一名患有AL淀粉样变性和严重X因子缺乏症的患者接受VAD加SCT治疗后的长期结局
The Long-term Outcomes after VAD plus SCT Therapy in a Patient with AL Amyloidosis and Severe Factor X Deficiency.
作者信息
Iwadate Dosuke, Hasegawa Eiko, Hoshino Junichi, Hayami Noriko, Sumida Keiichi, Yamanouchi Masayuki, Sekine Akinari, Kawada Masahiro, Hiramatsu Rikako, Suwabe Tatsuya, Sawa Naoki, Yuasa Mitsuhiro, Wake Atsushi, Fujii Takeshi, Ohashi Kenichi, Takaichi Kenmei, Ubara Yoshifumi
机构信息
Nephrology Center, Toranomon Hospital, Japan.
Department of Hematology, Toranomon Hospital, Japan.
出版信息
Intern Med. 2018 Mar 1;57(5):701-706. doi: 10.2169/internalmedicine.9263-17. Epub 2017 Nov 1.
A 55-year-old man was admitted to our institute to undergo evaluation for proteinuria (5.4 g/day) with lambda-type Bence-Jones protein (BJP). Primary amyloid light chain (AL) amyloidosis and acquired factor X deficiency were diagnosed. High-dose melphalan combined with autologous stem cell transplantation was performed. After three years, the patient's proteinuria normalized, he was negative for urinary BJP, and his factor X activity improved to 105%. Serial renal biopsy showed no progression of amyloid deposition at a biopsy after 5 years, but showed a slight increase in the amyloid deposition after 11 years. This therapy can improve the prognosis of AL amyloidosis; however, there are limitations to the strategy.
一名55岁男性因蛋白尿（5.4克/天）伴λ型本-周蛋白（BJP）入住我院接受评估。诊断为原发性淀粉样轻链（AL）淀粉样变性和获得性因子X缺乏症。进行了大剂量美法仑联合自体干细胞移植。三年后，患者蛋白尿恢复正常，尿BJP转阴，因子X活性提高到105%。系列肾活检显示，5年后活检时淀粉样沉积无进展，但11年后淀粉样沉积略有增加。这种治疗方法可以改善AL淀粉样变性的预后；然而，该策略存在局限性。
Zotero 插件