Advances in new drug therapies for the management of sickle cell disease

Kenneth Ataga, Payal C. Desai

Research output: Contribution to journalReview article

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.

LanguageEnglish (US)
Pages329-343
Number of pages15
JournalExpert Opinion on Orphan Drugs
Volume6
Issue number5
DOIs
StatePublished - May 4 2018
Externally publishedYes

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Medication Therapy Management
Sickle Cell Anemia
Pharmaceutical Preparations
Rare Diseases
Hydroxyurea
Africa South of the Sahara
Expert Testimony
Acute Pain
Glutamine
India
Biomarkers
Drug Therapy
Therapeutics

All Science Journal Classification (ASJC) codes

  • Pharmacology, Toxicology and Pharmaceutics (miscellaneous)
  • Health Policy
  • Pharmacology (medical)

Cite this

Advances in new drug therapies for the management of sickle cell disease. / Ataga, Kenneth; Desai, Payal C.

In: Expert Opinion on Orphan Drugs, Vol. 6, No. 5, 04.05.2018, p. 329-343.

Research output: Contribution to journalReview article

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