Primer İmmün Yetmezlik Hastalıkları ve Hematopoetik Kök Hücre Nakli

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.

Anahtar Kelimeler:

Hematopoetik kök hücre nakli, primer immün yetmezlik

Primary Immunodeficiency Diseases and Hematopoietic Stem Cell Transplantation

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.

Keywords:

Hematopoietic stem cell transplantation, primary immunodeficiency,

PDF

___

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.