高リスクニューロブラストーマ患者の無事故生存に対するタンデム・オトログ性幹細胞移植と単一の移植の効果: ランダム化臨床試験
Julie R Park1,2, Susan G Kreissman3, Wendy B London4
1Department of Pediatrics, Seattle Children's Hospital, Seattle, Washington.
JAMA
|August 28, 2019
まとめ
単独移植と比較して,高リスクの神経芽細胞腫患者の無事生存率 (EFS) が著しく改善されました. しかし,ランダム化率の低さは,神経芽細胞腫の治療におけるこれらの結果の一般化を制限する可能性があります.
科学分野:
- 小児腫瘍学
- 血液学
- 免疫学
背景:
- 高リスクの神経芽細胞腫の治療には,化学療法,自己幹細胞移植による高用量療法,および免疫療法が含まれます.
- 現在の治療基準にもかかわらず,高リスクの神経芽細胞腫の生存率は低いままです.
研究 の 目的:
- 高リスクの神経芽細胞腫患者における単一の自己移植と比較して,タンデム自己移植がイベントフリー生存率 (EFS) を改善するかどうかを評価する.
主な方法:
- 高リスクの神経芽細胞腫の患者355人を対象としたランダム化臨床試験です.
- 患者はランダムに,タンデム移植 (チオテパ/ サイクロフォスファミド,その後,減量カルボプラチン/ エトポシド/ メルファラン) または単発移植 (カルボプラチン/ エトポシド/ メルファラン) に割り当てられました.
- 主なアウトカムはEFSで,最初のイベントまでの時間 (再発,進行,二次悪性腫瘍,または死亡) と定義されました.
主要な成果:
- 3年間のEFSは,単一移植グループ (48. 4%) と比較して,タンデム移植グループ (61. 6%) で有意に高かった.
- 最も一般的な毒性は粘膜および感染性であり,両群の割合は似ています.
- 該当する患者の有意な割合 (297/ 652) はランダム化されず,結果の一般化に潜在的に影響を与えた.
結論:
- タンデム・オートロゴス移植は,高リスクの神経芽細胞腫患者におけるEFSの改善に有意な効果を示しています.
- ランダム化率は低いため,より広範な高リスクニューロブラストーマ集団にこれらの結果を一般化する場合に注意が必要です.
- 移植戦略を最適化し,これらの患者の治療結果を改善するために,さらなる研究が必要になる可能性があります.
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