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Hydroxyurea And Sickle Cell

hydroxyurea and sickle cell

hydroxyurea and 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.

What are the benefits?

hydroxyurea and sickle cell is not a cure for sickle cell disease but

can help prevent many of the complications of the disease.

It helps to reduce the following:

  • number and severity of pain crises
  • number of hospital admissions
  • number of acute chest syndrome events (chest crisis).
  • need for blood transfusions.
  • It can also:
  • increase life expectancy
  • improve quality of life.

Mechanism of Action

Given the abundant evidence for laboratory and clinical efficacy of hydroxyurea and sickle cell 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.

Is hydroxyurea and sickle cell a cure for sickle cell disease?

No. Hydroxyurea does not cure sickle cell disease. Hydroxyurea can greatly reduce some of thecomplications of the disease. It does not work if it is not taken as instructed. Usually, it takes severalmonths before you will see results or get any benefi t from the medicine. It takes that long to reachthe right dose of hydroxyurea. It is important to remember that hydroxyurea must be taken every dayfor it to work well.

What tests are done to monitor hydroxyurea and sickle cell?

Patients taking hydroxyurea usually have physical exams and have their blood counts checkedevery month. They will be seen more often if needed. If your child is part of a research study,then more tests may be performed. These tests help to determine the long-term risks and benefi tsof hydroxyurea for people with sickle cell disease. Areas of the body tested can include the blood,brain, kidneys, heart, and other internal organs.

Cell Cohort, have documented SCA to be a severe, debilitating hematologic disorder.


The profound clinical effects of HU in children with SCD have been recently reviewed (9,10,11); these are summarized here in Table 1. Much of the work on HU in children with SCD has come from phase II and III trials trials led by Ware et al., including pivotal studies such as phase I/II trial of HU in children with sickle cell anemia (HUG KIDS) (12,13,14), Hydroxyurea Safety and Organ Toxicity Trial (HUSOFT) (15), Pediatric Hydroxyurea Phase III Clinical Trial (BABY HUG) (16,17,18) and an early pediatric trial published in 1999 (12). French investigators have also contributed insights into the impact of HU (19,20). Randomized pediatric trials with HU have demonstrated decreased occurrences of pain episodes (18), acute chest syndrome, hospitalization (8,11,18), transfusion, and splenic autoinfarction

Is hydroxyurea and sickle cell  the only treatment for sickle celldisease?

Hydroxyurea is only one (1) choice of treatment at this time. Red blood cell transfusions are anothertreatment choice. Transfusions have been used for many years to treat stroke and problems with thebrain in people with sickle cell disease.Another treatment choice is stem cell transplant (sometimes called bone marrow transplant).This is the only cure for sickle cell disease. Stem cell transplants replace the patient’s bonemarrow with normal bone marrow. This causes the body to make mostly Hemoglobin A instead ofabnormal Hb S. But transplants require a matched donor and can sometimes cause severe sideeffects, including occasional severe illness or death.

need for preventive therapy

Decades of observational data, including landmark natural history studies from the National Heart, Lung and Blood Institute (NHLBI)–sponsored Cooperative Study of Sickle Cell Disease and the Jamaican Sickle

side effects

The studies in the literature report only very minor side effects. My view is that untreated sickle cell disease is a worse disease for a child to have than acute leukaemia. My observations are that life expectancy and quality of life is worse for most children with sickle cell disease compared to children today with acute leukaemia. When treating leukaemia the patients and doctors accept very severe side effects because the benefits outweigh the side effects.

What should you do if your child misses a dose of hydroxyurea and sickle cell ?

If a dose is missed, do not give a make-up dose.

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