Biology of Blood and Marrow Transplantation
Volume 16, Issue 1, Supplement , Pages S64-S67 , January 2010

Severe Sickle Cell Disease—Pathophysiology and Therapy

  • George Buchanan

      Affiliations

    • University of Texas Southwestern at Dallas, Texas
  • ,
  • Elliott Vichinsky

      Affiliations

    • Children's Hospital and Research Center at Oakland, Oakland, California
  • ,
  • Lakshmanan Krishnamurti

      Affiliations

    • Children's Hospital of Pittsburgh, University of Pittsburgh, Pittsburgh, Pennsylvania
  • ,
  • Shalini Shenoy

      Affiliations

    • Washington University School of Medicine and St. Louis Children's Hospital, St. Louis, Missouri
    • Corresponding Author InformationCorrespondence and reprint requests: Shalini Shenoy, MD, Washington University School of Medicine and St. Louis Children's Hospital, Box 8116, 1 Children's Place, St. Louis, MO 63110.

References 

  1. Miller ST, Sleeper LA, Pegalow CH, et al. Prediction of adverse outcomes in children with sickle cell disease. N Eng J Med. 2000;342:83–89
  2. Quinn C, Lee NJ, Shull EP, et al. Prediction of adverse outcomes in children with sickle cell anemia: a study of the Dallas Newbron Cohort. Blood. 2008;111:544–548
  3. Boyd J, Macklin EA, Strunk RC, et al. Asthma is associated with acute chest syndrome and pain in children with sickle cell anemia. Blood. 2006;108:2923–2927
  4. Gladwin M, Vichinsky E. Pulmonary complications of sickle cell disease. N Engl J Med. 2008;359:2254–2265
  5. Panepinto J, Walters MC, Carreras J, et al. Matched-related transplantation for sickle cell disease: report from the Center for International Blood and Transplant Research. Br J Haematol. 2007;137:479–485
  6. Walters M, Patience M, Leisenring W, et al. Stable mixed hematopoietic chimerism after bone marrow transplantation for sickle cell anemia. Biol Blood Marrow Transplant. 2001;7:665–673
  7. Locatelli F, Rocha V, Reed W, et al. Related umbilical cord blood transplantation in patients with thalassemia and sickle cell disease. Blood. 2003;101:2137–2143
  8. Ohene-Frempong K, Weiner S, Sleeper L. Cerebrovascular accidents in sickle cell disease rates and risk factors. Blood. 1998;91:288–294
  9. Scothorn D, Price C, Schwartz D. Risk of recurrent stroke in children with sickle cell disease receiving blood transfusion therapy for at least five years after initial stroke. J Peds. 2002;140:348–354
  10. Mentzer W, Heller S, Pearle PR, et al. Availability of related donors for bone marrow transplantation in sickle cell anemia. Am J Pediatr Hematol Oncol. 1994;16:27–29
  11. Krishnamurti L, Abel S, Maiers M, et al. Availability of unrelated donors for hematopoietic stem cell transplantation for hemoglobinopathies. Bone Marrow Transplant. 2003;31:547–550
  12. Walters M, Sullivan KM, Bernaudin F, et al. Neurologic complications after allogeneic marrow transplantation for sickle cell anemia. Blood. 1995;85:879–884
  13. Walters M, Quirolo L, Trachtenberg ET, et al. Sibling donor cord blood transplantation for thalassemia major: experience of the sibling donor cord blood program. Ann N Y Acad Sci. 2005;1054:206–213
  14. Shenoy S, Grossman WJ, DiPersioet J. A novel reduced-intensity stem cell transplant regimen for non-malignant disorders. Bone Marrow Transplant. 2005;35:345–352
  15. Krishnamurti L, Kharbanda S, Biernacki MA, et al. Stable long-term donor engraftment following reduced-intensity hematopoietic cell transplantation for sickle cell disease. Biol Blood Marrow Transplant. 2008;14:1270–1278
  16. Barker JN, Scaradavou A, Stevens CE, Rubinstein P. Analysis of 608 umbilical cord blood (UCB) transplants: HLA-Match is a critical determinant of transplant related mortality (TRM) in the post-engraftment period even in the absence of acute graft versus host disease (aGVHD). Blood. 2005;106:Abstract #303
  17. Eapen M, Horowitz MM, Klein JP, et al. Higher mortality after allogeneic peripheral blood transplantation compared with bone marrow in children and adolescents: the Histocompatibility and Alternate Stem Cell Source working Committee of the International Bone Marrow transplant Registry. J Clin Oncol. 2004;22:4872–4880
  18. Walters M, Patience M, Leisenring W, et al. Barriers to bone marrow transplantation for sickle cell anemia. Biol Blood Marrow Transplant. 1996;2:100–104
  19. Kodish E, Lantos J, Stocking C, et al. Bone marrow transplantation for sickle cell disease. A study of parents' decisions. N Eng J Med. 1991;325:1349–1353
  20. Powars DR, Chan LS, Hiti A, et al. Outcome of sickle cell anemia: a 4-decade observational study of 1056 patients. Medicine (Baltimore). 2005;84:363–376
  21. Aduloju S, Palmer S, Eckman J. Mortality in sickle cell patient transitioning from pediatric to adult program: 10 years grady comprehensive sickle cell center experience. Blood. 2008;112:Abstract #1426
  22. Ballas SK, Dampier D. Outcome of transitioning pediatric patients with sickle cell disease to adult programs. Blood. 2004;104:Abstract 3743
  23. Gladwin MT, Vichinsky E. Pulmonary complications of sickle cell disease. N Engl J Med. 2008;359:2254–2265
  24. Klings ES, Wyszynski DF, Nolanet VG, et al. Abnormal pulmonary function in adults with sickle cell anemia. Am J Respir Crit Care Med. 2006;173:1264–1269
  25. Guasch A, Navarrete J, Nass K, et al. Glomerular involvement in adults with sickle cell hemoglobinopathies: prevalence and clinical correlates of progressive renal failure. J Am Soc Nephrol. 2006;17:2228–2235
  26. Miller RG, Segal JB, Asharet BH, et al. High prevalence and correlates of low bone mineral density in young adults with sickle cell disease. Am J Hematol. 2006;81:236–241
  27. Strouse J, Haywood C, Lanzkron S. Predictors of in-hospital mortality and charges in sickle cell disease: results from the California Discharge Databases 1998-2005. Blood. 2007;110:Abstract #432
  28. McClish DK, Penberthy LT, Bovbjerg VE, et al. Health related quality of life in sickle cell patients: the PiSCES project. Health Qual Life Outcomes. 2005;3:50

 Financial disclosure: See Acknowledgments on page S66.

PII: S1083-8791(09)00457-1

doi: 10.1016/j.bbmt.2009.10.001

Biology of Blood and Marrow Transplantation
Volume 16, Issue 1, Supplement , Pages S64-S67 , January 2010