Hematopoietic Stem Cell Transplantation in Adult Sickle Cell Disease: Problems and Solutions.
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.
Hakan
H
Özdoğu
Can
C
Boğa
D000293:Adolescent; D000328:Adult; D064591:Allografts; D000755:Anemia, Sickle Cell / Q000150:complications / Q000191:economics / Q000628:therapy*; D003131:Combined Modality Therapy; D005260:Female; D059247:Fertility Preservation; D006086:Graft vs Host Disease / Q000517:prevention & control; D017048:Health Care Costs; D018380:Hematopoietic Stem Cell Transplantation / Q000009:adverse effects / Q000191:economics; D006648:Histocompatibility; D006801:Humans; D008297:Male; D019653:Myeloablative Agonists / Q000009:adverse effects / Q000627:therapeutic use; D009927:Tissue and Organ Procurement; D019172:Transplantation Conditioning / Q000009:adverse effects / Q000379:methods; D016896:Treatment Outcome; D055815:Young Adult
D019653:Myeloablative Agonists