From the Guidelines
Hydroxyurea is primarily taken to treat sickle cell disease, as it increases total and fetal hemoglobin and decreases vaso-occlusive complications, including episodes of painful events and acute chest syndrome, hospitalizations, and need for transfusion. The medication is taken once a day orally and is supplied as a capsule, fast-dissolving tablet, or compounded liquid 1. Some key points to consider when taking hydroxyurea include:
- It has a positive impact on health-related quality of life in people with HbSS or Sb0-thalassemia 1
- The National Heart, Lung, and Blood Institute-funded Pediatric Hydroxyurea Phase III Clinical Trial demonstrated that hydroxyurea is of significant benefit presymptomatically to infants with HbSS or Sb0-thalassemia and has no unique adverse effects, even if initiated in early infancy and continued for more than a decade 1
- Myelosuppression is a potential adverse effect, and a CBC and reticulocyte count should be monitored every 1 to 3 months depending on how long the child has been taking the medication and whether the child has attained a stable dose 1
- The 2014 National Heart, Lung, and Blood Institute guidelines for SCD recommend offering hydroxyurea to every child with HbSS or Sb0-thalassemia at 9 months of age even without clinical symptoms 1.
From the FDA Drug Label
Hydroxyurea capsules are indicated for the treatment of: • Resistant chronic myeloid leukemia. • Locally advanced squamous cell carcinomas of the head and neck (excluding the lip) in combination with chemoradiation. Hydroxyurea is taken for:
- Resistant chronic myeloid leukemia
- Locally advanced squamous cell carcinomas of the head and neck (in combination with chemoradiation) 2
From the Research
Hydroxyurea Uses
- Hydroxyurea is used in the treatment of various myeloproliferative neoplasms (MPN), where it reduces cell proliferation by impairing DNA synthesis leading to decreased hematopoiesis 3
- It is used to treat Chronic myeloid leukemia (CML) and has been shown to have a better survival rate compared to busulphan in patients with Philadelphia chromosome (Ph)-positive CML 4
- Hydroxyurea is also used in the treatment of sickle cell disease, where it raises the level of HbF and haemoglobin, and decreases the rate of painful episodes and acute chest syndrome (ACS) episodes by approximately 50% 5
- Additionally, hydroxyurea has been used to treat other conditions such as melanoma, ovarian cancer, and myeloproliferative syndromes 5
Side Effects and Risks
- Common side effects of hydroxyurea include macrocytic anemia, leucopenia, thrombocytopenia, neutropenia, bone marrow suppression, elevation of hepatic enzymes, anorexia, nausea, vomiting, and infertility 3, 5
- Rare side effects include hemolytic anemia, which has been reported in a few cases 3
- There is also a potential risk of carcinogenesis or leukemogenic risk associated with long-term use of hydroxyurea, as evidenced by a case report of a patient with sickle cell disease who developed acute myeloid leukemia after 26 months of hydroxyurea use 6