Fenoglio Roberta, Baldovino Simone, Ferro Michela, Sciascia Savino, Rabajoli Gianluca, Quattrocchio Giacomo, Beltrame Giulietta, Naretto Carla, Rossi Daniela, Alpa Mirella, Barreca Antonella, Papotti Mario Giulio, Roccatello Dario
Nephrology and Dialysis Unit and CMID, University of Turin and San Giovanni Hospital, Piazza del Donatore di Sangue 3, 10054, Turin, Italy.
Department of Clinical and Biological Sciences, Center of Research of Immunopathology and Rare Diseases, Coordinating Center of the Network for Rare Diseases of Piedmont and Aosta Valley, University of Turin, Turin, Italy.
J Nephrol. 2021 Feb;34(1):231-240. doi: 10.1007/s40620-020-00748-7. Epub 2020 May 29.
AL amyloidosis is caused by a clone of plasma cell. Due to the impact of the disease on patient survival, careful evaluation of organ involvement is essential and treatment should be tailored to single patient's risk.
We analyzed the clinical, laboratory and histological characteristics of 21 elderly patients (pts) (mean age 74.7 ± 7.97 years, range 55-81) with AL amyloidosis, including 17 patients (81%) with biopsy-proven renal involvement, who were ineligible for bone marrow transplantation, and evaluated the impact of renal impairment on survival.
Cardiac and renal involvement was found in 14 (67%) cases. Among the 17 patients with renal involvement, 12 had renal failure with proteinuria, and one showed isolated renal failure and vascular amyloid deposition. Hematological response occurred in 57.1% after first line therapy (75% after three cycles). In six of the patients with renal involvement, proteinuria decreased from 4.2 to 1.1 g/24 h (range 0.2-3 g/24 h), serum Creatinina (sCr) levels declined or stabilized. Severe renal failure at diagnosis was found to directly influence patient survival, while the Staging System for Renal Outcome in AL Amyloidosis did not associate with outcomes.
To the best of our knowledge this is the first case series in which the whole cohort of patients with urinary or functional abnormalities underwent a histological evaluation. None of the patients were eligible for bone marrow transplantation. Hematologic response was 57.1%, while renal response was much lower (35%). Of note, the Staging System did not completely apply to this peculiar setting of patients in whom renal involvement was not presumptive but biopsy-proven. More aggressive approaches may be needed in these patients to avoid the inexorable progression of the disease.
AL淀粉样变性由浆细胞克隆引起。由于该疾病对患者生存的影响，仔细评估器官受累情况至关重要，且治疗应根据个体患者的风险进行调整。
我们分析了21例老年AL淀粉样变性患者（平均年龄74.7±7.97岁，范围55 - 81岁）的临床、实验室和组织学特征，其中17例（81%）经活检证实有肾脏受累，这些患者不符合骨髓移植条件，并评估了肾功能损害对生存的影响。
14例（67%）患者出现心脏和肾脏受累。在17例肾脏受累患者中，12例有肾衰竭伴蛋白尿，1例表现为孤立性肾衰竭和血管淀粉样沉积。一线治疗后血液学缓解率为57.1%（三个周期后为75%）。在6例肾脏受累患者中，蛋白尿从4.2降至1.1 g/24小时（范围0.2 - 3 g/24小时），血清肌酐（sCr）水平下降或稳定。诊断时的严重肾衰竭直接影响患者生存，而AL淀粉样变性肾脏结局分期系统与预后无关。
据我们所知，这是首个对所有有泌尿或功能异常的患者队列进行组织学评估的病例系列。所有患者均不符合骨髓移植条件。血液学缓解率为57.1%，而肾脏缓解率则低得多（35%）。值得注意的是，分期系统并不完全适用于这种特殊的患者群体，在这些患者中肾脏受累并非推测性而是经活检证实的。可能需要更积极的治疗方法来避免疾病的不可阻挡的进展。
