Hematopoietic Stem-Cell Transplantation for Sickle Cell Disease

Yıl 2014, Cilt: 23 Sayı: 1, 42 - 48, 30.10.2014

Atila Tanyeli

https://doi.org/10.17827/aktd.43132

Öz

Sickle cell anemia is one of the most common hemoglobinopathies in the worldwide. Sickle cell anemia characterized by crises and organ failure develops over time. Myeloablative stem cell transplantation is curative but it has been performed in children younger than 16 years of age. Modest modifications in the conditioning regimen and supportive care have improved outcome such that the majority of children with a suitable HLA-matched sibling donor can expect a cure from this approach. But nonmyeloablative protocols are crucial for the future of Hematopoietic Stem-Cell Transplantation for older sickle cell anemia patients with organ failure. A protocol for nonmyeloablative allogeneic hematopoietic stem-cell transplantation that includes total-body irradiation and treatment with alemtuzumab and sirolimus can achieve stable, mixed donor–recipient chimerism. Stem cell transplantation is recommended in the presence of HLA-matched siblings in patients at risk.

Anahtar Kelimeler

Sickle cell anemia, hematopoietic stem-cell transplantation, childhood

Orak Hücre Anemisinde Hematopoetik Kök Hücre Transplantasyonu

Öz

Orak hücre anemisi dünya genelinde en sık rastlanılan hemoglobinopatilerden biri olup anemi, krizler ve bunların sonucu gelişen organ yetmezlikleri ile seyreden bir hastalıktır. Myeloablatif kök hücre nakli ile tam tedavi sağlanabilmektedir. Fakat genelde 16 yaşından küçük çocuklarda uygulanmaktadır. Tüm vücut ışınlaması, alemtuzumab ve sirolimus içeren nonmyeloablatif protokollerin kullanılması ile erişkinlerde de iyi sonuçlar alınmaktadır. Erişkinler de, bu şekilde stabil ve mikskimerik hale getirilebilmektedir. Risk kriterleri taşıyan HLA uygun kardeşi olan hastalara kök hücre nakli önerilmektedir.

Anahtar Kelimeler

Orak hücre anemisi, hematopoetik kök hücre nakli, çocukluk çağı

Kaynakça

• Harmening DM. Hemoglobinopathies In: Clinical Hematology and Fundamentals of Hemostasis. 3rd Edition (Ed DM Harmening):173-192. Philadelphia, USA, FA Davis, 1997.
• Canatan D: Orak hücre anemisi. XXX. Ulusal Hematoloji Kongresi Mezuniyet Sonrası Eğitim Kursu kitabı. Sayfa: 93. İstanbul, 2003.
• Gaziev D., Lucarelli G. Stem cell transplantation for hemoglobinopathies. Curr Opin Pediatr. 2003; 15:24-31.
• Lucarelli G, Isgrò A, Sodani P, Gaziev J. Hematopoietic stem cell transplantation in thalassemia and sickle cell anemia. Cold Spring Harb Perspect Med. 2012; 2:a011825.
• Johnson FL, Look AT., Gockerman J, Ruggiero MR, Dalla-Pozza L, Billings FT. Bone marrow transplantation in a patient with sickle cell anemia. N Engl J Med. 1984; 311:780-3.
• Shenoy S. Hematopoietic stem cell transplantation for sickle cell disease: current practice and emerging trends. Hematology Am Soc Hematol Educ Program. 2011; 2011:273-9..
• Vermylen C., CornuG.,Ferster A., Sariban E. Hematopoetic stem cell transplantation for sickle cell anemia: the first 50 patients transplanted in Belgium. Bone Marrow Transplant. 1998; 22:1-6. 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; 110:2749-56. Panepinto JA, Walters MC, Carreras J, Marsh J, Bredeson CN, Gale RP et al. Matched-related donor transplantation for sickle cell disease. Br J Haematol. 2007; 137:479-85.
• Brachet C, Azzi N, Demulder A, Devalck C, Gourdin A, Gulbis B et al. Hydroxyurea treatment for sickle cell disease: impact on haematopoietic stem cell transplantation’soutcome. Bone Marrow Transplant. 2004; 33:799-803.
• Walters MC. Hematopoetic stem cell transplantation for sickle cell disease. In Thomas’ Hematopoetic Cell Transplantation, 3rd Edition (Eds KG Blume, S Forman, FR Appelbaum):1417 Oxford, Blackwell Publishing, 2004.
• Steen RG, Helton KJ, Horwitz EM, Benaim E, Thompson S, Bowman LC et al. Improved cerebrovascular patency following therapy in patients with sickle cell disease: initial results in four patients who received HLA-identical hematopoetic stem cell allografts. Ann Neurol. 2001; 49:222-9.