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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
SIMPLE SUMMARY: Patients with a metastatic rhabdomyosarcoma treated with a long-term maintenance therapy (LTMT) as a final additional treatment element show a similar outcome compared to a final high-dose chemotherapy regimen and a significantly better outcome compared to patients receiving an allog...
Autores principales: | , , , , , , , , , , , , , , , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
MDPI
2023
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10093505/ https://www.ncbi.nlm.nih.gov/pubmed/37046711 http://dx.doi.org/10.3390/cancers15072050 |
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author | Tramsen, Lars Bochennek, Konrad Sparber-Sauer, Monika Salzmann-Manrique, Emilia Scheer, Monika Dantonello, Tobias Borkhardt, Arndt Dirksen, Uta Thorwarth, Anne Greiner, Jeanette Ebinger, Martin Weclawek-Tompol, Jadwiga Ladenstein, Ruth Ljungman, Gustaf Hallmen, Erika Lehrnbecher, Thomas Koscielniak, Ewa Klingebiel, Thomas |
author_facet | Tramsen, Lars Bochennek, Konrad Sparber-Sauer, Monika Salzmann-Manrique, Emilia Scheer, Monika Dantonello, Tobias Borkhardt, Arndt Dirksen, Uta Thorwarth, Anne Greiner, Jeanette Ebinger, Martin Weclawek-Tompol, Jadwiga Ladenstein, Ruth Ljungman, Gustaf Hallmen, Erika Lehrnbecher, Thomas Koscielniak, Ewa Klingebiel, Thomas |
author_sort | Tramsen, Lars |
collection | PubMed |
description | SIMPLE SUMMARY: Patients with a metastatic rhabdomyosarcoma treated with a long-term maintenance therapy (LTMT) as a final additional treatment element show a similar outcome compared to a final high-dose chemotherapy regimen and a significantly better outcome compared to patients receiving an allogeneic hematopoietic stem cell transplantation. Particularly, special subgroups such as the very high-risk group defined by alveolar RMS and an Oberlin risk score of ≥ 2 seem to benefit from the LTMT, as they exhibit an overall survival of 37%, which has not been shown before. ABSTRACT: 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 (n = 21, EFS 19%, p = 0.02, OS 24%, p = 0.002). The outcome of LTML was slightly improved compared to HDCT (n = 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. |
format | Online Article Text |
id | pubmed-10093505 |
institution | National Center for Biotechnology Information |
language | English |
publishDate | 2023 |
publisher | MDPI |
record_format | MEDLINE/PubMed |
spelling | pubmed-100935052023-04-13 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 Tramsen, Lars Bochennek, Konrad Sparber-Sauer, Monika Salzmann-Manrique, Emilia Scheer, Monika Dantonello, Tobias Borkhardt, Arndt Dirksen, Uta Thorwarth, Anne Greiner, Jeanette Ebinger, Martin Weclawek-Tompol, Jadwiga Ladenstein, Ruth Ljungman, Gustaf Hallmen, Erika Lehrnbecher, Thomas Koscielniak, Ewa Klingebiel, Thomas Cancers (Basel) Article SIMPLE SUMMARY: Patients with a metastatic rhabdomyosarcoma treated with a long-term maintenance therapy (LTMT) as a final additional treatment element show a similar outcome compared to a final high-dose chemotherapy regimen and a significantly better outcome compared to patients receiving an allogeneic hematopoietic stem cell transplantation. Particularly, special subgroups such as the very high-risk group defined by alveolar RMS and an Oberlin risk score of ≥ 2 seem to benefit from the LTMT, as they exhibit an overall survival of 37%, which has not been shown before. ABSTRACT: 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 (n = 21, EFS 19%, p = 0.02, OS 24%, p = 0.002). The outcome of LTML was slightly improved compared to HDCT (n = 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. MDPI 2023-03-30 /pmc/articles/PMC10093505/ /pubmed/37046711 http://dx.doi.org/10.3390/cancers15072050 Text en © 2023 by the authors. https://creativecommons.org/licenses/by/4.0/Licensee MDPI, Basel, Switzerland. This article is an open access article distributed under the terms and conditions of the Creative Commons Attribution (CC BY) license (https://creativecommons.org/licenses/by/4.0/). |
spellingShingle | Article Tramsen, Lars Bochennek, Konrad Sparber-Sauer, Monika Salzmann-Manrique, Emilia Scheer, Monika Dantonello, Tobias Borkhardt, Arndt Dirksen, Uta Thorwarth, Anne Greiner, Jeanette Ebinger, Martin Weclawek-Tompol, Jadwiga Ladenstein, Ruth Ljungman, Gustaf Hallmen, Erika Lehrnbecher, Thomas Koscielniak, Ewa Klingebiel, Thomas 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 | 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 | Article |
url | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10093505/ https://www.ncbi.nlm.nih.gov/pubmed/37046711 http://dx.doi.org/10.3390/cancers15072050 |
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