Biology of Blood and Marrow Transplantation
Volume 14, Issue 11 , Pages 1270-1278 , November 2008

Stable Long-Term Donor Engraftment following Reduced-Intensity Hematopoietic Cell Transplantation for Sickle Cell Disease

  • Lakshmanan Krishnamurti

      Affiliations

    • Children's Hospital of Pittsburgh, University of Pittsburgh Medical Center, Pittsburgh, Pennsylvania
    • Corresponding Author InformationCorrespondence and reprint requests: Lakshmanan Krishnamurti, M.D., Division of Hematology/Oncology/Bone Marrow Transplantation, Children's Hospital of Pittsburgh, 3705 Fifth Avenue, Pittsburgh, PA 15213.
  • ,
  • Sandhya Kharbanda

      Affiliations

    • University of Alabama, Birmingham, Alabama
  • ,
  • Melinda A. Biernacki

      Affiliations

    • Dana Farber Cancer Institute, Boston, Massachusetts
  • ,
  • Wandi Zhang

      Affiliations

    • Dana Farber Cancer Institute, Boston, Massachusetts
  • ,
  • K. Scott Baker

      Affiliations

    • University of Minnesota, Minneapolis, Minnesota
  • ,
  • John E. Wagner

      Affiliations

    • University of Minnesota, Minneapolis, Minnesota
  • ,
  • Catherine J. Wu

      Affiliations

    • Dana Farber Cancer Institute, Boston, Massachusetts

Received 9 July 2008 ,Accepted 27 August 2008.

References 

  1. Platt OS, Brambilla DJ, Rosse WF, et al. Mortality in SCD. Life expectancy and risk factors for early death. N Engl J Med. 1994;330:1639–1644
  2. Powars DR, Chan LS, Hiti A, Ramicone E, Johnson C. Outcome of sickle cell anemia: a 4-decade observational study of 1056 patients. Medicine (Baltimore). 2005;84:363–376
  3. Hagar RW, Vichinsky EP. Major changes in SCD. Adv Pediatr. 2000;47:249–272
  4. Steinberg MH. Hydroxyurea treatment for SCD. ScientificWorldJournal. 2002;2:1706–1728
  5. Platt OS. Hydroxyurea for the treatment of sickle cell anemia. N Engl J Med. 2008;358:1362–1369
  6. Abboud MR, Jackson SM, Barredo J, Beatty J, Laver J. Bone marrow transplantation for sickle cell anemia. Am J Pediatr Hematol Oncol. 1994;16:86–89
  7. Bernaudin F, Socie G, Kuentz M, et al. Long-term results of related, myeloablative stem cell transplantation to cure sickle cell disease. Blood. 2007;110:27549–2756
  8. Ferster A, De Valck C, Azzi N, Fondu P, Toppet M, Sariban E. Bone marrow transplantation for severe sickle cell anaemia. Br J Haematol. 1992;80:102–105
  9. Giardini C, Galimberti M, Lucarelli G, et al. Bone marrow transplantation in sickle-cell anemia in Pesaro. Bone Marrow Transplant. 1993;12:122–123
  10. Panepinto JA, Walters MC, Carreras J, et al. Matched-related donor transplantation for SCD: report from the Center for International Blood and Transplant Research. Br J Haematol. 2007;137:479–485
  11. Walters MC. Stem cell therapy for SCD: transplantation and gene therapy. Hematology Am Soc Hematol Educ Program. 2005;66–73
  12. Walters MC. Cord blood transplantation for sickle cell anemia: Bust or boom?. Pediatr Transplant. 2007;11:582–583
  13. Walters MC, Patience M, Leisenring W, et al. Bone marrow transplantation for SCD. N Engl J Med. 1996;335:369–376
  14. Walters MC, Storb R, Patience M, et al. Impact of bone marrow transplantation for symptomatic SCD: an interim report. Multicenter investigation of bone marrow transplantation for SCD. Blood. 2000;95:1918–1924
  15. 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
  16. Andersson BS, Kashyap A, Couriel D, et al. Intravenous busulfan in pretransplant chemotherapy: bioavailability and patient benefit. Biol Blood Marrow Transplant. 2003;9:722–724
  17. Mielcarek M, Martin PJ, Leisenring W, et al. Graft-versus-host disease after nonmyeloablative versus conventional hematopoietic stem cell transplantation. Blood. 2003;102:756–762
  18. Franzoni E, Sarajlija J, Garone C, Malaspina E, Marchiani V. No kinetic interaction between levetiracetam and cyclosporine: a case report. J Child Neurol. 2007;22:440–442
  19. Wu CJ, Krishnamurti L, Kutok JL, et al. Evidence for ineffective erythropoiesis in severe sickle cell disease. Blood. 2005;106:3639–3645
  20. Wu CJ, Hochberg EP, Rogers SA, et al. Molecular assessment of erythroid lineage chimerism following nonmyeloablative allogeneic stem cell transplantation. Exp Hematol. 2003;31:924–933
  21. Krishnamurti L, Blazar BR, Wagner JE. Bone marrow transplantation without myeloablation for SCD. N Engl J Med. 2001;344:68
  22. Andreani M, Manna M, Lucarelli G, et al. Persistence of mixed chimerism in patients transplanted for the treatment of thalassemia. Blood. 1996;87:3494–3499
  23. Krishnamurti L. Hematopoietic cell transplantation for SCD: state of the art. Expert Opin Biol Ther. 2007;7:161–172
  24. 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
  25. Horan JT, Liesveld JL, Fenton P, Blumberg N, Walters MC. Hematopoietic stem cell transplantation for multiply transfused patients with SCD and thalassemia after low-dose total body irradiation, fludarabine, and rabbit anti-thymocyte globulin. Bone Marrow Transplant. 2005;35:171–177
  26. 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
  27. Iannone R, Ohene-Frempong K, Fuchs EJ, Casella JF, Chen AR. Bone marrow transplantation for sickle cell anemia: progress and prospects. Pediatr Blood Cancer. 2005;44:436–440
  28. van Besien K, Bartholomew A, Stock W, et al. Fludarabine-based conditioning for allogeneic transplantation in adults with SCD. Bone Marrow Transplant. 2000;26:445–449
  29. Liu YP, Li Z, Nador RG, Strober S. Simultaneous protection against allograft rejection and graft-versus-host disease after total lymphoid irradiation: role of natural killer T cells. Transplantation. 2008;85:607–614
  30. Lowsky R, Takahashi T, Liu YP, et al. Protective conditioning for acute graft-versus-host disease. N Engl J Med. 2005;353:1321–1331
  31. Lan F, Zeng D, Higuchi M, Higgins JP, Strober S. Host conditioning with total lymphoid irradiation and antithymocyte globulin prevents graft-versus-host disease: the role of CD1-reactive natural killer T cells. Biol Blood Marrow Transplant. 2003;9:355–363
  32. Scandling JD, Busque S, Dejbakhsh-Jones S, et al. Tolerance and chimerism after renal and hematopoietic-cell transplantation. N Engl J Med. 2008;358:362–368
  33. Mazur M, Kurtzberg J, Halperin E, Ciocci G, Szabolcs P. Transplantation of a child with sickle cell anemia with an unrelated cord blood unit after RIC conditioning. J Pediatr Hematol Oncol. 2006;28:840–844
  34. Hongeng S, Pakakasama S, Chaisiripoomkere W, Ungkanont A, Jootar S. Nonmyeloablative stem cell transplantation with a haploidentical donor in a class 3 lucarelli severe thalassemia patient. Bone Marrow Transplant. 2004;34:271–272
  35. Hongeng S, Pakakasama S, Chuansumrit A, et al. RIC stem cell transplantation for treatment of class 3 Lucarelli severe thalassemia patients. Am J Hematol. 2007;82:1095–1098
  36. Schleuning M, Stoetzer O, Waterhouse C, Schlemmer M, Ledderose G, Kolb HJ. Hematopoietic stem cell transplantation after reduced-intensity conditioning as treatment of SCD. Exp Hematol. 2002;30:7–10
  37. Shenoy S, Grossman WJ, DiPersio J, et al. A novel reduced-intensity stem cell transplant regimen for nonmalignant disorders. Bone Marrow Transplant. 2005;35:345–352
  38. Horwitz ME, Spasojevic I, Morris A, et al. Fludarabine-based nonmyeloablative stem cell transplantation for SCD with and without renal failure: clinical outcome and pharmacokinetics. Biol Blood Marrow Transplant. 2007;13:1422–1426
  39. Baron F, Little MT, Storb R. Kinetics of engraftment following allogeneic hematopoietic cell transplantation with reduced-intensity or nonmyeloablative conditioning. Blood Rev. 2005;19:153–164
  40. Childs R, Clave E, Contentin N, et al. Engraftment kinetics after nonmyeloablative allogeneic peripheral blood stem cell transplantation: full donor T-cell chimerism precedes alloimmune responses. Blood. 1999;94:3234–3241
  41. Kean LS, Manci EA, Perry J, et al. Chimerism and cure: hematologic and pathologic correction of murine SCD. Blood. 2003;102:4582–4593
  42. Wu CJ, Gladwin M, Tisdale J, et al. Mixed haematopoietic chimerism for SCD prevents intravascular haemolysis. Br J Haematol. 2007;139:504–507
  43. Myers GD, Krance RA, Weiss H, et al. Adenovirus infection rates in pediatric recipients of alternate donor allogeneic bone marrow transplants receiving either antithymocyte globulin (ATG) or alemtuzumab (Campath). Bone Marrow Transplant. 2005;36:1001–1008
  44. Penack O, Fischer L, Stroux A, et al. Serotherapy with thymoglobulin and alemtuzumab differentially influences frequency and function of natural killer cells after allogeneic stem cell transplantation. Bone Marrow Transplant. 2008;41:377–383
  45. Deeg HJ, Socie G, Schoch G, et al. Malignancies after marrow transplantation for aplastic anemia and fanconi anemia: a joint Seattle and Paris analysis of results in 700 patients. Blood. 1996;87:386–392
  46. Inamoto Y, Suzuki R, Kuwatsuka Y, et al. Long-term outcome after bone marrow transplantation for aplastic anemia using cyclophosphamide and total lymphoid irradiation as conditioning regimen. Biol Blood Marrow Transplant. 2008;14:43–49
  47. Slavin S, Naparstek E, Aker M, et al. The use of total lymphoid irradiation (TLI) for prevention of rejection of T-lymphocyte depleted bone marrow allografts in non-malignant hematological disorders. Transplant Proc. 1989;21:3053–3054
  48. Flowers ME, Traina F, Storer B, et al. Serious graft-versus-host disease after hematopoietic cell transplantation following nonmyeloablative conditioning. Bone Marrow Transplant. 2005;35:277–282
  49. Petersen SL, Madsen HO, Ryder LP, et al. Chimerism studies in HLA-identical nonmyeloablative hematopoietic stem cell transplantation point to the donor CD8(+) T-cell count on day + 14 as a predictor of acute graft-versus-host disease. Biol Blood Marrow Transplant. 2004;10:337–346
  50. Storb RF, Lucarelli G, McSweeney PA, Childs RW. Hematopoietic cell transplantation for benign hematological disorders and solid tumors. Hematology Am Soc Hematol Educ Program. 2003;372–397

PII: S1083-8791(08)00383-2

doi: 10.1016/j.bbmt.2008.08.016

Biology of Blood and Marrow Transplantation
Volume 14, Issue 11 , Pages 1270-1278 , November 2008