Sunkyung Kim

Corticosteroid Treatments in Males With Duchenne Muscular Dystrophy: Treatment Duration and Time to Loss of Ambulation.

This population-based study examines the association between corticosteroid treatment and time to loss of ambulation, stratifying by treatment duration (short: 0.25-3 years, long: >3 years), among 477 Duchenne muscular dystrophy cases identified by the Muscular Dystrophy Surveillance Tracking and Research Network (MDSTARnet). Those cases who received short-term corticosteroid treatment had a time to loss of ambulation that was 0.8 years shorter (t test) and an annual risk of losing ambulation 77% higher than the untreated (Cox regression). Conversely, cases who received long-term corticosteroid treatment had a time to loss of ambulation that was 2 years longer and an annual risk of losing ambulation 82% lower than the untreated, up to age 11 years; after which the risks were not statistically different. The relationship of corticosteroids and time to loss of ambulation is more complex than depicted by previous studies limited to treatment responders or subjects who lost ambulation during study follow-up.

Associations between timing of corticosteroid treatment initiation and clinical outcomes in Duchenne muscular dystrophy

The long-term efficacy of corticosteroid treatment and timing of treatment initiation among Duchenne muscular dystrophy (DMD) patients is not well-understood. We used data from a longitudinal, population-based DMD surveillance program to examine associations between timing of treatment initiation (early childhood [before or at age 5 years], late childhood [after age 5 years], and naïve [not treated]) and five clinical outcomes (age at loss of ambulation; ages at onset of cardiomyopathy, scoliosis, and first fracture; and pulmonary function). Hazard ratios (HRs) and 95% confidence intervals (CIs) were estimated using survival analysis. DMD patients who initiated corticosteroid treatment in early childhood had a higher risk of earlier onset cardiomyopathy compared to cases who initiated treatment in late childhood (HR = 2.0, 95% CI = [1.2, 3.4]) or treatment naïve patients (HR = 1.9, 95% CI = [1.1, 3.2]), and higher risk of suffering a fracture (HR = 2.3, 95% CI = [1.4, 3.7] and HR = 2.6, 95% CI = [1.6, 4.2], respectively). Patients with early childhood treatment had slightly decreased respiratory function compared with those with late childhood treatment. Ages at loss of ambulation or scoliosis diagnosis did not differ statistically among treatment groups. We caution that the results from our study are subject to several limitations, as they were based on data abstracted from medical records. Further investigations using improved reporting of disease onset and outcomes are warranted to obtain a more definitive assessment of the association between the timing of corticosteroid treatment and disease severity.

Metabolic risk factors in U.S. youth with low relative muscle mass.

AIMS To examine the association between relative muscle mass (RMM) and nine risk factors for cardiovascular disease and diabetes (CVD/DM) in U.S. youth. METHODS We used a sample representative of the U.S. population of youth, aged 8-20 years (NHANES 1999-2004). We compared the prevalence of adverse levels of nine CVD/DM risk factors between youths in the lowest quartile of RMM and their peers in the remaining quartiles, controlling for age, sex, and race/ethnicity. We also examined variations in the adjusted prevalence of these risk factors along the entire range of RMM. RESULTS The adjusted prevalence of adverse levels of risk factors among youths in the lowest quartile of RMM was significantly higher for seven of the nine risk factors examined compared with their peers in the other quartiles. Over the entire range of RMM, the adjusted prevalence of adverse levels of each of these seven risk factors decreased gradually with increasing RMM values (all p for trend <0.001). CONCLUSIONS RMM and prevalence of adverse risk factors for CVD/DM are highly and inversely associated in U.S. youth. Among youth with low RMM, the risk of these chronic diseases could be significantly high later in life.

Genitourinary health in a population-based cohort of males with Duchenne and Becker Muscular dystrophies.

Introduction: Genitourinary (GU) health among patients with Duchenne and Becker muscular dystrophies (DBMD) has not been explored using population‐based data. Methods: Medical records of 918 males ascertained by the Muscular Dystrophy Surveillance, Tracking, and Research Network were reviewed for documentation of GU‐related hospitalizations and prescribed medications. Percentages of males who received these medical interventions were calculated, and hazard ratios (HRs) and 95% confidence intervals (CIs) were estimated for associations with sociodemographics (study site, race/ethnicity), symptoms (early‐ vs. late‐onset, ambulation status, scoliosis), and treatments (respiratory support, steroids). Results: Among the 918 males, 81 (9%) had a GU condition, with voiding dysfunction (n = 40), GU tract infection (n = 19), and kidney/ureter calculus (n = 9) most frequently seen. The Kaplan‐Meier curve produced a cumulative probability of 27%. Cox regression showed GU conditions were more common when males were non‐ambulatory (HR 2.7, 95% CI 1.3–5.6). Conclusions: Our findings highlight the need for increased awareness of GU health and multidisciplinary care of DBMD patients. Muscle Nerve 52: 22–27, 2015

Integrating Household Water Treatment, Hand Washing, and Insecticide-Treated Bed Nets Into Pediatric HIV Care in Mombasa, Kenya: Impact on Diarrhea and Malaria Risk

Introduction: In developing countries, HIV-infected children are at higher risk of morbidity and mortality from opportunistic infections than HIV-uninfected children. To address this problem, the Healthy Living Initiative (HLI) in Mombasa, Kenya distributed basic care packages (BCPs) containing improved water storage vessels, water treatment solution, soap, and insecticide-treated bed nets to prevent diarrhea and malaria in children, and had community health workers (CHWs) make bimonthly home visits to encourage adherence to HLI interventions and antiretroviral (ARV) medicine use. Methods: To evaluate HLI, we enrolled 500 HIV-infected children from Bomu Hospital. In the implementation phase, from February to August 2011, we conducted surveys of caregivers, then provided free BCPs. In the evaluation phase, from September 2011 to August 2012, CHWs recorded observations of BCP use during home visits. We abstracted hospital data to compare diarrhea and malaria episodes, and pharmacy data on ARVs dispensed, between the 12-month preimplementation baseline phase (February 2010–January 2011) and the evaluation phase. Results: The retention rate of children in HLI was 78.4%. In a multivariable logistic regression model adjusting for demographic characteristics, number of CHW home visits, distance to clinic, orphan status, and number of ARVs dispensed, children in HLI had 71% lower risk of diarrhea (relative risk 0.29, P < 0.001) and 87% lower risk of malaria (relative risk 0.13, P = 0.001) during the evaluation phase than the baseline phase; there was no independent association between ARV use and illness. Conclusions: HIV-infected children in HLI were less likely to experience diarrhea and malaria during the evaluation phase than the baseline phase.

Impact of the Integration of Water Treatment, Hygiene, Nutrition, and Clean Delivery Interventions on Maternal Health Service Use.

AbstractReducing barriers associated with maternal health service use, household water treatment, and improved hygiene is important for maternal and neonatal health outcomes. We surveyed a sample of 201 pregnant women who participated in a clinic-based intervention in Kenya to increase maternal health service use and improve household hygiene and nutrition through the distribution of water treatment products, soap, protein-fortified flour, and clean delivery kits. From multivariable logistic regression analyses, the adjusted odds of ≥ 4 antenatal care (ANC4+) visits (odds ratio [OR] = 3.0, 95% confidence interval [CI] = 1.9-4.5), health facility delivery (OR = 5.3, 95% CI = 3.4-8.3), and any postnatal care visit (OR = 2.8, 95% CI = 1.9-4.2) were higher at follow-up than at baseline, adjusting for demographic factors. Women who completed primary school had higher odds of ANC4+ visits (OR = 1.8, 95% CI = 1.1-2.9) and health facility delivery (OR = 4.2, 95% CI = 2.5-7.1) than women with less education. For women who lived ≤ 2.5 km from the health facility, the estimated odds of health facility delivery (OR = 2.4, 95% CI = 1.5-4.1) and postnatal care visit (OR = 1.6, 95% CI = 1.0-2.6) were higher than for those who lived > 2.5 km away. Compared with baseline, a higher percentage of survey participants at follow-up were able to demonstrate proper handwashing (P = 0.001); water treatment behavior did not change. This evaluation suggested that hygiene, nutritional, clean delivery incentives, higher education level, and geographical contiguity to health facility were associated with increased use of maternal health services by pregnant women.