Abstract:Overall, the results of this review do not justify the use of HDC with SCR as a standard therapy for children with metastatic rhabdomyosarcoma. However, all reported studies were possibly subject to significant bias, especially selection bias. This might have underestimated the measured effect of HDC. As a result, a clinically important excess of adverse risk patients in the HDC arms may explain the non-beneficial effect of HDC. Only a large prospective RCT will be able to answer the question of whether HDC wi… Show more
“…An alternative method, reserved especially for patients with disseminated tumor disease (group IV) with the worst prognosis, is aggressive chemotherapy with subsequent autologous myogenic stem cell transplantation [ 72 , 73 ].…”
Rhabdomyosarcoma (RMS) is the most frequent soft tissue sarcoma in children. It is localized in the head and neck region in 40% of cases. Treatment of RMS is complex, including multi-drug chemotherapy, radiotherapy and surgery. The progress that has been accomplished in oncology in recent decades significantly improved outcomes. The 5-year survival rate raised from 25% in 1970 to 73% in 2001, according to IRS-IV data. The outcome is influenced by primary tumor localization, clinical staging, histological tumor type and age at the moment of diagnosis. The relatively rare incidence of these tumors resulted in difficulties in creating more standardized therapeutic protocols. Comparison of outcomes in large patients groups led to an increase in the number of patients with complete remission. Although survival rates of RMS patients have improved, searching for new therapeutic modalities and substances is still essential to improve outcomes in cases of more advanced stages and unfavorable tumor localizations.
“…An alternative method, reserved especially for patients with disseminated tumor disease (group IV) with the worst prognosis, is aggressive chemotherapy with subsequent autologous myogenic stem cell transplantation [ 72 , 73 ].…”
Rhabdomyosarcoma (RMS) is the most frequent soft tissue sarcoma in children. It is localized in the head and neck region in 40% of cases. Treatment of RMS is complex, including multi-drug chemotherapy, radiotherapy and surgery. The progress that has been accomplished in oncology in recent decades significantly improved outcomes. The 5-year survival rate raised from 25% in 1970 to 73% in 2001, according to IRS-IV data. The outcome is influenced by primary tumor localization, clinical staging, histological tumor type and age at the moment of diagnosis. The relatively rare incidence of these tumors resulted in difficulties in creating more standardized therapeutic protocols. Comparison of outcomes in large patients groups led to an increase in the number of patients with complete remission. Although survival rates of RMS patients have improved, searching for new therapeutic modalities and substances is still essential to improve outcomes in cases of more advanced stages and unfavorable tumor localizations.
“…Thirteen SRs assessed interventions with a curative intention [19, 20, 22-24, 27, 29, 32-36, 40], whereas in the other 11 the interventions was palliative [15,18,21,25,26,28,30,31,[37][38][39].…”
Section: Characteristics and Quality Of Systematic Reviewsmentioning
confidence: 99%
“…metastatic STS (First Line): Six SRs [18,21,30,31,37,38] included a total of 17 RCTs, 19 non-controlled trials and 5 observational studies, addressing mainly two therapeutic options in this scenario. Two SRs compared chemotherapy of doxorubicin-based combination versus doxorubicin alone.…”
Section: Patients With Locally Advanced Unresectable And/ormentioning
confidence: 99%
“…Four SRs [18,30,31,37] compared dose-intensive chemotherapy by autologous bone marrow support, stem cell transplantation or growth factor with standard-dose chemotherapy. Two of them in patients diagnosed of rhabdomyosarcoma [18,30]. In general, the results were stated by the authors as "harmful" and "inconclusive".…”
Section: Patients With Locally Advanced Unresectable And/ormentioning
Purpose Soft tissue sarcomas are a heterogeneous group of rare tumours of mesenchymal origin. Evidence mapping is one of the most didactic and friendly approaches to organise and summarise the range of research activity in broad topic fields. The objective of this evidence mapping is to identify, describe and organise the current available evidence about therapeutic interventions on soft tissues sarcomas. Methods We followed the methodology of global evidence mapping. We performed a search of the PubMed, EMBASE, The Cochrane Library and Epistemonikos to identify systematic reviews (SRs) with or without meta-analyses published between 1990 and March 2016. Two independent literature reviewers assessed eligibility and extracted data. Methodological quality of the included systematic reviews was assessed using AMSTAR. We organised the results according to identified PICO questions and used tables and a bubble plot to display the results. Results The map is based on 24 SRs that met eligibility criteria and included 66 individual studies. Three-quarters were either observational or uncontrolled clinical trials. The quality of the included SRs was in general moderate or high. We identified 64 PICO questions from them. The corresponding results mostly favoured the intervention arm. Conclusions This evidence mapping was built on the basis of SRs, which mostly included non-experimental studies and were qualified by the AMSTAR tool as of moderate quality. The evidence mapping created from PICO questions is a useful approach to describe complex and huge clinical topics through graphical media and orientate further research to fulfil the existing gaps. However, it is important to delimitate the steps of the evidence mapping in a pre-established protocol.
“…complete-partial response, as well as patients with sensitive relapse are good candidates to be considered for HDC, whenever possible within controlled studies. Finally, evidence of HDC role in rhabdomyosarcomas and osteosarcomas is still missing (49,50).…”
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