Thomas ED, Buckner CD, Sanders JE, et al. Marrow transplantation for thalassaemia. Lancet. 1982;2:227–9.
Lucarelli G, Polchi P, Galimberti M, et al. Marrow transplantation for thalassaemia following busulphan and cyclophosphamide. Lancet. 1985;1:1355–7.
Bhatia M, Walters MC. Hematopoietic cell transplantation for thalassemia and sickle cell disease: past, present and future. Bone Marrow Transplant. 2008;41:109–17.
Lucarelli G, Galimberti M, Polchi P, et al. Bone marrow transplantation in patients with thalassemia. N Engl J Med. 1990;322:417–21.
Lucarelli G, Galimberti M, Polchi P, et al. Bone marrow transplantation in adult thalassemia. Blood. 1992;80:1603–7.
Mathews V, George B, Deotare U, et al. A new stratification strategy that identifies a subset of class III patients with an adverse prognosis among children with beta thalassemia major undergoing a matched related allogeneic stem cell transplantation. Biol Blood Marrow Transplant. 2007;13:889–94.
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.
Fang JP, Xu LH. Hematopoietic stem cell transplantation for children with thalassemia major in China. Pediatr Blood Cancer. 2010;55:1062–5.
Sabloff M, Chandy M, Wang Z, et al. HLA-matched sibling bone marrow transplantation for beta-thalassemia major. Blood. 2010;117:1745–50.
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;16:622–8.
Lucarelli G, Clift RA, Galimberti M, et al. Marrow transplantation for patients with thalassemia: results in class 3 patients. Blood. 1996;87:2082–8.
Sodani P, Gaziev D, 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.
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;115:4597–604.
Bernardo ME, 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 treosulfan. Blood. 2012;120:473–6.
Choudhary D, Sharma SK, Gupta N, et al. Treosulfan-thiotepa-fludarabine-based conditioning regimen for allogeneic transplantation in patients with thalassemia major: a single-center experience from north India. Biol Blood Marrow Transplant. 2012;19:492–5.
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;19:1259–62.
Mathews V, George B, Viswabandya A, et al. Improved clinical outcomes of high risk beta thalassemia major patients undergoing a HLA matched related allogeneic stem cell transplant with a treosulfan based conditioning regimen and peripheral blood stem cell grafts. PLoS One. 2013;8:e61637.
Lawson SE, Roberts IA, Amrolia P, Dokal I, Szydlo R, Darbyshire PJ. Bone marrow transplantation for beta-thalassaemia major: the UK experience in two paediatric centres. Br J Haematol. 2003;120:289–95.
Bernardo ME, Zecca M, Piras E, et al. Treosulfan-based conditioning regimen for allogeneic haematopoietic stem cell transplantation in patients with thalassaemia major. Br J Haematol. 2008;143:548–51.
Iannone R, Casella JF, Fuchs EJ, et al. Results of minimally toxic nonmyeloablative transplantation in patients with sickle cell anemia and beta-thalassemia. Biol Blood Marrow Transplant. 2003;9:519–28.
Horan JT, Liesveld JL, Fenton P, et al. Hematopoietic stem cell transplantation for multiply transfused patients with sickle cell disease and thalassemia after low-dose total body irradiation, fludarabine, and rabbit anti-thymocyte globulin. Bone Marrow Transplant. 2005;35:171–7.
Danylesko I, Shimoni A, Nagler A. Treosulfan-based conditioning before hematopoietic SCT: more than a BU look-alike. Bone Marrow Transplant. 2012;47:5–14.
Hilger RA, Harstrick A, Eberhardt W, et al. Clinical pharmacokinetics of intravenous treosulfan in patients with advanced solid tumors. Cancer Chemother Pharmacol. 1998;42:99–104.
Glowka FK, Karazniewicz-Lada M, Grund G, et al. Pharmacokinetics of high-dose i.v. treosulfan in children undergoing treosulfan-based preparative regimen for allogeneic haematopoietic SCT. Bone Marrow Transplant. 2008;42(Suppl 2):S67–70.
Scheulen ME, Hilger RA, Oberhoff C, et al. Clinical phase I dose escalation and pharmacokinetic study of high-dose chemotherapy with treosulfan and autologous peripheral blood stem cell transplantation in patients with advanced malignancies. Clin Cancer Res. 2000;6:4209–16.
Cappelli B, Chiesa R, Evangelio C, et al. Absence of VOD in paediatric thalassaemic HSCT recipients using defibrotide prophylaxis and intravenous Busulphan. Br J Haematol. 2009;147:554–60.
Cesaro S, Pillon M, Talenti E, et al. A prospective survey on incidence, risk factors and therapy of hepatic veno-occlusive disease in children after hematopoietic stem cell transplantation. Haematologica. 2005;90:1396–404.
de Witte T. The role of iron in patients after bone marrow transplantation. Blood Rev. 2008;22(Suppl 2):S22–8.
Srivastava A, Poonkuzhali B, Shaji RV, et al. Glutathione S-transferase M1 polymorphism: a risk factor for hepatic venoocclusive disease in bone marrow transplantation. Blood. 2004;104:1574–7.
Chandy M, Balasubramanian P, Ramachandran SV, 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.
Rajasekar R, Mathews V, Lakshmi KM, et al. Cellular immune reconstitution and its impact on clinical outcome in children with beta thalassemia major undergoing a matched related myeloablative allogeneic bone marrow transplant. Biol Blood Marrow Transplant. 2009;15:597–609.
Iravani M, Tavakoli E, Babaie MH, et al. Comparison of peripheral blood stem cell transplant with bone marrow transplant in class 3 thalassemic patients. Exp Clin Transplant. 2010;8:66–73.
Ghavamzadeh A, Iravani M, 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;14:301–8.
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;25:815–21.
La Nasa G, Giardini C, Argiolu F, et al. Unrelated donor bone marrow transplantation for thalassemia: the effect of extended haplotypes. Blood. 2002;99:4350–6.
Fleischhauer K, Locatelli F, Zecca M, et al. Graft rejection after unrelated donor hematopoietic stem cell transplantation for thalassemia is associated with nonpermissive HLA-DPB1 disparity in host-versus-graft direction. Blood. 2006;107:2984–92.
Locatelli F, Rocha V, Reed W, et al. Related umbilical cord blood transplantation in patients with thalassemia and sickle cell disease. Blood. 2003;101:2137–43.
Lisini D, Zecca M, Giorgiani G, et al. Donor/recipient mixed chimerism does not predict graft failure in children with beta-thalassemia given an allogeneic cord blood transplant from an HLA-identical sibling. Haematologica. 2008;93:1859–67.
Walters MC, Quirolo L, Trachtenberg ET, et al. Sibling donor cord blood transplantation for thalassemia major: Experience of the Sibling Donor Cord Blood Program. Ann N Y Acad Sci. 2005;1054:206–13.
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;122:1072–8.
Pinto FO, Roberts I. Cord blood stem cell transplantation for haemoglobinopathies. Br J Haematol. 2008;141:309–24.
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;17:1375–82.
Bertaina A, Merli P, Rutella S, et al. HLA-haploidentical stem cell transplantation after removal of alphabeta+ T and B-cells in children with non-malignant disorders. Blood. 2014;124:822–6.
Bittencourt H, Rocha V, Chevret S, et al. Association of CD34 cell dose with hematopoietic recovery, infections, and other outcomes after HLA-identical sibling bone marrow transplantation. Blood. 2002;99:2726–33.
Rund D, Rachmilewitz E. Beta-thalassemia. N Engl J Med. 2005;353:1135–46.
Panigrahi I, Marwaha RK. Common queries in thalassemia care. Indian Pediatr. 2006;43:513–8.
Shatry AM, Jones M, Levy RB. The effect of the spleen on compartmental levels and distribution of donor progenitor cells after syngeneic and allogeneic bone marrow transplants. Stem Cells Dev. 2004;13:51–62.
Li Z, Gooley T, Applebaum FR, et al. Splenectomy and hematopoietic stem cell transplantation for myelofibrosis. Blood. 2001;97:2180–1.
Phrommintikul A, Sukonthasarn A, Kanjanavanit R, et al. Splenectomy: a strong risk factor for pulmonary hypertension in patients with thalassaemia. Heart. 2006;92:1467–72.
Tripatara A, Jetsrisuparb A, Teeratakulpisarn J, et al. Hemostatic alterations in splenectomized and non-splenectomized patients with beta-thalassemia/hemoglobin E disease. Thromb Res. 2007;120:805–10.
Pattanapanyasat K, Gonwong S, Chaichompoo P, et al. Activated platelet-derived microparticles in thalassaemia. Br J Haematol. 2007;136:462–71.
Singer ST, Kuypers FA, Styles L, et al. Pulmonary hypertension in thalassemia: association with platelet activation and hypercoagulable state. Am J Hematol. 2006;81:670–5.
Robin M, Guardiola P, Devergie A, et al. A 10-year median follow-up study after allogeneic stem cell transplantation for chronic myeloid leukemia in chronic phase from HLA-identical sibling donors. Leukemia. 2005;19:1613–20.
Mathews V, George B, Lakshmi KM, et al. Impact of pretransplant splenectomy on patients with beta-thalassemia major undergoing a matched-related allogeneic stem cell transplantation. Pediatr Transplant. 2009;13:171–6.
Bhatia M, Cairo MS. Splenectomy or no splenectomy prior to allogeneic stem-cell transplantation in patients with severe thalassemia: this is the question. Pediatr Transplant. 2009;13:143–5.
Angelucci E, Muretto P, Lucarelli G, et al. Phlebotomy to reduce iron overload in patients cured of thalassemia by bone marrow transplantation. Italian Cooperative Group for Phlebotomy Treatment of Transplanted Thalassemia Patients. Blood. 1997;90:994–8.