Generic Portal

TY - JOUR

T1 - The value of early tumor size response to chemotherapy in pediatric rhabdomyosarcoma

AU - van Ewijk, Roelof

AU - Vaarwerk, Bas

AU - Breunis, Willemijn B.

AU - Schoot, Reineke A.

AU - ter Horst, Simone A. J.

AU - van Rijn, Rick R.

AU - van der Lee, Johanna H.

AU - Merks, Johannes H. M.

N1 - Funding Information: Funding: This work has been supported by the KIKA foundation (Children Cancer-free, number 175) and Roelof van Ewijk by the SKOCA Foundation (Pediatric Oncology Center Amsterdam). Publisher Copyright: © 2021 by the authors. Licensee MDPI, Basel, Switzerland.

PY - 2021

Y1 - 2021

N2 - 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.

AB - 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.

KW - Biomarker

KW - Prognosis

KW - Response

KW - Rhabdomyosarcoma

KW - Sarcoma

UR - http://www.scopus.com/inward/record.url?scp=85099926104&partnerID=8YFLogxK

U2 - 10.3390/cancers13030510

DO - 10.3390/cancers13030510

M3 - Article

C2 - 33561094

VL - 13

SP - 1

EP - 15

JO - Cancers

JF - Cancers

SN - 2072-6694

IS - 3

M1 - 510

ER -