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      Treatment patterns and burden of complications associated with sickle cell disease: A US retrospective claims analysis

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          Abstract

          Complications associated with sickle cell disease (SCD) that are highly impactful for patients but until recently have been less understood include priapism, nephropathy, and neurologic injury. We conducted a retrospective study using US administrative claims data from July 01, 2013 through March 31, 2020 to analyze incidence of these complications, SCD treatment patterns, and healthcare resource utilization (HCRU) and costs among 2524 pediatric and adult patients with SCD (mean [SD] age 43.4 [22.4] years). The most common treatments during follow‐up were short‐acting opioids (54.0% of patients), red blood cell transfusion (15.9%), and hydroxyurea (11.0%). SCD complications occurred frequently; in the overall population, the highest follow‐up incidences per 1000 person‐years were for acute kidney injury (53.1), chronic kidney disease (40.6), and stroke (39.0). Complications occurred across all age groups but increased in frequency with age; notably, acute kidney injury was 69.7 times more frequent among ages 65+ than ages 0–15 ( p < 0.001). Follow‐up per‐patient‐per‐month HCRU also increased with age; however, all‐cause healthcare costs were similarly high for all age groups and were driven primarily by inpatient stays. Patients with SCD across the age spectrum have a high burden of complications with the use of current treatments, suggesting unmet needs for treatment management.

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          Most cited references53

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          Updating and validating the Charlson comorbidity index and score for risk adjustment in hospital discharge abstracts using data from 6 countries.

          With advances in the effectiveness of treatment and disease management, the contribution of chronic comorbid diseases (comorbidities) found within the Charlson comorbidity index to mortality is likely to have changed since development of the index in 1984. The authors reevaluated the Charlson index and reassigned weights to each condition by identifying and following patients to observe mortality within 1 year after hospital discharge. They applied the updated index and weights to hospital discharge data from 6 countries and tested for their ability to predict in-hospital mortality. Compared with the original Charlson weights, weights generated from the Calgary, Alberta, Canada, data (2004) were 0 for 5 comorbidities, decreased for 3 comorbidities, increased for 4 comorbidities, and did not change for 5 comorbidities. The C statistics for discriminating in-hospital mortality between the new score generated from the 12 comorbidities and the Charlson score were 0.825 (new) and 0.808 (old), respectively, in Australian data (2008), 0.828 and 0.825 in Canadian data (2008), 0.878 and 0.882 in French data (2004), 0.727 and 0.723 in Japanese data (2008), 0.831 and 0.836 in New Zealand data (2008), and 0.869 and 0.876 in Swiss data (2008). The updated index of 12 comorbidities showed good-to-excellent discrimination in predicting in-hospital mortality in data from 6 countries and may be more appropriate for use with more recent administrative data. © The Author 2011. Published by Oxford University Press on behalf of the Johns Hopkins Bloomberg School of Public Health. All rights reserved.
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            Sickle cell disease

            Sickle cell disease (SCD) is a group of inherited disorders caused by mutations in HBB, which encodes haemoglobin subunit β. The incidence is estimated to be between 300,000 and 400,000 neonates globally each year, the majority in sub-Saharan Africa. Haemoglobin molecules that include mutant sickle β-globin subunits can polymerize; erythrocytes that contain mostly haemoglobin polymers assume a sickled form and are prone to haemolysis. Other pathophysiological mechanisms that contribute to the SCD phenotype are vaso-occlusion and activation of the immune system. SCD is characterized by a remarkable phenotypic complexity. Common acute complications are acute pain events, acute chest syndrome and stroke; chronic complications (including chronic kidney disease) can damage all organs. Hydroxycarbamide, blood transfusions and haematopoietic stem cell transplantation can reduce the severity of the disease. Early diagnosis is crucial to improve survival, and universal newborn screening programmes have been implemented in some countries but are challenging in low-income, high-burden settings.
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              Sickle Cell Disease

              New England Journal of Medicine, 376(16), 1561-1573
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                Author and article information

                Contributors
                amy.j.anderson@optum.com
                Journal
                EJHaem
                EJHaem
                10.1002/(ISSN)2688-6146
                JHA2
                EJHaem
                John Wiley and Sons Inc. (Hoboken )
                2688-6146
                06 October 2022
                November 2022
                : 3
                : 4 ( doiID: 10.1002/jha2.v3.4 )
                : 1135-1144
                Affiliations
                [ 1 ] Albert Einstein College of Medicine The Children's Hospital at Montefiore The Bronx New York USA
                [ 2 ] Department of Urology The Johns Hopkins University School of Medicine Baltimore Maryland USA
                [ 3 ] Novartis Pharmaceuticals Corporation East Hanover New Jersey USA
                [ 4 ] Optum Life Sciences Eden Prairie Minnesota USA
                [ 5 ] Sickle Cell Center Division of Hematology and Oncology University of Illinois Hospital and Health Sciences System Chicago Illinois USA
                Author notes
                [*] [* ] Correspondence

                Amy Anderson, Optum Life Sciences, 11000 Optum Circle, Eden Prairie, MN 55344, USA.

                Email: amy.j.anderson@ 123456optum.com

                Author information
                https://orcid.org/0000-0002-8584-4194
                Article
                JHA2575
                10.1002/jha2.575
                9713207
                36467832
                31503a28-f837-4240-81eb-c9c33dd0de88
                © 2022 The Authors. eJHaem published by British Society for Haematology and John Wiley & Sons Ltd.

                This is an open access article under the terms of the http://creativecommons.org/licenses/by/4.0/ License, which permits use, distribution and reproduction in any medium, provided the original work is properly cited.

                History
                : 02 September 2022
                : 15 August 2022
                : 04 September 2022
                Page count
                Figures: 3, Tables: 5, Pages: 10, Words: 6462
                Categories
                Research Article
                Sickle Cell, Thrombosis, and Benign Haematology
                Research Articles
                Custom metadata
                2.0
                November 2022
                Converter:WILEY_ML3GV2_TO_JATSPMC version:6.2.1 mode:remove_FC converted:01.12.2022

                healthcare costs,opioid use,organ dysfunction,retrospective studies,sickle cell disease,united states

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