Advances in New Drug Therapies for the Management of Sickle Cell Disease

Overview

Journal Expert Opin Orphan Drugs

Publisher Informa Healthcare

Date 2019 Mar 16

PMID 30873300

Citations 8

Authors

Kenneth I Ataga

Payal C Desai

Affiliations

Soon will be listed here.

Abstract

Introduction: Sickle cell disease (SCD) is an orphan disease in the United States, but is highly prevalent worldwide. Only two drugs, hydroxyurea and L-glutamine, are approved for this disease. With an improved understanding of the pathophysiology of SCD as well as the success of several recently approved drugs for other orphan diseases, there is an increased interest in the development of drugs for SCD.

Areas Covered: This review summarizes published studies of drug therapies and ongoing trials of novel agents.

Expert Opinion: The development of drugs with different mechanisms of action offers opportunities for combination and individualized therapy in SCD. In addition to acute pain crisis, the evaluation of other SCD-related complications, exercise capacity, patient reported outcomes and validated surrogate endpoints are necessary to advance drug development. It is important to involve sites in sub-Saharan Africa and India, which have the highest burden of SCD, in trials of novel therapies.

Citing Articles

Hemoglobin Variants as Targets for Stabilizing Drugs.

Zoldakova M, Novotny M, Khakurel K, Zoldak G Molecules. 2025; 30(2).

PMID: 39860253 PMC: 11767434. DOI: 10.3390/molecules30020385.

mARC1 Is the Main Contributor to Metabolic Reduction of -Hydroxyurea.

Klopp C, Zhang X, Campbell M, Kvaskoff D, Struwe M, Warren C J Med Chem. 2024; 67(20):18090-18097.

PMID: 39397364 PMC: 11513889. DOI: 10.1021/acs.jmedchem.4c01148.

The Role of Antiplatelet in the Management of Sickle Cell Disease Patients.

Naramreddy S, Varma A, Taksande A, Meshram R Cureus. 2023; 15(7):e42058.

PMID: 37602132 PMC: 10434724. DOI: 10.7759/cureus.42058.

Determinants of hydroxyurea use among doctors, nurses and sickle cell disease patients in Nigeria.

Isa H, Nnebe-Agumadu U, Nwegbu M, Okocha E, Chianumba R, Brown B PLoS One. 2022; 17(11):e0276639.

PMID: 36355850 PMC: 9682971. DOI: 10.1371/journal.pone.0276639.

Modulating hemoglobin allostery for treatment of sickle cell disease: current progress and intellectual property.

Pagare P, Rastegar A, Abdulmalik O, Omar A, Zhang Y, Fleischman A Expert Opin Ther Pat. 2021; 32(2):115-130.

PMID: 34657559 PMC: 8881396. DOI: 10.1080/13543776.2022.1994945.

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