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- W3113131897 abstract "Abstract BACKGROUND Neurofibromatosis type 1 (NF1) is autosomal dominant neurogenetic disorder characterized by progressive cutaneous, neurologic, skeletal, and neoplastic manifestations. Plexiform neurofibromas (PN) are one of the different types of neurofibromas that occur in these patients. Complete surgical resection is difficult due to the tumor infiltrative behavior. We evaluated pegylated interferon- α-2b (PI) in patients with unresectable progressive or symptomatic PN. METHODS Pediatric patients (1–21 years old) affected by unresectable PN, followed at Bambino Gesù Hospital, were treated with PI. We administered PI as a weekly subcutaneous injection at a beginning dose of 1.0 mcg/kg/wk, increased to 3.0 mcg/kg/wk if well tolerated. Paracetamol (15mg/kg) was given 30 minutes prior the dose of PI and then every 4–6 hours as needed. Patients were evaluated with Magnetic Resonance Imaging (MRI) every 12 months after treatment start in case of stable disease. RESULTS 10 patients (3 females, 7 males) were enrolled. Median age was 12 years old. The median duration of treatment was 12,6 months. Grade 3 neutropenia (30%) and increased liver transaminases level (20%) were the most common toxicity. 6/10 patients experienced an improvement about pain. 7/10 patients showed clinical response. 1/10 patient had a radiological response at MRI, 1/10 experienced progression disease and 8/10 showed a stable disease at MRI evaluation. CONCLUSIONS Our study demonstrated that PI could be a suitable treatment for unresectable PN in terms of stabilization of the tumour size due to its antitumor activity although clinical response does not correlate with radiographic changes." @default.
- W3113131897 created "2020-12-21" @default.
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- W3113131897 date "2020-12-01" @default.
- W3113131897 modified "2023-09-26" @default.
- W3113131897 title "NFB-07. USE OF PEGYLATED INTERFERON α- 2b IN PEDIATRIC PATIENTS AFFECTED BY UNRESECTABLE PLEXIFORM NEUROFIBROMAS: MONOCENTRIC EXPERIENCE" @default.
- W3113131897 doi "https://doi.org/10.1093/neuonc/noaa222.611" @default.
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