Rationale, application and clinical qualification for NT-proBNP as a surrogate end point in pivotal clinical trials in patients with AL amyloidosis

被引:73
|
作者
Merlini, G. [1 ]
Lousada, I. [2 ]
Ando, Y. [3 ]
Dispenzieri, A. [4 ]
Gertz, M. A. [4 ]
Grogan, M. [4 ]
Maurer, M. S. [5 ]
Sanchorawala, V. [6 ,7 ]
Wechalekar, A. [8 ]
Palladini, G. [1 ]
Comenzo, R. L. [9 ]
机构
[1] Univ Pavia, IRCCS Policlin San Matteo, Dept Mol Med, Amyloid Res & Treatment Ctr, Pavia, Italy
[2] Amyloidosis Res Consortium Inc, Boston, MA USA
[3] Kumamoto Univ, Grad Sch Med Sci, Dept Neurol, Kumamoto, Japan
[4] Mayo Clin, Coll Med, Rochester, MN USA
[5] Columbia Univ, Med Ctr, Clin Cardiovasc Res Lab Elderly, New York, NY USA
[6] Boston Univ, Sch Med, Amyloidosis Ctr, Boston, MA USA
[7] Boston Univ, Med Ctr, Boston, MA USA
[8] UCL, Natl Amyloidosis Ctr, Med & Haematol, London, England
[9] Tufts Med Ctr, John C Davis Myeloma & Amyloid Program, 800 Washington St, Boston, MA 02111 USA
关键词
LIGHT-CHAIN AMYLOIDOSIS; BRAIN NATRIURETIC PEPTIDE; PRIMARY SYSTEMIC AMYLOIDOSIS; CARDIAC TROPONIN-T; BIOLOGICAL VARIATION; EARLY MORTALITY; STAGING SYSTEM; TASK-FORCE; SURVIVAL; HEART;
D O I
10.1038/leu.2016.191
中图分类号
R73 [肿瘤学];
学科分类号
100214 ;
摘要
Amyloid light-chain (LC) amyloidosis (AL amyloidosis) is a rare and fatal disease for which there are no approved therapies. In patients with AL amyloidosis, LC aggregates progressively accumulate in organs, resulting in organ failure that is particularly lethal when the heart is involved. A significant obstacle in the development of treatments for patients with AL amyloidosis, as well as for those with any disease that is rare, severe and heterogeneous, has been satisfying traditional clinical trial end points (for example, overall survival or progression-free survival). It is for this reason that many organizations, including the United States Food and Drug Administration through its Safety and Innovation Act Accelerated Approval pathway, have recognized the need for biomarkers as surrogate end points. The international AL amyloidosis expert community is in agreement that the N-terminal fragment of the probrain natriuretic peptide (NT-proBNP) is analytically validated and clinically qualified as a biomarker for use as a surrogate end point for survival in patients with AL amyloidosis. Underlying this consensus is the demonstration that NT-proBNP is an indicator of cardiac response in all interventional studies in which it has been assessed, despite differences in patient population, treatment type and treatment schedule. Furthermore, NT-proBNP expression is directly modulated by amyloidogenic LC-elicited signal transduction pathways in cardiomyocytes. The use of NT-proBNP will greatly facilitate the development of targeted therapies for AL amyloidosis. Here, we review the data supporting the use of NT-proBNP, a biomarker that is analytically validated, clinically qualified, directly modulated by LC and universally accepted by AL amyloidosis specialists, as a surrogate end point for survival.
引用
收藏
页码:1979 / 1986
页数:8
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