淀粉样变性
免疫球蛋白轻链
耐受性
中性粒细胞减少症
抗体
作者
Murielle Roussel,Giampaolo Merlini,Sylvie Chevret,Bertrand Arnulf,Anne-Marie Stoppa,Aurore Perrot,Giovanni Palladini,Lionel Karlin,Bruno Royer,Antoine Huart,Margaret Macro,Pierre Morel,Laurent Frenzel,Cyrille Touzeau,Eileen M Boyle,Véronique Dorvaux,Fabien Le Bras,David Lavergne,Frank Bridoux,Arnaud Jaccard
出处
期刊:Blood
[American Society of Hematology]
日期:2020-04-30
卷期号:135 (18): 1531-1540
被引量:56
标识
DOI:10.1182/blood.2019004369
摘要
Daratumumab is a human monoclonal antibody targeting CD38, an antigen uniformly expressed by plasma cells in multiple myeloma and light-chain amyloidosis (AL). We report the results of a prospective multicenter phase 2 study of daratumumab monotherapy in AL (NCT02816476). Forty previously treated AL patients with a difference between involved and uninvolved free light chains (dFLC) >50 mg/L were included in 15 centers between September of 2016 and April of 2018. Patients received 6 28-day cycles of IV daratumumab, every week for cycles 1 and 2 and every 2 weeks for cycles 3 through 6. Median age was 69 years (range, 45-83). Twenty-six patients had ≥2 organs involved, with heart in 24 and kidney in 26. Median time from diagnosis to enrollment was 23 months (interquartile range, 4-122), with a median of 3 prior therapies (range, 1-5). At data cutoff (September of 2019), all patients discontinued therapy; 33 received the planned 6 cycles. Overall, 22 patients had hematological response, and 19 patients (47.5%) achieved very good partial response (dFLC <40 mg/L) or better. Median time to hematological response was 1 week. Patients with no response after 4 doses were unlikely to respond further. Renal and cardiac responses occurred in 8 and 7 patients, respectively. Daratumumab was well tolerated, with no unexpected adverse events. With a median follow-up of 26 months, the 2-year overall survival rate was 74% (95% confidence interval, 62-81). Daratumumab monotherapy is associated with deep and rapid hematological responses in previously treated AL patients, with a good safety profile. Further studies of daratumumab in combination regimens are warranted.
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