High-dose melphalan and autologous stem cell transplantation for systemic light-chain amyloidosis: a single institution retrospective analysis of 40 cases

We report our retrospective analysis of 40 patients who received high-dose melphalan and autologous stem cell transplantation for systemic immunoglobulin light-chain (AL) amyloidosis. Between 2006 and 2013, 40 patients with AL amyloidosis were transplanted at our medical center. Their median age was 54 years (range 32–70 years): 18 were male. The dominant organs involved were the heart in 13 patients, and kidney in 22: and other organs were involved in five. The median melphalan dose administered was 129 (range 50–200) mg/m², and the median infused CD34⁺ cells was 2.69 (range 1.17–11.26) × 10⁶/kg. Of the 40 patients, 30 are alive after a median follow-up of 42 (range 12–94) months, and the 4-year estimated overall survival rate was 74 % (95 % CI 56–86 %). Four patients died ≤100 days post-ASCT (heart failure in three patients, bacteremia in one). The 4-year estimated survival of the patients with cardiac involvement was 54 %, significantly lower than that of the other patients (91 %). Hematological and organ responses were 52 and 50 %, respectively. Careful patient selection and experienced management are important, especially for patients with cardiac involvement. It is also important to develop additional treatment for patients who do not achieve a hematological and/or organ response.