シークル細胞病
Russell E Ware1, Mariane de Montalembert2, Léon Tshilolo3
1Division of Hematology, Department of Pediatrics, Cincinnati Children's Hospital Medical Center, Cincinnati, OH, USA.
Lancet (London, England)
|February 5, 2017
まとめ
シークル細胞病は 異常な赤血球が血流を阻害するので 激しい痛みと臓器損傷を引き起こします 新しい遺伝子治療は 改善された診断と管理戦略と共に 治療への希望をもたらします
科学分野:
- 血液学
- 遺伝学
- 血管生物学
背景:
- シークル細胞病 (Sickle cell disease,SCD) は,世界中で何百万もの患者を抱える,生命を脅かす血液学的疾患です.
- 異常な赤血球が血管閉塞を引き起こし 組織不血症,炎症,急性痛風を引き起こします
- 慢性臓器損傷と早期の死亡は 繰り返し発生する状血症と 血液溶解性貧血によるものです
研究 の 目的:
- 状細胞疾患の現在の治療戦略と新たな治療戦略を 検討する.
- 診断と病気管理の最近の進歩を強調する.
- SCDの病理生理学と治療における現在進行中の論争に対処するために.
主な方法:
- 状細胞疾患における現在の治療法と進行中の研究の文献レビュー.
- スクリーニング,診断,管理の最近の進歩の分析.
- SCDにおける論争の的側面の議論
主要な成果:
- 現在の治療には輸血とヒドロキシカルバミドが含まれます.幹細胞移植は治療の可能性があります.
- 新興治療には遺伝子治療と遺伝子編集が含まれます
- 最近の進歩には 普遍的な脳卒中リスクスクリーニング, 鉄過量管理の改善, 診療所の診断が含まれています
結論:
- 状細胞疾患の管理には大きな進展があり,新たな治療法が目前に迫っています.
- 病理生理学と管理における論争を解決することは 患者の成果を改善するために不可欠です
- SCDの治療の将来は 先進的な治療法と診断と 総合的なケアを組み合わせるものです
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