Hematopoietic stem cell transplantation for Thalassemia

Current Stem Cell Research and Therapy

Volumn 6, Issue 2, 2011, Pages 162-169

  Gaziev, Javid ^a   Lucarelli, Guido ^a  

^a TOR VERGATA UNIVERSITY HOSPITAL   (Italy)

Author keywords

Bone marrow transplantation; Ex thalassemics; Haploidentical donor; Thalassemia; Unrelated cord blood transplantation; Unrelated donor

Indexed keywords

AZATHIOPRINE; BUSULFAN; CYCLOPHOSPHAMIDE; CYCLOSPORIN A; FLUDARABINE; GRANULOCYTE COLONY STIMULATING FACTOR; HYDROXYUREA; METHOTREXATE; METHYLPREDNISOLONE; PEGINTERFERON ALPHA; RECOMBINANT ERYTHROPOIETIN; RIBAVIRIN;

ALLOGENEIC HEMATOPOIETIC STEM CELL TRANSPLANTATION; ANTIVIRAL THERAPY; ARTICLE; BETA THALASSEMIA; BLOOD DONOR; CHELATION THERAPY; CHIMERA; CLINICAL EFFECTIVENESS; DISEASE FREE SURVIVAL; DRUG DOSE REDUCTION; GRAFT FAILURE; GRAFT REJECTION; GRAFT SURVIVAL; GRAFT VERSUS HOST REACTION; GROWTH RETARDATION; HAPLOIDY; HEPATITIS C; HISTOCOMPATIBILITY; HISTOINCOMPATIBILITY; HLA MATCHING; HUMAN; HYPOGONADISM; IMMUNOSUPPRESSIVE TREATMENT; LIVER FIBROSIS; LOW DRUG DOSE; PATIENT SELECTION; PRIORITY JOURNAL; RISK ASSESSMENT; THALASSEMIA; TREATMENT OUTCOME; UMBILICAL CORD BLOOD;

ADULT; ANIMALS; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HUMANS; THALASSEMIA;

EID: 79955702763     PISSN: 1574888X     EISSN: None     Source Type: Journal    
DOI: 10.2174/157488811795495413     Document Type: Article

References (56)

1. Weatherall DJ, Clegg JB. The thalassemia Syndromes. 4 th ed.Oxford: Blackwell Science 2001.
2. Weatherall DJ, Clegg JB. Thalassemia - a global public healthproblem. Nat Med 1996; 2(8): 847-9.
3. Borgna- Pignatti C, Rugolotto S, De Stefano P, et al. Survival andcomplications in patients with thalassemia major treated with transfusionsand deferrioxamine. Haematologica 2004; 89: 1187-93.
4. Piga A, Longo F, Consolati A. Mortality and morbidity in thalassemiawith conventional treatment. Bone Marrow Transplant 1977;19(Suppl 2): 11-13
5. Modell B, Khan M, Darlison M. Survival in beta-thalassemia majorin the UK: data from the UK Thalassemia Register. Lancet 2000;355: 2051-52.
6. Piga A, Galanello R, Forni G, et al. Randomized phase II trial ofdeferasirox (Exjade, ICL 670), once daily, orally-administered ironchelator, in comparision to deferoximine in thalassemia patientswith transfusional iron overload. Haematologica 2006; 91: 873-80.
7. Lucarelli G, Galimberti M, Polchi P, et al. Bone marrow transplantation in patients with thalassemia. N Engl JMed 1990; 322: 417-21.
8. Lucarelli G, Andreani M, Angelucci E. The cure of thalassemia bybone marrow transplantation. Blood Rev 2002; 16: 81-5.
9. Olivieri NF, Nathan DG, MacMillan JH, et al. Survival in medically treated patients with homogygous beta-thalassemia. N Engl J Med 1994; 331: 574-8.
10. Cunningham MJ, Macklin EA, Neueld EJ, Cohen AR. Thalassemia Clinical Research Network. Complications of beta -thalassemia major in North America. Blood 2002; 99: 36-43.
11. Lucarelli G, Galimberti M, Polchi P, et al. Bone marrow transplantation in thalassemia. Hematol Oncol Clin North Am 1991; 5(3): 549-56.
12. Lucarelli G, Galimberti M, Polchi P, et al. Marrow transplantation in patients with thalassemia responsive to iron chelation therapy. N Engl J Med 1993; 329: 840-4.
13. Lucarelli G, Clift R, Galimberti M, et al. Marrow transplantation for patients with thalassemia: results in Class 3 patients. Blood 1996; 87: 2082-8.
14. Sodani P, Gaziev J, Polchi P, et al. New approach for bone marrow transplantation in patients with class 3 thalassemia aged younger than 17 years. Blood 2004; 104: 1201-3.
15. Lucarelli G, Galimberti M, Polchi P, et al. Bone marrow transplantation in adult thalassemia. Blood 1992; 80: 1603-07.
16. Lucarelli G, Clift RA, Galimberti M, et al. Bone marrow transplantation in adult thalassemic patients. Blood 1999; 93: 1164-7.
17. Gaziev J, Sodani P, Polchi P, Andreani M, Lucarelli G. Bone marrow transplantation in adults with thalassemia. Treatment and longterm follow-up. Ann NY Acad Sci 2005; 1054: 196-205.
18. Di Bartolomeo P, Santarone S, DiBartolomeo E, et al. Long-termresults of bone marrow transplantation for thalassemia major in-Pescara. Blood 2004; 104: 3332.
19. Argiolu F, Sanna MA, Cossu F, et al. Bone marrow transplant in thalassemia. The experience of Cagliari. Bone Marrow Transplant 1997; 19 (Suppl 2): 65-7.
20. Clift RA, Johnson FL. Marrow transplants for thalassemia. The USA experience. Bone Marrow Transplant 1997; 19(Suppl 2): 57-9.
21. Lawson SE, Roberts IAG, Amrolia P, Dokal I, Szydlo R, Darbyshire PJ. Bone marrow transplantation for?-thalassemia major: The UK experience in two paediatric centers. Br J Haematol 2003; 120:289-95.
22. Ghavamzadeh A, Bahar B, Djahani M, Kokabandeh A, Shahriari A. Bone marrow transplantation of thalassemia, the experience in Tehran (Iran). Bone Marrow Transplant 1997; 19(Suppl 2): 71-3.
23. Dennison D, Srivastava A, Chandy M. Bone marrow transplantation for thalassemia in India. Bone Marrow Transplant 1997;19(Suppl 2): 70.
24. Lin HP, Chan LL, Lam SK, Ariffin W, Menaka N, Looi LM. Bone marrow transplantation for thalassemia. The experience from Malaysia. Bone Marrow Transplant 1997; 19 (Suppl 2): 74-7.
25. Li CK, Shing MK, Chik KW, Lee V, Leung TF, Cheung AYK, Yuen PMP. Haematopoietic stem cell transplantation for thalassemia major in Hong Kong: prognostic factors and outcome. Bone Marrow Transplant 2002; 29: 101-5.
26. Issaragrisil S, Suvatte V, Visuthisakchai S, et al. Bone marrow and cord blood stem cell transplantation for thalassemia in Thailand. Bone Marrow Transplant 1997; 19(Suppl 2): 54-6.
27. Hongeng S, Pakakasama S, Chuansumrit A, et al. Outcomes of transplantation with related and unrelated-donor stem cells in children with severe thalassemia. Biol Blood Marrow Transplant 2006;12: 683-7.
28. Gaziev J, Nguyen L, Puozzo C, et al. Novel pharmacokinetic behavior of intravenous busulfan in children with thalassemia under-going hematopoietic stem cell transplantation: a prospective evaluation of pharmacokinetic and pharma codynamic profile with the rapeutic drug monitorino. Blood 2010; 115: 4597-7604.
29. Gaziev D, Galimberti M, Lucarelli G, et al. Bone marrow transplantation from alternative donors for thalassemia: HLA-phenotypically identical relative and HLA nonidentical sibling or parent transplants. Bone Marrow Transplant 2000; 25(8): 815-21.
30. Glukman E, Rocha E, Boyer-Chammard A, et al. Outcome of cord-blood transplantation from related and unrelated donors. Eurocord Transplant Group and the European Blood and Marrow Transplnatation Group. N Engl J Med 1997; 337: 373-81.
31. Rocha V, Cornish J, Sievers EL, et al. Comparison of outcomes of unrelated bone marrow and umbilical cord blood transplants in children with acute leukemia. Blood 2001; 97: 2962-71.
32. Rocha V, Wagner JE Jr, Sobochinski KA, et al. Graft-versus-hostdisease in children who have received a cord-blood or bone marrow transplant from and HLA-identical sibling. Eurocord and International Bone Marrow Transplant Registry Working Committee on Alternative Donor and Stem Cell Sources. N Engl J Med 2000;342: 1846-54.
33. Locatelli F, Rocha V, Reed W, et al. Related unbilical cord bloodtransplantation in patients with thalassemia and sickle cell disease. Blood 2003; 101: 2137-43.
34. Fang J, Huang S, Chen C, et al. Umbilical cord blood transplantation in Chinese children with beta-thalassemia. J Pediatr Hematol Oncol 2004; 26(3): 185-9.
35. Wagner JE, Kernan NA, Steinbuch M Broxmeyer HE, Glucman E. Allogeneic sibling umbilical cord blood transplantation in forty-four children with malignant and non-malignant diseases. Lancet 1995; 46: 214-9.
36. 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 NY Acad Sci 2005;1054: 186-95.
37. La Nasa G, Caocci G, Argiolu F, et al. Unrelated donor stem cell transplantation in adult patients with thalassemia. Bone Marrow Transplant 2005; 36: 971-5.
38. Wagner JE, Barker JN, De For TE, 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 2002; 100: 1611-8.
39. Jaing T-H, Chen SH-H, Tsai MH, et al. Transplantation of unrelated donor umbilical cord blood for nonmalignant diseases: a single institution's experience with 45 patients. Biol Blood Marrow Transplant 2010; 16: 102-7.
40. Sodani P, Isgro A, Gaziev J, et al. Purified T-depleted, CD34+peripheral blood and bone marrow cell transplantation from haploidentical mother to child with Thalassemia. Blood 2010; 115: 1296-1302.
41. Andreani M, Manna M, Lucarelli G, et al. Persistence of mixed chimerism in patients transplanted for the treatment of thalassemia. Blood 1996; 87: 3494-9.
42. Andreani M, Nesci S, Lucarelli G, et al. Long-term survival of ex-thalassemic patients with persistent mixed chimerism after marrow transplantation. Bone Marrow Transplant 2000; 25: 401-4.
43. Chandy M, Mathews V, George B, et al. Reduced intensity conditioningwith fludarabine, busulfan and cyclophosphamide for highrisk patients with thalassemia major undergoing allogeneic bonemarrow transplantation results in high rejection rates. Blood 2007;110: 594a. 404.
44. Gaziev D, Polchi P, Lucarelli G, et al. Second marrow transplants for graft failure in patients with thalassemia. Bone Marrow Transplant 1999; 24: 1299-06.
45. Gaziev J, Giardini C, Polchi P, Sodani P, Lucarelli G. New approaches to the second transplant for thalassemia. Bone Marrow Transplant 2004; 33(suppl 1): S153.
46. Gaziev J, Sodani P, Lucarelli G, et al. Second Stem Cell Transplantation for Thalassemia Recurrence Following Graft Rejection of the First Graft. Bone Marrow Transplant 2008; 42: 397-404.
47. Gaziev D, Polchi P, Galimberti M, et al. Graft-versus-host disease after bone marrow transplantation for thalassemia: an analysis of incidence and risk factors. Transplantation 1997; 63: 854-60.
48. Lucarelli G, Angelucci E, Giardini C, et al. Fate of iron stores in thalassemia after bone marrow transplantation. Lancet 1993; 342:1388-91.
49. Angelucci E, Muretto P, Nicolucci A, et al. Effects of iron overload and hepatitis C virus positivity in determining progression of liver fibrosis in thalassemia following bone marrow transplantation. Blood 2002; 100: 17-21.
50. Angelucci E, Muretto P, Lucarelli G, et al. Phlebotomy to reduceiron overload in patients cured of thalassemia by bone marrow transplantation. Italian Cooperative Group for Phlebotomy Treatment of Transplanted Thalassemia Patients. Blood 2002; 90(3):994-8.
51. Angelucci E, Muretto P, Lucarelli G, et al. Treatment of iron overload in the 'ex-thalassemic'. Report from the phlebotomy program. Ann NY Acad Sci 1998; 30(850): 288-93.
52. Giardini C, Galimberti M, Lucarelli G, et al. Desferrioxamine therapy accelerates clearance of iron deposits after bone marrow transplantation for thalassemia. Br J Haematol 1995; 89: 868-73.
53. Mariotti E, Angelucci E, Agostini A, Baronciani D, Sgarbi E, Lucarelli G. Evaluation of cardiac status in iron-loaded thalassemia patients following bone marrow transplantation: improvement in cardiac function during reduction in body iron burden. Br J Haematol 1998; 103(4): 916-21.
54. Gaziev D, Galimberti M, Giardini C, Baronciani D, Lucarelli G. Growth in children after bone marrow transplantation for thalassemia. Bone Marrow Transplant 1993; 12(Suppl 1): 100-1.
55. De Sanctis V, Galimberti M, Lucarelli G, et al. Growth and development in ex-thalassemic patients. Bone Marrow Transplant 1997;19(Suppl 2): 126-7.
56. Strader DB, Wright T, Thomas DL, Seef LB. Diagnosis, management, and treatment of hepatitis C. Hepatology 2004; 39(4): 1147-71.