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Sickle Cell Disease

Outcome of hematopoietic cell transplantation in children with sickle cell disease, a single center's experience

Bone Marrow Transplantation volume 45, pages 895–900 (2010)Cite this article

Abstract

Multicenter trials have shown that hematopoietic cell transplantation (HCT) has an excellent outcome in children with sickle cell disease (SCD). As a single center, we performed a total of 11 transplants in 10 patients (6 males, 4 females) with SCD between 1997 and 2005. Eight patients had hemoglobin SS disease and two patients had HbSβ0 thalassemia. The median age of HCT was 10.1 (range 2.8–16.3) years. All donors were HLA-identical siblings; six patients received bone marrow (BM), two patients received mobilized peripheral blood, one patient received umbilical cord blood (UCB) and one patient received both UCB and BM from the same donor. Myeloablative conditioning regimen consisted of busulfan, horse antithymocyte globulin and cyclophosphamide. One patient had a gradual decrease in donor chimerism to 15% and subsequently received a second bone marrow transplant using a reduced intensity conditioning regimen consisting of alemtuzumab, fludarabine and melphalan leading to stable full engraftment. Currently, 9 out of 10 patients are alive with a median follow-up of 5.5 (range 2.9–11) years. As a single institution's experience with HCT in children with SCD, we report an excellent outcome, and a second HCT may be considered for patients with impending engraftment failure as a cure for SCD.

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References

  1. Platt OS, Brambilla DJ, Rosse WF, Milner PF, Castro O, Steinberg MH et al. Mortality in sickle cell disease. Life expectancy and risk factors for early death. N Engl J Med 1994; 23: 1639–1644.

    Article  Google Scholar 

  2. Leikin SL, Gallagher D, Kinney TR, Sloane D, Klug P, Rida W . Mortality in children and adolescents with sickle cell disease. Cooperative Study of Sickle Cell Disease. Pediatrics 1989; 84: 500–508.

    CAS  PubMed  Google Scholar 

  3. Serjeant GR . Natural history and determinants of clinical severity of sickle cell disease. Curr Opin Hematol 1995; 2: 103–108 Review.

    Article  CAS  PubMed  Google Scholar 

  4. Adams R, McKie V, Nichols F, Carl E, Zhang DL, McKie K et al. The use of transcranial ultrasonography to predict stroke in sickle cell disease. N Engl J Med 1992; 326: 605–610.

    Article  CAS  PubMed  Google Scholar 

  5. Hankins JS, Ware RE, Rogers ZR, Wynn LW, Lane PA, Scott JP et al. Long-term hydroxyurea therapy for infants with sickle cell anemia: the HUSOFT extension study. Blood 2005; 106: 2269–2275.

    Article  CAS  PubMed  PubMed Central  Google Scholar 

  6. Adams RJ, McKie VC, Hsu L, Files B, Vichinsky E, Pegelow C et al. Prevention of a first stroke by transfusions in children with sickle cell anemia and abnormal results on transcranial Doppler ultrasonography. N Engl J Med 1998; 339: 5–11.

    Article  CAS  PubMed  Google Scholar 

  7. Walters MC, Storb R, Patience M, Leisenring W, Taylor T, Sanders JE et al. Impact of bone marrow transplantation for symptomatic sickle cell disease: an interim report. Multicenter investigation of bone marrow transplantation for sickle cell disease. Blood 2000; 95: 1918–1924.

    CAS  PubMed  Google Scholar 

  8. Vermylen C, Cornu G, Ferster A, Brichard B, Ninane J, Ferrant A et al. Haematopoietic stem cell transplantation for sickle cell anaemia: the first 50 patients transplanted in Belgium. Bone Marrow Transplant 1998; 22: 1–6.

    Article  CAS  PubMed  Google Scholar 

  9. Bernaudin F, Socie G, Kuentz M, Chevret S, Duval M, Bertrand Y et al. Long-term results of related myeloablative stem-cell transplantation to cure sickle cell disease. Blood 2007; 110: 2749–2756 Epub 2 July 2007.

    Article  CAS  PubMed  Google Scholar 

  10. Walters MC, Sullivan KM, Bernaudin F, Souillet G, Vannier JP, Johnson FL et al. Neurologic complications after allogeneic marrow transplantation for sickle cell anemia. Blood 1995; 85: 879–884.

    CAS  PubMed  Google Scholar 

  11. Przepiorka D, Weisdorf D, Martin P, Klingemann HG, Beatty P, Hows J et al. 1994 Consensus Conference on Acute GVHD Grading. Bone Marrow Transplant 1995; 15: 825–828 (Review).

    CAS  PubMed  Google Scholar 

  12. Akpek G, Lee SJ, Flowers ME, Pavletic SZ, Arora M, Lee S et al. Performance of a new clinical grading system for chronic graft-versus-host disease: a multicenter study. Blood 2003; 102: 802–809.

    Article  CAS  PubMed  Google Scholar 

  13. Schoenberg JB, Beck GJ, Bouhuys A . Growth and decay of pulmonary function in healthy blacks and whites. Respir Physiol 1978; 33: 367–393.

    Article  CAS  PubMed  Google Scholar 

  14. Woodard P, Helton KJ, Khan RB, Hale GA, Phipps S, Wang W et al. Brain parenchymal damage after haematopoietic stem cell transplantation for severe sickle cell disease. Br J Haematol 2005; 129: 550–552.

    Article  PubMed  Google Scholar 

  15. Panepinto JA, Walters MC, Carreras J, Marsh J, Bredeson CN, Gale RP et al. Non-Malignant Marrow Disorders Working Committee, Center for International Blood and Marrow TranMatched-related donor transplantation for sickle cell disease: report from the Center for International Blood and Transplant Research. Br J Haematol 2007; 137: 479–485.

    Article  PubMed  Google Scholar 

  16. Gaziev J, Sodani P, Lucarelli G . Hematopoietic stem cell transplantation in thalassemia. Bone Marrow Transplant 2008; 42: S41.

    Article  PubMed  Google Scholar 

  17. Soliman AT, elZalabany M, Amer M, Ansari BM . Growth and pubertal development in transfusion-dependent children and adolescents with thalassaemia major and sickle cell disease: a comparative study. J Trop Pediatr 1999; 45: 23–30.

    Article  CAS  PubMed  Google Scholar 

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Authors and Affiliations

  1. Division of Pediatric Hematology/Oncology, University of Mississippi Medical Center, Jackson, MS, USA

    S Majumdar, Z Robertson, A Robinson, S Starnes, R Iyer & G Megason

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  1. S Majumdar
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  2. Z Robertson
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  3. A Robinson
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  4. S Starnes
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  5. R Iyer
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  6. G Megason
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Correspondence to G Megason.

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Majumdar, S., Robertson, Z., Robinson, A. et al. Outcome of hematopoietic cell transplantation in children with sickle cell disease, a single center's experience. Bone Marrow Transplant 45, 895–900 (2010). https://doi.org/10.1038/bmt.2009.244

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