CML Pediatric ITK Response According to Molecular Identification at Diagnosis
CML Pediatric ITK Response According to Molecular Identification at Diagnosis (CML Piramid
University Hospital, Bordeaux
88 participants
Feb 27, 2023
OBSERVATIONAL
Conditions
Summary
Treatment of chronic myeloid leukemia (CML) has been revolutionized by tyrosine kinase inhibitor (TKI). Nevertheless, case of failure and suboptimal response are still observed even in children. Pediatric CML is a rare disease and differs from adult in terms of disease presentation and treatment response underlying a likely different CML biology. Molecular mechanisms that induce resistance to TKI are still poorly characterized except mutations in the tyrosine kinase domain of BCR::ABL1. We propose to search for a molecular signature to predict the response to TKI in the pediatric population.
Eligibility
Inclusion Criteria7
- Age at diagnosis less than or equal to 18 years
- Presence of a Philadelphia chromosome detected by cytogenetic analysis (conventional karyotype or Fluorescence In Situ Hybridization (FISH)) and a BCR ::ABL1 transcript e13a2 ou e14a2
- Diagnosis in chronic phase according to the European Leukemia Net (ELN) criteria
- First-line treatment with TKIs
- Possible pre-treatment with hydroxyurea
- DNA available at diagnosis
- RNA available for a sub-group patients (8 responders vs 8 no responders)
Exclusion Criteria3
- Age at diagnosis more than 18 years
- Diagnosis in accelerated phase or blastic phase
- First line treatment other than TKI
Interventions
Targeted Next Generation Sequencing (DNA and RNA)
Locations(1)
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NCT05605379