Haemophilia. 2020;26:487-493.wileyonlinelibrary.com/journal/hae | 487 | INTRODUC TI ONHaemophilia is an X-linked, inherited disorder that results from mutations in the genes that code for one of two proteins necessary for normal blood clotting, factor VIII (haemophilia A) or factor IX (haemophilia B). Haemophilia A (HA) is more common than haemophilia B (HB); however, both disorders primarily affect males and result in bleeding in organs, tissues and joints in response to trauma and Introduction: Estimates of the size and characteristics of the US haemophilia population are needed for healthcare planning and resource needs assessment. A network of comprehensive haemophilia treatment centres (HTCs) located throughout the United States receives federal support for diagnosis and management of haemophilia and other rare bleeding disorders.Aim: Estimate the incidence and prevalence of haemophilia among US males using the HTC network. Methods:During the period 2012-2018, de-identified surveillance data were collected on all males who visited an HTC that included year of birth, gender, race, Hispanic ethnicity, residence zip code, haemophilia type and severity. Data from all patients were used to calculate period prevalence by haemophilia type, severity and state of residence. Data from a subset of patients born 1995-2014 were used to estimate incidence rates over the 20-year period.

There are limited observational studies among children diagnosed with von Willebrand Disease (VWD). We analyzed differences in bleeding characteristics by sex and type of VWD using the largest reported surveillance database of children with VWD (n = 2712), ages 2 to 12 years old. We found that the mean ages of first bleed and diagnosis were lowest among children with type 3 VWD. It was even lower among boys than girls among all VWD types, with statistically significant difference among children with type 1 or type 3 VWD. Children with type 3 VWD also reported higher proportions of ever having a bleed compared to other VWD types, with statistically higher proportions of boys compared to girls reporting ever having a bleed with type 1 and type 2 VWD. A similar pattern was observed with the use of treatment product, showing higher usage among type 3 VWD, and among boys than girls with type 1 and type 2 VWD. While there were no differences in life quality or in well-being status by sex, children with type 3 VWD showed a greater need for mobility assistance compared to children with type 1 and type 2 VWD. In an adjusted analysis among children with type 1 VWD, boys showed a significant association of ever bleeding [hazard ratio 1.4; P-value <.001)] compared to girls. Understanding phenotypic bleeding characteristics, well-being status, treatment, and higher risk groups for bleeding among pre-adolescent children with VWD will aid physicians in efforts to educate families about bleeding symptoms.

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Problem/Condition Hemophilia is an X-linked genetic disorder that primarily affects males and results in deficiencies in blood-clotting proteins. Hemophilia A is a deficiency in factor VIII, and hemophilia B is a deficiency in factor IX. Approximately one in 5,000 males are born with hemophilia, and hemophilia A is about four times as common as hemophilia B. Both disorders are characterized by spontaneous internal bleeding and excessive bleeding after injuries or surgery. Hemophilia can lead to repeated bleeding into the joints and associated chronic joint disease, neurologic damage, damage to other organ systems, and death. Although no precise national U.S. prevalence estimates for hemophilia exist because of the difficulty identifying cases among persons who receive care from various types of health care providers, two previous state-based studies estimated hemophilia prevalence at 13.4 and 19.4 per 100,000 males. In addition, these studies showed that 67% and 82% of persons with hemophilia received care in a federally funded hemophilia treatment center (HTC), and 86% and 94% of those with the most severe cases of hemophilia (i.e., those with the lowest levels of clotting factor activity in the circulating blood) received care in a federally funded HTC. As of January 2020, the United States had 144 HTCs. Period Covered 1998–2019. Description of the System Surveillance for hemophilia, which is a complex, chronic condition, is challenging because of its low prevalence, the difficulty in ascertaining cases uniformly, and the challenges in routinely characterizing and tracking associated health complications. Over time, two systems involving many stakeholders have been used to conduct ongoing hemophilia surveillance. During 1998–2011, CDC and the HTCs collaborated to establish the Universal Data Collection (UDC) surveillance system. The purposes of the UDC surveillance system were to monitor human immunodeficiency virus (HIV) and bloodborne viral hepatitis in persons with hemophilia, thereby tracking blood safety, and to track the prevalence of and trends in complications associated with hemophilia. HTC staff collected clinical data and blood specimens from UDC participants and submitted them to CDC. CDC tested specimens for viral hepatitis and HIV. In 2011, the UDC surveillance system was replaced by a new hemophilia surveillance system called Community Counts. CDC and the HTCs established Community Counts to expand laboratory testing and the collection of clinical data to better identify and track emerging health issues in persons with hemophilia. Results This report is the first comprehensive summary of CDC’s hemophilia surveillance program, which comprises both UDC and Community Counts. Data generated from these surveillance systems have been used in the development of public health and clinical guidelines and practices to improve the safety of U.S. blood products and either prevent hemop...

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Objectives Federal open-data initiatives that promote increased sharing of federally collected data are important for transparency, data quality, trust, and relationships with the public and state, tribal, local, and territorial partners. These initiatives advance understanding of health conditions and diseases by providing data to researchers, scientists, and policymakers for analysis, collaboration, and use outside the Centers for Disease Control and Prevention (CDC), particularly for emerging conditions such as COVID-19, for which data needs are constantly evolving. Since the beginning of the pandemic, CDC has collected person-level, de-identified data from jurisdictions and currently has more than 8 million records. We describe how CDC designed and produces 2 de-identified public datasets from these collected data. Methods We included data elements based on usefulness, public request, and privacy implications; we suppressed some field values to reduce the risk of re-identification and exposure of confidential information. We created datasets and verified them for privacy and confidentiality by using data management platform analytic tools and R scripts. Results Unrestricted data are available to the public through Data.CDC.gov, and restricted data, with additional fields, are available with a data-use agreement through a private repository on GitHub.com. Practice Implications Enriched understanding of the available public data, the methods used to create these data, and the algorithms used to protect the privacy of de-identified people allow for improved data use. Automating data-generation procedures improves the volume and timeliness of sharing data.

Introduction: Sickle cell disease (SCD), a group of inherited blood cell disorders that primarily affects Black or African American persons, is associated with severe complications and a >20-year reduction in life expectancy. In 2014, an expert panel convened by the National Heart, Lung, and Blood Institute issued recommendations to prevent or reduce complications in children and adolescents with the most severe SCD subtypes, known as sickle cell anemia (SCA); recommendations included 1) annual screening of children and adolescents aged 2-16 years with transcranial Doppler (TCD) ultrasound to identify those at risk for stroke and 2) offering hydroxyurea therapy to children and adolescents aged ≥9 months to reduce the risk for several life-threatening complications. Methods: Data from the IBM MarketScan Multi-State Medicaid Database were analyzed. TCD screening and hydroxyurea use were examined for 3,352 children and adolescents with SCA aged 2-16 years and continuously enrolled in Medicaid during 2019. Percentage change during 2014-2019 and variation by health subgroups were assessed. Analyses were stratified by age. Results: During 2014-2019, TCD screening increased 27% among children and adolescents aged 10-16 years; hydroxyurea use increased 27% among children aged 2-9 years and 23% among children and adolescents aged 10-16 years. However, in 2019, only 47% and 38% of children and adolescents aged 2-9 and 10-16 years, respectively, had received TCD screening and 38% and 53% of children and adolescents aged 2-9 years and 10-16 years, respectively, used hydroxyurea. For both prevention strategies, usage was highest among children and adolescents with high levels of health care utilization and evidence of previous complications indicative of severe disease. Conclusion and Implications for Public Health Practice: Despite increases since 2014, TCD screening and hydroxyurea use remain low among children and adolescents with SCA. Health care providers should implement quality care strategies within their clinics and partner with patients, families, and community-based organizations to address barriers to delivering and receiving recommended care.* A more comprehensive summary of the genetics, epidemiology, and health outcomes associated with SCD, the health care needs of persons affected by SCD, and their challenges to receipt of optimal care can be found at National Academies of Sciences, Engineering, and Medicine. 2020. Addressing sickle cell disease: a strategic plan and blueprint for action. Washington, DC: The National Academies Press.

People with sickle cell disease (SCD) suffer from numerous acute complications that can result in multiple hospitalizations and emergency department (ED) and outpatient care visits. Priapism, a prolonged unwanted erection of the penis not due to sexual stimulation, is a serious complication among males with SCD. Variations in estimates of prevalence make it difficult to accurately assess the burden of this complication of SCD. We analyzed data from the Nationwide Emergency Department Sample (NEDS), a product of the Healthcare Cost and Utilization Project, for the years 2006 through 2010 to measure the numbers of ED visits and to examine patterns of subsequent hospitalizations associated with priapism among male patients with SCD. We find that among ED visits associated with males with SCD, those prompted by priapism are more likely to result in hospitalization than are those associated with pain.