Pokima Ngowari, Khattar Georges, Keesari Praneeth R, Khan Salman, Asogwa Nnedindu, Niazi Muhammad, Zheng Ruifang, Dai Qun
Internal Medicine, Staten Island University Hospital, Staten Island, USA.
Hematology and Medical Oncology, Northwell Health/Staten Island University Hospital, Staten Island, USA.
Cureus. 2024 May 12;16(5):e60161. doi: 10.7759/cureus.60161. eCollection 2024 May.
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.
我们报告一例成年男性病例,该患者表现为全血细胞减少并伴有症状性贫血,需要长期输血。他被诊断为系统性肥大细胞增多症并伴有相关血液肿瘤。在对米哚妥林治疗反应不佳后,该患者开始使用新批准的阿伐替尼。患者的所有三种血细胞系均有显著改善;然而,他在使用这种药物时出现了腿部水肿、眼睑水肿和牙龈出血。该病例强调了管理晚期系统性肥大细胞增多症患者的复杂性、高选择性KIT抑制在其治疗中的新兴作用以及药物不良反应的实际管理。