Midostaurin
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Various toxicities following an off label use: case report A female infant [exact age at the time of reaction onset not stated] developed nausea, diarrhoea, thrombocytopenia and vomiting following an off label use of midostaurin for systemic mastocytosis. The girl was initially diagnosed with cutaneous mastocytosis, and received treatment with unspecified antihistamines, sodium cromoglicate [cromolyn] and montelukast. At age 6 months, she developed numerous painful, haemorrhagic bullae on light touch or skin trauma. Consequently, she was admitted for detailed investigations. Following investigation, she was diagnosed with indolent systemic mastocytosis. The medications were continued, but after one month, she presented again due to worsening of the bullae, uncontrollable pruritus, irritability and episodes of flushing. She was treated with unspecified antibiotics and methylprednisolone without improvement. As a result, an approval for compassionate use of midostaurin was obtained, and she received liquid formulation of midostaurin 30 mg/m2 twice daily [route not stated]. Ondansetron was administered before each dose prophylactically with modest response. Over the subsequent 2 months, the dose of midostaurin was increased to 60 mg/m2 twice daily due to persistence of her symptoms. Following 12 weeks, significant clinical improvement was noted. The bullae resolved completely and serum tryptase levels were nearly normal. She tolerated midostaurin well. However, she developed side effects such as diarrhoea, nausea, thrombocytopenia and vomiting [duration of treatment to reactions onsets and outcomes not stated]. Author comment: "[C]ompassionate use approval by the manufacturer, we obtained a liquid formulation of midostaurin from the manufacturer." "[T]he patient is tolerating midostaurin well. Common side effects of midostaurin are nausea, vomiting, diarrhea, and thrombocytopenia." Liu MM, et al. Treatment of systemic mastocytosis in an infant with midostaurin. Journal of Allergy and Clinical Immunology: In Practice 7: 2929-2931e1, No. 8, Nov-Dec 2019. Available from: URL: http://doi.org/10.1016/j.jaip.2019.05.032 803438094 USA
0114-9954/19/1782-0001/$14.95 Adis © 2019 Springer Nature Switzerland AG. All rights reserved
Reactions 7 Dec 2019 No. 1782
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