High-dose melphalan followed by auto-SCT has favorable safety and efficacy in selected patients with light chain deposition disease and light and heavy chain deposition disease

摘要

Light chain and light and heavy chain deposition disease (LCDD and LHCDD) are plasma cell dyscrasias in which aggregation and deposition of Ig components leads to organ dysfunction. Although similar in principle to light chain (AL) amyloidosis, there are a number of distinguishing clinical and pathological features. Untreated, the natural history is usually progression to end-stage renal disease with death resulting either from light-chain-mediated organ impairment or complications of concomitant multiple myeloma (MM). Alkylator-based chemotherapy is generally ineffective in controlling disease progression, and high dose therapy with autologous stem cell transplant (ASCT) has been advocated as a means to effect a rapid reduction in pathological light chain production. Data supporting the safety and efficacy of this approach remain limited given the rarity of these conditions.