Skip to main content

Thank you for visiting nature.com. You are using a browser version with limited support for CSS. To obtain the best experience, we recommend you use a more up to date browser (or turn off compatibility mode in Internet Explorer). In the meantime, to ensure continued support, we are displaying the site without styles and JavaScript.

  • Original Article
  • Published:

Inherited and Genetic Disorders

Hemopoietic stem cell transplantation in thalassemia: a report from the European Society for Blood and Bone Marrow Transplantation Hemoglobinopathy Registry, 2000–2010

Bone Marrow Transplantation volume 51, pages 536–541 (2016)Cite this article

Subjects

Abstract

Allogeneic hemopoietic stem cell transplantation (HSCT) is the only method currently available to cure transfusion-dependent thalassemia major that has been widely used worldwide. To verify transplantation distribution, demography, activity, policies and outcomes inside the European Group for Blood and Marrow Transplantation (EBMT), we performed a retrospective non-interventional study, extracting data from the EBMT hemoglobinopathy prospective registry database. We included 1493 consecutive patients with thalassemia major transplanted between 1 January 2000 and 31 December 2010. In total, 1359 (91%) transplants were performed on patients <18 years old, 1061 were from a human leukocyte Ag-identical sibling donor. After a median observation time of 2 years, the 2-year overall survival (OS) and event-free survival (EFS; that is, thalassemia-free survival) were 88±1% and 81±1%, respectively. Transplantation from a human leukocyte Ag-identical sibling offered the best results, with OS and EFS of 91±1% and 83±1%, respectively. No significant differences in survival were reported between countries. The threshold age for optimal transplant outcomes was around 14 years, with an OS of 90–96% and an EFS of 83–93% when transplants were performed before this age. Allogeneic HSCT for thalassemia is a curative approach that is employed internationally and produces excellent results.

This is a preview of subscription content, access via your institution

Access options

Buy this article

  • Purchase on Springer Link
  • Instant access to full article PDF
Buy now

Prices may be subject to local taxes which are calculated during checkout

Figure 1
Figure 2
Figure 3
Figure 4
Figure 5
Figure 6

Similar content being viewed by others

References

  1. Thomas ED, Buckner CD, Sanders JE, Papayannopoulou T, Borgna-Pignatti C, De Stefano P et al. Marrow transplantation for thalassaemia. Lancet 1982; 2: 227–229.

    Article  CAS  PubMed  Google Scholar 

  2. Lucarelli G, Polchi P, Izzi T, Manna M, Agostinelli F, Delfini C et al. Allogeneic marrow transplantation for thalassemia. Exp Hematol 1984; 12: 676–681.

    CAS  PubMed  Google Scholar 

  3. Angelucci E, Matthes-Martin S, Baronciani D, Bernadine F, Bonanomi S, Cappellini MD et al. Hematopoietic stem cell transplantation in thalassemia major and sickle cell disease: Indications and management recommendations from an international expert panel. Haematologica 2014; 99: 811–820.

    Article  PubMed  PubMed Central  Google Scholar 

  4. Przepiorka D, Weisdorf D, Martin P, Klingemann HG, Beatty P, Hows J et al. 1994 Consensus Conference on Acute GVHD Grading. Bone Marrow Transplant 1995; 15: 825–828.

    CAS  PubMed  Google Scholar 

  5. Marubini E, Valsecchi MG (eds). Analysing Survival Data from Clinical Trials and Observational Studies. Wiley: New York, 2004.

    Google Scholar 

  6. 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 1990; 322: 417–421.

    Article  CAS  PubMed  Google Scholar 

  7. Lucarelli G, Galimberti M, Polchi P, Angelucci E, Baronciani D, Giardini C et al. Marrow transplantation in patients with thalassemia responsive to iron chelation therapy. N Engl J Med 1993; 329: 840–844.

    Article  CAS  PubMed  Google Scholar 

  8. Lucarelli G, Galimberti M, Polchi P, Giardini C, Politi P, Baronciani D et al. Marrow transplantation in patients with advanced thalassemia. N Engl J Med 1987; 316: 1050–1055.

    Article  CAS  PubMed  Google Scholar 

  9. Modell B, Darlison M . Global epidemiology of haemoglobin disorders and derived service indicators. Bull World Health Organ 2008; 86: 480–487.

    Article  PubMed  PubMed Central  Google Scholar 

  10. Sabloff M, Chandy M, Wang Z, Logan BR, Ghavamzadeh A, Li CK et al. HLA-matched sibling bone marrow transplantation for beta-thalassemia major. Blood 2011; 117: 1745–1750.

    Article  CAS  PubMed  PubMed Central  Google Scholar 

  11. Mathews V, George B, Deotare U, Lakshmi KM, Viswabandya A, Daniel D 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: 889–894.

    Article  PubMed  Google Scholar 

  12. Angelucci E . Hematopoietic stem cell transplantation in thalassemia. Hematology Am Soc Hematol Educ Program 2010; 2010: 456–462.

    Article  PubMed  Google Scholar 

  13. Lucarelli G, Clift RA, Galimberti M, Angelucci E, Giardini C, Baronciani D et al. Bone marrow transplantation in adult thalassemic patients. Blood 1999; 93: 1164–1167.

    CAS  PubMed  Google Scholar 

  14. Michlitsch JG, Walters MC . Recent advances in bone marrow transplantation in hemoglobinopathies. Curr Mol Med 2008; 8: 675–689.

    Article  CAS  PubMed  Google Scholar 

  15. Li C, Wu X, Feng X, He Y, Liu H, Pei F 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.

    Article  CAS  PubMed  Google Scholar 

  16. Choudhary D, Sharma S, Gupta N, Kharya G, Pavecha P, Handoo A 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 2013; 19: 492–495.

    Article  CAS  PubMed  Google Scholar 

  17. Agarwal MB . Advances in management of Thalassemia. Indian J Pediatr 2009; 76: 177–184.

    Article  CAS  PubMed  Google Scholar 

  18. Anurathapan U, Pakakasama S, Rujkijyanont P, Sirachainan N, Songdej D, Chuansumrit A 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: 1259–1262.

    Article  PubMed  PubMed Central  Google Scholar 

  19. Mathews V, George B, Viswabandya A, Abraham A, Ahmed R, Ganapule 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.

    Article  CAS  PubMed  PubMed Central  Google Scholar 

  20. Cavazzana-Calvo M, Payen E, Negre O, Wang G, Heir K, Fusil F et al. Transfusion independence and HMGA2 activation after gene therapy of human β-thalassaemia. Nature 2010; 467: 318–322.

    Article  CAS  PubMed  PubMed Central  Google Scholar 

Download references

Acknowledgements

We thank Arnaud Dalissier (study coordinator for the EBMT Paediatrics Working Party, Paris) for coordination of the study, data collection and data managements, and Evgenia Glocova (Children Cancer Research Institute, Vienna) for additional statistical support in the analysis. We thank Enago (www.enago.com) for the English language review.

Author information

Authors and Affiliations

  1. Ematologia e Centro Trapianti, Ospedale Oncologico di Riferimento Regionale ‘Armando Businco’, Azienda Ospedaliera ‘Brotzu’, Cagliari, Italy

    D Baronciani, E Angelucci, C Targhetta & F Pilo

  2. Department of Pediatrics, St Anna Children’s Hospital, Medical University, Vienna, Austria

    U Potschger & C Peters

  3. International Center for Transplantation in Thalassemia and Sickle Cell Anemia, Rome, Italy

    J Gaziev

  4. Pediatric Stem Cell Transplantation Unit, Akdeniz University School of Medicine, Antalya, Turkey

    A Yesilipek

  5. Department of Pediatric Hematology/Oncology, Fondazione IRCCS Policlinico ‘San Matteo’, Pavia, Italy

    M Zecca

  6. Department of Biomedical Science and Biotechnology, Second Paediatric Clinic, Bone Marrow Transplant Unit, University of Cagliari, Cagliari, Italy

    M G Orofino

  7. Ematologia e Centro Trapianti, Ospedali Riuniti Marche Nord, Pesaro, Italy

    C Giardini

  8. Department of PHO and Stem Cell Transplantation, King Faisal Specialist Hospital and Research Centre, Riyadh, Saudi Arabia

    A Al-Ahmari

  9. Stem Cells Programme, IRCCS San Raffaele Scientific Institute, Milan, Italy

    S Marktel

  10. Division of Paediatrics, St Mary Hospital, London, UK

    J de la Fuente

  11. Hematology, Oncology and Stem Cell Transplantation Research Center, Tehran University of Medical Sciences, Tehran, Iran

    A Ghavamzadeh

  12. Pediatric Bone Marrow and Stem Cell Transplant, King Hussein Cancer Center, Al-Jubeiha Amman, Jordan

    A A Hussein

  13. Department of Pediatric Hematology and Oncology IRCCS Ospedale Pediatrico Bambino Gesù, Rome, Italy

    F Locatelli

  14. EBMT member,

    G Dini

  15. Division for Stem Cell Transplantation and Immunology Hospital for Children and Adolescents, Frankfurt a. Main, Germany

    P Bader

Authors
  1. D Baronciani
    View author publications

    You can also search for this author in PubMed Google Scholar

  2. E Angelucci
    View author publications

    You can also search for this author in PubMed Google Scholar

  3. U Potschger
    View author publications

    You can also search for this author in PubMed Google Scholar

  4. J Gaziev
    View author publications

    You can also search for this author in PubMed Google Scholar

  5. A Yesilipek
    View author publications

    You can also search for this author in PubMed Google Scholar

  6. M Zecca
    View author publications

    You can also search for this author in PubMed Google Scholar

  7. M G Orofino
    View author publications

    You can also search for this author in PubMed Google Scholar

  8. C Giardini
    View author publications

    You can also search for this author in PubMed Google Scholar

  9. A Al-Ahmari
    View author publications

    You can also search for this author in PubMed Google Scholar

  10. S Marktel
    View author publications

    You can also search for this author in PubMed Google Scholar

  11. J de la Fuente
    View author publications

    You can also search for this author in PubMed Google Scholar

  12. A Ghavamzadeh
    View author publications

    You can also search for this author in PubMed Google Scholar

  13. A A Hussein
    View author publications

    You can also search for this author in PubMed Google Scholar

  14. C Targhetta
    View author publications

    You can also search for this author in PubMed Google Scholar

  15. F Pilo
    View author publications

    You can also search for this author in PubMed Google Scholar

  16. F Locatelli
    View author publications

    You can also search for this author in PubMed Google Scholar

  17. G Dini
    View author publications

    You can also search for this author in PubMed Google Scholar

  18. P Bader
    View author publications

    You can also search for this author in PubMed Google Scholar

  19. C Peters
    View author publications

    You can also search for this author in PubMed Google Scholar

Corresponding author

Correspondence to E Angelucci.

Ethics declarations

Competing interests

The authors declare no conflict of interest.

Additional information

This study was conducted on behalf of the EBMT Pediatric Disease Working Party. This study was presented as oral communication at the 2011 American Society of Hematology meeting in San Diego, CA, USA and at the plenary session of annual meeting of the Italian Association of Hematology Naples 2011.

Appendix

Appendix

Appendix Contributing centers

The number of patients registered in the study period (2000–2010) is shown in parentheses.

Frankfurt, J. W. Goethe Universität, Kinderheilkunde lll (3); Ankara, Gazi University, Besevle (1); Shiraz, Nemazee Hospital (16); Marseille, Hôpital Timone Enfants (4); Würzburg, Universitätsklinikum (1); Paris, Hôpital Neckar des enfants malades (3); Basel, Universitätsspital Basel (1); Leiden, University Hospital (40); Ulm, Kinderklinik Universitätsklinikum, Abt. Peds. II (4); London, Hammersmith Hospitals NHS Trust (1); Copenhagen, Rigshospitalet (10); Paris, Hôpital St. Louis (8); Leuven, University Hospital Gasthuisberg (1); Stockholm, Karolinska University Hospital, Huddinge (7); Brussels, Institut Jules Bordet, Childrens Hospital (3); London, The Royal Free Hospital (11); London, Royal Marsden Hospital (3); Rome, Università "La Sapienza", Hem Faculty 1 (1); Besancon, Hôpital Jean Minjoz and St Jacques (1); Brussels, Clinique Universitaire St Luc (4); Oslo, Rikshospitalet, Department of Medicine, The National Hospital (2); Nijmegen, University Hospital (4); Cordoba, Hospital Reina Sofia (2); Utrecht, University Hospital UMCU (1); Santander, Hospital Universitario M. de Valdecilla (1); London, Great Ormond Street Hospital (4); Pescara, Ospedale Civile (18); Nantes, Hotel Dieu, CHU Nantes (1); Leeds, St. James’s University Hospital and The General Infirmary (11); Jerusalem, Hadassah University Hospital (40); Essen, Universitätsklinikum (5); Barcelona, Santa Creu i Sant Pau (3); Geneva, Hôpital Cantonal Universitaire (1); London, The London Clinic (2); Uppsala, University Hospital, Medical Clinic (3); Grenoble, CHV Grenoble (1); Clermont Ferrand, CRCTCP, CHU Estaing (3); Genova, Istituto Giannina Gaslini (4); Newcastle upon Tyne, Royal Victoria Infirmary (1); Lille, Hôpital Claude Huriez (2); Monza, Ospedale San Gerardo (1); Lund, University Hospital (4); Rome, Ospedale S. Camillo (2); Goteborg, CHECT Sahlgrenska University Hospital (2); Hannover, Medizinische Hochschule (7); Lisbon, Instituto Portugues de Oncologia (4); Torino, University Hospital (14); Zurich, Universitäts Kinderklinik (10); Berlin, Charité, Campus Virchow Klinikum (1); Napels, National Cancer Institute (3); Haifa, Rambam Medical Center (24); Sofia, Pediatric Hospital for Oncohematology and BMT (7); Belgrade, Mother+Child Health Institute (1); Ankara, GATA BMT Center, Etlik (10); Rome, Policlinico Tor Vergata, Fond. IME (146); Bristol, Royal Hospital for Sick Children (1); Riyadh, King Faisal Specialist Hospital (64); Ankara, Ihsan Dogramaci Childrens Hospital (Hacettepe) (21); Istanbul, University of Istanbul (10); Madrid, Hospital Universitario Materno Infantil Gregorio Maranon (1); Barcelona, Hospital M. Infantil, Vall d'Hebron (5); Münster, Universitätklinikum Münster (1); München, Universitätsklinikum Grosshadern, Med Klin lll (1); Manchester, Royal Children's Hospital (17); Nice, Hôpital de l'Archet 1 (2); Trieste, Istituto per I'Infanzia (4); San Giovanni Rotondo, Hospital Casa Sollievo Sofferenza (1); Barcelona, Hospital General Vall d'Hebron (7); Vienna, St. Anna Kinderspital (6); Pesaro, Ospedale San Salvatore (67); Tübingen, Universitätsklinikum, Pädiatrie (2); London, St George’s Hospital (2); Pavia, Policlinico IRCCS St Matteo (113); Tel Hashomer, Chaim Sheba Medical Cebter (10); Amman, King Hussein Cancer Centre (44); Klinik fuer Knochenmarktransplantation und Haematologie/Onkologie, Idar-Obestein, Germany (15); San Sebastian, Hospital Nostra Senora de Aranzazu (1); Miskolc, Postgraduate Medical School (1); Firenze, Azienda Ospedaliero Universitaria Meyer, Department of Paediatric Haematology Oncology (11); Bremen, Klinikum Bremen-Mitte, Inn. Med l (1); Hamburg, Universitätsklinikum Eppendorf (4); Antalya, Akdeniz University Medical School (123); Ankara, University of Ankara (26); Izmir, Ege University Medical Faculty, Bornova (22); Athens, Evanghelismos Hospital (1); Brussels, University Hospital (1); Paris, Hôpital Robert Debré, Hem-Immuno (3); Teheran, Shariati Hospital (52); Düsseldorf, Universitätsklinikum (4); Valencia, Hospital Universitario La Fe (1); Lyon, Hôpital Edouard Herriot (3); Strasbourg, Hôpital Hautpierre (2); Vandoeuvre-les-Nancy, Hôpital d'Enfants (1); Randwick, Sydney Children’s Hospital, Centre for Children’s Cancer (2); Alger, Centre Pierre et Marie Curie (26); Glasgow, Royal Hospital for Sick Children (3); Sidney, The Children’s Hospital at Westmead (2); Palma de Mallorca, Hospital Universitario Son Espases (1); Salamanca, Complejo Hospital (3); Madrid, Hospital Niño Jesus (1); Madrid, Hospital La Paz (5); Brescia, Universitá degli Studi di Brescia (2); Buenos Aires CEHT (1); Gent, University Hospital (1); Athens, Aghia Sophia Childrens Hospital (21); Petach-Tikva, Childrens Medical center (6); Rome, Rome Transplant Network¨Tor Vergata¨ University of Rome Stem Cell Transplant Unit (12); Istanbul Tip Fakultesi Iç (1); Sevilla, Hospital Universitario Virgen del Rocio (1); Sheffield, Teaching Hospitals NHS Trust, Royal Hallamshire Hospital (4); Birmingham, The Birmingham Children's Hospital (15); Bologna, Policlinico S. Orsola, Clin. Pediatrica lll (7); Pisa, University of Pisa (4); Gdansk, Medical University (1); Lyon, Institut d'Hématologie et d'Oncologie Pédiatrique (9); Dresden, Universitätsklinikum Carl Gustav Carus (3); Freiburg, Universitatsklinikum, Kinderklinik (5); Cagliari, Binaghi BMT Centre (42); Cagliari, Univer Studi (70); Milan, Istituto Scientifico H.S. Raffaele (58); Catania, Pediatric Unit-Policlinico (1); London, St Mary's Hospital (58); Antalya, Medical Park Hospitals (2); Rouen, Hôpital Charles Nicolle (2); Toulouse, Hôpital Purpan (1); Strasbourg H Hautepierre (3); Bordeaux, CHU Hopitalier Pellegrin-Enfants (1).

Rights and permissions

Reprints and permissions

About this article

Check for updates. Verify currency and authenticity via CrossMark

Cite this article

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 51, 536–541 (2016). https://doi.org/10.1038/bmt.2015.293

Download citation

  • Received:

  • Revised:

  • Accepted:

  • Published:

  • Issue Date:

  • DOI: https://doi.org/10.1038/bmt.2015.293

This article is cited by

Search

Advanced search

Quick links