Beyond Midostaurin: Role of Avapritinib in Managing Systemic Mastocytosis

We present a case of an adult male who presented with pancytopenia accompanied by symptomatic anemia, necessitating chronic transfusions. He was diagnosed with systemic mastocytosis with an associated hematologic neoplasm. Following an inadequate response to midostaurin therapy, the patient was init...

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Published inCurēus (Palo Alto, CA) Vol. 16; no. 5; p. e60161
Main Authors Pokima, Ngowari, Khattar, Georges, Keesari, Praneeth R, Khan, Salman, Asogwa, Nnedindu, Niazi, Muhammad, Zheng, Ruifang, Dai, Qun
Format Journal Article
LanguageEnglish
Published Palo Alto Cureus Inc 12.05.2024
Cureus
Subjects
Adults
Allergy/Immunology
Anemia
Biopsy
Blood platelets
Bone marrow
Case reports
Edema
Hematology
Hemoglobin
Kinases
Medical prognosis
Mutation
Oncology
Osteoporosis
Patients
Remission (Medicine)
Response rates
Thrombocytopenia
Tumors
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Summary:We present a case of an adult male who presented with pancytopenia accompanied by symptomatic anemia, necessitating chronic transfusions. He was diagnosed with systemic mastocytosis with an associated hematologic neoplasm. Following an inadequate response to midostaurin therapy, the patient was initiated on the newly approved avapritinib. The patient showed significant improvements in all three blood cell lines; however, he developed leg edema, blepharedema, and gum bleeding on this medication. This case underscores the intricacies of managing a patient with advanced systemic mastocytosis, the emerging role of highly selective KIT inhibition in its treatment, and the practical management of adverse medication effects.
Bibliography:ObjectType-Case Study-2
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ISSN:2168-8184
2168-8184
DOI:10.7759/cureus.60161