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SUTIMLIMAB, A COMPLEMENT C1S INHIBITOR, PROVIDES SUSTAINED IMPROVEMENTS IN PATIENT-REPORTED OUTCOMES IN PATIENTS WITH COLD AGGLUTININ DISEASE (CAD): 2 YEAR FOLLOW-UP FROM THE CARDINAL STUDY
Author(s): ,
Alexander Röth
Affiliations:
Department of Hematology and Stem Cell Transplantation,West German Cancer Center, University Hospital Essen, University of Duisburg-Essen,Essen,Allemagne;Department of Hematology and Stem Cell Transplantation,West German Cancer Center, University Hospital Essen, University of Duisburg-Essen,Essen,Deutschland;Department of Hematology and Stem Cell Transplantation,West German Cancer Center, Universi
,
Catherine M. Broome
Affiliations:
Division of Hematology,MedStar Georgetown University Hospital,Washington, DC,États-unis;Division of Hematology,MedStar Georgetown University Hospital,Washington, DC,Vereinigte Staaten;Division of Hematology,MedStar Georgetown University Hospital,Washington, DC,Stati Uniti;Division of Hematology,MedStar Georgetown University Hospital,Washington, DC,United States;Division of Hematology,MedStar Georg
,
Wilma Barcellini
Affiliations:
Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico,Milan,Italie;Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico,Milan,Italien;Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico,Milan,Italia;Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico,Milan,Italy;Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico,Milan,Italia;Fondazione IRCCS Ca' Granda Ospedale Maggiore
,
Tor Henrik Anderson Tvedt
Affiliations:
Section for Hematology, Department of Medicine,Haukeland University Hospital,Bergen,Norvège;Section for Hematology, Department of Medicine,Haukeland University Hospital,Bergen,Norwegen;Section for Hematology, Department of Medicine,Haukeland University Hospital,Bergen,Norvegia;Section for Hematology, Department of Medicine,Haukeland University Hospital,Bergen,Norway;Section for Hematology, Departm
,
Yoshitaka Miyakawa
Affiliations:
Thrombosis and Hemostasis Center, Saitama Medical University Hospital,Saitama,Japon;Thrombosis and Hemostasis Center, Saitama Medical University Hospital,Saitama,Japan;Thrombosis and Hemostasis Center, Saitama Medical University Hospital,Saitama,Giappone;Thrombosis and Hemostasis Center, Saitama Medical University Hospital,Saitama,Japan;Thrombosis and Hemostasis Center, Saitama Medical University
,
Shirley D'Sa
Affiliations:
UCLH Centre for Waldenström's Macroglobulinemia and Related Conditions, University College London Hospitals NHS Foundation Trust,London,Royaume-uni;UCLH Centre for Waldenström's Macroglobulinemia and Related Conditions, University College London Hospitals NHS Foundation Trust,London,Vereinigtes Königreich;UCLH Centre for Waldenström's Macroglobulinemia and Related Conditions, University College Lo
,
David Cella
Affiliations:
Department of Medical Social Sciences,Center for Patient-Centered Outcomes, Institute for Public Health and Medicine, Feinberg School of Medicine, Northwestern University,Chicago, IL,États-unis;Department of Medical Social Sciences,Center for Patient-Centered Outcomes, Institute for Public Health and Medicine, Feinberg School of Medicine, Northwestern University,Chicago, IL,Vereinigte Staaten;Depa
,
Florence Joly
Affiliations:
Sanofi,Chilly-Mazarin,France;Sanofi,Chilly-Mazarin,Frankreich;Sanofi,Chilly-Mazarin,Francia;Sanofi,Chilly-Mazarin,France;Sanofi,Chilly-Mazarin,Francia;Sanofi,Chilly-Mazarin,Frankrijk;Sanofi,Chilly-Mazarin,França;Sanofi,Chilly-Mazarin,Франция ;Sanofi,Chilly-Mazarin,Frankrike
,
Jennifer Wang
Affiliations:
Sanofi,Cambridge, MA,États-unis;Sanofi,Cambridge, MA,Vereinigte Staaten;Sanofi,Cambridge, MA,Stati Uniti;Sanofi,Cambridge, MA,United States;Sanofi,Cambridge, MA,Estados Unidos;Sanofi,Cambridge, MA,Verenigde Staten;Sanofi,Cambridge, MA,Estados Unidos;Sanofi,Cambridge, MA,United States;Sanofi,Cambridge, MA,USA
,
Timothee Sourdille
Affiliations:
Sanofi,Cambridge, MA,États-unis;Sanofi,Cambridge, MA,Vereinigte Staaten;Sanofi,Cambridge, MA,Stati Uniti;Sanofi,Cambridge, MA,United States;Sanofi,Cambridge, MA,Estados Unidos;Sanofi,Cambridge, MA,Verenigde Staten;Sanofi,Cambridge, MA,Estados Unidos;Sanofi,Cambridge, MA,United States;Sanofi,Cambridge, MA,USA
,
Frank Shafer
Affiliations:
Sanofi,Bridgewater, NJ,États-unis;Sanofi,Bridgewater, NJ,Vereinigte Staaten;Sanofi,Bridgewater, NJ,Stati Uniti;Sanofi,Bridgewater, NJ,United States;Sanofi,Bridgewater, NJ,Estados Unidos;Sanofi,Bridgewater, NJ,Verenigde Staten;Sanofi,Bridgewater, NJ,Estados Unidos;Sanofi,Bridgewater, NJ,United States;Sanofi,Bridgewater, NJ,USA
,
Marek Wardęcki
Affiliations:
Sanofi,Warsaw,Pologne;Sanofi,Warsaw,Polen;Sanofi,Warsaw,Polonia;Sanofi,Warsaw,Poland;Sanofi,Warsaw,Polonia;Sanofi,Warsaw,Polen;Sanofi,Warsaw,Polonia;Sanofi,Warsaw,Польша;Sanofi,Warsaw,Polen
Ilene C. Weitz
Affiliations:
Keck School of Medicine of USC,Los Angeles, CA,États-unis;Keck School of Medicine of USC,Los Angeles, CA,Vereinigte Staaten;Keck School of Medicine of USC,Los Angeles, CA,Stati Uniti;Keck School of Medicine of USC,Los Angeles, CA,United States;Keck School of Medicine of USC,Los Angeles, CA,Estados Unidos;Keck School of Medicine of USC,Los Angeles, CA,Verenigde Staten;Keck School of Medicine of USC
(Abstract release date: 05/12/22) EHA Library. Röth A. 06/11/22; 357168; S304
Alexander Röth
Alexander Röth
Contributions
Abstract
Presentation during EHA2022: All Oral presentations will be presented between Friday, June 10 and Sunday, June 12 and will be accessible for on-demand viewing from Monday, June 20 until Monday, August 15, 2022 on the Congress platform.

Abstract: S304

Type: Oral Presentation

Session title: Quality of life and health econimics in hematological patients

Background
CAD is a rare chronic autoimmune hemolytic anemia characterized by classical complement pathway (CP)-mediated hemolysis, anemia, fatigue, and poor quality of life (QOL). Sutimlimab is a first-in-class humanized monoclonal antibody that selectively inhibits C1s of the C1 complex, preventing CP activation, while leaving the alternative and lectin pathways intact. One-year interim follow-up from the CARDINAL study (NCT03347396) have previously demonstrated continuous classical CP inhibition with sutimlimab resulted in rapid, sustained improvements in all patient-reported outcomes (PROs) measures evaluated.

Aims
To report sutimlimab effect on PROs at 2 years, from the CARDINAL Part B extension.

Methods
CARDINAL was a Phase 3, open-label, single-arm study with a 26-week treatment period (Part A) and a 2-year extension (Part B) after the last patient finishes Part A. Sutimlimab was administered through intravenous infusions on Days 0 and 7, followed by biweekly dosing. PRO data through Week 135, the last data recording within the 2-year Part B time period, are reported here. Efficacy endpoints included hemolytic markers and the Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-Fatigue) as a measure of QOL. Exploratory QOL endpoints included mean change from baseline in EuroQol 5-dimension 5-level (EQ-5D-5L) scores, 12-Item Short Form Health Survey (SF-12), Patient Global Impression of Change (PGIC) and Patient Global Impression of Fatigue Severity (PGIS). PRO measures were evaluated every 3 months and conducted in the following order: FACIT-Fatigue, PGIS, PGIC, SF-12 and EQ-5D-5L. Descriptive statistics, frequency, or percentage were used to analyze outcomes.

Results
Overall, 24 patients enrolled and 22 finished Part A and entered Part B, with 19 (86.4%) patients completing Part B. The mean (SD) FACIT-Fatigue score at baseline was 32 (11) and improved by 7 (8) within 1 week of sutimlimab treatment; the mean change score from baseline remained ≥5 from Week 1 to Week 135 (minimum score of 38 (9) at Week 123, maximum score of 44 (5) at Week 7), consistent with a clinically meaningful improvement. Efficacy for the EQ-5D-5L visual analogue scale (VAS) was sustained over 2 years; the mean (SD) change from baseline in EQ-5D-5L visual analogue scale (VAS) score (n=15/22) at Week 135 was 17.1 (21.6) (Figure A). The majority of evaluable patients (n=13/15) indicated an improved disease state compared to baseline on the PGIC at Week 135. No or mild fatigue was reported in patients (80%, n=12/15) on completing PGIS at Week 135, compared with one-third at baseline (n=2/6). The mean increase in SF-12 physical and mental component scores (n=6/22) from baseline to Week 123 were 4.7 (6.9) and 3.8 (14.1) (Figure B), consistent with the clinically important changes of 3.9 and 2.8 respectively.

Conclusion
From this CARDINAL follow-up extension study, sutimlimab has shown to produce rapid and sustained improvements in FACIT-Fatigue and other PRO measures evaluated up to 2 years, demonstrating the continued meaningful impact of sutimlimab on patient QOL.

Keyword(s): Autoimmune hemolytic anemia (AIHA), Complement, Monoclonal antibody, Patient reported outcomes

Presentation during EHA2022: All Oral presentations will be presented between Friday, June 10 and Sunday, June 12 and will be accessible for on-demand viewing from Monday, June 20 until Monday, August 15, 2022 on the Congress platform.

Abstract: S304

Type: Oral Presentation

Session title: Quality of life and health econimics in hematological patients

Background
CAD is a rare chronic autoimmune hemolytic anemia characterized by classical complement pathway (CP)-mediated hemolysis, anemia, fatigue, and poor quality of life (QOL). Sutimlimab is a first-in-class humanized monoclonal antibody that selectively inhibits C1s of the C1 complex, preventing CP activation, while leaving the alternative and lectin pathways intact. One-year interim follow-up from the CARDINAL study (NCT03347396) have previously demonstrated continuous classical CP inhibition with sutimlimab resulted in rapid, sustained improvements in all patient-reported outcomes (PROs) measures evaluated.

Aims
To report sutimlimab effect on PROs at 2 years, from the CARDINAL Part B extension.

Methods
CARDINAL was a Phase 3, open-label, single-arm study with a 26-week treatment period (Part A) and a 2-year extension (Part B) after the last patient finishes Part A. Sutimlimab was administered through intravenous infusions on Days 0 and 7, followed by biweekly dosing. PRO data through Week 135, the last data recording within the 2-year Part B time period, are reported here. Efficacy endpoints included hemolytic markers and the Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-Fatigue) as a measure of QOL. Exploratory QOL endpoints included mean change from baseline in EuroQol 5-dimension 5-level (EQ-5D-5L) scores, 12-Item Short Form Health Survey (SF-12), Patient Global Impression of Change (PGIC) and Patient Global Impression of Fatigue Severity (PGIS). PRO measures were evaluated every 3 months and conducted in the following order: FACIT-Fatigue, PGIS, PGIC, SF-12 and EQ-5D-5L. Descriptive statistics, frequency, or percentage were used to analyze outcomes.

Results
Overall, 24 patients enrolled and 22 finished Part A and entered Part B, with 19 (86.4%) patients completing Part B. The mean (SD) FACIT-Fatigue score at baseline was 32 (11) and improved by 7 (8) within 1 week of sutimlimab treatment; the mean change score from baseline remained ≥5 from Week 1 to Week 135 (minimum score of 38 (9) at Week 123, maximum score of 44 (5) at Week 7), consistent with a clinically meaningful improvement. Efficacy for the EQ-5D-5L visual analogue scale (VAS) was sustained over 2 years; the mean (SD) change from baseline in EQ-5D-5L visual analogue scale (VAS) score (n=15/22) at Week 135 was 17.1 (21.6) (Figure A). The majority of evaluable patients (n=13/15) indicated an improved disease state compared to baseline on the PGIC at Week 135. No or mild fatigue was reported in patients (80%, n=12/15) on completing PGIS at Week 135, compared with one-third at baseline (n=2/6). The mean increase in SF-12 physical and mental component scores (n=6/22) from baseline to Week 123 were 4.7 (6.9) and 3.8 (14.1) (Figure B), consistent with the clinically important changes of 3.9 and 2.8 respectively.

Conclusion
From this CARDINAL follow-up extension study, sutimlimab has shown to produce rapid and sustained improvements in FACIT-Fatigue and other PRO measures evaluated up to 2 years, demonstrating the continued meaningful impact of sutimlimab on patient QOL.

Keyword(s): Autoimmune hemolytic anemia (AIHA), Complement, Monoclonal antibody, Patient reported outcomes

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