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.

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