Consensus guidelines for the conduct and reporting of clinical trials in systemic light-chain amyloidosis

• Published in: Leukemia Vol. 26; no. 11; pp. 2317 - 2325
• Main Authors: Comenzo, R L, Reece, D, Palladini, G, Seldin, D, Sanchorawala, V, Landau, H, Falk, R, Wells, K, Solomon, A, Wechalekar, A, Zonder, J, Dispenzieri, A, Gertz, M, Streicher, H, Skinner, M, Kyle, R A, Merlini, G
• Format: Journal Article
• Language: English
• Published: London Nature Publishing Group UK 01.11.2012; Nature Publishing Group
• Subjects: 692/308/2779/109; 692/699/317; 692/700/1538; Amyloidosis; Amyloidosis - therapy; Biological and medical sciences; Blood diseases; Cancer; Cancer Research; Chains; Clinical trials; Clinical Trials as Topic; Collaboration; Critical Care Medicine; Drug development; Endpoint Determination; Guidelines; Heart; Hematologic and hematopoietic diseases; Hematology; Humans; Immunoglobulins; Intensive; Internal Medicine; Leukemia; Light; Medical sciences; Medicine; Medicine & Public Health; Multiple myeloma; Oncology; Plasma; Population studies; Practice Guidelines as Topic; Proteins; review; Toxicity; Canada; United States; Italy; United States > US; clinical trials; amyloidosis; free light chains; clinical research; AL amyloidosis; Hematology; Clinical trial; Clinical research; Light peptide chain; Consensus; Recommendation
• ISSN: 0887-6924; 1476-5551; 1476-5551
• DOI: 10.1038/leu.2012.100

This manuscript summarizes the recommendations that emerged from the first Roundtable on Clinical Research in Immunoglobulin Light-chain Amyloidosis (AL), a meeting sponsored by the Amyloidosis Foundation (Clarkston, MI, USA) to develop a consensus of experts on a modern framework for clinical trial design and drug development in AL. Recent diagnostic and technical advances in AL, and updated consensus guidelines for assessing hematologic and organ responses, enable us to define study populations, appropriate end points, and other criteria for all phases of clinical research. This manuscript provides a framework for the design and conduct of systematic collaborative clinical research in AL to encourage more rapid testing of therapies and to expedite new drug development and approval.