Department of Hematology, Pitié-Salpétrière Hospital APHP, Sorbonne Université, Boulevard de l'hôpital, 75013, Paris, France.
Department of Hematology, Centre-Hospitalier Sud-Francilien, Corbeil-Essonnes, France.
Curr Oncol Rep. 2019 Feb 26;21(3):27. doi: 10.1007/s11912-019-0768-4.
Waldenström macroglobulinemia (WM) is a rare lymphoproliferative disorder. Up to now, therapeutic choice was not influenced by the biological characteristics of the disease. Here, we will review how recent advances in biology in WM may affect therapy strategy.
Recently, WM has been described as a new oncogenic model. MyD88 mutation has been described as a key driver mutation and has functional consequences which could be targeted. Other mutations, such as CXCR4 or TP53, have been reported. These mutations are associated with different clinical presentation, prognosis, and treatment response. Mutational status may influence therapeutic choice in some patients but additional data are required. New targeted therapies are on development.
华氏巨球蛋白血症(WM)是一种罕见的淋巴增殖性疾病。到目前为止,治疗选择并未受到疾病生物学特征的影响。在这里,我们将回顾 WM 中生物学的最新进展如何影响治疗策略。
最近,WM 已被描述为一种新的致癌模型。MyD88 突变已被描述为关键驱动突变,并具有可靶向的功能后果。其他突变,如 CXCR4 或 TP53,也有报道。这些突变与不同的临床表现、预后和治疗反应相关。突变状态可能会影响某些患者的治疗选择,但需要更多的数据。新的靶向治疗正在开发中。