Bethesda, Maryland

June 2, 2003


Dr. Oswaldo Castro, J. Hoxi Jones, Dr. Peter Lane, Dr. Herbert J. Meiselman, Dr. Russell Ware, Dr. Joseph Telfair, Dr. Theodore Wun


Dr. Gilda Barabino, Dr. Marie Stuart


Dr. Marie Mann, Dr. Robert Sheffler, Dr. Joseph Desimone


Dr. William Hannon


Barbara Adam, CDC; Dr. Herman Branson, DBDR/BDP, Dr. Duane Bonds, DBDR/SCDSRG, Dr. Gregory Evans, DBDR/SCDSRG; Dr. Charles Peterson, DBDR/BDP; Ms. Sonya Ross, SCDAA, Dr. Ellen Werner, DBDR/BDP; Dr. William P. Winter, Howard University, Dr. Morton Goldberg, Johns Hopkins, Gilian Engelson, HRSA, Onyinye Onyekwere, Howard University, Daniel Prepetit, AMCHP, Dr. Robert Thompson, Duke University, Larry & Michelle Allen, Equal Voices.

Executive Secretary - Dr. Charles M. Peterson

Secretary - Ms. Petronella A. Barrow

Dr. Charles Peterson called the meeting to order at 8:30 am and read the required notification regarding conflicts of interest and reminded those present to sign in on the required sign-in sheets. Dr. Peterson thanked and provided acknowledgment to Drs. Lane, Meiselman and Stuart for their contributions to the committee during their three years of service.

  1. I. Dr. Peter Lane called for review of the minutes of June , 2002 and they were reviewed and approved.

  2. I. Dr. Lane welcomed the members and guests and introduced the Scientific Presentations.

    A. Dr. Morton Goldberg presented Sickle Cell Hyphema or hemorrhage into the anterior chamber of the eye. Hyphema is a relatively common occurrence as a result of ocular trauma but may have devastating consequences in an individual with a sickle cell hemoglobinopathy including those with sickle cell trait. The primary danger in the condition lies in the potential for irreversible optic nerve damage as the result of increased ocular pressure that persists over time. While normal red cells are able to diapedese into the Canal of Schlemm for ultimate return to the normal circulation, sickled cells accumulate en route to the canal and obstruct flow leading to increased pressure and glaucoma. The anterior chamber of the eye promotes sickle cell formation even in sickle cell trait individuals as a result of the properties of anterior fluid that includes a low oxygen, high C02, and low pH environment exacerbated by increased ascorbate levels. The goals of therapy are to monitor ocular pressure every 6 hours in hospital and intervene if pressures exceed 24mmHg for 24 hours. Intervention involves paracentesis of the anterior chamber that may require repeat procedures several times until the condition resolves. Standard ocular approaches to medical treatment of glaucoma have not been useful in sickle cell hyphema. Since in non-sickle cell hemoglobinopathy patients the condition usually resolves spontaneously, the major pitfall is failure to recognize the presence of a sickle cell abnormality especially in individuals with sickle cell trait.

    B. Dr. Robert Thompson spoke about research in psychosocial issues in Sickle Cell Disease. The field has generally focused on identification of outcome risks, and formulation of conceptual models and delineation of correlates of risk and resiliency as potential intervention targets. Increased risk is generally broken into two basic areas: neurocognitive functioning and psychological adjustment. Each has ben the subject of increasingly fruitful studies. Intervention trials to enhance outcomes are still in preliminary stages.

  3. Chairman’s Report: Dr Lane thanked the committee and in particular Ms. Barrow for facilitating the meetings. He noted that he would miss working with the committee and noted that there were still several areas of unfinished business. The issue of achieving translation of therapies into clinical practice through the use of clinical trials still remains a challenge. The reimbursement issues in sickle cell disease and the provision of realistic ICD 9 codes remain to be achieved. The success of the current effort to subsume clinical trials into the Centers program remains to be determined and he recommended that the centers program be reviewed with this question in mind at the appropriate time interval.

  4. DBDR Director’s Report: Dr Peterson introduced Dr. Herman Branson the newly appointed Director of the Blood Diseases Program of the Division of Blood Diseases and Resources and Dr. Jean Henslee-Downey newly appointed Director of the Blood Resources Program. The Division expects that budgets in FY04 and FY05 will not benefit from the rate of rise seen in former years and that the number of larger studies that will be possible will definitely decrease. Nevertheless there are a number of activities underway which promised considerable scientific momentum toward the ultimate development of therapies and an eventual cure for Sickle Cell Disease. The new Sickle Cell Centers program is just underway. There are several clinical trials in process. New partnerships are being developed that will bring increasing attention to sickle cell patients and the disease. The strategic plan for a gene based cure of the hemoglobinopathies continues to move forward.

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

    A. Ms. Sonya Ross on behalf of the sickle Cell Disease Association of America gave a report on the evaluation of the last September joint meeting of the Association and the Centers Investigators. There were over 1,000 attendees of whom 240 returned evaluation forms. In general the attendees comments were very positive. Suggestions for improvement included avoiding simultaneous presentations that made it difficult to choose which to attend, the development of more handouts and outlines, and the avoidance of multiple forms of media presentations since switching from, for example, slides to digital presentations was often cumbersome. There was a request for a social work interest group. Tapes of many of the presentations are available on the web and through the Association. The group felt that having a joint meeting of the lay and scientific audiences was valuable and ought to be considered every year.

  6. Agency Reports

    A. Communicable Disease Center: Ms. Barbara Adam noted that a Sickle Cell Screening Symposium will take place next May in Atlanta, GA that will include sessions on Quality Control Workshops and on Newborn Screening. Those interested may request further information from her by e-mail: bwa1@cdc.gov.

    B. Health Resources and Services Administration: Dr. Marie Mann of the Maternal and Child Health Bureau discussed the new program in newborn screening with $4 million targeted towards sickle cell disease beginning in 2002 and continuing with an equal level of funding through 2003. The program involves follow up and counseling programs. At present 44 states have universal newborn screening and Alaska, Idaho, New Hampshire, North Dakota, and West Virginia are all considering programs. There is also interest on the part of HRSA in funding reference laboratories for hemoglobinopathies based on filter paper blood spot testing. Ultimately such a reference laboratory would have to become self sufficient.

    C. Department of Veteran Affairs: Dr. Joseph DeSimone noted that there was a new director who was highly interested in outcomes and management strategies. One target area was in the area of cardiac arrest. While there is little activity in the area of sickle cell disease, it was noted that the VA might be able to provide some data on the prevalence of hyphema in the sickle cell trait population.

    D. Department of the Army: Dr. Robert Sheffler reported that the recommendations of the SCDAC regarding screening for sickle cell trait in the military had been noted and that the approach to the issue had not markedly changed among the services. It was also noted that the armed services might be able to provide some data on the prevalence of hyphema and outcome in the sickle cell trait population vs. the baseline population of recruits.

  7. Update on Program Activities

    A. Comprehensive Sickle Cell Centers Renewal Progress: Dr. Evans noted that the new funding cycle for the Centers began in April, 2003. The Clinical Trials Network that is part of the Centers Program has met 5 times to begin to prioritize protocols and identifying those for early implementation by the Network. Dr. Evans indicated that the new Network would have a somewhat flexible infrastructure and it is anticipated that 3-6 clinical trials would be completed during this 5 year cycle. Dr. Evans stressed his interest in receiving input from outside the 10 centers regarding research priorities.

    B. Strategic Plan for a Gene-Based Cure for Beta-Chain Hemoglobinopathies: As reported to this committee previously, extramural investigators have proposed a two-armed, 11 part Strategic Plan for Gene-Based Cures for Beta-Chain Hemoglobinopathies. One arm deals with various aspects of the study of the mechanistic basis of induction of endogenous fetal hemoglobin genes, and the other arm deals with the development of gene therapy as a therapeutic modality for sickle cell disease and Cooley’s anemia. To date, two RFAs have been funded and a PA announced.

    C. Pediatric Hydroxyurea Phase III Clinical Trial (BABY HUG): This contract began in August, 2000 with the funding of 10 peripheral clinical sites and a medical coordinating center. The investigator group has spent the first two years of the contract finalizing the protocol and trial design. The protocol has been revised and is now approved by the Protocol Review Committee.

    D. MSH Patients’ Follow-up: This prospective epidemiologic cohort study of the subjects who were originally enrolled in the Multicenter Study of Hydroxyurea (MSH Trial) has been renewed for another 5 years of follow up. Manuscripts were demonstrated showing decreased mortality in those subjects who were begun on hydroxyurea in 1992 and who have continued on the drug with good fetal hemoglobin response since that time. Long term efficacy has been demonstrated in terms of persistence of fetal hemoglobin response, total hemoglobin response, and decreased morbidity secondary to consequences of decreased sickling. However, this patient population continues to have a high mortality rate secondary to the severity of their sickle cell anemia at time of study entry (3 or more crises per year). The leading causes of death in this cohort are death in acute crisis and pulmonary hypertension/chronic sickle lung disease.

    E. Parvovirus B19 Study: This prospective epidemiologic cohort study involves pediatric subjects enrolled at 7 of the 10 NHLBI funded Comprehensive Sickle Cell Centers. These subjects are being followed to ascertain the seroconversion rate from negative to positive after B19 exposure. The study is scheduled to end in 2003 at which time a vaccine trial is being contemplated.

    F. STOP II Trial: This investigator initiated randomized clinical trial is in progress and is designed to answer the question of whether or not chronic transfusion therapy can be safely withdrawn from subjects after 30 months of blood transfusions and normal transcranial doppler (TCD) surveillance studies.

    G. Hip Core Trial: This investigator initiated randomized clinical trial is designed to ascertain the possible utility of hip coring as a treatment for patients with avascular necrosis of the hip. It ended with the current funding cycle of the Comprehensive Sickle Cell Centers in April 2003 and data analysis is underway.

    H. Psychosocial Aspects and Outcomes Research: The report of the workshop that occurred June 13-14, 2002 on Adults with Sickle Cell Disease: Meeting Unmet Needs is available as an executive summary that will be distributed to the committee and a full report will be available soon. There was considerable interest in these issues and the Institute was encouraged to develop a Strategic Plan in the area. Specific suggestions from the group included the development and validation of data collection instruments.

  8. Other

    It was suggested that priorities could be worked on by the committee by circulating a suggested list of activities that could be prioritized by the group. Dr. Lane, and Telfair and Wun agreed to coordinate this activity. Drs. Lane, Ware, and Wun would also make recommendations regarding the next step for ICD 9 coding suggestions.

  9. Suggestions for future speakers

    One suggestion was for a talk on womens health issues in sickle cell disease. Members of the committee were encouraged to forward suggestions to staff.

  10. Dates of Next Meetings
  11. November 3, 2003

    June 7, 2004

  12. Summary List of Action Items

    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.

    B. Drs. Lane, Wun and Telfair will work on an evaluation of clinical trials needs and assessments and recommendations for priorities made.

    C. Dr. Helena Mishoe will be asked to present her new Office and its role to the committee.

    D. An organization chart for the Centers Program will be provided to the committee by Dr. Evans.

    E. Follow up on the Pulmonary Disease Initiative and others that have not moved forward will be provided by staph at the next meeting. The pulmonary initiative was felt by the committee to still be of the highest priority.

    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.

    G. Drs. Wun, Lane and Ware will continue to address the issue of ICD 9 codes and 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

    I. Ms. Sonya Ross will report back from SCDAA on the idea of yearly joint meetings in the future.


    Peter Lane, M.D.


    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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