Rasch analysis of clinical outcome measures in spinal muscular atrophy.

INTRODUCTION: Trial design for SMA depends on meaningful rating scales to assess outcomes. In this study Rasch methodology was applied to 9 motor scales in spinal muscular atrophy (SMA). METHODS: Data from all 3 SMA types were provided by research groups for 9 commonly used scales. Rasch methodology assessed the ordering of response option thresholds, tests of fit, spread of item locations, residual correlations, and person separation index. RESULTS: Each scale had good reliability. However, several issues impacting scale validity were identified, including the extent that items defined clinically meaningful constructs and how well each scale measured performance across the SMA spectrum. CONCLUSIONS: The sensitivity and potential utility of each SMA scale as outcome measures for trials could be improved by establishing clear definitions of what is measured, reconsidering items that misfit and items whose response categories have reversed thresholds, and adding new items at the extremes of scale ranges.

A Systematic Review of Peer Support Interventions for Adolescents Living with HIV in Sub-Saharan Africa.

Despite widespread availability of life-saving antiretroviral therapy (ART) in sub-Saharan Africa, AIDS remains one of the leading causes of death among adolescents living with HIV (ALHIV) in sub-Saharan Africa. The purpose of this article was to review the state of the science regarding interventions to improve ART adherence and/or HIV care retention among ALHIV throughout sub-Saharan Africa. The primary aim of this review was to describe the impact of peer support interventions in improving treatment outcomes (i.e., ART adherence and retention in HIV care) among ALHIV in sub-Saharan Africa. The secondary aim of this review was to determine whether these interventions may be efficacious at improving mental health outcomes. We identified 27 articles that met the eligibility criteria for our review, and categorized each article based on the type of peer support provided to ALHIV-individualized peer support, group-based support, and individualized plus group-based support. Results regarding the efficacy of these interventions are mixed and most of the studies included were deemed moderate in methodological quality. Although studies evaluating group-based peer support interventions were the most common, most of these studies were not associated with retention, adherence, or mental health outcomes. More robust, fully powered studies are needed to strengthen our knowledge base regarding peer support for ALHIV.

Characteristics and outcomes of pediatric patients presenting at Cambodian referral hospitals without appointments: an observational study.

BACKGROUND: Emergency medicine is a young specialty in many low- and middle-income countries (LMICs). Although many patients seeking emergency or acute care are children, little information is available about the needs and current treatment of this group in LMICs. In this observational study, we sought to describe characteristics, chief complaints, management, and outcomes of children presenting for unscheduled visits to two Cambodian public hospitals. METHODS: Children enrolled in the study presented without appointment for treatment at one of two Cambodian public referral hospitals during a 4-week period in 2012. Researchers used standardized questionnaires and hospital records to collect demographic and clinical data. Patients were followed up at 48 h and 14 days after initial presentation. Multivariate logistic regression identified factors associated with hospital admission. RESULTS: This study included 867 unscheduled visits. Mean patient age was 5.7 years (standard deviation 4.8 years). Of the 35 different presenting complaints, fever (63%), respiratory problems (25%), and skin complaints (24%) were most common. The majority of patients were admitted (51%), while 1% were transferred to another facility. Seven patients (1%) died within 14 days. Follow-up rates were 83% at 48 h and 75% at 14 days. Predictors of admission included transfer or referral from another health provider, seeking prior care for the presenting problem, low socioeconomic status, onset of symptoms within 24 h of seeking care, abnormal vital signs or temperature, and chief complaint of abdominal pain or fever. CONCLUSIONS: While the admission rate in this study was high, mortality was low. More effective identification and management of children who can be treated and released may free up scarce inpatient resources for children who warrant admission.