期刊
BLOOD REVIEWS
卷 27, 期 6, 页码 279-287出版社
CHURCHILL LIVINGSTONE
DOI: 10.1016/j.blre.2013.09.001
关键词
Sickle cell disease; Haematology; Adult; Paediatric
类别
资金
- Novartis Pharmaceuticals
Sickle cell disease (SCD) is a genetic disorder characterised by anaemia and sickling of red blood cells, leading to chronic haemolytic anaemia, vascular injury, and organ dysfunction. Although children and adults experience many similar symptoms and problems, complications increase with age, leading to early mortality. Hydroxyurea (hydroxycarbamide), the only US Food and Drug Administration-approved treatment continues to be under-utilised and other treatments available to children are often inaccessible for adults. Haematopoietic stem-cell transplantation is a curative option, but is limited by a lack of donors and concerns for transplant-related toxicities. Although comprehensive programs exist in paediatrics, affected adults may not have access to preventative and comprehensive healthcare because of a lack of providers or care coordination. They are often forced to rely on urgent care, leading to increased healthcare utilisation costs and inappropriate treatment. This problem highlights the importance of primary care during the transition from paediatrics to adulthood. (C) 2013 Elsevier Ltd. All rights reserved.
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