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Chapter 54 - Allogeneic Hematopoietic Cell Transplants for Thalassemia

from Section 15 - Hematopoietic Cell Transplants for Non-Neoplastic Diseases

Published online by Cambridge University Press:  24 May 2017

Hillard M. Lazarus
Affiliation:
Case Western Reserve University, Ohio
Robert Peter Gale
Affiliation:
Imperial College London
Armand Keating
Affiliation:
University of Toronto
Andrea Bacigalupo
Affiliation:
Ospedale San Martino, Genoa
Reinhold Munker
Affiliation:
Louisiana State University, Shreveport
Kerry Atkinson
Affiliation:
University of Queensland
Syed Ali Abutalib
Affiliation:
Midwestern Regional Medical Center, Cancer Treatment Centers of America, Chicago
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Hematopoietic Cell Transplants
Concepts, Controversies and Future Directions
, pp. 513 - 523
Publisher: Cambridge University Press
Print publication year: 2000

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References

Weatheral, D.J., Clegg, J.B.. The Thalassemia Syndromes. 4th ed. Oxford: Blackwell Science. 2001.CrossRefGoogle Scholar
Modell, B., Khan, M., Darlison, M.. Survival in beta-thalassemia major in the UK: data from the UK Thalassemia Register. Lancet 2000; 355: 2051–2.CrossRefGoogle ScholarPubMed
Cunningham, M.J., Macklin, E.A., Neueld, E.J., et al. Thalassemia Clinical Research Network. Complications of beta–thalassemia major in North America. Blood 2002; 99 : 3643.Google Scholar
Thomas, E.D., Buckner, C.D., Sanders, J.E., et al. Marrow transplantation for thalassaemia. Lancet 1982; ii: 227–5.Google Scholar
Lucarelli, G., Galimberti, M., Delfini, C., et al. Marrow transplantation for thalassemia following busulfan and cyclophosphamide. Lancet 1985, 1(8442): 13551357.CrossRefGoogle ScholarPubMed
Lucarelli, G., Galimberti, M., Polchi, P., et al. Marrow transplantation in patients with advanced thalassemia. N Engl J Med 1987; 316: 1050–5.CrossRefGoogle ScholarPubMed
Tutschka, P.J., Elfenbein, G.J., Sensenbrenner, L.L., et al. Preparative regimens for marrow transplantation in acute leukemia and aplastic anemia. Baltimore experience. Am J Ped Hematol Oncol 1980; 2: 363–70.Google Scholar
Santos, G.W., Tutschka, P.J., Brookmeyer, R., et al. Marrow transplantation for acute nonlymphocytic leukemia after treatment with busulfan and cyclophosphamide. N Engl J Med 1983; 309: 1347–53.CrossRefGoogle ScholarPubMed
Bolinger, A.M., Zangwill, A.B., Slattery, J.T., et al. Target dose adjustment of busulfan in pediatric patients undergoing bone marrow transplantation. Bone Marrow Transplant 2001; 28: 1013–18.CrossRefGoogle ScholarPubMed
Chandy, M., Balasubramanian, P., Ramachandran, S.V ., et al. Randomized trial of two different conditioning regimens for bone marrow transplantation in thalassemia-the role of busulfan pharmacokinetics in determining outcome. Bone Marrow Transplant 2005; 36: 839–45.CrossRefGoogle ScholarPubMed
Andersson, B.S., Kashyap, A., Gian, V., et al. Conditioning therapy with intravenous busulfan and cyclophosphamide (IV BuCy2) for hematologic malignancies prior to allogeneic stem cell transplantation: a phase II study. Biol Blood Marrow Transplant 2002; 8: 145–54.CrossRefGoogle ScholarPubMed
Gaziev, J., Nguyen, L., Puozzo, C., et al. Novel pharmacokinetic behavior of intravenous busulfan in children with thalassemia undergoing hematopoietic stem cell transplantation: a prospective evaluation of pharmacokinetic and pharmacodynamic profile with therapeutic drug monitoring. Blood 2010; 115(22): 4597–604.CrossRefGoogle ScholarPubMed
Bernardo, M.E., Piras, E., Vacca, A., et al. Allogeneic hematopoietic stem cell transplantation in thalassemia major: results of a reduced-toxicity conditioning regimen based on the use of threosulfan. Blood 2014; 120: 473–6.Google Scholar
Choudhary, D., Sharma, S.K., Gupta, N., et al. Treosulfan-thiotepa-fludarabinee based conditioning regimen for allogeneic aransplantation in patients with thalassemia major: a single-Center experience from North India. Biol Blood Marrow Transplant 2013; 9: 492503.CrossRefGoogle Scholar
Mathews, V., George, V ., Viswabandya, A., et al. Improved clinical outcomes of high risk b thalassemia major patients undergoing a HLA-matched related allogeneic stem cell transplant with a treosulfan based conditioning regimen and peripheral blood stem cell grafts. PlosOne 2013; 8: 18.CrossRefGoogle Scholar
Anurathapan, U., Pakakasama, S., Rujkijyanont, P., et al. Pretransplant immunosuppression followed by reduced-toxicity conditioning and stem cell transplantation in high-risk thalassemia: a safe approach to disease control. Biol Blood Marrow Transplant 2013; 19: 1254–70.CrossRefGoogle ScholarPubMed
Lucarelli, G., Galimberti, M., Polchi, P., et al. Bone marrow transplantation in patients with thalassemia. N Engl J Med 1990; 322: 417–21.CrossRefGoogle ScholarPubMed
Lucarelli, G., Galimberti, M., Polchi, P., et al. Bone marrow transplantation in thalassemia. Hematol Oncol Clin North Am 1991; 5(3): 549–56.CrossRefGoogle ScholarPubMed
Lucarelli, G., Andreani, M., Angelucci, E.. The cure of thalassemia by bone marrow transplantation. Blood Reviews 2002; 16: 81–5.CrossRefGoogle ScholarPubMed
Lucarelli, G., Clift, R., Galimberti, M., et al. Marrow transplantation for patients with thalassemia: results in Class 3 patients. Blood 1996; 87: 2082–88.CrossRefGoogle ScholarPubMed
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.CrossRefGoogle ScholarPubMed
Gaziev, J., Lucarelli, G.. Stem cell transplantation for thalassemia. RBM Online 2005;10:111–15.Google Scholar
Lucarelli, G., Clift, R.A., Galimberti, M., et al. Bone marrow transplantation in adult thalassemic patients. Blood 1999; 93: 1164–7.CrossRefGoogle ScholarPubMed
Gaziev, J., Sodani, P., Polchi, P., et al. Bone marrow transplantation in adults with thalassemia. Treatment and long-term follow-up. Ann NY Acad Sci 2005; 1054: 196205.CrossRefGoogle ScholarPubMed
Gaziev, J., Isgrò, A., Sodani, P., et al. Optimal outcomes in young class 3 patients with thalassemia undergoing HLA-identical sibling bone marrow transplantation. Transplantation 2016; 100(4): 925–32.CrossRefGoogle ScholarPubMed
Lawson, S.E., Roberts, I.A., Amrolia, P., Dokal, I., Szydlo, R., Darbyshire, P.J.. Bone marrow transplantation for beta-thalassaemia major: the UK experience in two paediatric centres. Br J Haematol 2003; 120(2): 289–95.CrossRefGoogle ScholarPubMed
Di Bartolomeo, P., Santarone, S., Di Bartolomeo, E., et al. Long-term results of survival in patients with thalassemia major treated with bone marrow transplantation. Am J Hematol 2008; 83(7): 528–30.CrossRefGoogle ScholarPubMed
Ghavamzadeh, A., Iravani, A., Ashouri, A., et al. Peripheral blood versus bone marrow as a source of hematopoietic stem cells for allogeneic transplantation in children with class I and II beta thalassemia major. Biol Blood Marrow Transplant 2008; 14(3): 301–8.CrossRefGoogle Scholar
Irfan, M., Hashmi, K., Adil, S., et al. Beta-thalassaemia major: bone marrow versus peripheral blood stem cell transplantation. J Pak Med Assoc 2008; 58(3): 107–10.Google ScholarPubMed
Chiesa, R., Cappelli, B., Crocchiolo, R., et al. Unpredictability of intravenous busulfan pharmacokinetics in children undergoing hematopoietic stem cell transplantation for advanced beta thalassemia: limited toxicity with a dose-adjustment policy. Biol Blood Marrow Transplant 2010;16(5):622–8.CrossRefGoogle ScholarPubMed
Iravani, M., Tavakoli, E., Babaie, M.H., Ashouri, A., Khatami, F., Ghavamzadeh, A.. Comparison of peripheral blood stem cell transplant with bone marrow transplant in class 3 thalassemic patients. Exp Clin Transplant 2010; 8(1): 6673Google ScholarPubMed
Sabloff, M., Chandy, M., Wang, Z., et al. HLA-matched sibling bone marrow transplantation for beta-thalassemia major. Blood 2011; 117(5): 1745–50.CrossRefGoogle ScholarPubMed
Yesilipek, M.A., Ertem, M., Cetin, M., et al. HLA-matched family hematopoetic stem cell transplantation in children with beta thalassemia major: the experience of the Turkish Pediatric Bone Marrow Transplantation Group. Pediatr Transplant 2012; 16(8): 846–51.CrossRefGoogle ScholarPubMed
Goussetis, E., Peristeri, I., Kitra, V., et al. HLA-matched sibling stem cell transplantation in children with ß-thalassemia with anti-thymocyte globulina s part of the preparative regimen: the Greek experience. Bone Marrow Transplant 2012; 47: 1061–6.CrossRefGoogle Scholar
Galambrun, C., Pondarre, C., Bertrand, Y., et al. French multicenter 22-year experience in stem cell transplantation for beta-thalassemia major: lessons and future directions. Biol Blood Marrow Transplant 2013; 19(1): 62–8.CrossRefGoogle ScholarPubMed
Hussein, A.A., Al-Zaben, A., Ghatasheh, L., et al. Risk adopted allogeneic hematopoietic stem cell transplantation using a reduced intensity regimen for children with thalassemia major. Pediatr Blood Cancer 2013; 60(8): 1345–9.CrossRefGoogle ScholarPubMed
Shaw, P.J., Kan, F., Ahn, K.W., et al. Outcomes of pediatric bone marrow transplantation for leukemia and myelodysplasia using matched sibling, mismatched related, or matched unrelated donors. Blood 2010; 116(11):4007–15.CrossRefGoogle ScholarPubMed
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.CrossRefGoogle ScholarPubMed
Gaziev, J., Sodani, P., Lucarelli, G., et al. Second hematopoietic SCT in patients with thalassemia recurrence following rejection of the first graft. Bone Marrow Transplant 2008; 42(6): 397404.CrossRefGoogle ScholarPubMed
Gaziev, J., Marziali, M., Isgrò, A., et al. Bone marrow transplantation for thalassemia from alternative related donors: improved outcomes with a new approach. Blood 2013; 122(15): 2751–6.Google ScholarPubMed
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: 1296–302.CrossRefGoogle ScholarPubMed
Locatelli, F., Rocha, V., Reed, W., et al. Related unbilical cord blood transplantation in patients with thalassemia and sickle cell disease. Blood 2003; 101: 2137–43.CrossRefGoogle Scholar
Soni, S., Boulad, F., Cowan, M.D., 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(9): 1690–4.CrossRefGoogle ScholarPubMed
Jaing, T-H., Hung, I-J., Yang, C.H., et al. Unrelated cord blood transplantation for thalassemia: a single institution experience of 35 patients. Bone Marrow Transplant 2012; 47: 3339.CrossRefGoogle ScholarPubMed
Ruggeri, A., Eapen, M., Scaravadou, A. et al. Umblical cord blood transplantation for children with thalassemia and sickle cell disease. The Eurocord Registry, the Center for Iternational Blood and Marrow Transplant Research, and the New York Blood Center. Biol Blood Marrow Transplant 2011; 17(9): 1375–82.CrossRefGoogle Scholar
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: 577–92.CrossRefGoogle ScholarPubMed
Parikh, S.H., Mendizabal, A., Benjamin, C.L., et al. A novel reduced-intensity conditioning regimen for unrelated umbilical cord blood transplantation in children with non-malignant diseases. Biol Blood Marrow Transplant 2014; 20: 326–36.CrossRefGoogle Scholar
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): 4350–6.CrossRefGoogle ScholarPubMed
Chunfu, Li., Xuedong, Wu., Feng. Xiaoping, et al. A novel conditioning regimen improves outcomes in ß-thalassemia major patients using unrelated donor peripheral blood stem cell transplantation. Blood 2012, 120: 3875–81.CrossRefGoogle Scholar
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.CrossRefGoogle ScholarPubMed
Andreani, M., Testi, M., Battarra, M., et al. Realtionship between mixed chimerism and rejection after bone marrow transplantation in thalassemia. Blood Transfus 2008; 6: 143–9.Google Scholar
Gaziev, J., Isgro, A., Marziali, M., et al. High CD3+ and CD34+ cell doses in the graft increase the incidence of acute GVHD in children receiving BMT for thalassemia. Bone Marrow Transplant 2012; 47: 107–14.CrossRefGoogle ScholarPubMed
Lucarelli, G., Angelucci, E., Giardini, C., et al. Fate of iron stores in thalassemia after bone marrow transplantation. Lancet 1993; 342: 1388–91.CrossRefGoogle ScholarPubMed
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: 1721.CrossRefGoogle ScholarPubMed

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