Melphalan, Etoposide, and Carboplatin Megatherapy with Autologous Stem Cell Transplantation in Children with Relapsing or Therapy-Resistant Extracranial Germ-Cell Tumors—A Retrospective Analysis

Pediatric germ cell tumors (GCTs) are a group of chemosensitive malignancies with a 90% curability rate. We report a series of children with relapsing or therapy-resistant GCT treated with melphalan–etoposide–carboplatin high-dose chemotherapy (HDCT) and autologous stem cell transplantation. This co...

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Bibliographic Details
Main Authors: Marek Ussowicz, Monika Mielcarek-Siedziuk, Jakub Musiał, Mateusz Stachowiak, Jadwiga Węcławek-Tompol, Dorota Sęga-Pondel, Jowita Frączkiewicz, Joanna Trelińska, Anna Raciborska
Format: Article
Language:English
Published: MDPI AG 2020-12-01
Series:Cancers
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Online Access:https://www.mdpi.com/2072-6694/12/12/3841
Description
Summary:Pediatric germ cell tumors (GCTs) are a group of chemosensitive malignancies with a 90% curability rate. We report a series of children with relapsing or therapy-resistant GCT treated with melphalan–etoposide–carboplatin high-dose chemotherapy (HDCT) and autologous stem cell transplantation. This consisted of 18 children, either with GCTs after relapse (nine patients) or with an unsatisfactory response to first-line chemotherapy (nine patients), who underwent HDCT. The HDCT regimens MEC1 (carboplatin 1500 mg/m<sup>2</sup>, etoposide 1800 mg/m<sup>2</sup>, and melphalan 140 mg/m<sup>2</sup>) and MEC2 (carboplatin 800 mg/m<sup>2</sup>, etoposide 800 mg/m<sup>2</sup>, and melphalan 140 mg/m<sup>2</sup>) were each used in nine patients. The median observation time was 81 months, the 5-year overall survival (OS) was 76%, and the event-free survival (EFS) was 70.8%. Non-relapse mortality was 0%, and four patients died after HDCT due to progression of the malignancy. No difference in OS or EFS was noted between the MEC1 and MEC2 protocols. The 5-year OS and 5-year EFS were higher in children treated with autologous stem cell transplantation before the age of four years. The presence of metastatic disease or time of HDCT consolidation during first/subsequent line chemotherapy did not affect patient survival. The melphalan–etoposide–carboplatin protocol is feasible in pediatric GCT, but is associated with potentially life-threatening complications. In conclusion, the use of HDCT must be examined in well-designed clinical trials, and the identification of patients who can benefit from this approach is critical to avoid overtreatment.
ISSN:2072-6694