Betibeglogene Autotemcel基因治疗Non-beta(0) /β(0)基因型beta-Thalassemia。

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F右路放倒,汤普森AA, Kwiatkowski杰,波特JB,脱粒机AJ, Hongeng年代,萨奥尔MG, Thuret我Lal,身上M,施耐德曼J,奥尔森TS,木匠B, Amrolia PJ, Anurathapan U, Schambach, Chabannon C,施密特M, Labik我,艾略特H,郭R, Asmal M,科尔文RA,沃尔特斯MC

Betibeglogene Autotemcel基因治疗Non-beta(0) /β(0)基因型beta-Thalassemia。

郑传经地中海J。2022年2月3,386 (5):415 - 427。doi: 10.1056 / NEJMoa2113206。Epub 2021年12月11日。

PubMed ID

34891223 (在PubMed

]

文摘

背景:Betibeglogene autotemcel (beti-cel)基因治疗transfusion-dependent beta-thalassemia包含自体CD34 +造血干细胞和祖细胞转导BB305慢病毒载体编码β球蛋白(β(A-T87Q))的基因。方法:在这个非盲、三期研究中,我们评估的有效性和安全性beti-cel在成人和儿科患者transfusion-dependent beta-thalassemia和non-beta(0) /β(0)基因型。患者接受myeloablation白消安(剂量调整的基础上,药代动力学分析)并收到beti-cel静脉注射。主要终点是输血(即独立。加权平均血红蛋白水平> / = 9克/分升没有红细胞输血> / = 12个月)。结果:共有23名患者登记和接受治疗,平均随访29.5个月(范围,13.0到48.2)。输血独立发生在20 22可以评估的患者(91%),包括6 7例(86%)比12年的年龄更小。平均血红蛋白水平在输血独立是11.7克/分升(范围9.5 - 12.8)。beti-cel灌注后12个月,平均水平的基因therapy-derived成人血红蛋白(HbA) T87Q氨基酸替换(HbA (T87Q))是8.7克/分升(范围5.2 - 10.6)患者输血的独立性。beti-cel的安全性是一致的与busulfan-based myeloablation。 Four patients had at least one adverse event that was considered by the investigators to be related or possibly related to beti-cel; all events were nonserious except for thrombocytopenia (in 1 patient). No cases of cancer were observed. CONCLUSIONS: Treatment with beti-cel resulted in a sustained HbA(T87Q) level and a total hemoglobin level that was high enough to enable transfusion independence in most patients with a non-beta(0)/beta(0) genotype, including those younger than 12 years of age. (Funded by Bluebird Bio; HGB-207 ClinicalTrials.gov number, NCT02906202.).

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Betibeglogene autotemcel