Imatinib treatment and longitudinal growth in pediatric patients with chronic myeloid leukemia: influence of demographic, pharmacological, and genetic factors in the German CML-PAED cohort

Haematologica. 2024 Aug 1;109(8):2555-2563. doi: 10.3324/haematol.2023.284668.

Abstract

In children and adolescents, impaired growth due to tyrosine kinase inhibitor therapy remains an insufficiently studied adverse effect. This study examines demographic, pharmacological, and genetic factors associated with impaired longitudinal growth in a uniform pediatric cohort treated with imatinib. We analyzed 94 pediatric patients with chronic myeloid leukemia (CML) diagnosed in the chronic phase and treated with imatinib for >12 months who participated in the Germany-wide CML-PAEDII study between February 2006 and February 2021 (clinicaltrials gov. Identifier: NCT00445822). During imatinib treatment, significant height reduction occurred, with medians of -0.35 standard deviation score (SDS) at 12 months and -0.76 SDS at 24 months. Cumulative height SDS change (Δ height SDS) showed a more pronounced effect in prepubertal patients during the first year but were similar between prepubertal and pubertal subgroups by the second year (-0.55 vs. -0.50). From months 12 to 18 on imatinib, only 18% patients achieved individually longitudinal growth adequate to the growth standard (Δ height SDS ≥0). When patients were divided into two subgroups based on median Δ height SDS (classifier Δ height SDS > or ≤-0.37) after 1 year on imatinib therapy, cohort 1 (Δ height SDS ≤-0.37) showed younger age at diagnosis, a higher proportion of prepubertal children, but also better treatment response and higher imatinib serum levels. Exploring the association of growth parameters with pharmacokinetically relevant single nucleotide polymorphisms, known for affecting imatinib response, showed no correlation. This retrospective study provides new insights into imatinib-related growth impairment. We emphasize the importance of optimizing treatment strategies for pediatric patients to realize their maximum growth potential.

Publication types

  • Clinical Trial
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adolescent
  • Antineoplastic Agents / adverse effects
  • Antineoplastic Agents / therapeutic use
  • Body Height / drug effects
  • Child
  • Child, Preschool
  • Female
  • Germany / epidemiology
  • Humans
  • Imatinib Mesylate* / therapeutic use
  • Infant
  • Leukemia, Myelogenous, Chronic, BCR-ABL Positive* / drug therapy
  • Leukemia, Myelogenous, Chronic, BCR-ABL Positive* / genetics
  • Longitudinal Studies
  • Male
  • Protein Kinase Inhibitors / adverse effects
  • Protein Kinase Inhibitors / therapeutic use
  • Retrospective Studies
  • Treatment Outcome

Substances

  • Antineoplastic Agents
  • Imatinib Mesylate
  • Protein Kinase Inhibitors

Associated data

  • ClinicalTrials.gov/NCT00445822

Grants and funding

Funding: The study was supported by the Interdisciplinary Center for Clinical Research (IZKF) at the University Hospital of the University of Erlangen-Nürnberg (Laboratory Rotation, to SSe). The continuous financial support in data collection by “Sonnenstrahl e.V. Dresden - Förderkreis für krebskranke Kinder und Jugendliche” (Dresden, Germany), “Schornsteinfeger helfen krebskranken Kindern e.V.” (Dörfles-Esbach, Germany), and the “Madeleine-Schickedanz-Kinderkrebs-stiftung” (Fürth, Germany) is highly acknowledged.