Cord blood transplantation in patients with hemoglobinopathies

Transfusion and Apheresis Science

Volumn 42, Issue 3, 2010, Pages 277-281

  Boncimino, Agata ^a   Bertaina, Alice ^a   Locatelli, Franco ^a  

^a UNIVERSITY OF PAVIA   (Italy)

Author keywords

[No Author keywords available]

Indexed keywords

CYCLOSPORIN A; METHOTREXATE; THIOTEPA;

ALLOGENEIC STEM CELL TRANSPLANTATION; ARTICLE; BIOETHICS; BONE MARROW CELL; BONE MARROW TRANSPLANTATION; CORD BLOOD STEM CELL TRANSPLANTATION; GRAFT VERSUS HOST REACTION; HEMOGLOBINOPATHY; HLA MATCHING; HUMAN; INCIDENCE; OUTCOME ASSESSMENT; SAFETY; SICKLE CELL ANEMIA; THALASSEMIA; ANEMIA, SICKLE CELL; DIRECTED TISSUE DONATION; DISEASE FREE SURVIVAL; SIBLING;

ANEMIA, SICKLE CELL; CORD BLOOD STEM CELL TRANSPLANTATION; DIRECTED TISSUE DONATION; DISEASE-FREE SURVIVAL; HUMANS; SIBLINGS; THALASSEMIA;

EID: 77953022728     PISSN: 14730502     EISSN: None     Source Type: Journal    
DOI: 10.1016/j.transci.2010.03.006     Document Type: Article

References (50)

1. Olivieri N.F. The β-thalassemia. New Engl J Med 34 (1999) 99-109
2. Platt O.S., Brambilla D.J., Rosse W.F., Milner P.F., Castro O., Steinberg M.H., et al. Mortality in sickle cell disease. Life expectancy and risk factors for early death. New Engl J Med 330 (1994) 1639-1644
3. Walters M.C., Nienhuis A.W., and Vichinsky E. Novel therapeutic approaches in sickle cell disease. Hematology (Am Soc Hematol Educ Program) (2002) 319-338
4. Zurlo M.G., De Stefano P., Borgna-Pignatti C., Di Palma A., Piga A., Melevendi C., et al. Survival and causes of death in thalassemia major. Lancet 2 (1989) 27-30
5. Borgna-Pignatti C., Rugolotto S., De Stefano P., Piga A., Di Gregorio F., Gamberoni M.R., et al. Survival and disease complications in thalassemia major. Ann NY Acad Sci 850 (1998) 227-231
6. Olivieri N.F., and Brittenham G.M. Iron-chelating therapy and the treatment of thalassemia. Blood 89 (1997) 739-761
7. Modell B., Khan M., and Darlison M. Survival in beta-thalassaemia major in the UK: data from the UK Thalassaemia register. Lancet 355 (2000) 2051-2052
8. Borgna-Pignatti C., Rugolotto S., De Stefano P., Zhao H., Cappellini M.D., Del Vecchio G.C., et al. Survival and complications in patients with thalassemia major treated with transfusion and deferoxamine. Haematologica 89 (2004) 1187-1193
9. Cappellini M.D., Cohen A., Piga A., Bejaoui M., Perrotta S., Agaoglu L., et al. A phase 3 study of deferasirox (ICL670), a once-daily oral iron chelator, in patients with beta-thalassemia. Blood 107 (2006) 3455-3462
10. Borgna-Pignatti C., Cappellini M.D., De Stefano P., Del Vecchio G.C., Forni G.L., Gamberini M.R., et al. Cardiac morbidity and mortality in deferoxamine- or deferiprone-treated patients with thalassemia major. Blood 107 (2006) 3733-3737
11. Piga A., Longo F., and Consolati A. Mortality and morbidity in thalassaemia with conventional treatment. Bone Marrow Transplant 19 (1997) 11-13
12. Locatelli F., and De Stefano P. New insights into haematopoietic stem cell transplantation for patients with haemoglobinopathies. Br J Haematol 125 (2004) 3-11
13. Thomas E.D., Buckner C.D., Sanders J.E., Papayannopoulou T., Borgna-Pignatti C., De Stefano P., et al. Marrow transplantation for thalassemia. Lancet 2 (1982) 227-229
14. Lucarelli G., Galimberti M., Polchi P., Angelucci E., Baronciani D., Giardini C., et al. Bone marrow transplantation in patients with thalassemia. N Engl J Med 322 (1990) 417-421
15. Lucarelli G., Clift R.A., Galimberti M., Polchi P., Angelucci E., Baronciani D., et al. Marrow transplantation for patients with thalassemia: results in class 3 patients. Blood 87 (1996) 2082-2088
16. Giardini C., and Lucarelli G. Bone marrow transplantation for beta-thalassemia. Hematol Oncol Clin North Am 13 (1999) 1059-1064
17. Walters M.C., Patience M., Leisenring W., Eckman J.R., Scott J.P., Mentzer W.C., et al. Bone marrow transplantation for sickle cell disease. N Engl J Med 335 (1996) 369-376
18. Hoppe C.C., and Walters M.C. Bone marrow transplantation in sickle cell anemia. Curr Opin Oncol 13 (2001) 85-90
19. Walters M.C., Patience M., Leisenring W., Rogers Z.R., Aquino V.M., Buchanan G.R., et al. Multicenter investigation of bone marrow transplantation for sickle cell disease. Stable mixed haematopoietic chimerism after bone marrow transplantation for sickle cell anemia. Biol Blood Marrow Transplant 7 (2001) 665-673
20. Storb R.F., Lucarelli G., McSweeney P.A., and Childs R.W. Hematopoietic cell transplantation for benign hematological disorders and solid tumors. Hematology (Am Soc Hematol Educ Program) (2003) 372-397
21. Locatelli F. Reduced-intensity regimens in allogeneic hematopoietic stem cell transplantation for hemoglobinopathies. Hematology (Am Soc Hematol Educ Program) (2006) 398-401
22. Lucarelli G., Clift R.A., Galimberti M., Angelucci E., Giardini C., Baronciani D., et al. Bone marrow transplantation in adult thalassemic patients. Blood 93 (1999) 1164-1167
23. Bernaudin F., Socié G., Kuentz M., Chevret S., Duval M., Bertrand Y., et al. SFGM-TC. Long-term results of related myeloablative stem-cell transplantation to cure sickle cell disease. Blood 110 (2007) 2749-2756
24. Gluckman E., and Locatelli F. Umbilical cord blood transplants. Curr Opin Haematol 7 (2000) 353-357
25. Kurtzberg J. Update on umbilical cord blood transplantation. Curr Opin Pediatr 21 (2009) 22-29
26. Locatelli F. Improving cord blood transplantation in children. Br J Haematol 147 (2009) 217-226
27. Ferrara J.L., Levine J.E., Reddy P., and Holler E. Graft-versus-host disease. Lancet 373 (2009) 1550-1561
28. Rocha V., Wagner J.E., Sobocinski K.A., Klein J.P., Zhang M.J., Horowitz M.M., et al. Graft-versus-host disease in children who have received a cord-blood or bone marrow transplant from an HLA-identical sibling. Eurocord and international bone marrow transplant registry working committee on alternative donor and stem cell sources. N Engl J Med 342 (2000) 1846-1854
29. Gluckman E., Rocha V., Boyer-Chammard A., Locatelli F., Arcese W., Pasquini R., et al. Outcome of cord-blood transplantation from related and unrelated donors. Eurocord transplant group and the European blood and marrow transplantation group. N Engl J Med 337 (1997) 373-381
30. Wagner J.E., Barker J.N., DeFor T.E., Baker K.S., Blazar B.R., Eide C., et al. Transplantation of unrelated donor umbilical cord blood in 102 patients with malignant and nonmalignant diseases: influence of CD34 cell dose and HLA disparity on treatment-related mortality and survival. Blood 100 (2002) 1611-1618
31. Eapen M., Rubinstein P., Zhang M.J., Stevens C., Kurtzberg J., Scaradavou A., et al. Outcomes of transplantation of unrelated donor umbilical cord blood and bone marrow in children with acute leukaemia: a comparison study. Lancet 369 (2007) 1947-1954
32. Gluckman E. Cord blood transplantation. Biol Blood Marrow Transplant 12 (2006) 808-812
33. Issaragrisil S., Visuthisakchai S., Suvatte V., Tanphaichitr V.S., Chandanayingyong D., Schreiner T., et al. Brief report: transplantation of cord-blood stem cells into a patient with severe thalassemia. N Engl J Med 332 (1995) 367-369
34. Lau Y.L., Ma E.S., Ha S.Y., Chan G.C., Chiu D., Tang M., et al. Sibling HLA-matched cord blood transplant for beta-thalassemia: report of two cases, expression of fetal hemoglobin and review of the literature. J Pediatr Hematol Oncol 20 (1998) 477-481
35. Brichard B., Vermylen C., Ninane J., and Cornu G. Persistence of fetal hemoglobin production after successful transplantation of cord blood stem cells in a patient with sickle cell anemia. J Pediatr 128 (1996) 241-243
36. Locatelli F., Rocha V., Reed W., Bernaudin F., Ertem M., Grafakos S., et al. Eurocord transplant group. Related umbilical cord blood transplantation in patients with thalassemia and sickle cell disease. Blood 101 (2003) 2137-2143
37. Gaziev D., Polchi P., Galimberti M., Angelucci E., Giardini C., Baronciani D., et al. Graft-versus-host disease after bone marrow transplantation for thalassemia: an analysis of incidence and risk factors. Transplantation 63 (1997) 854-860
38. Sullivan K.M., Parkman R., and Walters M.C. Bone marrow transplantation for non-malignant disease. Hematology (Am Soc Hematol Educ Program) (2000) 319-338
39. Hall J.G., Martin P.L., Wood S., and Kurtzberg J. Unrelated umbilical cord blood transplantation for an infant with beta-thalassemia major. J Pediatr Hematol Oncol 26 (2004) 382-385
40. Jaing T.H., Hung I.J., Yang C.P., Chen S.H., Sun C.F., and Chow R. Rapid and complete donor chimerism after unrelated mismatched cord blood transplantation in 5 children with beta-thalassemia major. Biol Blood Marrow Transplant 11 (2005) 349-353
41. Gluckman E., Rocha V., Ionescu I., Bierings M., Harris R.E., Wagner J., et al. Results of unrelated cord blood transplant in Fanconi anemia patients: risk factor analysis for engraftment and survival. Biol Blood Marrow Transplant 13 (2007) 1073-1082
42. Rocha V., and Locatelli F. Searching for alternative donors of haematopoietic stem cell for paediatric patients. Bone Marrow Transplant 41 (2008) 207-214
43. Andreani M., Manna M., Lucarelli G., Tonucci P., Agostinelli F., Ripalti M., et al. Persistence of mixed chimerism in patients transplanted for treatment of thalassemia. Blood 87 (1996) 3494-3499
44. Andreani M., Nesci S., Lucarelli G., Tonucci P., Rapa S., Angelucci E., et al. Long term survival of ex-thalassemic patients with persistent mixed chimerism after bone marrow transplantation. Bone marrow transplantation 25 (2000) 401-404
45. Li C.K., Chik K.W., Tsang K.S., Pong H., Shing M.M., and Yuen P.M. Mixed chimerism after bone marrow transplantation for thalassemia major. Haematologica 87 (2002) 781-782
46. Lisini D., Zecca M., Giorgiani G., Montagna D., Cristantielli R., Labirio M., et al. Donor/recipient mixed chimerism does not predict graft failure in children with beta-thalassemia given allogeneic cord blood transplantation from an HLA-identical sibling. Haematologica 93 (2008) 1859-1867
47. Hill R.S., Petersen F.B., Storb R., Appelbaum F.R., Doney K., Dahlberg S., et al. Mixed hematologic chimerism after allogeneic marrow transplantation for severe aplastic anemia is associated with a higher risk of graft rejection and a lessened incidence of acute graft-versus-host disease. Blood 67 (1986) 811-816
48. Locatelli F., Bruno B., Zecca M., Van-Lint M.T., McCann S., Arcese W., et al. Cyclosporin A and short-term methotrexate versus cyclosporine A as graft versus host disease prophylaxis in patients with severe aplastic anemia given allogeneic bone marrow transplantation from HLA-identical sibling: results of a GITMO/EBMT randomized trial. Blood 96 (2000) 1690-1697
49. Reed W., Smith R., Dekovic F., Lee J.Y., Saba J.D., Trachtenberg E., et al. Comprehensive banking of sibling donor cord blood for children with malignant and non-malignant disease. Blood 101 (2003) 351-357
50. Burgio G.R., Gluckman E., and Locatelli F. Ethical reappraisal of 15 years of cord blood transplantation. Lancet 361 (2003) 250-252