Magnano Laura, Rivero Andrea, Matutes Estella
Department of Hematology, Hospital Clínic, Barcelona, Spain.
Institut d'Investigacions Biomèdiques August Pi i Sunyer (IDIBAPS), Barcelona, Spain.
Curr Oncol Rep. 2022 May;24(5):633-644. doi: 10.1007/s11912-021-01159-y. Epub 2022 Feb 25.
This manuscript aims at updating the knowledge on the clinico-biological characteristics, pathogenesis, and the diagnostic challenges of T-LGLL and CLPD-NK disorders and reviews the advances in the management and treatment of these patients.
It has been shown that clonal large granular lymphocyte (LGL) expansions arise from chronic antigenic stimulation, leading to resistance to apoptosis. All the above findings have facilitated the diagnosis of LGLL and provided insights in the pathogenesis of the disease. At present, there is no standard first-line therapy for the disease. Immunosuppressive agents are the treatment routinely used in clinical practice. However, these agents have a limited capacity to eradicate the LGL clone and induce long-lasting remission. Advances in the knowledge of pathogenesis have made it possible to explore new therapeutic targets with promising results. Since LGLL is a rare disease, international efforts are needed to carry on prospective clinical trials with new potentially active drugs that could include a large number of patients.
本手稿旨在更新有关T-LGLL和CLPD-NK疾病的临床生物学特征、发病机制及诊断挑战的知识，并回顾这些患者管理和治疗方面的进展。
已表明克隆性大颗粒淋巴细胞（LGL）扩增源于慢性抗原刺激，导致对细胞凋亡产生抗性。上述所有发现都有助于LGLL的诊断，并为该疾病的发病机制提供了见解。目前，该疾病尚无标准的一线治疗方法。免疫抑制剂是临床实践中常规使用的治疗药物。然而，这些药物根除LGL克隆并诱导长期缓解的能力有限。发病机制知识的进展使得探索新的治疗靶点成为可能，并取得了有前景的结果。由于LGLL是一种罕见疾病，需要国际间共同努力开展前瞻性临床试验，使用可能包括大量患者的新的潜在活性药物。
