Stem Cell Therapy Good for Amyloidosis

U.S. scientists have found high-dose chemotherapy and blood stem cell transplantation can result in long-term survival for light chain amyloidosis patients.

Researchers from at Boston University Medical Center said they first began using the treatment in 1994 and have become the first to publish such long-term outcome results.

Light chain amyloidosis occurs when plasma cells in bone marrow produce proteins that misfold and deposit in tissues, leading to organ failure and death. Between 1,200 and 3,200 new cases are reported annually in the United States, although researchers believe the disease is highly under-diagnosed.

The researchers reviewed the records of 80 patients who received high-dose chemotherapy and blood stem cell transplantation from July 1994 through July 1997. The median survival for all 80 patients was 57 months. Eighteen of these patients are still alive 10 or more years after undergoing the treatment. By comparison, only two percent of patients treated with oral melphalan and prednisone have survived more than 10 years.

The findings appear pre-published in the online version of the journal Blood.