What is the role of autologous stem cell transplantation in the treatment of light-chain deposition disease (LCDD)?

Updated: Sep 26, 2019
  • Author: Swapna Boppana, MD; Chief Editor: Emmanuel C Besa, MD  more...
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Answer

Stem cell transplantation can produce durable responses in patients with LCDD. [14, 41, 42] Stem cells are mobilized using granulocyte colony-stimulating factor (G-CSF), and high-dose chemotherapy with melphalan is given. The dose of melphalan is adjusted to the renal function to decrease morbidity.

A long-term analysis of 6 patients with LCDD who underwent ASCT demonstrated that this is an effective therapy for patients with renal dysfunction due to LCDD. [14] Proteinuria was reduced by 92%, and the glomerular filtration rate improved by 95% in these patients. The authors also suggest if kidney dysfunction persists after ASCT, a hematological response may permit successful kidney transplantation with improved graft viability and decreased risk of recurrence. Another study of patients with LCDD treated with high-dose melphalan followed by ASCT also demonstrated that patients with renal dysfunction have improvement in renal function following ASCT. [43] Of the 5 evaluable patients with hematological response, one had complete response and four with partial response.

The use of high dose chemotherapy followed by ASCT is associated with toxicities such as mucositis, sepsis, bacteremia, and diarrhea. In general, LCDD patients are younger; thus, ASCT should be considered in these patients. However, associated comorbidities, presence of cardiac involvement, concomitant presence of multiple myeloma, and number of organs affected may predict for a worse outcome. Therefore, age and comorbidities should be considered prior to ASCT. Multiorgan failure following ASCT has been reported in patients with extrarenal disease. [14]


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