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Phase 3 CADENZA Trial Validates QOL Improvements Reported by Patients With CAD Receiving Sutimlimab


Mental and physical health aspects of quality of life (QOL) improved after sutimlimab treatment in patients with cold agglutin disease (CAD) during a phase 3 trial.

Results of the phase 3 CADENZA trial, published in the European Journal of Hematology, show that patients reported improvements in cold agglutin disease (CAD) symptoms after receiving sutimlimab intravenous infusions compared with a placebo group. The results of this trial further confirm the effectiveness of this therapy in improving quality of life for patients with CAD.

CAD is a rare chronic autoimmune hemolytic anemia, causing extreme fatigue and poor quality of life, for which there is currently only 1 approved therapy in the United States, sutimlimab.

“Sutimlimab is a first-in-class, humanized, monoclonal antibody that selectively inhibits C1s and prevents classical complement pathway activation, leaving alternative and lectin pathways intact,” the study authors wrote.

The full 26-week CADENZA study included a total of 22 patients who received sutimlimab and 20 patients who received the placebo. The majority of patients were female, and participants’ ages ranged from 46 to 88 years. Approximately three-quarters of patients had received at least 1 CAD therapy within the last 5 years.

The full study consisted of part A and part B phases. Part A included a randomized, double-blind, placebo-controlled phase, conducted from March 2018 to September 2020. Part B included an open-label extension period for patients to receive sutimlimab treatment for at least 1 year from the last patient completion of part A.

Patients self-reported outcomes through several questionnaires: Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-Fatigue), 12-Item Short Form Health Survey (SF-12), EuroQol visual analogue scale (EQ-VAS), Patient Global Impression of Change (PGIC), and Patient Global Impression of [fatigue] Severity (PGIS).

The effectiveness of sutimlimab was measured by a significant increase from baseline scores. In the sutimlimab group, the mean change of baseline FACIT-Fatigue score increased by more than 5 points. Least-squares mean change in FACIT-Fatigue score from baseline to treatment assessment endpoint was 10.8 v 1.9 points (sutimlimab v placebo, P < .001).

Significant mental component score (MCS) and physical component score (PCS) improvements were reported in the SF-12, with mean changes from baseline to week 26 of 5.65 for sutimlimab and –0.48 for placebo for MCS (P = .065) and 5.54 and 1.57, respectively, for PCS (P = .064).

These improvements reported from baseline scores to the end of treatment assessment validated sutimlimab as effective in improving quality of life for patients with CAD, the authors concluded.

“Along with quick and sustained responses to treatment in hemoglobin and hemolytic markers, treatment with sutimlimab resulted in rapid improvements in PROs (FACIT-Fatigue, SF-12, EQ VAS, PGIC and PGIS) versus placebo-treated patients,” the authors wrote. “These improvements followed improvements in hemoglobin and tracked with normalization of bilirubin and represented [clinically important change] for FACIT-Fatigue and SF-12.”


Röth A, Broome CM, Barcellini W, et al. Sutimlimab provides clinically meaningful improvements in patient‐reported outcomes in patients with cold agglutinin disease: results from the randomized, placebo‐controlled, phase 3 CADENZA study. Eur J Hematol. Published online November 20, 2022. doi:10.1111/ejh.13903

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