Safety and Effectiveness of Sutimlimab in Cold Agglutinin Disease: A Real‐World International Experience

Sutimlimab is a monoclonal antibody against complement fraction C1s approved for the treatment of hemolytic anemia due to cold agglutinin disease (CAD). Here, we analyzed and report the largest international CAD cohort of sutimlimab-treated patients ever reported to highlight its safety and effectiveness in the real-world setting. We accrued a cohort of 57 CAD patients (median age 73.5 years, 56% females). At baseline, patients had severe to moderate anemia (median Hb 8.9 g/dL) and active hemolysis, with a substantial transfusion burden despite a median of 2 prior therapies, including corticosteroids and rituximab. After sutimlimab initiation, median Hb increased by 2 g/dL within 2 weeks and reached 12 g/dL in 4 weeks, remaining stable up to 24 months. This improvement was paralleled by an early and durable normalization of hemolytic markers. Objective responses were observed in most patients by week 2, with complete responses in approximately 50% by Week 4 and 55%-60% during long-term follow-up. Peripheral cold-induced symptoms did not improve and were associated with reduced response rates. Inadequate reticulocytosis also predicted poorer response and suggests the combination with recombinant erythropoietin. Sutimlimab was generally well tolerated. Infections were the most frequent adverse events (23%); severe infections predominantly occurred in previously rituximab-treated individuals. Hemolytic exacerbations occurred in 16% of cases, mostly due to infections. Thrombotic complications were rare. Overall, sutimlimab demonstrated rapid, durable effectiveness and a favorable safety profile in heavily pretreated real-world CAD patients.

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Publication Details

Journal
American Journal of Hematology
Published
2026-04-16
DOI
https://doi.org/10.1002/ajh.70307
Primary Topic
Blood groups and transfusion
Type
article
Field-Weighted Citation Impact
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article

Safety and Effectiveness of Sutimlimab in Cold Agglutinin Disease: A Real‐World International Experience

Uroš Markovic, B Fattizzo, Fabrizio Pappagallo, George Kannourakis et al.
American Journal of Hematology
Blood groups and transfusion
article

Safety and Effectiveness of Sutimlimab in Cold Agglutinin Disease: A Real‐World International Experience

Uroš Markovic, B Fattizzo, Fabrizio Pappagallo, George Kannourakis, Cinzia Bitetti, Antonio De Vivo, Luana Fianchi, Michael Fillitz, Antonio Giovanni Solimando, Nilla Maschio, Fabrizio Vianello, Giacinto Luca Pedone, Jahanzaib Khwaja, Antonella Sau, Dorela Lame, Bernd Jilma, Elisa Sambruna, Nicola Tumedei, Laura Pavan, Catherine Broome, Luigi Curreli, Yoshitaka Miyakawa, Ulrich Jaeger, Arbana Dizdari, Andrea Patriarca, Riccardo Mario Colombo, Carlo Visco, Francesco Lanza, Maria Bruna Greve, Moritz Kleemiss, Guillaume Moulis, Davide Rapezzi, Jalink Marit, Marc Michel, Johan Cid, Esther N. Oliva, Wilma Barcellini, Vincenzo Sammartano, Francesco Passamonti
article en

Abstract

Sutimlimab is a monoclonal antibody against complement fraction C1s approved for the treatment of hemolytic anemia due to cold agglutinin disease (CAD). Here, we analyzed and report the largest international CAD cohort of sutimlimab-treated patients ever reported to highlight its safety and effectiveness in the real-world setting. We accrued a cohort of 57 CAD patients (median age 73.5 years, 56% females). At baseline, patients had severe to moderate anemia (median Hb 8.9 g/dL) and active hemolysis, with a substantial transfusion burden despite a median of 2 prior therapies, including corticosteroids and rituximab. After sutimlimab initiation, median Hb increased by 2 g/dL within 2 weeks and reached 12 g/dL in 4 weeks, remaining stable up to 24 months. This improvement was paralleled by an early and durable normalization of hemolytic markers. Objective responses were observed in most patients by week 2, with complete responses in approximately 50% by Week 4 and 55%-60% during long-term follow-up. Peripheral cold-induced symptoms did not improve and were associated with reduced response rates. Inadequate reticulocytosis also predicted poorer response and suggests the combination with recombinant erythropoietin. Sutimlimab was generally well tolerated. Infections were the most frequent adverse events (23%); severe infections predominantly occurred in previously rituximab-treated individuals. Hemolytic exacerbations occurred in 16% of cases, mostly due to infections. Thrombotic complications were rare. Overall, sutimlimab demonstrated rapid, durable effectiveness and a favorable safety profile in heavily pretreated real-world CAD patients.

American Journal of Hematology
Università Cattolica del Sacro Cuore (IT), University of Verona (IT), Università degli Studi del Piemonte Orientale “Amedeo Avogadro” (IT), Mater Health Services (AU), University of Padua (IT), Université Fédérale de Toulouse Midi-Pyrénées (FR), Georgetown University (US), University of Milan (IT), Université Paris-Est Créteil (FR), University Hospital Bonn (DE), Leiden University Medical Center (NL), London North West Healthcare NHS Trust (GB), MedStar Georgetown University Hospital (US), Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico (IT), Ospedale "Santa Maria delle Croci" di Ravenna (IT), IRCCS Azienda Ospedliero-Universitaria di Bologna Policlinico di Sant'Orsola (IT), Hanusch Hospital (AT), Ospedale di Santo Spirito (IT), Azienda Unità Sanitaria Locale Della Romagna (IT), Ballarat Health Services (AU), Azienda Ospedaliero Universitaria Ospedali Riuniti (IT), Istituto Oncologico Veneto (IT), Federico II University Hospital (IT), Azienda Ospedaliero Universitaria Maggiore della Carita (IT), Ospedale Madonna Delle Grazie (IT), Hôpitaux Universitaires Henri-Mondor (FR), Azienda Sanitaria Ospedaliera S.Croce e Carle Cuneo (IT), Associazione Italiana Ematologia Oncologia Pediatrica (IT), Azienda Ospedaliera Ospedale Maggiore (IT), Istituti di Ricovero e Cura a Carattere Scientifico (IT), University of Bari Aldo Moro (IT), University of Reggio Calabria (IT), University of Naples Federico II (IT), Universitat de Barcelona (ES), Medical University of Vienna (AT), Saitama Medical University (JP)
Ministero della Salute
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Openalex Percentile: Top 19%
Blood groups and transfusion
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