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.
authors.affiliation: Division of Hematology/Oncology, Tufts Medical Center, Boston, MA 02111, USA. rcomenzo@tuftsmedicalcenter.org
authors.forename: R L
authors.initials: RL
authors.lastname: Comenzo
authors.forename: D
authors.initials: D
authors.lastname: Reece
authors.forename: G
authors.initials: G
authors.lastname: Palladini
authors.forename: D
authors.initials: D
authors.lastname: Seldin
authors.forename: V
authors.initials: V
authors.lastname: Sanchorawala
authors.forename: H
authors.initials: H
authors.lastname: Landau
authors.forename: R
authors.initials: R
authors.lastname: Falk
authors.forename: K
authors.initials: K
authors.lastname: Wells
authors.forename: A
authors.initials: A
authors.lastname: Solomon
authors.forename: A
authors.initials: A
authors.lastname: Wechalekar
authors.forename: J
authors.initials: J
authors.lastname: Zonder
authors.forename: A
authors.initials: A
authors.lastname: Dispenzieri
authors.forename: M
authors.initials: M
authors.lastname: Gertz
authors.forename: H
authors.initials: H
authors.lastname: Streicher
authors.forename: M
authors.initials: M
authors.lastname: Skinner
authors.forename: R A
authors.initials: RA
authors.lastname: Kyle
authors.forename: G
authors.initials: G
authors.lastname: Merlini
mesh_terms: D000686:Amyloidosis / Q000628:therapy*; D002986:Clinical Trials as Topic; D023381:Endpoint Determination; D006801:Humans; D017410:Practice Guidelines as Topic