[Cheryl G]

Stem cell transplants for AL amyloidosis

by Cheryl G on Sat Nov 01, 2014 2:46 pm

While poking around a bit to answer another question here in the forum, I came across a couple of articles and resources that people here in the forum might find useful. I thought I would post them so that we all have them as references.

First, here is a review of stem cell transplantation for AL amyloidosis. It's about three years old, so perhaps slightly out of date. But it's written in language that I found very easy to follow, and there are some interesting statistics in it that I didn't know. For example, it notes that about 50 percent of AL amyloidosis patients who undergo a transplant and reach a complete response live 10 years or longer, suggesting that the transplant may practically curative.

SO Schönland et al., "Current status of hematopoietic cell transplantation in the treatment of systemic amyloid light-chain amyloidosis," Bone Marrow Transplantation, 2012, 47, 895–905 (full text; pdf also available)

Abstract:

Systemic amyloid light-chain (AL) amyloidosis is a protein conformation disorder caused by clonal plasma cell dyscrasias. Symptoms result from fibrillar extracellular deposits in various tissues. The deposits disrupt organ function and ultimately lead to death. The prognosis is poor and depends mostly on the severity of cardiac involvement. The treatment is derived from the therapy of multiple myeloma with the main goal being to reach a complete hematological remission (CR). High-dose melphalan (HDM) and autologous hematopoietic cell transplantation can induce CR rates in about 40%. The main concern was the high transplant-related mortality of up to 40% due to organ dysfunction, which could be reduced to <12% by careful patient selection in experienced centers. However, >50% of patients in CR survive longer than 10 years, suggesting that HDM has the potential to change the natural course of the disease. As there is evidence that ‘graft-versus-plasma-cell-dyscrasia’ effects are active in AL amyloidosis, allogeneic hematopoietic cell transplantation might be an option for younger patients with preserved organ functions who have relapsed after HDM.