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      A Phase 3 Randomized Trial of Voxelotor in Sickle Cell Disease

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          Abstract

          Deoxygenated sickle hemoglobin (HbS) polymerization drives the pathophysiology of sickle cell disease. Therefore, direct inhibition of HbS polymerization has potential to favorably modify disease outcomes. Voxelotor is an HbS polymerization inhibitor.

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          Sickle cell disease: renal manifestations and mechanisms.

          Karl A. Nath, Robert Hebbel (2015)
          Sickle cell disease (SCD) substantially alters renal structure and function, and causes various renal syndromes and diseases. Such diverse renal outcomes reflect the uniquely complex vascular pathobiology of SCD and the propensity of red blood cells to sickle in the renal medulla because of its hypoxic, acidotic, and hyperosmolar conditions. Renal complications and involvement in sickle cell nephropathy (SCN) include altered haemodynamics, hypertrophy, assorted glomerulopathies, chronic kidney disease, acute kidney injury, impaired urinary concentrating ability, distal nephron dysfunction, haematuria, and increased risks of urinary tract infections and renal medullary carcinoma. SCN largely reflects an underlying vasculopathy characterized by cortical hyperperfusion, medullary hypoperfusion, and an increased, stress-induced vasoconstrictive response. Renal involvement is usually more severe in homozygous disease (sickle cell anaemia, HbSS) than in compound heterozygous types of SCD (for example HbSC and HbSβ(+)-thalassaemia), and is typically mild, albeit prevalent, in the heterozygous state (sickle cell trait, HbAS). Renal involvement contributes substantially to the diminished life expectancy of patients with SCD, accounting for 16-18% of mortality. As improved clinical care promotes survival into adulthood, SCN imposes a growing burden on both individual health and health system costs. This Review addresses the renal manifestations of SCD and focuses on their underlying mechanisms.
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            Mortality Rates and Age at Death from Sickle Cell Disease: U.S., 1979–2005

            Sophie Lanzkron, C Carroll, Carlton Haywood (2013)
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              Cardiovascular complications and risk of death in sickle-cell disease

              Mark Gladwin (2016)
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                Author and article information

                Journal
                Title: New England Journal of Medicine
                Abbreviated Title: N Engl J Med
                Publisher: Massachusetts Medical Society
                ISSN (Print): 0028-4793
                ISSN (Electronic): 1533-4406
                Publication date Created: June 13 2019
                Publication date (Electronic): June 13 2019
                Affiliations
                [1 ]From the University of California, San Francisco (UCSF) Benioff Children’s Hospital Oakland, Oakland (E.V.), and Global Blood Therapeutics, South San Francisco (C.C.H., J.L.-G., M.T., A.I., B.T.) — both in California; the University of Tennessee Health Science Center at Memphis, Memphis (K.I.A.); Cincinnati Children’s Hospital and University of Cincinnati, Cincinnati (R.E.W.); Kenya Medical Research Institute, Kisumu, Kenya (V.N.); Cairo University, Cairo (A.E.-B.), and the Pediatric Department and...
                Article
                DOI: 10.1056/NEJMoa1903212
                PubMed ID: 31199090
                SO-VID: 189a3564-d36a-4e60-9af2-c732c0293f99
                Copyright © © 2019
                License:

                http://www.nejmgroup.org/legal/terms-of-use.htm

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