Sutimlimab provides clinically meaningful improvements in patient‐reported outcomes in patients with cold agglutinin disease: Results from the randomised, placebo‐controlled, Phase 3 CADENZA study

• DOI: 10.1111/ejh.13903
• Publication Date: 2022-11-20T11:33:30Z
• Authors: Alexander Röth; Catherine M. Broome; Wilma Barcellini; Bernd Jilma; Quentin A. Hill; David Cella; Tor Henrik Anders...; Masaki Yamaguchi; Michelle Lee; Frank Shafer; Marek Wardęcki; Xiaoyu Jiang; Parija Patel; Florence Joly; Ilene C. Weitz
• Keywords: Clinical Global Impression; Cold agglutinin disease

Cold agglutinin disease (CAD) is a rare chronic autoimmune haemolytic anaemia, driven mainly by classical complement pathway activation, leading to profound fatigue and poor quality of life. In the Phase 3 CADENZA trial, sutimlimab-a C1s inhibitor-rapidly halted haemolysis, increased haemoglobin levels improved versus placebo in patients with CAD without recent history transfusion. Patient-reported outcomes (PROs) included Functional Assessment Chronic Illness Therapy-Fatigue (FACIT-Fatigue), 12-Item Short Form Health Survey (SF-12), EuroQol visual analogue scale (EQ-VAS), Patient Global Impression Change (PGIC) (fatigue) Severity (PGIS). Sutimlimab resulted significant rapid meaningful improvements PROs. From Week 1, FACIT-Fatigue mean score >5 points above baseline (considered clinically important change [CIC]). Least-squares (LS) from treatment assessment timepoint was 10.8 vs. 1.9 (sutimlimab placebo; p < 0.001). Improvements physical (PCS) mental (MCS) component scores SF-12 were also considered CICs (LS changes 26: PCS 5.54 1.57 [p = 0.064]; MCS 5.65 -0.48 0.065]). These findings demonstrate that addition improving haematologic parameters, sutimlimab demonstrates patient-reported benefits. Study registered at www.clinicaltrials.gov: NCT03347422.