Voxelotor: A Hemoglobin S Polymerization Inhibitor for the Treatment of Sickle Cell Disease

Yenamandra, Avani; Marjoncu, Dennis

doi:10.6004/jadpro.2020.11.8.7

Cited by 7 publications

(7 citation statements)

References 11 publications

(16 reference statements)

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“…It functions by binding reversibly to Hb and increasing affinity for oxygen. This prevents polymerization and sickling, which minimizes blood viscosity and improves blood cell deformability -reducing episodes of hemolysis and improving anemia [11]. A phase 3 randomized, doubleblind, placebo-controlled study evaluating the safety and efficacy of voxelotor treatment showed a mean change in hemoglobin from baseline of 1.1 g/dL compared with −0.1 for placebo (P < 0.001).…”

Section: Discussionmentioning

confidence: 99%

Voxelotor Treatment Providing Transfusion Independence for Patient With Combined Sickle Cell Disease and Lipopolysaccharide-Responsive and Beige-Like Anchor (LRBA) Deficiency

Larkin,

Sharma,

Castro

et al. 2023

Cureus

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We present a 22-year-old female with transfusion-dependent anemia due to sickle cell disease (SCD) with lipopolysaccharide-responsive and beige-like anchor protein (LRBA) deficiency whose treatment frequency was moderated with voxelotor (Oxbryta®). The patient was transfusion dependent, initially thought to be secondary only to SCD. After the diagnosis of LRBA deficiency, her regimen included abatacept, sirolimus, hydroxyurea, and folic acid, but she still required intermittent transfusion. She was started on voxelotor in January 2020. Since initiation, her baseline hemoglobin level has increased and she is no longer transfusion dependent.

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Section: Discussionmentioning

confidence: 99%

Voxelotor Treatment Providing Transfusion Independence for Patient With Combined Sickle Cell Disease and Lipopolysaccharide-Responsive and Beige-Like Anchor (LRBA) Deficiency

Larkin,

Sharma,

Castro

et al. 2023

Cureus

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“…Reduce the burden of sickled cells [94,95] Hydroxyurea Increase fetal hemoglobin (HbF) to stop polymers forming in the sickle hemoglobin [96] Hematopoietic stem cell transplantation Reverse the sickle phenotype [97,98] L-glutamine Antioxidant effects [99] Hemoglobin S (HbS) polymerization inhibitors Prevent HbS polymerization [100] Monoclonal antibody (crizanlizumab) Reduce selectin-mediated adhesion [101] Gene editing therapy (Casgevy™) Editing faulty gene in a patient's bone marrow stem cells [102] Recent Advanced Therapeutic Approaches for Sickle Cell Disease…”

Section: Blood Transfusionmentioning

confidence: 99%

Sickle Cell Disease Update: New Treatments and Challenging Nutritional Interventions

Bell,

Varzakas,

Psaltopoulou

et al. 2024

Nutrients

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Sickle cell disease (SCD), a distinctive and often overlooked illness in the 21st century, is a congenital blood disorder characterized by considerable phenotypic diversity. It comprises a group of disorders, with sickle cell anemia (SCA) being the most prevalent and serious genotype. Although there have been some systematic reviews of global data, worldwide statistics regarding SCD prevalence, morbidity, and mortality remain scarce. In developed countries with a lower number of sickle cell patients, cutting-edge technologies have led to the development of new treatments. However, in developing settings where sickle cell disease (SCD) is more prevalent, medical management, rather than a cure, still relies on the use of hydroxyurea, blood transfusions, and analgesics. This is a disease that affects red blood cells, consequently affecting most organs in diverse manners. We discuss its etiology and the advent of new technologies, but the aim of this study is to understand the various types of nutrition-related studies involving individuals suffering from SCD, particularly in Africa. The interplay of the environment, food, gut microbiota, along with their respective genomes collectively known as the gut microbiome, and host metabolism is responsible for mediating host metabolic phenotypes and modulating gut microbiota. In addition, it serves the purpose of providing essential nutrients. Moreover, it engages in direct interactions with host homeostasis and the immune system, as well as indirect interactions via metabolites. Nutrition interventions and nutritional care are mechanisms for addressing increased nutrient expenditures and are important aspects of supportive management for patients with SCD. Underprivileged areas in Sub-Saharan Africa should be accompanied by efforts to define and promote of the nutritional aspects of SCD. Their importance is key to maintaining well-being and quality of life, especially because new technologies and products remain limited, while the use of native medicinal plant resources is acknowledged.

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“…Among the paediatric group patients who received voxelotor in the HOPE-KIDS 1 trial, 47% had >1 g/dl (gram per decilitre) increase in haemoglobin concentration from baseline by week 24, 35% had >1.5 g/dl increase and 21% had >2.0 g/dl increase with reductions in levels of indirect bilirubin, lactate dehydrogenase and percentage reticulocytes which are all markers of haemolysis. [32] [33] Crinzalizumab and voxelotor has been approved by the Food and Drug administration in the United States [31] [34] but is yet to be widely available for use in Nigeria. These novel agents will improve the quality of life of SCD patients and reduce the frequency of vasoocclusive crises and need for opioid analgesics.…”

Section: Disease Modifying Therapiesmentioning

confidence: 99%

Management of Opioid Use Disorder in Sickle Cell Anaemia amidst Growing Menace in the General Population

Aboluwarin,

Ojuawo,

Akanbi

et al. 2023

OJPed

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Nigeria has a very high number of sickle cell disease (SCD) population with addition of 150,000 babies born annually with the disease. Early infant diagnosis and good care make many of these babies survive to adulthood. Severe pain requiring moderately strong or very strong analgesics is a common presentation of patients with Sickle Cell Anaemia. Paediatricians find ready usefulness of Opioids which are very useful for the painful episodes among these patients. Therefore, the chances of abuse and addiction to these medications become very high and constitute additional burden on the deficient manpower in the health sector. Opioid Use Disorder among Sickle Cell Disease patients has subtle presentation, so a high index of suspicion is required to make both the diagnosis and referral to treatment centres. In this review, the epidemiology, pain pathophysiology, behavioural and pharmacologic therapy have been re-examined.

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Voxelotor: A Hemoglobin S Polymerization Inhibitor for the Treatment of Sickle Cell Disease

Cited by 7 publications

References 11 publications

Voxelotor Treatment Providing Transfusion Independence for Patient With Combined Sickle Cell Disease and Lipopolysaccharide-Responsive and Beige-Like Anchor (LRBA) Deficiency

Voxelotor Treatment Providing Transfusion Independence for Patient With Combined Sickle Cell Disease and Lipopolysaccharide-Responsive and Beige-Like Anchor (LRBA) Deficiency

Sickle Cell Disease Update: New Treatments and Challenging Nutritional Interventions

Management of Opioid Use Disorder in Sickle Cell Anaemia amidst Growing Menace in the General Population

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