Effects of voxelotor: A Synthesis of Findings from 24 Studies

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This information is not medical advice and is not a substitute for diagnosis or treatment by a physician.Data sources and disclaimers (data limitations, copyright, etc.)The analysis on "Effects of voxelotor: A Synthesis of Findings from 24 Studies" on this page is based on PubMed data provided by the U.S. National Library of Medicine (NLM). However, NLM does not endorse or verify these analyses.

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Original Abstract of the Article

Major Research Findings

Voxelotor is a new drug that is being studied for the treatment of sickle cell disease. Voxelotor works by binding to hemoglobin, which increases the hemoglobin's affinity for oxygen and reduces its tendency to polymerize. 1 , 17 Several studies have shown that voxelotor can increase hemoglobin levels and decrease markers of hemolysis. 5 , 11 , 16 Voxelotor may also help to reduce the number of painful crises and hospitalizations in people with sickle cell disease. 5 In addition, voxelotor has been shown to improve intestinal health in mice with sickle cell disease. 22

Benefits and Risks

Benefit Summary

Voxelotor is a promising new treatment for sickle cell disease. It has been shown to increase hemoglobin levels, reduce hemolysis, and potentially decrease the number of painful crises and hospitalizations. 5 , 16 It may also improve intestinal health. 22

Risk Summary

Voxelotor can cause side effects, such as headache, rash, and diarrhea. 5 It may also raise liver enzyme levels and decrease white blood cell counts. 12

Comparison Across Studies

Similarities Across Studies

Studies on voxelotor have consistently shown that it can increase hemoglobin levels and decrease markers of hemolysis. 5 , 17 , 18

Differences Across Studies

There are some differences in the findings of voxelotor studies. For example, the effectiveness of voxelotor may vary depending on the dosage and duration of treatment.

Consistency and Contradictions in Results

The findings on voxelotor are generally consistent. It has been shown to increase hemoglobin levels and decrease markers of hemolysis in multiple studies. 5 , 17 , 18 However, there is some variability in the reported side effects. For example, some studies have reported headache, rash, and diarrhea as side effects, while others have reported increases in liver enzyme levels and decreases in white blood cell counts. 5 , 12

Real-World Application Considerations

Voxelotor is a promising new treatment for sickle cell disease, but it is important to be aware of the potential side effects. Voxelotor is not a cure for sickle cell disease, and it is not effective for everyone with the disease. It is important to talk to your doctor to see if voxelotor is right for you.

Limitations of Current Research

Research on voxelotor is still in its early stages. More research is needed to understand the long-term effects and safety of the drug. More research is also needed to determine if voxelotor is effective for everyone with sickle cell disease.

Future Research Directions

Future research should focus on understanding the long-term effects and safety of voxelotor. Researchers should also investigate whether voxelotor is effective for everyone with sickle cell disease.

Conclusions

Voxelotor is a promising new treatment for sickle cell disease. It has been shown to increase hemoglobin levels and decrease markers of hemolysis. 5 , 17 , 18 However, it is important to be aware of the potential side effects. If you are considering voxelotor, it is important to talk to your doctor about the risks and benefits of the drug.

Literature analysis of 24 papers
Positive Content
23
Neutral Content
1
Negative Content
0
Article Type
4
0
0
7
23
1.

[Impact of voxelotor on hemoglobin electrophoretic and chromatographic profiles].

Author: FellahiSoraya, MouriNadir, GiraudBaptiste, MartinoSuella, de LunaGonzalo, SakkaMehdi, JolyPhilippe, BastardJean-Philippe, GalacterosFrédéric, MoutereauStéphane

Improved oxygen carrying capacity
Reduced sickle cell symptoms

This study provides images of hemoglobin profiles in sickle cell disease patients treated with voxelotor, which can be helpful for monitoring their treatment.

English AbstractA summary of a research paper/publication written in English for a wider audience.
Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.

Language : French

5.

Recent Advances in Sickle-Cell Disease Therapies: A Review of Voxelotor, Crizanlizumab, and L-glutamine.

Author: MigotskyMichael, BeestrumMolly, BadawySherif M

Hemoglobin increase
Pain crisis reduction
SCD treatment
Infusion reactions
Insurance denial
Intolerability

This article reviews post-approval studies of crizanlizumab, voxelotor, and L-glutamine for sickle-cell disease (SCD). All three medications have shown promise in reducing pain crises and improving other SCD-related symptoms. However, barriers to access such as insurance approval and cost remain significant challenges.

Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.
ReviewAn article that critically examines existing research on a particular topic and summarizes the current state of knowledge in the field.

Language : English

6.

Rheological Impact of GBT1118 Cessation in a Sickle Mouse Model.

Author: KanneCeleste K, NeborDanitza, PochronMira, OksenbergDonna, SheehanVivien A

Improved RBC deformability
Sickle cell disease management
Rebound hyperviscosity

Voxelotor is a drug that increases hemoglobin levels in sickle cell disease. This study investigated the potential for rebound hyperviscosity after cessation of voxelotor treatment.

Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.

Language : English

7.

Voxelotor: A Novel Treatment for Sickle Cell Disease.

Author: HerityLeah B, VaughanDaleMarie M, RodriguezLindsey Ritenour, LoweDenise Kozella

Increased Hb levels
Safe and effective
Costly treatment

Voxelotor, a novel agent targeting the pathophysiology of sickle cell disease (SCD), was found to be safe and effective as monotherapy or in combination with hydroxyurea. It may be considered for patients with SCD who have continued anemia and hemolysis despite being on maximum tolerated dose of hydroxyurea or in those who are hydroxyurea intolerant.

Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.
ReviewAn article that critically examines existing research on a particular topic and summarizes the current state of knowledge in the field.

Language : English

8.

An integrated therapeutic approach to sickle cell disease management beyond infancy.

Author: RibeilJean-Antoine, PollockGalia, FrangoulHaydar, SteinbergMartin H

Sickle cell disease treatment

This article discusses the use of hydroxyurea, voxelotor, and crizanlizumab in the treatment of sickle cell disease. The authors recommend that all patients should be started first on maximal doses of hydroxyurea and that voxelotor should be added when the response to hydroxyurea is insufficient.

Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.
ReviewAn article that critically examines existing research on a particular topic and summarizes the current state of knowledge in the field.

Language : English

9.

Advances in the Management of Sickle Cell Disease: New Concepts and Future Horizons.

Author: HigginsTara, MendittoMelissa A, KatartzisStephanie, MatsonKelly L

Sickle cell treatment

This review discusses the FDA-approved pharmacotherapies for sickle cell disease, including hydroxyurea, L-glutamine, crizanlizumab, and voxelotor, outlining their clinical trials, dosing, interactions, side effects, access, and cost.

Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.

Language : English

11.

Voxelotor in adolescents and adults with sickle cell disease (HOPE): long-term follow-up results of an international, randomised, double-blind, placebo-controlled, phase 3 trial.

Author: HowardJo, AtagaKenneth I, BrownRobert C, AchebeMaureen, NdubaVidelis, El-BeshlawyAmal, HassabHoda, AgodoaIrene, TondaMargaret, GraySarah, Lehrer-GraiwerJoshua, VichinskyElliott

Sickle cell disease treatment

This report describes the long-term efficacy and safety of voxelotor, a sickle haemoglobin polymerization inhibitor, for the treatment of sickle cell disease. The results from the HOPE trial showed that voxelotor was effective and safe at 72 weeks of treatment.

Clinical Trial, Phase IIIA Phase III trial is a trial that further rigorously verifies the efficacy and safety of a new drug or treatment, whose efficacy and safety have been confirmed in a Phase II trial, in a large number of patients, and evaluates whether it is superior to existing treatments.
Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.
Multicenter StudyResearch conducted in collaboration with multiple research institutions. It aims to collect a wide range of data and obtain more generalizable results.
Randomized Controlled TrialA clinical trial that randomly assigns participants to treatment and control groups to evaluate the effects of an intervention.

Language : English

12.

Model-informed drug development of voxelotor in sickle cell disease: Exposure-response analysis to support dosing and confirm mechanism of action.

Author: GreenMichelle L, SavicRadojka M, TondaMargaret, JorgaKarin, WashingtonCarla B

Hemolysis reduction
SCD treatment
Alanine aminotransferase increase
White blood cell count decrease

This study evaluated the relationship between voxelotor whole blood concentration and clinical measures of anemia and hemolysis in patients with sickle cell disease (SCD). The results support 1500 mg q.d. as the therapeutic dose for voxelotor in adults and adolescents.

Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.
Research Support, Non-U.S. Gov'tResearch funding provided by government agencies outside the United States.

Language : English

13.

The effect of the antisickling compound GBT1118 on the permeability of red blood cells from patients with sickle cell anemia.

Author: Al BalushiHalima, DufuKobina, ReesDavid C, BrewinJohn N, HannemannAnke, OksenbergDonna, LuDavid C-Y, GibsonJohn S

Sickle cell treatment

GBT1118, a novel compound that increases oxygen affinity and reduces sickling of red blood cells, shows promise for treating sickle cell anemia. It reduces the activity of transport systems involved in red blood cell dehydration, increases red blood cell hydration, and reduces hemolysis.

Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.
Research Support, Non-U.S. Gov'tResearch funding provided by government agencies outside the United States.

Language : English

14.

Research in Sickle Cell Disease: From Bedside to Bench to Bedside.

Author: Salinas CisnerosGabriel, TheinSwee Lay

HbF induction
SCD treatment

Research on sickle cell disease has resulted in new drugs and gene therapies, with the development of hydroxyurea as a standard of care for the disease. Further research on the mechanisms of HU and other treatments led to the approval of three new drugs and promising genetic therapies for the disease.

Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.
ReviewAn article that critically examines existing research on a particular topic and summarizes the current state of knowledge in the field.

Language : English

15.

An Investigation of Structure-Activity Relationships of Azolylacryloyl Derivatives Yielded Potent and Long-Acting Hemoglobin Modulators for Reversing Erythrocyte Sickling.

Author: OmarAbdelsattar M, AbdulmalikOsheiza, GhatgeMohini S, MuhammadYosra A, ParedesSteven D, El-ArabyMoustafa E, SafoMartin K

Antisickling activity
Increased Hb oxygen affinity
SCD treatment
Metabolic instability

This study describes the development of novel antisickling agents that bind to sickle hemoglobin (HbS) to increase its oxygen affinity and inhibit erythrocyte sickling. These agents, designated MMA, show significantly enhanced and sustained pharmacologic activities in vitro.

Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.
Research Support, Non-U.S. Gov'tResearch funding provided by government agencies outside the United States.

Language : English

16.

A phase 1/2 ascending dose study and open-label extension study of voxelotor in patients with sickle cell disease.

Author: HowardJo, HemmawayClaire Jane, TelferPaul, LaytonD Mark, PorterJohn, AwogbadeMoji, MantTimothy, GretlerDaniel D, DufuKobina, HutchaleelahaAthiwat, PatelMira, SiuVincent, DixonSandra, LandsmanNoel, TondaMargaret, Lehrer-GraiwerJoshua

Treats sickle cell disease

This report describes results from a Phase 1/2 trial of voxelotor (GBT440), a first-in-class oral therapy for sickle cell disease. Voxelotor improved hematologic measures, including increased hemoglobin levels and reduced hemolysis and sickling, with no treatment-related serious adverse events.

Clinical Trial, Phase IThe first stage of clinical trials in humans. Evaluates the safety, tolerability, and pharmacokinetics of a new drug/treatment.
Clinical Trial, Phase IIA Phase II trial is a trial that further evaluates the efficacy and safety of a new drug or treatment, whose safety has been confirmed to some extent in a Phase I trial, in a small number of patients.
Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.
Randomized Controlled TrialA clinical trial that randomly assigns participants to treatment and control groups to evaluate the effects of an intervention.
Research Support, Non-U.S. Gov'tResearch funding provided by government agencies outside the United States.

Language : English

17.

Pharmacokinetics and pharmacodynamics of voxelotor (GBT440) in healthy adults and patients with sickle cell disease.

Author: HutchaleelahaAthiwat, PatelMira, WashingtonCarla, SiuVincent, AllenElizabeth, OksenbergDonna, GretlerDaniel D, MantTimothy, Lehrer-GraiwerJosh

SCD treatment

Voxelotor, a haemoglobin (Hb) modulator for sickle cell disease (SCD), showed promising safety, tolerability, pharmacokinetics, and pharmacodynamics profiles in both healthy volunteers and SCD patients.

Clinical Trial, Phase IThe first stage of clinical trials in humans. Evaluates the safety, tolerability, and pharmacokinetics of a new drug/treatment.
Clinical Trial, Phase IIA Phase II trial is a trial that further evaluates the efficacy and safety of a new drug or treatment, whose safety has been confirmed to some extent in a Phase I trial, in a small number of patients.
Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.
Multicenter StudyResearch conducted in collaboration with multiple research institutions. It aims to collect a wide range of data and obtain more generalizable results.
Randomized Controlled TrialA clinical trial that randomly assigns participants to treatment and control groups to evaluate the effects of an intervention.
Research Support, Non-U.S. Gov'tResearch funding provided by government agencies outside the United States.

Language : English

18.

A Phase 3 Randomized Trial of Voxelotor in Sickle Cell Disease.

Author: VichinskyElliott, HoppeCarolyn C, AtagaKenneth I, WareRussell E, NdubaVidelis, El-BeshlawyAmal, HassabHoda, AchebeMaureen M, AlkindiSalam, BrownR Clark, DiuguidDavid L, TelferPaul, TsitsikasDimitris A, ElghandourAshraf, GordeukVictor R, KanterJulie, AbboudMiguel R, Lehrer-GraiwerJoshua, TondaMargaret, IntondiAllison, TongBarbara, HowardJo,

Increased hemoglobin
Reduced hemolysis

Voxelotor, an HbS polymerization inhibitor, significantly increased hemoglobin levels and reduced markers of hemolysis in patients with sickle cell disease. These findings suggest that voxelotor has the potential to modify the course of the disease.

Clinical Trial, Phase IIIA Phase III trial is a trial that further rigorously verifies the efficacy and safety of a new drug or treatment, whose efficacy and safety have been confirmed in a Phase II trial, in a large number of patients, and evaluates whether it is superior to existing treatments.
Comparative StudyResearch that compares multiple groups/interventions to determine differences/similarities in outcomes/characteristics.
Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.
Multicenter StudyResearch conducted in collaboration with multiple research institutions. It aims to collect a wide range of data and obtain more generalizable results.
Randomized Controlled TrialA clinical trial that randomly assigns participants to treatment and control groups to evaluate the effects of an intervention.
Research Support, Non-U.S. Gov'tResearch funding provided by government agencies outside the United States.

Language : English

19.

Voxelotor for the treatment of sickle cell disease in pediatric patients.

Author: BrownClark, TondaMargaret, AbboudMiguel R

SCD treatment

This review provides an overview of sickle cell disease (SCD) and the role of voxelotor, a novel oral therapy, in managing the disease. Voxelotor inhibits the polymerization of sickle hemoglobin, reducing the incidence of end-organ damage (EOD).

Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.
ReviewAn article that critically examines existing research on a particular topic and summarizes the current state of knowledge in the field.
Research Support, Non-U.S. Gov'tResearch funding provided by government agencies outside the United States.

Language : English

20.

GBT440 improves red blood cell deformability and reduces viscosity of sickle cell blood under deoxygenated conditions.

Author: DufuKobina, PatelMira, OksenbergDonna, CabralesPedro

Anti-sickling effect

GBT440, a novel anti-polymerization and anti-sickling agent, is being investigated for the treatment of sickle cell disease, a genetic blood disorder.

Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.

Language : English

21.

Effects of an allosteric hemoglobin affinity modulator on arterial blood gases and cardiopulmonary responses during normoxic and hypoxic low-intensity exercise.

Author: StewartGlenn M, ChaseSteven, CrossTroy J, Wheatley-GuyCourtney M, JoynerMichael J, CurryTimothy, Lehrer-GraiwerJosh, DufuKobina, VlahakisNicholas E, JohnsonBruce D

Improved oxygen saturation
Hyperventilation

Voxelotor, an allosteric hemoglobin modulator, enhanced arterial oxygen saturation during low-intensity exercise in hypoxia. It did not affect oxygen consumption or central hemodynamics but did stimulate hyperventilation.

Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.
Research Support, Non-U.S. Gov'tResearch funding provided by government agencies outside the United States.

Language : English

23.

Emerging pharmacotherapeutic approaches for the management of sickle cell disease.

Author: TorresLidiane, ConranNicola

Reduced VOE frequency

Several new pharmacotherapies for sickle cell disease (SCD) are in late-stage development, targeting specific pathophysiological mechanisms. These include voxelotor, prasugrel, and crizanlizumab, which show promise in preventing vaso-occlusive episodes.

Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.
ReviewAn article that critically examines existing research on a particular topic and summarizes the current state of knowledge in the field.

Language : English

24.

Hope on the Horizon: New and Future Therapies for Sickle Cell Disease.

Author: KuririFahd A

SCD treatment
SCD treatment side effects

This article reviews treatment options for sickle cell disease (SCD), including conventional therapies like hydroxyurea and newer treatments like voxelotor. The article also explores promising future treatments like gene therapy.

Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.
ReviewAn article that critically examines existing research on a particular topic and summarizes the current state of knowledge in the field.

Language : English

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