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<p>Before 2000, AL amyloidosis was considered an incurable disease. However, in the 2000s, autologous peripheral blood stem cell transplantation therapy using vincristine + doxorubicin + dexamethasone with high-dose melphalan (VAD + SCT) was initiated. Bone marrow cells are the site of AL amyloid production. By normalizing bone marrow cells, VAD + SCT not only reduces amyloid production but also improves renal tissue damaged by amyloid deposition. However, VAD + SCT was only applied to patients who met the criteria, such as being under 65&#xa0;years of age and not affecting the heart. At our hospital, VAD + SCT was applied in 20 patients who met this criteria. After treatment, 5 died and 2 required hemodialysis, but the remaining 13 patients survived for more than 10&#xa0;years, with resolution of proteinuria and maintenance of renal function. These outcomes indicate that suppression of amyloid production is an effective treatment for AL amyloidosis. In the 2020s, molecularly targeted drugs against the plasma cell CD38 began to be administered for the treatment of AL amyloidosis.</p>

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Trend of treatment strategy for amyloid light-chain amyloidosis: a-single center experience

  • 期刊论文

  • 发表日期：

    2025年5月15日

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  • Yoshifumi Ubara,
  • Kei Kono,
  • Junichi Hoshino,
  • Takehiko Wada,
  • Atsushi Wake,
  • Kenichi Ohashi,
  • Naoki Sawa

摘要

Before 2000, AL amyloidosis was considered an incurable disease. However, in the 2000s, autologous peripheral blood stem cell transplantation therapy using vincristine + doxorubicin + dexamethasone with high-dose melphalan (VAD + SCT) was initiated. Bone marrow cells are the site of AL amyloid production. By normalizing bone marrow cells, VAD + SCT not only reduces amyloid production but also improves renal tissue damaged by amyloid deposition. However, VAD + SCT was only applied to patients who met the criteria, such as being under 65 years of age and not affecting the heart. At our hospital, VAD + SCT was applied in 20 patients who met this criteria. After treatment, 5 died and 2 required hemodialysis, but the remaining 13 patients survived for more than 10 years, with resolution of proteinuria and maintenance of renal function. These outcomes indicate that suppression of amyloid production is an effective treatment for AL amyloidosis. In the 2020s, molecularly targeted drugs against the plasma cell CD38 began to be administered for the treatment of AL amyloidosis.