Bethesda, Maryland

November 4, 2002


Dr. Gilda Barabino, Dr. Oswaldo Castro, Dr. Peter Lane, Dr. Russell Ware, Dr. Joseph Telfair, Dr. Theodore Wun


J. Hoxi Jones-Carranza, Dr. Marie Stuart, Dr. Meiselman


Dr. Marie_Mann, Dr. Robert Sheffler


Dr. William Hannon, Dr. Joseph Desimone


Dr. Herman Branson, DBDR/BDP, Dr. Duane Bonds, DBDR/SCDSRG; Dr. Gillian Engelson, HRSA, Dr. Gregory Evans, DBDR/SCDSRG; Dr. John Kark, Howard University, Dr. Charles Peterson, DBDR/BDP; Ms. Susan Pucie, DBDR/BDP; Ms. Sonya Ross, SCDAA, Dr. Paul Swerdlow, Wayne State University, Dr. Ellen Werner, DBDR/BDP; Dr. William P. Winter, Howard University

Executive Secretary - Dr. Charles M. Peterson

Secretary - Ms. Petronella A. Barrow

Dr. Charles Peterson called the meeting to order at 9:05 am and read the required notification regarding conflicts of interest and reminded those present to sign in on the required sign-in sheets.

  1. Dr. Peter Lane called for review of the minutes of June , 2002 and they were reviewed and approved.
  2. Dr. Lane welcomed the members and guests and gave the Chairman’s report. He noted that he had found it useful to attend the NHLBI Advisory Council Meeting for October as it clarified how initiatives are handled and reviewed by the Institute. He encouraged other members of the SCDAC to attend a Council Meeting if possible. The combined meeting in September of the Sickle Cell Disease Association of America was discussed. The Committee asked that a report be presented at the next meeting from SCDAA regarding the official feedback from the meeting, to be followed by a discussion about whether any changes might be recommended for future annual meetings. The general feeling of the group was that the meetings were highly successful and well received. Further discussion occurred on the Sickle Cell Disease Advisory Committee meeting format and way of doing business. The consensus was that the meeting organization worked well but it was recommended that the meetings begin at 8:30 AM. It was further recommended that a speaker in the area of psychosocial issues be scheduled and that an Orientation Fact Sheet be developed to be distributed to all new members that would describe the Division and the role of the SCDAC. The status of the American Academy of Pediatrics Universal Screening (for sickle cell disease) Initiative was reviewed. This work with financial support from HRSA is ongoing and Dr. Lane will continue to update the committee on any progress. Finally, the shortage of pneumococcal vaccine was noted. This shortage is of special concern to patients with sickle cell disease especially in view of the increasing prevalence of penicillin resistant organisms. The committee noted that the Prenvar shortage is a significant public health problem and recommended development and implementation of a system that preferentially ensures vaccine availability to sickle cell patients.
  3. Dr. Lane introduced the Scientific Presentations.

  4. A. Dr. Mark Gladwin spoke on the Pulmonary Complications of Sickle Cell Disease. His work has focused on the prevalence of pulmonary hypertension and the markedly increased risk for mortality that accompanies this diagnosis in persons with sickle cell disease.

    B. Dr. Russell Ware spoke on Secondary Stroke Prevention with Hydroxyurea. This approach combined with phlebotomy to remove iron overload resulting from transfusion therapy may have benefit for patients and appears worthy of a phase III clinical trial.

  5. 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. Dr. Helena Mishoe has left the Division to become Director of the Office of Minority Health Affairs for NHLBI. The committee requested that Dr. Mishoe be invited to the next meeting to review the status and function of her new Office. Dr. Morton Goldberg of Johns Hopkins has been contacted and will address the group at the next meeting on Sickle Cell Eye Disease with comparison to the eye disease seen in diabetes. Additional suggestions for topics for presentation were also solicited.
  6. Action Items from previous meeting were completed or discussed as follows:
  7. A. Dr. Swerdlow presented an initial proposal to change the ICD 9 codes for sickle cell disease and the sickle beta thalassemia disorders. The suggested codes would expand the 282.60 list of diagnoses and associated conditions to include SS, SC, S-beta thalassemia, S-other, Pain acute, Pain chronic, Acute Chest Syndrome, Splenic or Liver Sequestration and Acute Multiorgan Failure Syndrome. In addition it was noted that hemolytic leg ulcers and aplastic A “crisis” both need separate coding. Drs. Swerdlow, Ware, and Lane will continue these efforts to bring these issues to the attention of the AMA by very early in 2003.

    B. The BABY HUG Protocol Review Committee/Data and Safety Monitoring Board are still reviewing the protocol, age of participants, and considerations of therapeutic endpoints and potential toxicities. Since the protocol is still under review, this clinical trial is on hold.

    Dr. Bonds asked that a full report be postponed until the next meeting. Dr. Bonds will contact The Committee in December after speaking with Dr. Stuart and after getting a sense about the direction of the new center clinical trials.

    C. Dr. Marie Stuart was not present to review the two SC initiatives on Membrane Active Drugs and Hydroxyurea. This discussion will be postponed until the next meeting at which time discussion of these issues will have occurred in the Sickle Cell Centers Network.

    D. NHLBI will consider ways by which the utility of the CSSCD DNA resource can be prolonged and establish a minimum amount below which no more samples will be withdrawn. Dr. Bonds noted that the Institute is seeking advice on this issue from a number of sources including the convening of a workshop. It was recommended that Dr. Roy Wu from NCI also be contacted to see if they might have suggestions as to how the resource might be amplified. Meanwhile the Institute has several outstanding commitments to honor regarding samples distribution so the problem is increasing.

    E. Dr. Bonds noted that the initiative on Sickle Cell Disease and the Lung received a favorable rating from the Board of Extramural Advisors as well as Council and will be released soon. The letters of intent will be due January 24, 2003 and the applications due February 24, 2003.

  8. Agency Reports
  9. A. Health Resources and Services Administration: Dr. Marie Mann discussed the new program in newborn screening with $4 million targeted towards sickle cell disease. This program involves follow up and counseling programs.

    B. 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 issue was still under consideration.

  10. Update on Program Activities
  11. A. Comprehensive Sickle Cell Centers Renewal Progress: Dr. Evans noted that the new funding cycle for the Centers will begin in April, 2003. The Clinical Trials Network that is part of the Centers Program will be meeting December 5, 2002 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, and he asked The Committee to provide a list of priorities for clinical trials and suggested that Dr. Lane attend the December 5th meeting as an observer.

    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. Additional RFAs are under consideration by the Institute.

    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 under consideration by a newly constituted 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. The investigator group has demonstrated 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. The plans for the next 5 years include obtaining studies to ascertain the prevalence of pulmonary hypertension in subjects on hydroxyurea continuously vs. patients who never took hydroxyurea for the past 10 years. In addition, offspring neurodevelopmental follow-up studies are planned for the extension.

    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 is scheduled to end with the current funding cycle of the Comprehensive Sickle Cell Centers in April 2003.

    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 was presented at the SCDAA Combined Meetings in September. There are a number of follow up activities planned including the recently completed consumers working group on quality of life issues. 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.

  12. Dates of Next Meetings
  13. June 2, 2003

    November 3, 2003

  14. Summary List of Action Items
  15. A. A report will be sought from SCDAA on the September Meeting feedback with a discussion about potential improvements.

    B. The next meeting of the Committee will begin at 8:30 AM. Minutes from the previous meeting and the draft agenda will be distributed ahead of time. A fact sheet will be developed detailing the Committee’s function and the Division for inclusion in the packet for each meeting.

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

    D. A speaker will be sought who can address the issue of psychosocial research in Sickle Cell Disease.

    E. Drs. Swerdlow, Lane and Ware will continue to address the issue of ICD 9 codes and sickle cell disease with the goal of contacting the AMA with suggestions early in 2003.

    F. Dr. Bonds will contact The Committee in December regarding the two proposed RFAs.


    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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