Paper Details

  1. Home
  2. Paper Details

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

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

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.

Overview

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.
Paper Details　
Original Abstract of the Article :
Sickle cell disease (SCD) is characterized by the production of sickle hemoglobin (HbS), which when deoxygenated, polymerizes leading to red blood cell damage and hemolytic anemia, a defining feature of SCD. Voxelotor (Oxbryta) is a small molecule inhibitor of HbS polymerization that disrupts the po...See full text at original site
Dr.Camel IconDr.Camel's Paper Summary Blogラクダ博士について

ラクダ博士は、Health Journal が論文の内容を分かりやすく解説するために作成した架空のキャラクターです。
難解な医学論文を、専門知識のない方にも理解しやすいように、噛み砕いて説明することを目指しています。

* ラクダ博士による解説は、あくまで論文の要点をまとめたものであり、原論文の完全な代替となるものではありません。詳細な内容については、必ず原論文をご参照ください。
* ラクダ博士は架空のキャラクターであり、実際の医学研究者や医療従事者とは一切関係がありません。
* 解説の内容は Health Journal が独自に解釈・作成したものであり、原論文の著者または出版社の見解を反映するものではありません。


引用元：
https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9197532/

データ提供：米国国立医学図書館（NLM）

Voxelotor: A Model-Informed Drug Development for Sickle Cell Disease

Sickle cell disease (SCD) is a debilitating genetic disorder characterized by red blood cell abnormalities. This study explores the development of voxelotor, a small molecule inhibitor of sickle hemoglobin (HbS) polymerization, as a treatment for SCD. Like a resourceful camel adapting to a harsh desert environment, this research aims to develop innovative therapies to alleviate the challenges of SCD.

Voxelotor: Efficacy and Safety in Sickle Cell Disease

The study found that voxelotor was effective in increasing hemoglobin levels and reducing hemolysis in patients with SCD. The 1500 mg once-daily dose was found to be well-tolerated, with most side effects being mild. These findings support the use of voxelotor as a promising treatment for SCD.

Developing Personalized Treatment Strategies for Sickle Cell Disease

This study demonstrates the potential of model-informed drug development to optimize treatment strategies for SCD. By carefully evaluating the relationship between drug exposure and clinical response, researchers can refine dosing recommendations and ensure that patients receive the most effective and safe treatment.

Dr. Camel's Conclusion

This research offers a beacon of hope for those living with SCD. Voxelotor emerges as a promising treatment option, showcasing the potential of scientific innovation to improve the lives of patients with this challenging condition.

Date :
  1. Date Completed 2022-06-16
  2. Date Revised 2022-08-18
Further Info :

Pubmed ID

35447017

DOI: Digital Object Identifier

PMC9197532

Related Literature

Article Analysis
・Effects of voxelotor
SNS
PICO Info
in preparation
Languages

English

Positive IndicatorAn AI analysis index that serves as a benchmark for how positive the results of the study are. Note that it is a benchmark and requires careful interpretation and consideration of different perspectives.

This site uses cookies. Visit our privacy policy page or click the link in any footer for more information and to change your preferences.