Using poverty maps to improve the design of household surveys: the evidence from Tunisia.

In this paper we aim to propose a new method for improving the design effect of household surveys based on a two-stage design in which the first stage clusters, or Primary Selection Units (PSUs), are stratified along administrative boundaries. Improvement of the design effect can result in more precise survey estimates (smaller standard errors and confidence intervals) or in the reduction of the necessary sample size, i.e. a reduction in the budget needed for a survey. The proposed method is based on the availability of a previously conducted poverty maps, i.e. spatial descriptions of the distribution of per capita consumption expenditures, that are finely disaggregated in small geographic units, such as cities, municipalities, districts or other administrative partitions of a country that are directly linked to PSUs. Such information is then used to select PSUs with systematic sampling by introducing further implicit stratification in the survey design, so as to maximise the improvement of the design effect. Since per capita consumption expenditures estimated at PSU level from the poverty mapping are affected by (small) standard errors, in the paper we also perform a simulation study in order to take into account this addition variability.

The 'Double Fuzzy Set' Approach to Multidimensional Poverty Measurement: With a Focus on the Health Dimension.

In more recent times, there is an increasing consensus in the field of development study to view poverty as a multidimensional deprivation beyond the more commonly used monetary perspective. Although the multidimensional poverty measurement is gaining more acceptance among policy makers, it is still based on the clear distinction between the poor and non-poor through an arbitrary threshold. One alternative to this shortcoming is offered by a multidimensional poverty measurement with a fuzzy-set approach in which it is possible to recognize deprivations as a matter of degree. The integrated fuzzy approach allows this possibility, although there is an unexplored opportunity of recognising that two or more dimensions can be attributed to a single item or attribute. This paper aims to contribute to this strand of research by introducing a 'Double-Fuzzy' approach. The methodology is applied to Tunisia using the Tunisian National Survey on Household Budget and Consumption (HBS) 2015.

Social and medical needs of rare metabolic patients: results from a MetabERN survey.

BACKGROUND: Many surveys have been performed over the years to assess the medical and social requirements of patients with a rare disease, but no studies have focused specifically on patients in Europe or with an inherited metabolic disease (IMD). To obtain a comprehensive overview of the social and psychological status and needs of IMD patients, especially in Europe, the European Reference Network for Hereditary Metabolic Disorders (MetabERN) has performed a dedicated survey among its metabolic patients. RESULTS: A total of 924 patients and caregivers responded to the questionnaire. Most participants were from 25 European countries, with Spain, Italy, and Germany being the most represented; only eight participants were extra-European. The survey showed that most social assistance services, from free educational/development services for those with intellectual disability to transition from childhood to adult care and job placement support, are available for a limited number of patients or are unknown to the majority of patients or their parents/caregivers. Similarly, psychological assistance for the patient or the parent/caregiver is available for a small fraction of respondents, despite the fact that the majority considers this type of support necessary for both the patient and the caregiver. In addition, for most IMD patients local specialised or emergency medical assistance is lacking, although national clinical pathways are defined, and medical professionals of reference are readily available when needed. Lastly, while most national health services in Europe cover all or part of the expenses for medications, medical devices, food supplements, dietary integrators, physiotherapy, and speech therapy, significant gaps in the economic support for healthcare and other expenses still exist. CONCLUSIONS: Overall, our survey reveals a widespread lack of social, psychological, and economic support for IMD patients in Europe. More needs to be done to provide daily assistance to IMD patients in order to alleviate the burden on caregivers and to allow patients to become independent and productive adults. Where support is actually available locally or nationally, most IMD patients are not aware of it, so an active dissemination of this information among the metabolic community is essential.

A randomized trial of oral betamethasone to reduce ataxia symptoms in ataxia telangiectasia.

No controlled studies exist regarding the pharmaceutical reduction of ataxia symptoms in ataxia telangiectasia (A-T). In a multicenter, double-blind, randomized, placebo-controlled crossover trial, oral betamethasone (BETA) and placebo were compared in terms of their reduction of ataxia symptoms as assessed with the International Cooperative Ataxia Rating Scale (ICARS). In this study of 13 A-T children, betamethasone reduced the ICARS total score by a median of 13 points in the intent-to-treat population and 16 points in the per-protocol population (ie, median percent decreases of ataxia symptoms of 28% and 31%, respectively). In conclusion, Oral betamethasone could be a promising therapy to relieve ataxia symptoms in A-T patients; however, long-term effectiveness and safety must be established. (Current Controlled Trials, number ISRCTN08774933.)

Silent increase of urinary ethylmalonic acid is an indicator of nonspecific brain dysfunction.

Our aim was to compare urinary ethylmalonic acid (EMA) levels in subjects who had no apparent clinical reason to have increased levels of this substance but were suffering from non-specific CNS impairment, and healthy controls. Urinary EMA concentrations detected by (1)H-NMR spectroscopy were studied in 130 subjects with CNS impairment of unknown origin (with no definite diagnosis, no specific symptoms or signs, and normal common biochemical and metabolic screening results) and 130 age- and sex-matched healthy subjects. EMA levels exceeding two standard deviations (SD) above normal (i.e. 8.1 mmol/molCn) were found in a subgroup of CNS-impaired patients and healthy controls. EMA levels exceeding 2 SD above normal were fourfold prevalent in the urine of patients with non-specific CNS impairment compared to from the EMA levels in healthy controls. Moreover, we found that the level exceeding > 8.1 mmol/molCn (i.e. > + 2 SD) had sufficient discrimination accuracy in identifying subjects with non-specific CNS impairment; the level exceeding 12 mmol/molCn (i.e. > + 6 SD) reaches suitable accuracy (i.e. 100% specificity and 78.6% sensitivity). These observations are of importance, as we found that subtle increases in urinary EMA levels are frequent in patients with non-specific CNS impairment. The reasons for this association remain unknown.