Chairs:
Joel Buxbaum, La Jolla, CA, USA
Violaine Plante-Bordeneuve, Créteil, France
ATTR AMYLOIDOSIS
OP 08Long-term safety and efficacy of patisiran: Global open-label extension 24-month data in patients with hereditary transthyretin-mediated amyloidosis
David Adams, Le Kremlin Bicêtre, France
OP 09Long-term impact of tafamidis in patients with late-onset hereditary transthyretin amyloidosis with stage I polyneuropathy
Roberta Mussinelli, Pavia, Italy
OP 10External validation of the national amyloidosis center score in an international cohort of patients with transthyretin cardiac amyloidosis
Adrián Rivas-Pérez, Madrid, Spain
OP 11Evaluation of patisiran with concomitant or prior use of transthyretin stabilizers in patients with hereditary transthyretin-mediated amyloidosis
Hollis Lin, Cambridge, MA, USA
OP 12Open-label study of patisiran in patients with hereditary transthyretin-mediated amyloidosis with polyneuropathy post-orthotopic liver transplant
Julian Gillmore, London, UK
OP 13High resolution nerve ultrasound as a diagnostic tool for differential diagnosis and progression recognition in TTR-related familial amyloidosis
Natalie Winter, Tübingen, Germany
OP 14Origin of val30met in familial amyloid polyneuropathy (TTR-FAP) in Portugal: a walk through the mutational path
Carolina Lemos, Porto, Portugal
OP 1599mTc-DPD scintigraphy predicts amyloid fibril type in hereditary transthyretin amyloidosis
Jonas Wixner, Umeå, Sweden
14:56 – 15:30Discussion
Multidisciplinary treatment approach in the management of patients with AL amyloidosis
Chair:
Giovanni Palladini, Pavia, Italy
Diagnostic pit-falls and risk stratification in AL amyloidosis
Efstathios Kastritis, Athens, Greece
Monoclonal antibody treatment for AL amyloidosis
Ashutosh Wechalekar, London, UK
Novel targets and drugs for AL amyloidosis
Giovanni Palladini, Pavia, Italy
Experts’ discussion on the Treatment of Patients with AL Amyloidosis non-eligible for ASCT: burning questions
Chair:
Morie A. Gertz, Rochester, MN, USA
Panelists:
Shaji Kumar, Rochester, MN, USA
Monique C. Minnema, Utrecht, the Netherlands
Paolo Milani, Pavia, Italy
Maria Teresa Cibeira, Barcelona, Spain
Arnaud Jaccard, Limoges, France
Maria Gavriatopoulou, Athens, Greece
Topics:
• Best initial therapy for fit patients
• Best inicial therapy for unfit patients
• When to start therapy at relapse or progression
• Treatment at first relapse
• Treatment at later relapses or refractory disease
• Best novel emerging agents
• Role of anti-amyloid therapy
Chairs:
Matthias Schmidt, Ulm, Germany
Francesca Lavatelli, Pavia, Italy
BASIC SCIENCE II
OP16Hepatic expression of mutant transthyretin remodels proteostasis machinery in hereditary ATTR amyloidosis
Richard Giadone, Boston, MA, USA
OP17Diagnostic potential of a novel RT-QPCR-based assay to measure CCND1 MRNA expression levels in bone marrow plasma cells from al amyloidosis patients
Alice Nevone, Pavia, Italy
OP18Machine learning predicts immunoglobulin light chain toxicity through somatic mutations
Maura Garofalo, Bellinzona, Switzerland
OP19Drosophila melanogaster as a model organism for ATTR amyloidosis
Xiaohong Gu, Uppsala, Sweden
OP20Eleven different amyloid types identified in cutaneous amyloidosis by proteomics-based typing
Surendra Dasari, Rochester, MN, USA
19:05 - 19:20Discussion