Prognostic Factors Working Group

Prognostic Factors for Drug Development and Clinical Care Management

Goals

To develop prognostic factors for transthyretin (ATTR) and light chain (AL ) amyloidosis clinical trial endpoints, with quantitative assessments and validation that reflects current standards of care.

Rationale

  • Understanding prognostic factors is central to optimizing clinical trial designs in terms of enrichment, inclusion/exclusion criteria, baseline stratification, trial durations, and potential use of external control data. These design specifications rely on quantitative understanding of baseline prognostic factors for the trial’s endpoints.
  • Combinations of prognostic factors into composite prognostic scores with better predictive performance than any single factor can also improve trial designs.
  • While there is confidence in our shared knowledge of prognostic factors for survival and other outcomes, specific quantification of prognostic factors is needed for common trial endpoints such as changes in function (e.g., Six-Minute Walk Duration [6MWD]) or quality of life (e.g., Kansas City Cardiomyopathy Questionnaire [KCCQ]).
  • Quantitative relationships between known prognostic factors and outcomes (e.g., heart failure events, survival) also need to be updated for current standard of care.

Chairs

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Mathew Maurer
Working Group Chair
Columbia University Medical Center
BIO+
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Ashutosh Wechalekar
Working Group Co-Chair
National Amyloidosis Centre, University College of London
BIO+

Members

  • Brian Boursiquot

    Columbia University

  • Chafic Karam

    Abramson Cancer Center of the University of Pennsylvania

  • David Wolinsky

    Cleveland Clinic Florida

  • Dimitrios Bampatsias

    Columbia University

  • Helen Lachmann

    Royal Free London NHS Foundation Trust

  • James Hoffman

    University of Miami Sylvester Cancer Center

  • Jamie Zigterman

    Amyloidosis Research Consortium

  • Jan Griffin

    Medical University of South Carolina

  • Jenni Li

    FDA

  • Joban Vaishnav

    Johns Hopkins

  • Jovian Yu

    AbbVie

  • Mazen Hanna

    Cleveland Clinic

  • Mike Petrassi

    Protego

  • Morie Gertz

    Mayo Clinic

  • Pablo Garcia-Pavia

    Hospital Universitario Puerta de Hierro-Majadahonda

  • Rajshekhar Chakraborty

    Columbia University Irving Medical Center

  • Ricardo Rocha

    Intellia

  • Ron Witteles

    Stanford University School of Medicine

  • Yevgeniy Brailovsky

    Columbia University