Atypical teratoid rhabdoid tumor: improved long‐term survival with an intensive multimodal therapy and delayed radiotherapy. The Medical University of Vienna Experience 1992–2012

Abstract

Atypical teratoid rhabdoid tumors (ATRTs) are recently defined highly aggressive embryonal central nervous system tumors with a poor prognosis and no definitive guidelines for treatment. We report on the importance of an initial correct diagnosis and disease-specific therapy on outcome in 22 consecutive patients and propose a new treatment strategy. From 1992 to 2012, nine patients initially diagnosed correctly as ATRT (cohort A, median age 24 months) were treated according to an intensive multimodal regimen (MUV-ATRT) consisting of three 9-week courses of a dose-dense regimen including doxorubicin, cyclophosphamide, vincristine, ifosfamide, cisplatin, etoposide, and methotrexate augmented with intrathecal therapy, followed by high-dose chemotherapy (HDCT) and completed with local radiotherapy. Thirteen patients were treated differently (cohort B, median age 30 months) most of whom according to protocols in use for their respective diagnoses. As of July 2013, 5-year overall survival (OS) and event-free survival (EFS) for all 22 consecutive patients was 56.3 ± 11.3% and 52.9 ± 11.0%, respectively. For MUV-ATRT regimen-treated patients (cohort A) 5-year OS was 100% and EFS was 88.9 ± 10.5%. For patients treated differently (cohort B) 5-year OS and EFS were 28.8 ± 13.1%. All nine MUV-ATRT regimen-treated patients are alive for a median of 76 months (range: 16–197), eight in first complete remission. Our results compare favorably to previously published data. The drug combination and sequence used in the proposed MUV-ATRT regimen appear to be efficacious in preventing early relapses also in young children with M1–M3 stage disease allowing postponement of radiotherapy until after HDCT.

Improved 6‐year overall survival in AT/RT – results of the registry study Rhabdoid 2007

“…Rather provocative data have been published by Slavc et al with OS rates of 100% in nine patients. Independent confirmation is currently missing [15].…”

Atypical teratoid rhabdoid tumor: long-term survival after chemoradiotherapy

“…Higher powered studies may likely affirm that long-term overall survival can be reached in these patients with the use of intensive chemoradiotherapy after surgical debulking [12]. However, even in the face of aggressive treatment, it has already been reported in some cases that these tumors can recur up to 20 years from initial therapy [13].…”

“…Though impairments did not affect daily functioning, radiation may be incompletely associated with sequelae. This raises the question that though most clinical trials for this tumor have used photon beam radiotherapy [8,12], the growth of radiotherapy technology necessitates questions of whether other radiation modalities are superior. A case report from Scandinavia has described long-term survival with trimodality therapy using Gamma Knife radiosurgery with a dose of 18 Gy in one fraction (biologically equal to 50.…”

A case of relapsing spinal atypical teratoid/rhabdoid tumor (AT/RT) responding to vinorelbine, cyclophosphamide, and celecoxib

“…Because of its rapid progression and its severe prognosis, front-line multimodal intensive treatment is the widest adopted therapeutic option [3,[6][7][8]. Recent and most successful studies consist, after surgery, with the goal of achieving gross tumor resection, intensive systemic and intrathecal chemotherapy, followed by highdose chemotherapy (HDCT) with autologous hemopoietic stem cells rescue [3,[6][7][8], and radiotherapy. The role of HDCT is still unclear because limited data are available.…”