Immune checkpoint inhibition for pediatric patients with recurrent/refractory CNS tumors: a single institution experience.
J Neurooncol
; 149(1): 113-122, 2020 Aug.
Article
in En
| MEDLINE
| ID: mdl-32627129
ABSTRACT
INTRODUCTION:
Immune checkpoint inhibition through PD-1 and CTLA-4 blockade has shown efficacy in some adult malignancies and generated interest in pediatrics, including central nervous system (CNS) tumors. We describe our experience with immune checkpoint inhibition in recurrent/refractory pediatric CNS tumors.METHODS:
We performed a retrospective chart review of pediatric patients with recurrent or refractory CNS tumors treated with ipilimumab, nivolumab and/or pembrolizumab at Dana-Farber/Boston Children's Hospital between 2018 and 2019.RESULTS:
Eleven patients were identified. Diagnoses included diffuse intrinsic pontine glioma (DIPG) (n = 2), high-grade glioma (HGG) (n = 5), ependymoma (n = 1), craniopharyngioma (n = 1), high-grade neuroepithelial tumor (n = 1) and non-germinomatous germ cell tumor (NGGCT) (n = 1). Eight patients had recurrent disease, while three had refractory disease. Nine patients received combination therapy (ipilimumab/nivolumab); two patients received either nivolumab or pembrolizumab. Median time from diagnosis-to-treatment was 8 months (range 0.8-156). All patients received prior radiation therapy (RT), with median time from RT-to-immunotherapy was 3.8 years. One patient received concurrent then adjuvant immunotherapy with RT. Median duration of treatment was 6.1 months (range 1-25). Therapy was discontinued in nine patients seven due to disease progression and two due to toxicity (colitis; transaminitis). Other pertinent toxicities included Type 1 diabetes mellitus, hypothyroidism and skin toxicity. Based on iRANO criteria, best responses included partial response (n = 3), stable disease (n = 7) and progressive disease (n = 1). Durable response was noted in two patients.CONCLUSION:
Immune checkpoint inhibition was relatively well tolerated in a cohort of pediatric patients spanning several CNS tumor diagnoses. Results from prospective clinical trials will be critical to inform clinical decisions.Key words
Full text:
1
Collection:
01-internacional
Database:
MEDLINE
Main subject:
Salvage Therapy
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Central Nervous System Neoplasms
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Drug Resistance, Neoplasm
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Immune Checkpoint Inhibitors
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Neoplasm Recurrence, Local
Type of study:
Observational_studies
/
Prognostic_studies
/
Risk_factors_studies
Limits:
Adolescent
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Adult
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Child
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Child, preschool
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Female
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Humans
/
Male
Language:
En
Journal:
J Neurooncol
Year:
2020
Type:
Article
Affiliation country:
United States