REAL-WORLD EXPERIENCE OF VOXELOTOR FOR THE TREATMENT OF PATIENTS WITH SICKLE CELL DISEASE – A SINGLE-CENTER STUDY
Author(s): ,
Kathryn Muschick
Affiliations:
Prisma Health – Upstate Comprehensive SCD Program,Greenville,United States
,
Tranaka Fuqua
Affiliations:
Prisma Health – Upstate Comprehensive SCD Program,Greenville,United States
,
Carrianne Stoker-Postier
Affiliations:
Prisma Health – Upstate Comprehensive SCD Program,Greenville,United States
Alan R. Anderson
Affiliations:
Prisma Health – Upstate Comprehensive SCD Program, University of South Carolina School of Medicine,Greenville,United States
EHA Library. Anderson A. 06/09/21; 324930; EP1209
Alan Anderson
Alan Anderson
Contributions
Abstract
Presentation during EHA2021: All e-poster presentations will be made available as of Friday, June 11, 2021 (09:00 CEST) and will be accessible for on-demand viewing until August 15, 2021 on the Virtual Congress platform.

Abstract: EP1209

Type: E-Poster Presentation

Session title: Sickle cell disease

Background
Sickle cell disease (SCD) is an inherited disorder of the red blood cells (RBC) in which sickle hemoglobin (HbS) polymerizes and leads to a detrimental shape change in the RBC. This change, known as sickling, can trigger serious consequences including chronic hemolysis, anemia, and episodic vaso-occlusion and often leads to complications such as cumulative organ damage, disability, and accelerated mortality. Voxelotor is a first-in-class therapy that interferes with sickle cell polymerization, increases hemoglobin (Hb) levels, and reduces hemolysis. It is FDA approved for the treatment of SCD in patients 12 years of age and older.

Aims
The objective of this study was to quantify the clinical response to voxelotor treatment in a single-center series of 73 adult and adolescent patients with SCD.

Methods
We collected real-world clinical data from 73 adult and adolescent patients with SCD at the Prisma Health – Upstate Comprehensive SCD Program, Greenville, South Carolina, USA, under a data collection protocol for standard-of-care clinical data. Laboratory data were collected before initiation of voxelotor and were compared to the most recent laboratory value collected during treatment. All patients included in the analysis had received voxelotor for at least 2 consecutive weeks. Data from 13 patients were not included in the analysis, 7 patients who received less than 2 weeks of treatment, 3 after reporting adverse events (hypersensitivity reaction [n=2], diarrhea [n=1]), and 6 additional patients who did not receive medications due to insurance issues.

Results
Sixty patients were included in the current analysis. The age range was 12 to 70 years, average (SD) age was 31.1 years (15.36), and 61.7% were female. SCD genotypes represented included HbSS (85%), HbSC (6.7%), and HbSβ0 (6.7%). Mean baseline Hb was 8.2 g/dL. A stable dose of hydroxyurea (HU) was reported in 80.0% of patients at the time of voxelotor initiation and 26.7% of patients received exogenous erythropoietin (EPO) treatment plus HU. At the time of this analysis, patients received voxelotor for an average (range) of 3.9 months (0.5‑13.6 months). Levels of Hb increased (post- vs pre-voxelotor, mean [SD]) by 2.0 g/dL (1.02 g/dL); whereas reticulocyte percentage decreased by 4.6% (3.7%), and total bilirubin decreased by 1.4 mg/dL (2.2 mg/dL). All patients who received exogenous EPO were able to either discontinue (n=11) or reduce the frequency (n=5) of EPO therapy after voxelotor initiation. The clinical improvements reported by patients included increased energy, improved activity tolerance, decreased pain symptoms, and reduced scleral icterus. The most common reported side effects were diarrhea, nausea, and rash. These side effects were mild and self-limited in most patients. Dose modification from 1500 mg to 1000 mg was only necessary in 3 patients with diarrhea.

Conclusion
The effects of voxelotor on Hb, reticulocyte percentage, and total bilirubin were of similar magnitude to values reported in the HOPE trial. In addition, consistent evidence of improved clinical status was associated with a robust hematologic response, including a reduction in EPO therapy. In summary, our study suggests that patients with SCD achieve meaningful clinical benefit with voxelotor therapy.

Keyword(s): Clinical data, Hemoglobin, Patient, Sickle cell disease

Presentation during EHA2021: All e-poster presentations will be made available as of Friday, June 11, 2021 (09:00 CEST) and will be accessible for on-demand viewing until August 15, 2021 on the Virtual Congress platform.

Abstract: EP1209

Type: E-Poster Presentation

Session title: Sickle cell disease

Background
Sickle cell disease (SCD) is an inherited disorder of the red blood cells (RBC) in which sickle hemoglobin (HbS) polymerizes and leads to a detrimental shape change in the RBC. This change, known as sickling, can trigger serious consequences including chronic hemolysis, anemia, and episodic vaso-occlusion and often leads to complications such as cumulative organ damage, disability, and accelerated mortality. Voxelotor is a first-in-class therapy that interferes with sickle cell polymerization, increases hemoglobin (Hb) levels, and reduces hemolysis. It is FDA approved for the treatment of SCD in patients 12 years of age and older.

Aims
The objective of this study was to quantify the clinical response to voxelotor treatment in a single-center series of 73 adult and adolescent patients with SCD.

Methods
We collected real-world clinical data from 73 adult and adolescent patients with SCD at the Prisma Health – Upstate Comprehensive SCD Program, Greenville, South Carolina, USA, under a data collection protocol for standard-of-care clinical data. Laboratory data were collected before initiation of voxelotor and were compared to the most recent laboratory value collected during treatment. All patients included in the analysis had received voxelotor for at least 2 consecutive weeks. Data from 13 patients were not included in the analysis, 7 patients who received less than 2 weeks of treatment, 3 after reporting adverse events (hypersensitivity reaction [n=2], diarrhea [n=1]), and 6 additional patients who did not receive medications due to insurance issues.

Results
Sixty patients were included in the current analysis. The age range was 12 to 70 years, average (SD) age was 31.1 years (15.36), and 61.7% were female. SCD genotypes represented included HbSS (85%), HbSC (6.7%), and HbSβ0 (6.7%). Mean baseline Hb was 8.2 g/dL. A stable dose of hydroxyurea (HU) was reported in 80.0% of patients at the time of voxelotor initiation and 26.7% of patients received exogenous erythropoietin (EPO) treatment plus HU. At the time of this analysis, patients received voxelotor for an average (range) of 3.9 months (0.5‑13.6 months). Levels of Hb increased (post- vs pre-voxelotor, mean [SD]) by 2.0 g/dL (1.02 g/dL); whereas reticulocyte percentage decreased by 4.6% (3.7%), and total bilirubin decreased by 1.4 mg/dL (2.2 mg/dL). All patients who received exogenous EPO were able to either discontinue (n=11) or reduce the frequency (n=5) of EPO therapy after voxelotor initiation. The clinical improvements reported by patients included increased energy, improved activity tolerance, decreased pain symptoms, and reduced scleral icterus. The most common reported side effects were diarrhea, nausea, and rash. These side effects were mild and self-limited in most patients. Dose modification from 1500 mg to 1000 mg was only necessary in 3 patients with diarrhea.

Conclusion
The effects of voxelotor on Hb, reticulocyte percentage, and total bilirubin were of similar magnitude to values reported in the HOPE trial. In addition, consistent evidence of improved clinical status was associated with a robust hematologic response, including a reduction in EPO therapy. In summary, our study suggests that patients with SCD achieve meaningful clinical benefit with voxelotor therapy.

Keyword(s): Clinical data, Hemoglobin, Patient, Sickle cell disease

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