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Primary Immunodeficiency Diseases and Hematopoietic Stem Cell Transplantation

Year 2014, Volume: 23 Issue: 1, 108 - 117, 30.10.2014
https://doi.org/10.17827/aktd.81569

Abstract

Hematopoietic stem cell transplantation (HSCT) is the only curative therapy for primary immunodeficiency diseases. Early diagnosis, including prenatally, and early transplantation improve HSCT outcomes. Survival rates improve with advances in the methods of preparing hosts and donor cells, and in supportive and conditioning regimes.

References

  • Small TN, Friedrich W, O’Reilly RJ. Hematopoietic cell transplantation for immunodeficiency diseases , in Thomas’ Hematopoietic Cell Transplantation Fourth Edition (Eds FR Appelbaum, SJ Forman, RS Negrin, KG Blume):1105-24. UK, Wiley-Blackwell, 2008.
  • Kalman L, Lindegren ML, Kobrynski L, Vogt R, Hannon H, Howard JT et al. Mutations in genes required for T-cell development: IL7R, CD45, IL2RG, JAK3, RAG1, RAG2, ARTEMIS and ADA and severe combined ımmunodeficiency: HuGE review. Genet Med. 2004; 6:16-26.
  • Fischer A, Landais P, FriedrichW, Morgan G, Gerritsen B, Fasth A et al. European experience of bone-marrow transplantation for severe combined immunodeficiency. Lancet. 1990; 336:850-4. Antoine C, Müller S, Cant A, Cavazzana-Calvo M, Veys P, Vossen J et al. European Group for Blood and Marrow Transplantation; European Society for Immunodeficiency. Long-term survival and transplantation of haemopoietic stem cells for immunodeficiencies: report of the European experience 1968-99. Lancet. 2003; 361:553-60.
  • Gennery AR, Slatter MA, Grandin L, Taupin P, Cant AJ, Veys P et al. Inborn Errors Working Party of the European Group for Blood and Marrow Transplantation; European Society for Immunodeficiency.Transplantation of hematopoetic stem cells and long term survival for primary immunodeficiencies in Europe: Entering a new century, do we do better? J Allergy Clin Immunol. 2010; 126:602-10.
  • Grunebaum E, Mazzolari, Porta F, Dallera D, Atkinson A, Reid B et al. Bone marrow transplantation for severe combined immun deficiency. JAMA. 2006; 295:508-18.
  • Bucley RH, Schiff SE, Schiff RI, Markert L, Williams LW, Roberts JL et al. Hematopoetic stem cell transplantation for the treatment of severe combined immunodeficiency. N Engl J Med. 1999; 340:508-16.
  • Slatter MA, Gennery AR. Umblical cord stem cell transplantation for primary immunodeficiencies. Expert Opin Biol Ther. 2006; 6:555-65.
  • Slatter MA, Rao K, Amrolia P, Flood T, Abinun M, Hambleton S et al. Treosulfan-based conditioning regimens for hematopoietic stem cell transplantation in children with primary immunodeficiency: United Kingdom experience. Blood. 2011; 117:4367-75.

Primer İmmün Yetmezlik Hastalıkları ve Hematopoetik Kök Hücre Nakli

Year 2014, Volume: 23 Issue: 1, 108 - 117, 30.10.2014
https://doi.org/10.17827/aktd.81569

Abstract

Hematopoetik kök hücre nakli (HKHN) birçok primer immün yetmezlik hastalıkları için tek küratif tedavi yöntemidir. Prenatal dönemi kapsayan erken tanı ve erken nakil ile HKHN ile daha iyi sonuçlar alınmaktadır. Hazırlık ve destek tedavilerinin, alıcıların ve verici hücrelerinin hazırlanmasındaki metodların geliştirilmesi ile sağkalım oranlarında artış olmaktadır.

References

  • Small TN, Friedrich W, O’Reilly RJ. Hematopoietic cell transplantation for immunodeficiency diseases , in Thomas’ Hematopoietic Cell Transplantation Fourth Edition (Eds FR Appelbaum, SJ Forman, RS Negrin, KG Blume):1105-24. UK, Wiley-Blackwell, 2008.
  • Kalman L, Lindegren ML, Kobrynski L, Vogt R, Hannon H, Howard JT et al. Mutations in genes required for T-cell development: IL7R, CD45, IL2RG, JAK3, RAG1, RAG2, ARTEMIS and ADA and severe combined ımmunodeficiency: HuGE review. Genet Med. 2004; 6:16-26.
  • Fischer A, Landais P, FriedrichW, Morgan G, Gerritsen B, Fasth A et al. European experience of bone-marrow transplantation for severe combined immunodeficiency. Lancet. 1990; 336:850-4. Antoine C, Müller S, Cant A, Cavazzana-Calvo M, Veys P, Vossen J et al. European Group for Blood and Marrow Transplantation; European Society for Immunodeficiency. Long-term survival and transplantation of haemopoietic stem cells for immunodeficiencies: report of the European experience 1968-99. Lancet. 2003; 361:553-60.
  • Gennery AR, Slatter MA, Grandin L, Taupin P, Cant AJ, Veys P et al. Inborn Errors Working Party of the European Group for Blood and Marrow Transplantation; European Society for Immunodeficiency.Transplantation of hematopoetic stem cells and long term survival for primary immunodeficiencies in Europe: Entering a new century, do we do better? J Allergy Clin Immunol. 2010; 126:602-10.
  • Grunebaum E, Mazzolari, Porta F, Dallera D, Atkinson A, Reid B et al. Bone marrow transplantation for severe combined immun deficiency. JAMA. 2006; 295:508-18.
  • Bucley RH, Schiff SE, Schiff RI, Markert L, Williams LW, Roberts JL et al. Hematopoetic stem cell transplantation for the treatment of severe combined immunodeficiency. N Engl J Med. 1999; 340:508-16.
  • Slatter MA, Gennery AR. Umblical cord stem cell transplantation for primary immunodeficiencies. Expert Opin Biol Ther. 2006; 6:555-65.
  • Slatter MA, Rao K, Amrolia P, Flood T, Abinun M, Hambleton S et al. Treosulfan-based conditioning regimens for hematopoietic stem cell transplantation in children with primary immunodeficiency: United Kingdom experience. Blood. 2011; 117:4367-75.
There are 8 citations in total.

Details

Primary Language Turkish
Journal Section Review
Authors

Ayşe Özkan

Publication Date October 30, 2014
Published in Issue Year 2014 Volume: 23 Issue: 1

Cite

AMA Özkan A. Primer İmmün Yetmezlik Hastalıkları ve Hematopoetik Kök Hücre Nakli. aktd. March 2014;23(1):108-117. doi:10.17827/aktd.81569