Based on findings from the phase 1/2 BCHILD trial, the FDA approved bosutinib for pediatric chronic myelogenous leukemia.
This article was originally published by CancerNetwork.
The FDA has granted approval to bosutinib (Bosulif) for the treatment of pediatric patients aged 1 year or older with newly diagnosed or resistant/previous treatment intolerant chronic myelogenous leukemia (CML) that is Philadelphia chromosome (Ph) positive and in chronic phase, according to a press release from the FDA.1
Findings from the phase 1/2 BCHILD study (NCT04258943)—in which 28 patients with PH-positive resistant or intolerant CML were treated with 300 mg/m2 to 400 mg/m2 and 21 patients with newly diagnosed, chronic phase disease received 300 mg/m2—supported the approval.2 The recommended doses were 400 mg/m2 and 300 mg/m2, respectively.
In the newly diagnosed population, the major cytogenetic response rate was (MCyR) was 76.2% (95% CI, 52.8%-91.8%) and the complete cytogenetic response rate (CCyR) was 71.4% (95% CI, 47.8%-88.7%). Additionally, investigators reported a major molecular response (MMR) rate of 28.6% (95% CI, 11.3%-52.3%) with a median duration of response of 14.2 months (range, 1.1-26.3).
In the resistant/intolerant population, the MCyR and CCyR rates were 82.1% (95% CI, 63.1%-93.9%) and 78.6% (95% CI, 59.0%-91.7%), respectively. Moreover, investigators reported an MMR of 50.0% (95% CI, 30.6%-69.4%) in this population. Of 14 patients, 2 lost an MMR following 13.6 months and 24.7 months of treatment with bosutinib, respectively. The median follow-up was 23.2 months (range, 1-61.5 months).
In terms of safety, frequent adverse effects included diarrhea, abdominal pain, vomiting, nausea, rash, fatigue, hepatic dysfunction, headache, pyrexia, decreased appetite, and constipation. Additionally, investigators reported several frequent laboratory abnormalities, including increased creatinine, increased alanine aminotransferase or aspartate aminotransferase, decreased white blood cell count, and decreased platelet count.
References
1. FDA approves bosutinib for pediatric patients with chronic myelogenous leukemia. News release. FDA. September 26, 2023. Accessed September 27, 2023. https://bit.ly/46madME
2. Pennesi E, Brivio E, Willemse ME, et al. Preliminary results from the first-in-child phase II trial (ITCC-054/COG-AAML1921) of bosutinib in pediatric patients with newly diagnosed (ND) chronic myeloid leukemia (CML). J Clin Oncol. 2023; 41(supp 16):10017. doi:10.1200/JCO.2023.41.16_suppl.10017
CMS Medicare Final Rule: Advancing Benefits, Competition, and Consumer Protection
May 7th 2024On this episode of Managed Care Cast, we're talking with Karen Iapoce, senior director of government products and programs at ZeOmega, about the recent CMS final rule on Medicare Part D and Medicare Advantage.
Listen
Persistence Pays Off With Zanubrutinib: A Challenging CLL Case With a Prior BTK Inhibitor Failure
May 10th 2024The case of a 77-year-old woman with a long chronic lymphocytic leukemia (CLL) history illustrates the novel use of zanubrutinib as a potential option for some patients who have failed first-generation Bruton tyrosine kinase (BTK) inhibitors and venetoclax.
Read More
Posters Characterize DMD Caregiver Experiences, Impact of Gene Therapy on Caregiving Demands
May 10th 2024Posters presented at the ISPOR—The Professional Society for Health Economics and Outcomes Research meeting explored Duchenne muscular dystrophy (DMD) caregiver experiences and gene therapy’s impact on work opportunities for caregivers.
Read More