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      Curative Therapies for Sickle Cell Disease

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          Abstract

          Background: Sickle cell disease (SCD) is an inherited hemoglobinopathy associated with severe morbidity, impaired quality of life, and premature mortality. Hematopoietic stem cell transplantation (HSCT) is the only curative treatment available for patients with SCD and has a >90% event-free survival when a matched related donor is used. However, availability of human leukocyte antigen (HLA)–identical sibling donors for the SCD population is limited. The use of HLA-matched unrelated donors or related haploidentical donors has the potential to expand the donor pool.

          Methods: We reviewed the current literature on the indications for SCD transplantation, donor options, and the emerging use of gene therapy as a treatment option. Google Scholar and PubMed were searched using the terms SCD, bone marrow transplantation, donor sources, gene therapy, HSCT, and HLA matching. Additional articles were identified from the bibliographies of retrieved articles. All articles were reviewed for pertinent information related to SCD and transplantation.

          Results: HSCT has the potential to establish donor-derived normal erythropoiesis with stable long-term engraftment, amelioration of symptoms, and stabilization of organ damage. The majority of HSCT has been performed in children from HLA-identical sibling donors and has resulted in excellent rates of survival. The use of alternate donors such as HLA-matched unrelated donors and haploidentical donors has the potential to expand the applicability of HSCT for SCD. Early results in gene therapy for SCD are encouraging.

          Conclusion: Evaluation of the long-term benefits of curative therapies for SCD requires comparative clinical trials and studies of late effects.

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          Author and article information

          Journal
          Ochsner J
          Ochsner J
          TOJ
          ochjnl
          The Ochsner Journal
          Academic Division of Ochsner Clinic Foundation
          1524-5012
          Summer 2019
          Summer 2019
          : 19
          : 2
          : 131-137
          Affiliations
          [ 1 ]Department of Pediatric Hematology-Oncology-Bone Marrow Transplant, Aflac Cancer and Blood Disorders Center, Emory University School of Medicine , Atlanta, GA
          [ 2 ] Children's Healthcare of Atlanta , Atlanta, GA
          Author notes
          Address correspondence to Lakshmanan Krishnamurti, MD, Department of Pediatric Hematology-Oncology-Bone Marrow Transplant, Aflac Cancer and Blood Disorders Center, Emory University School of Medicine, 2015 Uppergate Drive, Suite 400, Atlanta, GA 30322. Tel: (404) 785-0914. Email: lkrishn@ 123456emory.edu
          Article
          PMC6584191 PMC6584191 6584191 toj.18.0044
          10.31486/toj.18.0044
          6584191
          31258425
          a6464280-335c-404a-9a95-de9a190ff4a6
          © Academic Division of Ochsner Clinic Foundation
          History
          Page count
          Tables: 2, References: 51, Pages: 7
          Categories
          Reviews and Contemporary Updates

          genetic therapy , stem cell transplantation , bone marrow transplantation , Anemia–sickle cell

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