25 research outputs found

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

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    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

    Whooping cough in school age children with persistent cough: prospective cohort study in primary care

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    Objective To estimate the proportion of school age children with a persistent cough who have evidence of a recent Bordetella pertussis infection. Design Prospective cohort study (October 2001 to March 2005). Setting General practices in Oxfordshire, England. Participants 172 children aged 5-16 years who presented to their general practitioner with a cough lasting 14 days or more who consented to have a blood test. Main outcome measures Serological evidence of a recent Bordetella pertussis infection; symptoms at presentation; duration and severity of cough; sleep disturbance (parents and child). Results 64 (37.2%, 95% confidence interval 30.0% to 44.4%) children had serological evidence of a recent Bordetella pertussis infection; 55 (85.9%) of these children had been fully immunised. At presentation, children with whooping cough were more likely than others to have whooping (odds ratio 2.85, 95% confidence interval 1.39 to 5.82), vomiting (4.35, 2.04 to 9.25), and sputum production (2.39, 1.14 to 5.02). Children with whooping cough were also more likely to still be coughing two months after the start of their illness (85% v 48%; P = 0.001), continue to have more than five coughing episodes a day (P = 0.049), and cause sleep disturbance for their parents (P = 0.003). Conclusions For school age children presenting to primary care with a cough lasting two weeks or more, a diagnosis of whooping cough should be considered even if the child has been immunised. Making a secure diagnosis of whooping cough may prevent inappropriate investigations and treatment

    Estimated operational performance of CEA in clinical practice at currently recommended intervals if further investigation is triggered by thresholds of 2.5 and 5 μg/L.

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    <p>Estimated operational performance of CEA in clinical practice at currently recommended intervals if further investigation is triggered by thresholds of 2.5 and 5 μg/L.</p

    Comparison of the diagnostic performance in detecting recurrence of a rise in blood CEA level above a threshold of 5μg/L in relation to i) all CEA measurements taken prior to diagnosis of recurrence and ii) the final CEA measurement taken prior to diagnosis of recurrence.

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    <p>Comparison of the diagnostic performance in detecting recurrence of a rise in blood CEA level above a threshold of 5μg/L in relation to i) all CEA measurements taken prior to diagnosis of recurrence and ii) the final CEA measurement taken prior to diagnosis of recurrence.</p
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