New Directions for Sickle Cell Therapy in the Genome Era: Attendee Voting for Research Therapies

National Human Genome Research Institute

National Institutes of Health
U.S. Department of Health and Human Services


New Therapies for Sickle Cell Anemia in the Genome Era

Attendees' Votes Among Options for Research on New Therapies for Sickle Cell Disease

Conference attendees were asked to cast votes among approximately 30 priorities that had emerged during the meeting as options for furthering research aimed at developing new, more effective therapies for SCD. All participants were given 100 votes each, which they could then split among any of the possible priorities. Tabulation of those votes is shown below:

Twenty-two (22) percent of all votes were cast in favor of an innovative multidisciplinary Sickle Cell Disease Research Network with a central prospective registry of several thousand well phenotyped patients.

Features of this might include:

Ten (10) percent of all votes were cast in favor of bringing new people and disciplines into the field and training the next generation of researchers. This would include integrating genomics, proteomics, and high-throughput screening expertise into sickle cell disease research. It might also include increasing the number of basic, clinical, and social science researchers, as well as nurses and allied health professionals, doing research on sickle cell disease. Possible mechanisms for accomplishing this might include grant supplements and loan repayment programs.

Eight (8) percent of all votes cast were cast in favor of defining the genetic basis of phenotypic variability. Methods to this might include case/control studies and/or studies of monozygotic and dizygotic twins, of sib pairs and of individuals with unusually mild phenotypes. International collaboration would be particularly helpful here.

Five (5) percent of all votes were cast in favor of establishing a working group to define SCD severity by strict standardized criteria. This might include innovative techniques for more precise definition of phenotypes, e.g., for acute chest syndrome and for stroke, as well as the use of laboratory measures.

Four (4) percent of all votes cast were cast for each of the following:

Three (3) percent of all votes cast were cast for each of the following:

Two (2) percent of all votes cast were cast for each of the following:

One (1) percent of all votes cast were cast for each of the following:

Less than once (<1) percent of all votes cast were cast for each of the following:

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Last Reviewed: November 22, 2013