Central Nervous System Atypical Teratoid/Rhabdoid Tumor: Results of Therapy in Children Enrolled in a Registry

Hilden, Joanne M.; Meerbaum, Sharon O.; Burger, Peter C.; Finlay, Jonathan L.; Janss, Anna J.; Scheithauer, Bernd W.; Walter, Andrew W.; Rorke, Lucy B.; Biegel, Jaclyn A.

doi:10.1200/jco.2004.07.073

Cited by 391 publications

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“…11,14 There is also evolving evidence that dose-intensive chemotherapy and autologous hematopoietic stem cell rescue, particularly with the use of high-dose methotrexate, may be of benefit in young children with ATRT. 1,15,24,25 Despite its benefits, tumor progression during adjuvant chemotherapy, particularly locoregionally, is relatively common and is associated with decreased survival. 1,3,9,13 Thus, interest in a potential role for adjuvant RT in the prevention of local recurrence and cerebrospinal fluid dissemination has continued to grow.…”

Section: Discussionmentioning

confidence: 99%

“…2,5,7 Although ATRT comprises <5% of CNS tumors in children aged <18 years, it reportedly represents up to 20% of CNS tumors in children aged <3 years. 1,4,8 However, it is believed that the true incidence may be higher because of misdiagnosis given similarities in imaging and histologic appearance to other high-grade embryonal CNS malignancies. 2,3,8,9 Current knowledge describing clinical characteristics, prognosis, and therapy for ATRT is derived mostly from small, retrospective, observational studies.…”

Section: Introductionmentioning

confidence: 99%

“…[1][2][3][4][5] ATRT is extremely rare in adults, and only 31 patients have been reported in the literature. 6 Originally described in the 1980s, ATRT has been widely recognized as a rare but important clinical entity only over the past decade.…”

Section: Introductionmentioning

confidence: 99%

“…The prognosis for patients with ATRT remains dismal, and the historic median overall survival (OS) ranges from 6 months to 18 months. 1,3,4,9,10 Therapeutic approaches for ATRT historically have relied on data from treatments of other childhood CNS tumors, particularly primitive neuroendocrine tumors and medulloblastoma, which have a considerably better prognoses. 5 However, preliminary results from the first prospective clinical trial in children with ATRT indicate that intensive multimodality treatment, including resection, CNS radiotherapy (RT), and chemotherapy based on the Intergroup Rhabdomyosarcoma Study Group (IRS) III protocol, may lead to longer survival, with an estimated 2-year OS survival rate of 70% AE 10%.…”

Section: Introductionmentioning

confidence: 99%

“…Given the rarity of ATRT, large retrospective analyses are important sources of information to guide the development of treatment strategies. 1,9,10,[12][13][14][15] The objectives of the current study were to identify clinical and prognostic factors for ATRT and to determine whether the use of RT has changed in the last decade in light of information suggesting its importance in the management of ATRT using the National Cancer Institute (NCI) Surveillance, Epidemiology, and End Results (SEER) database. The SEER Program collects cancer survival and incidence information from population-based cancer registries that encompass approximately 28% of the US population, which provides an opportunity to study rare entities like ATRT.…”

Section: Introductionmentioning

confidence: 99%

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Survival outcomes in atypical teratoid rhabdoid tumor for patients undergoing radiotherapy in a Surveillance, Epidemiology, and End Results analysis

Buscariollo

Park

Roberts

et al. 2011

Cancer

155

161

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BACKGROUND: Atypical teratoid rhabdoid tumor (ATRT) is a rare central nervous system malignancy with a poor prognosis that affects mostly young children. Although radiotherapy (RT) historically has been delayed in patients aged <3 years, emerging evidence suggests a role for RT to achieve long-term survivorship. Clinical features and age-dependent trends of RT use were evaluated for patients with ATRT. METHODS: The National Cancer Institute's Surveillance, Epidemiology, and End Results database was used to identify 144 patients with ATRT from 1973 to 2008. The primary endpoint was median overall survival (OS). Clinical and treatment variables were assessed for an association with OS using Cox proportional hazards models. Landmark analysis was used to correct for immortal time bias of adjuvant RT. RESULTS: The median age at diagnosis was 1 year (range, 0-67 years). Gross total resection of the primary tumor was achieved in 39% of patients, and 33% of patients received RT. From 1992 to 2008, RT use increased 2.4-fold in patients aged 3 years. The median OS for was 10 months. In multivariate analyses, metastatic disease (hazard ratio, 2.83; 95% confidence interval, 1.53-5.23; P ¼ .001) and RT (hazard ratio, 0.10; 95% confidence interval, 0.01-0.73; P ¼ .02) were identified as independent predictors of survival. Landmark analysis confirmed a robust association between RT use and survival, which was attenuated in patients ages 4 to 17 years compared with younger patients. CONCLUSIONS: The current results indicated that RT may offer a significant survival benefit for patients with ATRT and that patients aged 3 years may derive more benefit from initial RT compared with older children. The authors concluded that prospective clinical trials are needed to examine the role of RT in the initial management of ATRT in patients aged <3 years. Cancer 2012;118:4212-9. V C 2011 American Cancer Society.KEYWORDS: atypical teratoid rhabdoid tumor, central nervous system, epidemiology, radiotherapy, children. INTRODUCTIONAtypical teratoid rhabdoid tumor (ATRT) of the central nervous system (CNS) is a highly malignant embryonal neoplasm (grade 4 according to the World Health Organization classification) that typically occurs in children aged <3 years. 1-5 ATRT is extremely rare in adults, and only 31 patients have been reported in the literature. 6 Originally described in the 1980s, ATRT has been widely recognized as a rare but important clinical entity only over the past decade. 2,4 The diagnosis of ATRT has been greatly facilitated by the rise in accessibility of immunohistochemical testing in the late 1990s, which takes advantage of the frequent loss of switch/sucrose nonfermentable (SWI/SNF)-related, matrix-associated, actin-dependent regulator of chromatin, subfamily b, member 1 (SMARCB1) (also known as SNF5 homolog [hSNF5] and INI1) tumor suppressor gene products in these tumors. 2,5,7 Although ATRT comprises <5% of CNS tumors in children aged <18 years, it reportedly represents up to 20% of CNS tumors in children aged <3 years...

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