and cytogenetic tests and frequency in the population studied
次要结局
- Number of participants with adverse events as a measure of safety and tolerability(1 year)
- Response rate(1 month)
- Cumulative Incidence of Transformation to Acute Myeloid Leukemia(5 years)
- Overall survival(5 years)
- Cumulative incidence of relapse and non-relapse mortality(5 years)
- Disease Free Survival(5 years)
研究者
Fabio Pires de Souza Santos
MD
Hospital Israelita Albert Einstein