Suzette O. Oyeku

Sickle Cell Disease: A Question of Equity and Quality

Thirty years ago, the first major federal legislation concerning sickle cell disease treatment was passed, resulting in the development of comprehensive sickle cell centers. We are now at another watershed moment in the treatment of this illness with the passage in October 2004 of the Sickle Cell Treatment Act, designed to substantially expand specialized sickle cell treatment programs. This legislation offers a remarkable opportunity to significantly improve health outcomes for individuals with sickle cell disease if it is implemented with a specific focus on the distinct but related issues of equity and quality. Despite major advances in sickle cell disease treatment that have occurred over the past 3 decades, important gaps exist both in the equity of government and private philanthropic support for research and in the uniform provision of high quality clinical care. This article assesses the current gaps in funding support and in the implementation of improvements in clinical care in order to suggest strategies for making optimal use of the opportunity that the new legislation presents to improve the health of all individuals affected by this disease.

Administrative data sets and health services research on hemoglobinopathies: a review of the literature.

CONTEXT Large administrative healthcare data sets are an important source of data for health services research on sickle cell disease (SCD) and thalassemia. This paper identifies and describes major U.S. healthcare administrative databases and their use in published health services research on hemoglobinopathies. EVIDENCE ACQUISITION Publications that used U.S. administrative healthcare data sets to assess healthcare use or expenditures were identified through PubMed searches using key words for SCD and either costs, expenditures, or hospital discharges; no additional articles were identified by using thalassemia as a key word. Additional articles were identified through manual searches of related articles or reference lists. EVIDENCE SYNTHESIS A total of 26 original health services research articles were identified. The types of administrative data used for health services research on hemoglobinopathies included federal- and state-specific hospital discharge data sets and public and private health insurance claims databases. Gaps in recent health services research on hemoglobin disorders included a paucity of research related to thalassemia, few studies of adults with hemoglobinopathies, and few studies focusing on emergency department or outpatient clinic use. CONCLUSIONS Administrative data sets provide a unique means to study healthcare use among people with SCD or thalassemia because of the ability to examine large sample sizes at fairly low cost, resulting in greater generalizability than is the case with clinic-based data. Limitations of administrative data in general include potential misclassification, under-reporting, and lack of sociodemographic information.

Parental and other factors associated with hydroxyurea use for pediatric sickle cell disease

Hydroxyurea (HU) is highly effective treatment for sickle cell disease (SCD). While pediatric use of HU is accepted clinical practice, barriers to use may impede its potential benefit.

Increased prevalence of osteonecrosis of the femoral head in children and adolescents with sickle-cell disease.

anemia. Blood 2010;115:5300–5311. 10. Thornburg CD, Calatroni A, Telen M, Kemper AR. Adherence to hydroxyurea therapy in children with sickle cell anemia. J Pediatr 2010;156:415–419. 11. Sichle Cell Disease Guidelines-Hydroxyurea Chapter. http://rover.nhlbi.nih. gov/guidelines/scd/about.htm. Accessed November 21, 2011. 12. de Montalembert M, Brousse V, Elie C, et al. Long-term hydroxyurea treatment in children with sickle cell disease: Tolerance and clinical outcomes. Haematologica 2006;91:125–128. 13. Zimmerman SA, Schultz WH, Davis JS, et al. Sustained long-term hematologic efficacy of hydroxyurea at maximum tolerated dose in children with sickle cell disease. Blood 2004;103:2039–2045. 14. Burgess SW SP, Morawska A, Devadason SG. Assessing adherence and factors associated with adherence in young children with asthma. Respirology 2008;13:559–563. 15. Jentzsch NS, Camargos PA, Colosimo EA, Bousquet J. Monitoring adherence to beclomethasone in asthmatic children and adolescents through four different methods. Allergy 2009;64:1458–1462. 16. Thornburg CD, Calatroni A, Telen M, Kemper AR. Adherence to hydroxyurea therapy in children with sickle cell anemia. J Pediatr 2010;156:415– 419. 17. Osterberg L, Blaschke T. Adherence to medication. N Engl J Med 2005;353: 487–497. 18. Franklin VL, Waller A, Pagliari C, Greene SA. A randomized controlled trial of Sweet Talk, a text-messaging system to support young people with diabetes. Diabet Med 2006;23:1332–1338. 19. Lee SJ, Joffe S, Kim HT, et al. Physicians’ attitudes about quality-of-life issues in hematopoietic stem cell transplantation. Blood 2004;104:2194– 2200. 20. Wong EC, Perez-Albuerne E, Moscow JA, Luban NL. Transfusion management strategies: A survey of practicing pediatric hematology/oncology specialists. Pediatr Blood Cancer 2005;44:119–127. 21. Streiff MB, Smith B, Spivak JL. The diagnosis and management of polycythemia vera in the era since the Polycythemia Vera Study Group: A survey of American Society of Hematology members’ practice patterns. Blood 2002;99: 1144–1149.

Hepatic Iron Quantification on 3 Tesla (3 T) Magnetic Resonance (MR): Technical Challenges and Solutions

MR has become a reliable and noninvasive method of hepatic iron quantification. Currently, most of the hepatic iron quantification is performed on 1.5 T MR, and the biopsy measurements have been paired with R 2 and R 2* values for 1.5 T MR. As the use of 3 T MR scanners is steadily increasing in clinical practice, it has become important to evaluate the practicality of calculating iron burden at 3 T MR. Hepatic iron quantification on 3 T MR requires a better understanding of the process and more stringent technical considerations. The purpose of this work is to focus on the technical challenges in establishing a relationship between T 2* values at 1.5 T MR and 3 T MR for hepatic iron concentration (HIC) and to develop an appropriately optimized MR protocol for the evaluation of T 2* values in the liver at 3 T magnetic field strength. We studied 22 sickle cell patients using multiecho fast gradient-echo sequence (MFGRE) 3 T MR and compared the results with serum ferritin and liver biopsy results. Our study showed that the quantification of hepatic iron on 3 T MRI in sickle cell disease patients correlates well with clinical blood test results and biopsy results. 3 T MR liver iron quantification based on MFGRE can be used for hepatic iron quantification in transfused patients.

Sickle cell disease incidence among newborns in New York State by maternal race/ethnicity and nativity

Purpose:Sickle cell disease is estimated to occur in 1:300–400 African-American births, with higher rates among immigrants from Africa and the Caribbean, and is less common among Hispanic births. This study determined sickle cell disease incidence among New York State newborns stratified by maternal race/ethnicity and nativity.Methods:Newborns with confirmed sickle cell disease born to New York State residents were identified by the New York State newborn screening program for the years 2000–2008 and matched to birth records to obtain birth and maternal information. Annual incidence rates were computed and bivariate analyses were conducted to examine associations with maternal race/ethnicity and nativity.Results:From 2000 to 2008, 1,911 New York State newborns were diagnosed with sickle cell disease and matched to the birth certificate files. One in every 1,146 live births was diagnosed with sickle cell disease. Newborns of non-Hispanic black mothers accounted for 86% of sickle cell disease cases whereas newborns of Hispanic mothers accounted for 12% of cases. The estimated incidence was 1:230 live births for non-Hispanic black mothers, 1:2,320 births for Hispanic mothers, and 1:41,647 births for non-Hispanic white mothers. Newborns of foreign-born non-Hispanic black mothers had a twofold higher incidence of sickle cell disease than those born to US-born non-Hispanic black mothers (P < 0.001).Conclusion:This study provides the first US estimates of sickle cell disease incidence by maternal nativity. Women born outside the United States account for the majority of children with sickle cell disease born in New York State. Such findings identify at-risk populations and inform outreach activities that promote ongoing, high-quality medical management to affected children.Genet Med 2013:15(3):222–228

Hemoglobinopathy learning collaborative: Using quality improvement (QI) to achieve equity in health care quality, coordination, and outcomes for sickle cell disease

Care and outcomes for individuals living with sickle cell disease (SCD) vary across institutions and communities. The Hemoglobinopathy Learning Collaborative (HLC) seeks to improve outcomes across the life course through improvement science. Faculty identified five key drivers of improved outcomes: a strong community network; knowledgeable, proactive individuals, families and providers; reliable identification and follow-up; seamless co-management between primary and specialty care; and appropriate treatment for acute episodes. Using a modified Delphi process, we selected improvement measures aligned with the drivers. Data are collected via a Web-based system linked to a reporting portal. Participating teams include consumers, community organizations and primary and specialty care providers. This commentary reviews the context of SCD in the U.S.; describes the framework, measures, and technology infrastructure already created for the HLC; reports on the early experience of teams; highlights the initiative’s challenges and opportunities; and reflects on its implications in the setting of health reform.

Trends in blood transfusion among hospitalized children with sickle cell disease

Blood transfusions represent a major therapeutic option in acute management of sickle cell disease (SCD). Few data exist documenting trends in transfusion among children with SCD, particularly during hospitalization.

Sickle cell disease pain management and the medical home

Pain is the most common cause for hospitalization and acute morbidity in sickle cell disease (SCD). The consequences of SCD-related pain are substantial, affecting both the individual and the health care system. The emergence of the patient-centered medical home (PCMH) provides new opportunities to align efforts to improve SCD management with innovative and potentially cost-effective models of patient-centered care. The Department of Health and Human Services has designated SCD as a priority area with emphasis on creating PCMHs for affected patients. The question for patients, clinicians, scientists, and policy-makers is how the PCMH can be designed to address pain, the hallmark feature of SCD. This article provides a framework of pain management within the PCMH model. We present an overview of pain and pain management in SCD, gaps in pain management, and current care models used by patients and discuss core PCMH concepts and multidisciplinary team-based PCMH care strategies for SCD pain management.

Mortality of New York children with sickle cell disease identified through newborn screening

Purpose:Long-term follow-up of newborn screening for conditions such as sickle cell disease can be conducted using linkages to population-based data. We sought to estimate childhood sickle cell disease mortality and risk factors among a statewide birth cohort with sickle cell disease identified through newborn screening.Methods:Children with sickle cell disease identified by newborn screening and born to New York residents in 2000–2008 were matched to birth and death certificates. Mortality rates were calculated (using numbers of deaths and observed person-years at risk) and compared with mortality rates for all New York children by maternal race/ethnicity. Stratified analyses were conducted to examine associations between selected factors and mortality.Results:Among 1,911 infants with sickle cell disease matched to birth certificates, 21 deaths were identified. All-cause mortality following diagnosis was 3.8 per 1,000 person-years in the first 2 years of life and 1.0 per 1,000 person-years at ages 2–9 years. The mortality rate was significantly lower among children of foreign-born mothers and was significantly higher among preterm infants with low birth weight. The mortality rates were not significantly higher for infants after 28 days with sickle cell disease than for all New York births, but they were 2.7–8.4 times higher for children 1 through 9 years old with homozygous sickle cell disease than for those of all non-Hispanic black or Hispanic children born to New York residents.Conclusion:Estimated mortality risk in children with homozygous sickle cell disease remains elevated even after adjustment for maternal race/ethnicity. These results provide evidence regarding the current burden of child mortality among children with sickle cell disease despite newborn screening.Genet Med 17 6, 452–459.