Health Care Transition Experiences of Males with Childhood-onset Duchenne and Becker Muscular Dystrophy: Findings from the Muscular Dystrophy Surveillance Tracking and Research Network (MD STARnet) Health Care Transitions and Other Life Experiences Survey.

Introduction: As the proportion of males with Duchenne muscular dystrophy (DMD) surviving into adulthood increases, more information is needed regarding their health care transition planning, an essential process for adolescents and young adults with DMD. The objective of this study was to describe the health care transition experiences of a population of males living with Duchenne or Becker muscular dystrophy (DBMD). Methods: The eligible participants, identified through the Muscular Dystrophy Surveillance Tracking and Research Network (MD STARnet) surveillance project, were 16-31 years old and lived in Arizona, Colorado, Georgia, Iowa, or western New York (n=258). The MD STARnet Health Care Transitions and Other Life Experiences Survey was conducted in 2013 and administered online or in a telephone interview. Sixty-five males (25%) completed the survey. Among non-ambulatory males, response differences were compared by age group. Statistical comparisons were conducted using Fisher's exact test, or when appropriate, the Chisquare test. Results: Twenty-one percent of non-ambulatory males aged 16-18 years, 28% of non-ambulatory males aged 19-23 years, 25% of non-ambulatory males aged 24-30 years, and 18 ambulatory males had a written transition plan. Nineteen percent of non-ambulatory males aged 24-30 years had delayed or gone without needed health care in the past 12 months. Among non-ambulatory males aged 24-30 years, 75% had cardiology providers and 69% had pulmonology providers involved in their care in the past 12 months. Twentyeight percent of non-ambulatory males aged 19-23 years and 25% of non-ambulatory males aged 24-30 years reported that they did not receive health care or other services at least once because they were unable to leave their home. Non-ambulatory males aged 16-18 years (29%) were less likely to have ever discussed how to obtain or keep health insurance as they get older compared to non-ambulatory males aged 24-30 years (69%) (p <0.01). Discussion: This study identified potential barriers to the successful health care transition of males with DBMD. The results of this study may indicate a lack of targeted informational resources and education focused on supporting the transition of young men with DBMD as they age from adolescence into adulthood within the healthcare system. Future studies could determine the reasons for the potential barriers to health care and identify the optimal transition programs for males with DBMD. There are a few online resources on transition available to adolescents and young adults with special health care needs.

Quantitative Bias Analysis in Regulatory Settings.

Nonrandomized studies are essential in the postmarket activities of the US Food and Drug Administration, which, however, must often act on the basis of imperfect data. Systematic errors can lead to inaccurate inferences, so it is critical to develop analytic methods that quantify uncertainty and bias and ensure that these methods are implemented when needed. "Quantitative bias analysis" is an overarching term for methods that estimate quantitatively the direction, magnitude, and uncertainty associated with systematic errors influencing measures of associations. The Food and Drug Administration sponsored a collaborative project to develop tools to better quantify the uncertainties associated with postmarket surveillance studies used in regulatory decision making. We have described the rationale, progress, and future directions of this project.

Gynecologic oncologists involvement on ovarian cancer standard of care receipt and survival.

AIM: To examine the influence of gynecologic oncologists (GO) in the United States on surgical/chemotherapeutic standard of care (SOC), and how this translates into improved survival among women with ovarian cancer (OC). METHODS: Surveillance, Epidemiology, and End Result (SEER)-Medicare data were used to identify 11688 OC patients (1992-2006). Only Medicare recipients with an initial surgical procedure code (n = 6714) were included. Physician specialty was identified by linking SEER-Medicare to the American Medical Association Masterfile. SOC was defined by a panel of GOs. Multivariate logistic regression was used to determine predictors of receiving surgical/chemotherapeutic SOC and proportional hazards modeling to estimate the effect of SOC treatment and physician specialty on survival. RESULTS: About 34% received surgery from a GO and 25% received the overall SOC. One-third of women had a GO involved sometime during their care. Women receiving surgery from a GO vs non-GO had 2.35 times the odds of receiving the surgical SOC and 1.25 times the odds of receiving chemotherapeutic SOC (P < 0.01). Risk of mortality was greater among women not receiving surgical SOC compared to those who did [hazard ratio = 1.22 (95%CI: 1.12-1.33), P < 0.01], and also was higher among women seen by non-GOs vs GOs (for surgical treatment) after adjusting for covariates. Median survival time was 14 mo longer for women receiving combined SOC. CONCLUSION: A survival advantage associated with receiving surgical SOC and overall treatment by a GO is supported. Persistent survival differences, particularly among those not receiving the SOC, require further investigation.

Anaphylaxis in Schools: Results of the EPIPEN4SCHOOLS Survey Combined Analysis.

A pilot survey described the characteristics of anaphylactic events occurring in an initial set of participating U.S. schools during the 2013-2014 school year. This survey was subsequently readministered to large school districts, which were underrepresented in initial results. A cross-sectional survey was administered to the U.S. schools that were participating in the EPIPEN4SCHOOLS® program (Mylan Specialty L.P., Canonsburg, PA) to assess characteristics of anaphylactic events. Data from large school districts were added to initial findings in this comprehensive combined analysis. A total of 1,140 anaphylactic events were reported among 6,574 responding schools. Of 1,063 anaphylactic events with data on who experienced the event, it was observed that it occurred mostly in students (89.5%, 951/1,063). For students, anaphylactic events were reported across all grades, with 44.9% (400/891) occurring in high school students, 18.9% (168/891) in middle school students, and 32.5% (290/891) in elementary school students. Food was identified as the most common trigger (60.1%, 622/1,035). A majority of schools (55.0%, 3,332/6,053) permitted only the school nurse and select staff to administer epinephrine to treat anaphylaxis. The unpredictability of anaphylaxis is emphasized by its high occurrence in individuals with no known allergies (25.0%). A majority of schools permitted only the school nurse and select staff to treat anaphylaxis. Thus, individuals experiencing an anaphylactic event may frequently encounter staff members not being permitted to administer potentially life-saving epinephrine. Epinephrine auto-injectors provided by the EPIPEN4SCHOOLS program were used to treat 38.0% of events. Anaphylaxis can occur in children with no previously known allergies, illustrating the importance of public access to epinephrine.

The Effect of Gynecologic Oncologist Availability on Ovarian Cancer Mortality.

AIM: To determine the association between the distribution of gynecologic oncologist (GO) and population-based ovarian cancer death rates. MATERIALS AND METHODS: Data on ovarian cancer incidence and mortality in the United States (U.S.) was supplemented with U.S. census data, and analyzed in relation to practicing GOs. GO locations were geocoded to link association between county variables and GO availability. Logistic regression was used to measure areas of high and low ovarian cancer mortality, adjusting for contextual variables. RESULTS: Practicing GOs were unevenly distributed in the United States, with the greatest numbers in metropolitan areas. Ovarian cancer incidence and death rates increased as distance to a practicing GO increased. A relatively small number (153) of counties within 24 miles of a GO had high ovarian cancer death rates compared to 577 counties located 50 or more miles away with high ovarian cancer death rates. Counties located 50 or more miles away from a GO practice had an almost 60% greater odds of high ovarian cancer mortality compared to those with closer practicing GOs (OR 1.59, 95% CI 1.18-2.15). CONCLUSION: The distribution of GOs across the United States appears to be significantly associated with ovarian cancer mortality. Efforts that facilitate outreach of GOs to certain populations may increase geographic access. Future studies examining other factors associated with lack of GO access (e.g. insurance and other socioeconomic factors) at the individual level will assist with further defining barriers to quality ovarian cancer care in the United States.

False positive rate of carbon monoxide saturation by pulse oximetry of emergency department patients.

BACKGROUND: Symptoms of carbon monoxide (CO) poisoning are non-specific. Diagnosis requires suspicion of exposure, confirmed by measuring ambient CO levels or carboxyhemoglobin (COHb). An FDA-approved pulse oximeter (Rad-57) can measure CO saturation (S(pCO)). The device accuracy has implications for clinical decision-making. METHODS: From April 1 to August 15, 2008, study personnel measured S(pCO) and documented demographic factors at time of clinical blood draw, in a convenience sample of 1,363 subjects presenting to the emergency department at Intermountain Medical Center, Murray, Utah. The technician then assayed COHb. COHb and S(pCO) values were compared by subject; false positive or negative values were defined as S(pCO) at least 3 percentage points greater or less than COHb level, reported by the manufacturer to be ± 1 SD in performance. RESULTS: In 1,363 subjects, 613 (45%) were male, 1,141 (84%) were light-skinned, 14 in shock, 4 with CO poisoning, and 122 (9%) met the criteria for a false positive value (range 3-19 percentage points), while 247 (18%) met the criteria for a false negative value (-13 to -3 percentage points). Risks for a false positive S(pCO) reading included being female and having a lower perfusion index. Methemoglobin, body temperature, and blood pressure also appear to influence the S(pCO) accuracy. There was variability among monitors, possibly related to technician technique, as rotation of monitors among technicians was not enforced. CONCLUSIONS: While the Rad-57 pulse oximeter functioned within the manufacturer's specifications, clinicians using the Rad-57 should expect some S(pCO) readings to be significantly higher or lower than COHb measurements, and should not use S(pCO) to direct triage or patient management. An elevated S(pCO) could broaden the diagnosis of CO poisoning in patients with non-specific symptoms. However, a negative S(pCO) level in patients suspected of having CO poisoning should never rule out CO poisoning, and should always be confirmed by COHb.

A matched-cohort evaluation of a bedside asthma intervention for patients hospitalized at a large urban children's hospital.

Emergency care and hospitalizations account for 36% of asthma-related medical expenses for children. National asthma guidelines emphasize the need for asthma self-management education at multiple points of care, including the hospital, to help prevent acute exacerbations. The integration of a bedside asthma education program into discharge planning at a busy urban children's hospital aimed to reduce repeat emergency department (ED) visits and hospitalizations by educating the community's highest-risk children and their families about asthma. A trained respiratory professional provided 45 minutes of individualized bedside education to families at the hospital and one follow-up support phone call within 3 weeks after discharge. Children receiving the intervention were matched to a control group of children not receiving the intervention by age and 2 markers of past utilization using data obtained from hospital records. Repeat ED utilization was analyzed using a Cox proportional hazards model controlling for sex, residence, race or ethnicity, and year. Compared to 698 matched controls, no significant improvement was observed in the 698 intervention participants or any subgroups followed for 12 months after the intervention.

Contextual Analysis of Breast Cancer Stage at Diagnosis Among Women in the United States, 2004.

BACKGROUND: To explore contextual effects and to test for interactions, this study examined how breast cancer stage at diagnosis among U.S. women related to individual- and county-level (contextual) variables associated with access to health care and socioeconomic status. METHODS: Individual-level incidence data were obtained from the National Program of Cancer Registries (NPCR) and the Surveillance, Epidemiology and End-Results (SEER) program. The county of residence of women with diagnosed breast cancer (n = 217,299) was used to link NPCR and SEER data with county-level measures of health care access from the 2004 Area Resource File (ARF). In addition to individual-level covariates such as age, race, and Hispanic ethnicity, we examined county-level covariates (residence in a Health Professional Shortage Area, urban/rural residence; race/ethnicity; and number of health centers/clinics, mammography screening centers, primary care physicians, and obstetrician-gynecologists per 100,000 female population or per 1000 square miles) as predictors of stage of breast cancer at diagnosis. RESULTS: Both individual-level and contextual variables are associated with later stage of breast cancer at diagnosis. Black women and women of "other race" had higher odds of receiving a diagnosis of regional or distant stage breast cancer (P <0.0001 and P = 0.02). With adjustment for age, Hispanics were more likely to receive a diagnosis of later stage breast cancer than non-Hispanics (P <0.0.001). Women living in areas with a higher proportion of black women had greater odds of receiving a diagnosis of regional or late stage breast cancer compared with women living in areas with the lowest proportion of black women. The same was noted for women living in areas with intermediate proportions of Hispanic women (age-adjusted odds ratio [OR], 0.94; 95% confidence interval [CI], 0.92-0.97]. Other important contextual variables associated with stage at diagnosis included the percentage of persons living below the poverty level and the number of office-based physicians per 100,000 women. Women living in counties with a higher proportion of persons living below the poverty level or fewer office-based physicians were more likely to receive a diagnosis of later stage breast cancer than those living in other counties (P < 0.001). In multivariable analysis, residence in areas with a higher proportion of non-Hispanic black women modified the associations of age and Hispanic ethnicity with later stage breast cancer (P = 0.0159 and P = 0.0002, respectively). CONCLUSIONS: This study found that county-level contextual variables related to the availability and accessibility of health care providers and health services can affect the timeliness of breast cancer diagnosis. This information could help public health officials develop interventions to reduce the burden of breast cancer among U.S. women.