ObServatory Children Acute RElated Therapy Leukemia (OSCARE)

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.

Study Overview

• Status: Not yet recruiting
• Conditions: Leukemia, Myeloid, Acute
• Intervention / Treatment: Other: collection of the clinical and biological characteristics of secondary AMLs in children

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.

• Study Type: Observational
• Enrollment (Anticipated): 40

Contacts and Locations

• Study Contact: Name: Stéphane S DUCASSOU, MD PhD; Phone Number: 05 57 82 04 38; Email: stephane.ducassou@chu-bordeaux.fr

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: No older than 18 years (ADULT, CHILD)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

• Sampling Method: Non-Probability Sample

Study Population

children and adolescents aged 0-18 years diagnosed with secondary AML

Description

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

Study Plan

How is the study designed?

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
Secondary Acute Myeloid Leukemias (AML) of the child	Other: collection of the clinical and biological characteristics of secondary AMLs in children; to record scientific and medical information on cases of secondary Acute Myeloid Leukemias that have occurred in France since 2013

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
potential clinical-biological prognostic factors - patient characterics	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)	through study completion, an average of 6 months
potential clinical-biological prognostic factors - first cancer characteristics	First cancer characteristics are age of onset, determined from the date of diagnosis, and histology, determined by anatomo-pathological diagnosis.	through study completion, an average of 6 months after patient inclusion
potential clinical-biological prognostic factors :first cancer treatment : types of treatments received	type of treatment: chemotherapy (Anthracyclines, Alkylating agents) and / or radiotherapy and/or Marrow autograft or allograft	through study completion, an average of 6 months after patient inclusion
potential clinical-biological prognostic factors - first cancer treatments: response to treatment	response measured by bone marrow assessment using morphology and minimal residual disease (MRD) assessment : no response, partial response, complete remission	through study completion, an average of 6 months after patient inclusion
potential clinical-biological prognostic factors - first cancer treatments: Number of participants with treatment-related adverse events as assessed by CTCAE v4.0	nature, incidence and severity of adverse events (AEs)	through study completion, an average of 6 months after patient inclusion
potential clinical-biological prognostic factors - first cancer treatments: duration of treatment	duration of treatment determined from the start and end dates	through study completion, an average of 6 months after patient inclusion
potential clinical-biological prognostic factors - first cancer treatments: cumulative dose received	cumulative dose of each type of treatment received ( chemotherapy (Anthracyclines, Alkylating agents) and / or radiotherapy and/or Marrow autograft or allograft)	through study completion, an average of 6 months after patient inclusion
potential clinical-biological prognostic factors - secondary AML characteristics: date of diagnosis	date of diagnosis	through study completion, an average of 6 months after patient inclusion
potential clinical-biological prognostic factors - secondary AML characteristics: median time of onset compared to the date of end of treatment for the 1st cancer	median time of onset compared to the date of end of treatment for the 1st cancer	through study completion, an average of 6 months after patient inclusion
potential clinical-biological prognostic factors - secondary AML characteristics : hematological data assessed by morphology	Leukocytes at diagnosis of secondary AML (G/L)	through study completion, an average of 6 months after patient inclusion
potential clinical-biological prognostic factors - secondary AML characteristics: cytogenetic data	karyotype, exclusive and cumulative anomalies	through study completion, an average of 6 months after patient inclusion
potential clinical-biological prognostic factors - secondary AML characteristics: molecular data	molecular data assessed by Next-Generation Sequencing panel	through study completion, an average of 6 months after patient inclusion
potential clinical-biological prognostic factors - secondary AML treatment: duration of treatment	duration of treatment determined from the start and end dates	through study completion, an average of 6 months after patient inclusion
potential clinical-biological prognostic factors - secondary AML treatment: type of treatment	chemotherapy received and bone marrow transplantation	through study completion, an average of 6 months after patient inclusion
potential clinical-biological prognostic factors - secondary AML treatment : response to treatment	response measured by bone marrow assessment using morphology and minimal residual disease (MRD) assessment : no response, partial response, complete remission	through study completion, an average of 6 months after patient inclusion
potential clinical-biological prognostic factors - secondary AML treatment: Number of participants with treatment-related adverse events as assessed by CTCAE v4.0	nature, incidence and severity of adverse events (AEs)	through study completion, an average of 6 months after patient inclusion
potential clinical-biological prognostic factors - Overall Survival (OS)	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	at the end of the 2 years of the inclusion period

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
feasibility of setting up a French national database of secondary AMLs for children and adolescents - Number of cases included over the period	Number of cases included over the period	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 - Missing Data	Proportion and type of missing data in the database after the base freeze	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	Centre participation rates	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	Recurrence of the disease : Relapse criteria (in a patient who has had complete remission): ≥ 5% blasts in the bone marrow (not attributable to post chemotherapy regeneration)	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	Treatment-related toxicities : Metabolic / endocrine complications in particular: growth retardation, hypothyroidism, gonadal insufficiency; Organic complications, in particular: renal failure, heart failure, respiratory failure ; Graft versus host reaction (GvH)	at the end of the 2 years of the inclusion period

Collaborators and Investigators

• Sponsor: University Hospital, Bordeaux

Study record dates

Study Major Dates

• Study Start (ANTICIPATED): September 1, 2020
• Primary Completion (ANTICIPATED): September 1, 2024
• Study Completion (ANTICIPATED): September 1, 2024

Study Registration Dates

• First Submitted: June 9, 2020
• First Submitted That Met QC Criteria: June 24, 2020
• First Posted (ACTUAL): June 30, 2020

Study Record Updates

• Last Update Posted (ACTUAL): July 1, 2020
• Last Update Submitted That Met QC Criteria: June 29, 2020
• Last Verified: June 1, 2020

More Information

Terms related to this study

• Keywords: Children; Secondary; Acute myeloid Leukemia
• Additional Relevant MeSH Terms: Neoplasms by Histologic Type; Neoplasms; Leukemia; Leukemia, Myeloid; Leukemia, Myeloid, Acute
• Other Study ID Numbers: CHUBX 2018/31

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No