TY - JOUR
T1 - Metastatic adult-type non-rhabdomyosarcoma soft tissue sarcomas in children and adolescents
T2 - A cohort study from the European paediatric Soft tissue sarcoma Study Group
AU - Ferrari, Andrea
AU - Orbach, Daniel
AU - Casanova, Michela
AU - van Noesel, Max M
AU - Berlanga, Pablo
AU - Brennan, Bernadette
AU - Corradini, Nadege
AU - Schoot, Reineke A
AU - Ramirez-Villar, Gema L
AU - Hjalgrim, Lisa Lyngsie
AU - Alaggio, Rita
AU - Guillen Burrieza, Gabriela
AU - Safwat, Akmal
AU - Cameron, Alison L
AU - van Rijn, Rick R
AU - Minard-Colin, Veronique
AU - Zanetti, Ilaria
AU - Bisogno, Gianni
AU - Chisholm, Julia C
AU - Merks, Johannes H M
N1 - © 2023 American Cancer Society.
PY - 2023/8/15
Y1 - 2023/8/15
N2 - BACKGROUND: Limited data exist on the clinical behavior of pediatric non-rhabdomyosarcoma soft tissue sarcomas (NRSTS) with distant metastases at onset, and a clear standard of care has not yet been defined.METHODS: This cohort study reports on pediatric adult-type metastatic NRSTS enrolled in two concurrent prospective European studies, i.e., the randomized BERNIE study and the single-arm MTS 2008 study developed by the European paediatric Soft tissue sarcoma Study Group. Treatment programs were originally designed for patients with metastatic rhabdomyosarcoma, i.e., nine courses of multidrug chemotherapy (with or without bevacizumab in the BERNIE study), followed by 12 cycles of maintenance therapy, whereas radiotherapy and/or surgery (on primary tumor and/or metastases) were delayed until after seven courses of chemotherapy had been administered.RESULTS: The study included 61 patients <21 years old treated from July 2008 to December 2016. The lung was the site of metastases in 75% of the cases. All patients received multi-agent chemotherapy, 44% had local therapy to primary tumor, and 18% had treatment of metastases. Median time to progression/relapse was 6 months. A high rate of tumor progression was observed during the initial part of the chemotherapy program. With a median follow-up of 41.5 months (range, 2-111 months), 3-year event-free survival and overall survival were 15.4% (95% confidence interval [CI], 7.6-25.7) and 34.9% (95% CI, 22.7-47.5), respectively. There were no statistically significant differences in outcome depending on the type of treatment administered.CONCLUSIONS: The study confirmed the overall poor outcome for patients with metastatic NRSTS, whose treatment remains a challenge.PLAIN LANGUAGE SUMMARY: Pediatric non-rhabdomyosarcoma soft tissue sarcomas form a heterogeneous group of rare tumors. Although recent international studies have defined the standard of care for patients with localized disease, limited data are available on the clinical behavior of patients with distant metastases. This study on 61 metastatic cases treated on two prospective European protocols confirms that the chances of survival of such patients are often dismal and a standard treatment is still lacking.
AB - BACKGROUND: Limited data exist on the clinical behavior of pediatric non-rhabdomyosarcoma soft tissue sarcomas (NRSTS) with distant metastases at onset, and a clear standard of care has not yet been defined.METHODS: This cohort study reports on pediatric adult-type metastatic NRSTS enrolled in two concurrent prospective European studies, i.e., the randomized BERNIE study and the single-arm MTS 2008 study developed by the European paediatric Soft tissue sarcoma Study Group. Treatment programs were originally designed for patients with metastatic rhabdomyosarcoma, i.e., nine courses of multidrug chemotherapy (with or without bevacizumab in the BERNIE study), followed by 12 cycles of maintenance therapy, whereas radiotherapy and/or surgery (on primary tumor and/or metastases) were delayed until after seven courses of chemotherapy had been administered.RESULTS: The study included 61 patients <21 years old treated from July 2008 to December 2016. The lung was the site of metastases in 75% of the cases. All patients received multi-agent chemotherapy, 44% had local therapy to primary tumor, and 18% had treatment of metastases. Median time to progression/relapse was 6 months. A high rate of tumor progression was observed during the initial part of the chemotherapy program. With a median follow-up of 41.5 months (range, 2-111 months), 3-year event-free survival and overall survival were 15.4% (95% confidence interval [CI], 7.6-25.7) and 34.9% (95% CI, 22.7-47.5), respectively. There were no statistically significant differences in outcome depending on the type of treatment administered.CONCLUSIONS: The study confirmed the overall poor outcome for patients with metastatic NRSTS, whose treatment remains a challenge.PLAIN LANGUAGE SUMMARY: Pediatric non-rhabdomyosarcoma soft tissue sarcomas form a heterogeneous group of rare tumors. Although recent international studies have defined the standard of care for patients with localized disease, limited data are available on the clinical behavior of patients with distant metastases. This study on 61 metastatic cases treated on two prospective European protocols confirms that the chances of survival of such patients are often dismal and a standard treatment is still lacking.
KW - Adolescent
KW - Antineoplastic Combined Chemotherapy Protocols
KW - Child
KW - Cohort Studies
KW - Humans
KW - Neoplasm Recurrence, Local/drug therapy
KW - Prospective Studies
KW - Randomized Controlled Trials as Topic
KW - Rhabdomyosarcoma/drug therapy
KW - Sarcoma/drug therapy
KW - Soft Tissue Neoplasms/pathology
KW - Young Adult
KW - treatment
KW - adolescents
KW - prognostic factors
KW - non-rhabdomyosarcoma soft tissue sarcomas
KW - metastases
KW - children
KW - outcome
UR - http://www.scopus.com/inward/record.url?scp=85153508549&partnerID=8YFLogxK
U2 - 10.1002/cncr.34814
DO - 10.1002/cncr.34814
M3 - Journal article
C2 - 37084075
SN - 0008-543X
VL - 129
SP - 2542
EP - 2552
JO - Cancer
JF - Cancer
IS - 16
ER -