Improved 6-year overall survival in AT/RT - results of the registry study Rhabdoid 2007
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Improved 6-year overall survival in AT/RT - results of the registry study Rhabdoid 2007. / Bartelheim, Kerstin; Nemes, Karolina; Seeringer, Angela; Kerl, Kornelius; Buechner, Jochen; Boos, Joachim; Graf, Norbert; Dürken, Matthias; Gerss, Joachim; Hasselblatt, Martin; Kortmann, Rolf-Dieter; Teichert von Luettichau, Irene; Nagel, Inga; Nygaard, Randi; Oyen, Florian; Quiroga, Eduardo; Schlegel, Paul-Gerhardt; Schmid, Irene; Schneppenheim, Reinhard; Siebert, Reiner; Solano-Paez, Palma; Timmermann, Beate; Warmuth-Metz, Monika; Frühwald, Michael Christoph.
In: CANCER MED-US, Vol. 5, No. 8, 08.2016, p. 1765-75.Research output: SCORING: Contribution to journal › SCORING: Journal article › Research › peer-review
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TY - JOUR
T1 - Improved 6-year overall survival in AT/RT - results of the registry study Rhabdoid 2007
AU - Bartelheim, Kerstin
AU - Nemes, Karolina
AU - Seeringer, Angela
AU - Kerl, Kornelius
AU - Buechner, Jochen
AU - Boos, Joachim
AU - Graf, Norbert
AU - Dürken, Matthias
AU - Gerss, Joachim
AU - Hasselblatt, Martin
AU - Kortmann, Rolf-Dieter
AU - Teichert von Luettichau, Irene
AU - Nagel, Inga
AU - Nygaard, Randi
AU - Oyen, Florian
AU - Quiroga, Eduardo
AU - Schlegel, Paul-Gerhardt
AU - Schmid, Irene
AU - Schneppenheim, Reinhard
AU - Siebert, Reiner
AU - Solano-Paez, Palma
AU - Timmermann, Beate
AU - Warmuth-Metz, Monika
AU - Frühwald, Michael Christoph
N1 - © 2016 The Authors. Cancer Medicine published by John Wiley & Sons Ltd.
PY - 2016/8
Y1 - 2016/8
N2 - Atypical teratoid rhabdoid tumors (AT/RT) are characterized by mutations and subsequent inactivation of SMARCB1 (INI1, hSNF5), a predilection for very young children and an unfavorable outcome. The European Registry for rhabdoid tumors (EU-RHAB) was established to generate a common European database and to establish a standardized treatment regimen as the basis for phase I/II trials. Thus, genetic analyses, neuropathologic and radiologic diagnoses, and a consensus treatment regimen were prospectively evaluated. From 2005 to 2009, 31 patients with AT/RT from four countries were recruited into the registry study Rhabdoid 2007 and treated with systemic and intraventricular chemotherapy. Eight patients received high-dose chemotherapy, 23 radiotherapy, and 17 maintenance therapy. Reference evaluations were performed in 64% (genetic analyses, FISH, MLPA, sequencing) up to 97% (neuropathology, INI1 stain). Germ-line mutations (GLM) were detected in 6/21 patients. Prolonged overall survival was associated with age above 3 years, radiotherapy and achievement of a complete remission. 6-year overall and event-free survival rates were 46% (±0.10) and 45% (±0.09), respectively. Serious adverse events and one treatment-related death due to insufficiency of a ventriculo peritoneal shunt (VP-shunt) and consecutive herniation were noted. Acquisition of standardized data including reference diagnosis and a standard treatment schedule improved data quality along with a survival benefit. Treatment was feasible with significant but manageable toxicity. Although our analysis is biased due to heterogeneous adherence to therapy, EU-RHAB provides the best available basis for phase I/II clinical trials.
AB - Atypical teratoid rhabdoid tumors (AT/RT) are characterized by mutations and subsequent inactivation of SMARCB1 (INI1, hSNF5), a predilection for very young children and an unfavorable outcome. The European Registry for rhabdoid tumors (EU-RHAB) was established to generate a common European database and to establish a standardized treatment regimen as the basis for phase I/II trials. Thus, genetic analyses, neuropathologic and radiologic diagnoses, and a consensus treatment regimen were prospectively evaluated. From 2005 to 2009, 31 patients with AT/RT from four countries were recruited into the registry study Rhabdoid 2007 and treated with systemic and intraventricular chemotherapy. Eight patients received high-dose chemotherapy, 23 radiotherapy, and 17 maintenance therapy. Reference evaluations were performed in 64% (genetic analyses, FISH, MLPA, sequencing) up to 97% (neuropathology, INI1 stain). Germ-line mutations (GLM) were detected in 6/21 patients. Prolonged overall survival was associated with age above 3 years, radiotherapy and achievement of a complete remission. 6-year overall and event-free survival rates were 46% (±0.10) and 45% (±0.09), respectively. Serious adverse events and one treatment-related death due to insufficiency of a ventriculo peritoneal shunt (VP-shunt) and consecutive herniation were noted. Acquisition of standardized data including reference diagnosis and a standard treatment schedule improved data quality along with a survival benefit. Treatment was feasible with significant but manageable toxicity. Although our analysis is biased due to heterogeneous adherence to therapy, EU-RHAB provides the best available basis for phase I/II clinical trials.
KW - Journal Article
U2 - 10.1002/cam4.741
DO - 10.1002/cam4.741
M3 - SCORING: Journal article
C2 - 27228363
VL - 5
SP - 1765
EP - 1775
JO - CANCER MED-US
JF - CANCER MED-US
SN - 2045-7634
IS - 8
ER -
