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Review
. 2021 Jan 29;13(3):510.
doi: 10.3390/cancers13030510.

The Value of Early Tumor Size Response to Chemotherapy in Pediatric Rhabdomyosarcoma

Roelof van Ewijk  1   2 , Bas Vaarwerk  1   2 , Willemijn B Breunis  2   3 , Reineke A Schoot  1 , Simone A J Ter Horst  1   4 , Rick R van Rijn  5 , Johanna H van der Lee  6   7 , Johannes H M Merks  1   2
Affiliations
Review

The Value of Early Tumor Size Response to Chemotherapy in Pediatric Rhabdomyosarcoma

Roelof van Ewijk et al. Cancers (Basel). .

Abstract

Rhabdomyosarcoma is the most common soft tissue sarcoma in childhood. Results of clinical trials, with three-year event-free and overall survival as primary outcomes, often take 7 to 10 years. Identification of an early surrogate biomarker, predictive for survival, is therefore crucial. We conducted a systematic review to define the prognostic value of early tumor size response in children with IRSG group III rhabdomyosarcoma. The search included MEDLINE/EMBASE from inception to 18 November 2020. In total, six studies were included, describing 2010 patients, and assessed by the Quality in Prognosis Studies (QUIPS) instrument. Four studies found no prognostic value for tumor size response, whereas two studies reported a prognostic effect. In these two studies, the survival rate of patients with progressive disease was not separately analyzed from patients with stable disease, potentially explaining the difference in study outcome. In conclusion, our findings support that early progression of disease is associated with poorer survival, justifying adaptation of therapy. However, in patients with non-progressive disease, there is no evidence that the degree of response is a prognostic marker for survival. Because the vast majority of patients do not have progressive disease, early tumor size response should be reconsidered for assessment of treatment efficacy. Therefore, at present, early surrogate biomarkers for survival are still lacking.

Keywords: biomarker; prognosis; response; rhabdomyosarcoma; sarcoma.

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Conflict of interest statement

The authors declare no conflict of interest. The funders had no role in the design of the study; in the collection, analyses, or interpretation of data; in the writing of the manuscript, or in the decision to publish the results.

Figures

Figure 1
Figure 1
Flowchart of study inclusion.
Figure 2
Figure 2
Grouping of response categories for the different studies. Categories expressed as change in tumor size, as measured in two-dimensional measurements %. Studies describing a different response measurement (i.e., one-dimensional or three-dimensional measurement) were recalculated. Abbreviations: CR, complete response; NR, non-response; OR, objective response; PD, progressive disease; PR/PAR, partial response; SPD, stable/progressive disease; SR, sufficient response. In three studies (Dantonello et al. [10], Ferrari et al. [12], and Vaarwerk et al. [14]) treatment after response assessment was adjusted in case of insufficient response based on the tumor size response.
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