After this therapy, two patients with primary amyloid polyneuropathy displayed improvements in neuropathy and overall health.
Although primary amyloidosis is a rare cause of polyneuropathy, about 17% of patients with this condition develop neuropathy that is progressive and refractory to treatment. These authors describe the diagnosis, treatment with high-dose melphalan plus autologous stem cell transplantation (HDM/SCT), and follow-up of two patients with well-characterized primary systemic amyloidosis and polyneuropathy.
Both patients had extensive evaluations to characterize their underlying disease, including determination of the underlying amyloid protein, bone marrow biopsy revealing clonal expansion of CD38++CD19– cells, and demonstration of amyloid deposits in other organs. One patient had the characteristic autonomic involvement associated with this condit…