TY  - JOUR
A1  - Bartelheim, Kerstin
A1  - Nemes, Karolina
A1  - Seeringer, Angela
A1  - Kerl, Kornelius
A1  - Buechner, Jochen
A1  - Boos, Joachim
A1  - Graf, Norbert
A1  - Dürken, Matthias
A1  - Gerss, Joachim
A1  - Hasselblatt, Martin
A1  - Kortmann, Rolf-Dieter
A1  - Teichert von Luettichau, Irene
A1  - Nagel, Inga
A1  - Nygaard, Randi
A1  - Oyen, Florian
A1  - Quiroga, Eduardo
A1  - Schlegel, Paul-Gerhardt
A1  - Schmid, Irene
A1  - Schneppenheim, Reinhard
A1  - Siebert, Reiner
A1  - Solano-Paez, Palma
A1  - Timmermann, Beate
A1  - Warmuth-Metz, Monika
A1  - Frühwald, Michael Christoph
T1  - Improved 6-year overall survival in AT/RT - results of the registry study Rhabdoid 2007
JF  - Cancer Medicine
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.
KW  - AT/RT
KW  - EU‐RHAB Registry
KW  - pediatric brain tumor
KW  - Rhabdoid 2007
Y1  - 2016
U6  - http://nbn-resolving.de/urn/resolver.pl?urn:nbn:de:bvb:20-opus-164799
VL  - 5
IS  - 8
ER  -