Basset Marco, Hummedah Kamal, Kimmich Christoph, Veelken Kaya, Dittrich Tobias, Brandelik Simone, Kreuter Michael, Hassel Jessica, Bosch Nikolaus, Stuhlmann-Laeisz Christiane, Blank Norbert, Müller-Tidow Carsten, Röcken Christoph, Hegenbart Ute, Schönland Stefan
Medical Department V, Hematology, Oncology and Rheumatology, University Hospital Heidelberg, Heidelberg, Germany.
Amyloidosis Center Heidelberg, University Hospital Heidelberg, Heidelberg, Germany.
Am J Hematol. 2020 Oct;95(10):1158-1169. doi: 10.1002/ajh.25915. Epub 2020 Aug 12.
In localized light chain amyloidosis (locAL), amyloidogenic light chains (aLC) are produced and deposited locally by a B-cell clone. We present 293 patients with immunohistochemically confirmed locAL. Lung (nodular pulmonary) with 63 patients was the most involved organ. The aLC was λ in 217 cases (κ:λ ratio 1:3). A local B-cell clone was identified in 30% of cases. Sixty-one (21%) had a concomitant autoimmune disorder (cAD). A monoclonal component (MC) were present in 101 (34%) patients and were more frequent in subjects with cAD (51% vs 34%; P = .03). Cigarette smoking was more prevalent in lung locAL (54% vs 37%; P = .018). After a median follow-up of 44 months, 16 patients died and 5- and 10-years locAL progression-free survival (PFS) were 62% and 44%. Interestingly, locAL-PFS was shorter among patients with an identified clonal infiltrate at amyloid deposition site (40 vs 109 months; P = .02) and multinuclear giant cells and/or an inflammatory infiltrate resulted in longer locAL-PFS in lung involvement (65 vs 42 months; P = .01). However, no differences in locAL PFS were observed in patients with cAD, a MC and involved organ site. Treatment was administered in 163 (54%) patients and was surgical in 135 (46%). Median locAL-PFS after first treatment was 56 months. Responders had longer locAL-PFS (78 vs 17 months; P < .001). Three patients with lung locAL and a MC were diagnosed as systemic AL amyloidosis at follow-up. In summary, locAL pathogenesis seems to be heterogeneous and the clonal infiltrate leads local progression.
在局限性轻链淀粉样变性（locAL）中，淀粉样变性轻链（aLC）由B细胞克隆产生并局部沉积。我们报告了293例经免疫组织化学确诊的locAL患者。肺部（结节性肺淀粉样变性）受累患者最多，有63例。217例患者的aLC为λ型（κ:λ比例为1:3）。30%的病例中发现了局部B细胞克隆。61例（21%）患者伴有自身免疫性疾病（cAD）。101例（34%）患者存在单克隆成分（MC），在伴有cAD的患者中更常见（51%对34%；P = 0.03）。吸烟在肺部locAL中更为普遍（54%对37%；P = 0.018）。中位随访44个月后，16例患者死亡，5年和10年locAL无进展生存期（PFS）分别为62%和44%。有趣的是，在淀粉样蛋白沉积部位发现克隆浸润的患者中，locAL-PFS较短（40个月对109个月；P = 0.02），而多核巨细胞和/或炎性浸润导致肺部受累患者的locAL-PFS更长（65个月对42个月；P = 0.01）。然而，在伴有cAD、MC和受累器官部位的患者中，未观察到locAL PFS的差异。163例（54%）患者接受了治疗，其中135例（46%）接受了手术治疗。首次治疗后的中位locAL-PFS为56个月。有反应者的locAL-PFS更长（78个月对17个月；P < 0.001）。3例肺部locAL且伴有MC的患者在随访中被诊断为系统性AL淀粉样变性。总之，locAL的发病机制似乎是异质性的，克隆浸润导致局部进展。
