MINUTES OF THE EIGHTY-FIFTH MEETING OF THE SICKLE CELL DISEASE ADVISORY COMMITTEE

Bethesda, Maryland

November 3, 2003


COMMITTEE MEMBERS PRESENT

Dr. Paul Frenette, Dr. Oswaldo Castro, Dr. Theodore Wun, Dr. Joseph Telfair, Ms. J. Hoxi Jones, Dr. Russell Ware, Dr. Micheal DeBaun, Dr. Gilda Barabino.

COMMITTEE MEMBERS ABSENT

Dr. Johnson Haynes

EX-OFFICIO MEMBERS PRESENT

Dr. Robert Sheffler, Dr. Joseph Desimone

EX-OFFICIO MEMBERS ABSENT

Dr. William Hannon, Dr. Marie Mann

PROGRAM STAFF AND AFFILIATED ORGANIZATION REPRESENTATIVES:

Dr. Duane Bonds, DBDR/BDP; Dr. Gregory Evans, DBDR/BDP; Dr. Blaine Moore DBDR/BDP; Dr. Charles Peterson, DBDR; Ms. Sonya Ross, SCDAA; Dr. Ellen Werner, DBDR/BDP ; Dr. Georgia J. Persinos, PG Associates; Dr. William P. Winter, Howard University; Dr. Carl Hunt, NHLBI; Dr. Jeanne Smith, Harlem, NY; Dr. Dorothy Moore, UMDNJ/NJMS & SCDAA; Ms. Mae Osburn, SCDAD

Executive Secretary - Dr. Charles M. Peterson

Secretary - Ms. Petronella A. Barrow

Dr. Charles Peterson called the meeting to order at 8:30 am and referred members to the required notification regarding conflicts of interest and reminded those present to sign in on the required sign-in sheets.

  1. Dr. Ted Wun called for review of the minutes of June 3, 2003 and they were reviewed and approved.
  2. Dr. Wun welcomed the members and guests and introduced the Scientific Presentations.

    A. Dr. Gabriel Haddad: “Sleep Disordered Breathing and Hypoxemia” Dr. Haddad reviewed the complications of sickle cell disease with special emphasis on the lung and the central nervous system. Disordered breathing and sleep disorder in persons with sickle cell disease may result from damage to these critical organ systems and in turn may contribute to the sequential accumulative damage seen in each. For these reasons, persons with sickle cell disease and with sleep dysfunction including snoring and obstruction should be evaluated by a sleep specialist. The critical nature of these interactions remains to be defined and presents an opportunity for a number of investigations in the area.

    B. Dr. Jeanne Smith “Women’s Health Issues in Sickle Cell Disease” Women with sickle cell disease are vulnerable to the same common end organ sequelae as their male counterpart. Many studies of the issues of reproductive health are confounded by the overlapping issues associated with access to health care. The Cooperative Study of Sickle Cell Disease did document an increased prevalence of small for gestational age infants in children born to sickle cell disease mothers. The reasons for this difference are unknown. The C-section rate also tends to be high in these women. Optimum birth control strategies and use or non use of hydroxyurea in women contemplating pregnancy are among the critical issues that require further work.

  3. Chairman’s Report: Dr Wun noted that he looked forward to working with the Committee and helping move forward science in the area of Sickle Cell Disease.

  4. DBDR Director’s Report: Dr Peterson reported on the state of the budget and the second Continuing Resolution for FY 2004. Until a budget is approved, NHLBI will have a very conservative payline.

  5. Action Items from previous meeting were completed or discussed as follows:

    A. Staff will check the web site regarding the need for and update regarding sickle cell hyphema and Dr. Goldberg will be asked to critique the minutes and make recommendations for appropriate references and recommendations by other professional bodies. This was done by staff.

    B. Drs. Lane, Wun and Telfair will work on an evaluation of clinical trials needs and assessments and recommendations for priorities made. This effort is ongoing and will be continued by Drs. Wun and Telfair

    C. Dr. Helena Mishoe will be asked to present her new Office of Minority Health Affairs and its role to the committee. Dr. Mishoe gave an overview of her office and its responsibilities. While the office is relatively new, its responsibilities span intra- and extramural affairs. The goals for the Office include not only advocating approaches to recruitment and retention of minorities in the scientific workplace but also winning hearts and minds for participation in the research endeavor at every level including participation in clinical trials.

    D. An organization chart for the Centers Program will be provided to the committee by Dr. Evans. The Centers Program was reviewed in detail by Dr. Evans under the Update on Program Activities.

    E. Follow up on the Pulmonary Disease Initiative and others that have not moved forward will be provided by staff at the next meeting. The pulmonary initiative was felt by the committee to still be of the highest priority. The initiative has been published in the Early Notification System and will be published soon in The Guide to Grants and Contracts

    F. Dr. Werner will forward the executive summary of the Workshop on Unmet Needs to the committee and provide the complete write-up as soon as it is available. The full report was distributed. In addition to the Executive Summary and Recommendations, which were posted on the NHLBI website in the summer, the full report describes the follow-up activities that have ensued since June, 2002. Planning Committee members and workshop presenters received a copy via email and were asked to provide comments by Friday, November 7, 2003. The report will be posted on the NHLBI website by the end of November, 2003.

    G. Drs. Wun, Lane and Ware will continue to address the issue of ICD 9 codes and sickle cell disease. This issue remains outstanding in part due to the difficulty of understanding and influencing the process. Dr Wun will contact Drs. Swerdlow and Lane and see if further progress can be made in this area. Dr. Ware will also help. The Committee concurred that the codes along with DRGs often create barriers to optimum care for persons with sickle cell disease.

    H. Dr. Lane will draft a list of items to be included in an orientation booklet for new members to the Sickle Cell Disease Advisory Committee. Dr. Lane had not been able to accomplish this task. The Committee felt that inclusion of the charter in the meeting folders would be helpful and Dr. Evans volunteered to put together a flow chart of the initiative process that could be helpful to new members.*

  6. Agency Reports

    A. Communicable Disease Center: No representation was present for the meeting but materials on a new educational format were distributed that includes sickle cell disease. These materials can be accessed at .

    B. Health Resources and Services Administration: No representation was present. The Committee hoped that an update on reimbursement issues for persons under the age of 21 might be discussed at the next meeting by the HRSA representative.*

    C. Department of Veteran Affairs: Dr. Joseph DeSimone reported that there were no new initiatives to report to the Committee.

    D. Department of the Army: Dr. Robert Sheffler reported that the recommendations of the committee regarding screening for sickle cell hemoglobinopathies by the army was still under consideration. No further action had been taken.

  7. Update on Program Activities

    A. Trans-NIH Workshop: New Directions in Sickle Cell Therapy in the Genome Era: Dr. Evans gave an overview of this Strategic Planning workshop planned for November 19-21 at NIH, with lead sponsorship by the National Human Genome Research Institute. NHLBI, NIDDK, the Fogarty International Center and the Office of Rare Diseases of the NIH/DHHS. are also contributing. One outcome from this three day conference November 19-21 is a planned reevaluation and update of NHLBI’s strategic plan in the area.

    B. The new R21 PA in Rare Diseases was reviewed by Ms. Elizabeth Wagner.

    C. Comprehensive Sickle Cell Centers Integration Committee Report, and High School Student Summer Program: Dr. Evans handed out an organizational chart for the new NHLBI sickle cell center program that now features collaborative clinical trials. He described the functions of the Steering Committee and five subcommittees, and mentioned five protocols under development. Dr. Qasba gave an update on the Summer for Sickle Cell Science Program, that provides Summer research experiences for high school students. 26 students are presently supported at nine centers in the first year of this program.

    D. Strategic Plan for a Gene-Based Cure for Beta-Chain Hemoglobinopathies: Dr. Evans gave an update on the status of this NHLBI Strategic Plan. A joint RFA PI meeting was held in Bethesda Sept. 28-30 that featured over 30 scientific presentations, for three different RFAs.

    E. MSH Patients’ Follow-up: Dr. Bonds noted that the follow-up study continued. A motion was proposed by Dr. Moore and seconded by Dr. DeBaun that “the feasibility of a registry of those receiving hydroxyurea be evaluated with special emphasis on related neoplasias.” The motion passed unanimously. Dr. DeBaun volunteered to work with Dr. Bonds and NHLBI statisticians to determine how this might best be done and report back to the Committee.*

    F. Pediatric Hydroxyurea Phase III Clinical Trial (BABY HUG): Dr. Bonds noted that the study had enrolled the first patient.

    G. Psychosocial Aspects and Outcomes Research: Dr. Werner coordinated a discussion of these efforts. The Committee was enthusiastic about seeing a Quality of Life Instrument that was specific for Sickle Cell Disease in addition to more traditional generic instruments. The Institute was encouraged to continue its efforts in the area.

    H. Parvovirus B19 Studies: Dr. Evans reported that the B19 parvovirus seroprevalance study conducted during the previous funding cycle of the NHLBI Centers is nearly complete. The data is being collated and analyzed. Secondly, a new NHLBI contract for production of anti-B19 vaccine is now being negotiated.

    I. STOP II Trial: The trial is past the 50% recruitment milestone.

    J. The CSSCD Cohort Study and DNA Repository Samples was discussed. The problem is lack of critical sample in a number of vials and thought has been given to expanding the genomic DNA available in these samples to provide an ongoing resource. It was noted that it is difficult to work with the CSSCD data base since there are few individuals from the previous Data Coordinating Center who can access the data in a meaningful manner. Nevertheless it was felt that the effort to preserve sample should be pursued. It was noted that the issue of funding ancillary studies from existing data bases of completed studies was becoming an increasing need and challenge. The previously available Ancillary Studies funding has expired and the question raised if NHLBI had any further plans to address the need.

  8. Other: The Committee requested a list of planned initiatives for proposal to the institute for funding be provided in a format such that they could be rank ordered and given relative rankings of enthusiasm by the Committee. Drs. Evans and Bonds volunteered to provide such a list by e-mail within the next two weeks. Done

  9. Suggestions for future speakers: Members of the committee were encouraged to forward suggestions to staff. One suggestion was to consider speakers who might be able to address the issues of neoplasia associated with hydroxyurea therapy in other areas such as PNH or other experiences with an hydroxyurea registry.
  10. Dates of Next Meetings
  11. June 7, 2004

    November 1, 2004

  12. Summary List of Action Items (*above)

    A. A motion was proposed by Dr. Moore and seconded by Dr. DeBaun that “the feasibility of a registry of those receiving hydroxyurea be evaluated with special emphasis on related neoplasias.” The motion passed unanimously. Dr. DeBaun volunteered to work with Dr. Bonds and NHLBI statisticians to determine how this might best be done and report back to the Committee.

    B. Drs. Lane, Wun and Telfair will work on an evaluation of clinical trials needs and assessments and recommendations for priorities made. This effort is ongoing and will be continued by Drs. Wun and Telfair

    C. The full report on Adults with Sickle Cell: Meeting Unmet Needs will be published on the NHLBI website by the end of November, 2003.

    D. Dr. Evans volunteered to put together a flow chart of the initiative process that could be helpful to new members.

    E. The Committee hoped that an update on reimbursement issues for persons under the age of 21 might be discussed at the next meeting by the HRSA representative.

    F. The Committee requested a list of planned initiatives for proposal to the institute for funding be provided in a format such that they could be rank ordered and given relative rankings of enthusiasm by the Committee. Drs. Evans and Bonds volunteered to provide such a list by e-mail within the next two weeks. Done


    _____________________________

    Theodore Wun, M.D.

    Chairman

    Sickle Cell Disease Advisory Committee


    _____________________________

    Charles M. Peterson, M.D.

    Executive Secretary

    Sickle Cell Disease Advisory Committee

    Last updated: October 28, 2007

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