Inadequate compliance with ICT in thalassemia major is common and results in substantial morbidity and mortality, as well as increased costs.

Results of this analysis of the cost effectiveness of oral deferasirox versus infusional deferoxamine suggest that deferasirox is a cost effective iron chelator from a US healthcare perspective.

Background: Causal mediation analysis can improve understanding of the mechanisms underlying epidemiologic associations. However, the utility of natural direct and indirect effect estimation has been limited by the assumption of no confounder of the mediator-outcome relationship that is affected by prior exposure-an assumption frequently violated in practice.Methods: We build on recent work that identified alternative estimands that do not require this assumption and propose a flexible and double robust semiparametric targeted minimum loss-based estimator for data-dependent stochastic direct and indirect effects. The proposed method treats the intermediate confounder affected by prior exposure as a time-varying confounder and intervenes stochastically on the mediator using a distribution which conditions on baseline covariates and marginalizes over the intermediate confounder. In addition, we assume the stochastic intervention is given, conditional on observed data, which results in a simpler estimator and weaker identification assumptions.Results: We demonstrate the estimator's finite sample and robustness properties in a simple simulation study. We apply the method to an example from the Moving to Opportunity experiment. In this application, randomization to receive a housing voucher is the treatment/instrument that influenced moving to a low-poverty neighborhood, which is the intermediate confounder. We estimate the data-dependent stochastic direct effect of randomization to the voucher group on adolescent marijuana use not mediated by change in school district and the stochastic indirect effect mediated by change in school district. We find no evidence of mediation.Conclusions: Our estimator is easy to implement in standard statistical software, and we provide annotated R code to further lower implementation barriers.

Acute malnutrition accounts for an immense disease burden and is implicated as a key, underlying cause of child mortality in low resource settings. Child wasting, defined as weight-for-length more than 2 standard deviations below international standards, is a leading indicator to measure the Sustainable Development Goal target to end malnutrition by 2030. Prevailing methods to measure wasting rely on cross-sectional surveys that are unable to measure onset, recovery, and persistence - key features of wasting epidemiology that could inform preventive interventions and disease burden estimates. Here, we show through an analysis of 18 longitudinal cohorts that child wasting is a highly dynamic process of incident onset and recovery, and that peak incidence is between birth and 3 months - far earlier than peak prevalence at 12-15 months. By age 24 months the proportion of children who had ever experienced a wasting episode (33%) was more than 5-fold higher than prevalence (6%), suggesting that the wasting burden is likely far higher than cross-sectional surveys suggest. Seasonally driven changes in population mean weight-for-length were large (>0.5 z in some cohorts) and were synchronous with rainfall across diverse settings, creating potential for seasonally targeted interventions. Our results motivate a new focus on extending preventive interventions for child wasting to pregnant and lactating mothers, and for preventive and therapeutic interventions to include children below age 6 months in addition to current targets of ages 6-59 months.

Abatacept is cost-effective by current standards of medical practice in patients with moderately to severely active RA and inadequate response to MTX.

BackgroundHealthcare claims databases have been used in several studies to characterize the risk and burden of chemotherapy-induced febrile neutropenia (FN) and effectiveness of colony-stimulating factors against FN. The accuracy of methods previously used to identify FN in such databases has not been formally evaluated.MethodsData comprised linked electronic medical records from Geisinger Health System and healthcare claims data from Geisinger Health Plan. Subjects were classified into subgroups based on whether or not they were hospitalized for FN per the presumptive “gold standard” (ANC <1.0×109/L, and body temperature ≥38.3°C or receipt of antibiotics) and claims-based definition (diagnosis codes for neutropenia, fever, and/or infection). Accuracy was evaluated principally based on positive predictive value (PPV) and sensitivity.ResultsAmong 357 study subjects, 82 (23%) met the gold standard for hospitalized FN. For the claims-based definition including diagnosis codes for neutropenia plus fever in any position (n=28), PPV was 100% and sensitivity was 34% (95% CI: 24–45). For the definition including neutropenia in the primary position (n=54), PPV was 87% (78–95) and sensitivity was 57% (46–68). For the definition including neutropenia in any position (n=71), PPV was 77% (68–87) and sensitivity was 67% (56–77).ConclusionsPatients hospitalized for chemotherapy-induced FN can be identified in healthcare claims databases--with an acceptable level of mis-classification--using diagnosis codes for neutropenia, or neutropenia plus fever.

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Child growth failure is associated with a higher risk of illness and mortality, which contributed to the United Nations Sustainable Development Goal 2.2 to end malnutrition by 2030. Current prenatal and postnatal interventions, such as nutritional supplementation, have been insufficient to eliminate growth failure in low resource settings -motivating a search for key age windows and population subgroups in which to focus future preventive efforts. Quantifying the effect of early growth failure on severe outcomes is important to assess burden and longer-term impacts on the child. Here we show through an analysis of 35 longitudinal cohorts (108,336 children) that maternal and child characteristics at birth accounted for the largest attributable differences in growth. Yet, postnatal growth failure was larger than differences at birth, and characteristics of the child's household environment were additional determinants of growth failure after age 6 months. Children who experienced early ponderal or linear growth failure were at much higher risk of persistent growth failure and were 2.0 to 4.8 times more likely to die by age 24 months. High attributable risk from prenatal causes, and severe consequences for children who experienced early growth failure, support a focus on pre-conception and pregnancy as key opportunities for new preventive interventions. Our results suggest that broad improvements in wellbeing will be necessary to eliminate growth failure in low resource settings, but that screening based on weight could help identify children at highest risk of death before age 24 months.