Dasatinib in pediatric patients with chronic myeloid leukemia in chronic phase: long-term efficacy and endocrine analysis
In brief
Dasatinib brought deep molecular responses to up to 55% of children
In this phase 2 trial, after about eight years, 55% of newly diagnosed children had a deep molecular response; the rate was 38% among children whose leukemia resisted or did not tolerate imatinib. Height scores fell most in prepubertal children, while other reported endocrine effects were modest. The findings support durable disease control, but the growth effects warrant attention during long-term treatment.
- Journal
- Blood advances (Q1)
- Published
- 5 November 2026
- Study design
- Non-randomized / quasi-experimental trial
- Evidence level
- Level 2, Moderate (CEBM 2b)
- Authors
- Jill Samis, Briana C Patterson, Lia Gore, C Michel Zwaan, Patricia Martín-Regueira, Yu Tian, et al.
- PMID
- 42987920
- DOI
- 10.1182/bloodadvances.2026021359
Why clinicians should know about it
- Picked for Hematology (paper of the day, 6 November 2026): Long-term dasatinib efficacy and endocrine effects in pediatric CML
Abstract
Chronic myeloid leukemia (CML) accounts for 2-3% of childhood leukemias. CA180 226 is a prospective phase 2 trial evaluating dasatinib in pediatric patients with newly diagnosed (ND) or imatinib resistant/intolerant (IM R/I) chronic phase CML (CML CP). Analyses showed rapid molecular responses, but long term efficacy, deep molecular responses (DMR), and endocrine effects of tyrosine kinase inhibitors (TKIs) in children have not been reported. This study reports long term outcomes in the largest pediatric cohort to date with endocrine assessment during TKI therapy. ND patients received dasatinib tablets (Cohort 3A) or oral suspension (Cohort 3B), while IM R/I patients were enrolled in Cohort 1. Cohorts 3A and 1 received 60 mg/m² daily; Cohort 3B received 72 mg/m² daily. Median follow-up was 94.8 months for Cohort 3 and 92.4 months for Cohort 1. Height z scores declined over the first few years of therapy particularly in prepubertal children, whereas BMI and bone density changes were mild. Thyroid and gonadal abnormalities were infrequent. Major molecular responses (MMR) occurred within the first 2 years, with 96 month MMR rates of 83.3% in Cohort 3 and 69.0% in Cohort 1. DMR accumulated, with MR4 and MR4.5 rates of 54.8% and 41.7% in Cohort 3 and 37.9% and 27.6% in Cohort 1 at 96 months, respectively. Median progression free and disease free survival were not reached. These long-term data demonstrate durable dasatinib efficacy to induce DMR alongside initial height z-score decline and modest additional endocrine effects, providing important insight into TKI therapy in pediatric CML-CP. NCT00777036.
Abstract as published, via PubMed.
For healthcare professionals. The summary is generated by AI from the published abstract, and the evidence level is assigned automatically from the study design on the Oxford CEBM hierarchy. Neither is medical advice. Read the full paper before changing practice.