Opinion Article, J Pharm Sci Emerg Drugs Vol: 11 Issue: 1

Managing Sickle Cell Disease with Drug Therapies

Wenzi Cai^*

Department of Pharmacy, West China Hospital, Sichuan University, Sichuan, China

*Corresponding Author: Wenzi Cai
Department of Pharmacy, West China Hospital, Sichuan University, Sichuan, China
E-mail: caiw@su.edu.cn

Received date: 22 January, 2023, Manuscript No. JPSED-23-92362;

Editor assigned date: 24 January, 2023, Pre QC. JPSED-23-92362(PQ);

Reviewed date: 14 February, 2023, QC No. JPSED-23-92362;

Revised date: 21 February, 2023, Manuscript No. JPSED-23-92362(R);

Published date: 28 February, 2023, DOI: 10.4172/2324-8955.1000124.

Citation: Chai W (2023) Managing Sickle Cell Disease with Drug Therapies. J Pharm Sci Emerg Drugs 11:1.

Description

Sickle cell disease is a genetic blood disorder that affects the hemoglobin molecule in red blood cells. Hemoglobin is a protein that carries oxygen from the lungs to the body's tissues and organs. People with sickle cell disease have an abnormal form of hemoglobin, which causes red blood cells to become abnormal and rigid.These abnormal cells can get stay in small blood vessels, leading to pain, organ damage, and a range of other complications.

Sickle cell disease is caused by mutations in the HBB gene, which provides instructions for making the beta-globin subunit of hemoglobin. The mutations cause the beta-globin subunits to stick together, forming long, rigid rods that distort the shape of the red blood cells. This can lead to a range of health problems, including pain, anemia, and organ damage. While there is currently no cure for SCD, there are several drug therapies available to manage the symptoms and improve quality of life for people with the condition.

Types of drug therapies

Hydroxyurea: This medication is an oral chemotherapy drug that can increase the production of fetal hemoglobin, which can reduce the severity and frequency of painful episodes in sickle cell disease. Hydroxyurea also has other beneficial effects, such as reducing the risk of stroke and improving survival rates.

L-glutamine: This medication is an oral powder that can also increase the production of fetal hemoglobin. It has been shown to reduce the frequency of painful episodes in sickle cell disease and improve patients' quality of life.

Crizanlizumab: This is a new medication that was recently approved by the FDA for the prevention of Vaso-Occlusive Crises (VOCs) in sickle cell disease. It is a monoclonal antibody that targets a molecule called P-selectin, which is involved in the adhesion of sickle cells to blood vessel walls.

Voxelotor: This medication is an oral drug that was recently approved by the FDA for the treatment of sickle cell disease. It works by increasing the affinity of hemoglobin for oxygen, which can help to prevent the formation of sickle cells and reduce the frequency of painful episodes.

Blood transfusions: In some cases, regular blood transfusions may be recommended for patients with sickle cell disease to help reduce the risk of complications such as stroke or organ damage.

In addition to these medications, other treatments may be recommended to manage symptoms of sickle cell disease, such as pain management with non-opioid and opioid medications, oxygen therapy, and hydration. It's important for patients to work closely with their healthcare team to determine the best treatment plan for their individual needs.

Conclusion

Sickle cell disease is a complex genetic blood disorder that affects millions of people worldwide. While there is currently no cure for sickle cell disease, drug therapies have been developed to help manage the symptoms and reduce the risk of complications. Hydroxyurea, Lglutamine, Crizanlizumab, Voxelotor, and blood transfusions are among the drug therapies that have been shown to be effective in treating sickle cell disease. Treatment plans should be individualized based on the patient's specific symptoms and medical history. Ongoing research is focused on developing new drug therapies and improving existing treatments to help improve the lives of individuals living with sickle cell disease. With proper medical care and management, individuals with sickle cell disease can lead healthy and fulfilling lives.