A prospective phase II of daratumumab in previously treated systemic light chain amyloidosis (AL) patients

• Published in: Blood 2020-02
• Main Authors: Roussel, Murielle, Merlini, Giampaolo, Chevret, Sylvie, Arnulf, Bertrand, Stoppa, Anne Marie, Perrot, Aurore, Palladini, Giovanni, Karlin, Lionel, Royer, Bruno, Huart, Antoine, Macro, Margaret, Morel, Pierre, Frenzel, Laurent, Touzeau, Cyrille, Boyle, Eileen, Dorvaux, Véronique, Le Bras, Fabien, Lavergne, David, Bridoux, Frank, Jaccard, Arnaud
• Format: Article
• Language: English
• Subjects: Life Sciences
• ISSN: 0006-4971; 1528-0020
• DOI: 10.1182/blood.2019004369

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