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The Management of Sickle Cell Disease

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Division of Blood Diseases and Re s o u rc e s T H E M A N A G E M E N T O F S I C K L E C E L L D I S E A S E N A TIONAL INSTITUTES OF HEALT H N A T IONAL HEART, LUNG, AND BLOOD INSTITUTE T HE M ANAGEMENT OF S ICKLE C ELL D ISEASE NATIONAL INSTITUTES OF HEALTH National Heart, Lung, and Blood Institute Division of Blood Diseases and Resources NIH PUBLICATION NO. 02-2117 ORIGINALLY PRINTED 1984 PREVIOUSLY REVISED 1989, 1995 REPRINTED JUNE 1999 REVISED JUNE 2002 (FOURTH EDITION) II CONTENTS Preface . V Contributors . VII Introduction . 1 DIAGNOSIS AND COUNSELING 1. World Wide Web Resources. 5 2. Neonatal Screening . 7 3. Sickle Cell Trait. 15 4. Genetic Counseling . 19 HEALTH MAINTENANCE 5. Child Health Care Maintenance. 25 6. Adolescent Health Care and Transitions . 35 7. Adult Health Care Maintenance . 41 8. Coordination of Care: Role of Mid-Level Practitioners . 47 9. Psychosocial Management . 53 TREATMENT OF ACUTE AND CHRONIC COMPLICATIONS 10. Pain . 59 11. Infection. 75 12. Transient Red Cell Aplasia . 81 13. Stroke and Central Nervous System Disease . 83 14. Sickle Cell Eye Disease . 95 15. Cardiovascular Manifestations. 99 16. Acute Chest Syndrome and Other Pulmonary Complications . 103 17. Gall Bladder and Liver . 111 III 18. Splenic Sequestration. 119 19. Renal Abnormalities in Sickle Cell Disease. 123 20. Priapism . 129 21. Bones and Joints . 133 22. Leg Ulcers. 139 SPECIAL TOPICS 23. Contraception and Pregnancy . 145 24. Anesthesia and Surgery . 149 25. Transfusion, Iron Overload, and Chelation . 153 26. Fetal Hemoglobin Induction . 161 27. Hematopoietic Cell Transplantation . 167 28. Genetic Modulation of Phenotype by Epistatic Genes . 173 29. Highlights from Federally Funded Studies. 181 IV PREFACE Enclosed is the fourth edition of a book that iduals with sickle cell disease and provides is dedicated to the medical and social issues of relevant online resources at the end of the individuals with sickle cell disease. This publi- chapters, is to serve as an adjunct to recent cation, which was developed by physicians, textbooks that delve more deeply into all nurses, psychologists, and social workers who aspects of the disorder. specialize in the care of children and adults with sickle cell disease, describes the current The authors hope that this book will be used approach to counseling and also to manage- by medical students, house staff, general practi- ment of many of the medical complications tioners, specialists, nurses, social workers, psy- of sickle cell disease. chologists, and other professionals as well as the families and patients who are coping with Each chapter was prepared by one or more the complexities of sickle cell disease on a daily experts and then reviewed by several others basis. The book, any part of which can be in the field. Additional experts reviewed the copied freely, will be placed on the National entire volume. This book is not the result of a Heart, Lung, and Blood Institute (NHLBI) formalized consensus process but rather repre- Web site and will be updated as needed. sents the efforts of those who have dedicated their professional careers to the care of indi- Research is essential to provide the knowledge viduals with sickle cell disease. The names of required to improve the care of individuals the authors, their affiliations, and their e-mail with sickle cell disease, but it is the physicians addresses are listed in the front of the book. and other health care personnel who must ensure that the very best care is actually Multiple new therapies are now available for delivered to each child and adult who has children and adults with sickle cell disease, this disorder. We hope that this book will and often the options to be chosen present help to achieve this goal. a dilemma for both patients and physicians. This book does not provide answers to many of these newer questions but rather explains the choices available. The book, which focuses Claude Lenfant, M.D. primarily on the basic management of indiv- Director, NHLBI V VI CONTRIBUTORS Robert Adams, M.D. Henny Billett, M.D. Associate Professor, Department of Neurology Director, Clinical Hematology Room HB-2060 Albert Einstein College of Medicine Medical College of Georgia Comprehensive Sickle Cell Center Augusta, GA 30912 Montefiore Hospital Medical Center Tel: (706) 721-4670 111 East 210th Street Fax: (706) 721-6757 Bronx, NY 10467 E-mail: [email protected] Tel: (718) 920-7373 Fax: (718) 920-5095 Kenneth I. Ataga, M.D. E-mail: [email protected] Division of Hematology/Oncology E-mail: [email protected] CB# 7305, 3009 Old Clinic Building University of North Carolina at Chapel Hill Carine Boehme, M.S. Chapel Hill, NC 27599-7305 Associate Professor Tel: (919) 843-7708 The Johns Hopkins University School of Medicine E-mail: [email protected] CMSC Room 1004 600 North Wolfe Street Harold Ballard, M.D. Baltimore, MD 21287-9278 Assistant Chief, Hematology Division Tel: (410) 955-0483 New York Veterans Administration Fax: (410) 955-0484 Medical Services, 12th Floor E-mail: [email protected] New York, NY 10010 Tel: (212) 951-3484 Kenneth Bridges, M.D. Fax: (212) 951-5981 Associate Professor of Medicine Director, Joint Center for Sickle Cell Lennette Benjamin, M.D. and Thrombosis and Hemostasis Disorders Associate Professor of Medicine Brigham and Women’s Hospital Montefiore Hospital Medical Center Harvard Medical School Comprehensive Sickle Cell Center 75 Francis Street 111 East 210th Street Boston, MA 02115 Bronx, NY 10467-2490 Tel: (617) 732-5842 Tel: (718) 920-7375 Fax: (617) 975-0876 Fax: (718) 798-5095 E-mail: [email protected] E-mail: [email protected] VII Oswaldo Castro, M.D. Joseph DeSimone, Ph.D. Professor of Medicine/Pediatrics Geneticist Howard University School of Medicine VA West Side Medical Center Comprehensive Sickle Cell Center Hematology Research (151C) 2121 Georgia Avenue, NW 820 South Damen Avenue Washington DC 20059 Chicago, IL 60612 Tel: (202) 806-7930 Tel: (312) 666-6500 x2683 Fax: (202) 806-4517 Fax: (312) 455-5877 E-mail: [email protected] E-mail: [email protected] Samuel Charache, M.D. Ann Earles, R.N., P.N.P. 2006 South Road Research Nurse Baltimore, MD 21209-4510 Children’s Hospital of Oakland Tel: (410) 466-6405 747 52nd Street Fax: (410) 466-4330 Oakland, CA 94609-1809 E-mail: [email protected] Tel: (510) 428-3453 Fax: (510) 450-5635 Wesley Covitz, M.D. E-mail: [email protected] Professor of Pediatrics Wake Forest School of Medicine James Eckman, M.D. Medical Center Boulevard Professor of Medicine Winston-Salem, NC 27157-1081 Emory University School of Medicine Tel: (336) 716-4267 69 Butler Street Fax: (336) 716-0533 Atlanta, GA 30303 E-mail: [email protected] Tel: (404) 616-5982 Fax: (404) 577-9107 Gary R. Cutter, Ph.D. E-mail: [email protected] Professor of Medicine Director, Center for Research Design Morton Goldberg, M.D. and Statistical Methods Director, Department of Ophthalmology UNR School of Medicine, Mail Stop 199 The Johns Hopkins Hospital Reno, Nevada 89557 The Wilmer Ophthamological Institute Tel: (775) 784-1565 Maumanee Building, Room 729 Fax: (775) 784-1142 600 North Wolfe Street E-mail: [email protected] Baltimore, MD 21287-9278 Tel: (410) 955-6846 Carlton Dampier, M.D. Fax: (410) 955-0675 Director, Marian Anderson SCC E-mail: [email protected] Hematology/Oncology St. Christopher’s Hospital for Children Harry E. Jergesen, M.D. Erie Avenue at Front Street Department of Orthopaedic Surgery Philadelphia, PA 19134 University of California, San Francisco Tel: (215) 427-5096 500 Parnassus Avenue, MU-320W Fax: (215) 427-6684 San Francisco, CA 94143 E-mail: [email protected] Tel: (415) 476-8938 Fax: (415) 476-1301 E-mail: [email protected] VIII Cage Johnson, M.D. Peter Lane, M.D. Professor of Medicine Director, Colorado Sickle Cell Treatment University of Southern California and Research Center 2025 Zonal Avenue Campus Box C-222 Los Angeles, CA 90033 University of Colorado Health Sciences Center Tel: (323) 442-1259 4200 East Ninth Avenue Fax: (323) 442-1255 Denver, CO 80262 E-mail: [email protected] Tel: (303) 372-9070 Fax: (303) 372-9161 Adrena Johnson-Telfair, P.A.C. E-mail: [email protected] Associate Director for Clinical Services University of Alabama at Birmingham Dimitris Loukopoulos, M.D., D.Sci. Comprehensive Sickle Cell Center First Department of Medicine 1900 University Boulevard University of Athens School of Medicine 513 Tinsley Harrison Laikon Hospital Birmingham, AL 35294-0006 Athens 11527 GREECE Tel: (205) 975-2281 Tel: +30 1 7771 161 Fax: (205) 975-5264 Fax: +30 1 7295 065 E-mail: [email protected] E-mail: [email protected] Clinton Joiner, M.D., Ph.D. Bertram Lubin, M.D. Associate Professor of Pediatrics Director of Medical Research Director, Comprehensive Sickle Cell Center Children’s Hospital Oakland Research Institute Children’s Hospital Medical Center 5700 Martin Luther King Junior Way 3333 Burnett Avenue, OSB 4 Oakland, CA 94609 Cincinnati, OH 45229-3039 Tel: (510) 450-7601 Tel: (513) 636-4541 Fax: (510) 450-7910 Fax: (513) 636-5562 E-mail: [email protected] E-mail: [email protected] Elysse Mandell, M.S.N. John Kark, M.D. Division of Hematology Professor, Hematology/Oncology Brigham and Women’s Hospital Howard University Hospital 75 Francis Street 5145 Tower Building Boston, MA 02115 2041 Georgia Avenue, NW Tel: (617) 732-8485 Washington, DC 20060 Fax: (617) 975-0876 Tel: (202) 865-1511 E-mail: [email protected] Fax: (202) 865-4607 E-mail: [email protected] Vipul Mankad, M.D.
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