Hematopoietic Stem Cell Transplantation in Adult Sickle Cell Disease: Problems and Solutions

• Published in: Turkish journal of haematology Vol. 32; no. 3; pp. 195 - 205
• Main Authors: Özdoğu, Hakan, Boğa, Can
• Format: Journal Article
• Language: English
• Published: Turkey Galenos Publishing 01.09.2015; Galenos Publishing House
• Subjects: Adolescent; Adult; Allografts; Anemia, Sickle Cell - complications; Anemia, Sickle Cell - economics; Anemia, Sickle Cell - therapy; Combined Modality Therapy; conditioning; Female; Fertility Preservation; graft rejection; Graft vs Host Disease - prevention & control; graft-versus-host disease; Health Care Costs; hematopoietic stem cell transplantation; Hematopoietic Stem Cell Transplantation - adverse effects; Hematopoietic Stem Cell Transplantation - economics; Histocompatibility; Humans; Male; Myeloablative Agonists - adverse effects; Myeloablative Agonists - therapeutic use; Review; sickle cell disease; Tissue and Organ Procurement; Transplantation Conditioning - adverse effects; Transplantation Conditioning - methods; Treatment Outcome; Young Adult
• ISSN: 1300-7777; 1308-5263
• DOI: 10.4274/tjh.2014.0311

Sickle cell disease-related organ injuries cannot be prevented despite hydroxyurea use, infection prophylaxis, and supportive therapies. As a consequence, disease-related mortality reaches 14% in adolescents and young adults. Hematopoietic stem cell transplantation is a unique curative therapeutic approach for sickle cell disease. Myeloablative allogeneic hematopoietic stem cell transplantation is curative for children with sickle cell disease. Current data indicate that long-term disease-free survival is about 90% and overall survival about 95% after transplantation. However, it is toxic in adults due to organ injuries. In addition, this curative treatment approach has several limitations, such as difficulties to find donors, transplant-related mortality, graft loss, graft-versus-host disease (GVHD), and infertility. Engraftment effectivity and toxicity for transplantations performed with nonmyeloablative reduced-intensity regimens in adults are being investigated in phase 1/2 trials at many centers. Preliminary data indicate that GVHD could be prevented with transplantations performed using reduced-intensity regimens. It is necessary to develop novel regimens to prevent graft loss and reduce the risk of GVHD.