TY - JOUR
T1 - Efficacy and Safety of Bimagrumab in Sporadic Inclusion Body Myositis Long-term Extension of RESILIENT
AU - RESILIENT Study Extension Group
AU - Amato, Anthony A.
AU - Hanna, Michael G.
AU - Machado, Pedro M.
AU - Badrising, Umesh A.
AU - Chinoy, Hector
AU - Benveniste, Olivier
AU - Karanam, Ananda Krishna
AU - Wu, Min
AU - Tankó, László B.
AU - Schubert-Tennigkeit, Agnes Annette
AU - Papanicolaou, Dimitris A.
AU - Lloyd, Thomas E.
AU - Needham, Merrilee
AU - Liang, Christina
AU - Reardon, Katrina A.
AU - de Visser, Marianne
AU - Ascherman, Dana P.
AU - Barohn, Richard J.
AU - Dimachkie, Mazen M.
AU - Miller, James A.L.
AU - Kissel, John T.
AU - Oskarsson, Björn
AU - Joyce, Nanette C.
AU - Van den Bergh, Peter
AU - Baets, Jonathan
AU - De Bleecker, Jan L.
AU - Karam, Chafic
AU - David, William S.
AU - Mirabella, Massimiliano
AU - Nations, Sharon P.
AU - Jung, Hans H.
AU - Pegoraro, Elena
AU - Maggi, Lorenzo
AU - Rodolico, Carmelo
AU - Filosto, Massimiliano
AU - Shaibani, Aziz I.
AU - Sivakumar, Kumaraswamy
AU - Goyal, Namita A.
AU - Mori-Yoshimura, Madoka
AU - Yamashita, Satoshi
AU - Suzuki, Naoki
AU - Aoki, Masashi
AU - Katsuno, Masahisa
AU - Morihata, Hirokazu
AU - Murata, Kenya
AU - Nodera, Hiroyuki
AU - Nishino, Ichizo
AU - Romano, Carla D.
AU - Williams, Valerie S.L.
AU - Vissing, John
N1 - Publisher Copyright:
© 2021 Lippincott Williams and Wilkins. All rights reserved.
PY - 2021
Y1 - 2021
N2 - Objective To assess long-term (2 years) effects of bimagrumab in participants with sporadic inclusion body myositis (sIBM). Methods Participants (aged 36–85 years) who completed the core study (RESILIENT [Efficacy and Safety of Bimagrumab/BYM338 at 52 Weeks on Physical Function, Muscle Strength, Mobility in sIBM Patients]) were invited to join an extension study. Individuals continued on the same treatment as in the core study (10 mg/kg, 3 mg/kg, 1 mg/kg bimagrumab or matching placebo administered as IV infusions every 4 weeks). The co–primary outcome measures were 6-minute walk distance (6MWD) and safety. Results Between November 2015 and February 2017, 211 participants entered double-blind placebo-controlled period of the extension study. Mean change in 6MWD from baseline was highly variable across treatment groups, but indicated progressive deterioration from weeks 24–104 in all treatment groups. Overall, 91.0% (n = 142) of participants in the pooled bimagrumab group and 89.1% (n = 49) in the placebo group had ≥1 treatment-emergent adverse event (AE). Falls were slightly higher in the bimagrumab 3 mg/kg group vs 10 mg/kg, 1 mg/kg, and placebo groups (69.2% [n = 36 of 52] vs 56.6% [n = 30 of 53], 58.8% [n = 30 of 51], and 61.8% [n = 34 of 55], respectively). The most frequently reported AEs in the pooled bimagrumab group were diarrhea 14.7% (n = 23), involuntary muscle contractions 9.6% (n = 15), and rash 5.1% (n = 8). Incidence of serious AEs was comparable between the pooled bimagrumab and the placebo group (18.6% [n = 29] vs 14.5% [n = 8], respectively). Conclusion Extended treatment with bimagrumab up to 2 years produced a good safety profile and was well-tolerated, but did not provide clinical benefits in terms of improvement in mobility. The extension study was terminated early due to core study not meeting its primary endpoint.
AB - Objective To assess long-term (2 years) effects of bimagrumab in participants with sporadic inclusion body myositis (sIBM). Methods Participants (aged 36–85 years) who completed the core study (RESILIENT [Efficacy and Safety of Bimagrumab/BYM338 at 52 Weeks on Physical Function, Muscle Strength, Mobility in sIBM Patients]) were invited to join an extension study. Individuals continued on the same treatment as in the core study (10 mg/kg, 3 mg/kg, 1 mg/kg bimagrumab or matching placebo administered as IV infusions every 4 weeks). The co–primary outcome measures were 6-minute walk distance (6MWD) and safety. Results Between November 2015 and February 2017, 211 participants entered double-blind placebo-controlled period of the extension study. Mean change in 6MWD from baseline was highly variable across treatment groups, but indicated progressive deterioration from weeks 24–104 in all treatment groups. Overall, 91.0% (n = 142) of participants in the pooled bimagrumab group and 89.1% (n = 49) in the placebo group had ≥1 treatment-emergent adverse event (AE). Falls were slightly higher in the bimagrumab 3 mg/kg group vs 10 mg/kg, 1 mg/kg, and placebo groups (69.2% [n = 36 of 52] vs 56.6% [n = 30 of 53], 58.8% [n = 30 of 51], and 61.8% [n = 34 of 55], respectively). The most frequently reported AEs in the pooled bimagrumab group were diarrhea 14.7% (n = 23), involuntary muscle contractions 9.6% (n = 15), and rash 5.1% (n = 8). Incidence of serious AEs was comparable between the pooled bimagrumab and the placebo group (18.6% [n = 29] vs 14.5% [n = 8], respectively). Conclusion Extended treatment with bimagrumab up to 2 years produced a good safety profile and was well-tolerated, but did not provide clinical benefits in terms of improvement in mobility. The extension study was terminated early due to core study not meeting its primary endpoint.
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U2 - 10.1212/WNL.0000000000011626
DO - 10.1212/WNL.0000000000011626
M3 - Article
C2 - 33597289
AN - SCOPUS:85103473554
SN - 0028-3878
VL - 96
SP - E1595-E1607
JO - Neurology
JF - Neurology
IS - 12
ER -