My Cart

0 Item(s): $0.00

You have no items in your shopping cart.

Hydroxyurea For Sickle Cell

hydroxyurea for sickle cell

Hydroxyurea, a myelosuppressive agent, is the only effective drug proven to reduce the frequency of painful episodes. It raises the level of HbF and the haemoglobin level. It usually decreases the rate of painful episodes by 50 %. It was first tested in sickle cell disease in 1984.

Mechanism of Action

Given the abundant evidence for laboratory and clinical efficacy of hydroxyurea therapy in children and adults with SCA, it is perhaps surprising that its mechanisms of action for HbF induction remain incompletely understood. Current evidence suggests that several potential mechanisms of action by hydroxyurea may be relevant for patients with SCA, which together lead not only to HbF induction but also to additional benefits. Perhaps the most important mechanism of action is inhibition of ribonucleotide reductase (RR), the enzyme involved in transforming ribonucleosides into deoxyribonucleosides that serve as building blocks for DNA synthesis.

How does hydroxyurea work?

Red blood cells contain hemoglobin. Hemoglobin helps red blood cells carry oxygen from the lungsto other parts of the body. People with normal hemoglobin have mostly Hemoglobin A in their redblood cells. People with sickle cell disease have mostly sickle or Hemoglobin S (Hb S) in their redblood cells. Hb S is an abnormal type of hemoglobin. In people with sickle cell disease, Hb S causesthe red blood cells to change from a round shape to a sickle or banana shape. Also, Hb S causes thered blood cells to become rigid and sticky. This leads to blockage of blood fl ow to important bodyorgans, muscles, and tissues.

Use of hydroxyurea

Hydroxyurea time line

Clinical experience has been accumulating for more than 25 years regarding the safety and efficacy of hydroxyurea therapy for patients with SCA. Figure 1 illustrates a time line for hydroxyurea treatment in this patient population, beginning with early “proof-of-principle” studies in adults.11–14 In the early 1990s, an important prospective phase 1/2 study in adults15 using hydroxyurea at maximum tolerated dose (MTD) was followed by the pivotal NHLBI-sponsored double-blinded, placebo-controlled phase 3 MSH trial.

Have we underestimated Sickle Cell Disease?

In the past there hasn’t really been any effective treatment for sickle cell disease. Therefore, I think doctors and nurses, including myself, have not always talked enough about the seriousness of sickle cell, the long term complications, or the risk of dying from its complications. It almost seemed cruel to just go on about future problems when there was, in truth, very little we could do about it.

What other important information should you know about hydroxyurea?

The drug has to be taken every day, not just when a painful crisis starts. It will not take away the pain in an active crisis. It works by making changes to the blood cells which prevent crises. It reduces sickling, decreases red cell breakdown, and increases protective fetal hemoglobin.

At the 1-month visit, the red blood cells are larger. This is seen on testing and looking under the microscope. These red cells are able to move through the tiny blood vessels more easily and break less easily. Parents often report that their child's appetite has increased.

side effects

  • Bleeding under the skin
  • blisters on the skin
  • bluish or pale color on the skin of the fingers or toes
  • coldness of the fingers or toes
  • 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

Overdose information

Disclaimer: The information in this Family Med-aid is accurate at the time of printing. It provides a summary of information about hydroxyurea and does not contain all possible information about this medicine. Not all side effects are listed. If you have any questions or want more information about hydroxyurea, speak to your healthcare provider.

Is hydroxyurea the only treatment for sickle cell disease?

Hydroxyurea is the only non-invasive option at this time. Chronic red blood cell transfusions have been used for many yearsto treat severe problems of sickle cell disease; however, this introduces the slim but still real chance of transfusion-acquiredHIV or Hepatitis C as well as other problems such as iron overload with liver and heart damage. Also, bone marrow or stem celltransplantation (replacing a patient’s bone marrow with normal bone marrow) can cure sickle cell disease, but requires a suitable donor and can be associated with many severe complications


The evidence shows that hydroxyurea is likely to be effective in the short term at decreasing the frequency of painful episodes and raising fetal haemoglobin levels in the blood in people with SCD. Hydroxyurea is also likely to be effective in preventing first strokes for those at an increased risk of stroke and does not seem to be associated with an increase in any side effects (including serious and life‐threatening side effects).

There is currently not much evidence on whether hydroxyurea is beneficial over a long period of time, what the best dose to take is, or whether treatment causes any long‐term or serious side effects. More studies are needed to answer these questions.

Know More About This Medicine and Buy Now :