Targeted Therapy with Dalpiciclib in a Pediatric Patient with Anaplastic Ependymoma: A Case Study and Literature Review

Introduction: Ependymoma is the third most common brain tumor in children, with a high recurrence rate and poor prognosis. The traditional treatment of ependymoma is surgery and radiation therapy. However, the effectiveness of conventional chemotherapy has been modest. Recent breakthroughs in molecular biology and genetics have opened doors to more targeted and effective therapeutic approaches. Complex mutations, such as CDK4 amplification in anaplastic ependymoma, are infrequently documented, warranting further investigation. Case Presentations: This case study presents a twelve-year-old girl with a WHO Grade III anaplastic ependymoma. She underwent two surgical procedures, followed by radiotherapy and chemotherapy. Despite this comprehensive treatment, she experienced a relapse after one year. Genetic testing identified CDK4 amplification in the tumor tissue. Subsequently, based on the genetic finding, she was treated with a CDK4/6 inhibitor, Dalpiciclib, in combination with bevacizumab. So far, she has survived for over 48 months following her surgery and continues to be monitored, underscoring the remarkable efficacy of the treatment. Conclusions: This report represents the first use of the CDK4/6 inhibitor Dalpiciclib for the treatment of anaplastic ependymoma. The treatment was guided by insights from next-generation sequencing (NGS) analysis and has shown promising results in terms of patient survival.