Philippa G. Sprinz

Sickle Cell Disease–Related Pediatric Medical Expenditures in the U.S.

BACKGROUND Although it is known that people with sickle cell disease (SCD) have relatively high utilization of medical care, most previous estimates of SCD-attributable expenditures have been limited to either inpatient care or single-state data. PURPOSE To extend known findings by measuring the attributable or incremental expenditures per child with SCD compared to children without this illness and to thereby estimate SCD-attributable expenditures among children in the U.S. METHODS MarketScan Medicaid and Commercial Claims databases for 2005 were used to estimate total medical expenditures of children with and without SCD. Expenditures attributable to SCD were calculated as the difference in age-adjusted mean expenditures during 2005 for children with SCD relative to children without SCD in the two databases. RESULTS Children with SCD incurred medical expenditures that were

Quality-of-Care Indicators for Children With Sickle Cell Disease

9369 and

Health care utilization and expenditures for privately and publicly insured children with sickle cell disease in the United States.

13,469 higher than those of children without SCD enrolled in Medicaid and private insurance, respectively. In other words, expenditures of children with SCD were 6 and 11 times those of children without SCD enrolled in Medicaid and private insurance, respectively. CONCLUSIONS Using a large, multistate, multipayer patient sample, SCD-attributable medical expenditures in children were conservatively and approximately estimated at

Cerebral metastasis and other central nervous system complications of pleuropulmonary blastoma

335 million in 2005.

Communication of Positive Newborn Screening Results for Sickle Cell Disease and Sickle Cell Trait: Variation Across States

OBJECTIVE: To develop a set of quality-of-care indicators for the management of children with sickle cell disease (SCD) who are cared for in a variety of settings by addressing the broad spectrum of complications relevant to their illness. METHODS: We used the Rand/University of California Los Angeles appropriateness method, a modified Delphi method, to develop the indicators. The process included a comprehensive literature review with ratings of the evidence and 2 rounds of anonymous ratings by an expert panel (nominated by leaders of various US academic societies and the National Heart, Lung, and Blood Institute). The panelists met face-to-face to discuss each indicator in between the 2 rounds. RESULTS: The panel recommended 41 indicators that cover 18 topics; 17 indicators described routine health care maintenance, 15 described acute or subacute care, and 9 described chronic care. The panel identified 8 indicators most likely to have a large positive effect on improving quality of life and/or health outcomes for children with SCD, which covered 6 topics: timely assessment and treatment of pain and fever; comprehensive planning; penicillin prophylaxis; transfusion; and the transition to adult care. CONCLUSIONS: Children with SCD are at risk for serious morbidities and early mortality, yet efforts to assess and improve the quality of their care have been limited compared with other chronic childhood conditions. This set of 41 indicators can be used to assess quality of care and provide a starting point for quality-improvement efforts.

Management of Children With Sickle Cell Disease: A Comprehensive Review of the Literature

There are no current national estimates on health care utilization and expenditures for US children with sickle cell disease (SCD).

Transition from pediatric to adult care for sickle cell disease: Results of a survey of pediatric providers†

Pleuropulmonary blastoma (PPB) is a rare tumor of pleura and lung in young children. Central nervous system (CNS) complications, particularly cerebral parenchymal metastases, occur in aggressive forms of PPB: Types II and III PPB. This article evaluates cerebral and meningeal metastases, cerebrovascular events (CVA) caused by tumor emboli, spinal cord complications, and intracranial second malignancies in PPB.

Improving the Management of Vaso-Occlusive Episodes in the Pediatric Emergency Department.

In the US, all states and the District of Columbia have universal newborn screening (NBS) programs for sickle cell disease (SCD), which also identify sickle cell trait (trait). In this project, we surveyed follow‐up coordinators, including one in the District of Columbia and two in Georgia, about protocols for stakeholder notification for SCD and trait. The primary outcomes were total number and type of stakeholder informed of a positive screen. We received 52 completed surveys (100% response). Primary care providers (PCPs) (100%), hematologists (81%), hospitals (73%), and families (40%) were the most commonly notified stakeholders of positive SCD screens, while PCPs (88%), hospitals (63%), and families (37%) were most commonly notified for trait. On average, 3.4 stakeholders were notified for a positive screening for SCD, compared to 2.4 stakeholders for sickle cell trait (P < 0.001). In multivariate analyses for SCD, we found a 2.9% increase in stakeholders notified for each additional year of universal screening mandated in a state (95% CI: 1.4–4.4%). For trait, we found an 8.5% increase in stakeholders notified for each additional follow‐up staff (95% CI: 1.3–15.7%), and a 1.3% increase for each additional percent of black births in the state (95% CI: 0.1–2.5%). Wide variation exists in stakeholder notification by NBS programs of positive screenings for SCD and trait. This variation may alter the effectiveness of NBS programs by location of birth.

Pregnancy in bone marrow failure syndromes: Diamond-Blackfan anaemia and Shwachman-Diamond syndrome.

OBJECTIVE: Sickle cell disease (SCD) affects 70 000 to 100 000 people in the United States, and 2000 infants are born with the disease each year. The purpose of this study was to review the quality of the literature for preventive interventions and treatment of complications for children with SCD to facilitate the use of evidence-based medicine in clinical practice and identify areas in need of additional research. METHODS: We searched the Ovid Medline database and the Cochrane Library for articles published between January 1995 and April 2010 for English-language abstracts on 28 topics thought to be important for the care of children with SCD. We also added pertinent references cited by studies identified in our search. Each abstract was reviewed independently by 2 authors. Data from articles retrieved for full review were abstracted by using a common form. RESULTS: There were 3188 abstracts screened, and 321 articles underwent full review. Twenty-six articles (<1% of abstracts initially screened), which consisted of 25 randomized controlled trials and 1 meta-analysis, were rated as having level I evidence. Eighteen of the 28 topics selected for this review did not have level I evidence studies published. The management and prevention of pain episodes accounted for more than one-third of the level I studies. CONCLUSIONS: Although significant strides have been made in the care of children with SCD in the past 2 decades, more research needs to be performed, especially for acute events associated with SCD, to ensure that the health and well-being of children with SCD continues to improve.

Zinc Protoporphyrin and Iron Deficiency Screening: Trends and Therapeutic Response in an Urban Pediatric Center

As recently as the 1970s, children born with sickle cell disease (SCD) were unlikely to survive into adulthood. With advances in medical care, most patients now survive childhood and live into their forties or beyond [1,2]. A better transition from pediatric- to adult-focused care is therefore increasingly important [3]. Despite recent awareness of the importance of transition by the SCD community, little is known about existing transition programs. We conducted a survey of pediatric sickle cell clinics to describe current transition practices and identify areas for improvement. Survey topics included program demographics, transition logistics, assessment of patient transition readiness and independence, transition preparation, and program evaluation. Twenty-three clinics (77%) report having a transition program, although half have been in place for under 2 years. There is wide variation in specific transition practices. Most centers (97%) have an identified accepting adult provider, however, only 60% routinely transfer their patients to an adult hematologist specializing in SCD. Although there has been a recent effort to establish transition programs in pediatric sickle cell clinics, specific practices vary widely. As anticipated, lack of an accepting adult hematologist with an interest in SCD emerged as a common barrier to transition.