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Review
. 2016 Oct 20;8(1):e2016048.
doi: 10.4084/MJHID.2016.048. eCollection 2016.

What Unrelated Hematopoietic Stem Cell Transplantation in Thalassemia Taught us about Transplant Immunogenetics

Giorgio La Nasa  1 , Adriana Vacca  2 , Roberto Littera  3 , Eugenia Piras  2 , Sandro Orru  4 , Marianna Greco  2 , Carlo Carcassi  4 , Giovanni Caocci  1
Affiliations
Review

What Unrelated Hematopoietic Stem Cell Transplantation in Thalassemia Taught us about Transplant Immunogenetics

Giorgio La Nasa et al. Mediterr J Hematol Infect Dis. .

Abstract

Although the past few decades have shown an improvement in the survival and complication-free survival rates in patients with beta-thalassemia major and gene therapy is already at an advanced stage of experimentation, hematopoietic stem cell transplantation (HSCT) continues to be the only effective and realistic approach to the cure of this chronic non-malignant disease. Historically, human leukocyte antigen (HLA)-matched siblings have been the preferred source of donor cells owing to superior outcomes compared with HSCT from other sources. Nowadays, the availability of an international network of voluntary stem cell donor registries and cord blood banks has significantly increased the odds of finding a suitable HLA matched donor. Stringent immunogenetic criteria for donor selection have made it possible to achieve overall survival (OS) and thalassemia-free survival (TFS) rates comparable to those of sibling transplants. However, acute and chronic graft-versus-host disease (GVHD) remains the most important complication in unrelated HSCT in thalassemia, leading to significant rates of morbidity and mortality for a chronic non-malignant disease. A careful immunogenetic assessment of donors and recipients makes it possible to individualize appropriate strategies for its prevention and management. This review provides an overview of recent insights about immunogenetic factors involved in GVHD, which seem to have a potential role in the outcome of transplantation for thalassemia.

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Conflict of interest statement

Financial and Competing Interests Disclosure. The authors have no relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript. This includes employment, consultancies, honoraria, stock ownership or options, expert testimony, grants or patents received or pending, or royalties.

Figures

Figure 1
Figure 1
Risk assessment of acute graft-versus-host disease and rejection according to permissive and nonpermissive HLA-DPB1 mismatches in donor and recipient pairs.
Figure 2
Figure 2
Algorithm showing the risk of developing acute GvHD or rejection after unrelated HSCT according to recipient KIR ligands and the number of donors activating KIRs. aGVHD= acute Graft Versus Host Disease.
Figure 3
Figure 3
The path from GvHD to immune tolerance is like a bridge where many variables are still missing.
See this image and copyright information in PMC

References

    1. Rund D. Thalassemia 2016: Modern medicine battles an ancient disease. Am J Hematol. 2016;91(1):15–21. doi: 10.1002/ajh.24231. - DOI - PubMed
    1. Borgna-Pignatti C. The life of patients with thalassemia major. Haematologica. 2010;95(3):345–8. doi: 10.3324/haematol.2009.017228. - DOI - PMC - PubMed
    1. Caocci G, Efficace F, Ciotti F, Roncarolo MG, Vacca A, Piras E, et al. Health related quality of life in Middle Eastern children with beta-thalassemia. BMC Blood Disord. 2012;12:6. doi: 10.1186/1471-2326-12-6. - DOI - PMC - PubMed
    1. Modell B, Khan M, Darlison M, Westwood MA, Ingram D, Pennell DJ. Improved survival of thalassaemia major in the UK and relation to T2* cardiovascular magnetic resonance. J Cardiovasc Magn Reson. 2008;10:42. doi: 10.1186/1532-429X-10-42. - DOI - PMC - PubMed
    1. Borgna-Pignatti C, Marsella M. Iron Chelation in Thalassemia Major. Clin Ther. 2015;37(12):2866–77. doi: 10.1016/j.clinthera.2015.10.001. - DOI - PubMed