You are currently viewing Hydroxyurea in sickle cell disease: These features make it a perfect therapeutic agent: Part 2.

Hydroxyurea in sickle cell disease: These features make it a perfect therapeutic agent: Part 2.

If you’ve read the previous article about the vital facts about hydroxyurea in sickle cell disease, you now have an idea about the drug. There are features that any therapeutic intervention in chronic disease, must-have for it be ideal. These fall under laboratory efficacy, clinical efficacy, treatment goals, sustainability, safety, and side effects. Let’s look at what hydroxyurea has in today’s article.

Hydroxyurea is a single agent, that is orally administered, on once-daily dosing. Yes, it is expensive for the common man, but not as expensive when compared to meropenem in terms of what either drug achieves as a therapeutic agent.

Hydroxyurea increases the concentrations of fetal haemoglobin and total haemoglobin. It reduces white blood cells and reticulocytes, as well as lowering lactate dehydrogenase levels in the blood. It reduces the severity of anaemia, leads to fewer vaso-occlusive events, and hospitalization. It also decreases haemolysis.

The drug works in all age groups prevent acute events and chronic organ dysfunction. Its benefits continue over time without medication resistance or tolerance. There are few short-term toxicities that might affect adherence. The drug has a favourable therapeutic index. There are no severe toxicities in the short-term, and no known long-term sequelae or complications directly to the drug.

The above features enable hydroxyurea to reduce the number of painful events, as well as hospitalizations in adults and children. It preserves splenic function in infants who receive it. It is associated with excellent growth and development when used in both school-age and very young patients. Sexual maturation occurs without delay among adolescents taking hydroxyurea.

That said, there are a couple of indications for the use of hydroxyurea in sickle cell anaemia. They include;
-frequent pain crises,
– acute chest syndrome,
– severe or symptomatic anaemia,
– hemolytic alloantibodies or autoantibodies,
– severe complications without benefit from standard therapy like priapism and leg ulcers.

Hydroxyurea may be beneficial in some conditions, though, its clinical efficacy isn’t yet proven. These include chronic transfusion therapy precluded by the presence of multiple alloantibodies, abnormal transcranial Doppler velocities refusing transfusion therapy as well as any history of stroke, non-compliance with a transfusion or chelation therapy.

Contraindications to hydroxyurea use are;
-pregnancy or sexually active women unwilling to use contraception,
-active liver disease like hepatitis B or C,
-documented severe hypersensitivity or toxicity to the drug, and
-history of significant non-compliance with recommended medical care.

In a nutshell, hydroxyurea ticks all the boxes that constitute a perfect therapeutic regimen for sickle cell disease. Next is an article about how we administer this drug and the specifics of monitoring and follow-up.

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MBChB (MUK), Graduate Fellow, Department of Physiology, Makerere University Founder and Content Creator Peer reviewer, Associate Editor

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