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Hydroxyurea In Sickle Cell

hydroxyurea in sickle cell

hydroxyurea in sickle cell, 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. It also decreases the rate of ACS episodes and blood transfusions by ~50 % in adults. It was developed as an anticancer drug and has been used to treat myeloproliferative syndromes-leukemia, melanoma, and ovarian cancer. It was approved for use by FDA in adults. Side effects includes neutropenia, bone marrow suppression, elevation of hepatic enzymes, anorexia, nausea, vomiting and infertility.

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 haemoglobin called hydroxyurea in sickle cell 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.


To make sure hydroxyurea in sickle cell is safe for you, tell your doctor if you have:

  • bone marrow suppression or anemia (low red blood cells);
  • low levels of platelets in your blood;
  • a history of skin cancer;
  • high levels of uric acid in your blood;
  • HIV or AIDS;
  • kidney disease (or if you are on dialysis);
  • liver disease;
  • a pancreas disorder; or
  • if you are receiving chemotherapy or radiation.

What is the evidence?

We have known since the early days of the 1990’s that hydroxyurea in sickle celltreatment works in adults. It prevents crisis and reduces the frequency of crisis. It also was known early on to reduce the hospital admissions and reduce the need for blood transfusions. Very importantly it was proven early to reduce acute chest crisis which is the commonest cause of death in children with sickle cell disease.

Side Effects

  • anemia,
  • myelosuppression, and
  • leukemia.
  • nausea,
  • vomiting,
  • upset stomach,
  • diarrhea,
  • constipation,
  • skin changes such as peeling or discoloration,
  • flu-like symptoms,
  • hair loss,
  • rash,
  • headache,
  • dizziness,
  • drowsiness,

How I use hydroxyurea to treat young patients with sickle cell anemia

Lung and Blood Institute–sponsored Multicenter Study of hydroxyurea in sickle celltrial proved clinical efficacy for preventing acute vaso-occlusive events in severely affected adults. Based on this cumulative experience, hydroxyurea has emerged as an important therapeutic option for children and adolescents with recurrent vaso-occlusive events; recent evidence documents sustained long-term benefits with prevention or reversal of chronic organ damage. Despite abundant evidence for its efficacy, however, hydroxyurea has not yet translated into effective therapy for SCA.


The evidence shows that hydroxyurea in sickle cell 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).

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