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Allografting

Allogeneic cellular gene therapy in hemoglobinopathies—evaluation of hematopoietic SCT in sickle cell anemia

Bone Marrow Transplantation volume 47, pages 227–230 (2012)Cite this article

Abstract

Many patients with thalassemia have been cured with BMT since the first successful transplant in 1981. Allogeneic stem cell gene therapy is the only treatment option for patients with sickle cell anemia (SCA). A total of 11 patients with a median age of 12 years (range, 2–16), affected by SCA, received hematopoietic SCT from HLA-identical, related donors following a myeloablative-conditioning regimen. Indications for transplantation were vaso-occlusive crisis, acute chest syndrome, avascular bone necrosis, chronic RBC transfusions, or hemorrhagic stroke. All patients had sustained engraftment. One patient became a stable mixed chimera with 25% of donor cells at 4 years after transplantation. One patient died at 1 year after transplantation. The probability of survival, SCA-free survival and TRM at 5 years after transplant were 90, 90 and 10%, respectively. All 10 surviving patients remained free of any SCA-related events after transplantation. In conclusion, these data confirm SCT from a suitable HLA-matched, related donor should become the primary option for curing children with SCA. There is an excellent survival rate and a return to normal life, free of SCA-related events.

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References

  1. Roseff SD . Sickle cell disease: a review. Immunohematology 2009; 25: 67–74.

    CAS  PubMed  Google Scholar 

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

    Article  CAS  Google Scholar 

  3. Lucarelli G, Izzi T, Polchi P, Manna A, Agostinelli F, Delfini C et al. Bone Marrow Transplantation in Thalassemia. J Exp Clin Cancer Res 1983; 3: 313–315.

    Google Scholar 

  4. Lucarelli G, Gaziev J . Advances in the allogeneic transplantation for thalassemia. Blood Rev 2008; 22: 53–63.

    Article  Google Scholar 

  5. Walters MC, Patience M, Leisenring W, Eckman JR, Scott JP, Mentzer WC et al. Bone marrow transplantation for sickle cell disease. N Engl J Med 1996; 335: 369–376.

    Article  CAS  PubMed  Google Scholar 

  6. Giardini C, Galimberti M, Lucarelli G, Polchi P, Angelucci E, Baronciani D et al. Bone marrow transplantation in sickle cell disorders in Pesaro. Bone Marrow Transplant 1997; 19 (Suppl 2): 106–109.

    Google Scholar 

  7. Vermylen C, Cornu G, Ferster A, Brichard B, Ninane J, Ferrant A et al. Haematopoietic stem cell transplantation for sickle cell anaemia: the first 50 patients transplanted in Belgium. Bone Marrow Transplant 1998; 22: 1–6.

    Article  CAS  Google Scholar 

  8. Bernaudin F, Socie G, Kuentz M, Chevret S, Duval M, Bertrand Y et al. Long-term results of related myeloablative stem-cell transplantation to cure sickle cell disease. Blood 2007; 110: 2749–2756.

    Article  CAS  Google Scholar 

  9. Majumdar S, Robertson Z, Robinson A, Starnes S, Iyer R, Megason G . Outcome of ematopoietic cell transplantation in children with sickle cell disease, a single center's experience. Bone Marrow Transplant 2010; 45: 895–900.

    Article  CAS  PubMed  Google Scholar 

  10. McPherson ME, Hutcherson D, Olson E, Haight AE, Horan J, Chiang KY . Safety and efficacy of targeted busulfan therapy in children undergoing myeloablative matched sibling donor BMT for sickle cell disease. Bone Marrow Transplant. Bone Marrow Transplantation 2010; 46: 27–33.

    Article  PubMed  Google Scholar 

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Acknowledgements

We thank Gianluca Baldassarri for Technical Assistance.

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Authors and Affiliations

  1. International Center for Transplantation in Thalassemia and Sickle Cell Anemia, Mediterranean Institute of Hematology, Policlinic of the University of Rome Tor Vergata, Rome, Italy

    G Lucarelli, J Gaziev, A Isgrò, P Sodani, K Paciaroni, C Alfieri, G De Angelis, M Marziali, M D Simone, C Gallucci, A Roveda, F Saltarelli, F Torelli & M Andreani

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  1. G Lucarelli
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  2. J Gaziev
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  3. A Isgrò
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  4. P Sodani
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  5. K Paciaroni
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  6. C Alfieri
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  7. G De Angelis
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  8. M Marziali
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  9. M D Simone
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  10. C Gallucci
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  11. A Roveda
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  12. F Saltarelli
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  13. F Torelli
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  14. M Andreani
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Correspondence to G Lucarelli.

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Lucarelli, G., Gaziev, J., Isgrò, A. et al. Allogeneic cellular gene therapy in hemoglobinopathies—evaluation of hematopoietic SCT in sickle cell anemia. Bone Marrow Transplant 47, 227–230 (2012). https://doi.org/10.1038/bmt.2011.79

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  • DOI: https://doi.org/10.1038/bmt.2011.79

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