A MULTICENTER, RETROSPECTIVE STUDY ON REAL-WORLD EXPERIENCE OF PATIENTS WITH SICKLE CELL DISEASE TREATED WITH VOXELOTOR
Author(s): ,
Biree Andemariam
Affiliations:
New England Sickle Cell Institute, University of Connecticut Health,Farmington,États-unis;New England Sickle Cell Institute, University of Connecticut Health,Farmington,Vereinigte Staaten;New England Sickle Cell Institute, University of Connecticut Health,Farmington,Stati Uniti;New England Sickle Cell Institute, University of Connecticut Health,Farmington,United States;New England Sickle Cell Inst
,
Modupe Idowu
Affiliations:
University of Texas Health,Houston,États-unis;University of Texas Health,Houston,Vereinigte Staaten;University of Texas Health,Houston,Stati Uniti;University of Texas Health,Houston,United States;University of Texas Health,Houston,Estados Unidos;University of Texas Health,Houston,Verenigde Staten;University of Texas Health,Houston,Estados Unidos;University of Texas Health,Houston,United States;Uni
,
Nirmish Shah
Affiliations:
Duke University School of Medicine,Durham,États-unis;Duke University School of Medicine,Durham,Vereinigte Staaten;Duke University School of Medicine,Durham,Stati Uniti;Duke University School of Medicine,Durham,United States;Duke University School of Medicine,Durham,Estados Unidos;Duke University School of Medicine,Durham,Verenigde Staten;Duke University School of Medicine,Durham,Estados Unidos;Duk
,
Richard Drachtman
Affiliations:
Rutgers Robert Wood Johnson Medical School,New Brunswick,États-unis;Rutgers Robert Wood Johnson Medical School,New Brunswick,Vereinigte Staaten;Rutgers Robert Wood Johnson Medical School,New Brunswick,Stati Uniti;Rutgers Robert Wood Johnson Medical School,New Brunswick,United States;Rutgers Robert Wood Johnson Medical School,New Brunswick,Estados Unidos;Rutgers Robert Wood Johnson Medical School,N
,
Archana Sharma
Affiliations:
Rutgers Robert Wood Johnson Medical School,New Brunswick,États-unis;Rutgers Robert Wood Johnson Medical School,New Brunswick,Vereinigte Staaten;Rutgers Robert Wood Johnson Medical School,New Brunswick,Stati Uniti;Rutgers Robert Wood Johnson Medical School,New Brunswick,United States;Rutgers Robert Wood Johnson Medical School,New Brunswick,Estados Unidos;Rutgers Robert Wood Johnson Medical School,N
,
Alexander Glaros
Affiliations:
Division of Pediatric Hematology/Oncology,Central Michigan University,Detroit,États-unis;Division of Pediatric Hematology/Oncology,Central Michigan University,Detroit,Vereinigte Staaten;Division of Pediatric Hematology/Oncology,Central Michigan University,Detroit,Stati Uniti;Division of Pediatric Hematology/Oncology,Central Michigan University,Detroit,United States;Division of Pediatric Hematology
,
Maureen Achebe
Affiliations:
Brigham and Women's Hospital,Boston,États-unis;Brigham and Women's Hospital,Boston,Vereinigte Staaten;Brigham and Women's Hospital,Boston,Stati Uniti;Brigham and Women's Hospital,Boston,United States;Brigham and Women's Hospital,Boston,Estados Unidos;Brigham and Women's Hospital,Boston,Verenigde Staten;Brigham and Women's Hospital,Boston,Estados Unidos;Brigham and Women's Hospital,Boston,United St
,
Alecia Nero
Affiliations:
University of Texas Southwestern Medical Center,Dallas,États-unis;University of Texas Southwestern Medical Center,Dallas,Vereinigte Staaten;University of Texas Southwestern Medical Center,Dallas,Stati Uniti;University of Texas Southwestern Medical Center,Dallas,United States;University of Texas Southwestern Medical Center,Dallas,Estados Unidos;University of Texas Southwestern Medical Center,Dallas
,
Susanna Curtis
Affiliations:
Albert Einstein College of Medicine,Bronx,États-unis;Albert Einstein College of Medicine,Bronx,Vereinigte Staaten;Albert Einstein College of Medicine,Bronx,Stati Uniti;Albert Einstein College of Medicine,Bronx,United States;Albert Einstein College of Medicine,Bronx,Estados Unidos;Albert Einstein College of Medicine,Bronx,Verenigde Staten;Albert Einstein College of Medicine,Bronx,Estados Unidos;Alb
Caterina Minnitti
Affiliations:
Albert Einstein College of Medicine,Bronx,États-unis;Albert Einstein College of Medicine,Bronx,Vereinigte Staaten;Albert Einstein College of Medicine,Bronx,Stati Uniti;Albert Einstein College of Medicine,Bronx,United States;Albert Einstein College of Medicine,Bronx,Estados Unidos;Albert Einstein College of Medicine,Bronx,Verenigde Staten;Albert Einstein College of Medicine,Bronx,Estados Unidos;Alb
(Abstract release date: 05/12/22) EHA Library. Andemariam B. 06/10/22; 358343; P1485
Dr. Biree Andemariam
Dr. Biree Andemariam
Contributions
Abstract
Presentation during EHA2022: All (e)Poster presentations will be made available as of Friday, June 10, 2022 (09:00 CEST) and will be accessible for on-demand viewing until Monday, August 15, 2022 on the Congress platform.

Abstract: P1485

Type: Poster presentation

Session title: Sickle cell disease

Background
Sickle cell disease (SCD) is an inherited systemic disorder, with pathology driven by polymerization of sickle hemoglobin (HbS). Voxelotor, a HbS polymerization inhibitor, is approved in the United States for treatment of SCD in adults and pediatric patients aged ≥4 years and in the European Union for the treatment of hemolytic anemia due to SCD in adult and pediatric patients ≥12 years of age as monotherapy or in combination with hydroxycarbamide. Efficacy and safety data from the randomized, placebo-controlled HOPE trial demonstrated the effectiveness of voxelotor in increasing hemoglobin (Hb) levels and reducing markers of hemolysis. Real-world studies complement and expand upon information gathered in randomized clinical trials by providing evidence of treatment safety and efficacy in clinical practice. 

Aims
The Retrospective Study to Evaluate Outcomes in Patients With Sickle Cell Disease Treated With Oxbryta (RETRO) aims to characterize real-world safety and effectiveness of voxelotor in adults and adolescents (aged ≥12 years) with SCD treated with voxelotor as part of their usual care.

Methods
RETRO is a multicenter, post-marketing, retrospective study that collected laboratory and clinical data from patients’ medical records 1 year before and 1 year or more after initiation of voxelotor treatment. Patients with documented SCD who received voxelotor for ≥2 consecutive weeks were included in this analysis.

Results
Data from 216 patients across 9 US sites were collected and analyzed. The mean (SD) patient age was 33.5 (14.2) years, and the mean (SD) duration of voxelotor treatment was 51.1 (25.6) weeks. Reasons for voxelotor prescription (n, %) included reducing the following: anemia (151, 69.9%), pain (51, 23.6%), frequency of vaso-occlusive crises (45, 20.8%), and the need for blood transfusions (17, 7.9%); multiple reasons may have been selected. Most patients were prescribed an initial voxelotor dose of 1500 mg (n=187, 86.6%), and 68.1% (n=147) of patients used hydroxyurea concomitantly. A total of 25.0% (n=54) of patients had a dosage interruption or adjustment. Reasons for dosage change (n, %) included adverse event (AE; 37, 17.1%), other (22, 10.2%), pill burden (2, 0.9%), and lack of efficacy (1, 0.5%); multiple reasons may have been selected.

A total of 198 patients had recorded baseline and post-treatment Hb values. In these patients, the mean (SD) peak observed post-treatment Hb level increased from baseline by 1.4 (1.6) g/dL, from 7.8 (1.5) g/dL to 9.2 (2.0) g/dL (Figure). In patients with recorded baseline and post-treatment indirect bilirubin levels (n=80) and reticulocyte percentages (n=178), the mean (SD) minimum observed post-treatment value for indirect bilirubin decreased from baseline by 1.1 (1.9) mg/dL, from 3.1 (2.0) mg/dL to 1.9 (1.9) mg/dL, and reticulocyte percentage decreased from baseline by 3.8% (5.8%), from 11.6 % (6.8%) to 7.7% (5.1%). The safety and tolerability of voxelotor in the real-world setting will be presented. The most common non-SCD-related treatment-emergent AEs were diarrhea, headache, and rash; 37.0% (n=80) of patients reported ≥1 non-SCD-related AE, and most AEs were mild in severity.

Conclusion
RETRO is the first multicenter study to collect and analyze retrospective data from patients with SCD treated with voxelotor in a real-world setting. These interim results are consistent with the HOPE trial, showing that voxelotor treatment was associated with increased Hb levels and decreased hemolytic markers. The safety data are also consistent with those from the HOPE trial.

Keyword(s): Adverse reaction, Clinical outcome, Hemoglobin, Sickle cell

Presentation during EHA2022: All (e)Poster presentations will be made available as of Friday, June 10, 2022 (09:00 CEST) and will be accessible for on-demand viewing until Monday, August 15, 2022 on the Congress platform.

Abstract: P1485

Type: Poster presentation

Session title: Sickle cell disease

Background
Sickle cell disease (SCD) is an inherited systemic disorder, with pathology driven by polymerization of sickle hemoglobin (HbS). Voxelotor, a HbS polymerization inhibitor, is approved in the United States for treatment of SCD in adults and pediatric patients aged ≥4 years and in the European Union for the treatment of hemolytic anemia due to SCD in adult and pediatric patients ≥12 years of age as monotherapy or in combination with hydroxycarbamide. Efficacy and safety data from the randomized, placebo-controlled HOPE trial demonstrated the effectiveness of voxelotor in increasing hemoglobin (Hb) levels and reducing markers of hemolysis. Real-world studies complement and expand upon information gathered in randomized clinical trials by providing evidence of treatment safety and efficacy in clinical practice. 

Aims
The Retrospective Study to Evaluate Outcomes in Patients With Sickle Cell Disease Treated With Oxbryta (RETRO) aims to characterize real-world safety and effectiveness of voxelotor in adults and adolescents (aged ≥12 years) with SCD treated with voxelotor as part of their usual care.

Methods
RETRO is a multicenter, post-marketing, retrospective study that collected laboratory and clinical data from patients’ medical records 1 year before and 1 year or more after initiation of voxelotor treatment. Patients with documented SCD who received voxelotor for ≥2 consecutive weeks were included in this analysis.

Results
Data from 216 patients across 9 US sites were collected and analyzed. The mean (SD) patient age was 33.5 (14.2) years, and the mean (SD) duration of voxelotor treatment was 51.1 (25.6) weeks. Reasons for voxelotor prescription (n, %) included reducing the following: anemia (151, 69.9%), pain (51, 23.6%), frequency of vaso-occlusive crises (45, 20.8%), and the need for blood transfusions (17, 7.9%); multiple reasons may have been selected. Most patients were prescribed an initial voxelotor dose of 1500 mg (n=187, 86.6%), and 68.1% (n=147) of patients used hydroxyurea concomitantly. A total of 25.0% (n=54) of patients had a dosage interruption or adjustment. Reasons for dosage change (n, %) included adverse event (AE; 37, 17.1%), other (22, 10.2%), pill burden (2, 0.9%), and lack of efficacy (1, 0.5%); multiple reasons may have been selected.

A total of 198 patients had recorded baseline and post-treatment Hb values. In these patients, the mean (SD) peak observed post-treatment Hb level increased from baseline by 1.4 (1.6) g/dL, from 7.8 (1.5) g/dL to 9.2 (2.0) g/dL (Figure). In patients with recorded baseline and post-treatment indirect bilirubin levels (n=80) and reticulocyte percentages (n=178), the mean (SD) minimum observed post-treatment value for indirect bilirubin decreased from baseline by 1.1 (1.9) mg/dL, from 3.1 (2.0) mg/dL to 1.9 (1.9) mg/dL, and reticulocyte percentage decreased from baseline by 3.8% (5.8%), from 11.6 % (6.8%) to 7.7% (5.1%). The safety and tolerability of voxelotor in the real-world setting will be presented. The most common non-SCD-related treatment-emergent AEs were diarrhea, headache, and rash; 37.0% (n=80) of patients reported ≥1 non-SCD-related AE, and most AEs were mild in severity.

Conclusion
RETRO is the first multicenter study to collect and analyze retrospective data from patients with SCD treated with voxelotor in a real-world setting. These interim results are consistent with the HOPE trial, showing that voxelotor treatment was associated with increased Hb levels and decreased hemolytic markers. The safety data are also consistent with those from the HOPE trial.

Keyword(s): Adverse reaction, Clinical outcome, Hemoglobin, Sickle cell

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