News in AL Amyloidosis ASH 2016: A rapidly evolving field of investigation

Abstract

Amyloidosis is a rare but life-threatening protein misfolding disease. The early diagnosis and enrollment of patients into multicentre trials is of great importance, as is the need for intensive collaboration between multiple medical departments and experienced specialists. In the following review, the most interesting abstracts from the annual American Society of Hematology (ASH) meeting in 2016 are presented. The topics include the limitations of established biomarkers in risk assessment and response evaluation, the introduction of a new biomarker, the comparison of different treatment sequences and the efficacy of a multiple drug regimen in light-chain (AL) amyloidosis.

Keywords: 11-1F4; Anti-SAP antibody; Antifibrillary antibody; NEOD001; Protein misfolding disease.

Fig. 1

Transthoracic echocardiography. The typical features of cardiac amyloidosis in transthoracic echocardiography in a four-chamber view: a speckled appearance of the hypertrophied intraventricular septum, dilated atria and pericardial effusion (a). Pulsed-waved Doppler echocardiography illustrating a restrictive filling pattern with an E/A ratio of 5.05 (b). Two-dimensional speckle-tracking revealing the apical sparing of the longitudinal strain (c). Courtesy of Franz Ducka, MD (Medical University Vienna, Department of Internal Medicine II, Division of Cardiology)

Fig. 2

Cardiac MRI features in amyloidosis. The cine imaging shows hypertrophic left and right ventricles as well as severely dilated atria (a). Typical late enhancement pattern 15 min after gadolinium application (b). Native T1 mapping, which is used for extracellular volume quantification (native T1 time myocardium: 1180 ms; extracellular volume: 56.8%; c). Courtesy of Franz Ducka, MD (Medical University Vienna, Department of Internal Medicine II, Division of Cardiology)

Fig. 3

Colonic AL amyloidosis. a Histology and a hematoxylin and eosin (H&E) stain; original magnification (x10). Focal eosinophilic homogenisation of the submucosa. b Immunohistological amyloid typing with a kappa light-chain antibody revealing the specific positive staining of the deposits. c Congo red staining of the deposits with d characteristic apple-green birefringence under polarised light. Courtesy of Ingrid Simonitsch-Klupp, MD (Medical University Vienna, Institute of Pathology)