Breaking the Matches in a Paired T-Test for Community Interventions When the Number of Pairs is Small

There is considerable interest in community interventions for health promotion, where the community is the experimental unit. Because such interventions are expensive, the number of experimental units (communities) is usually small. Because of the small number of communities involved, investigators often match treatment and control communities on demographic variables before randomization to minimize the possibility of a bad split. Unfortunately, matching has been shown to decrease the power of the design when the number of pairs is small, unless the matching variable is very highly correlated with the outcome variable (in this case, with change in the health behavior). We used computer simulation to examine the performance of an approach in which we matched communities but performed an unmatched analysis. If the appropriate matching variables are unknown, and there are fewer than ten pairs, an unmatched design and analysis has the most power. If, however, one prefers a matched design, then for N \u3c 10, power can be increased by performing an unmatched analysis of the matched data. We also discuss a variant of this procedure, in which an unmatched analysis is performed only if the matching didn\u27t work

A Community-Wide Collaboration to Reduce Cardiovascular Disease Risk: The Hearts of Sonoma County Initiative.

PURPOSE AND OBJECTIVES: Collaboration across multiple sectors is needed to bring about health system transformation, but creating effective and sustainable collaboratives is challenging. We describe outcomes and lessons learned from the Hearts of Sonoma County (HSC) initiative, a successful multi-sector collaborative effort to reduce cardiovascular disease (CVD) risk in Sonoma County, California. INTERVENTION APPROACH: HSC works in both clinical systems and communities to reduce CVD risk. The initiative grew out of a longer-term county-wide collaborative effort known as Health Action. The clinical component involves activating primary care providers around management of CVD risk factors; community activities include community health workers conducting blood pressure screenings and a local heart disease prevention campaign. EVALUATION METHODS: The impact of the clinical improvement efforts was tracked using blood pressure data from the 4 health systems participating in HSC. Descriptive information on the community-engagement efforts was obtained from program records. Lessons learned in developing and maintaining the collaborative were gathered through document review and interviews with key informants. RESULTS: Favorable trends were seen in blood pressure control among patients with hypertension in the participating health systems: patients with controlled blood pressure increased from 58% in 2014 to 67% in 2016 (P \u3c .001). Between 2017 and 2019, the community engagement effort conducted 99 outreach events, reaching 1,751 individuals, and conducted 1,729 blood pressure screenings, with 441 individuals referred to clinical providers for follow-up care. HSC scored highly on 6 essential elements of an effective coalition and achieved a degree of sustainability that has eluded many other collaboratives. IMPLICATIONS FOR PUBLIC HEALTH: Factors contributing to the success of HSC include 1) starting small and focused to build trust among participants and demonstrate value, 2) working within the framework of a larger effort, and 3) providing long-term, open-ended backbone support

Rett syndrome with and without detected MECP2 mutations: an attempt to redefine phenotypes

Background: The diagnosis of Rett syndrome (RTT) is based on a set of clinical criteria, irrespective of mutation status. The aims of this study were (1) to define the clinical differences existing between patients with Rett syndrome with (Group I) and without a MECP2 mutation (Group II), and (2) to characterize the phenotypes associated with the more common MECP2 mutations. Patients and methods: We analyzed 87 patients fulfilling the clinical criteria for RTT. All were observed and videotaped by the same paediatric neurologist. Seven common mutations were considered separately, and associated clinical features analysed. Results: Comparing Group I and II, we found differences concerning psychomotor development prior to onset, acquisition of propositive manipulation and language, and evolving autistic traits. Based on age at observation, we found differences in eye pointing, microcephaly, growth, number of stereotypies, rigidity, ataxia and ataxic-rigid gait, and severity score. Patients with truncating differed from those with missense mutations regarding acquisition of propositive words and independent gait, before the beginning of the disease, and microcephaly, growth, foot length, dystonia, rigidity and severity score, at the time of observation. Patients with the R168X mutation had a more severe phenotype, whereas those with R133C showed a less severe one. Patients with R294X had a hyperactive behaviour, and those with T158M seemed to be particularly ataxic and rigid. Conclusion: A clear regressive period (with loss of prehension and language, deceleration of growth) and the presence of more than three different stereotypies, rigidity and ataxic-rigid gait seemed to be very helpful in differentiating Group I from Group II

Convalescent plasma in patients admitted to hospital with COVID-19 (RECOVERY): a randomised controlled, open-label, platform trial

Background: Many patients with COVID-19 have been treated with plasma containing anti-SARS-CoV-2 antibodies. We aimed to evaluate the safety and efficacy of convalescent plasma therapy in patients admitted to hospital with COVID-19. Methods: This randomised, controlled, open-label, platform trial (Randomised Evaluation of COVID-19 Therapy [RECOVERY]) is assessing several possible treatments in patients hospitalised with COVID-19 in the UK. The trial is underway at 177 NHS hospitals from across the UK. Eligible and consenting patients were randomly assigned (1:1) to receive either usual care alone (usual care group) or usual care plus high-titre convalescent plasma (convalescent plasma group). The primary outcome was 28-day mortality, analysed on an intention-to-treat basis. The trial is registered with ISRCTN, 50189673, and ClinicalTrials.gov, NCT04381936. Findings: Between May 28, 2020, and Jan 15, 2021, 11558 (71%) of 16287 patients enrolled in RECOVERY were eligible to receive convalescent plasma and were assigned to either the convalescent plasma group or the usual care group. There was no significant difference in 28-day mortality between the two groups: 1399 (24%) of 5795 patients in the convalescent plasma group and 1408 (24%) of 5763 patients in the usual care group died within 28 days (rate ratio 1·00, 95% CI 0·93–1·07; p=0·95). The 28-day mortality rate ratio was similar in all prespecified subgroups of patients, including in those patients without detectable SARS-CoV-2 antibodies at randomisation. Allocation to convalescent plasma had no significant effect on the proportion of patients discharged from hospital within 28 days (3832 [66%] patients in the convalescent plasma group vs 3822 [66%] patients in the usual care group; rate ratio 0·99, 95% CI 0·94–1·03; p=0·57). Among those not on invasive mechanical ventilation at randomisation, there was no significant difference in the proportion of patients meeting the composite endpoint of progression to invasive mechanical ventilation or death (1568 [29%] of 5493 patients in the convalescent plasma group vs 1568 [29%] of 5448 patients in the usual care group; rate ratio 0·99, 95% CI 0·93–1·05; p=0·79). Interpretation: In patients hospitalised with COVID-19, high-titre convalescent plasma did not improve survival or other prespecified clinical outcomes. Funding: UK Research and Innovation (Medical Research Council) and National Institute of Health Research

The Potential of Partnerships for Health Advocacy and Policy Change: The Legacy of the Partnership for the Public’s Health Initiative

· This article reports on a study of 11 partnerships between public health departments and community organizations that were funded by The California Endowment to support advocacy and organizing to improve health outcomes in the communities. · The evaluation examined the sustainability of the partnerships as well as the policy and advocacy work of the organizations. · Almost 90 percent of the activities in policy change and community capacity building was sustained, whereas partnership and health department capacity building activities were the least likely to be sustained. · The policy change legacies at the community level were strong and included empowerment of community members, the creation of healthier environments, increased access to services, and increased focus on health by local governments