title: Primary intracranial soft tissue sarcomas in children, adolescents, and young adults: single institution experience and review of the literature.
abstract: There is a paucity of literature reporting the outcome of intracranial sarcomas (IS) in children, adolescents, and young adults (CAYA). A multimodal therapeutic approach is commonly used, with no well-established treatment consensus. We conducted a retrospective review of CAYA with IS, treated at our institution, to determine their clinical findings, treatments, and outcomes. Immunohistochemistry (PDGFRA and EGFR) and DNA sequencing were performed on 5 tumor samples. A literature review of IS was also conducted. We reviewed 13 patients (median age, 7 years) with a primary diagnosis of IS between 1990 and 2015. Diagnoses included unclassified sarcoma (n = 9), chondrosarcoma (n = 2), and rhabdomyosarcoma (n = 2). Five patients underwent upfront gross total resection (GTR) of the tumor. The 5-drug regimen (vincristine, doxorubicin, cyclophosphamide, etoposide, and ifosfamide) was the most common treatment used. Nine patients died due to progression or recurrence (n = 8) or secondary malignancy (n = 1). The median follow-up period of the 4 surviving patients was 1.69 years (range 1.44-5.17 years). The 5-year progression-free survival and overall survival rates were 21 and 44 %, respectively. BRAF, TP53, KRAS, KIT, ERBB2, MET, RET, ATM, and EGFR mutations were detected in 4 of the 5 tissue samples. All 5 samples were immunopositive for PDGFRA, and only 2 were positive for EGFR. IS remain a therapeutic challenge due to high progression and recurrence rates. Collaborative multi-institutional studies are warranted to delineate a treatment consensus and investigate tumor biology to improve the disease outcome.
authors.affiliation: Department of Pediatrics, The University of Texas MD Anderson Cancer Center, 1515 Holcombe Blvd., Unit 87, Houston, TX, 77030, USA. omaher@mdanderson.org.
authors.forename: Ossama M
authors.initials: OM
authors.lastname: Maher
authors.affiliation: Department of Pediatrics, The University of Texas MD Anderson Cancer Center, 1515 Holcombe Blvd., Unit 87, Houston, TX, 77030, USA.
authors.forename: Soumen
authors.initials: S
authors.lastname: Khatua
authors.affiliation: Department of Pediatrics, The University of Texas MD Anderson Cancer Center, 1515 Holcombe Blvd., Unit 87, Houston, TX, 77030, USA.
authors.forename: Devashis
authors.initials: D
authors.lastname: Mukherjee
authors.affiliation: Department of Pathology, The University of Texas MD Anderson Cancer Center, Houston, TX, USA.
authors.forename: Adriana
authors.initials: A
authors.lastname: Olar
authors.affiliation: Department of Pathology, The University of Texas MD Anderson Cancer Center, Houston, TX, USA.
authors.forename: Alexander
authors.initials: A
authors.lastname: Lazar
authors.affiliation: Department of Hematopathology, Molecular Diagnostic Laboratory, The University of Texas MD Anderson Cancer Center, Houston, TX, USA.
authors.forename: Raja
authors.initials: R
authors.lastname: Luthra
authors.affiliation: Department of Biostatistics, The University of Texas MD Anderson Cancer Center, Houston, TX, USA.
authors.forename: Diane
authors.initials: D
authors.lastname: Liu
authors.affiliation: Department of Biostatistics, The University of Texas MD Anderson Cancer Center, Houston, TX, USA.
authors.forename: Jimin
authors.initials: J
authors.lastname: Wu
authors.affiliation: Department of Radiology, The University of Texas MD Anderson Cancer Center, Houston, TX, USA.
authors.forename: Leena
authors.initials: L
authors.lastname: Ketonen
authors.affiliation: Department of Pediatrics, The University of Texas MD Anderson Cancer Center, 1515 Holcombe Blvd., Unit 87, Houston, TX, 77030, USA. wzaky@mdanderson.org.
authors.forename: Wafik
authors.initials: W
authors.lastname: Zaky
mesh_terms: D000293:Adolescent; D000328:Adult; D014408:Biomarkers, Tumor / Q000378:metabolism*; D001932:Brain Neoplasms / Q000378:metabolism / Q000473:pathology* / Q000628:therapy; D002648:Child; D002675:Child, Preschool; D003131:Combined Modality Therapy; D005260:Female; D005500:Follow-Up Studies; D006801:Humans; D007124:Immunoenzyme Techniques; D007223:Infant; D007231:Infant, Newborn; D008297:Male; D009364:Neoplasm Recurrence, Local / Q000378:metabolism / Q000473:pathology* / Q000628:therapy; D009367:Neoplasm Staging; D011379:Prognosis; D012983:Soft Tissue Neoplasms / Q000378:metabolism / Q000473:pathology* / Q000628:therapy; D015996:Survival Rate; D055815:Young Adult
chemical_list: D014408:Biomarkers, Tumor
keywords: Children; Intracranial; Multimodality therapy; Sarcoma; Young adults