医学
恶心
呕吐
不利影响
内科学
中性粒细胞减少症
口炎
胃肠病学
布苏尔班
单采
发热性中性粒细胞减少症
外科
儿科
化疗
造血干细胞移植
疾病
血小板
标识
DOI:10.1016/s2352-3026(20)30407-5
摘要
Alexis Thompson (Northwestern University Feinberg School of Medicine, Chicago, IL, USA) and colleagues reported results from the ongoing phase 1–2 HGB-206 study (NCT02140554) testing LentiGlobin BB305 in patients with sickle cell disease. CD34+ cells collected by plerixafor mobilisation and apheresis were transduced with BB305 lentiviral vector and LentiGlobin was infused following myeloablative busulfan conditioning. 25 (63%) of 40 patients were treated with LentiGlobin; in 16 evaluable patients with 6 or more months of follow-up, total haemoglobin (Hb) was 11·5 g/dL (9·6–16·2), with HbAT87Q contribution of 5·2 g/dL (2·7–9·4), sickle Hb (HbS) of 6·1 g/dL (4·9–7·8), and median HbS of 60% or less of total Hb. Most common non-haematological grade 3 or worse adverse events were stomatitis (n=15) and febrile neutropenia (n=11). Serious adverse events reported in two or more patients were nausea, opioid withdrawal syndrome, and vomiting (n=2 for all).
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