@article{240a58190ca44f2891843856537b42f1,
title = "Consensus guidelines for the conduct and reporting of clinical trials in systemic light-chain amyloidosis",
abstract = "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.",
keywords = "Amyloidosis, Clinical research, Clinical trials, Free light chains",
author = "Comenzo, {R. L.} and D. Reece and G. Palladini and D. Seldin and V. Sanchorawala and H. Landau and R. Falk and K. Wells and A. Solomon and A. Wechalekar and J. ZonDer and A. Dispenzieri and M. Gertz and H. Streicher and M. Skinner and Kyle, {R. A.} and G. Merlini",
note = "Funding Information: 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. Funding Information: Systemic immunoglobulin light-chain amyloidosis (AL) is the most common type of systemic amyloidosis with an estimated incidence of 8–10 cases per million person-years.1,2 Although there have been recent advances in AL, there is a major need for a framework for clinical research to encourage the development of new therapies. Access to novel agents and their optimal and safe use are challenges for patients with this rare disease. Well-designed clinical trials with multicenter collaboration will accelerate timely testing of novel agents and combinations, will improve access and will accelerate pharmaceutical company efforts and acceptance by payers. We in the United States are heartened by the continued interest and support of the National Cancer Institute study groups, particularly the Southwest and Eastern Oncology Groups, in developing phase II and III trials for patients with AL, and in the European Union by the collaboration of national plasma-cell disease networks and of the European Myeloma Network. These efforts should be complemented by the development of a phase I pipeline to nurture the development of phase II and III trials.",
year = "2012",
month = nov,
doi = "10.1038/leu.2012.100",
language = "English (US)",
volume = "26",
pages = "2317--2325",
journal = "Leukemia",
issn = "0887-6924",
publisher = "Nature Publishing Group",
number = "11",
}