Hematopoietic stem cell transplantation for patients with sickle cell disease. Progress and future directions

Hematology/Oncology Clinics of North America

Volumn 28, Issue 6, 2014, Pages 1171-1185

  Fitzhugh, Courtney D ^a   Abraham, Allistair A ^b   Tisdale, John F ^a   Hsieh, Matthew M ^a  

^a George Washington University School of Medicine and Health Sciences ^*  (United States)

^b GEORGE WASHINGTON UNIVERSITY SCHOOL OF MEDICINE AND HEALTH SCIENCES   (United States)

Author keywords

Cord blood (CB) donor; Haploidentical donor; Matched sibling donor; Matched unrelated donor; Sickle cell disease

Indexed keywords

ACUTE GRAFT VERSUS HOST DISEASE; CLINICAL EVALUATION; CORD BLOOD STEM CELL TRANSPLANTATION; DISEASE FREE SURVIVAL; GENE THERAPY; HEMATOPOIETIC STEM CELL; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HLA MATCHING; HUMAN; MORTALITY; REVIEW; SIBLING RELATION; SICKLE CELL ANEMIA; TREATMENT INDICATION; UNRELATED DONORS; ALLOTRANSPLANTATION; ANEMIA, SICKLE CELL; FORECASTING; HISTOCOMPATIBILITY TEST; PROCEDURES; SURVIVAL; TREATMENT OUTCOME; TRENDS;

ANEMIA, SICKLE CELL; CORD BLOOD STEM CELL TRANSPLANTATION; FORECASTING; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HISTOCOMPATIBILITY TESTING; HUMANS; SURVIVAL ANALYSIS; TRANSPLANTATION, HOMOLOGOUS; TREATMENT OUTCOME; UNRELATED DONORS;

EID: 84912059866     PISSN: 08898588     EISSN: 15581977     Source Type: Journal    
DOI: 10.1016/j.hoc.2014.08.014     Document Type: Review

References (63)

1. Johnson F.L., Look A.T., Gockerman J., et al. Bone-marrow transplantation in a patient with sickle-cell anemia. NEngl J Med 1984, 311(12):780-783.
2. Vermylen C., Fernandez Robles E., Ninane J., et al. Bone marrow transplantation in five children with sickle cell anaemia. Lancet 1988, 1(8600):1427-1428.
3. Vermylen C., Cornu G. Bone marrow transplantation for sickle cell disease. The European experience. Am J Pediatr Hematol Oncol 1994, 16(1):18-21.
4. Ferster A., De Valck C., Azzi N., et al. Bone marrow transplantation for severe sickle cell anaemia. Br J Haematol 1992, 80(1):102-105.
5. Johnson F.L., Mentzer W.C., Kalinyak K.A., et al. Bone marrow transplantation for sickle cell disease. The United States experience. Am J Pediatr Hematol Oncol 1994, 16(1):22-26.
6. Walters M.C., Patience M., Leisenring W., et al. Bone marrow transplantation for sickle cell disease. NEngl J Med 1996, 335(6):369-376.
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(7):2749-2756.
8. Hsieh M.M., Fitzhugh C.D., Weitzel R., et al. Nonmyeloablative HLA-matched sibling allogeneic hematopoietic stem cell transplantation for severe sickle cell phenotype. JAMA 2014, 312(1):48-56.
9. Brand A., Doxiadis I.N., Roelen D.L. On the role of HLA antibodies in hematopoietic stem cell transplantation. Tissue Antigens 2013, 81(1):1-11.
10. Ciurea S.O., Thall P.F., Wang X., et al. Donor-specific anti-HLA Abs and graft failure in matched unrelated donor hematopoietic stem cell transplantation. Blood 2011, 118(22):5957-5964.
11. Takanashi M., Atsuta Y., Fujiwara K., et al. The impact of anti-HLA antibodies on unrelated cord blood transplantations. Blood 2010, 116(15):2839-2846.
12. Thomas E.D., Buckner C.D., Sanders J.E., et al. Marrow transplantation for thalassaemia. Lancet 1982, 2(8292):227-229.
13. Lucarelli G., Galimberti M., Polchi P., et al. Marrow transplantation in patients with advanced thalassemia. NEngl J Med 1987, 316(17):1050-1055.
14. McPherson M.E., Hutcherson D., Olson E., et al. Safety and efficacy of targeted busulfan therapy in children undergoing myeloablative matched sibling donor BMT for sickle cell disease. Bone Marrow Transplant 2011, 46(1):27-33.
15. Maheshwari S., Kassim A., Yeh R.F., et al. Targeted busulfan therapy with a steady-state concentration of 600-700 ng/mL in patients with sickle cell disease receiving HLA-identical sibling bone marrow transplant. Bone Marrow Transplant 2014, 49(3):366-369.
16. Walters M.C., Patience M., Leisenring W., et al. Stable mixed hematopoietic chimerism after bone marrow transplantation for sickle cell anemia. Biol Blood Marrow Transplant 2001, 7(12):665-673.
17. Iannone R., Casella J.F., Fuchs E.J., et al. Results of minimally toxic nonmyeloablative transplantation in patients with sickle cell anemia and beta-thalassemia. Biol Blood Marrow Transplant 2003, 9(8):519-528.
18. 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(4):445-449.
19. Locatelli F., Kabbara N., Ruggeri A., et al. Outcome of patients with hemoglobinopathies given either cord blood or bone marrow transplantation from an HLA-identical sibling. Blood 2013, 122(6):1072-1078.
20. Locatelli F., Rocha V., Reed W., et al. Related umbilical cord blood transplantation in patients with thalassemia and sickle cell disease. Blood 2003, 101(6):2137-2143.
21. Soni S., Boulad F., Cowan M.J., et al. Combined umbilical cord blood and bone marrow from HLA-identical sibling donors for hematopoietic stem cell transplantation in children with hemoglobinopathies. Pediatr Blood Cancer 2014, 61:1690-1694.
22. Dedeken L., Le P.Q., Azzi N., et al. Haematopoietic stem cell transplantation for severe sickle cell disease in childhood: a single centre experience of 50 patients. Br J Haematol 2014, 165(3):402-408.
23. Gooley T.A., Chien J.W., Pergam S.A., et al. Reduced mortality after allogeneic hematopoietic-cell transplantation. NEngl J Med 2010, 363(22):2091-2101.
24. Hahn T., McCarthy P.L., Hassebroek A., et al. Significant improvement in survivalafter allogeneic hematopoietic cell transplantation during a period of significantly increased use, older recipient age, and use of unrelated donors. JClin Oncol 2013, 31(19):2437-2449.
25. Ballen K.K., Gluckman E., Broxmeyer H.E. Umbilical cord blood transplantation: the first 25 years and beyond. Blood 2013, 122(4):491-498.
26. Cavazzana-Calvo M., Andre-Schmutz I., Fischer A. Haematopoietic stem cell transplantation for SCID patients: where do we stand?. Br J Haematol 2013, 160(2):146-152.
27. Krishnamurti L., Abel S., Maiers M., et al. Availability of unrelated donors for hematopoietic stem cell transplantation for hemoglobinopathies. Bone Marrow Transplant 2003, 31(7):547-550.
28. Justus D, Perez E, Dioguardi J, etal. Allogeneic donor availability for hematopoietic stem cell transplantation in patients with sickle cell disease. World Cord Blood Congress IV and Innovative Therapies for Sickle Cell Disease. Monaco, October 24-27, 2013.
29. Mynarek M., Bettoni da Cunha Riehm C., Brinkmann F., et al. Normalized transcranial Doppler velocities, stroke prevention and improved pulmonary function after stem cell transplantation in children with sickle cell anemia. Klin Padiatr 2013, 225(3):127-132.
30. Kharbanda S., Smith A.R., Hutchinson S.K., et al. Unrelated donor allogeneic hematopoietic stem cell transplantation for patients with hemoglobinopathies using a reduced-intensity conditioning regimen and third-party mesenchymal stromal cells. Biol Blood Marrow Transplant 2014, 20(4):581-586.
31. La Nasa G., Giardini C., Argiolu F., et al. Unrelated donor bone marrow transplantation for thalassemia: the effect of extended haplotypes. Blood 2002, 99(12):4350-4356.
32. La Nasa G., Argiolu F., Giardini C., et al. Unrelated bone marrow transplantation for beta-thalassemia patients: the experience of the Italian Bone Marrow Transplant Group. Ann N Y Acad Sci 2005, 1054:186-195.
33. Li C., Wu X., Feng X., et al. Anovel conditioning regimen improves outcomes in beta-thalassemia major patients using unrelated donor peripheral blood stem cell transplantation. Blood 2012, 120(19):3875-3881.
34. Ruggeri A., Eapen M., Scaravadou A., et al. Umbilical cord blood transplantation for children with thalassemia and sickle cell disease. Biol Blood Marrow Transplant 2011, 17(9):1375-1382.
35. Luznik L., Engstrom L.W., Iannone R., et al. Posttransplantation cyclophosphamide facilitates engraftment of major histocompatibility complex-identical allogeneic marrow in mice conditioned with low-dose total body irradiation. Biol Blood Marrow Transplant 2002, 8(3):131-138.
36. Luznik L., O'Donnell P.V., Symons H.J., et al. HLA-haploidentical bone marrow transplantation for hematologic malignancies using nonmyeloablative conditioning and high-dose, posttransplantation cyclophosphamide. Biol Blood Marrow Transplant 2008, 14(6):641-650.
37. Munchel A.T., Kasamon Y.L., Fuchs E.J. Treatment of hematological malignancies with nonmyeloablative, HLA-haploidentical bone marrow transplantation and high dose, post-transplantation cyclophosphamide. Best Pract Res Clin Haematol 2011, 24(3):359-368.
38. Bolanos-Meade J., Fuchs E.J., Luznik L., et al. HLA-haploidentical bone marrow transplantation with posttransplant cyclophosphamide expands the donor pool for patients with sickle cell disease. Blood 2012, 120(22):4285-4291.
39. Dallas M.H., Triplett B., Shook D.R., et al. Long-term outcome and evaluation of organ function in pediatric patients undergoing haploidentical and matched related hematopoietic cell transplantation for sickle cell disease. Biol Blood Marrow Transplant 2013, 19(5):820-830.
40. 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):1-6.
41. Ciurea S.O., de Lima M., Cano P., et al. High risk of graft failure in patients with anti-HLA antibodies undergoing haploidentical stem-cell transplantation. Transplantation 2009, 88(8):1019-1024.
42. Gladstone D.E., Zachary A.A., Fuchs E.J., et al. Partially mismatched transplantation and human leukocyte antigen donor-specific antibodies. Biol Blood Marrow Transplant 2013, 19(4):647-652.
43. Yoshihara S., Taniguchi K., Ogawa H., et al. The role of HLA antibodies in allogeneic SCT: is the 'type-and-screen' strategy necessary not only for blood type but also for HLA?. Bone Marrow Transplant 2012, 47(12):1499-1506.
44. de Lima M., McNiece I., Robinson S.N., et al. Cord-blood engraftment with exvivo mesenchymal-cell coculture. NEngl J Med 2012, 367(24):2305-2315.
45. Horwitz M.E., Chao N.J., Rizzieri D.A., et al. Umbilical cord blood expansion with nicotinamide provides long-term multilineage engraftment. JClin Invest 2014, 124:3121-3128.
46. Cavazzana-Calvo M., Payen E., Negre O., et al. Transfusion independence and HMGA2 activation after gene therapy of human beta-thalassaemia. Nature 2010, 467(7313):318-322.
47. Panepinto J.A., Walters M.C., Carreras J., et al. Matched-related donor transplantation for sickle cell disease: report from the Center for International Blood and Transplant Research. Br J Haematol 2007, 137(5):479-485.
48. 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 2010, 45(5):895-900.
49. Soni S., Gross T.G., Rangarajan H., et al. Outcomes of matched sibling donor hematopoietic stem cell transplantation for severe sickle cell disease with myeloablative conditioning and intermediate-dose of rabbit anti-thymocyte globulin. Pediatr Blood Cancer 2014, 61:1685-1689.
50. Horwitz M.E., Spasojevic I., Morris A., et al. Fludarabine-based nonmyeloablative stem cell transplantation for sickle cell disease with and without renal failure: clinical outcome and pharmacokinetics. Biol Blood Marrow Transplant 2007, 13(12):1422-1426.
51. Krishnamurti L., Kharbanda S., Biernacki M.A., et al. Stable long-term donor engraftment following reduced-intensity hematopoietic cell transplantation for sickle cell disease. Biol Blood Marrow Transplant 2008, 14(11):1270-1278.
52. Matthes-Martin S., Lawitschka A., Fritsch G., et al. Stem cell transplantation after reduced-intensity conditioning for sickle cell disease. Eur J Haematol 2013, 90(4):308-312.
53. Bhatia M., Jin Z., Baker C., et al. Reduced toxicity, myeloablative conditioning with BU, fludarabine, alemtuzumab and SCT from sibling donors in children with sickle cell disease. Bone Marrow Transplant 2014, 49:913-920.
54. Brichard B., Vermylen C., Ninane J., et al. Persistence of fetal hemoglobin production after successful transplantation of cord blood stem cells in a patient with sickle cell anemia. JPediatr 1996, 128(2):241-243.
55. Miniero R., Rocha V., Saracco P., et al. Cord blood transplantation (CBT) in hemoglobinopathies. Eurocord. Bone Marrow Transplant 1998, 22(Suppl 1):S78-79.
56. Gore L., Lane P.A., Quinones R.R., et al. Successful cord blood transplantation for sickle cell anemia from a sibling who is human leukocyte antigen-identical: implications for comprehensive care. JPediatr Hematol Oncol 2000, 22(5):437-440.
57. Walters M.C., Quirolo L., Trachtenberg E.T., 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.
58. Mazur M., Kurtzberg J., Halperin E., et al. Transplantation of a child with sickle cell anemia with an unrelated cord blood unit after reduced intensity conditioning. JPediatr Hematol Oncol 2006, 28(12):840-844.
59. Adamkiewicz T.V., Szabolcs P., Haight A., et al. Unrelated cord blood transplantation in children with sickle cell disease: review of four-center experience. Pediatr Transplant 2007, 11(6):641-644.
60. Sauter C., Rausen A.R., Barker J.N. Successful unrelated donor cord blood transplantation for adult sickle cell disease and Hodgkin lymphoma. Bone Marrow Transplant 2010, 45(7):1252.
61. Kamani N.R., Walters M.C., Carter S., et al. Unrelated donor cord blood transplantation for children with severe sickle cell disease: results of one cohort from the phase II study from the Blood and Marrow Transplant Clinical Trials Network (BMT CTN). Biol Blood Marrow Transplant 2012, 18(8):1265-1272.
62. Radhakrishnan K., Bhatia M., Geyer M.B., et al. Busulfan, fludarabine, and alemtuzumab conditioning and unrelated cord blood transplantation in children with sickle cell disease. Biol Blood Marrow Transplant 2013, 19(4):676-677.
63. Raj A., Bertolone S., Cheerva A. Successful treatment of refractory autoimmune hemolytic anemia with monthly rituximab following nonmyeloablative stem cell transplantation for sickle cell disease. JPediatr Hematol Oncol 2004, 26(5):312-314.