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Household financial burden of phenylketonuria and its impact on treatment in China: a cross-sectional study.

ABSTRACT: BACKGROUND:Phenylketonuria (PKU) is a rare inborn disease, which, untreated, leading to severe neurobehavioral dysfunction. Considering its complexity, the management of PKU may bring a formidable economic burden to parents and caregivers. It is still unknown what the out-of-pocket expenses are for a patient with PKU in China. This paper explores the household financial burden of classical PKU and its impact on Chinese families in a quantitative manner for the first time. METHODS:A non-interventional and observational study was conducted at the China-Japan Friendship Hospital, one of the national centers for inherited metabolic disorders in China. The medical and non-medical household financial burdens were consolidated into a questionnaire to evaluate the out-of-pocket costs (OOPCs) of PKU treatment and follow-up. FINDINGS:The total OOPCs were USD$3766.1 (0y), USD$3795.2 (1-2 ys), USD$4657.7 (3-4 ys), USD$5979.9 (5-8 ys), and USD$5588.7 (9 ys and older) for PKU patients of different age groups. The median economic burden of classical PKU was 75.0 % of total annual family income (range 1.0-779.1 %), and 94.4 % of the families exceeding the threshold considered as catastrophic expenditure. There was a negative correlation between the financial burden and the proportion of time when Phe concentrations were in the desired target range (120-250 ?mol/L) in 0-4-ys group (r?=?-0.474, p?=?0.026). CONCLUSIONS:The management of PKU is associated with a severe financial burden on patients' families, which may lead to insufficient treatment or variation of blood Phe concentration. The current reimbursement policies are as yet inadequate. A national reimbursement system targeting treatment practices for PKU patients and other rare diseases across China is imperative.

SUBMITTER: Wang L

PROVIDER: S-EPMC5393103 | biostudies-literature | 2017 May

REPOSITORIES: biostudies-literature

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Household financial burden of phenylketonuria and its impact on treatment in China: a cross-sectional study.

Wang Lin L Zou Hui H Ye Fang F Wang Kundi K Li Xiaowen X Chen Zhihua Z Chen Jie J Han Bingjuan B Yu Weimin W He Chun C Shen Ming M

Journal of inherited metabolic disease 20161110 3

<h4>Background</h4>Phenylketonuria (PKU) is a rare inborn disease, which, untreated, leading to severe neurobehavioral dysfunction. Considering its complexity, the management of PKU may bring a formidable economic burden to parents and caregivers. It is still unknown what the out-of-pocket expenses are for a patient with PKU in China. This paper explores the household financial burden of classical PKU and its impact on Chinese families in a quantitative manner for the first time.<h4>Methods</h4> ...[more]

PMID: 27832415

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Project description:ObjectivesTo determine the incidence and intensity of household impoverishment induced by cancer treatment in China.DesignAverage income and daily consumption per capita of the households and out-of-pocket payments for cancer care were estimated. Household impoverishment was determined by comparing per capita daily consumption against the Chinese poverty line (CPL, US$1.2) and the World Bank poverty line (WBPL, US$1.9) for 2015. Both pre-treatment and post-treatment consumptions were calculated assuming that the households would divert daily consumption money to pay for cancer treatment.ParticipantsCancer patients diagnosed initially from 1 January 2015 to 31 December 2016 who had received cancer treatment subsequently. Those with multiple cancer diagnoses were excluded.Data sourcesA household questionnaire survey was conducted on 2534 cancer patients selected from nine hospitals in seven provinces through two-stage cluster/convenience sampling.Findings5.89% (CPL) to 12.94% (WBPL) households were impoverished after paying for cancer treatment. The adjusted OR (AOR) of post-treatment impoverishment was higher for older patients (AOR=2.666-4.187 for ≥50 years vs <50 years, p<0.001), those resided in central region (AOR=2.619 vs eastern, p<0.01) and those with lower income (AOR=0.024-0.187 in higher income households vs the lowest 20%, p<0.001). The patients without coverage from social health insurance had higher OR (AOR=1.880, p=0.040) of experiencing post-treatment household impoverishment than those enrolled with the insurance for urban employees. Cancer treatment is associated with an increase of 5.79% (CPL) and 12.45% (WBPL) in incidence of household impoverishment. The median annual consumption gap per capita underneath the poverty line accumulated by the impoverished households reached US$128 (CPL) or US$212 (WBPL). US$31 170 395 (CPL) or US$115 238 459 (WBPL) were needed to avoid household impoverishment induced by cancer treatment in China.ConclusionsThe financial burden of cancer treatment imposes a significant risk of household impoverishment despite wide coverage of social health insurance in China.

Project description:BackgroundPhenylketonuria (PKU) imposes a substantial burden on people living with the condition and their families. However, little is known about the time cost and financial burden of having PKU or caring for a child with the condition.Methods and findingsPrimary data were collected with a detailed cost and utilization survey. Primary outcomes included utilization and out-of-pocket costs of medical services, medical formula, and prescribed low-protein food consumption, as well as the time and perceived effort involved in following the PKU diet. Respondents were people living with PKU or parents of children with PKU identified through a state newborn screening program database. Secondary administrative claims data were also used to calculate mean total, insurer, and out-of-pocket payments in inpatient, outpatient (office visits, emergency room, and laboratory tests), and pharmacy settings for privately insured persons with PKU. Payments were calculated for sapropterin and for PKU formula.In primary data analysis (children n = 32, adults n = 52), annual out-of-pocket costs were highest for low-protein foods (child = $1651; adult = $967) compared with other categories of care. The time burden of PKU care was high; families reported spending more than 300 h per year shopping for and preparing special diet foods.In secondary data analysis, children 12-17 years old had the highest average medical expenditures ($54,147; n = 140) compared to children 0-11 years old ($19,057; n = 396) and adults 18 years and older ($40,705; n = 454). Medication costs were the largest contributor to medical costs, accounting for 61-81% of total costs across age groups. Sapropterin was the largest driver of medication costs, accounting for 85% of child medication costs and 92% of adult medication costs.ConclusionTreatment for PKU incurs a substantial time and cost burden on persons with PKU and their families. Estimated medical expenditures using claims data varied by age group, but sapropterin represented the largest cost for PKU treatment from a payer perspective across age groups.

Project description:BackgroundIn rural China ~607 million people drink boiled water, yet little is known about prevailing household water treatment (HWT) methods or their effectiveness. Boiling, the most common HWT method globally, is microbiologically effective, but household air pollution (HAP) from burning solid fuels causes cardiovascular and respiratory disease, and black carbon emissions exacerbate climate change. Boiled water is also easily re-contaminated. Our study was designed to identify the HWT methods used in rural China and to evaluate their effectiveness.MethodsWe used a geographically stratified cross-sectional design in rural Guangxi Province to collect survey data from 450 households in the summer of 2013. Household drinking water samples were collected and assayed for Thermotolerant Coliforms (TTC), and physicochemical analyses were conducted for village drinking water sources. In the winter of 2013-2104, we surveyed 120 additional households and used remote sensors to corroborate self-reported boiling data.FindingsOur HWT prevalence estimates were: 27.1% boiling with electric kettles, 20.3% boiling with pots, 34.4% purchasing bottled water, and 18.2% drinking untreated water (for these analyses we treated bottled water as a HWT method). Households using electric kettles had the lowest concentrations of TTC (73% lower than households drinking untreated water). Multilevel mixed-effects regression analyses showed that electric kettles were associated with the largest Log10TTC reduction (-0.60, p<0.001), followed by bottled water (-0.45, p<0.001) and pots (-0.44, p<0.01). Compared to households drinking untreated water, electric kettle users also had the lowest risk of having TTC detected in their drinking water (risk ratio, RR = 0.49, 0.34-0.70, p<0.001), followed by bottled water users (RR = 0.70, 0.53-0.93, p<0.05) and households boiling with pots (RR = 0.74, 0.54-1.02, p = 0.06).ConclusionAs far as we are aware, this is the first HWT-focused study in China, and the first to quantify the comparative advantage of boiling with electric kettles over pots. Our results suggest that electric kettles could be used to rapidly expand safe drinking water access and reduce HAP exposure in rural China.

Project description:Cluster headaches (CH) are recurrent severe headaches, which impose a major burden on the life of patients. We investigated the impact of CH on employment status and job burden.The study was a sub-study of the Korean Cluster Headache Registry. Patients with CH were enrolled from September 2016 to February 2018 from 15 headache clinics in Korea. We also enrolled a headache control group with age-sex matched patients with migraine or tension-type headache. Moreover, a control group including individuals without headache complaints was recruited. All participants responded to a questionnaire that included questions on employment status, type of occupation, working time, sick leave, reductions in productivity, and satisfaction with current occupation. The questionnaire was administered to participants who were currently employed or had previous occupational experience.We recruited 143 patients with CH, 38 patients with other types of headache (migraine or tension-type headache), and 52 headache-free controls. The proportion of employees was lower in the CH group compared with the headache and headache-free control groups (CH: 67.6% vs. headache controls: 84.2% vs. headache-free controls: 96.2%; p?=?0.001). The CH group more frequently experienced difficulties at work and required sick leave than the other groups (CH: 84.8% vs. headache controls: 63.9% vs. headache-free controls: 36.5%; p?<? 0.001; CH: 39.4% vs. headache controls: 13.9% vs. headache-free controls: 3.4%; p?<? 0.001). Among the patients with CH, sick leave was associated with younger age at CH onset (25.8 years vs. 30.6 years, p?=?0.014), severity of pain rated on a visual analogue scale (9.3 vs. 8.8, p?=?0.008), and diurnal periodicity during the daytime (p?=?0.003). There were no significant differences with respect to the sick leave based on sex, age, CH subtypes, and CH recurrence.CH might be associated with employment status. Most patients with CH experienced substantial burdens at work.

Project description:BACKGROUND: In regions of declining malaria transmission, new strategies for control are needed to reduce transmission and achieve elimination. Artemisinin-combination therapy (ACT) is active against immature gametocytes and can reduce the risk of transmission. We sought to determine whether household screening and treatment of infected individuals provides protection against infection for household members. METHODOLOGY/PRINCIPAL FINDINGS: The study was conducted in two areas in Southern Province, Zambia in 2007 and 2008/2009. To determine the impact of proactive case detection, households were randomly selected either to join a longitudinal cohort, in which participants were repeatedly screened throughout the year and those infected treated with artemether-lumefantrine, or a cross-sectional survey, in which participants were visited only once. Cross-sectional surveys were conducted throughout the year. The prevalence of RDT positivity was compared between the longitudinal and cross-sectional households at baseline and during follow-up using multilevel logistic regression. In the 2007 study area, 174 and 156 participants enrolled in the cross-sectional and longitudinal groups, respectively. In the 2008/2009 study area, 917 and 234 participants enrolled in the cross-sectional and longitudinal groups, respectively. In both study areas, participants and households in the longitudinal and cross-sectional groups were similar on demographic characteristics and prevalence of RDT positivity at baseline (2007: OR?=?0.97; 95% CI:0.46, 2.03 | 2008/2009: OR?=?1.28; 95% CI:0.44, 3.79). After baseline, the prevalence of RDT positivity was significantly lower in longitudinal compared to cross-sectional households in both study areas (2007: OR?=?0.44; 95% CI:0.20, 0.96 | 2008/2009: OR?=?0.16; 95% CI:0.05, 0.55). CONCLUSIONS/SIGNIFICANCE: Proactive case detection, consisting of screening household members with an RDT and treating those positive with ACT, can reduce transmission and provide indirect protection to household members. A targeted test and treat strategy could contribute to the elimination of malaria in regions of low transmission.

Dataset Information

Household financial burden of phenylketonuria and its impact on treatment in China: a cross-sectional study.

Publications

Household financial burden of phenylketonuria and its impact on treatment in China: a cross-sectional study.

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