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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MDPI AG
2020-12-01
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| Series: | Cancers |
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| Online Access: | https://www.mdpi.com/2072-6694/12/12/3841 |
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| author | Marek Ussowicz Monika Mielcarek-Siedziuk Jakub Musiał Mateusz Stachowiak Jadwiga Węcławek-Tompol Dorota Sęga-Pondel Jowita Frączkiewicz Joanna Trelińska Anna Raciborska |
| author_facet | Marek Ussowicz Monika Mielcarek-Siedziuk Jakub Musiał Mateusz Stachowiak Jadwiga Węcławek-Tompol Dorota Sęga-Pondel Jowita Frączkiewicz Joanna Trelińska Anna Raciborska |
| author_sort | Marek Ussowicz |
| collection | DOAJ |
| description | 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. |
| first_indexed | 2024-03-10T13:55:31Z |
| format | Article |
| id | doaj.art-bea7def8dc46433588644f3fc8e3820b |
| institution | Directory Open Access Journal |
| issn | 2072-6694 |
| language | English |
| last_indexed | 2024-03-10T13:55:31Z |
| publishDate | 2020-12-01 |
| publisher | MDPI AG |
| record_format | Article |
| series | Cancers |
| spelling | doaj.art-bea7def8dc46433588644f3fc8e3820b2023-11-21T01:40:48ZengMDPI AGCancers2072-66942020-12-011212384110.3390/cancers12123841Melphalan, Etoposide, and Carboplatin Megatherapy with Autologous Stem Cell Transplantation in Children with Relapsing or Therapy-Resistant Extracranial Germ-Cell Tumors—A Retrospective AnalysisMarek Ussowicz0Monika Mielcarek-Siedziuk1Jakub Musiał2Mateusz Stachowiak3Jadwiga Węcławek-Tompol4Dorota Sęga-Pondel5Jowita Frączkiewicz6Joanna Trelińska7Anna Raciborska8Department of Paediatric Bone Marrow Transplantation, Oncology and Hematology, Wrocław Medical University, 50-367 Wrocław, PolandDepartment of Paediatric Bone Marrow Transplantation, Oncology and Hematology, Wrocław Medical University, 50-367 Wrocław, PolandDepartment of Pediatric Oncology and Hematology, Clinical Hospital No. 2, 35-301 Rzeszów, PolandDepartment of Paediatric Bone Marrow Transplantation, Oncology and Hematology, Wrocław Medical University, 50-367 Wrocław, PolandDepartment of Paediatric Bone Marrow Transplantation, Oncology and Hematology, Wrocław Medical University, 50-367 Wrocław, PolandDepartment of Paediatric Bone Marrow Transplantation, Oncology and Hematology, Wrocław Medical University, 50-367 Wrocław, PolandDepartment of Paediatric Bone Marrow Transplantation, Oncology and Hematology, Wrocław Medical University, 50-367 Wrocław, PolandDepartment of Pediatrics, Oncology, Hematology and Diabetology, Medical University of Lodz, 91-738 Łódź, PolandDepartment of Oncology and Surgical Oncology for Children and Youth, Institute of Mother and Child, 01-211 Warsaw, PolandPediatric 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.https://www.mdpi.com/2072-6694/12/12/3841germ cell tumorshigh dose chemotherapyautologous stem cell transplantation |
| spellingShingle | Marek Ussowicz Monika Mielcarek-Siedziuk Jakub Musiał Mateusz Stachowiak Jadwiga Węcławek-Tompol Dorota Sęga-Pondel Jowita Frączkiewicz Joanna Trelińska Anna Raciborska Melphalan, Etoposide, and Carboplatin Megatherapy with Autologous Stem Cell Transplantation in Children with Relapsing or Therapy-Resistant Extracranial Germ-Cell Tumors—A Retrospective Analysis Cancers germ cell tumors high dose chemotherapy autologous stem cell transplantation |
| title | Melphalan, Etoposide, and Carboplatin Megatherapy with Autologous Stem Cell Transplantation in Children with Relapsing or Therapy-Resistant Extracranial Germ-Cell Tumors—A Retrospective Analysis |
| title_full | Melphalan, Etoposide, and Carboplatin Megatherapy with Autologous Stem Cell Transplantation in Children with Relapsing or Therapy-Resistant Extracranial Germ-Cell Tumors—A Retrospective Analysis |
| title_fullStr | Melphalan, Etoposide, and Carboplatin Megatherapy with Autologous Stem Cell Transplantation in Children with Relapsing or Therapy-Resistant Extracranial Germ-Cell Tumors—A Retrospective Analysis |
| title_full_unstemmed | Melphalan, Etoposide, and Carboplatin Megatherapy with Autologous Stem Cell Transplantation in Children with Relapsing or Therapy-Resistant Extracranial Germ-Cell Tumors—A Retrospective Analysis |
| title_short | Melphalan, Etoposide, and Carboplatin Megatherapy with Autologous Stem Cell Transplantation in Children with Relapsing or Therapy-Resistant Extracranial Germ-Cell Tumors—A Retrospective Analysis |
| title_sort | melphalan etoposide and carboplatin megatherapy with autologous stem cell transplantation in children with relapsing or therapy resistant extracranial germ cell tumors a retrospective analysis |
| topic | germ cell tumors high dose chemotherapy autologous stem cell transplantation |
| url | https://www.mdpi.com/2072-6694/12/12/3841 |
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