Rare Daily Staff

Researchers report that for highly selected patients, treatment including high-dose chemotherapy and autologous stem cell transplantation produces prolonged survival with improved safety in patients with AL amyloidosis, a rare life-threatening disease.

The scientists from the Boston University/Boston Medical Center Amyloidosis Center reported that as part of their work they have also developed a predictive score to assess event free survival following stem cell transplantation. Event-free survival is defined as the time between stem cell transplantation and initiation of next line of therapy or death, whichever occurs first. The researchers published their findings online in the American Journal of Hematology.

AL amyloidosis is a life-threatening disease related to the bone marrow cancer multiple myeloma. It is caused when a person’s antibody-producing white blood cells do not function properly and generate abnormal protein that misfolds to form amyloid fibrils and deposits made of components of antibodies called light chains, which then deposit in various organs of the body.

The researchers reviewed collected data from a prospectively maintained database of patients with AL amyloidosis consecutively treated with HDM/SCT between July 1994 and September 2021.They found that hematologic complete remission (CR) was achieved in 39 percent of patients with a median overall survival of 15 years with 30 percent of these patients surviving greater than 20 years.

“This treatment, first developed here at BU/BMC in the 1990s, leads to long term survival especially for those who achieve a complete hematologic response after SCT,” says corresponding author Vaishali Sanchorawala, director of the Boston University/Boston Medical Center Amyloidosis Center. “The risk of future hematologic relapse was also low (five percent) amongst patients with a sustained hematologic CR for 15 years, suggesting that the majority of patients who reach this milestone may be operationally cured by HDM/SCT.”

The researcher also developed a prognostic score for event free survival that incorporated measuring the patients’ light chain levels and bone marrow plasma cell percentage. Patients with low-risk (0 factors) had a median event-free survival estimate of 5.3 years, compared to 2.8 years for patients with intermediate-risk (1 factor), and 1 year for patients with high-risk (2 factors) disease.

Photo: Vaishali Sanchorawala, director of the Boston University/Boston Medical Center Amyloidosis Center

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