Clinical predictors of survival for patients with atypical teratoid/rhabdoid tumors

Vismaya S Bachu; Pavan Shah; Adrian E Jimenez; Adham M Khalafallah; Jignesh Tailor; Debraj Mukherjee; Alan R Cohen

doi:10.1007/s00381-022-05511-w

Clinical predictors of survival for patients with atypical teratoid/rhabdoid tumors

Childs Nerv Syst. 2022 Jul;38(7):1297-1306. doi: 10.1007/s00381-022-05511-w. Epub 2022 Apr 1.

Authors

Vismaya S Bachu¹, Pavan Shah², Adrian E Jimenez², Adham M Khalafallah², Jignesh Tailor^{3

4}, Debraj Mukherjee², Alan R Cohen²

Affiliations

¹ Department of Neurosurgery, The Johns Hopkins University School of Medicine, 1800 Orleans Street, Baltimore, MD, 21287, USA. vbachu2@jh.edu.
² Department of Neurosurgery, The Johns Hopkins University School of Medicine, 1800 Orleans Street, Baltimore, MD, 21287, USA.
³ Department of Neurosurgery, Indiana University School of Medicine, Indianapolis, IN, USA.
⁴ Division of Pediatric Neurosurgery, Riley Hospital for Children, Indianapolis, IN, USA.

PMID: 35362829
DOI: 10.1007/s00381-022-05511-w

Abstract

Purpose: Atypical teratoid/rhabdoid tumors (AT/RTs) are malignant central nervous system (CNS) neoplasms of the young. Our study analyzed a large AT/RT cohort from the National Cancer Database (NCDB) to elucidate predictors of short-term mortality and overall survival (OS).

Methods: Information was collected on patients with histologically confirmed AT/RT using the NCDB (2004-2016). Kaplan-Meier analysis indicated OS. Prognostic factors for 30-day mortality, 90-day mortality, and OS were determined via multivariate Cox proportional hazards (CPH) and logistic regression models.

Results: Our cohort of 189 patients had a median age of 1 year (IQR [1, 4]) and tumor size of 4.7 ± 2.0 cm at diagnosis. Seventy-two percent were under 3 years old; 55.6% were male and 71.0% were Caucasian. Fifty (27.2%) patients received only surgery (S) (OS = 5.91 months), 51 (27.7%) received surgery and chemotherapy (S + CT) (OS = 11.2 months), and 9 (4.89%) received surgery and radiotherapy (S + RT) (OS = 10.3 months). Forty-five (24.5%) received S + CT + RT combination therapy (OS = 45.4 months), 13 (17.1%) received S + CT + BMT/SCT (bone marrow or stem cell transplant) (OS = 55.5 months), and 16 (8.70%) received S + CT + RT + BMT/SCT (OS = 68.4 months). Bivariate analysis of dichotomized age (HR = 0.550, 95% CI [0.357, 0.847], p = 0.0067) demonstrated significantly increased patient survival if diagnosed at or above 1 year old. On multivariate analysis, administration of S + CT + RT, S + CT + BMT/SCT, or S + CT + RT + BMT/SCT combination therapy predicted significantly (p < 0.05) increased OS compared to surgery alone.

Conclusion: AT/RTs are CNS tumors where those diagnosed under 1 year old have a significantly worse prognosis. Our study demonstrates that while traditional CT, RT, and BMT/SCT combination regimens prolong life, overall survival in this population is still low.

Keywords: National Cancer Database Study; Pediatric NCDB; Pediatric brain tumor; Pediatric tumor.

MeSH terms

Central Nervous System Neoplasms* / therapy
Child, Preschool
Cohort Studies
Combined Modality Therapy
Female
Humans
Infant
Male
Prognosis
Rhabdoid Tumor* / therapy