Anvisa approves a new pioneer treatment for sickle cell disease, with precise dosage for patients aged 2 years and over

December 23, 2021

The first good news in the health area in 2022 is the approval by Anvisa of the first and only treatment in Brazil for sickle cell disease, based on hydroxyurea, which allows the customization of the dosage according to the age and weight of each patient. The new drug is a hope for thousands of families with young children, especially the black population – an ethnic group in which sickle cell disease is more common – which represents 54% of Brazilians.1  

Sickle cell disease is a serious public health problem in Brazil, due to its high rate of morbidity and mortality by affecting red blood cells, leaving them with a rigid, crescent-shaped shape, which causes them to become trapped in blood vessels. blocking the flow of blood and causing crippling pain and damage to various organs of the body, which requires frequent hospitalization and successive blood transfusions.2 

The effectiveness of hydroxyurea in reducing the mortality of people with sickle cell disease has been known since the beginning of the millennium, but until now its administration had the drawbacks of handling the drug at home and often wasting part of the dose, in addition to allowing errors. in its administration due to the difficulty in understanding the dilution methodology. 

 

Benefits of hydroxyurea in the treatment of sickle cell disease and the importance of an accurate dosage for each patient. 

The first scientific evidence regarding the effectiveness of hydroxyurea in reducing the mortality of people with sickle cell disease was published in 2002, in the United States, at the HU study center of MSH – Management Sciences for Health. The use of hydroxyurea raises the levels of fetal hemoglobin (Hb F) in patients with sickle cell syndrome (SF) and the drug has been studied since then in several groups of patients, adults and children, proving its effectiveness as it leads to improvement. clinical and hematological by reducing the incidence of vaso-occlusive episodes.3  

One of the fundamental aspects to obtain the best results in the treatment of the disease and to minimize the unwanted side effects is the individualized dosage according to the age and weight of each patient. This aspect is particularly important in children, where the administration of hydroxyurea has reduced the frequency of blood transfusions by up to 80% and prevented organ damage.4  

 

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References: 

  1. IBGE – Brazilian Institute of Geography and Statistics. Continuous National Household Sample Survey. Available in: https://biblioteca.ibge.gov.br/visualizacao/livros/liv101651_notas_tecnicas.pdf Accessed in January 2022.

  2. Fiocruz. Sickle cell disease and the use of hydroxyurea. Available in: https://portal.fiocruz.br/video/doenca-falciforme-e-o-uso-da-hidroxiureia Access in January 2022.

  3. Ministry of Health – Federal Government. Sickle cell disease. Hydroxyurea: use and access. Available in: https://www.nupad.medicina.ufmg.br/wp-content/uploads/2016/12/Hidroxiureia-Uso-e-Acesso.pdf Accessed in January 2022.

  4. Scielo Brazil. Efficacy and toxicity of hydroxyurea in children with sickle cell anemia. Available in: https://www.scielo.br/j/rbhh/a/XJgdT3cg4SrdTCgqBPQdZwR/?lang=pt Accessed in January 2022. 

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