Department of Hematology, Peking Union Medical College Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, No.1 Shuaifuyuan, Dong Cheng District, Beijing 100730, China; Medical College, Chinese Academy of Medical Sciences, Beijing, China.
Department of Pathology, Peking Union Medical College Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Beijing, China.
Neoplasia. 2024 Nov;57:101048. doi: 10.1016/j.neo.2024.101048. Epub 2024 Sep 13.
Indolent natural killer cell lymphoproliferative disorder of the gastrointestinal tract (iNKLPD-GI) is an uncommon, recently recognized lymphoid proliferation of mature NK cells primarily manifesting in the GI tract. Unlike NK/T lymphoma, iNKLPD-GI exhibits a rather indolent clinical course, underscoring the need for cautious management to prevent unnecessary interventions. However, clinical and molecular features of this entity have not been thoroughly understood. This study aimed to add more information to the current knowledge of this disease. Seven patients with iNKLPD-GI were included in our study. Clinical data included initial symptoms, endoscopic manifestations, pathological features, and therapies. Besides, next-generation sequencing was arranged to explore the underlying genetic mechanism of this disease. In our study, iNKLPD-GI in the urinary bladder was first identified. Edema of extremities (3, 42.8 %) was the most prevalent onset symptom which was reported for the first time. Pathological and immunohistological features were found to display the phenotype of NK cells. Unlike extranodal NK/T cell lymphoma, Epstein-Barr virus-encoded small RNA (EBER) were negative in all patients. Moreover, we found that two patients harbored JAK3 mutation. Apart from JAK3 K563_C565del previously reported in the literature, we discovered new JAK3 mutation sites. Other mutations including BRAF, KRAS, and SH2B3 were also identified. In conclusion, iNKLPD-GI was an indolent atypical NK-cell proliferation with diverse clinical characteristics. "Watch and wait" therapy was preferable to intense chemotherapy. Recurrent JAK3 mutation may be the underlying mechanism responsible for the neoplastic nature of the disease and may serve as a potential target for patients with severe symptoms.
惰性自然杀伤细胞(NK)细胞胃肠型淋巴组织增生性疾病(iNKLPD-GI)是一种少见的、新近认识的成熟 NK 细胞增生性疾病,主要表现为胃肠道受累。与 NK/T 细胞淋巴瘤不同,iNKLPD-GI 具有相对惰性的临床病程,这突出强调了谨慎管理的必要性,以避免不必要的干预。然而,该疾病的临床和分子特征尚未被充分了解。本研究旨在为该疾病的现有知识增加更多信息。我们的研究纳入了 7 例 iNKLPD-GI 患者。临床资料包括首发症状、内镜表现、病理学特征和治疗方案。此外,还进行了下一代测序,以探讨该疾病的潜在遗传机制。在本研究中,我们首次发现了膀胱 iNKLPD-GI。四肢水肿(3 例,42.8%)是最常见的首发症状。病理学和免疫组织化学特征显示 NK 细胞表型。与结外 NK/T 细胞淋巴瘤不同,所有患者的 EBV 编码的小 RNA(EBER)均为阴性。此外,我们发现 2 例患者存在 JAK3 突变。除了文献中报道的 JAK3 K563_C565del 突变外,我们还发现了新的 JAK3 突变位点。其他突变包括 BRAF、KRAS 和 SH2B3。总之,iNKLPD-GI 是一种惰性的非典型 NK 细胞增生性疾病,具有多种临床特征。“观察与等待”疗法优于强化化疗。复发性 JAK3 突变可能是该疾病发生肿瘤的潜在机制,并可能成为症状严重患者的潜在治疗靶点。