Sustained complete molecular remission after imatinib discontinuation in children with chronic myeloid leukemia
Olga Moser1, Manuela Krumbholz, Christian Thiede
1Department of Pediatric Hematology and Oncology, University Hospital Bonn, Bonn, Germany.
Insights
Approximately 40% of adults with chronic myeloid leukemia (CML) achieve sustained remission after stopping imatinib. This study explored similar possibilities in children, finding low-level disease fluctuations in two pediatric CML patients after treatment cessation.
Area of Science:
- Pediatric Hematology
- Oncology
- Clinical Research
Background:
- Complete molecular response (CMR) is achievable in adults with chronic myeloid leukemia (CML) on imatinib therapy.
- Approximately 40% of adults maintain CMR after imatinib discontinuation.
- Data on imatinib discontinuation in pediatric CML patients are limited.
Purpose of the Study:
- To investigate the feasibility of imatinib discontinuation in children with CML.
- To assess disease status in pediatric patients achieving CMR after imatinib cessation.
- To evaluate the potential for minimizing imatinib exposure and side effects in low-risk children.
Main Methods:
- Case study of two pediatric patients with CML in sustained CMR.
- Monitoring of disease levels via RNA transcript and genomic DNA.
- Assessment of patient risk stratification based on adult criteria.
Main Results:
- Two pediatric CML patients maintained CMR for 48 and 19 months post-imatinib discontinuation.
- Both patients exhibited low-level, fluctuating disease at molecular levels.
- Patients were classified as low-risk according to established adult criteria.
Conclusions:
- Imatinib discontinuation may be a viable option for select low-risk pediatric CML patients.
- Clinical trials are warranted to further evaluate treatment cessation in this population.
- Close monitoring is essential to manage potential molecular relapses and minimize long-term imatinib exposure.
Abstract:
Approximately 40% of adults with chronic myeloid leukemia (CML) in prolonged complete molecular response (CMR) remain in CMR after imatinib discontinuation. Corresponding information in children is lacking. Two children with CML in CMR for 48 and 19 months after imatinib discontinuation showed low-level fluctuating disease at RNA transcript and genomic DNA levels. Both patients were low risk according to adult criteria. Since adults with molecular relapse responded to re-introduction of imatinib, we postulated that treatment discontinuation in low risk children might be justified within clinical trials with close monitoring. This may help to minimize exposure to imatinib and its potential side effects.
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