慢性リンパ球性白血病および小リンパ球性リンパ腫 (TRANSCEND CLL 004) のリソカバトゲンマレウセル:多センター,オープン,単一アーム,第1段階試験
Tanya Siddiqi1, David G Maloney2, Saad S Kenderian3
1Department of Hematology and Hematopoietic Cell Transplantation, City of Hope National Medical Center, Duarte, CA, USA.
Lancet (London, England)
|June 9, 2023
まとめ
リソカバテゲン・マラレウセル (リソセル) は,再発性または耐性慢性リンパ球性白血病または小リンパ球性リンパ腫の患者で有意な完全応答率を示した. このCAR T細胞治療は,前回のブルトンチロシンキナーゼ阻害剤およびヴェネトクラックス治療に失敗した患者に有効な選択肢を提供します.
科学分野:
- 腫瘍学
- 免疫療法
- 血液学
背景:
- 慢性リンパ球性白血病 (CLL) と小リンパ球性リンパ腫 (SLL) の患者は,ブルトンチロシンキナーゼ (BTK) 阻害剤とヴェネトクラックス不全の後に再発または耐性疾患を有するが,治療の選択肢は限られている.
- リソカバトゲン・マラレウセル (リソセル) は,試験中のキメリック抗原受容体 (CAR) T細胞療法である.
研究 の 目的:
- BTK阻害剤とヴェネトクラックスの両方で進行した再発または耐性CLL患者におけるリソセルの有効性と安全性を評価する.
- リソセルの推奨フェーズ2用量 (DL2) で完全な応答率 (CR) を決定する.
主な方法:
- TRANSCEND CLL 004はオープン・シングル・アーム・フェーズ1-2試験である.
- 主要な有効性分析は,BTK阻害剤の進行とヴェネトクラックス不全を先行した,第2レベル (DL2) の患者49人に焦点を当てた.
- 主要エンドポイントは,独立したレビューによって評価されたCR率でした.
主要な成果:
- 統計的に有意なCR率18% (患者49人中9人) がDL2 (p=0. 0006) で設定された一次有効性分析で観察されました.
- グレード3のサイトカイン放出症候群は患者の9%で,グレード3の神経学的イベントは18%で発生しました.
- マクロファージ活性化症候群による治療関連死亡が1件発生しました.
結論:
- リゾセルは有意な有効性を示し,再発性/耐性性CLL患者集団において完全な応答を誘導した.
- リソセルの安全性プロファイルは管理可能で,サイトカイン放出症候群と神経学的イベントの割合は管理可能でした.
- Liso- celは,他の治療法を使い果たしたCLL/ SLL患者にとって有望な治療法です.
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