Systemic chemotherapy of pediatric recurrent ependymomas: results from the German HIT-REZ studies.

Abstract

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.

• Authors: Jonas E Adolph, Gudrun Fleischhack, Christine Gaab, Ruth Mikasch, Martin Mynarek, Stefan Rutkowski, Ulrich Schüller, Stefan M Pfister, Kristian W Pajtler, Till Milde, Olaf Witt, Brigitte Bison, Monika Warmuth-Metz, Rolf-Dieter Kortmann, Stefan Dietzsch, Torsten Pietsch, Beate Timmermann, Stephan Tippelt, German GPOH HIT-Network
• Journal: Journal of neuro-oncology (J Neurooncol) Vol. 155 Issue 2 Pg. 193-202 (Nov 2021) ISSN: 1573-7373 [Electronic] United States
• PMID: 34657224 (Publication Type: Journal Article)
• Copyright: © 2021. The Author(s).

Chemical References

• Sirolimus

Topics

• Adolescent
• Brain Neoplasms (drug therapy, pathology)
• Child
• Child, Preschool
• Ependymoma (drug therapy, pathology)
• Germany
• Humans
• Neoplasm Recurrence, Local (drug therapy)
• Sirolimus (therapeutic use)
• Treatment Outcome