Hematopoietic stem cell transplantation in thalassemia and sickle cell anemia

Cold Spring Harbor Perspectives in Medicine

Volumn 2, Issue 5, 2012, Pages

  Lucarelli, Guido ^a   Isgrò, Antonella ^a   Sodani, Pietro ^a   Gaziev, Javid ^a  

^a TOR VERGATA UNIVERSITY HOSPITAL   (Italy)

Author keywords

[No Author keywords available]

Indexed keywords

AZATHIOPRINE; BUSULFAN; CYCLOPHOSPHAMIDE; CYCLOSPORIN; FLUDARABINE; HYDROXYUREA; METHOTREXATE; METHYLPREDNISOLONE; THIOTEPA; THYMOCYTE ANTIBODY; MYELOABLATIVE AGENT;

ARTICLE; BLOOD TRANSFUSION; DISEASE FREE SURVIVAL; ERYTHROPOIESIS; GRAFT REJECTION; GRAFT SURVIVAL; GRAFT VERSUS HOST REACTION; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HUMAN; LOW DRUG DOSE; MORTALITY; RISK FACTOR; SICKLE CELL ANEMIA; THALASSEMIA; ADULT; LIVING DONOR; METHODOLOGY; REVIEW;

ADULT; ANEMIA, SICKLE CELL; GRAFT VS HOST DISEASE; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HUMANS; LIVING DONORS; MYELOABLATIVE AGONISTS; THALASSEMIA;

EID: 84878958557     PISSN: None     EISSN: 21571422     Source Type: Journal    
DOI: 10.1101/cshperspect.a011825     Document Type: Article

References (44)

1. Adamkiewicz TV, Szabolcs P, Haight A, Baker KS, Staba S, Kedar A, Chiange KY, Krishnamurti L, Boyer MW, Kurtzberg J, et al. 2007. Unrelated cord blood transplantation in children with sickle cell disease: Review of four-center experience. Pediatr Transplant 11: 641-644.
2. Andreani M, Manna M, Lucarelli G, Tonucci P, Agostinelli F, Ripalti M, Rapa S, Talevi N, Galimberti M, Nesci S. 1996. Persistence of mixed chimerism in patients transplanted for the treatment of thalassemia. Blood 87: 3494-3499.
3. Andreani M, Nesci S, Lucarelli G, Tonucci P, Rapa S, Angelucci E, Persini B, Agostinelli F, Donati M, Manna M. 2000. Long-term survival of ex-thalassemic patients with persistent mixed cjimerism after marrow transplantation. Bone Marrow Transplant 25: 401-404.
4. Andreani M, Testi M, Battarra M, Lucarelli G. 2011a. Split chimerism between nucleated and red blood cells after bone marrow transplantation for haemoglobinopathies. Chimerism 2: 21-22.
5. Andreani M, Testi M, Gaziev J, Condello R, Bontadini A, Tazzari PL, Ricci F, De Felice L, Agostini F, Fraboni D, et al. 2011b. Quantitatively different red cell/nucleated cell chimerism in patients with long-term, persistent hematopoietic mixed chimerism after bone marrow transplantation for thalassemia major or sickle cell disease. Haematologica 96: 128-133.
6. Bank A. 2006. Regulation of human fetal haemoglobin: New players, new complexities. Blood 107: 435-443.
7. Bernaudin F, Socie G, Kuentz M, Chevret S, Duval M, Bertrand Y, Vannier JP, Yakouben K, Thuret I, Bordigoni P, et al. 2007. Long-term results of related myeloablative stem-cell transplantation to cure sickle cell disease. Blood 110: 2749-2756.
8. Ferster A, Corazza F, Vertongen F, Bujan W, Devalck C, Fondu P, Cochaux P, Lambermont M, Khaladji Z, Sariban E. 1995. Transplanted sickle-cell disease patients with autologous bone marrow recovery after graft failure develop increased levels of fetal haemoglobin which corrects disease severity. Brit J Haematol 90: 804-808.
9. Gaziev J, Sodani P, Polchi P, Andreani M, Lucarelli G. 2005. Bone marrow transplantation in adults with thalassemia. Treatment and long-term follow-up. Ann NY Acad Sci 1054: 196-205.
10. Ghavamzadeh A, Nasseri P, Eshraghian MR, Jahani M, Baybordi I, Nateghi J, Khodabandeh A, Sadjadi AR, Mohyeddin M, Khademi Y. 1997. Prognostic factors in bone marrow transplantation for β thalassemia major: Experiences from Iran. Bone Marrow Transplant 22: 1167-1169.
11. Giardini C, Galimberti M, Lucarelli G, Polchi P, Angelucci E, Baronciani F, Agostinelli F, Giorni G, Muretto P. 1993. Bone marrow transplantation in sickle-cell anemia in Pesaro. Bone Marrow Transplant 12: 122-123.
12. Hobbs JR, Hugh-Jones K, Shaw PJ, Downie CJC, Williamson S. 1986. Engraftment rates related to busulphan and cyclophosphamide dosages for displacement bone marrow transplants in fifty children. Bone Marrow Transplant 1: 201-208.
13. Hongeng S, Pakakasama S, Chuansumrit A, Sirachainan N, Kitpoka P, Udomsubpayakul U, Ungkanont A, Jootar S. 2006. Outcomes of transplantation with related and unrelated-donor stem cells in children with severe thalassemia. Biol Blood Marrow Transplant 12: 683-687.
14. Hsieh MM, Kang EM, Fitzhugh CD, et al. 2009. Allogeneic hematopoietic stem-cell transplantation for sickle cell disease. N Engl J Med 361: 2309-2317.
15. Hsieh MM, Fitzhugh CD, Tisdale JF. 2011. Allogeneic hematopoietic stem cell transplantation for sickle cell disease: The time is now. Blood 118: 1197-1207.
16. Iannone R, Casella JF, Fuchs EJ, Chen AR, Jones RJ, Woolfrey A, Amylon M, Sullivan KM, Storb RF, Walters MC. 2003. Results of minimally toxic nonmyeloablative transplantation in patients with sicule cell anemia and β-thalassemia. Biol Blood Marrow Transplant 9: 519-528.
17. Isgrò A, Gaziev J, Sodani P, Lucarelli G. 2010. Progress in hematopoietic stem cell transplantation as allogeneic cellular gene therapy in thalassemia. Ann NY Acad Sci 1202: 149-154.
18. Kapoor N, Kirkpatrick D, Blaese RM, Oleske J, Hilgartner MH, Changanti RS, Good RA, O'Reilly RJ. 1981. Reconstitution of normal megakaryocytopoiesis and immunologic functions in Wiskott-Aldrich syndrome by marrow transplantation following myeloablation and immunosuppression with busulfan and cyclophosphamide. Blood 57: 692-696.
19. Krishnamurti L, Kharbanda S, Biernacki M, Zhang W, Baker KS, Wagner JE, WuCJ. 2008. Stable long-term donor engraftment following reduced-intensity hematopoietic cell transplantation for sickle cell disease. Biol Blood Marrow Transplant 14: 1270-1278.
20. La Nasa G, Argiolu F, Giardini C, Pession A, Fagioli F, Caocci G, Vacca A, De Stefano P, Piras E, Ledda A, et al. 2005a. Unrelated bone marrow transplantation for β-thalassemia patients: The experience of the Italian Bone Marrow Tranplant Group. Ann NY Acad Sci 1054: 186-195.
21. La Nasa G, Caocci G, Argiolu F, Giardini C, Locatelli F, Vacca A, Ororfino MG, Piras E, Addari MC, Ledda A, et al. 2005b. Unrelated donor stem cell transplantation in adult patients with thalassemia. Bone Marrow Transplant 36: 971-975.
22. Locatelli F, Rocha V, Reed W, Bernaudin F, Ertem M, Grafakos S, Brichard B, Li X, Nagler A, Giorgiani G, et al. 2003. Related umbilical cord blood transplantation in patients with thalassemia and sickle cell disease. Blood 101: 2137-2143.
23. Lucarelli G, Gaziev J. 2008. Advances in the allogeneic transplantation for thalassemia. Blood Rev 22: 53-63.
24. Lucarelli G, Clift RA, Galimberti M, Angelucci E, Giardini C, Baronciani D, PolchiP, Andreani M, Gazlev D, Erer B, et al. 1990. Bone marrow transplantation in patients with thalassemia. N Engl J Med 322: 417-421.
25. Lucarelli G, Galimberti M, Polchi P, Angelucci E, Baronciani D, Durazzi SM, Giardini C, Albertini F, Clift RA. 1992. Bone marrow transplantation in adult thalassemia. Blood 80: 1603-1607.
26. Lucarelli G, Clift RA, Galimberti M, Angelucci E, Giardini C, Baronciani D, PolchiP, Andreani M, Gazlev D, Erer B, et al. 1999. Bone marrow transplantation in adult thalassemic patients. Blood 93: 1164-1167.
27. Lucarelli G, Andreani M, Angelucci E. 2002. The cure of thalassemia by bone marrow transplantation. Blood Rev 16: 81-85.
28. Lucarelli G, Gaziev J, Isgrò A, Sodani P, Paciaroni K, Alfieri C, De Angelis G, Marziali M, Simone MD, Gallucci C, et al. 2011. Allogeneic cellular gene therapy in hemoglobinopathies-evaluation of hematopoietic SCT in sickle cell anemia. Bone Marrow Transplant doi: 10. 1038/bmt. 2011. 79.
29. Nesci S, Manna M, Lucarelli G, Tonucci P, Donati M, Buffi O, Agostinelli F, Andreani M. 1998. Mixed chimerism after bone marrow transplantation in thalassemia. Ann NY Acad Sci 850: 495-497.
30. 0-thalassemia: A clinical remission despite genetic disease and transplant rejection. Am J Hematol 84: 372-373.
31. Panepinto JA, Walters MC, Carreras J, Marsh J, Bredeson CN, Gale RP, Hale GA, Horan J, Hows JM, Klein JP, et al. 2007. Matched-related donor transplantation for sickle cell disease: Report from the Center for International Blood and Transplant Research. Br J Haematol 137: 479-485.
32. Parkman R, Rappeport J, Geha R, Belli J, Cassady R, Levey R, Nathan DG, RosenFS. 1978. Complete correction of the Wiskott-Aldrich syndrome by allogeneic bone-marrow transplantation. N Engl J Med 298: 921-927.
33. Sabloff M, Chandy M, Wang Z, Logan BR, Ghavamzadeh A, Li CK, Irfan SM, Bredeson CN, Cowan MJ, Gale RP, et al. 2011. HLA-matched sibling bone marrow transplantation for β-thalassemia major. Blood 117: 1745-1750.
34. Sodani P, Isgrò A, Gaziev J, Polchi P, Paciaroni K, Marziali M, Simone MD, Roveda A, Montuoro A, Alfieri C, et al. 2010. Purified T-depleted, CD34+ peripheral blood and bone marrow cell transplantation from haploidentical mother to child with thalassemia. Blood 115: 1296-1302 (Erratum in Blood 116: 1627).
35. Sodani P, Isgrò A, Gaziev J, Paciaroni K, Marziali M, Simone MD, Roveda A, De Angelis G, Gallucci C, Torelli F, et al. 2011. T cell-depleted HLA-haploidentical stem cell transplantation in thalassemia young patients. Pediatr Rep 22: e13.
36. Storb R, Champlin RE. 1991. Bone marrow transplantation for severe aplastic anemia. Bone Marrow Transplant 8: 69-72.
37. Storb R, Weiden PL, Graham TC, Lerner KG, Nelson N, Thomas ED. 1977. Hemopoietic grafts between DLA-identical canine littermates following dimethyl myleran. Evidence for resistance to grafts not associated with DLA and abrogated by antithymocyte serum. Transplantation 24: 349-357.
38. Thomas ED, Storb R, Fefer A, Slichter SJ, Bryant JI, Buckner CD, Neiman PE, Clift RA, Funk DD, Lerner KE. 1972. Aplastic anaemia treated by marrow transplantation. Lancet 1: 284-289.
39. van Beisen K, Bartholomew A, Stock W, Peace D, Devine S, Sher D, Sosman J, Chen YH, Koshy M, Hoffman R. 2000. Fludarabine-based conditioning regimen for allogeneic transplantation in adult with sickle cell disease. Bone Marrow Transplant 26: 445-449.
40. Vermylen C, Cornu G. 1993. Bone marrow transplantation in sickle cell anemia. Blood Rev 7: 1-3.
41. Vermylen C, Cornu G, Ferster A, Brichard B, Ninane J, Ferrant A, Zenebergh A, Maes P, Dhooge C, Benoit Y, et al. 1998. Haematopoietic stem cell transplantation for sickle cell anaemia: The first 50 patients transplanted in Belgium. Bone Marrow Transplant 22: 1-6.
42. Walters MC, Storb R, Patience M, Leisenring W, Taylor T, Sanders JE, Buchanan GE, Rogers ZR, Dinndorf P, Davies SC, et al. 2000. Impact of bone marrow transplantation for symptomatic sickle cell disease: An interim report. Multicenter investigation of bone marrow transplantation for sickle cell disease. Blood 95: 1918-1924.
43. Walters MC, Patience M, Leisenring W, Rogers ZR, Aquino VM, Buchanan GR, Roberts IA, Yeager AM, Hsu L, Adamkiewicz T, et al. 2001. Stable mixed hematopoietic chimerism after bone marrow transplantation for sickle cell anemia. Biol Blood Marrow Transplant 7: 665-673.
44. Williams T. 2012. World distribution, population genetics, and burden of thalassemic and sickle cell syndromes. Cold Spring Harb Perspect Med doi: 10. 1101/cshperspect. a011692.