Pediatric Patients with Stage IV Rhabdomyosarcoma Significantly Benefit from Long-Term Maintenance Therapy: Results of the CWS-IV 2002 and the CWS DOK IV 2004-Trials
Rhabdomyosarcoma (RMS) is the most common soft tissue sarcoma (STS) in childhood. Whereas more than 90% of patients with localized low-risk RMS can be cured, metastatic RMS have a dismal outcome, with survival rates of less than 30%. The HD CWS-96 trial showed an improved outcome for patients receiv...
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| Format: | Article |
| Language: | English |
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MDPI AG
2023-03-01
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| Series: | Cancers |
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| Online Access: | https://www.mdpi.com/2072-6694/15/7/2050 |
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| author | Lars Tramsen Konrad Bochennek Monika Sparber-Sauer Emilia Salzmann-Manrique Monika Scheer Tobias Dantonello Arndt Borkhardt Uta Dirksen Anne Thorwarth Jeanette Greiner Martin Ebinger Jadwiga Weclawek-Tompol Ruth Ladenstein Gustaf Ljungman Erika Hallmen Thomas Lehrnbecher Ewa Koscielniak Thomas Klingebiel |
| author_facet | Lars Tramsen Konrad Bochennek Monika Sparber-Sauer Emilia Salzmann-Manrique Monika Scheer Tobias Dantonello Arndt Borkhardt Uta Dirksen Anne Thorwarth Jeanette Greiner Martin Ebinger Jadwiga Weclawek-Tompol Ruth Ladenstein Gustaf Ljungman Erika Hallmen Thomas Lehrnbecher Ewa Koscielniak Thomas Klingebiel |
| author_sort | Lars Tramsen |
| collection | DOAJ |
| description | Rhabdomyosarcoma (RMS) is the most common soft tissue sarcoma (STS) in childhood. Whereas more than 90% of patients with localized low-risk RMS can be cured, metastatic RMS have a dismal outcome, with survival rates of less than 30%. The HD CWS-96 trial showed an improved outcome for patients receiving maintenance therapy after completing intensive chemotherapy. Consequently, the international clinical trials CWS-IV 2002 and CWS DOK IV 2004 on metastatic disease of STS of the Cooperative Weichteilsarkom Studiengruppe (CWS) were designed in addition to the CWS-2002P trial for localized RMS disease. All patients received a multimodal intensive treatment regimen. To maintain remission, three options were compared: long-term maintenance therapy (LTMT) versus allogeneic hematopoietic stem cell transplantation (alloHSCT) versus high-dose chemotherapy (HDCT). A total of 176 pediatric patients with a histologically confirmed diagnosis of metastatic RMS or RMS-like tumor were included. A total of 89 patients receiving LTML showed a significantly better outcome, with an event-free survival (EFS) of 41% and an overall survival (OS) of 53%, than alloHSCT (<i>n</i> = 21, EFS 19%, <i>p</i> = 0.02, OS 24%, <i>p</i> = 0.002). The outcome of LTML was slightly improved compared to HDCT (<i>n</i> = 13, EFS 35%, OS 34%). In conclusion, our data suggest that in patients suffering from metastatic RMS, long-term maintenance therapy is a superior strategy in terms of EFS and OS compared to alloHSCT. EFS and OS of HDCT are similar in these strategies; however, the therapeutic burden of LTMT is much lower. |
| first_indexed | 2024-03-11T05:41:15Z |
| format | Article |
| id | doaj.art-bb14379d8b3c4a5ca8ae45d1f21c1f44 |
| institution | Directory Open Access Journal |
| issn | 2072-6694 |
| language | English |
| last_indexed | 2024-03-11T05:41:15Z |
| publishDate | 2023-03-01 |
| publisher | MDPI AG |
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| series | Cancers |
| spelling | doaj.art-bb14379d8b3c4a5ca8ae45d1f21c1f442023-11-17T16:25:21ZengMDPI AGCancers2072-66942023-03-01157205010.3390/cancers15072050Pediatric Patients with Stage IV Rhabdomyosarcoma Significantly Benefit from Long-Term Maintenance Therapy: Results of the CWS-IV 2002 and the CWS DOK IV 2004-TrialsLars Tramsen0Konrad Bochennek1Monika Sparber-Sauer2Emilia Salzmann-Manrique3Monika Scheer4Tobias Dantonello5Arndt Borkhardt6Uta Dirksen7Anne Thorwarth8Jeanette Greiner9Martin Ebinger10Jadwiga Weclawek-Tompol11Ruth Ladenstein12Gustaf Ljungman13Erika Hallmen14Thomas Lehrnbecher15Ewa Koscielniak16Thomas Klingebiel17Department of Pediatrics, University Hospital Schleswig-Holstein, Campus Kiel, 24105 Kiel, GermanyDepartment for Children and Adolescents, University Hospital, Goethe-University, 60590 Frankfurt, GermanyCenter for Pediatric, Adolescent and Women’s Medicine, Pediatric 5 (Oncology, Hematology, Immunology), Hospital of the State Capital Stuttgart, Olgahospital, Stuttgart Cancer Center, 70174 Stuttgart, GermanyDepartment for Children and Adolescents, University Hospital, Goethe-University, 60590 Frankfurt, GermanyDepartment of Pediatric Oncology and Hematology, Charité–Universitätsmedizin Berlin, Augustenburger Platz 1, 13353 Berlin, GermanyDepartment of Pediatrics, Division of Pediatric Hematology and Oncology, Inselspital, Bern University Hospital, University of Bern, CH-3010 Bern, SwitzerlandDepartment of Pediatric Oncology, Hematology and Clinical Immunology, Medical Faculty, Heinrich Heine University, 40225 Duesseldorf, GermanyPediatrics III, University Hospital Essen, West German Cancer Center, 45147 Essen, GermanyDepartment of Pediatric Oncology and Hematology, Charité–Universitätsmedizin Berlin, Augustenburger Platz 1, 13353 Berlin, GermanyPediatric Oncology and Hematology, Children’s Hospital, Kantonsspital Aarau AG, CH-5001 Aarau, SwitzerlandDepartment of General Pediatrics and Pediatric Oncology and Hematology, University Children’s Hospital, 72076 Tuebingen, GermanyDepartment of Bone Marrow Transplantation, Pediatric Oncology and Haematology, University of Medicine Wroclaw, 50556 Wroclaw, PolandSt. Anna-Kinderspital, Children’s Cancer Research Institute (CCRI), 1090 Vienna, AustriaDepartment of Women’s and Children’s Health, Pediatric Oncology, Uppsala University, 75185 Uppsala, SwedenCenter for Pediatric, Adolescent and Women’s Medicine, Pediatric 5 (Oncology, Hematology, Immunology), Hospital of the State Capital Stuttgart, Olgahospital, Stuttgart Cancer Center, 70174 Stuttgart, GermanyDepartment for Children and Adolescents, University Hospital, Goethe-University, 60590 Frankfurt, GermanyCenter for Pediatric, Adolescent and Women’s Medicine, Pediatric 5 (Oncology, Hematology, Immunology), Hospital of the State Capital Stuttgart, Olgahospital, Stuttgart Cancer Center, 70174 Stuttgart, GermanyDepartment for Children and Adolescents, University Hospital, Goethe-University, 60590 Frankfurt, GermanyRhabdomyosarcoma (RMS) is the most common soft tissue sarcoma (STS) in childhood. Whereas more than 90% of patients with localized low-risk RMS can be cured, metastatic RMS have a dismal outcome, with survival rates of less than 30%. The HD CWS-96 trial showed an improved outcome for patients receiving maintenance therapy after completing intensive chemotherapy. Consequently, the international clinical trials CWS-IV 2002 and CWS DOK IV 2004 on metastatic disease of STS of the Cooperative Weichteilsarkom Studiengruppe (CWS) were designed in addition to the CWS-2002P trial for localized RMS disease. All patients received a multimodal intensive treatment regimen. To maintain remission, three options were compared: long-term maintenance therapy (LTMT) versus allogeneic hematopoietic stem cell transplantation (alloHSCT) versus high-dose chemotherapy (HDCT). A total of 176 pediatric patients with a histologically confirmed diagnosis of metastatic RMS or RMS-like tumor were included. A total of 89 patients receiving LTML showed a significantly better outcome, with an event-free survival (EFS) of 41% and an overall survival (OS) of 53%, than alloHSCT (<i>n</i> = 21, EFS 19%, <i>p</i> = 0.02, OS 24%, <i>p</i> = 0.002). The outcome of LTML was slightly improved compared to HDCT (<i>n</i> = 13, EFS 35%, OS 34%). In conclusion, our data suggest that in patients suffering from metastatic RMS, long-term maintenance therapy is a superior strategy in terms of EFS and OS compared to alloHSCT. EFS and OS of HDCT are similar in these strategies; however, the therapeutic burden of LTMT is much lower.https://www.mdpi.com/2072-6694/15/7/2050soft tissue sarcomarhabdomyosarcomametastaticchildrenlong-term maintenance therapyoutcome |
| spellingShingle | Lars Tramsen Konrad Bochennek Monika Sparber-Sauer Emilia Salzmann-Manrique Monika Scheer Tobias Dantonello Arndt Borkhardt Uta Dirksen Anne Thorwarth Jeanette Greiner Martin Ebinger Jadwiga Weclawek-Tompol Ruth Ladenstein Gustaf Ljungman Erika Hallmen Thomas Lehrnbecher Ewa Koscielniak Thomas Klingebiel Pediatric Patients with Stage IV Rhabdomyosarcoma Significantly Benefit from Long-Term Maintenance Therapy: Results of the CWS-IV 2002 and the CWS DOK IV 2004-Trials Cancers soft tissue sarcoma rhabdomyosarcoma metastatic children long-term maintenance therapy outcome |
| title | Pediatric Patients with Stage IV Rhabdomyosarcoma Significantly Benefit from Long-Term Maintenance Therapy: Results of the CWS-IV 2002 and the CWS DOK IV 2004-Trials |
| title_full | Pediatric Patients with Stage IV Rhabdomyosarcoma Significantly Benefit from Long-Term Maintenance Therapy: Results of the CWS-IV 2002 and the CWS DOK IV 2004-Trials |
| title_fullStr | Pediatric Patients with Stage IV Rhabdomyosarcoma Significantly Benefit from Long-Term Maintenance Therapy: Results of the CWS-IV 2002 and the CWS DOK IV 2004-Trials |
| title_full_unstemmed | Pediatric Patients with Stage IV Rhabdomyosarcoma Significantly Benefit from Long-Term Maintenance Therapy: Results of the CWS-IV 2002 and the CWS DOK IV 2004-Trials |
| title_short | Pediatric Patients with Stage IV Rhabdomyosarcoma Significantly Benefit from Long-Term Maintenance Therapy: Results of the CWS-IV 2002 and the CWS DOK IV 2004-Trials |
| title_sort | pediatric patients with stage iv rhabdomyosarcoma significantly benefit from long term maintenance therapy results of the cws iv 2002 and the cws dok iv 2004 trials |
| topic | soft tissue sarcoma rhabdomyosarcoma metastatic children long-term maintenance therapy outcome |
| url | https://www.mdpi.com/2072-6694/15/7/2050 |
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