Cytogenetic and survival analysis in 315 multiple myeloma patients; five with orbital and eight with extramedullary disease.

PURPOSE: To describe cytogenetic abnormalities in five patients with orbital involvement of multiple myeloma (MM) and to determine if high-risk cytogenetic abnormalities were more prevalent in patients with extramedullary disease (EMD). The final goal was to determine predictors of poor survival in the entire cohort. METHODS: Retrospective cohort study of 315 patients diagnosed with multiple myeloma over a 9-year period at a single institution. RESULTS: Males comprised 61% of the 315 patients and the majority (54.3%) were stage II at diagnosis of MM. Follow-up ranged 6 months to 18 years, mean 5.3 ± 3.7 years. Eight patients (2.5%), two of which had orbital involvement, developed EMD and an additional three patients had orbital involvement from adjacent bone disease. No genetic abnormality reached statistical significance between non-EMD and EMD groups. At the time of analysis 127 (41%) non-EMD patients and 6 (75%) EMD patients have died. The median survival time for the EMD group was 2 years, whereas for the non-EMD group it was 9 years (P-value of log rank test = .00015). Stage at time of MM diagnosis and age were associated with worse prognosis. Median survival for patients over 65 years of age was 7 years, compared to 12 years for patients 65 or younger (P-value for the log-ranked test <.001). CONCLUSIONS: Cytogenetic abnormalities are described in five patients with orbital involvement of MM. No genetic abnormality reached statistical significance between non-EMD and EMD groups. EMD status, stage of MM at diagnosis and age are associated with poorer overall survival.Abbreviation: MM: Multiple Myeloma; EMD: Extramedullary disease; FISH: fluorescence in situ hybridization; B2M: beta-2 microglobulin; LDH: lactate dehydrogenase; OS: overall survival.

Exploring association of mobile phone access with positive health outcomes and behaviors amongst post-partum mothers in rural Malawi.

BACKGROUND: Access to mass media and emerging technologies (e.g., cell phones, the internet, and social media) is a social determinant of health that has been shown to profoundly influence women's health outcomes. In the African region, where women in rural settings with limited access to care are most vulnerable to maternal mortality and other pregnancy-related morbidities, mobile phone access can be an important and life-saving health determinant. OBJECTIVE: The goal of this study was to examine the association between mobile/cellular phone ownership and health behaviors of post-partum mothers in rural Malawi. METHODS: In this cross-sectional study, we recruited and consented a convenient sample of 174 post-partum mothers of 4- and 5-month-olds who were attending well-child clinics in Gowa, situated in the rural Ntcheu district of Malawi. Using logistic regression models, we hypothesized that compared to non-cell phone owners, mobile phone ownership will be predictive (greater odds) of antenatal visit frequency, exclusive breastfeeding knowledge and practices, health-seeking behaviors, and involvement in motherhood support groups; and protective (lower odds) of infant illnesses, breastfeeding challenges, and post-partum depressive symptoms. RESULTS: Mobile phones were highly prevalent in this rural setting, with 45% (n = 79) of post-partum women indicating they owned at least one cell phone. Cell phone owners tended to have higher levels of education (p < 0.012) and wealth (p < 0.001). Interestingly, mobile phone ownership was only associated with exclusive breastfeeding practices; and phone owners had 75% lower odds of exclusively breastfeeding (adj. OR 0.25; 95% CI: 0.07-0.92, p = 0.038) in multivariable models. Though not statistically significant but clinically meaningful, cell phone ownership was associated with fewer depressive symptoms (adj. OR 0.84; 95% CI: 0.39-1.84, p = 0.67) and more social support (adj. OR 1.14; 95% CI: 0.61-2.13, p = 0.70). CONCLUSIONS: Digital literacy and internet connectivity are social determinants of health, thus delving deeper into mothers' digital experiences to identify and ameliorate their unique barriers to full digital access will be crucial to successful implementation of digital interventions to address post-partum challenges for women in hard-to-reach settings such as ours. Such interventions are of even greater relevance as the Covid-19 pandemic has increased the urgency of reaching vulnerable, marginalized populations.

The Effectiveness of Web-Based Asthma Self-Management System, My Asthma Portal (MAP): A Pilot Randomized Controlled Trial.

BACKGROUND: Whether Web-based technologies can improve disease self-management is uncertain. My Asthma Portal (MAP) is a Web-based self-management support system that couples evidence-based behavioral change components (self-monitoring of symptoms, physical activity, and medication adherence) with real-time monitoring, feedback, and support from a nurse case manager. OBJECTIVE: The aim of this study was to compare the impact of access to a Web-based asthma self-management patient portal linked to a case-management system (MAP) over 6 months compared with usual care on asthma control and quality of life. METHODS: A multicenter, parallel, 2-arm, pilot, randomized controlled trial was conducted with 100 adults with confirmed diagnosis of asthma from 2 specialty clinics. Asthma control was measured using an algorithm based on overuse of fast-acting bronchodilators and emergency department visits, and asthma-related quality of life was assessed using the Mini-Asthma Quality of Life Questionnaire (MAQLQ). Secondary mediating outcomes included asthma symptoms, depressive symptoms, self-efficacy, and beliefs about medication. Process evaluations were also included. RESULTS: A total of 49 individuals were randomized to MAP and 51 to usual care. Compared with usual care, participants in the intervention group reported significantly higher asthma quality of life (mean change 0.61, 95% CI 0.03 to 1.19), and the change in asthma quality of life for the intervention group between baseline and 3 months (mean change 0.66, 95% CI 0.35 to 0.98) was not seen in the control group. No significant differences in asthma quality of life were found between the intervention and control groups at 6 (mean change 0.46, 95% CI -0.12 to 1.05) and 9 months (mean change 0.39, 95% CI -0.2 to 0.98). For poor control status, there was no significant effect of group, time, or group by time. For all self-reported measures, the intervention group had a significantly higher proportion of individuals, demonstrating a minimal clinically meaningful improvement compared with the usual care group. CONCLUSIONS: This study supported the use of MAP to enhance asthma quality of life but not asthma control as measured by an administrative database. Implementation of MAP beyond 6 months with tailored protocols for monitoring symptoms and health behaviors as individuals' knowledge and self-management skills improve may result in long-term gains in asthma control. CLINICALTRIAL: International Standard Randomized Controlled Trial Number (ISRCTN): 34326236; http://www.isrctn.com/ISRCTN34326236 (Archived by Webcite at http://www.webcitation.org/6mGxoI1R7).

Derivation and validation of clinical phenotypes for COPD: a systematic review.

BACKGROUND: The traditional classification of COPD, which relies solely on spirometry, fails to account for the complexity and heterogeneity of the disease. Phenotyping is a method that attempts to derive a single or combination of disease attributes that are associated with clinically meaningful outcomes. Deriving phenotypes entails the use of cluster analyses, and helps individualize patient management by identifying groups of individuals with similar characteristics. We aimed to systematically review the literature for studies that had derived such phenotypes using unsupervised methods. METHODS: Two independent reviewers systematically searched multiple databases for studies that performed validated statistical analyses, free of definitive pre-determined hypotheses, to derive phenotypes among patients with COPD. Data were extracted independently. RESULTS: 9156 citations were retrieved, of which, 8 studies were included. The number of subjects ranged from 213 to 1543. Most studies appeared to be biased: patients were more likely males, with severe disease, and recruited in tertiary care settings. Statistical methods used to derive phenotypes varied by study. The number of phenotypes identified ranged from 2 to 5. Two phenotypes, with poor longitudinal health outcomes, were common across multiple studies: young patients with severe respiratory disease, few cardiovascular co-morbidities, poor nutritional status and poor health status, and a phenotype of older patients with moderate respiratory disease, obesity, cardiovascular and metabolic co-morbidities. CONCLUSIONS: The recognition that two phenotypes of COPD were often reported may have clinical implications for altering the course of the disease. This review also provided important information on limitations of phenotype studies in COPD and the need for improvement in future studies.

Tailoring interventions: identifying predictors of poor asthma control.

BACKGROUND: Determining the factors that will predict long-term asthma control is essential for improving health outcomes and decreasing the burden on the health care system. Patient-reported outcomes (PROs) on health behaviors can provide valuable information about future asthma control but have rarely been considered in previous analyses. OBJECTIVE: To develop statistical models for evaluating the predictors of long-term asthma control using PROs such as scores of the Asthma Control Test and the Asthma Self-Efficacy Scale. METHODS: Of 1,437 individuals contacted, 566 (39%) at baseline and 486 (34%) at follow-up completed the questionnaires, including 4 PROs (Asthma Control Test, Asthma Self-Efficacy Scale, Mini-Asthma Quality of Life Questionnaire, and Beliefs about Medication Questionnaire). Long-term asthma control was evaluated by assessing overuse of rescue medication and emergency department visits. A multivariate logistic generalized estimating equation model was fitted to evaluate the possible effect of the studied factors on asthma control. RESULTS: The complete case generalized estimating equation analysis included 286 participants who had complete PROs at the 2 evaluation times. After adjusting for socioeconomic status and smoking status, the Mini-Asthma Quality of Life Questionnaire was a significant predictor of asthma exacerbation. For each 1-point increase on the Mini-Asthma Quality of Life Questionnaire, there was a 0.25 decrease in the odds of a patient's asthma getting out of control. CONCLUSION: These findings suggest opportunities to decrease the burden on health care by tailoring interventions that combine PROs with other clinical and sociodemographic variables.

Interval estimation of some epidemiological measures of association.

In epidemiological cohort studies, the probability of developing a disease for individuals in a treatment/intervention group is compared with that of a control group. The groups involve varying cluster sizes, and the binary responses within each cluster cannot be assumed independently. Three major measures of association used to report the efficacy of treatments or effectiveness of public health intervention programs in case of prospective studies are Risk Difference (RD), Risk Ratio (RR) and Relative Risk Difference (RED). The preference of one measure of association over the other in drawing statistical inference depends on design of study. Lui (2004) discusses a number of methods of constructing confidence intervals for each of these measures. Specifically, Lui (2004) discusses four methods for RD, four methods for RR and three methods for RED. For the construction of confidence intervals for RD, Paul and Zaihra (2008) compare the four methods discussed by Lui (2004), using extensive simulations with a method based on an estimator of the variance of a ratio estimator by Cochran (1977) and a method based on a sandwich estimator of the variance of the regression estimator using the generalized estimating equations approach of Zeger and Liang (1986). Paul and Zaihra (2008) conclude that the method based on an estimate of the variance of a ratio estimator performs best overall. In this paper, we extend the two new methodologies introduced in Paul and Zaihra (2008) to confidence interval construction of the risk measures RR and RED. Extensive simulations show that the method based on an estimate of the variance of a ratio estimator performs best overall for constructing confidence interval for the other two risk measures RR and RED as well. This method involves a very simple variance expression which can be implemented with a very few computer codes. Therefore, it can be considered as an easily implementable alternative for all the three measures of association.

Interval estimation of risk difference for data sampled from clusters.

Risk difference (RD) is an important measure in epidemiological studies where the probability of developing a disease for individuals in an exposed group, for example, is compared with that in a control group. There are varying cluster sizes in each group and the binary responses within each cluster cannot be assumed independent. Under the cluster sampling scenario, Lui (Statistical Estimation of Epidemiological Risk. Wiley: CA, 2004; 7-27) discusses four methods for the construction of a confidence interval for the RD. In this paper we introduce two very simple methods. One method is based on an estimator of the variance of a ratio estimator (Sampling Techniques (3rd edn). Wiley: New York, 1977; 30-67) and the other method is based on a sandwich estimator of the variance of the regression estimator using the generalized estimating equations approach of Zeger and Liang (Biometrics 1986; 42:121-130). These two methods are then compared, by simulation, in terms of maintaining nominal coverage probability and average coverage length, with the four methods discussed by Lui (Statistical Estimation of Epidemiological Risk. Wiley: CA, 2004; 7-27). Simulations show at least as good properties of these two methods as those of the others. The method based on an estimate of the variance of a ratio estimator performs best overall. It involves a very simple variance expression and can be implemented with a very few computer codes. Therefore, it can be considered as an easily implementable alternative.