Biology of Blood and Marrow Transplantation
Volume 10, Issue 1 , Pages 23-31 , January 2004

Will developments in allogeneic transplantation influence treatment of adult patients with sickle cell disease?

  • Suparno Chakrabarti

      Affiliations

    • Corresponding Author InformationCorrespondence and reprint requests: Suparno Chakrabarti, MD, Department of Haematology, Birmingham Children’s HospitalBirmingham, Edgbaston, Birmingham B4 6NH, United Kingdom
    • Cancer Research UK Institute for Cancer Studies, University of Birmingham, Birmingham, United Kingdom
    • Department of Haematology, Birmingham Children’s Hospital, Birmingham, United Kingdom
  • ,
  • David Bareford

      Affiliations

    • Regional Sickle Cell and Thalassemia Centre, City Hospital, Birmingham, United Kingdom

Received 20 August 2003 ,Accepted 15 September 2003.

References 

  1. 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
  2. Platt OS, Brambilla DJ, Rosse WF, et al.  Mortality in sickle cell disease. Life expectancy and risk factors for early death. N Engl J Med. 1994;330:1639–1644
  3. Perronne V, Roberts-Harewood M, Bachir D, et al.  Patterns of mortality in sickle cell disease in adults in France and England. Hematol J. 2002;3:56–60
  4. Steinberg MH, Barton F, Castro O, et al.  Effect of hydroxyurea on mortality and morbidity in adult sickle cell anemia (risks and benefits up to 9 years of treatment). JAMA. 2003;289:1645–1651
  5. Johnson FL, Look AT, Gockerman J, et al.  Bone marrow transplantation in a patient with sickle cell anemia. N Engl J Med. 1984;311:780–783
  6. Walters MC, Storb R, Patience M, 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
  7. Vermylen C, Cornu G, Ferster 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
  8. Bernaudin F, Vernant JP, Vilmer E, et al.  Results of myeloablative allogeneic stem cell transplant for severe sickle cell disease in France. Blood. 2002;100(suppl 1):5a
  9. Walters MC, Sullivan KM, Bernaudin F, et al.  Neurologic complications after allogeneic marrow transplantation for sickle cell anemia. Blood. 1995;85:879–884
  10. Mentzer WC, Heller S, Pearle PR, Hackney E, Vichinsky E. Availability of related donors for bone marrow transplantation in sickle cell anemia. Am J Pediatr Hematol Oncol. 1994;16:27–29
  11. Walters MC, Patience M, Leisenring W, et al.  Barriers to bone marrow transplantation for sickle cell anemia. Biol Blood Marrow Transplant. 1996;2:100–104
  12. Kean LS, Durham MM, Adams AB, et al.  A cure for murine sickle cell disease through stable mixed chimerism and tolerance induction after nonmyeloablative conditioning and major histocompatibility complex-mismatched bone marrow transplantation. Blood. 2002;99:1840–1849
  13. McSweeney PA, Storb R. Mixed chimerism (preclinical studies and clinical applications). Biol Blood Marrow Transplant. 1999;5:192–203
  14. McSweeney PA, Niederwieser D, Shizuru JA, et al.  Hematopoietic cell transplantation in older patients with hematologic malignancies (replacing high-dose cytotoxic therapy with graft-versus-tumor effects). Blood. 2001;97:3390–3400
  15. Giralt S, Thall PF, Khouri I, et al.  Melphalan and purine analog-containing preparative regimens (reduced-intensity conditioning for patients with hematologic malignancies undergoing allogeneic progenitor cell transplantation). Blood. 2001;97:631–637
  16. Slavin S, Nagler A, Naparstek E, et al.  Nonmyeloablative stem cell transplantation and cell therapy as an alternative to conventional bone marrow transplantation with lethal cytoreduction for the treatment of malignant and nonmalignant hematologic diseases. Blood. 1998;91:756–763
  17. Graido-Gonzalez E, Doherty JC, Bergreen EW, et al.  Plasma endothelin-1, cytokine, and prostaglandin E2 levels in sickle cell disease and acute vaso-occlusive sickle crisis. Blood. 1998;92:2551–2555
  18. Ferrera JLM, Antin JH. Pathophysiology of graft-versus-host disease. In:  Thomas ED,  Blume KG,  Forman SJ editor. Haematopoetic Stem Cell Transplantation. Boston: Blackwell Scientific Publications; 1999;p. 305–315
  19. Easaw SJ, Lake DE, Beer M, et al.  Graft-versus-host disease (Possible higher risk for African American patients). Cancer. 1996;78:1492–1497
  20. Klingemann HG, Deeg HJ, Self S, Thomas ED, Storb R. Is race a risk factor for allogeneic marrow transplantation?. Bone Marrow Transplant. 1986;1:87–94
  21. Walters MC, 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
  22. Iannone R, Luznik L, Engstrom LW, et al.  Effects of mixed hematopoietic chimerism in a mouse model of bone marrow transplantation for sickle cell anemia. Blood. 2001;97:3960–3965
  23. Iannone R, Casella JF, Fuchs EJ, et al.  Results of minimally toxic nonmyeloablative transplantation in patients with sickle cell anemia and beta-thalassemia. Biol Blood Marrow Transplant. 2003;9:519–528
  24. van Besien K, Bartholomew A, Stock W, et al.  Fludarabine-based conditioning for allogeneic transplantation in adults with sickle cell disease. Bone Marrow Transplant. 2000;26:445–449
  25. van Besien K, Stock W, Smith S, et al.  Allogeneic stem cell transplantation with fludarabine melphalan and campath conditioning for adults with advanced sickle cell disease. Blood. 2002;100(suppl 1):430b
  26. Krishnamurti L, Blazar BR, Wagner JE. Bone marrow transplantation without myeloablation for sickle cell disease. N Engl J Med. 2001;344:68
  27. Schleuning M, Stoetzer O, Waterhouse C, Schlemmer M, Ledderose G, Kolb HJ. Hematopoietic stem cell transplantation after reduced-intensity conditioning as treatment of sickle cell disease. Exp Hematol. 2002;30:7–10
  28. Walters MC. Stem cell transplantation for sickle cell disease (how and when to intervene). In: Hematology (American Society of Haematology Education Program Book). 2002;p. 10–34
  29. Barrett J, Childs R. Non-myeloablative stem cell transplants. Br J Haematol. 2000;11:6–17
  30. Kottaridis PD, Milligan DW, Chopra R, et al.  In vivo CAMPATH-1H prevents graft-versus-host disease following nonmyeloablative stem cell transplantation. Blood. 2000;96:2419–2425
  31. Kreiter S, Winkelmann N, Schneider PM, et al.  Failure of sustained engraftment after non-myeloablative conditioning with low-dose TBI and T cell-reduced allogeneic peripheral stem cell transplantation. Bone Marrow Transplant. 2001;28:157–161
  32. Chakrabarti S, McDonald D, Milligan DW. T cell-depleted nonmyeloablative stem cell transplantation (what is the optimum balance between the intensity of host conditioning and the degree of T cell depletion of the graft?). Bone Marrow Transplant. 2001;28:313–314
  33. Chakrabarti S, Mackinnon S. The relevance of nonmyeloablative transplantation in developing countries (the lessons from the UK experience). Transplant Proc. 2002;35:172–173
  34. Chakrabarti S, Mackinnon S, Chopra R, et al.  High incidence of CMV infection after nonmyeloablative stem cell transplantation (potential role of CAMPATH-1H in delaying immune reconstitution). Blood. 2002;99:4357–4363
  35. Rebello P, Cwynarsky K, Varughese M, et al.  Pharmacokinetics of Campath-1H in bone marrow transplant patients. Cytotherapy. 2001;3:261–267
  36. Weiss L, Abdul-Hai A, Or R, Amir G, Poliiack A. Fludarabine in combination with cyclophosphamide decreases incidence of GVHD and maintains effective graft-versus-leukemia effect after allogeneic stem cell transplantation in murine lymphocytic leukaemia. Bone Marrow Transplant. 2003;31:11–15
  37. Lucarelli G, Andreani M, Angelucci E. The cure of the thalassemia with bone marrow transplantation. Bone Marrow Transplant. 2001;28(suppl 1):S11–S13
  38. van Besien K, Koshy M, Anderson-Shaw L, et al.  Allogeneic stem cell transplantation for sickle cell disease (A study of patients’ decisions). Bone Marrow Transplant. 2001;28:545–549
  39. Ballas SK. Iron overload is a determinant of morbidity and mortality in adult patients with sickle cell disease. Semin Hematol. 2001;38(suppl 1):30–36
  40. La Nasa G, Giardini C, Argiolu F, et al.  Unrelated donor bone marrow transplantation for thalassemia (the effect of extended haplotypes). Blood. 2002;99:4350–4356
  41. 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
  42. Barker JN, Wagner JE. Umbilical cord blood transplantation (current state of the art). Curr Opin Oncol. 2002;14:160–164
  43. Aversa F, Tabilio A, Velardi A, et al.  Treatment of high-risk acute leukemia with T-cell-depleted stem cells from related donors with one fully mismatched HLA haplotype. N Engl J Med. 1998;339:1186–1193
  44. Ruggeri L, Capanni M, Urbani E, et al.  Effectiveness of donor natural killer cell alloreactivity in mismatched hematopoietic transplants. Science. 2002;295:2097–2100
  45. Ruggeri L, Capanni M, Tosti A, et al.  Natural killer cells (biology and application in stem-cell transplantation). Cytotherapy. 2002;4:445–446
  46. Einsele H, Roosnek E, Rufer N, et al.  Infusion of cytomegalovirus (CMV)-specific T cells for the treatment of CMV infection not responding to antiviral chemotherapy. Blood. 2002;99:3916–3922
  47. Rooney CM, Smith CA, Ng CYC, et al.  Infusion of cytotoxic T cells for the prevention and treatment of Epstein-Barr virus-induced lymphoma in allogeneic transplant recipients. Blood. 1998;92:1549–1555
  48. Hsieh MH, Varadi G, Flomenberg N, Korngold R. Leucyl-leucine methyl ester-treated haploidentical donor lymphocyte infusions can mediate graft-versus-leukemia activity with minimal graft-versus-host disease risk. Biol Blood Marrow Transplant. 2002;8:303–315
  49. Shimazaki C, Ochiai N, Uchida R, et al.  Non-T-cell-depleted HLA haploidentical stem cell transplantation in advanced hematologic malignancies based on the feto-maternal michrochimerism. Blood. 2003;101:3334–3336
  50. Tanaka J, Asaka M, Imamura M. T-cell co-signalling molecules in graft-versus-host disease. Ann Hematol. 2000;79:283–290
  51. Blazar BR, Taylor PA, Noelle RJ, Vallera DA. CD4(+) T cells tolerized ex vivo to host alloantigen by anti-CD40 ligand (CD40L: CD154) antibody lose their graft-versus-host disease lethality capacity but retain nominal antigen responses. J Clin Invest. 1998;102:473–482
  52. Guinan EC, Boussiotis VA, Neuberg D, et al.  Transplantation of anergic histoincompatible bone marrow allografts. N Engl J Med. 1999;340:1704–1714
  53. Koc ON, Lazarus HM. Mesenchymal stem cells (heading into the clinic). Bone Marrow Transplant. 2001;27:235–239
  54. Panoskaltsis-Mortari A, Taylor PA, Rubin JS, et al.  Keratinocyte growth factor facilitates alloengraftment and ameliorates graft-versus-host disease in mice by a mechanism independent of repair of conditioning-induced tissue injury. Blood. 2000;96:4350–4356

PII: S1083-8791(03)00336-7

doi: 10.1016/j.bbmt.2003.09.004

Biology of Blood and Marrow Transplantation
Volume 10, Issue 1 , Pages 23-31 , January 2004