ハイドロキシカルバミドが状性貧血の非常に幼い子供に:多センター,ランダム化,制御試験 (BABY HUG)
Winfred C Wang1, Russell E Ware, Scott T Miller
1St Jude Children's Research Hospital, Memphis, TN 38105, USA. winfred.wang@stjude.org
Lancet (London, England)
|May 17, 2011
まとめ
ハイドロキシカルバミドの治療は,状細胞貧血の非常に幼い子供に痛みを大幅に軽減し,ダクチライト. と腎機能のプライマリエンドポイントは有意な違いを示さなかったが,この薬は
科学分野:
- 小児血液学 小児血液学
- 薬理学 薬理学とは
- クリニック・トライアル 臨床試験
背景:
- シークル細胞貧血は,臓器機能不全や急性合併症を含む乳児の有意な死亡率を引き起こします.
- ハイドロキシカルバミドは,状細胞貧血の成人の合併症を軽減することが知られている.
- この研究では,状血球性貧血の非常に幼い子供におけるヒドロキシカルバミドの効果を調査しました.
研究 の 目的:
- ハイドロキシカルバミドの臓器機能不全および状細胞貧血の乳児における臨床合併症に対する効果を評価する.
- この年齢層におけるヒドロキシカルバミドの検査結果と潜在的な毒性効果を検証する.
主な方法:
- 193人の乳児 (9~18ヶ月) を対象としたランダム化,ダブルブラインド,プラセボ対照試験で,状細胞性貧血 (HbSSまたはHbSβ) やタラセミア (thalassaemia) が確認されました.
- 参加者は2年間,液体ヒドロキシカルバミド (20mg/kg/日) またはプラセボを投与された.
- 主要エンドポイントには臓機能と腎臓機能が含まれ,次要エンドポイントには臨床イベント,血液検査,安全性が含まれていました.
主要な成果:
- ヒドロキシカルバミドは,プラセボと比較して痛みを有意に軽減した (p=0.002) とダクチライト (p<0.0001).
- 主要エンドポイントである臓機能 (p=0.21) と球の濾過率 (p=0.84) で有意な差異は認められなかった.
- ハイドロキシカルバミドは胎児のヘモグロビンを増加させ,白血球の数を減少させ,毒性が限られている (軽度から中等度の中性子減少症).
結論:
- ハイドロキシカルバミドは,状細胞貧血の非常に幼い子供の痛みを軽減する安全性と有効性を実証しています.
- この結果は,状血球貧血と診断された非常に幼いすべての子供に対して,ヒドロキシカルバミドを考慮することを支持しています.
- この小児集団における臓器機能に対する長期的な影響について,さらなる研究が行われる可能性がある.
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