Spinal cord atypical teratoid/rhabdoid tumors in children
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Spinal cord atypical teratoid/rhabdoid tumors in children : Clinical, genetic, and outcome characteristics in a representative European cohort. / Benesch, Martin; Nemes, Karolina; Neumayer, Petra; Hasselblatt, Martin; Timmermann, Beate; Bison, Brigitte; Ebetsberger-Dachs, Georg; Bourdeaut, Franck; Dufour, Christelle; Biassoni, Veronica; Morales La Madrid, Andrés; Entz-Werle, Natacha; Laithier, Véronique; Quehenberger, Franz; Weis, Serge; Sumerauer, David; Siebert, Reiner; Bens, Susanne; Schneppenheim, Reinhard; Kool, Marcel; Modena, Piergiorgio; Fouyssac, Fanny; C Frühwald, Michael.
In: PEDIATR BLOOD CANCER, Vol. 67, No. 1, 01.2020, p. e28022.Research output: SCORING: Contribution to journal › SCORING: Journal article › Research › peer-review
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TY - JOUR
T1 - Spinal cord atypical teratoid/rhabdoid tumors in children
T2 - Clinical, genetic, and outcome characteristics in a representative European cohort
AU - Benesch, Martin
AU - Nemes, Karolina
AU - Neumayer, Petra
AU - Hasselblatt, Martin
AU - Timmermann, Beate
AU - Bison, Brigitte
AU - Ebetsberger-Dachs, Georg
AU - Bourdeaut, Franck
AU - Dufour, Christelle
AU - Biassoni, Veronica
AU - Morales La Madrid, Andrés
AU - Entz-Werle, Natacha
AU - Laithier, Véronique
AU - Quehenberger, Franz
AU - Weis, Serge
AU - Sumerauer, David
AU - Siebert, Reiner
AU - Bens, Susanne
AU - Schneppenheim, Reinhard
AU - Kool, Marcel
AU - Modena, Piergiorgio
AU - Fouyssac, Fanny
AU - C Frühwald, Michael
N1 - © 2019 Wiley Periodicals, Inc.
PY - 2020/1
Y1 - 2020/1
N2 - BACKGROUND: Case reports have portrayed spinal cord atypical teratoid/rhabdoid tumor (spATRT) as an aggressive form of ATRT. We conducted a retrospective European survey to collect data on clinical characteristics, molecular biology, treatment, and outcome of children with intramedullary spATRT.METHODS: Scrutinizing a French national series and the European Rhabdoid Registry database, we identified 13 patients (median age 32 months; metastatic disease at diagnosis, n = 6). Systemic postoperative chemotherapy was administered to all patients; three received intrathecal therapy and six were irradiated (craniospinal, n = 3; local, n = 3).RESULTS: Median observation time was 8 (range, 1-93) months. Progression-free and overall survival rates at 1 and (2 years) were 35.2% ± 13.9% (26.4% ± 12.9%) and 38.5% ± 13.5% (23.1% ± 11.7%). Four patients (ATRT-SHH, n = 2; ATRT-MYC, n = 1; DNA methylation subgroup not available, n = 1) achieved complete remission (CR); two of them are alive in CR 69 and 72 months from diagnosis. One patient relapsed after CR and is alive with progressive disease (PD) and one died of the disease. Three patients (ATRT-MYC, n = 2; subgroup not available, n = 1) died after 7 to 22 months due to PD after having achieved a partial remission (n = 1) or stabilization (n = 2). Five patients (ATRT-MYC, n = 2; subgroup not available, n = 3) developed early PD and died. One patient (ATRT-MYC) died of intracerebral hemorrhage prior to response evaluation.CONCLUSIONS: Long-term survival is achievable in selected patients with spATRT using aggressive multimodality treatment. Larger case series and detailed molecular analyses are needed to understand differences between spATRT and their inracranial counterparts and the group of extradural malignant rhabdoid tumors.
AB - BACKGROUND: Case reports have portrayed spinal cord atypical teratoid/rhabdoid tumor (spATRT) as an aggressive form of ATRT. We conducted a retrospective European survey to collect data on clinical characteristics, molecular biology, treatment, and outcome of children with intramedullary spATRT.METHODS: Scrutinizing a French national series and the European Rhabdoid Registry database, we identified 13 patients (median age 32 months; metastatic disease at diagnosis, n = 6). Systemic postoperative chemotherapy was administered to all patients; three received intrathecal therapy and six were irradiated (craniospinal, n = 3; local, n = 3).RESULTS: Median observation time was 8 (range, 1-93) months. Progression-free and overall survival rates at 1 and (2 years) were 35.2% ± 13.9% (26.4% ± 12.9%) and 38.5% ± 13.5% (23.1% ± 11.7%). Four patients (ATRT-SHH, n = 2; ATRT-MYC, n = 1; DNA methylation subgroup not available, n = 1) achieved complete remission (CR); two of them are alive in CR 69 and 72 months from diagnosis. One patient relapsed after CR and is alive with progressive disease (PD) and one died of the disease. Three patients (ATRT-MYC, n = 2; subgroup not available, n = 1) died after 7 to 22 months due to PD after having achieved a partial remission (n = 1) or stabilization (n = 2). Five patients (ATRT-MYC, n = 2; subgroup not available, n = 3) developed early PD and died. One patient (ATRT-MYC) died of intracerebral hemorrhage prior to response evaluation.CONCLUSIONS: Long-term survival is achievable in selected patients with spATRT using aggressive multimodality treatment. Larger case series and detailed molecular analyses are needed to understand differences between spATRT and their inracranial counterparts and the group of extradural malignant rhabdoid tumors.
U2 - 10.1002/pbc.28022
DO - 10.1002/pbc.28022
M3 - SCORING: Journal article
C2 - 31571386
VL - 67
SP - e28022
JO - PEDIATR BLOOD CANCER
JF - PEDIATR BLOOD CANCER
SN - 1545-5009
IS - 1
ER -
