ReviewLeukemia2016
Rationale, application and clinical qualification for NT-proBNP as a surrogate end point in pivotal clinical trials in patients with AL amyloidosis.
Review in Leukemia, 2016. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 46 papers, 1 of them a synthesis that pooled it.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
46 citing papers in PubMed, 1 synthesis or guideline pooled it, 99 citations in OpenAlex.
- Diagnostic Role of NT-proBNP in Patients with Cardiac Amyloidosis Involvement: A Meta-Analysis.Arquivos brasileiros de cardiologia · 2022Pooled it
- Effects of Patisiran, an RNA Interference Therapeutic, on Cardiac Parameters in Patients With Hereditary Transthyretin-Mediated Amyloidosis.Circulation · 2019Trial
- Recent developments in systemic light-chain amyloidosis prognosis and treatment.Future cardiology · 2025Review
- Screening for Systemic Light-Chain Amyloidosis in Patients Over 60 with λ Monoclonal Gammopathies.Journal of clinical medicine · 2025Article
- A 68-Year-Old Colombian Man Presenting with Heart Failure and a Diagnosis of Cardiac Transthyretin Amyloidosis.The American journal of case reports · 2024Article
- Autologous stem cell transplantation in AL amyloidosis: Muddy waters.Blood reviews · 2024Review
- Cardiac Amyloidosis: State-of-the-Art Review in Molecular Pathology.Current issues in molecular biology · 2024Review
- Liver stiffness as a prognostic parameter and tool for risk stratification in advanced cardiac transthyretin amyloidosis.Clinical research in cardiology : official journal of the German Cardiac Society · 2024Article
- A Simple Frailty Score Predicts Survival and Early Mortality in Systemic AL Amyloidosis.Cancers · 2024Article
- Natriuretic Peptides and Cardiac Troponins: Markers of Disease Progression and Risk in Light Chain Cardiac Amyloidosis.Current heart failure reports · 2023Review
- Systemic AL amyloidosis: current approach and future direction.Oncotarget · 2023Review
- RNA Targeting and Gene Editing Strategies for Transthyretin Amyloidosis.BioDrugs : clinical immunotherapeutics, biopharmaceuticals and gene therapy · 2023Review
- Patisiran for the Treatment of Transthyretin-mediated Amyloidosis with Cardiomyopathy.Heart international · 2023Review
- Immunoglobulin Light Chain Amyloidosis: Diagnosis and Risk Assessment.Journal of the National Comprehensive Cancer Network : JNCCN · 2023Review
- Cardiovascular magnetic resonance in light-chain amyloidosis to guide treatment.European heart journal · 2022Article
- Nuclear Molecular Imaging of Disease Burden and Response to Treatment for Cardiac Amyloidosis.Biology · 2022Review
- Targeting Amyloid Fibrils by Passive Immunotherapy in Systemic Amyloidosis.BioDrugs : clinical immunotherapeutics, biopharmaceuticals and gene therapy · 2022Review
- Molecular Mechanism of Pathogenesis and Treatment Strategies for AL Amyloidosis.International journal of molecular sciences · 2022Review
- Proposed Cardiac End Points for Clinical Trials in Immunoglobulin Light Chain Amyloidosis: Report From the Amyloidosis Forum Cardiac Working Group.Circulation. Heart failure · 2022Review
- Biomarkers in AL Amyloidosis.International journal of molecular sciences · 2021Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
11 authors at 8 institutions in 4 countries.
Funding
Abstract
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 pro-brain 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.
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What OpenQuestion holds
Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.