Workshop on Sickle Cell Disease Cochairs: George R' Buchanan, MD and Marilyn J' Telen, MD Objective: - PowerPoint PPT Presentation

1 / 14
About This Presentation
Title:

Workshop on Sickle Cell Disease Cochairs: George R' Buchanan, MD and Marilyn J' Telen, MD Objective:

Description:

American Society of Hematology (ASH ) Workshop on Sickle Cell Disease. Co ... Members include clinicians, researchers, academicians, and students of hematology. ... – PowerPoint PPT presentation

Number of Views:64
Avg rating:3.0/5.0
Slides: 15
Provided by: ssuthe
Category:

less

Transcript and Presenter's Notes

Title: Workshop on Sickle Cell Disease Cochairs: George R' Buchanan, MD and Marilyn J' Telen, MD Objective:


1
Workshop on Sickle Cell DiseaseCo-chairs
George R. Buchanan, MD and Marilyn J. Telen, MD
Objective To construct a research agenda
aimed at better defining risk factors which
predict later adverse outcomes in sickle cell
disease, with particular emphasis on end organ
failure in adults.May 8-9, 2007Washington,
DC
2
About ASH
  • ASH is the worlds largest professional society
    (over 15,000 members worldwide) concerned with
    the causes and treatment of blood disorders.
    Members include clinicians, researchers,
    academicians, and students of hematology.
  • ASHs Mission To further the understanding,
    diagnosis, treatment, and prevention of disorders
    affecting the blood, bone marrow, and the
    immunologic, hemostatic and vascular systems, by
    promoting research, clinical care, education,
    training, and advocacy in hematology.

3
Background to ASH Participation in the Sickle
Cell Summit
  • 2005 ASPHO invited ASH to participate in a
    Sickle Cell Disease Summit.
  • December 2006 ASH chose to concentrate on the
    area of research.
  • February 2007 Drs. George Buchanan and Marilyn
    Telen were asked to chair a workshop on setting a
    research agenda for sickle cell disease

4
Workshop FocusMethods to Predict Risk of Later
Complications at an Early Age
  • Life expectancy of patients with SCD continues to
    increase.
  • As patients live longer, they develop more
    end-organ damage as a consequence of SCD.
  • There is a large degree of variability among
    patients regarding the complications of SCD they
    experience.
  • We need to understand risk in order to test
    preventive as well as treatment approaches.

5
Workshop Hypotheses
  • Advances in the prevention and treatment of
    sequelae of SCD will require understanding the
    processes that produce early and often undetected
    end-organ damage during childhood.
  • Clinical studies of diagnostic tools, as well as
    preventive and treatment strategies, will have to
    span the pediatric-adult divide in order to
    succeed in improving organ function. Improved
    knowledge of the natural history of organ
    pathology over time is also needed.

6
Methodology
  • Goal To promote stimulating, focused and
    well-informed discussion of key topics and
    research questions.
  • Strategy
  • Discussion leaders and discussants were
    identified for a number of key topics/organ
    systems.
  • Topics and talking points were established and
    distributed prior to the workshop, through small
    group teleconferences.
  • Reference lists were also distributed.

7
Discussion Topics
  • Target organs known to be affected by SCD, as
    well as pain.
  • Traditional and new methods for monitoring organ
    function and predicting risks of complications
  • Currently available preventive therapies.
  • What is in the pipeline.

8
Outcomes of the Workshop
  • Overwhelming support for a larger and more
    inclusive multi-institutional collaborative
    clinical research group, modeled after the
    cooperative oncology trials groups.
  • A detailed report with prioritized research areas
    will be presented to the ASH Executive Committee
    by September and then forwarded to interested
    agencies, including NHLBI, NIDDK, CDC, as well as
    published.
  • It is hoped that this effort will also lead to
    one or more RFAs from NIH.

9
ASH Research Agenda
  • Establishment of a Sickle Cell Disease
    Collaborative Research Group
  • Infrastructure and operation How will it work?
  • Mission What should it do?
  • Vision What elements are necessary for its
    success?

10
ASH Research Agenda
Sickle Cell Disease Collaborative Research
Group (The Group)
  • New organization needed to spearhead research
    agenda
  • Focus on prevention of organ damage in adults
  • Supplement existing NIH-funded clinical research
    initiatives
  • Modeled after NCI cooperative trials groups
    and/or comprehensive hemophilia center network
    (MCHB/CDC)
  • Inter-agency partnerships and other linkages
    required

11
ASH Research Agenda
Infrastructure and Operation of The Group
  • Necessary Components
  • National database/registry of patients with SCD
  • Inclusive
  • Representative
  • Secure
  • Nimble
  • Laboratory bank or repository linked to registry
    for biomarkers and genomic studies
  • Availability of database and specimens for
    translational research
  • Highly skilled research staff (faculty, nurses,
    data coordinators) at Group sites
  • Robust training environment
  • Novel peer-reviewed funding mechanisms

12
ASH Research Agenda
Sickle Cell Disease Collaborative Research
Group (The Group)
  • The Mission What are the Groups Aims?
  • Establish valid and reliable definitions of
    disease severity (overall and organ-specific)
  • Characterize disease phenotypes (e.g.,
    vaso-occlusive and hemolytic) and their
    pathophysiology
  • Develop better means of measuring early organ
    damage and foster conduct of organ-specific
    intervention trials (Example pulmonary
    hypertension)

13
ASH Research Agenda
Sickle Cell Disease Collaborative Research
Group (The Group)
  • Execute large scale trials of hydroxyurea and
    chronic transfusion, including careful study of
    effectiveness as well as efficacy
  • Better characterize clinically relevant
    endpoints, including mortality, event rates,
    hematologic measures, and HRQOL
  • Understand sickle cell pain, collaborating with
    pain experts in other disciplines
  • Design small molecules that inhibit sickling
  • Expand stem cell transplantation opportunities
  • Collaborate far more with the international SCD
    community

14
ASH Research Agenda
Sickle Cell Disease Collaborative Research
Group (The Group)
  • The Vision What Elements are Necessary for The
    Groups Success?
  • Collaborative spirit among investigators and
    institutions
  • Training of more clinical and translational
    investigators
  • Increased funding from NIH starting with RFAs
    for pilot projects
  • Involvement of HRSA, CDC, CMS in supporting the
    research agenda
  • Active engagement of relevant advocacy groups,
    community organizations, and professional
    societies
  • Recognizing and overcoming barriers to success
Write a Comment
User Comments (0)
About PowerShow.com