Scherber Robyn M, Borate Uma
Department of Hematology and Oncology, Oregon Health and Science University, Portland, OR, USA.
Department of Hematology and Oncology, Mayo Clinic, Scottsdale, AZ, USA.
Br J Haematol. 2018 Jan;180(1):11-23. doi: 10.1111/bjh.14967. Epub 2017 Oct 19.
Rapid advances in the understanding of the molecular biology, data from translational and clinical trials, and retrospective analyses has influenced the diagnosis and treatment of systemic mastocytosis (SM). Many options have existed for the symptomatic management of SM patients, but recent evolution in regards to the molecular underpinnings of this disease and our ability to distinguish clonal mastocytosis from mast cell activation syndrome has changed our treatment paradigm and opened new opportunities for understanding genetic risk, transformation to mast cell leukaemia, and treatment choices. Key to this change has been the discovery of the KIT mutation and the use of next generation sequencing to evaluate for co-existing molecular mutations that may define the disease course. Careful diagnosis, judicious symptom management and close surveillance of those who may have yet undiagnosed disease is paramount in providing optimal management. In this article, we review the diagnosis and provide a paradigm for the management of SM patients.
对分子生物学理解的迅速进展、转化和临床试验数据以及回顾性分析影响了系统性肥大细胞增多症(SM)的诊断和治疗。对于SM患者的症状管理存在多种选择,但最近关于该疾病分子基础的进展以及我们区分克隆性肥大细胞增多症与肥大细胞活化综合征的能力改变了我们的治疗模式,并为理解遗传风险、转化为肥大细胞白血病及治疗选择带来了新机遇。这一变化的关键在于KIT突变的发现以及使用新一代测序来评估可能决定疾病进程的共存分子突变。在提供最佳管理中,仔细诊断、明智的症状管理以及对可能尚未确诊疾病的患者进行密切监测至关重要。在本文中,我们回顾了SM患者的诊断并提供了一种管理模式。