Publication details

Evaluation of age-dependent treatment strategies for children and young adults with pineoblastoma: analysis of pooled European Society for Paediatric Oncology (SIOP-E) and US Head Start data

Authors	MYNAREK Martin PIZER Barry DUFOUR Christelle VUURDEN Dannis van GARAMI Miklos MASSIMINO Maura FANGUSARO Jason DAVIDSON Tom GIL-DA-COSTA Maria Joao ŠTĚRBA Jaroslav BENESCH Martin GERBER Nicolas JUHNKE B. Ole KWIECIEN Robert PIETSCH Torsten KOOL Marcel CLIFFORD Steve ELLISON David W. GIANGASPERO Felice WESSELING Pieter GILLES Floyd GOTTARDO Nicholas FINLAY Jonathan L. RUTKOWSKI Stefan HOFF Katja von
Year of publication	2017
Type	Article in Periodical
Magazine / Source	Neuro-Oncology
MU Faculty or unit	Faculty of Medicine
Citation
Web	http://dx.doi.org/10.1093/neuonc/now234
Doi	http://dx.doi.org/10.1093/neuonc/now234
Field	Oncology and hematology
Keywords	high-dose chemotherapy; pediatric; pineoblastoma; radiotherapy; treatment
Description	Background. Pineoblastoma is a rare pineal region brain tumor. Treatment strategies have reflected those for other malignant embryonal brain tumors. Patients and Methods. Original prospective treatment and outcome data from international trial groups were pooled. Cox regression models were developed considering treatment elements as time-dependent covariates. Results. Data on 135 patients with pineoblastoma aged 0.01-20.7 (median 4.9) years were analyzed. Median observation time was 7.3 years. Favorable prognostic factors were age >= 4 years (hazard ratio [HR] for progression-free survival [PFS] 0.270, P < .001) and administration of radiotherapy (HR for PFS 0.282, P < .001). Metastatic disease (HR for PFS 2.015, P =.006), but not postoperative residual tumor, was associated with unfavorable prognosis. In 57 patients < 4 years old, 5-year PFS/overall survival (OS) were 11 +/- 4%/12 +/- 4%. Two patients survived after chemotherapy only, while 3 of 16 treated with craniospinal irradiation (CSI) with boost, and 3 of 5 treated with high-dose chemotherapy (HDCT) and local radiotherapy survived. In 78 patients aged >= 4 years, PFS/OS were 72 +/- 7%/73 +/- 7% for patients without metastases, and 50 +/- 10%/55 +/- 10% with metastases. Seventy-three patients received radiotherapy (48 conventionally fractionated CSI, median dose 35.0 [ 18.0-45.0] Gy, 19 hyperfractionated CSI, 6 local radiotherapy), with (n = 68) or without (n = 6) chemotherapy. The treatment sequence had no impact; application of HDCT had weak impact on survival in older patients. Conclusion. Survival is poor in young children treated without radiotherapy. In these patients, combination of HDCT and local radiotherapy may warrant further evaluation in the absence of more specific or targeted treatments. CSI combined with chemotherapy is effective for older non-metastatic patients.