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Impact of voxelotor (GBT440) on unconjugated bilirubin and jaundice in sickle cell disease

Paul Telfer, Irene Agodoa, Kathleen M. Fox, Laurie Burke, Timothy Mant, Marzena Jurek, Margaret Tonda, Josh Lehrer-Graiwer
  • Paul Telfer
    Centre for Genomics and Child Health, Blizard Institute, Barts and The London School of Medicine and Dentistry, Queen Mary University of London,, United Kingdom | paul.telfer@bartshealth.nhs.uk
  • Irene Agodoa
    GBT, South San Francisco, CA, United States
  • Kathleen M. Fox
    LORA Group, Royal Oak, MD, United States
  • Laurie Burke
    LORA Group, Royal Oak, MD, United States
  • Timothy Mant
    IQVIA, Reading, United Kingdom
  • Marzena Jurek
    GBT, South San Francisco, CA, United States
  • Margaret Tonda
    GBT, South San Francisco, CA, United States
  • Josh Lehrer-Graiwer
    GBT, South San Francisco, CA, United States

Abstract

For many patients with sickle cell disease (SCD), jaundice is a significant clinical disease manifestation that impacts on patient well-being. We report a case of a patient with SCD and chronic jaundice treated with voxelotor (GBT440), a novel small molecule hemoglobin oxygen affinity modulator and potential disease-modifying therapy for SCD. The case patient is a 27- year-old Black male with a long history of SCD with clinical jaundice and scleral icterus. After starting voxelotor, the patient reported that his jaundice cleared within one week, and that he felt much better with more energy, and was relieved after his eyes cleared. Voxelotor reduced bilirubin and unconjugated bilirubin (by up to 76%), and hemoglobin improved from 9.9 g/dL at baseline to 11.1 g/dL at 90 days. Jaundice impacts many adults with SCD, significantly impacting self-image. Voxelotor treatment reduced bilirubin levels and improved jaundice, resulting in an improved sense of well-being in our case patient.

Keywords

sickle-cell disease, jaundice, scleral icterus, voxelotor

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Submitted: 2018-02-23 04:46:57
Published: 2018-05-22 09:57:37
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Copyright (c) 2018 Paul Telfer, Irene Agodoa, Kathleen M. Fox, Laurie Burke, Tomothy Mant, Marzena Jurek, Margaret Tonda, Josh Lehrer-Graiwer

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