Pediatric patients with chronic myelogenous leukemia (CML) may have attenuated growth when receiving treatment with nilotinib, according to an updated assessment published in Blood Advances.

The phase 2 open-label DIALOG study found nilotinib to have sustained efficacy in patients with newly diagnosed or imatinib/dasatinib resistant/intolerant CML. The updated assessment evaluated growth attenuation and safety profiles of patients in the trial.

Patients included had either discontinued the study or had completed 48 or more treatment cycles (n=58). A total of 33 patients were in the resistant/intolerant (R/I) cohort and 25 were in the newly diagnosed cohort. 

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Both cohorts had a negative slope in height standard deviation score (SDS). Patients in the R/I cohort were shorter at baseline than those in the newly diagnosed cohort. After treatment with at least 48 cycles, patients in the R/I cohort had a median change in height SDS of -0.54, and patients in the newly diagnosed cohort had a median change of -0.91.

A total of 3 growth-related adverse events (AEs) were reported in the course of the study, despite a trend in growth deceleration. All patients in the study reported at least 1 AE, with increased blood bilirubin, headache, pyrexia, and increase in alamine aminotransferase as the most common.

This follow-up analysis found a safety profile consistent with previous results. The main finding from the longer-term analysis is a clear deceleration in growth over time. Growth-related AEs or growth deceleration may be underreported, as it is not a common calculation in clinical trials.

The authors recommend that future studies include analysis of growth data for patients with CML receiving nilotinib.

Disclosure: This research was supported by Novartis Pharmaceuticals Corporation. Please see the original reference for a full list of disclosures.


Hijiya N, Maschan A, Rizzari C, et al. A phase 2 study of nilotinib in pediatric patients with CML: long-term update on growth retardation and safety. Blood Adv. 2021;5(14):2925-2934. doi:10.1182/bloodadvances.2020003759