Long-Term Clinical Outcome and Prognostic Factors of Children and Adolescents with Localized Rhabdomyosarcoma Treated on the CWS-2002P Protocol
We report here the results of the prospective, non-randomized, historically controlled CWS-2002P study in patients ≤ 21 years with localized RMS developed with the aim to improve the long-term outcome by adapting the burden of therapy to risk profile and to investigate the feasibility and relation t...
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| Language: | English |
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MDPI AG
2022-02-01
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| Series: | Cancers |
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| Online Access: | https://www.mdpi.com/2072-6694/14/4/899 |
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| author | Ewa Koscielniak Bernd Blank Christian Vokuhl Bernarda Kazanowska Ruth Ladenstein Felix Niggli Gustaf Ljungman Rupert Handgretinger Guido Seitz Jörg Fuchs Birgit Fröhlich Monika Scheer Rüdiger Wessalowski Irene Schmid Monika Sparber-Sauer Thomas Klingebiel |
| author_facet | Ewa Koscielniak Bernd Blank Christian Vokuhl Bernarda Kazanowska Ruth Ladenstein Felix Niggli Gustaf Ljungman Rupert Handgretinger Guido Seitz Jörg Fuchs Birgit Fröhlich Monika Scheer Rüdiger Wessalowski Irene Schmid Monika Sparber-Sauer Thomas Klingebiel |
| author_sort | Ewa Koscielniak |
| collection | DOAJ |
| description | We report here the results of the prospective, non-randomized, historically controlled CWS-2002P study in patients ≤ 21 years with localized RMS developed with the aim to improve the long-term outcome by adapting the burden of therapy to risk profile and to investigate the feasibility and relation to the outcome of maintenance therapy (MT) in the high-risk groups. Patients were allocated into low-risk (LR), standard-risk (SR), high-risk (HR), and very high-risk (VHR) groups. Chemotherapy consisted of vincristine (VCR) and dactinomycin (ACTO-D) for all patients with the addition of ifosfamide (IFO) in the SR, HR, and VHR and doxorubicin (DOX) in the HR and VHR groups. Low-dose cyclophosphamide and vinblastine maintenance therapy (MT) over 6 months was recommended in the HR and VHR groups. A total of 444 patients have been included in this analysis. With a median follow-up of 9·6 years (IQR 7·6–10·9) for patients alive, the 5-year EFS and OS for the whole group was 73% (95% CI 69–77) and 80% (95% CI 76–84), respectively. The 5-year EFS by risk group was 100% in the LR, 79% (95% CI 72–84) in the SR, 69% (95% CI 63–75) in the HR, and 42% (95% CI 23–61) in the VHR (log-rank <i>p</i> = 0.000). The 5-year EFS was 77% (95% CI 70–84) for 155 patients in the HR group who received MT as compared to 63% (95% CI 50–76) for 49 patients who did not (log-rank <i>p</i> = 0.015). Neither the reduction in the IFO dose in the SR nor the increased dose intensity of DOX in HR groups influenced the outcome when compared to the previous CWS and other European studies. MT was feasible, seemed to have an impact on prognosis, and should be studied in a well-controlled prospective trial in this patient population. The weighting of risk factors used for therapy stratification needs to be reevaluated. |
| first_indexed | 2024-03-09T22:24:14Z |
| format | Article |
| id | doaj.art-8e8bbdb20c9344e2976e9ddb827cb553 |
| institution | Directory Open Access Journal |
| issn | 2072-6694 |
| language | English |
| last_indexed | 2024-03-09T22:24:14Z |
| publishDate | 2022-02-01 |
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| series | Cancers |
| spelling | doaj.art-8e8bbdb20c9344e2976e9ddb827cb5532023-11-23T19:08:11ZengMDPI AGCancers2072-66942022-02-0114489910.3390/cancers14040899Long-Term Clinical Outcome and Prognostic Factors of Children and Adolescents with Localized Rhabdomyosarcoma Treated on the CWS-2002P ProtocolEwa Koscielniak0Bernd Blank1Christian Vokuhl2Bernarda Kazanowska3Ruth Ladenstein4Felix Niggli5Gustaf Ljungman6Rupert Handgretinger7Guido Seitz8Jörg Fuchs9Birgit Fröhlich10Monika Scheer11Rüdiger Wessalowski12Irene Schmid13Monika Sparber-Sauer14Thomas Klingebiel15Pediatrics 5 (Oncology, Hematology, Immunology), Klinikum Stuttgart-Olgahospital, 70174 Stuttgart, GermanyPediatrics 5 (Oncology, Hematology, Immunology), Klinikum Stuttgart-Olgahospital, 70174 Stuttgart, GermanySection of Pediatric Pathology, Department of Pathology, University Bonn, 53127 Bonn, GermanyDepartment of Pediatric Hematology/Oncology and BMT, University of Wroclaw, 50556 Wroclaw, PolandPediatric Oncology, St. Anna Kinderspital, 1090 Vienna, AustriaDepartment of Pediatric Oncology, University of Zürich, 8032 Zurich, SwitzerlandDepartment of Women’s and Children’s Health, Pediatric Oncology, Uppsala University, 75185 Uppsala, SwedenDepartment of Pediatric Hematology and Oncology, University of Tübingen, Hospital for Children and Adolescents, 72076 Tuebingen, GermanyDepartment of Pediatric Surgery, University Children’s Hospital Marburg, 35043 Marburg, GermanyDepartment of Pediatric Surgery and Urology, Hospital for Children and Adolescents, University Tuebingen, 72076 Tübingen, GermanyDepartment of Pediatric Hematology and Oncology, University Hospital Münster, 48149 Muenster, GermanyDepartment of Pediatric Oncology and Hematology, Charité-Universitätsmedizin, 13353 Berlin, GermanyDepartment of Pediatric Oncology, Medical Faculty, Heinrich Heine University, Hematologyand Clinical Immunology, 40225 Duesseldorf, GermanyDepartment of Pediatrics, Division of Pediatric Hematology and Oncology, Dr. von Hauner Children’s Hospital, University Hospital Munich, LMU Munich, 80337 Munich, GermanyPediatrics 5 (Oncology, Hematology, Immunology), Klinikum Stuttgart-Olgahospital, 70174 Stuttgart, GermanyDepartment for Children and Adolescents, University Hospital Frankfurt, Goethe University, 60590 Frankfurt, GermanyWe report here the results of the prospective, non-randomized, historically controlled CWS-2002P study in patients ≤ 21 years with localized RMS developed with the aim to improve the long-term outcome by adapting the burden of therapy to risk profile and to investigate the feasibility and relation to the outcome of maintenance therapy (MT) in the high-risk groups. Patients were allocated into low-risk (LR), standard-risk (SR), high-risk (HR), and very high-risk (VHR) groups. Chemotherapy consisted of vincristine (VCR) and dactinomycin (ACTO-D) for all patients with the addition of ifosfamide (IFO) in the SR, HR, and VHR and doxorubicin (DOX) in the HR and VHR groups. Low-dose cyclophosphamide and vinblastine maintenance therapy (MT) over 6 months was recommended in the HR and VHR groups. A total of 444 patients have been included in this analysis. With a median follow-up of 9·6 years (IQR 7·6–10·9) for patients alive, the 5-year EFS and OS for the whole group was 73% (95% CI 69–77) and 80% (95% CI 76–84), respectively. The 5-year EFS by risk group was 100% in the LR, 79% (95% CI 72–84) in the SR, 69% (95% CI 63–75) in the HR, and 42% (95% CI 23–61) in the VHR (log-rank <i>p</i> = 0.000). The 5-year EFS was 77% (95% CI 70–84) for 155 patients in the HR group who received MT as compared to 63% (95% CI 50–76) for 49 patients who did not (log-rank <i>p</i> = 0.015). Neither the reduction in the IFO dose in the SR nor the increased dose intensity of DOX in HR groups influenced the outcome when compared to the previous CWS and other European studies. MT was feasible, seemed to have an impact on prognosis, and should be studied in a well-controlled prospective trial in this patient population. The weighting of risk factors used for therapy stratification needs to be reevaluated.https://www.mdpi.com/2072-6694/14/4/899rhabdomyosarcomapediatricsoft tissue sarcomaclinical trialmaintenance therapyrisk grouping |
| spellingShingle | Ewa Koscielniak Bernd Blank Christian Vokuhl Bernarda Kazanowska Ruth Ladenstein Felix Niggli Gustaf Ljungman Rupert Handgretinger Guido Seitz Jörg Fuchs Birgit Fröhlich Monika Scheer Rüdiger Wessalowski Irene Schmid Monika Sparber-Sauer Thomas Klingebiel Long-Term Clinical Outcome and Prognostic Factors of Children and Adolescents with Localized Rhabdomyosarcoma Treated on the CWS-2002P Protocol Cancers rhabdomyosarcoma pediatric soft tissue sarcoma clinical trial maintenance therapy risk grouping |
| title | Long-Term Clinical Outcome and Prognostic Factors of Children and Adolescents with Localized Rhabdomyosarcoma Treated on the CWS-2002P Protocol |
| title_full | Long-Term Clinical Outcome and Prognostic Factors of Children and Adolescents with Localized Rhabdomyosarcoma Treated on the CWS-2002P Protocol |
| title_fullStr | Long-Term Clinical Outcome and Prognostic Factors of Children and Adolescents with Localized Rhabdomyosarcoma Treated on the CWS-2002P Protocol |
| title_full_unstemmed | Long-Term Clinical Outcome and Prognostic Factors of Children and Adolescents with Localized Rhabdomyosarcoma Treated on the CWS-2002P Protocol |
| title_short | Long-Term Clinical Outcome and Prognostic Factors of Children and Adolescents with Localized Rhabdomyosarcoma Treated on the CWS-2002P Protocol |
| title_sort | long term clinical outcome and prognostic factors of children and adolescents with localized rhabdomyosarcoma treated on the cws 2002p protocol |
| topic | rhabdomyosarcoma pediatric soft tissue sarcoma clinical trial maintenance therapy risk grouping |
| url | https://www.mdpi.com/2072-6694/14/4/899 |
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