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Selumetinib for plexiform neurofibromas in neurofibromatosis type 1: a single-institution experience

2020-04Journal article Restricted access
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dc.contributor.authorEspirito Santo, Vera
dc.contributor.authorPassos, Joao
dc.contributor.authorNzwalo, Hipólito
dc.contributor.authorCarvalho, Ines
dc.contributor.authorSantos, Filipa
dc.contributor.authorMartins, Carmo
dc.contributor.authorSalgado, Lucilia
dc.contributor.authorSilva, Conceicao e
dc.contributor.authorVinhais, Sofia
dc.contributor.authorVilares, Miguel
dc.contributor.authorSalgado, Duarte
dc.contributor.authorNunes, Sofia
dc.date.accessioned2021-06-24T11:35:53Z
dc.date.available2021-06-24T11:35:53Z
dc.date.issued2020-04
dc.description.abstractBackground Plexiform neurofibromas (PN) are the most frequent tumors associated with Neurofibromatosis type 1 (NF-1). PN can cause significant complications, including pain, functional impairment, and disfigurement. There is no efficient medical treatment and, surgical resection of large PN is frequently infeasible. Selumetinib (AZD6244/ARRY-142886) is a mitogen-activated protein kinase enzyme (MEK1/2) inhibitor and works by targeting the MAPK pathway. It is an investigational treatment option for inoperable symptomatic PN associated with NF-1. Herein, we describe a single institutional experience with selumetinib for inoperable PN in NF-1. Methods Case series study of demographics, clinical, baseline characteristics, treatment effect, and follow-up of consecutive genetically confirmed NF1 patients with inoperable PN associated with significant or potential significant morbidity treated with selumetinib (April 2018 to April 2019). Results Nineteen patients were treated with selumetinib. Predominant target locations were head and neck (31.6%, 6/19), chest (26.3%, 5/19) and pelvis (21%, 4/19) and the most important comorbidities were disfigurement (47.4%, 9/19) and pain (26.3%, 5/19). The mean follow-up time was 223 days (range 35-420 days). All but one had sustained clinical improvement, mainly in the first 60-90 days of treatment. In one patient, the treatment was suspended after 168 days (lack of clear benefit and left ventricular ejection fraction drop). There were no adverse effects leading to treatment suspension. Conclusions In the first observational study of selumetinib for NF-1 associated PN we showed that the drug was associated with clinical and radiological improvement. Our study also confirms the safety described in the clinical trials.
dc.identifier.doi10.1007/s11060-020-03443-6
dc.identifier.issn0167-594X
dc.identifier.urihttp://hdl.handle.net/10400.1/16563
dc.language.isoeng
dc.peerreviewedyes
dc.publisherSpringer
dc.subjectSelumetinib
dc.subjectPlexiform neurofibromas
dc.subjectNeurofibromatosis
dc.subjectTreatment
dc.subject.otherOncology; Neurosciences & Neurology
dc.titleSelumetinib for plexiform neurofibromas in neurofibromatosis type 1: a single-institution experience
dc.typejournal article
dspace.entity.typePublication
oaire.citation.endPage463
oaire.citation.issue2
oaire.citation.startPage459
oaire.citation.titleJournal of Neuro-Oncology
oaire.citation.volume147
person.familyNameNzwalo
person.givenNameHipólito
person.identifier337064
person.identifier.ciencia-id2C1F-E4F3-2C79
person.identifier.orcid0000-0002-1502-3534
person.identifier.ridAAG-3931-2020
person.identifier.scopus-author-id36057285600
rcaap.rightsrestrictedAccess
rcaap.typearticle
relation.isAuthorOfPublication287f7d4e-5ad8-4794-b526-c61d32c00446
relation.isAuthorOfPublication.latestForDiscovery287f7d4e-5ad8-4794-b526-c61d32c00446

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