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1.
J Investig Med ; 70(5): 1316-1319, 2022 06.
Article in English | MEDLINE | ID: covidwho-1909801

ABSTRACT

Patients with sickle cell disease (SCD) experience a range of clinical symptoms, including acute and chronic pain, fatigue, and respiratory problems, as well as chronic organ complications that can lead to disability and accelerated mortality. Voxelotor is a first-in-class therapy that targets sickle hemoglobin polymerization, the root cause of SCD. It is approved by the US Food and Drug Administration for treatment of SCD in patients aged 4 years and older and in the European Union and United Arab Emirates for the treatment of SCD in patients aged 12 years and older. Here, we report the single-center experience of both clinician-determined and patient-reported benefits of voxelotor in 27 consecutive patients treated for at least 8 weeks. Clinical Global Impression of Change and Patient Global Impression of Change rating scales were used to capture clinicians' and patients' perceptions of change in overall patient health-related quality-of-life with voxelotor treatment. Laboratory data were also collected to assess clinical response to treatment. As observed in previous clinical studies, hemoglobin concentrations and markers of hemolysis were improved in patients treated with voxelotor. Most patients reported marked improvement in disease symptoms, which correlated well with the clinicians' assessments. Although limited by the retrospective open-label study design, these findings suggest that voxelotor use has a positive impact on outcomes in patients with SCD.


Subject(s)
Anemia, Sickle Cell , Benzaldehydes , Pyrazines , Pyrazoles , Anemia, Sickle Cell/drug therapy , Benzaldehydes/therapeutic use , Hemoglobin, Sickle , Humans , Patient Reported Outcome Measures , Pyrazines/therapeutic use , Pyrazoles/therapeutic use , Quality of Life , Retrospective Studies
2.
Biomed Pharmacother ; 147: 112658, 2022 Mar.
Article in English | MEDLINE | ID: covidwho-1641135

ABSTRACT

The unexpected emergence of the new Coronavirus disease (COVID-19) has affected more than three hundred million individuals and resulted in more than five million deaths worldwide. The ongoing pandemic has underscored the urgent need for effective preventive and therapeutic measures to develop anti-viral therapy. The natural compounds possess various pharmaceutical properties and are reported as effective anti-virals. The interest to develop an anti-viral drug against the novel severe acute respiratory syndrome Coronavirus (SARS-CoV-2) from natural compounds has increased globally. Here, we investigated the anti-viral potential of selected promising natural products. Sources of data for this paper are current literature published in the context of therapeutic uses of phytoconstituents and their mechanism of action published in various reputed peer-reviewed journals. An extensive literature survey was done and data were critically analyzed to get deeper insights into the mechanism of action of a few important phytoconstituents. The consumption of natural products such as thymoquinone, quercetin, caffeic acid, ursolic acid, ellagic acid, vanillin, thymol, and rosmarinic acid could improve our immune response and thus possesses excellent therapeutic potential. This review focuses on the anti-viral functions of various phytoconstituent and alkaloids and their potential therapeutic implications against SARS-CoV-2. Our comprehensive analysis provides mechanistic insights into phytoconstituents to restrain viral infection and provide a better solution through natural, therapeutically active agents.


Subject(s)
Antiviral Agents/therapeutic use , COVID-19 Drug Treatment , Phytochemicals/therapeutic use , Phytotherapy , Alkaloids/therapeutic use , Benzaldehydes/therapeutic use , Benzoquinones/therapeutic use , Caffeic Acids/therapeutic use , Cinnamates/therapeutic use , Depsides/therapeutic use , Ellagic Acid/therapeutic use , Humans , Quercetin/therapeutic use , SARS-CoV-2 , Thymol/therapeutic use , Triterpenes/therapeutic use
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