Clinical trial NCT04450784: ObServatory Children Acute RElated Therapy Leukemia
Official title: ObServatory Children Acute RElated Therapy Leukemia
Study type: OBSERVATIONAL
Overall status: UNKNOWN
Conditions: Leukemia, Myeloid, Acute
Keywords: Secondary
Acute myeloid Leukemia
Children
Lead sponsor: University Hospital, Bordeaux
Sponsor class: OTHER
First posted: 2020-06-30
Enrollment: 40
Sex: ALL
Maximum age (years): 18
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: OTHER
Sampling method: NON_PROBABILITY_SAMPLE
Study population: children and adolescents aged 0-18 years diagnosed with secondary AML

Brief summary: 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.

Detailed description: 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.

Eligibility criteria: Inclusion Criteria:

* 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

Exclusion Criteria: None

Primary outcomes:
measure: potential clinical-biological prognostic factors - patient characterics
timeFrame: through study completion, an average of 6 months
measure: potential clinical-biological prognostic factors - first cancer characteristics
timeFrame: through study completion, an average of 6 months after patient inclusion
measure: potential clinical-biological prognostic factors :first cancer treatment : types of treatments received
timeFrame: through study completion, an average of 6 months after patient inclusion
measure: potential clinical-biological prognostic factors - first cancer treatments: response to treatment
timeFrame: through study completion, an average of 6 months after patient inclusion
measure: potential clinical-biological prognostic factors - first cancer treatments: Number of participants with treatment-related adverse events as assessed by CTCAE v4.0
timeFrame: through study completion, an average of 6 months after patient inclusion
measure: potential clinical-biological prognostic factors - first cancer treatments: duration of treatment
timeFrame: through study completion, an average of 6 months after patient inclusion
measure: potential clinical-biological prognostic factors - first cancer treatments: cumulative dose received
timeFrame: through study completion, an average of 6 months after patient inclusion
measure: potential clinical-biological prognostic factors - secondary AML characteristics: date of diagnosis
timeFrame: through study completion, an average of 6 months after patient inclusion
measure: potential clinical-biological prognostic factors - secondary AML characteristics: median time of onset compared to the date of end of treatment for the 1st cancer
timeFrame: through study completion, an average of 6 months after patient inclusion
measure: potential clinical-biological prognostic factors - secondary AML characteristics : hematological data assessed by morphology
timeFrame: through study completion, an average of 6 months after patient inclusion
measure: potential clinical-biological prognostic factors - secondary AML characteristics: cytogenetic data
timeFrame: through study completion, an average of 6 months after patient inclusion
measure: potential clinical-biological prognostic factors - secondary AML characteristics: molecular data
timeFrame: through study completion, an average of 6 months after patient inclusion
measure: potential clinical-biological prognostic factors - secondary AML treatment: duration of treatment
timeFrame: through study completion, an average of 6 months after patient inclusion
measure: potential clinical-biological prognostic factors - secondary AML treatment: type of treatment
timeFrame: through study completion, an average of 6 months after patient inclusion
measure: potential clinical-biological prognostic factors - secondary AML treatment : response to treatment
timeFrame: through study completion, an average of 6 months after patient inclusion
measure: potential clinical-biological prognostic factors - secondary AML treatment: Number of participants with treatment-related adverse events as assessed by CTCAE v4.0
timeFrame: through study completion, an average of 6 months after patient inclusion
measure: potential clinical-biological prognostic factors - Overall Survival (OS)
timeFrame: at the end of the 2 years of the inclusion period
Secondary outcomes:
measure: feasibility of setting up a French national database of secondary AMLs for children and adolescents - Number of cases included over the period
timeFrame: at the end of the 2 years of the inclusion period
measure: feasibility of setting up a French national database of secondary AMLs for children and adolescents - Missing Data
timeFrame: at the end of the 2 years of the inclusion period
measure: feasibility of setting up a French national database of secondary AMLs for children and adolescents - Centre participation rates
timeFrame: at the end of the 2 years of the inclusion period
measure: association of potential prognostic factors with event free survival and the occurrence of treatment-related toxicities- Recurrence of the disease
timeFrame: at the end of the 2 years of the inclusion period
measure: association of potential prognostic factors with event free survival and the occurrence of treatment-related toxicities- Treatment-related toxicities
timeFrame: at the end of the 2 years of the inclusion period
Other outcomes:
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