Trimodality therapy for atypical teratoid/rhabdoid tumor is associated with improved overall survival: A surveillance, epidemiology, and end results analysis.
Adolescent
Child
Child, Preschool
Cohort Studies
Combined Modality Therapy
Female
Follow-Up Studies
Humans
Incidence
Infant
Infant, Newborn
Male
Michigan
/ epidemiology
Neoplasm Recurrence, Local
/ diagnosis
Population Surveillance
Prognosis
Rhabdoid Tumor
/ diagnosis
Survival Rate
Teratoma
/ diagnosis
adjuvant chemotherapy
atypical teratoid rhabdoid tumor
chemoradiotherapy
cohort studies
combined (triple) modality therapy
primary brain tumor
propensity score
Journal
Pediatric blood & cancer
ISSN: 1545-5017
Titre abrégé: Pediatr Blood Cancer
Pays: United States
ID NLM: 101186624
Informations de publication
Date de publication:
12 2019
12 2019
Historique:
received:
01
07
2019
revised:
29
07
2019
accepted:
31
07
2019
pubmed:
30
8
2019
medline:
17
3
2020
entrez:
30
8
2019
Statut:
ppublish
Résumé
Atypical teratoid/rhabdoid tumors (AT/RTs) are rare aggressive central nervous system tumors. The use of radiation therapy (RT) remains controversial, especially for patients younger than three years of age. The purpose of the current investigation is to robustly analyze the impact of RT among pediatric AT/RT patients using the Surveillance, Epidemiology, and End Results (SEER) database. SEER 18 Custom Data registries were queried for AT/RT (ICD-0-3 9508/3). A total of 190 pediatric AT/RT patients were identified, of whom 102 underwent surgery + chemotherapy and 88 underwent trimodality therapy. Univariate and multivariable analyses using Kaplan-Meier and Cox proportional hazards regression modeling were performed. Propensity-score matched analysis with inverse probability of treatment weighting was performed to account for indication bias. The landmark method was used to account for immortal time bias. The majority of patients were <3 years old (75.8%). Patients <3 were more likely to be treated without RT as compared with older patients (62% vs 38%). Doubly robust MVA identified distant disease as a negative prognostic factor (HR 2.1, P = 0.003), whereas trimodality therapy was strongly protective (HR 0.39, P < 0.001). Infants (<1), toddlers (1-2), and older children (3+) all benefited from trimodality therapy, with largest benefit for infants (HR 0.34, P = 0.02) and toddlers (HR 0.31, P < 0.001). The current study provides further evidence that trimodality therapy improves clinical outcomes among patients with AT/RT. This finding was most pronounced for younger patients; therefore, further studies are needed to confirm this finding in this vulnerable population.
Sections du résumé
BACKGROUND
Atypical teratoid/rhabdoid tumors (AT/RTs) are rare aggressive central nervous system tumors. The use of radiation therapy (RT) remains controversial, especially for patients younger than three years of age. The purpose of the current investigation is to robustly analyze the impact of RT among pediatric AT/RT patients using the Surveillance, Epidemiology, and End Results (SEER) database.
METHODS
SEER 18 Custom Data registries were queried for AT/RT (ICD-0-3 9508/3). A total of 190 pediatric AT/RT patients were identified, of whom 102 underwent surgery + chemotherapy and 88 underwent trimodality therapy. Univariate and multivariable analyses using Kaplan-Meier and Cox proportional hazards regression modeling were performed. Propensity-score matched analysis with inverse probability of treatment weighting was performed to account for indication bias. The landmark method was used to account for immortal time bias.
RESULTS
The majority of patients were <3 years old (75.8%). Patients <3 were more likely to be treated without RT as compared with older patients (62% vs 38%). Doubly robust MVA identified distant disease as a negative prognostic factor (HR 2.1, P = 0.003), whereas trimodality therapy was strongly protective (HR 0.39, P < 0.001). Infants (<1), toddlers (1-2), and older children (3+) all benefited from trimodality therapy, with largest benefit for infants (HR 0.34, P = 0.02) and toddlers (HR 0.31, P < 0.001).
CONCLUSION
The current study provides further evidence that trimodality therapy improves clinical outcomes among patients with AT/RT. This finding was most pronounced for younger patients; therefore, further studies are needed to confirm this finding in this vulnerable population.
Types de publication
Journal Article
Langues
eng
Sous-ensembles de citation
IM
Pagination
e27969Informations de copyright
© 2019 Wiley Periodicals, Inc.
Références
Packer RJ, Biegel JA, Blaney S, et al. Atypical teratoid/rhabdoid tumor of the central nervous system: report on workshop. J Pediatr Hematol Oncol. 2002;24(5):337-342.
Tekautz TM, Fuller CE, Blaney S, et al. Atypical teratoid/rhabdoid tumors (ATRT): improved survival in children 3 years of age and older with radiation therapy and high-dose alkylator-based chemotherapy. J Clin Oncol. 2005;23(7):1491-1499.
Chen Y, Wong T, Ho DM, et al. Impact of radiotherapy for pediatric CNS atypical teratoid/rhabdoid tumor (single institute experience). Int J Radiat Oncol Biol Phys. 2006;64(4):1038-1043.
Chi SN, Zimmerman MA, Yao X, et al. Intensive multimodality treatment for children with newly diagnosed CNS atypical teratoid rhabdoid tumor. J Clin Oncol. 2009;27(3):385-389.
Ginn KF, Gajjar A. Atypical teratoid rhabdoid tumor: current therapy and future directions. Front Oncol. 2012;2:114.
Ostrom QT, Chen YM, de Blank P, et al. The descriptive epidemiology of atypical teratoid/rhabdoid tumors in the united states, 2001-2010. Neuro Oncol. 2014;16(10):1392-1399.
Hilden JM, Meerbaum S, Burger P, et al. Central nervous system atypical teratoid/rhabdoid tumor: results of therapy in children enrolled in a registry. J Clin Oncol. 2004;22(14):2877-2884.
Biggs PJ, Garen PD, Powers JM, Garvin JJ. Malignant rhabdoid tumor of the central nervous system. Human Pathology. 1987;18(4):332-337.
Lee RS, Stewart C, Carter SL, et al. A remarkably simple genome underlies highly malignant pediatric rhabdoid cancers. J Clin Invest. 2012;122(8):2983-2988.
Hasselblatt M, Isken S, Linge A, et al. High-resolution genomic analysis suggests the absence of recurrent genomic alterations other than SMARCB1 aberrations in atypical teratoid/rhabdoid tumors. Genes Chromosomes Cancer. 2013;52(2):185-190.
Hasselblatt M, Nagel I, Oyen F, et al. SMARCA4-mutated atypical teratoid/rhabdoid tumors are associated with inherited germline alterations and poor prognosis. Acta Neuropathol. 2014;128(3):453-456.
Louis DN, Perry A, Reifenberger G, et al. The 2016 World Health Organization classification of tumors of the central nervous system: a summary. Acta Neuropathol. 2016;131(6):803-820.
Frühwald MC, Biegel JA, Bourdeaut F, Roberts CWM, Chi SN. Atypical teratoid/rhabdoid tumors-current concepts, advances in biology, and potential future therapies. Neuro-oncology. 2016;18(6):764-778.
Ho DM, Hsu CY, Wong TT, Ting LT, Chiang H. Atypical teratoid/rhabdoid tumor of the central nervous system: a comparative study with primitive neuroectodermal tumor/medulloblastoma. Acta Neuropathol. 2000;99(5):482-488.
Duffner PK, Cohen ME, Thomas PR, Lansky SB. The long-term effects of cranial irradiation on the central nervous system. Cancer. 1985;56(7 Suppl):1841-1846.
Ris MD, Packer R, Goldwein J, Jones-Wallace D, Boyett JM. Intellectual outcome after reduced-dose radiation therapy plus adjuvant chemotherapy for medulloblastoma: a children's cancer group study. J Clin Oncol. 2001;19(15):3470-3476.
Oyharcabal-Bourden V, Kalifa C, Gentet JC, et al. Standard-risk medulloblastoma treated by adjuvant chemotherapy followed by reduced-dose craniospinal radiation therapy: a French Society of Pediatric Oncology Study. J Clin Oncol. 2005;23(21):4726-4734.
Bishop AJ, McDonald MW, Chang AL, Esiashvili N. Infant brain tumors: incidence, survival, and the role of radiation based on Surveillance, Epidemiology, and End Results (SEER) data. Int J Radiat Oncol Biol Phys. 2012;82(1):341-347.
De Amorim Bernstein K, Sethi R, Trofimov A, et al. Early clinical outcomes using proton radiation for children with central nervous system atypical teratoid rhabdoid tumors. Int J Radiat Oncol Biol Phys. 2013;86(1):114-120.
Elsayad K, Kriz J, Samhouri L, et al. Long-term survival following additive radiotherapy in patients with atypical teratoid rhabdoid tumors. Strahlenther Onkol. 2016;192(8):569-581.
Lau CS, Mahendraraj K, Chamberlain RS. Atypical teratoid rhabdoid tumors: a population-based clinical outcomes study involving 174 patients from the Surveillance, Epidemiology, and End Results database (1973-2010). Cancer Manag Res. 2015;7:301-309.
Lee J, Kim D-S, Han JW, Suh C-O. Atypical teratoid/rhabdoid tumors in children treated with multimodal therapies: the necessity of upfront radiotherapy after surgery. Pediatr Blood Cancer. 2017;64(12):e26663.
McGovern SL, Okcu MF, Munsell MF, et al. Outcomes and acute toxicities of proton therapy for pediatric atypical teratoid/rhabdoid tumor of the central nervous system. Int J Radiat Oncol Biol Phys. 2014;90(5):1143-1152.
Weber DC, Ares C, Malyapa R, et al. Tumor control and QoL outcomes of very young children with atypical teratoid/rhabdoid tumor treated with focal only chemo-radiation therapy using pencil beam scanning proton therapy. J Neurooncol. 2015;121(2):389-397.
Fischer-Valuck BW, Chen I, Srivastava AJ, et al. Assessment of the treatment approach and survival outcomes in a modern cohort of patients with atypical teratoid rhabdoid tumors using the national cancer database. Cancer. 2017;123(4):682-687.
Austin PC. Goodness-of-fit diagnostics for the propensity score model when estimating treatment effects using covariate adjustment with the propensity score. Pharmacoepidemiol Drug Saf. 2008;17(12):1202-1217.
Rosenbaum PR. Propensity score. In: Encyclopedia of Biostatistics. Atlanta, GA: American Cancer Society; 2005.
Cole SR, Hernán MA. Constructing inverse probability weights for marginal structural models. Am J Epidemiol. 2008;168(6):656-664.
Suissa S. Immortal time bias in pharmacoepidemiology. Am J Epidemiol. 2008;167(4):492-499.
Anderson JR, Cain KC, Gelber RD. Analysis of survival by tumor response. J Clin Oncol. 1983;1(11):710-719.
Squire SE, Chan MD, Marcus KJ. Atypical teratoid/rhabdoid tumor: the controversy behind radiation therapy. J Neurooncol. 2007;81(1):97-111.
Schrey D, Carceller Lechón F, Malietzis G, et al. Multimodal therapy in children and adolescents with newly diagnosed atypical teratoid rhabdoid tumor: individual pooled data analysis and review of the literature. J Neurooncol. 2016;126(1):81-90.
Park ES, Sung KW, Baek HJ, et al. Tandem high-dose chemotherapy and autologous stem cell transplantation in young children with atypical teratoid/rhabdoid tumor of the central nervous system. J Korean Med Sci. 2012;27(2):135-140.
Buscariollo DL, Park HS, Roberts KB, Yu JB. Survival outcomes in atypical teratoid rhabdoid tumor for patients undergoing radiotherapy in a Surveillance, Epidemiology, and End Results analysis. Cancer. 2012;118(17):4212-4219.
Wegner RE, Abel S, Horne ZD, et al. National trends in radiation dose escalation for glioblastoma. Radiat Oncol J. 2019;37(1):13-21.
Bilimoria KY, Bentrem DJ, Stewart AK, Winchester DP, Ko CY. Comparison of commission on cancer-approved and -nonapproved hospitals in the united states: implications for studies that use the national cancer data base. J Clin Oncol. 2009;27(25):4177-4181.
Slavc I, Chocholous M, Leiss U, et al. Atypical teratoid rhabdoid tumor: improved long-term survival with an intensive multimodal therapy and delayed radiotherapy. The Medical University of Vienna experience 1992-2012. Cancer Med. 2014;3(1):91-100.
Johann PD, Erkek S, Zapatka M, et al. Atypical teratoid/rhabdoid tumors are comprised of three epigenetic subgroups with distinct enhancer landscapes. Cancer Cell. 2016;29(3):379-393.
Gondi V, Yock TI, Mehta MP. Proton therapy for paediatric CNS tumours - improving treatment-related outcomes. Nat Rev Neurol. 2016;12(6):334-345.