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

被引：311

作者：

Comenzo, R. L. ^{[1
]}

Reece, D. ^{[2
]}

Palladini, G. ^{[3
]}

Seldin, D. ^{[4
]}

Sanchorawala, V. ^{[4
]}

Landau, H. ^{[5
]}

Falk, R. ^{[6
]}

Wells, K. ^{[7
]}

Solomon, A. ^{[7
]}

Wechalekar, A. ^{[8
]}

Zonder, J. ^{[9
]}

Dispenzieri, A. ^{[10
]}

Gertz, M. ^{[10
]}

Streicher, H. ^{[11
]}

Skinner, M. ^{[4
]}

Kyle, R. A. ^{[10
]}

Merlini, G. ^{[3
]}

机构：

[1] Tufts Med Ctr, Blood Bank, Div Hematol Oncol, Boston, MA 02111 USA

[2] Princess Margaret Hosp, Toronto, ON M4X 1K9, Canada

[3] Univ Pavia, Fdn IRCCS Policlin San Matteo, I-27100 Pavia, Italy

[4] Boston Univ, Sch Med, Boston, MA 02118 USA

[5] Mem Sloan Kettering Canc Ctr, New York, NY 10021 USA

[6] Brigham & Womens Hosp, Boston, MA 02115 USA

[7] Univ Tennessee, Grad Sch Med, Knoxville, TN USA

[8] UCL, London, England

[9] Barbara Ann Karmanos Canc Inst, Detroit, MI USA

[10] Mayo Clin, Rochester, MN USA

[11] NCI, Washington, DC USA

来源：

LEUKEMIA | 2012年 / 26卷 / 11期

关键词：

amyloidosis; free light chains; clinical trials; clinical research; STEM-CELL TRANSPLANTATION; BRAIN NATRIURETIC PEPTIDE; HIGH-DOSE MELPHALAN; TWICE-WEEKLY BORTEZOMIB; AL AMYLOIDOSIS; UNDETERMINED SIGNIFICANCE; MONOCLONAL GAMMOPATHIES; INTRAVENOUS MELPHALAN; MOLECULAR-MECHANISMS; CARDIAC BIOMARKERS;

D O I：

10.1038/leu.2012.100

中图分类号：

R73 [肿瘤学];

学科分类号：

100214 ;

摘要：

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.

引用

页码：2317 / 2325

页数：9

共 53 条

[1] Light and electron microscopy immunohistochemical characterization of amyloid deposits [J].

Arbustini, E ;

Morbini, P ;

Verga, L ;

Concardi, M ;

Porcu, E ;

Pilotto, A ;

Zorzoli, I ;

Garini, P ;

Anesi, E ;

Merlini, G .

AMYLOID-INTERNATIONAL JOURNAL OF EXPERIMENTAL AND CLINICAL INVESTIGATION, 1997, 4 (03) :157-170

[2] Independent Predictors of Survival in Primary Systemic (AL) Amyloidosis, Including Cardiac Biomarkers and Left Ventricular Strain Imaging: An Observational Cohort Study [J].

Bellavia, Diego ;

Pellikka, Patricia A. ;

Al-Zahrani, Ghormallah B. ;

Abraham, Theodore P. ;

Dispenzieri, Angela ;

Miyazaki, Chinami ;

Lacy, Martha ;

Scott, Christopher G. ;

Oh, Jae K. ;

Miller, Fletcher A., Jr. .

JOURNAL OF THE AMERICAN SOCIETY OF ECHOCARDIOGRAPHY, 2010, 23 (06) :643-652

[3] Evaluation of the cytogenetic aberration pattern in amyloid light chain amyloidosis as compared with monoclonal gammopathy of undetermined significance reveals common pathways of karyotypic instability [J].

Bochtler, Tilmann ;

Hegenbart, Ute ;

Cremer, Friedrich W. ;

Heiss, Christiane ;

Benner, Axel ;

Hose, Dirk ;

Moos, Marion ;

Bila, Jelena ;

Bartram, Claus R. ;

Ho, Anthony D. ;

Goldschmidt, Hartmut ;

Jauch, Anna ;

Schonland, Stefan O. .

BLOOD, 2008, 111 (09) :4700-4705

[4] Antibodies to human serum amyloid P component eliminate visceral amyloid deposits [J].

Bodin, Karl ;

Ellmerich, Stephan ;

Kahan, Melvyn C. ;

Tennent, Glenys A. ;

Loesch, Andrzej ;

Gilbertson, Janet A. ;

Hutchinson, Winston L. ;

Mangione, Palma P. ;

Gallimore, J. Ruth ;

Millar, David J. ;

Minogue, Shane ;

Dhillon, Amar P. ;

Taylor, Graham W. ;

Bradwell, Arthur R. ;

Petrie, Aviva ;

Gillmore, Julian D. ;

Bellotti, Vittorio ;

Botto, Marina ;

Hawkins, Philip N. ;

Pepys, Mark B. .

NATURE, 2010, 468 (7320) :93-97

[5] Reliable typing of systemic amyloidoses through proteomic analysis of subcutaneous adipose tissue [J].

Brambilla, Francesca ;

Lavatelli, Francesca ;

Di Silvestre, Dario ;

Valentini, Veronica ;

Rossi, Rossana ;

Palladini, Giovanni ;

Obici, Laura ;

Verga, Laura ;

Mauri, Pierluigi ;

Merlini, Giampaolo .

BLOOD, 2012, 119 (08) :1844-1847

[6] Translocation t(11;14) and survival of patients with light chain (AL) amyloidosis [J].

Bryce, Alan H. ;

Ketterling, Rhett P. ;

Gertz, Morie A. ;

Lacy, Martha ;

Knudson, Ryan A. ;

Zeldenrust, Steven ;

Kumar, Shaji ;

Hayman, Suzanne ;

Buadi, Francis ;

Kyle, Robert A. ;

Greipp, Philip R. ;

Lust, John A. ;

Russell, Stephen ;

Rajkumarl, S. Vincent ;

Fonseca, Rafael ;

Dispenzieri, Angela .

HAEMATOLOGICA-THE HEMATOLOGY JOURNAL, 2009, 94 (03) :380-386

[7] Risk-adapted autologous stem cell transplantation with adjuvant dexamethasone ± thalidomide for systemic light-chain amyloidosis:: results of a phase II trial [J].

Cohen, Adam D. ;

Zhou, Ping ;

Chou, Joanne ;

Teruya-Feldstein, Julie ;

Reich, Lilian ;

Hassoun, Hani ;

Levine, Beth ;

Filippa, Daniel A. ;

Riedel, Elyn ;

Kewalramani, Tarun ;

Stubblefield, Michael D. ;

Fleisher, Martin ;

Nimer, Stephen ;

Comenzo, Raymond L. .

BRITISH JOURNAL OF HAEMATOLOGY, 2007, 139 (02) :224-233

[8] Seeking confidence in the diagnosis of systemic AL (Ig light-chain) amyloidosis: patients can have both monoclonal gammopathies and hereditary amyloid proteins [J].

Comenzo, RL ;

Zhou, P ;

Fleisher, M ;

Clark, B ;

Teruya-Feldstein, J .

BLOOD, 2006, 107 (09) :3489-3491

[9]

Comenzo RL, 1998, BLOOD, V91, P3662

[10] Effect of dose-intensive intravenous melphalan and autologous blood stem-cell transplantation on AL amyloidosis-associated renal disease [J].

Dember, LM ;

Sanchorawala, V ;

Seldin, DC ;

Wright, DG ;

LaValley, M ;

Berk, JL ;

Falk, RH ;

Skinner, M .

ANNALS OF INTERNAL MEDICINE, 2001, 134 (09) :746-753

← 1 2 3 4 5 6 →