慢性免疫性血栓減少症 (PETIT2) の子供に対するエルトロンボパグ: ランダム化され,多センター,プラセボ対照試験
John D Grainger1, Franco Locatelli2, Thirachit Chotsampancharoen3
1Department of Haematology, Royal Manchester Children's Hospital, University of Manchester, Manchester, UK; Central Manchester Hospitals NHS Foundation Trust, NIHR/Wellcome Trust Manchester CRF, Royal Manchester Children's Hospital, University of Manchester, Manchester, UK.
Lancet (London, England)
|August 2, 2015
まとめ
Eltrombopagは慢性免疫性血小板減少症の小児の血小板数を効果的に増加させ,安全で適切な治療法を示した. 副作用による治療中止は少ない.
科学分野:
- 小児血液学
- 薬理学について
- 臨床試験
背景:
- 慢性免疫性血小板減少症 (ITP) は,小児の血小板数に影響する.
- エルトロンボパグは,トランポエチン受容体アゴニストであり,成人のITPの治療に承認されています.
- 小児のITP治療の有効性と安全性については,さらなる調査が必要である.
研究 の 目的:
- 慢性 ITP の子供におけるエルトロンボパグの安全性と有効性を評価する.
- 適切な血小板数を達成し維持するエルトロンボパグの能力を評価する.
主な方法:
- 92人の小児患者 (1~17歳) を対象としたランダム化,ダブルブラインド,プラセボ対照多センター試験 (PETIT2)
- 患者は13週間エルトロンボパグまたはプラセボを受け,その後はオープンラベルエルトロンボパグ期間でした.
- 5~12週間の6週間にわたって血小板数 ≥50×10^9/ Lを達成した患者の割合
主要な成果:
- プラセボ受験者の3%に対して,エルトロンボパグ受験者の40%が主要な結果を達成した (OR18. 0; p=0. 0004).
- 血小板の反応は,各年齢 (12-17歳,6-11歳,1-5歳) において一貫していました.
- 死亡,悪性腫瘍,または血栓症は発生しなかった.
結論:
- Eltrombopagは,慢性的な症状のITPを有する,持続的な血小板反応を誘発する子供に適した治療法です.
- この薬は小児患者で好ましい安全性プロファイルを示し,安全性に関する新たな懸念は認められなかった.
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