Pediatric Sickle Cell Program

SUNY - Health Science Center @ Brooklyn

Children's Medical Center of Brooklyn, Brooklyn, New York

For many years we at SUNY have advocated a comprehensive approach to health education and treatment of children with sickle cell disease. Each year, 15-30 infants are newly diagnosed at our institution and introduced to comprehensive care. By offering "well-child" care in addition to sickle cell-specific counseling and education, we (contact members of our staff) have succeeded in substantially improving the health and survival of our children. Continued provision of comprehensive sickle cell care in an era of managed care and "down-sizing" is a major challenge to all urban health care providers.

SUNY-Brooklyn has participated in the Cooperative Study of Sickle Cell Disease, a project designed to document the "natural history" of sickle cell disease sponsored by the Heart, Lung and Blood Institute of the National Instates of Health, since 1978. A review of some of the goals and accomplishments of the CSSCD has been distributed to parents and participants, and numerous important publications have resulted from the efforts of all involved. Recently, CSSCD investigators have reported that early manifestations of sickle cell disease may be used to predict severe complications later in childhood.  It is hoped that awareness of these factors will allow doctors to determine which children may most appropriately be considered for, or excluded from, a number of recently available, but potentially dangerous, treatments.  In the past, SUNY researchers have contributed to a number of publications related to sickle cell disease; Dr.'s Margaret Robinson and Janet Watson were the first to describe the increased susceptibility of individuals with sickle cell disease to pneumococcal meningitis. Click for a list of publications by current faculty.

We were one of 14 institutions to participate in The Stroke Prevention Trial (STOP) sponsored by the Medical College of Ga. at Augusta. The study was stopped early, on September 2, 1997, when it became clear that regular blood transfusion does prevent stroke in children with sickle cell anemia found to be at high risk due to an abnormal transcranial doppler (TCD) study. Details are provided in a news release and Clinical Alert from the Heart, Lung and Blood Institute of the NIH. It is important to understand that at present TCD must be performed at a site qualified to do the Doppler studies as they were done for STOP, and that physicians familiar with STOP results be involved in treatment discussions and recommendations. All of the STOP centers, including our own, are able to assist and advise and are listed in the links above. There is optimism that a second STOP study is imminent, which will determine whether or not transfusion therapy might safely be stopped in some children who achieve normal TCD studies while receiving transfusions.  The NIH has posted some guidelines for transfusion and a brief review of risks which may be of interest.

For over 20 years, the Wadsworth Center has been screening all newborns in New York State for sickle cell disease; newborn screening results are available to licensed physicians in NY State by calling 1-800-535-3079. Information about various abnormal hemoglobins encountered can be accessed at the Globin Gene Server Homepage.  See also the Human Genome Project for information about sickle cell disease, updates as to genome mapping progress, and ethical considerations related to genetic research.

Another important resource supported by the NY State Department of Health is the NY State Sickle Cell Advisory Committee, which works in many ways to address concerns of the sickle cell community. It is a standing committee of GENES, the Genetic Network for New York State, Puerto Rico, and the Virgin Islands, a federally designated region that is funded by the federal government to coordinate genetic services and provide education about genetic disorders to medical professionals and the lay population. GENES is one of ten regions represented by CORN (the Council of Regional Networks for Genetics Services).

An excellent reference as to care of children with sickle cell disease is available to review or download (Sickle Cell Disease (Clinical Guidelines) NIH, MCHB); additional Guidelines related to other issues, including pain management, are also available. An updated Black Book, Management and Therapy of Sickle Cell Disease, was published (in orange and purple), but additional printings are on hold. A modified, maneuverable text version is available here, and a PDF or text download is offered by the NIH/NHLBI. Finally, there is an online summary of the medical needs of infants with sickle cell disease, Consumer Guide 6: Sickle Cell Disease, that can be read by parents. For additional information about the Black Book or other sickle cell information call the sickle cell branch of the NHLBI by  Comprehensive Sickle Cell Centers phone at 301-435-0055, send email to warew@gwgate.nhlbi.nih.gov, or try the developing Sickle Cell Centers web site.

New York state is in the process of requiring that all Medicaid recipients enroll with a managed care organization (MCO) to continue to receive benefits; if families don't pick a provider within 60 days of being requested to do so, an MCO will be assigned by the State.   It is important that parents of children with sickle cell disease or other chronic medical conditions be certain that managed care providers they are considering can provided the kind of sickle cell care they expect and require.  The Childrens Hospital at Oakland as posted some recommendations regarding choosing a provider.  As we become progressively challenged by bureaucratic demands, it may be helpful to healthcare providers to have access to ICD-9-CM International (Disease) Codes; sickle cell anemia is 282.6. Thanks to the anonymous provider.

Information related to new treatments for sickle cell disease is also available online:

Pain and acute chest syndrome remain the most common complication of sickle cell disease. Hydroxyurea was effective in reducing both complications in adults with moderate to severe sickle cell anemia.  Questions and answers about the Multicenter Trial have been posted by the NIH/NHLBI.  We at SUNY currently offer hydroxyurea therapy to selected children with severe disease manifestations, and although we are concerned about long-term risks of treatment, recent studies suggest safety and efficacy in children.  A trial of hydroxyurea in infants with sickle cell disease has been proposed to determine whether early implementation of therapy might prevent damage to brain, spleen, lungs, kidney and other organs which are early targets of sickle cells.  The NIH/NHLBI has also suggested guidelines for use of hydroxyurea in children.

Bone marrow transplantation can cure sickle cell disease (NEJM article), but is associated with substantial risk and is still experimental. Children who have suffered severe complications and who have a compatible sibling donor might consider transplant. An exciting new alternative source of stem cells for potential transplantation is umbilical cord (placental) blood. Cord blood transplantation may be associated with lower risks of complications, and potentially will increase the possibility of finding a donor for an unrelated stem cell transplant; the NIH has recently supported several programs to routinely store cord blood. Mothers of children with sickle cell disease who are pregnant may want to discuss with their physician the possibility of storing cord (placental) blood at the time of delivery; the Childrens Hospital of Oakland is providing this service free to eligible, expectant mothers anywhere in the country who already have a child with sickle cell disease or other transplant-treatable condition.

While we do not advocate homeopathic alternatives to standard care, healthy life choices are important for individuals with sickle cell disease, and a wealth of information is available via Healthy.Net Home Page .

Joan Fleitas, Assistant Professor of Nursing at Fairfield University, has developed a page that will amuse and inform children, adults, parents and providers interested in sickle cell disease and other chronic conditions entitled Bandaides and Blackboards. Ivor and Deborah Pannell are friends of the SUNY program who are founders of S.C.A.R.E. (Sickle Cell Advocates for Research and Empowerment) and its web page at defiers.com. The page is an extremely comprehensive resource for those interested in sickle cell disease and recently has been expanded to include access to a sickle cell chat room and message board. Mr. Platt at Emory has expanded their sickle cell web page,  and we are soliciting ideas as to how to best utilize sickle cell.org.

The availability of beneficial but potentially dangerous therapies for sickle cell disease raises complicated ethical issues. An excellent series of review articles on several bioethical topics is provided on-line by the Canadian Medical Association Journal (link updated 9/98), and additional information and resources are available from the Medical College of Wisconsin.

This page is referenced in PedInfo , an enlarging database for pediatricians, geneticists and others. Pediatric hematologists should be aware of the web site by the American Society of Pediatric Hematology/Oncology, the American Society of Hematology, and Bloodline for meeting abstracts and relevant links. And you know about the London and New York Times.

Finally, years ago Brooklyn baseball fans were devastated by the loss of the Dodgers. Though they now have the Cyclones, a long-suffering Cleveland Indians fan (didn't it take the NY Rangers 49 years to win their most recent World Championship?) suggests Brooklynites might want to adopt the Tribe, particularly given their recent success. Or, perhaps, if you like to suffer, you may adopt the new Browns!  My Dad's old paper, the Plain Dealer is also finally online, for all displaced Clevelanders.

Copyright 1996, 1997, 1998 by Scott T. Miller