Consensus and controversies regarding the treatment of rhabdomyosarcoma

Borinstein, Scott C.; Steppan, Diana; Hayashi, Masanori; Loeb, David M.; Isakoff, Michael S.; Binitie, Odion; Brohl, Andrew S.; Bridge, Julia A.; Stavas, Mark J.; Shinohara, Eric T.; Meyer, William H.; Reed, Damon R.; Wagner, Lars M.

doi:10.1002/pbc.26809

Cited by 77 publications

(89 citation statements)

References 60 publications

(108 reference statements)

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“…They can be very challenging to treat, depending on the subtype [90]. The loss of PTCH1 in Gorlin syndrome is associated with a range of rare tumours in addition to BCC and MB, including fetal rhabdomyoma [91].…”

Section: The Curious Case Of Rhabdomyosarcomamentioning

confidence: 99%

Reproducibility of academic preclinical translational research: lessons from the development of Hedgehog pathway inhibitors to treat cancer

Curran¹

2018

Open Biol.

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Academic translational research is growing at a great pace at a time in which questions have been raised about the reproducibility of preclinical findings. The development of Hedgehog (HH) pathway inhibitors for the treatment of cancer over the past two decades offers a case study for understanding the root causes of failure to predict clinical outcomes arising from academic preclinical translational research. Although such inhibitors were once hoped to be efficacious in up to 25% of human cancer, clinical studies showed responses only in basal cell carcinoma and the HH subtype of medulloblastoma. Close examination of the published studies reveals limitations in the models used, lack of quantitative standards, utilization of high drug concentrations associated with non-specific toxicities and improper use of cell line and mouse models. In part, these issues arise from scientific complexity, for example, the failure of tumour cell lines to maintain HH pathway activity in vitro, but a greater contributing factor appears to be the influence of unconscious bias. There was a strong expectation that HH pathway inhibitors would make a profound impact on human cancer and experiments were designed with this assumption in mind.

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Section: The Curious Case Of Rhabdomyosarcomamentioning

confidence: 99%

Reproducibility of academic preclinical translational research: lessons from the development of Hedgehog pathway inhibitors to treat cancer

Curran¹

2018

Open Biol.

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“…All of these factors might pose additional disadvantages to adult RMS outcomes. [30] Furthermore, we have shown that survival was significantly different according to risk group stratification used in pediatric RMS. As a result, risk stratification used in pediatric cases also might be valid in adult RMS.…”

Section: Discussionmentioning

confidence: 95%

Adult Rhabdomyosarcoma: Clinical Features and Radiotherapy Outcomes-The Turkish Oncology Group (TOG) Bone and Soft Tissue Sarcoma Study Group

Kıraklı¹

2018

TJ Oncol

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“…Distal metastases remains the most adverse prognostic factor despite continuous improvement of the diagnostic capabilities and the use of more intense treatments [27][28][29]. Within this group of patients, outcome further differs significantly when multiple risk factors are combined, such as patient's age, primary tumor site, number of metastases, and bone or bone marrow involvement [30,31]. However, individual outcome is still difficult to be predicted at the time of diagnosis.…”

Section: Discussionmentioning

confidence: 99%

Prognostic Value of Circulating IGFBP2 and Related Autoantibodies in Children with Metastatic Rhabdomyosarcomas

et al. 2020

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Insulin-like growth factor-binding protein 2 (IGFBP2) is a tumor-associated protein measurable in patients’ biopsies and blood samples. Increased IGFBP2 expression correlates with tumor severity in rhabdomyosarcoma (RMS). Thus, we examined the plasmatic IGFBP2 levels in 114 RMS patients and 15 healthy controls by ELISA assay in order to evaluate its value as a plasma biomarker for RMS. Additionally, we looked for the presence of a humoral response against IGBFP2 protein measurable by the production of anti-IGFBP2 autoantibodies. We demonstrated that both circulating IGFBP2 protein and autoantibodies were significantly higher in RMS patients with respect to controls and their combination showed a better discriminative capacity. IGFBP2 protein identified metastatic patients with worse event-free survival, whereas both IGFBP2 and anti-IGFBP2 antibodies negatively correlated with overall survival. Our study suggests that IGFBP2 and anti-IGFBP2 antibodies are useful for diagnostic and prognostic purposes, mainly as independent negative prognostic markers in metastatic patients. This is the first study that reports a specific humoral response in RMS plasma samples and proves the value of blood-based biomarkers in improving risk assessment and outcome of metastatic RMS patients.

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Consensus and controversies regarding the treatment of rhabdomyosarcoma

Cited by 77 publications

References 60 publications

Reproducibility of academic preclinical translational research: lessons from the development of Hedgehog pathway inhibitors to treat cancer

Reproducibility of academic preclinical translational research: lessons from the development of Hedgehog pathway inhibitors to treat cancer

Adult Rhabdomyosarcoma: Clinical Features and Radiotherapy Outcomes-The Turkish Oncology Group (TOG) Bone and Soft Tissue Sarcoma Study Group

Prognostic Value of Circulating IGFBP2 and Related Autoantibodies in Children with Metastatic Rhabdomyosarcomas

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