Efficacy and safety profile of selumetinib in symptomatic inoperable plexiform neurofibromas

Panda, Prateek Kumar; Sihag, Rakesh Kumar; Sharawat, Indar Kumar

doi:10.23736/s0390-5616.21.05528-4

Cited by 3 publications

(3 citation statements)

References 21 publications

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“…6 Finally, in a study from India, the verdict given was that selumetinib can produce sustained shrinkage in most patients with NF1 and symptomatic plexiform neurofibroma and provide meaningful benefit in functional ability, acceptable safety profile and absence of cumulative toxic effects, with more robust evidence in children than previous studies. 7 The estimated progressionfree survival at 3 years was 84%. 8…”

Section: Medical Management With Mitogen-activated Protein Kinase Inh...mentioning

confidence: 98%

Plexiform neurofibromatosis of face – Recent advances in the management

Bhattacharya¹,

Bhattacharya²,

Bhattacharya³

2023

IJASHNB

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Plexiform neurofibromas (PNs) are benign tumors that grow in the nerves of people with neurofibromatosis type 1 (NF1), a genetic disorder that affects the development and growth of nerve cell tumors. PNs can occur anywhere in the body but are most commonly found in the skin, nerves, and bones. They can cause disfigurement and functional impairment, and may also increase the risk of malignant transformation. Treatment options include surgery, radiation therapy, and medications to manage symptoms.

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Section: Medical Management With Mitogen-activated Protein Kinase Inh...mentioning

confidence: 98%

Plexiform neurofibromatosis of face – Recent advances in the management

Bhattacharya¹,

Bhattacharya²,

Bhattacharya³

2023

IJASHNB

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“…1 Chronic paronychia is challenging to treat and may lead to early therapy discontinuation. 2 Although this cutaneous reaction is well-known, no histopathological characterisations of nail unit toxicity in children have been reported.…”

Section: Introductionmentioning

confidence: 99%

“…A common cutaneous reaction to selumetinib is the development of paronychia, possibly evolving into onychocryptosis with onset time ranging from weeks to several months after the start of treatment 1 . Chronic paronychia is challenging to treat and may lead to early therapy discontinuation 2 . Although this cutaneous reaction is well‐known, no histopathological characterisations of nail unit toxicity in children have been reported.…”

Section: Introductionmentioning

confidence: 99%

Histopathologic features of selumetinib‐induced paronychia in a child with neurofibromatosis type 1

Borgia,

Ferro,

Piccolo

et al. 2023

JEADV Clinical Practice

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We report a 4‐year‐old girl developing therapy refractory paronychia induced by selumetinib, an oral selective inhibitor of mitogen‐activated protein kinase 1/2 prescribed for a large submandibular plexiform neurofibroma. Although this cutaneous reaction is well‐known and more prevalent in children than in adults, no histopathological characterisations of nail unit toxicity in children on selumetinib have been reported so far. We show histopathological studies on patient‐derived periungual inflamed skin to investigate the cutaneous impact of selumetinib therapy. Our findings are consistent with those of epidermal growth factor receptor inhibitors and support the role of a non‐specific immune activation rather than opportunistic infection in the underlying mechanism of the disease. Partial bilateral matricectomies with electrocautery were resolutive, and the child restarted selumetinib with no recurrence of paronychia during a follow‐up period of 3 months after nail surgery.

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A single-center case study series assessing the effect of selumetinib use in patients with neurofibromatosis-related plexiform neurofibromas

Passos,

Soares,

Salgado

et al. 2024

Neuro-Oncology Advances

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Background Neurofibromatosis type 1 (NF1) is a common genetic disorder of phenotypic variability with age-dependent penetrance. This study describes the diagnosis, clinical characterization, management, and outcomes of a large patient cohort with plexiform neurofibroma (PN) treated with selumetinib in a real-world clinical setting. Methods This single-center observational study consecutively enrolled patients with NF1-PN treated with selumetinib from April 2018 to April 2023. Data on clinical features, tumor types and locations, and results from genetic tests were recorded at baseline; details of disease management with selumetinib and surgical intervention and disease evolution including imaging data and evaluations of pain and function were documented. Results Overall, 54 patients with a median age (range) of 16.4 (4.5–58.0) years were enrolled. Most had cutaneous manifestations (88.9%), including cutaneous neurofibromas and PN. Patients underwent [18F]fluorodeoxyglucose (FDG)-PET/CT imaging before treatment to rule out malignant lesions. Initial evaluations included directed MRI, which facilitated future comparisons and allowed for the assessment of PN resectability. Pharmacological treatment with selumetinib (with surgery, without surgery) resulted in the following proportion of patients achieving stable disease (58.8%, 54.3%), partial response (29.4%, 28.6%), and improved pain (58.8%, 37.1%), deformity (17.6%, 20.0%), and functional (17.6%, 20.0%) outcomes, respectively. Conclusions Results from this study demonstrate that NF1-PN can be managed effectively with selumetinib with surgical intervention in some patients. Most patients achieved tumor stability and improved symptom control, and the majority of patients continue under treatment. Effective diagnosis and management were achieved through individualized utility of FDG-PET/CT and MRI imaging and targeted resource allocation.

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Efficacy and safety profile of selumetinib in symptomatic inoperable plexiform neurofibromas

Cited by 3 publications

References 21 publications

Plexiform neurofibromatosis of face – Recent advances in the management

Plexiform neurofibromatosis of face – Recent advances in the management

Histopathologic features of selumetinib‐induced paronychia in a child with neurofibromatosis type 1

A single-center case study series assessing the effect of selumetinib use in patients with neurofibromatosis-related plexiform neurofibromas

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