Treatment of children with metastatic soft tissue sarcoma with oral maintenance compared to high dose chemotherapy: Report of the HD CWS‐96 trial

Klingebiel, Thomas; Boos, Joachim; Beske, Florian; Hallmen, Erika; Int-Veen, Christoph; Dantonello, Tobias; Treuner, J.; Gadner, Helmut; Márky, Ildikó; Kazanowska, Bernarda; Kościelniak, Ewa

doi:10.1002/pbc.21494

Cited by 113 publications

(99 citation statements)

References 25 publications

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“…STS can develop in any part of the body and the prognosis depends on their histological subtype, patient's age, stage of disease, localisation and feasibility of complete resection of the primary tumour, response to chemo-and radiotherapy and others (Koscielniak et al 2002). These clinical variables imply the choice of therapeutic strategy which in Poland is based on the protocols of the German Paediatric STS Study Group (CWS) (Klingebiel et al 2008).…”

Section: Introductionmentioning

confidence: 99%

The serum levels of soluble interleukin-2 receptor α and lactate dehydrogenase but not of B2-microglobulin correlate with selected clinico-pathological prognostic factors and response to therapy in childhood soft tissue sarcomas

Bień

Rąpała

Krawczyk

et al. 2009

J Cancer Res Clin Oncol

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sIL-2R alpha and LDH were [corrected] proven to be promising markers [corrected] for diagnosis and treatment monitoring in children with STS. The markers [corrected] correlated also with some [corrected] important prognostic clinico-pathological factors for childhood [corrected] STS; however, they [corrected] failed to predict EFS and OS. Measurements of serum [corrected] B2-M were shown [corrected] to have no clinical value in the diagnostics, prognostics and treatment monitoring in paediatric STS.

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Section: Introductionmentioning

confidence: 99%

The serum levels of soluble interleukin-2 receptor α and lactate dehydrogenase but not of B2-microglobulin correlate with selected clinico-pathological prognostic factors and response to therapy in childhood soft tissue sarcomas

Bień

Rąpała

Krawczyk

et al. 2009

J Cancer Res Clin Oncol

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“…Studies: Bader 1989 [106]; Bisogno 2009 [39]; Carli 1999 [36]; Carli 2004 [107]; Dumontet 1992 [93]; Hosoi 2007 [37]; Klingebiel 2008 [38]; Koscielniak 1997 [108]; Matsubara 2003 [109]; Suita 2005 [110]; Yamada 2007 [40]. Abbreviations.…”

Section: Methodsmentioning

confidence: 99%

High-Dose Chemotherapy Followed by Autologous Stem Cell Transplantation for Metastatic Rhabdomyosarcoma—A Systematic Review

et al. 2011

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IntroductionPatients with metastatic rhabdomyosarcoma (RMS) have a poor prognosis. The aim of this systematic review is to investigate whether high-dose chemotherapy (HDCT) followed by autologous hematopoietic stem cell transplantation (HSCT) in patients with metastatic RMS has additional benefit or harm compared to standard chemotherapy.MethodsSystematic literature searches were performed in MEDLINE, EMBASE, and The Cochrane Library. All databases were searched from inception to February 2010. PubMed was searched in June 2010 for a last update. In addition to randomized and non-randomized controlled trials, case series and case reports were included to complement results from scant data. The primary outcome was overall survival. A meta-analysis was performed using the hazard ratio as primary effect measure, which was estimated from Cox proportional hazard models or from summary statistics of Kaplan Meier product-limit estimations.ResultsA total of 40 studies with 287 transplant patients with metastatic RMS (age range 0 to 32 years) were included in the assessment. We identified 3 non-randomized controlled trials. The 3-year overall survival ranged from 22% to 53% in the transplant groups vs. 18% to 55% in the control groups. Meta-analysis on overall survival in controlled trials showed no difference between treatments. Result of meta-analysis of pooled individual survival data of case series and case reports, and results from uncontrolled studies with aggregate data were in the range of those from controlled data. The risk of bias was high in all studies due to methodological flaws.ConclusionsHDCT followed by autologous HSCT in patients with RMS remains an experimental treatment. At present, it does not appear justifiable to use this treatment except in appropriately designed controlled trials.

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“…3 Most patients achieve a remission after conventional therapy, but relapse is frequent, indicating persistence of minimal residual disease. 4 Although RMS is a chemosensitive disease, the benefits of high-dose chemotherapy followed by autologous haematopoietic stem cell rescue for children with stage IV or relapsed RMS are not clear because randomized trials comparing this treatment with standard chemotherapy are lacking. In a meta-analysis of 389 patients identified from 22 publications, there was no significant survival advantage of high-dose chemotherapy followed by autologous haematopoietic stem cell rescue for patients with relapsed or metastatic RMS as compared with standard chemotherapy.…”

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confidence: 99%

Metastatic rhabdomyosarcoma cured after chemotherapy and allogeneic SCT

Donker

Hoogerbrugge

Mavinkurve‐Groothuis

et al. 2008

Bone Marrow Transplant

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Rhabdomyosarcoma (RMS) is the most common soft tissue sarcoma in children and adolescents. Successful treatment of children with RMS requires multimodal therapy. However, despite advances in combined chemotherapy, radiation therapy and surgery, children with stage IV RMS have a uniformly poor outcome. 1 In case of nonembryonal histology in combination with metastatic involvement in three or more organ systems, the prognosis is dismal (3-year survival 5%). 2 Other unfavourable risk factors include the age at diagnosis of more than 10 years, bone or BM metastases and RMS of the extremities. Patients with more than two of these risk factors have a 5-year survival of 9%. 3 Most patients achieve a remission after conventional therapy, but relapse is frequent, indicating persistence of minimal residual disease. 4 Although RMS is a chemosensitive disease, the benefits of high-dose chemotherapy followed by autologous haematopoietic stem cell rescue for children with stage IV or relapsed RMS are not clear because randomized trials comparing this treatment with standard chemotherapy are lacking. In a meta-analysis of 389 patients identified from 22 publications, there was no significant survival advantage of high-dose chemotherapy followed by autologous haematopoietic stem cell rescue for patients with relapsed or metastatic RMS as compared with standard chemotherapy. 5 GVL effect is well documented after allogeneic SCT for haematological malignancies. Recently, the evidence of graft-versus-tumour effect has also been described in patients with a variety of solid tumours. 6 Only sporadic cases have been reported of allogeneic SCT in high-risk RMS patients. Wiegel et al. 7 report on two children with relapsed stage IV RMS who received allogeneic SCT after a myeloablative regimen in a non-remission status. Both patients died of disease progression. However, outcome of patients with ALL is also poor when allogeneic SCT is performed in a non-remission status or in a status with high levels of minimal residual disease. 8 Misawa et al. describe a patient with refractory RMS for which an allogeneic SCT was performed after a myeloablative regimen. The patient reached a PR. As tumour regression occurred after the reduction of immunosuppressants on day þ 30, the authors suggest the existence of a graft-versus-tumour effect. However, 80 days after transplantation, disease progression occurred. On day 165, the patient died of progressive disease in the final stage accompanied by fungal pneumonia. 9 In this letter we report, a patient with stage IV RMS who achieved CR and is in ongoing remission for 4 years after combined therapy followed by allogeneic SCT.An 8-year-old Caucasian girl was diagnosed with an alveolar RMS of the right gastrocnemical muscle (Figure 1). PAX-3 and PAX-7 fusion genes, which are specific for alveolar RMS were detected by reverse-transcription PCR. Staging revealed stage IV with extensive local, abdominal and thoracic lymph node metastases in combination with skeletal metastases (Figure 2). Bilateral crystal b...

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Treatment of children with metastatic soft tissue sarcoma with oral maintenance compared to high dose chemotherapy: Report of the HD CWS‐96 trial

Abstract: Oral maintenance therapy seems to be a promising option for patients with RMS-like stage IV tumors.

Cited by 113 publications

References 25 publications

The serum levels of soluble interleukin-2 receptor α and lactate dehydrogenase but not of B2-microglobulin correlate with selected clinico-pathological prognostic factors and response to therapy in childhood soft tissue sarcomas

The serum levels of soluble interleukin-2 receptor α and lactate dehydrogenase but not of B2-microglobulin correlate with selected clinico-pathological prognostic factors and response to therapy in childhood soft tissue sarcomas

High-Dose Chemotherapy Followed by Autologous Stem Cell Transplantation for Metastatic Rhabdomyosarcoma—A Systematic Review

Metastatic rhabdomyosarcoma cured after chemotherapy and allogeneic SCT

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