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Review Article| Volume 37, ISSUE 2, P413-432, April 2023

Hematopoietic Stem Cell Transplantation in Thalassemia

  • Author Footnotes
    1 These authors contributed equally.
    Mattia Algeri
    Correspondence
    Corresponding author.
    Footnotes
    1 These authors contributed equally.
    Affiliations
    Department of Hematology/Oncology, Cell and Gene Therapy – IRCCS, Bambino Gesù Children’s Hospital, Rome, Italy
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  • Author Footnotes
    1 These authors contributed equally.
    Mariachiara Lodi
    Footnotes
    1 These authors contributed equally.
    Affiliations
    Department of Hematology/Oncology, Cell and Gene Therapy – IRCCS, Bambino Gesù Children’s Hospital, Rome, Italy
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  • Author Footnotes
    1 These authors contributed equally.
    Franco Locatelli
    Footnotes
    1 These authors contributed equally.
    Affiliations
    Department of Hematology/Oncology, Cell and Gene Therapy – IRCCS, Bambino Gesù Children’s Hospital, Rome, Italy

    Department of Life Sciences and Public Health, Catholic University of the Sacred Heart, Rome, Italy
    Search for articles by this author
  • Author Footnotes
    1 These authors contributed equally.

      Keywords

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      References

        • Borgna-Pignatti C.
        • Cappellini M.D.
        • De Stefano P.
        • et al.
        Survival and complications in thalassemia.
        Ann N Y Acad Sci. 2005; 1054: 40-47
        • Kattamis A.
        • Kwiatkowski J.L.
        • Aydinok Y.
        Thalassaemia.
        Lancet Lond Engl. 2022; 399: 2310-2324
        • Taher A.T.
        • Musallam K.M.
        • Cappellini M.D.
        β-Thalassemias.
        N Engl J Med. 2021; 384: 727-743
        • Arian M.
        • Mirmohammadkhani M.
        • Ghorbani R.
        • et al.
        Health-related quality of life (HRQoL) in beta-thalassemia major (β-TM) patients assessed by 36-item short form health survey (SF-36): a meta-analysis.
        Qual Life Res Int J Qual Life Asp Treat Care Rehabil. 2019; 28: 321-334
        • Sobota A.
        • Yamashita R.
        • Xu Y.
        • et al.
        Quality of life in thalassemia: a comparison of SF-36 results from the thalassemia longitudinal cohort to reported literature and the US norms.
        Am J Hematol. 2011; 86: 92-95
        • Lal A.
        Challenges in chronic transfusion for patients with thalassemia.
        Hematol Am Soc Hematol Educ Program. 2020; 2020: 160-166
        • Taher A.T.
        • Cappellini M.D.
        How I manage medical complications of β-thalassemia in adults.
        Blood. 2018; 132: 1781-1791
        • Borgna-Pignatti C.
        • Rugolotto S.
        • De Stefano P.
        • et al.
        Survival and complications in patients with thalassemia major treated with transfusion and deferoxamine.
        Haematologica. 2004; 89: 1187-1193
        • Cappellini M.D.
        • Viprakasit V.
        • Taher A.T.
        • et al.
        A Phase 3 Trial of Luspatercept in Patients with Transfusion-Dependent β-Thalassemia.
        N Engl J Med. 2020; 382: 1219-1231
        • Weatherall D.J.
        The inherited diseases of hemoglobin are an emerging global health burden.
        Blood. 2010; 115: 4331-4336
        • Locatelli F.
        • Thompson A.A.
        • Kwiatkowski J.L.
        • et al.
        Betibeglogene Autotemcel Gene Therapy for Non-β0/β0 Genotype β-Thalassemia.
        N Engl J Med. 2022; 386: 415-427
        • Frangoul H.
        • Altshuler D.
        • Cappellini M.D.
        • et al.
        CRISPR-Cas9 Gene Editing for Sickle Cell Disease and β-Thalassemia.
        N Engl J Med. 2021; 384: 252-260
        • Locatelli F.
        • Frangoul H.
        • Corbacioglu S.
        • et al.
        EFFICACY AND SAFETY OF A SINGLE DOSE OF CTX001 FOR TRANSFUSION-DEPENDENT BETA-THALASSEMIA AND SEVERE SICKLE CELL DISEASE. EHA Library.
        (Available at:)
        • Baronciani D.
        • Angelucci E.
        • Potschger U.
        • et al.
        Hemopoietic stem cell transplantation in thalassemia: a report from the European Society for Blood and Bone Marrow Transplantation Hemoglobinopathy Registry, 2000-2010.
        Bone Marrow Transplant. 2016; 51: 536-541
        • Li C.
        • Mathews V.
        • Kim S.
        • et al.
        Related and unrelated donor transplantation for β-thalassemia major: results of an international survey.
        Blood Adv. 2019; 3: 2562-2570
        • Lucarelli G.
        • Galimberti M.
        • Polchi P.
        • et al.
        Bone marrow transplantation in patients with thalassemia.
        N Engl J Med. 1990; 322: 417-421
        • Giardini C.
        • Lucarelli G.
        Bone marrow transplantation for beta-thalassemia.
        Hematol Oncol Clin North Am. 1999; 13 (viii): 1059-1064
        • Lucarelli G.
        • Galimberti M.
        • Giardini C.
        • et al.
        Bone marrow transplantation in thalassemia. The experience of Pesaro.
        Ann N Y Acad Sci. 1998; 850: 270-275
        • Lucarelli G.
        • Clift R.A.
        • Galimberti M.
        • et al.
        Bone marrow transplantation in adult thalassemic patients.
        Blood. 1999; 93: 1164-1167
        • Angelucci E.
        • Pilo F.
        • Coates T.D.
        Transplantation in thalassemia: Revisiting the Pesaro risk factors 25 years later.
        Am J Hematol. 2017; 92: 411-413
        • 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 J Am Soc Blood Marrow Transplant. 2006; 12: 683-687
        • Fang J.-P.
        • Xu L.-H.
        Hematopoietic stem cell transplantation for children with thalassemia major in China.
        Pediatr Blood Cancer. 2010; 55: 1062-1065
        • 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 J Am Soc Blood Marrow Transplant. 2007; 13: 889-894
        • Andreani M.
        • Testi M.
        • Lucarelli G.
        Mixed chimerism in haemoglobinopathies: from risk of graft rejection to immune tolerance.
        Tissue Antigens. 2014; 83: 137-146
        • Angelucci E.
        • Baronciani D.
        Allogeneic stem cell transplantation for thalassemia major.
        Haematologica. 2008; 93: 1780-1784
        • Thompson A.A.
        • Cunningham M.J.
        • Singer S.T.
        • et al.
        Red cell alloimmunization in a diverse population of transfused patients with thalassaemia.
        Br J Haematol. 2011; 153: 121-128
        • Centis F.
        • Tabellini L.
        • Lucarelli G.
        • et al.
        The importance of erythroid expansion in determining the extent of apoptosis in erythroid precursors in patients with beta-thalassemia major.
        Blood. 2000; 96: 3624-3629
        • Isidori A.
        • Loscocco F.
        • Visani G.
        • et al.
        Iron Toxicity and Chelation Therapy in Hematopoietic Stem Cell Transplant.
        Transplant Cell Ther. 2021; 27: 371-379
        • Mathews V.
        • Savani B.N.
        Conditioning regimens in allo-SCT for thalassemia major.
        Bone Marrow Transplant. 2014; 49: 607-610
        • Lucarelli G.
        • Clift R.A.
        • Galimberti M.
        • et al.
        Marrow transplantation for patients with thalassemia: results in class 3 patients.
        Blood. 1996; 87: 2082-2088
        • 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 treosulfan.
        Blood. 2012; 120: 473-476
        • Li C.
        • Wu X.
        • Feng X.
        • et al.
        A novel conditioning regimen improves outcomes in β-thalassemia major patients using unrelated donor peripheral blood stem cell transplantation.
        Blood. 2012; 120: 3875-3881
        • Bertaina A.
        • Bernardo M.E.
        • Mastronuzzi A.
        • et al.
        The role of reduced intensity preparative regimens in patients with thalassemia given hematopoietic transplantation.
        Ann N Y Acad Sci. 2010; 1202: 141-148
        • 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 R.A.
        • Harstrick A.
        • Eberhardt W.
        • et al.
        Clinical pharmacokinetics of intravenous treosulfan in patients with advanced solid tumors.
        Cancer Chemother Pharmacol. 1998; 42: 99-104
        • Główka F.K.
        • Karaźniewicz-Łada 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: S67-S70
        • Mathews V.
        • George B.
        • Viswabandya A.
        • et al.
        Improved clinical outcomes of high risk β 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
        • Lüftinger R.
        • Zubarovskaya N.
        • Galimard J.-E.
        • et al.
        Busulfan-fludarabine- or treosulfan-fludarabine-based myeloablative conditioning for children with thalassemia major.
        Ann Hematol. 2022; 101: 655-665
        • Shenoy S.
        • Walters M.C.
        • Ngwube A.
        • et al.
        Unrelated Donor Transplantation in Children with Thalassemia using Reduced-Intensity Conditioning: The URTH Trial.
        Biol Blood Marrow Transplant J Am Soc Blood Marrow Transplant. 2018; 24: 1216-1222
        • 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: 2751-2756
        • Anurathapan U.
        • Hongeng S.
        • Pakakasama S.
        • et al.
        Hematopoietic Stem Cell Transplantation for Severe Thalassemia Patients from Haploidentical Donors Using a Novel Conditioning Regimen.
        Biol Blood Marrow Transplant J Am Soc Blood Marrow Transplant. 2020; 26: 1106-1112
        • Anurathapan U.
        • Pakakasama S.
        • Mekjaruskul P.
        • et al.
        Outcomes of thalassemia patients undergoing hematopoietic stem cell transplantation by using a standard myeloablative versus a novel reduced-toxicity conditioning regimen according to a new risk stratification.
        Biol Blood Marrow Transplant J Am Soc Blood Marrow Transplant. 2014; 20: 2066-2071
        • Lucarelli G.
        • Gaziev J.
        Advances in the allogeneic transplantation for thalassemia.
        Blood Rev. 2008; 22: 53-63
        • Isgrò A.
        • Gaziev J.
        • Sodani P.
        • et al.
        Progress in hematopoietic stem cell transplantation as allogeneic cellular gene therapy in thalassemia.
        Ann N Y Acad Sci. 2010; 1202: 149-154
        • 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: 1072-1078
        • Locatelli F.
        • Rocha V.
        • Reed W.
        • et al.
        Related umbilical cord blood transplantation in patients with thalassemia and sickle cell disease.
        Blood. 2003; 101: 2137-2143
        • Tucunduva L.
        • Volt F.
        • Cunha R.
        • et al.
        Combined cord blood and bone marrow transplantation from the same human leucocyte antigen-identical sibling donor for children with malignant and non-malignant diseases.
        Br J Haematol. 2015; 169: 103-110
        • Iravani M.
        • Tavakoli E.
        • Babaie M.H.
        • et al.
        Comparison of peripheral blood stem cell transplant with bone marrow transplant in class 3 thalassemic patients.
        Exp Clin Transplant Off J Middle East Soc Organ 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 J Am Soc Blood Marrow Transplant. 2008; 14: 301-308
        • Irfan M.
        • Hashmi K.
        • Adil S.
        • et al.
        Beta-thalassaemia major: bone marrow versus peripheral blood stem cell transplantation.
        JPMA J Pak Med Assoc. 2008; 58: 107-110
        • Baronciani D.
        • Pilo F.
        • Lyon-Caen S.
        • et al.
        Hematopoietic Stem Cell Transplantation in Thalassemia Major. Report from the EBMT Hemoglobinopathy Registry.
        Blood. 2011; 118: 905
        • He Y.
        • Jiang H.
        • Li C.
        • et al.
        Long-term results of the NF-08-TM protocol in stem cell transplant for patients with thalassemia major: A multi-center large-sample study.
        Am J Hematol. 2020; 95: E297-E299
        • Rocha V.
        • Locatelli F.
        Searching for alternative hematopoietic stem cell donors for pediatric patients.
        Bone Marrow Transplant. 2008; 41: 207-214
        • Gragert L.
        • Eapen M.
        • Williams E.
        • et al.
        HLA match likelihoods for hematopoietic stem-cell grafts in the U.S. registry.
        N Engl J Med. 2014; 371: 339-348
        • La Nasa G.
        • Giardini C.
        • Argiolu F.
        • et al.
        Unrelated donor bone marrow transplantation for thalassemia: the effect of extended haplotypes.
        Blood. 2002; 99: 4350-4356
        • La Nasa G.
        • Caocci G.
        • Argiolu F.
        • et al.
        Unrelated donor stem cell transplantation in adult patients with thalassemia.
        Bone Marrow Transplant. 2005; 36: 971-975
        • Sun L.
        • Wang N.
        • Chen Y.
        • et al.
        Unrelated Donor Peripheral Blood Stem Cell Transplantation for Patients with β-Thalassemia Major Based on a Novel Conditioning Regimen.
        Biol Blood Marrow Transplant J Am Soc Blood Marrow Transplant. 2019; 25: 1592-1596
        • Locatelli F.
        • Littera R.
        • Pagliara D.
        • et al.
        Outcome of unrelated donor bone marrow transplantation for thalassemia major patients.
        Blood. 2011; 118: 149
        • La Nasa G.
        • Littera R.
        • Locatelli F.
        • et al.
        Status of donor-recipient HLA class I ligands and not the KIR genotype is predictive for the outcome of unrelated hematopoietic stem cell transplantation in beta-thalassemia patients.
        Biol Blood Marrow Transplant J Am Soc Blood Marrow Transplant. 2007; 13: 1358-1368
        • La Nasa G.
        • Littera R.
        • Locatelli F.
        • et al.
        The human leucocyte antigen-G 14-basepair polymorphism correlates with graft-versus-host disease in unrelated bone marrow transplantation for thalassaemia.
        Br J Haematol. 2007; 139: 284-288
        • Littera R.
        • Orrù N.
        • Vacca A.
        • et al.
        The role of killer immunoglobulin-like receptor haplotypes on the outcome of unrelated donor haematopoietic SCT for thalassaemia.
        Bone Marrow Transplant. 2010; 45: 1618-1624
        • 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: 2984-2992
        • Ruggeri A.
        • Eapen M.
        • Scaravadou A.
        • et al.
        Umbilical cord blood transplantation for children with thalassemia and sickle cell disease.
        Biol Blood Marrow Transplant J Am Soc Blood Marrow Transplant. 2011; 17: 1375-1382
        • Jaing T.-H.
        • Hung I.-J.
        • Yang C.-P.
        • et al.
        Unrelated cord blood transplantation for thalassaemia: a single-institution experience of 35 patients.
        Bone Marrow Transplant. 2012; 47: 33-39
        • Gluckman E.
        Milestones in umbilical cord blood transplantation.
        Blood Rev. 2011; 25: 255-259
        • Shah S.A.
        • Shah K.M.
        • Patel K.A.
        • et al.
        Unrelated Umbilical Cord Blood Transplant for Children with β-Thalassemia Major.
        Indian J Hematol Blood Transfus. 2015; 31: 9-13
        • Eapen M.
        • Wang T.
        • Veys P.A.
        • et al.
        Allele-level HLA matching for umbilical cord blood transplantation for non-malignant diseases in children: a retrospective analysis.
        Lancet Haematol. 2017; 4: e325-e333
        • Sodani P.
        • Isgrò 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
        • Yee M.E.M.
        • Shah A.
        • Anderson A.R.
        • et al.
        Class I and II HLA antibodies in pediatric patients with thalassemia major.
        Transfusion (Paris). 2016; 56: 878-884
        • Ciurea S.O.
        • Al Malki M.M.
        • Kongtim P.
        • et al.
        The European Society for Blood and Marrow Transplantation (EBMT) consensus recommendations for donor selection in haploidentical hematopoietic cell transplantation.
        Bone Marrow Transplant. 2020; 55: 12-24
        • Handgretinger R.
        New approaches to graft engineering for haploidentical bone marrow transplantation.
        Semin Oncol. 2012; 39: 664-673
        • Schumm M.
        • Lang P.
        • Bethge W.
        • et al.
        Depletion of T-cell receptor alpha/beta and CD19 positive cells from apheresis products with the CliniMACS device.
        Cytotherapy. 2013; 15: 1253-1258
        • Bertaina A.
        • Lucarelli B.
        • Merli P.
        • et al.
        Clinical Outcome after Adoptive Infusion of BPX-501 Cells (donor T cells transduced with iC9 suicide gene) in Children with Thalassemia Major (TM) Given Alfa/Beta T-Cell Depleted HLA-Haploidentical Stem Cell Transplantation (HSCT).
        Biol Blood Marrow Transplant. 2016; 22: S306
        • Farnault L.
        • Gertner-Dardenne J.
        • Gondois-Rey F.
        • et al.
        Clinical evidence implicating gamma-delta T cells in EBV control following cord blood transplantation.
        Bone Marrow Transplant. 2013; 48: 1478-1479
        • Airoldi I.
        • Bertaina A.
        • Prigione I.
        • et al.
        γδ T-cell reconstitution after HLA-haploidentical hematopoietic transplantation depleted of TCR-αβ+/CD19+ lymphocytes.
        Blood. 2015; 125: 2349-2358
        • Gaziev J.
        • Isgrò A.
        • Sodani P.
        • et al.
        Haploidentical HSCT for hemoglobinopathies: improved outcomes with TCRαβ+/CD19+-depleted grafts.
        Blood Adv. 2018; 2: 263-270
        • Merli P.
        • Pagliara D.
        • Galaverna F.
        • et al.
        TCRαβ/CD19 depleted HSCT from an HLA-haploidentical relative to treat children with different nonmalignant disorders.
        Blood Adv. 2022; 6: 281-292
        • Merli P.
        • Caruana I.
        • De Vito R.
        • et al.
        Role of interferon-γ in immune-mediated graft failure after allogeneic hematopoietic stem cell transplantation.
        Haematologica. 2019; 104: 2314-2323
        • Sabulski A.
        • Myers K.C.
        • Bleesing J.J.
        • et al.
        Graft rejection markers in children undergoing hematopoietic cell transplant for bone marrow failure.
        Blood Adv. 2021; 5: 4594-4604
        • Galaverna F.
        • Li Pira G.
        • Algeri M.
        • et al.
        Alpha/beta T-cell depleted Haploidentical HSCT followed by infusion of donor lymphocytes transduced with inducible caspase9 gene is safe and effective for patients with erythroid disorders. Abstract 0039. The 44th Annual Meeting of the European Society for Blood and Marrow Transplantation: Physicians Oral Session.
        Bone Marrow Transplant. 2019; 53: 19
        • Luznik L.
        • Fuchs E.J.
        High-dose, posttransplantation cyclophosphamide to promote graft-host tolerance after allogeneic hematopoietic stem cell transplantation.
        Immunol Res. 2010; 47: 65-77
        • Bolaños-Meade J.
        • Fuchs E.J.
        • Luznik L.
        • et al.
        HLA-haploidentical bone marrow transplantation with posttransplant cyclophosphamide expands the donor pool for patients with sickle cell disease.
        Blood. 2012; 120: 4285-4291
        • Sun Q.
        • Wu B.
        • Lan H.
        • et al.
        Haploidentical haematopoietic stem cell transplantation for thalassaemia major based on an FBCA conditioning regimen.
        Br J Haematol. 2018; 182: 554-558
        • Andreani M.
        • Gregori S.
        The study of engraftment after hematopoietic stem cell transplantation: From the presence of mixed chimerism to the development of immunological tolerance.
        HLA. 2018; 92: 57-59
        • Fouzia N.A.
        • Edison E.S.
        • Lakshmi K.M.
        • et al.
        Long-term outcome of mixed chimerism after stem cell transplantation for thalassemia major conditioned with busulfan and cyclophosphamide.
        Bone Marrow Transplant. 2018; 53: 169-174
        • Andreani M.
        • Manna M.
        • Lucarelli G.
        • et al.
        Persistence of mixed chimerism in patients transplanted for the treatment of thalassemia.
        Blood. 1996; 87: 3494-3499
        • Andreani M.
        • Nesci S.
        • Lucarelli G.
        • et al.
        Long-term survival of ex-thalassemic patients with persistent mixed chimerism after bone marrow transplantation.
        Bone Marrow Transplant. 2000; 25: 401-404
        • Andreani M.
        • Testi M.
        • Gaziev J.
        • et al.
        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. 2011; 96: 128-133
        • Alfred A.
        • Vora A.J.
        What is the minimum level of donor chimerism necessary to sustain transfusion independence in thalassaemia?.
        Bone Marrow Transplant. 2011; 46: 769-770
        • Caocci G.
        • Orofino M.G.
        • Vacca A.
        • et al.
        Long-term survival of beta thalassemia major patients treated with hematopoietic stem cell transplantation compared with survival with conventional treatment.
        Am J Hematol. 2017; 92: 1303-1310
        • La Nasa G.
        • Caocci G.
        • Efficace F.
        • et al.
        Long-term health-related quality of life evaluated more than 20 years after hematopoietic stem cell transplantation for thalassemia.
        Blood. 2013; 122: 2262-2270
        • Shenoy S.
        • Gaziev J.
        • Angelucci E.
        • et al.
        Late Effects Screening Guidelines after Hematopoietic Cell Transplantation (HCT) for Hemoglobinopathy: Consensus Statement from the Second Pediatric Blood and Marrow Transplant Consortium International Conference on Late Effects after Pediatric HCT.
        Biol Blood Marrow Transplant J Am Soc Blood Marrow Transplant. 2018; 24: 1313-1321
        • Chaudhury S.
        • Ayas M.
        • Rosen C.
        • et al.
        A Multicenter Retrospective Analysis Stressing the Importance of Long-Term Follow-Up after Hematopoietic Cell Transplantation for β-Thalassemia.
        Biol Blood Marrow Transplant J Am Soc Blood Marrow Transplant. 2017; 23: 1695-1700
        • Rahal I.
        • Galambrun C.
        • Bertrand Y.
        • et al.
        Late effects after hematopoietic stem cell transplantation for β-thalassemia major: the French national experience.
        Haematologica. 2018; 103: 1143-1149
        • Thuret I.
        • Ruggeri A.
        • Angelucci E.
        • et al.
        Hurdles to the Adoption of Gene Therapy as a Curative Option for Transfusion-Dependent Thalassemia.
        Stem Cells Transl Med. 2022; 11: 407-414
        • Mathews V.
        • Srivastava A.
        • Chandy M.
        Allogeneic stem cell transplantation for thalassemia major.
        Hematol Oncol Clin North Am. 2014; 28: 1187-1200
        • Scalone L.
        • Mantovani L.G.
        • Krol M.
        • et al.
        Costs, quality of life, treatment satisfaction and compliance in patients with beta-thalassemia major undergoing iron chelation therapy: the ITHACA study.
        Curr Med Res Opin. 2008; 24: 1905-1917
        • John M.J.
        • Jyani G.
        • Jindal A.
        • et al.
        Cost Effectiveness of Hematopoietic Stem Cell Transplantation Compared with Transfusion Chelation for Treatment of Thalassemia Major.
        Biol Blood Marrow Transplant J Am Soc Blood Marrow Transplant. 2018; 24: 2119-2126