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

被引:13
作者
Tramsen, Lars [1 ,2 ]
Bochennek, Konrad [2 ]
Sparber-Sauer, Monika [3 ,4 ]
Salzmann-Manrique, Emilia [2 ]
Scheer, Monika [5 ]
Dantonello, Tobias [6 ]
Borkhardt, Arndt [7 ]
Dirksen, Uta [8 ,9 ]
Thorwarth, Anne [5 ]
Greiner, Jeanette [10 ]
Ebinger, Martin [11 ]
Weclawek-Tompol, Jadwiga [12 ]
Ladenstein, Ruth [13 ]
Ljungman, Gustaf [14 ]
Hallmen, Erika [3 ]
Lehrnbecher, Thomas [2 ]
Koscielniak, Ewa [3 ,4 ]
Klingebiel, Thomas [2 ]
机构
[1] Univ Hosp Schleswig Holstein, Dept Pediat, Campus Kiel, D-24105 Kiel, Germany
[2] Goethe Univ, Univ Hosp, Dept Children & Adolescents, D-60590 Frankfurt, Germany
[3] Olgahosp Stuttgart Canc Ctr, Hosp State Capital Stuttgart, Ctr Pediat Adolescent & Womens Med, Pediat Oncol Hematol Immunology 5, D-70174 Stuttgart, Germany
[4] Univ Tubingen, Fac Med, D-72016 Tubingen, Germany
[5] Charite Univ Med Berlin, Dept Pediat Oncol & Hematol, Augustenburger Pl 1, D-13353 Berlin, Germany
[6] Univ Bern, Bern Univ Hosp, Dept Pediat, Div Pediat Hematol & Oncol, CH-3010 Bern, Switzerland
[7] Heinrich Heine Univ, Med Fac, Dept Pediat Oncol Hematol & Clin Immunol, D-40225 Dusseldorf, Germany
[8] Univ Hosp Essen, West German Canc Ctr, Pediat 3, D-45147 Essen, Germany
[9] Natl Ctr Tumor Dis NCT Site Essen, German Canc Consortium Site Essen, D-45147 Essen, Germany
[10] Kantonsspital Aarau AG, Childrens Hosp, Pediat Oncol & Hematol, CH-5001 Aarau, Switzerland
[11] Univ Childrens Hosp, Dept Gen Pediat & Pediat Oncol & Hematol, D-72076 Tubingen, Germany
[12] Univ Med Wroclaw, Dept Bone Marrow Transplantat, Pediat Oncol & Haematol, Wroclaw, Poland
[13] St Anna Childrens Hosp, Childrens Canc Res Inst CCRI, A-1090 Vienna, Austria
[14] Uppsala Univ, Dept Womens & Childrens Hlth, Pediat Oncol, S-75185 Uppsala, Sweden
关键词
soft tissue sarcoma; rhabdomyosarcoma; metastatic; children; long-term maintenance therapy; outcome; SOFT-TISSUE SARCOMA; HIGH-DOSE CHEMOTHERAPY; STEM-CELL TRANSPLANTATION; PHASE-II WINDOW; METASTATIC RHABDOMYOSARCOMA; PROGNOSTIC-FACTORS; POOLED ANALYSIS; CHILDHOOD; CHILDREN; ONCOLOGY;
D O I
10.3390/cancers15072050
中图分类号
R73 [肿瘤学];
学科分类号
100214 ;
摘要
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.
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页数:14
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