Treatment of patients with advanced neurofibromatosis type 2 (NF2) with novel molecularly targeted therapies.

Subbiah, Vivek; Slopis, John M.; Hong, David S.; Angelo, Laura S.; McCutcheon, Ian E.; Kurzrock, Razelle

doi:10.1200/jco.2011.29.15_suppl.2100

Cited by 1 publication

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“…This study found that after 1 year of treatment with selumetinib, 70% of patients achieved a partial response and 56% had a durable response. 8 In terms of safety, the most common side effects were nausea, vomiting, diarrhea, asymptomatic rise in creatinine phosphokinase, acneiform rash, and paronychia, with only 10% of patients discontinuing treatment due to toxicities. 8 …”

Section: Discussionmentioning

confidence: 99%

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Images in Immunotherapy and Precision Oncology: A Case Report of Neurofibromatosis-1

Deshpande,

Munoz,

Dabak

et al. 2024

Journal of Immunotherapy and Precision Oncology

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Neurofibromatosis type 1 (NF1) is an autosomal dominant genetic disorder that primarily causes the growth of tumors along nerves. Additionally, the germline mutations involved in NF1 predispose patients to develop further malignancies. The mainstay initial treatment for these malignancies is surgical removal at diagnosis, although targeted therapies are under evaluation in the relapsed setting. We report a case of malignant peripheral nerve sheath tumor (MPNST), gastrointestinal stromal tumor (GIST), and pheochromocytoma in a patient with NF1 who presented with an infected right shoulder lesion that was confirmed to be spindle cell sarcoma via biopsy. She was treated with antibiotics; however, she rapidly deteriorated and opted for hospice care. NF1 germline mutations increase the risk of patients developing various types of cancer. Recent studies have shown that there is a role for using MEK inhibitors such as selumetinib for treating patients with NF1.

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Section: Discussionmentioning

confidence: 99%

“… 8 In terms of safety, the most common side effects were nausea, vomiting, diarrhea, asymptomatic rise in creatinine phosphokinase, acneiform rash, and paronychia, with only 10% of patients discontinuing treatment due to toxicities. 8 …”

Section: Discussionmentioning

confidence: 99%