Hydroxyurea: Effectively improve symptoms in children with sickle cell anemia in sub-Saharan Africa
Hydroxyurea: Effectively improve symptoms in children with sickle cell anemia in sub-Saharan Africa
December 17, 2018 Source: Ministry of Science and Technology
Window._bd_share_config={ "common":{ "bdSnsKey":{ },"bdText":"","bdMini":"2","bdMiniList":false,"bdPic":"","bdStyle":" 0","bdSize":"16"},"share":{ }};with(document)0[(getElementsByTagName('head')[0]||body).appendChild(createElement('script')) .src='http://bdimg.share.baidu.com/static/api/js/share.js?v=89860593.js?cdnversion='+~(-new Date()/36e5)];Sickle cell anemia (SCA) is a common, life-threatening genetic disorder. The glutamic acid, which is the sixth amino acid of the hemoglobin β-peptide chain, is replaced by valine to form sickle hemoglobin (HbS), which replaces normal Hb (HbA). Most of them have many acute and chronic complications that result in poor quality of life and early death. Globally, the incidence of sickle hemoglobin disease is highest in sub-Saharan Africa, with more than 300,000 babies afflicted each year, accounting for about 1% of newborns in the region. However, there are few screening programs for sickle cell anemia in the region, and only a few large urban centers offer specialized treatment.
Sickle cell
At present, the treatment of SCA is limited, although a large number of studies in recent years have shown that hydroxyurea is a safe and effective drug for controlling SCA and improving the quality of life of patients. However, most of these studies are concentrated in developed countries, and hydroxyurea is safe and effective in Africa. Not sure. Co-existing conditions such as malaria, other infectious diseases and malnutrition prevalent in the region may increase the incidence of toxic effects and limit the response to treatment.
Recently, a study published in the authoritative magazine New England Journal of Medicine has brought new hope. Studies have shown that taking hydroxyurea tablets daily can improve the symptoms of children with SCA in sub-Saharan Africa and can reduce the incidence of vascular occlusion events, infections, malaria, blood transfusions and death.
DOI: 10.1056/NEJMoa1813598
According to this latest study, a total of 606 children aged 1-10 years were screened and received daily hydroxyurea treatment, and 94.2% of patients completed 3 years of treatment and follow-up. Studies have shown that hydroxyurea treatment leads to a significant increase in hemoglobin and fetal hemoglobin levels, and hydroxyurea therapy is feasible and safe for children in sub-Saharan Africa. In addition, the study also showed that the use of hydroxyurea and vascular occlusive pain in SCA patients decreased by an average of 55%, infection by 38%, malaria by 51%, blood transfusion by 67%, and mortality by 70% compared with pre-treatment levels. related. Malaria is very common in Africa.
It is reported that the clinical trial has treated 4 children in sub-Saharan countries. The researchers will conduct long-term follow-up of these children to obtain additional data on growth and development and to collect any possible effects on organ function and fertility.
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