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Hematology Reports
Volume 10
Issue 2
10.4081/hr.2018.7643
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Hematology Reports is published by MDPI from Volume 14 Issue 1 (2022). Previous articles were published by another publisher in Open Access under a CC-BY (or CC-BY-NC-ND) licence, and they are hosted by MDPI on mdpi.com as a courtesy and upon agreement with PAGEPress.
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Case Report

Impact of Voxelotor (GBT440) on Unconjugated Bilirubin and Jaundice in Sickle Cell Disease

by
Paul Telfer
1,2,*,
Irene Agodoa
3,
Kathleen M. Fox
4,
Laurie Burke
4,
Timothy Mant
5,
Marzena Jurek
3,
Margaret Tonda
3 and
Josh Lehrer-Graiwer
3
1
Centre for Genomics and Child Health, Blizard Institute, Barts and The London School of Medicine and Dentistry, Queen Mary University of London, London E1 1BB, UK
2
Department of Haematology Barts Health NHS Trust, Royal London Hospital, Whitechapel Road, London E1 1BB, UK
3
GBT, South San Francisco, CA 94080, USA
4
LORA Group, Royal Oak, MD 20850, USA
5
IQVIA, Reading RG1 4LS, UK
*
Author to whom correspondence should be addressed.
Hematol. Rep. 2018, 10(2), 7643; https://doi.org/10.4081/hr.2018.7643
Submission received: 23 February 2018 / Revised: 21 May 2018 / Accepted: 21 May 2018 / Published: 22 May 2018

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 sickle-cell disease; jaundice; scleral icterus; voxelotor

Share and Cite

MDPI and ACS Style

Telfer, P.; Agodoa, I.; Fox, K.M.; Burke, L.; Mant, T.; Jurek, M.; Tonda, M.; Lehrer-Graiwer, J. Impact of Voxelotor (GBT440) on Unconjugated Bilirubin and Jaundice in Sickle Cell Disease. Hematol. Rep. 2018, 10, 7643. https://doi.org/10.4081/hr.2018.7643

AMA Style

Telfer P, Agodoa I, Fox KM, Burke L, Mant T, Jurek M, Tonda M, Lehrer-Graiwer J. Impact of Voxelotor (GBT440) on Unconjugated Bilirubin and Jaundice in Sickle Cell Disease. Hematology Reports. 2018; 10(2):7643. https://doi.org/10.4081/hr.2018.7643

Chicago/Turabian Style

Telfer, Paul, Irene Agodoa, Kathleen M. Fox, Laurie Burke, Timothy Mant, Marzena Jurek, Margaret Tonda, and Josh Lehrer-Graiwer. 2018. "Impact of Voxelotor (GBT440) on Unconjugated Bilirubin and Jaundice in Sickle Cell Disease" Hematology Reports 10, no. 2: 7643. https://doi.org/10.4081/hr.2018.7643

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