Current treatment of AL amyloidosis.
نویسندگان
چکیده
Immunoglobulin light chain systemic amyloidosis (AL) is a progressive disease caused by monoclonal light chains with specific mutations that confer a unique propensity to misfold from their native structure to less stable, partially folded intermediates that selfaggregate into oligomers and then into the highlyordered cross β-sheet structure which defines amyloid fibrils. At least 11 additional proteins, synthesized by different organs (liver, intestine, etc) can cause systemic amyloidoses which can be difficult to distinguish from AL amyloidosis on a clinical basis. These proteins form amyloid deposits that share the common tinctorial, green birefringence under polarized light after staining with Congo red, and ultrastructural features, rigid, nonbranching fibril with a distinct diameter of 7.5 to 10 nm (Figure 1). The unequivocal identification of the protein forming the amyloid fibril is essential for the choice of therapy. A mistake in protein typing may have catastrophic therapeutic consequences, such as performing an autologous stem cell transplant in a patient with transthyretin amyloidosis who should receive a liver transplant. Proteomics technology has significantly improved the typing of amyloid deposits and is routinely applied on abdominal fat aspirates at our Center. The amyloidogenic light chains are produced by a bone marrow plasma cell clone usually of limited size, enter the circulation, and target selected organs: heart, kidney, liver, soft tissues and peripheral nervous system, trough specific, but largely undetermined, interactions with local matrix components, such as glycosaminogly-
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عنوان ژورنال:
- Haematologica
دوره 94 8 شماره
صفحات -
تاریخ انتشار 2009