CASE REPORT article
Front. Oncol.
Sec. Pediatric Oncology
Volume 15 - 2025 | doi: 10.3389/fonc.2025.1637177
This article is part of the Research TopicCase Reports in Pediatric Oncology: 2025View all articles
Case Report: Relapsed alveolar rhabdomyosarcoma treated with abemaciclib, temozolomide, and irinotecan in the JPCS study
Provisionally accepted
Antonio Juan Ribelles1*
Nuria Benavent2Daniel Sanchez Mateos1Celine Pitou3Yanhong Zhou3Molly C. Hardebeck3Holly Knoderer3Adela Cañete1- 1Hospital Universitari i Politecnic La Fe, Valencia, Spain
- 2Instituto de Investigacion Sanitaria La Fe, Valencia, Spain
- 3Eli Lilly and Company, Indianapolis, United States
Select one of your emails
You have multiple emails registered with Frontiers:
Notify me on publication
Please enter your email address:
If you already have an account, please login
You don't have a Frontiers account ? You can register here
Background: Cyclin-dependent kinase (CDK) 4 and CDK6 play fundamental roles in cell cycle progression. The CDK4/6 inhibitor abemaciclib, in combination with temozolomide and irinotecan, was evaluated in pediatric and young adult patients with relapsed/refractory solid tumors in the phase 1b dose-escalation study, JPCS Part A (NCT04238819). This case report describes the notable results of a patient with relapsed alveolar rhabdomyosarcoma (ARMS) who experienced a prolonged complete response.Case presentation: An 8-year-old White male was initially diagnosed with stage IV ARMS with PAX3-FOXO1 fusion. Molecular characterization following a fourth relapse revealed CDK4, ERBB3, GLI1, MYCN, and FGFR4 amplifications and MYCN mutation. After five relapses, the patient enrolled in JPCS Part A and received abemaciclib (55 mg/m 2 twice daily continuously) in combination with temozolomide (100 mg/m 2 daily) and irinotecan (50 mg/m 2 daily) on days 1 to 5 of 21-day cycles. The patient received 12 cycles of the triplet combination, followed by 23 additional cycles of abemaciclib monotherapy. Complete response (CR) was achieved in less than 3 months, with a duration of response (DOR) of 22.6 months and progression-free survival (PFS) of 23.7 months. The study treatment was well tolerated.CDK4/6 inhibition with abemaciclib in combination with temozolomide and irinotecan provided a durable response in a patient with heavily pretreated ARMS. Additional studies may be warranted to further understand the role of CDK4/6 inhibitors for treatment of ARMS.
Keywords: Abemaciclib, CDK4/6 inhibitor, Rhabdomyosarcoma, JPCS, pediatric, case report
Received: 28 May 2025; Accepted: 31 Jul 2025.
Copyright: © 2025 Juan Ribelles, Benavent, Sanchez Mateos, Pitou, Zhou, Hardebeck, Knoderer and Cañete. This is an open-access article distributed under the terms of the Creative Commons Attribution License (CC BY). The use, distribution or reproduction in other forums is permitted, provided the original author(s) or licensor are credited and that the original publication in this journal is cited, in accordance with accepted academic practice. No use, distribution or reproduction is permitted which does not comply with these terms.
* Correspondence: Antonio Juan Ribelles, Hospital Universitari i Politecnic La Fe, Valencia, Spain
Disclaimer: All claims expressed in this article are solely those of the authors and do not necessarily represent those of their affiliated organizations, or those of the publisher, the editors and the reviewers. Any product that may be evaluated in this article or claim that may be made by its manufacturer is not guaranteed or endorsed by the publisher.