High dose chemotherapy for children and young adults with stage IV rhabdomyosarcoma

Admiraal, Rick; Paardt, M van der; Kobes, Jasmijn; Kremer, Leontien; Bisogno, Gianni; Merks, Johannes H. M.

doi:10.1002/14651858.cd006669

Cited by 4 publications

(4 citation statements)

References 9 publications

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“…Verma 2008 [20] investigated HDCT followed by autologous HSCT in patients with inoperable, locally advanced, or metastatic STS, reported the results of 2 single-arm studies on STS, and did not address specifically data on RMS. Admiraal 2007 [82] conducted a systematic review exclusively on patients with metastatic RMS.…”

Section: Discussionmentioning

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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Section: Discussionmentioning

confidence: 99%

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

et al. 2011

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“…A standard approach is still lacking, and there is a marked variability in how radiotherapy has reportedly been used, even in clinical trials. 20 As most studies over the years have focused on intensifying systemic treatments to improve the outcome of patients with metastatic RMS, [12][13][14][15][16][17][18] the potential role of radiotherapy may have sometimes been underestimated.…”

Section: -Year Os (%) Os P-value (Lor-rank)mentioning

confidence: 99%

“…[9][10][11] Different therapeutic approaches developed over the years have not substantially changed the prognosis for patients with distant metastases at diagnosis. Most studies have tried to improve patient outcome by increasing the intensity of systemic therapy (e.g., high-dose chemotherapy with stem cell support, dose-compressed chemotherapy), [12][13][14][15] or using alternative therapies (e.g., targeted agents, but also allogeneic transplantation). [16][17][18] Such approaches have sometimes made it difficult to combine the delivery of local treatments (surgery and radiotherapy to the sites of primary tumors and distant metastases) with aggressive systemic therapies.…”

Section: Introductionmentioning

confidence: 99%

Metastatic rhabdomyosarcoma: Evidence of the impact of radiotherapy on survival. A retrospective single‐center experience

Ferrari

Bergamaschi

Chiaravalli

et al. 2022

Pediatric Blood & Cancer

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Background The prognosis for patients with metastatic rhabdomyosarcoma (RMS) remains largely unsatisfactory despite the adoption of intensive multimodal therapy. To assess the role of different treatments adopted over the years, we retrospectively analyzed a cohort of patients <21 years old with metastatic RMS, treated from 1990 to 2020 at a referral center for pediatric sarcomas. Methods Patients were treated using a multimodal approach that included surgery, radiotherapy, and chemotherapy (both high‐dose chemotherapy and maintenance therapy in some cases). The type of radiotherapy administered was categorized as radical (to all sites of disease); partial (to at least one, but not all sites of disease); or none. A landmark analysis was used to examine the impact of radiotherapy on survival, that is, patients who had an event before day 221 were excluded from the analysis. Results The series included 80 patients. Event‐free survival (EFS) and overall survival (OS) rates at 5 years were 17.3% and 21.3%, respectively. Survival was significantly associated with radiotherapy to metastatic sites, and with the radiotherapy category. In particular, 5‐year EFS and OS rates were 70.6% and 76.0% for patients given radical radiotherapy, and 4.8% and 10.7%, respectively, for those given partial radiotherapy or none. Using the Cox multivariable analysis, OS correlated significantly with radiotherapy category. Conclusions While confirming the poor overall outcome of patients with metastatic RMS, this study identified radiotherapy—when given to all sites of disease (including metastases)—as the main variable influencing survival.

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“…Outcome in patients with localized disease is generally good, 1,2 but outcome for patients with metastatic RMS remains poor with 3-year overall survival (OS) of 34%-56%. 3,4 Various attempts to increase treatment intensity failed to improve survival (eg, high-dose chemotherapy with stem-cell support) [5][6][7][8] or resulted in very limited improvement in selected subgroups of patients (dose-compressed chemotherapy). 4 The European pediatric Soft tissue sarcoma Study Group (EpSSG) has collaborated in three studies in newly diagnosed RMS in recent years.…”

Section: Introductionmentioning

confidence: 99%

Metastatic Rhabdomyosarcoma: Results of the European Paediatric Soft Tissue Sarcoma Study Group MTS 2008 Study and Pooled Analysis With the Concurrent BERNIE Study

Schoot

Chisholm

Casanova

et al. 2022

JCO

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PURPOSE Outcome for patients with metastatic rhabdomyosarcoma (RMS) is poor. This study presents the results of the MTS 2008 study with a pooled analysis including patients from the concurrent BERNIE study. PATIENTS AND METHODS In MTS 2008, patients with metastatic RMS received four cycles of ifosfamide, vincristine, and actinomycin D (IVA) plus doxorubicin, five cycles of IVA, and 12 cycles of maintenance chemotherapy (low-dose cyclophosphamide and vinorelbine). The BERNIE study randomly assigned patients to the addition or not of bevacizumab to the same chemotherapy. Local therapy (surgery/radiotherapy) was given to the primary tumor and all metastatic sites when feasible. RESULTS MTS 2008 included 270 patients (median age, 9.6 years; range, 0.07-20.8 years). With a median follow-up of 50.3 months, 3-year event-free survival (EFS) and overall survival (OS) were 34.9% (95% CI, 29.1 to 40.8) and 47.9% (95% CI, 41.6 to 53.9), respectively. In pooled analyses on 372 patients with a median follow-up of 55.2 months, 3-year EFS and OS were 35.5% (95% CI, 30.4 to 40.6) and 49.3% (95% CI, 43.9 to 54.5), respectively. Patients with ≤ 2 Oberlin risk factors (ORFs) had better outcome than those with ≥ 3 ORFs: 3-year EFS was 46.1% versus 12.5% ( P < .0001) and 3-year OS 60.0% versus 26.0% ( P < .0001). Induction chemotherapy and maintenance appeared tolerable; however, about two third of patients needed dose adjustments during maintenance. CONCLUSION Outcome remains poor for patients with metastatic RMS and multiple ORFs. Because of the design of the studies, it was not possible to determine whether the intensive induction regimen and/or the addition of maintenance treatment resulted in apparent improvement of outcome compared with historical cohorts. Further studies, with novel treatment approaches are urgently needed, to improve outcome for the group of patients with adverse prognostic factors.

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High dose chemotherapy for children and young adults with stage IV rhabdomyosarcoma

Cited by 4 publications

References 9 publications

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

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

Metastatic rhabdomyosarcoma: Evidence of the impact of radiotherapy on survival. A retrospective single‐center experience

Metastatic Rhabdomyosarcoma: Results of the European Paediatric Soft Tissue Sarcoma Study Group MTS 2008 Study and Pooled Analysis With the Concurrent BERNIE Study

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