TY  - JOUR
A1  - Adolph, Jonas E.
A1  - Fleischhack, Gudrun
A1  - Gaab, Christine
A1  - Mikasch, Ruth
A1  - Mynarek, Martin
A1  - Rutkowski, Stefan
A1  - Schüller, Ulrich
A1  - Pfister, Stefan M.
A1  - Pajtler, Kristian W.
A1  - Milde, Till
A1  - Witt, Olaf
A1  - Bison, Brigitte
A1  - Warmuth-Metz, Monika
A1  - Kortmann, Rolf-Dieter
A1  - Dietzsch, Stefan
A1  - Pietsch, Torsten
A1  - Timmermann, Beate
A1  - Tippelt, Stephan
T1  - Systemic chemotherapy of pediatric recurrent ependymomas: results from the German HIT-REZ studies
JF  - Journal of Neuro-Oncology
N2  - Purpose
Survival in recurrent ependymoma (EPN) depends mainly on the extent of resection achieved. When complete resection is not feasible, chemotherapy is often used to extend progression-free and overall survival. However, no consistent effect of chemotherapy on survival has been found in patients with recurrent EPN.

Methods
Systemic chemotherapeutic treatment of 138 patients enrolled in the German HIT-REZ-studies was analyzed. Survival depending on the use of chemotherapy, disease-stabilization rates (RR), duration of response (DOR) and time to progression (TTP) were estimated.

Results
Median age at first recurrence was 7.6 years (IQR: 4.0–13.6). At first recurrence, median PFS and OS were 15.3 (CI 13.3–20.0) and 36.9 months (CI 29.7–53.4), respectively. The Hazard Ratio for the use of chemotherapy in local recurrences in a time-dependent Cox-regression analysis was 0.99 (CI 0.74–1.33). Evaluable responses for 140 applied chemotherapies were analyzed, of which sirolimus showed the best RR (50%) and longest median TTP [11.51 (CI 3.98; 14.0) months] in nine patients, with the strongest impact found when sirolimus was used as a monotherapy. Seven patients with progression-free survival > 12 months after subtotal/no-resection facilitated by chemotherapy were found. No definitive survival advantage for any drug in a specific molecularly defined EPN type was found.

Conclusion
No survival advantage for the general use of chemotherapy in recurrent EPN was found. In cases with incomplete resection, chemotherapy was able to extend survival in individual cases. Sirolimus showed the best RR, DOR and TTP out of all drugs analyzed and may warrant further investigation.
KW  - ependymoma
KW  - chemotherapy
KW  - recurrence
KW  - children
KW  - sirolimus
Y1  - 2021
U6  - http://nbn-resolving.de/urn/resolver.pl?urn:nbn:de:bvb:20-opus-308302
SN  - 0167-594X
SN  - 1573-7373
VL  - 155
IS  - 2
ER  -