Adamantinomatous craniopharyngioma associated with a compromised blood-brain barrier: patient series.
J Neurosurg Case Lessons
; 1(19): CASE2150, 2021 May 10.
Article
en En
| MEDLINE
| ID: mdl-35854837
ABSTRACT
BACKGROUND:
Adamantinomatous craniopharyngioma (ACP) is a highly morbid adult and pediatric brain tumor derived from epithelial remnants of the craniopharyngeal canal (Rathke's pouch), which gives rise to the anterior pituitary gland. Standard therapy includes maximal safe resection with or without radiation therapy. Systemic antitumor therapy remains elusive. Immune-related paracrine signaling involving the interleukin-6 receptor (IL-6R) may contribute to ACP pathogenesis. Tocilizumab, a recombinant humanized monoclonal antibody against IL-6R, is approved by the US Food and Drug Administration but does not cross an intact blood-brain barrier. OBSERVATIONS In a phase 0 trial design, a single dose of tocilizumab was delivered intravenously before clinically indicated surgical intervention in 3 children with ACP. The presence of tocilizumab was assayed in plasma, tumor tissue, tumor cyst fluid, and cerebrospinal fluid (n = 1) using a novel enzyme-linked immunosorbent assay. Tocilizumab reached ACP tumor tissue and/or cyst fluid after one systemic dose in every patient. LESSONS This finding helps explain extant data that indicate tocilizumab may contribute to ACP therapy. It further indicates that ACP does not reside behind an intact blood-brain barrier, dramatically broadening the range of potential antitumor therapies against this tumor. This has substantial implications for the design of future clinical trials for novel therapies against ACP in both children and adults.
ACP = adamantinomatous craniopharyngioma; BBB = bloodbrain barrier; CNS = central nervous system; CSF = cerebrospinal fluid; Cmax = maximum concentration; ELISA = enzyme-linked immunosorbent assay; IL-6 = interleukin-6; IL-6R = interleukin-6 receptor; adamantinomatous craniopharyngioma; bloodbrain barrier; craniopharyngioma; monoclonal antibody; tocilizumab
Texto completo:
1
Colección:
01-internacional
Base de datos:
MEDLINE
Tipo de estudio:
Risk_factors_studies
Idioma:
En
Revista:
J Neurosurg Case Lessons
Año:
2021
Tipo del documento:
Article