COLD AGGLUTININ DISEASE | Enjaymo Improves Life Quality And Lessens Fatigue, According to Phase 3 Trial Results

 People with cold agglutinin disease (CAD) reported clinically significant improvements in health-related quality of life after treatment with Sanofi’s Enjaymo (sutimlimab-jome), according to a new analysis of data from the Phase 3 CADENZA clinical trial.

These findings “further augment the primary efficacy outcomes of the placebo-controlled Phase 3 CADENZA study, demonstrating that in addition to providing improvements in hematologic [blood-related] parameters, treatment with [Enjaymo] also results in considerable benefits in PROs [patient-reported outcomes] and QoL [quality of life],” the researchers wrote.

The study, “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,” was published in the European Journal of Haematology.

Enjaymo became the first disease-modifying treatment approved in February 2022 by the US Food and Drug Administration (FDA) in the U.S. for the treatment of hemolytic anemia in adults with CAD. The European Commission also approved the use of Enjaymo in November 2022.

Administered directly into the bloodstream every other week, Enjaymo is designed to block the activation of the classic complement pathway, a group of inflammatory proteins that are overactive in CAD.

Patient-reported data and fatigue measurement

Researchers evaluated whether Enjaymo also improved the quality of life of CAD patients with no recent transfusion. They analyzed patient-reported life quality data from the 42 CADENZA participants: 22 given Enjaymo, and 20 on a placebo.

On a third standardized measure of health-related life quality called the Patient Global Impression of Change, the researchers noted that nearly three-quarters (73.7%) of Enjaymo-treated patients reported some level of improvement after 26 weeks, as compared with less than a third (31.6%) of those on placebo.

Of note, 10.5% of patients receiving Enjaymo reported that their disease had “’very much improved’, “compared with no patients in the placebo group,” the team wrote.

Measures of patient-reported fatigue — the Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-Fatigue) and Patient Global Impression of fatigue Severity — also showed better outcomes with Enjaymo than a placebo.

“Together, the results support the effectiveness of targeting the classical complement pathway in the management of CAD independent of transfusion status,” the researchers added.

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