The Long-term Outcomes after VAD plus SCT Therapy in a Patient with AL Amyloidosis and Severe Factor X Deficiency

Intern Med. 2018 Mar 1;57(5):701-706. doi: 10.2169/internalmedicine.9263-17. Epub 2017 Nov 1.

Abstract

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.

Keywords: AL amyloidosis; autologous stem cell transplantation (SCT); factor X deficiency; high-dose melphalan; vincristine, adriamycin, and dexamethasone (VAD).

Publication types

  • Case Reports

MeSH terms

  • Antineoplastic Agents, Alkylating / administration & dosage
  • Antineoplastic Combined Chemotherapy Protocols / therapeutic use*
  • Bence Jones Protein
  • Biopsy
  • Combined Modality Therapy
  • Doxorubicin / therapeutic use
  • Etoposide / therapeutic use
  • Factor X Deficiency / complications
  • Factor X Deficiency / diagnosis
  • Factor X Deficiency / therapy*
  • Hematopoietic Stem Cell Transplantation*
  • Humans
  • Immunoglobulin Light-chain Amyloidosis / complications
  • Immunoglobulin Light-chain Amyloidosis / diagnosis
  • Immunoglobulin Light-chain Amyloidosis / therapy*
  • Male
  • Melphalan / administration & dosage
  • Middle Aged
  • Remission Induction
  • Transplantation, Autologous

Substances

  • Antineoplastic Agents, Alkylating
  • Etoposide
  • Doxorubicin
  • Bence Jones Protein
  • Melphalan

Supplementary concepts

  • VAD combination