ObServatory Children Acute RElated Therapy Leukemia
试验速览
- 阶段
- 不适用
- 状态
- 尚未招募
- 发起方
- 入组人数
- 40
- 主要终点
- potential clinical-biological prognostic factors - secondary AML characteristics: molecular data
研究概览
简要总结
Acute Myeloid Leukemias (AML) of the child are a rare disease and its prognosis varies according to the subgroup. Secondary AMLs remain a subgroup of poor prognosis, whose cytogenetic and molecular characteristics and prognostic value differ in part from de novo AMLs. The purpose of this national observatory is to record scientific and medical information on cases of secondary AML that have occurred in France since 2013 in order to improve the treatment of children and adolescents with this disease in the years to come. This national observatory will contribute to better knowledge and progress in research into these diseases.
详细描述
Therapy-related myeloid neoplasms (t-MNs) are a group of hematologic diseases that arise after chemotherapy and/or radiation therapy for a previous cancer or rarely autoimmune diseases. t-MNs had been included in the group of AMLs and remain as a distinct category also in the recent 2016 revision of the WHO classification of myeloid neoplasm and acute leukemia. The latency between exposition to anticancer drugs and development of t-MN may vary from some months up to 10 years, even considering the age at diagnosis of the primary malignancy, the kind of cytotoxic treatment previously used, and the cumulative dose and dose intensity. The prognosis of s-AML is generally considered to be poorer than that of de novo AML. The disease tends to be refractory to chemotherapy, and patients' tolerance of treatment generally is reduced because of prior therapies. 5-year event-free survival (EFS) and overall survival (OS) rates of pediatric t-AML/t-MDS have been reported to be 14% to 30%. However, the results are conflicting and overall lacking when compared with those in adults. The purpose of this national observatory is to record scientific and medical information on cases of secondary Acute Myeloid Leukemias that have occurred in France since 2013 in order to improve the treatment of children and adolescents with this disease in the years to come. The primary objective is to evaluate the association of potential prognostic factors (including clinical-biological factors) with the overall survival of children and adolescents aged 0-18 years diagnosed with secondary AML. The secondary objectives are to test the feasibility of setting up a French national database of secondary AMLs for children and adolescents as a first step towards European implementation, to assess the association of potential prognostic factors with event free survival and the occurrence of treatment-related toxicities, to characterize the molecular abnormalities associated with secondary AMLs in children and adolescents.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Other
入排标准
- 年龄范围
- — 至 18 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patients aged 0-18 years
- •Patient with first cancer
- •Diagnosis of secondary AML
- •Patients treated in a SFCE (Société française des cancers de l'enfant) center
- •For cases included in the prospective from March 2019 onwards: Consent of holders of parental authority and consent of the child of understanding age
排除标准
- 未提供
结局指标
主要结局
potential clinical-biological prognostic factors - secondary AML characteristics: molecular data
时间窗: through study completion, an average of 6 months after patient inclusion
molecular data assessed by Next-Generation Sequencing panel
potential clinical-biological prognostic factors - patient characterics
时间窗: through study completion, an average of 6 months
patient characteristics are sex, patient's age at the start of treatment for secondary AML, date of birth, personal history of genetic predisposition (including brittle diseases - see below), family history of cancers (1st or 2nd degree)
potential clinical-biological prognostic factors :first cancer treatment : types of treatments received
时间窗: through study completion, an average of 6 months after patient inclusion
type of treatment: chemotherapy (Anthracyclines, Alkylating agents) and / or radiotherapy and/or Marrow autograft or allograft
potential clinical-biological prognostic factors - secondary AML characteristics: median time of onset compared to the date of end of treatment for the 1st cancer
时间窗: through study completion, an average of 6 months after patient inclusion
median time of onset compared to the date of end of treatment for the 1st cancer
potential clinical-biological prognostic factors - secondary AML characteristics: cytogenetic data
时间窗: through study completion, an average of 6 months after patient inclusion
karyotype, exclusive and cumulative anomalies
potential clinical-biological prognostic factors - first cancer characteristics
时间窗: through study completion, an average of 6 months after patient inclusion
First cancer characteristics are age of onset, determined from the date of diagnosis, and histology, determined by anatomo-pathological diagnosis.
potential clinical-biological prognostic factors - first cancer treatments: Number of participants with treatment-related adverse events as assessed by CTCAE v4.0
时间窗: through study completion, an average of 6 months after patient inclusion
nature, incidence and severity of adverse events (AEs)
potential clinical-biological prognostic factors - secondary AML treatment: duration of treatment
时间窗: through study completion, an average of 6 months after patient inclusion
duration of treatment determined from the start and end dates
potential clinical-biological prognostic factors - first cancer treatments: duration of treatment
时间窗: through study completion, an average of 6 months after patient inclusion
duration of treatment determined from the start and end dates
potential clinical-biological prognostic factors - secondary AML characteristics : hematological data assessed by morphology
时间窗: through study completion, an average of 6 months after patient inclusion
Leukocytes at diagnosis of secondary AML (G/L)
potential clinical-biological prognostic factors - secondary AML treatment: type of treatment
时间窗: through study completion, an average of 6 months after patient inclusion
chemotherapy received and bone marrow transplantation
potential clinical-biological prognostic factors - secondary AML treatment: Number of participants with treatment-related adverse events as assessed by CTCAE v4.0
时间窗: through study completion, an average of 6 months after patient inclusion
nature, incidence and severity of adverse events (AEs)
potential clinical-biological prognostic factors - first cancer treatments: response to treatment
时间窗: through study completion, an average of 6 months after patient inclusion
response measured by bone marrow assessment using morphology and minimal residual disease (MRD) assessment : no response, partial response, complete remission
potential clinical-biological prognostic factors - first cancer treatments: cumulative dose received
时间窗: through study completion, an average of 6 months after patient inclusion
cumulative dose of each type of treatment received ( chemotherapy (Anthracyclines, Alkylating agents) and / or radiotherapy and/or Marrow autograft or allograft)
potential clinical-biological prognostic factors - secondary AML characteristics: date of diagnosis
时间窗: through study completion, an average of 6 months after patient inclusion
date of diagnosis
potential clinical-biological prognostic factors - Overall Survival (OS)
时间窗: at the end of the 2 years of the inclusion period
Evaluated as time from diagnostic of the second AML to death from any cause or date last seen for patients who are alive at the end of the trial
potential clinical-biological prognostic factors - secondary AML treatment : response to treatment
时间窗: through study completion, an average of 6 months after patient inclusion
response measured by bone marrow assessment using morphology and minimal residual disease (MRD) assessment : no response, partial response, complete remission
次要结局
- feasibility of setting up a French national database of secondary AMLs for children and adolescents - Missing Data(at the end of the 2 years of the inclusion period)
- feasibility of setting up a French national database of secondary AMLs for children and adolescents - Centre participation rates(at the end of the 2 years of the inclusion period)
- feasibility of setting up a French national database of secondary AMLs for children and adolescents - Number of cases included over the period(at the end of the 2 years of the inclusion period)
- association of potential prognostic factors with event free survival and the occurrence of treatment-related toxicities- Treatment-related toxicities(at the end of the 2 years of the inclusion period)
- association of potential prognostic factors with event free survival and the occurrence of treatment-related toxicities- Recurrence of the disease(at the end of the 2 years of the inclusion period)