Hemoglobin and End-Organ Damage in Individuals with Sickle Cell Disease

ABSTRACT: Background: Sickle cell disease (SCD) is an inherited, chronic, multifaceted blood disorder. Patients with SCD develop anemia, which has been associated with end-organ damage (EOD). Objectives: This retrospective, observational, repeated-measures study systematically characterizes the rel...

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Main Authors: William B. Ershler, MD, Laura M. De Castro, MD, MHSc, Zahra Pakbaz, MD, Aaron Moynahan, MA, Derek Weycker, PhD, Thomas E. Delea, MSIA, Irene Agodoa, MD, Ze Cong, PhD
Format: Article
Language:English
Published: Elsevier 2023-01-01
Series:Current Therapeutic Research
Subjects:
Online Access:http://www.sciencedirect.com/science/article/pii/S0011393X23000061
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author William B. Ershler, MD
Laura M. De Castro, MD, MHSc
Zahra Pakbaz, MD
Aaron Moynahan, MA
Derek Weycker, PhD
Thomas E. Delea, MSIA
Irene Agodoa, MD
Ze Cong, PhD
author_facet William B. Ershler, MD
Laura M. De Castro, MD, MHSc
Zahra Pakbaz, MD
Aaron Moynahan, MA
Derek Weycker, PhD
Thomas E. Delea, MSIA
Irene Agodoa, MD
Ze Cong, PhD
author_sort William B. Ershler, MD
collection DOAJ
description ABSTRACT: Background: Sickle cell disease (SCD) is an inherited, chronic, multifaceted blood disorder. Patients with SCD develop anemia, which has been associated with end-organ damage (EOD). Objectives: This retrospective, observational, repeated-measures study systematically characterizes the relationship between hemoglobin (Hb) level and EOD in adolescent and adult patients with SCD. Methods: The study population comprised patients with SCD aged ≥12 years with available Hb data from a US provider-centric health care database. For each patient, each Hb value over time was included as a separate observation. Study outcomes—the onset of any new EOD, including chronic kidney disease, pulmonary hypertension, stroke, and leg ulcer—were ascertained during the 1-year period after each Hb assessment. The association between Hb levels and risk of new EOD was estimated using multivariable generalized estimating equations. Results: A total of 16,043 unique patients with SCD contributed 44,913 observations. Adjusted odds of any EOD during the 1-year follow-up were significantly lower with higher Hb level. Risk reductions with higher Hb levels for chronic kidney disease, pulmonary hypertension, and leg ulcer were comparable. The risk of new EOD was significantly lower among adolescent and adult patients with higher Hb levels. Conclusions: In patients with SCD, higher Hb levels are associated with a reduced risk of developing EOD. Therapeutic strategies that result in higher Hb levels may offer clinical and economic value for patients with SCD. (Curr Ther Res Clin Exp. 2023; 84:XXX–XXX)
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spelling doaj.art-525589fc430748bca4276d0196e5e4672023-06-21T06:50:44ZengElsevierCurrent Therapeutic Research0011-393X2023-01-0198100696Hemoglobin and End-Organ Damage in Individuals with Sickle Cell DiseaseWilliam B. Ershler, MD0Laura M. De Castro, MD, MHSc1Zahra Pakbaz, MD2Aaron Moynahan, MA3Derek Weycker, PhD4Thomas E. Delea, MSIA5Irene Agodoa, MD6Ze Cong, PhD7Department of Hematology, Inova Schar Cancer Institute, Fairfax, Virginia; Address correspondence to: William B. Ershler, MD, Inova Schar Cancer Institute, 8081 Innovation Park Dr, Suite 4408, Fairfax, VA 22031 (W. Ershler).Department of Medicine, University of Pittsburgh, Pittsburgh, PennsylvaniaDivision of Hematology/Oncology, UC Irvine Chao Family Cancer Center, Orange, CaliforniaPolicy Analysis Inc, Brookline, MassachusettsPolicy Analysis Inc, Brookline, MassachusettsPolicy Analysis Inc, Brookline, MassachusettsGlobal Blood Therapeutics, Inc., South San Francisco, CaliforniaGlobal Blood Therapeutics, Inc., South San Francisco, CaliforniaABSTRACT: Background: Sickle cell disease (SCD) is an inherited, chronic, multifaceted blood disorder. Patients with SCD develop anemia, which has been associated with end-organ damage (EOD). Objectives: This retrospective, observational, repeated-measures study systematically characterizes the relationship between hemoglobin (Hb) level and EOD in adolescent and adult patients with SCD. Methods: The study population comprised patients with SCD aged ≥12 years with available Hb data from a US provider-centric health care database. For each patient, each Hb value over time was included as a separate observation. Study outcomes—the onset of any new EOD, including chronic kidney disease, pulmonary hypertension, stroke, and leg ulcer—were ascertained during the 1-year period after each Hb assessment. The association between Hb levels and risk of new EOD was estimated using multivariable generalized estimating equations. Results: A total of 16,043 unique patients with SCD contributed 44,913 observations. Adjusted odds of any EOD during the 1-year follow-up were significantly lower with higher Hb level. Risk reductions with higher Hb levels for chronic kidney disease, pulmonary hypertension, and leg ulcer were comparable. The risk of new EOD was significantly lower among adolescent and adult patients with higher Hb levels. Conclusions: In patients with SCD, higher Hb levels are associated with a reduced risk of developing EOD. Therapeutic strategies that result in higher Hb levels may offer clinical and economic value for patients with SCD. (Curr Ther Res Clin Exp. 2023; 84:XXX–XXX)http://www.sciencedirect.com/science/article/pii/S0011393X23000061anemiachronic kidney diseaseleg ulcerpulmonary hypertensionsickle cell diseasestroke
spellingShingle William B. Ershler, MD
Laura M. De Castro, MD, MHSc
Zahra Pakbaz, MD
Aaron Moynahan, MA
Derek Weycker, PhD
Thomas E. Delea, MSIA
Irene Agodoa, MD
Ze Cong, PhD
Hemoglobin and End-Organ Damage in Individuals with Sickle Cell Disease
Current Therapeutic Research
anemia
chronic kidney disease
leg ulcer
pulmonary hypertension
sickle cell disease
stroke
title Hemoglobin and End-Organ Damage in Individuals with Sickle Cell Disease
title_full Hemoglobin and End-Organ Damage in Individuals with Sickle Cell Disease
title_fullStr Hemoglobin and End-Organ Damage in Individuals with Sickle Cell Disease
title_full_unstemmed Hemoglobin and End-Organ Damage in Individuals with Sickle Cell Disease
title_short Hemoglobin and End-Organ Damage in Individuals with Sickle Cell Disease
title_sort hemoglobin and end organ damage in individuals with sickle cell disease
topic anemia
chronic kidney disease
leg ulcer
pulmonary hypertension
sickle cell disease
stroke
url http://www.sciencedirect.com/science/article/pii/S0011393X23000061
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