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Hydroxyurea In Sickle Cell Disease

hydroxyurea in sickle cell disease


The major causes of morbidity and mortality in sickle cell disease (SCD) are the acute and long-term consequences of vaso-occlusion and hemolysis, many of which cannot be reversed (eg, tissue infarction, vasculopathy). The approaches that are available for reducing these pathophysiologic processes are regular red blood cell (RBC) transfusions, hydroxyurea therapy, and hematopoietic cell transplantation .

This topic review discusses hydroxyurea therapy in SCD, including the mechanism of action, administration, dosing, and adverse effects. Separate topic reviews present overviews of SCD clinical manifestations and management, and the use of regular RBC transfusions

How I use hydroxyurea in sickle cell disease

Hydroxyurea has many characteristics of an ideal drug for sickle cell anemia (SCA) and provides therapeutic benefit through multiple mechanisms of action. Over the past 25 years, substantial experience has accumulated regarding its safety and efficacy for patients with SCA. Early proof-of-principle studies were followed by prospective phase 1/2 trials demonstrating efficacy in affected adults, then adolescents and children, and more recently infants and toddlers. The phase 3 National Heart, Lung and Blood Institute–sponsored Multicenter Study of Hydroxyurea trial proved clinical efficacy for preventing acute vaso-occlusive events in severely affected adults.

How it works?

Parents may remember that when their child was born they had no sickle cell problems in the first 6 months and very few in the first year. This is because in babies blood there is a special hydroxyurea in sickle cell disease called haemoglobin F (HbF) which protects the sickle haemoglobin (HbS). This is nature’s natural protection against sickle problems. This is very effective as HbF actually carries oxygen around the body releasing it to any sickle blood under stress so preventing the fundamental process that causes almost all of the problems in sickle cell disease.

Hydroxyurea in Practice in Liverpool

In the last 5 years we have been putting more of our children with sickle cell disease on hydroxyurea in sickle cell disease.At Alder Hey in the sickle cell clinic we now have around 70% of all patients with sickle cell disease on hydroxyurea in sickle cell disease. We have not seen any serious problems and we have seen many lives massively improve. Hydroxyurea must be respected. It must be closely monitored to ensure the dose is effective and safe for the individual child. It is potentially dangerous if it is taken carelessly or intermittently. It is important to get the best effect that it is taken every day.

Does hydroxyurea in sickle cell disease Cause Cancer?

Historically, this has been a huge concern. The patients initially treated with hydroxyurea had conditions that increased their risk of malignancy (cancer) but because they developed cancer while on hydroxurea therapy it was attributed to the medication. In sickle cell disease, the answer is no. According to a study published in 2014 looking specifically at patients with sickle cell disease, those that took hydroxyurea were not at increased risk of cancer compared to those who did not take hydroxyurea.


hydroxyurea in sickle cell disease may be associated with certain idiosyncratic side effects in some patients. We reported an unusual desquamating, pruritic rash, decreased libido and a partial complex seizure in 4 patients with sickle cell anemia during treatment with hydroxyurea. Additionally, longitudinal melanonychia was noted in 2 patients on HU for sickle cell disease in our practice. In our experience of treating over 60 patients with hydroxyurea, these side effects are rare. The incidences of the described dermatologic and neurologic side effects

Side Effects

  • Black, tarry stools
  • blackening of the fingernails and toenails
  • blood in the urine or stools
  • pinpoint red spots on the skin
  • sores in the mouth and on the lips
  • unusual bleeding or bruising
  • crater-like lesions on the skin
  • fast heartbeat
  • hives, itching, skin rash
  • irritation
  • joint pain, stiffness, or swelling
  • numbness or tingling of the fingers or toes
  • pain in the fingers or toes
  • swelling of the eyelids, face, lips, hands, or feet
  • tightness in the chest
  • troubled breathing or swallowing
  • unusual tiredness or weakness
  • weight loss

Hydroxyurea in children

The use of hydroxyurea in children potentially brings the greatest rewards in terms of prevention of end organ damage but also carries greater potential risks (Vichinsky, 1997). The possibility of adverse effects on growth and development, and the risks of secondary malignancy in groups exposed to the drug for long periods led to caution. However, a number of promising phase I/II studies, showing profound and sustained increases in HbF without serious adverse events, led to larger scale trials

Dose and monitoring of hydroxyurea in sickle cell disease

The dose of hydroxyurea needed for maximal clinical benefit appears not to be the same as the MTD. Although dose‐dependent myelosuppression is rarely associated with adverse clinical events in the form of invasive infection or bleeding, it seems prudent to minimize doses to reduce the risk of long‐term toxicity, including secondary malignancies. Hydroxyurea is available as capsules of 500 mg and syrup can be made up for paediatric use (generally 100 mg/ml) but is not widely available commercially.

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