Article ; Online: Selumetinib side effects in children treated for plexiform neurofibromas: first case reports of peripheral edema and hair color change.
2021 Volume 21, Issue 1, Page(s) 67
Abstract: Background: Plexiform neurofibromas (PNs) are congenital tumors that affect around 50 % of the subjects with neurofibromatosis type 1. Despite being histologically benign, PNs can grow rapidly, especially in the pediatric age, and cause severe ... ...
| Abstract | Background: Plexiform neurofibromas (PNs) are congenital tumors that affect around 50 % of the subjects with neurofibromatosis type 1. Despite being histologically benign, PNs can grow rapidly, especially in the pediatric age, and cause severe morbidities. In the past, various therapeutic approaches have been proposed to treat these masses, none of which obtained valuable results. Selumetinib, an inhibitor of mitogen-activated protein kinase (MEK) 1 and 2, has been the first molecule to demonstrate the ability of tackling the growth of PNs. The drug's most common side effects, which usually are mild or moderate, include gastrointestinal symptoms (diarrhea, abdominal pain), dermatologic manifestations (maculo-papular and acneiform rash, paronychia, mucositis), and various laboratory test abnormalities (elevation of creatine kinase and aminotransferase). Cases presentation: We report two previously undescribed adverse events in pediatric patients: peripheral edema and hair color change. The first case of peripheral edema occurred in a 7-year-old boy affected by a severe form of NF1, after two years of treatment with selumetinib at the standard dose (25 mg/m Conclusions: Since the use of selumetinib to treat plexiform neurofibromas is increasing in the pediatric population, clinicians should be aware of its side effects, so to decide whether continuing the treatment, reducing the dose or even interrupting it, when appropriate. |
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| MeSH term(s) | Benzimidazoles ; Child ; Child, Preschool ; Edema/chemically induced ; Female ; Hair Color ; Humans ; Male ; Neurofibroma, Plexiform/chemically induced ; Neurofibroma, Plexiform/diagnostic imaging ; Neurofibroma, Plexiform/drug therapy |
| Chemical Substances | AZD 6244 ; Benzimidazoles |
| Language | English |
| Publishing date | 2021-02-06 |
| Publishing country | England |
| Document type | Case Reports ; Journal Article |
| ZDB-ID | 2041342-7 |
| ISSN | 1471-2431 ; 1471-2431 |
| ISSN (online) | 1471-2431 |
| ISSN | 1471-2431 |
| DOI | 10.1186/s12887-021-02530-5 |
| Database | MEDical Literature Analysis and Retrieval System OnLINE |
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