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Socioeconomic inequalities in mortality from conditions amenable to medical interventions: do they reflect inequalities in access or quality of health care?

BackgroundPrevious studies have reported large socioeconomic inequalities in mortality from conditions amenable to medical intervention, but it is unclear whether these can be attributed to inequalities in access or quality of health care, or to confounding influences such as inequalities in background risk of diseases. We therefore studied whether inequalities in mortality from conditions amenable to medical intervention vary between countries in patterns which differ from those observed for other (non-amenable) causes of death. More specifically, we hypothesized that, as compared to non-amenable causes, inequalities in mortality from amenable causes are more strongly associated with inequalities in health care use and less strongly with inequalities in common risk factors for disease such as smoking.MethodsCause-specific mortality data for people aged 30–74 years were obtained for 14 countries, and were analysed by calculating age-standardized mortality rates and relative risks comparing a lower with a higher educational group. Survey data on health care use and behavioural risk factors for people aged 30–74 years were obtained for 12 countries, and were analysed by calculating age-and sex-adjusted odds ratios comparing a low with a higher educational group. Patterns of association were explored by calculating correlation coefficients.ResultsIn most countries and for most amenable causes of death substantial inequalities in mortality were observed, but inequalities in mortality from amenable causes did not vary between countries in patterns that are different from those seen for inequalities in non-amenable mortality. As compared to non-amenable causes, inequalities in mortality from amenable causes are not more strongly associated with inequalities in health care use. Inequalities in mortality from amenable causes are also not less strongly associated with common risk factors such as smoking.ConclusionsWe did not find evidence that inequalities in mortality from amenable conditions are related to inequalities in access or quality of health care. Further research is needed to find the causes of socio-economic inequalities in mortality from amenable conditions, and caution should be exercised in interpreting these inequalities as indicating health care deficiencies.

Using ‘amenable mortality’ as indicator of healthcare effectiveness in international comparisons: results of a validation study

Background and study aims There is widespread consensus on the need for better indicators of the effectiveness of healthcare. We carried out an analysis of the validity of amenable mortality as an indicator of the effectiveness of healthcare, focusing on the potential use in routine surveillance systems of between-country variations in rates of mortality. We assessed whether the introduction of specific healthcare innovations coincided with declines in mortality from potentially amenable causes in seven European countries. In this paper, we summarise the main results of this study and illustrate them for four conditions. Data and methods We identified 14 conditions for which considerable declines in mortality have been observed and for which there is reasonable evidence in the literature of the effectiveness of healthcare interventions to lower mortality. We determined the time at which these interventions were introduced and assessed whether the innovations coincided with favourable changes in the mortality trends from these conditions, measured using Poisson linear spline regression. All the evidence was then presented to a Delphi panel. Main results The timing of innovation and favourable change in mortality trends coincided for only a few conditions. Other reasons for mortality decline are likely to include diffusion and improved quality of interventions and in incidence of diseases and their risk factors, but there is insufficient evidence to differentiate these at present. For most conditions, a Delphi panel could not reach consensus on the role of current mortality levels as measures of effectiveness of healthcare. Discussion and conclusions Improvements in healthcare probably lowered mortality from many of the conditions that we studied but occurred in a much more diffuse way than we assumed in the study design. Quantification of the contribution of healthcare to mortality requires adequate data on timing of innovation and trends in diffusion and quality and in incidence of disease, none of which are currently available. Given these gaps in knowledge, between-country differences in levels of mortality from amenable conditions should not be used for routine surveillance of healthcare performance. The timing and pace of mortality decline from amenable conditions may provide better indicators of healthcare performance.

Treatment options for lysosomal storage disorders: developing insights.

Introduction: Lysosomal storage disorders (LSDs) are clinically heterogeneous disorders that result primarily from lysosomal accumulation of macromolecules in various tissues. LSDs are always progressive, and often lead to severe symptoms and premature death. The identification of the underlying genetic and enzymatic defects has prompted the development of various treatment options. Areas covered: To describe the current treatment options for LSDs, the authors provide a focused overview of their pathophysiology. They discuss the current applications and challenges of enzyme-replacement therapy, stem-cell therapy, gene therapy, chaperone therapy and substrate-reduction therapy, as well as future therapeutic prospects. Expert opinion: Over recent decades, considerable progress has been made in the treatment of LSDs and in the outcome of patients. None of the current options are completely curative yet. They are complicated by the difficulty in efficiently targeting all affected tissues (particularly the central nervous system), in reaching sufficiently high enzyme levels in the target tissues, and by their high costs. The pathways leading from the genetic mutation to the clinical symptoms should be further elucidated, as they might prompt the development of new and ultimately curative therapies.

Pain in adult patients with Pompe disease A cross-sectional survey

BACKGROUND Pompe disease is a rare hereditary metabolic myopathy caused by a deficiency of acid-α-glucosidase. We investigated the presence and severity of pain and its interference with daily activities in a large group of adults with Pompe disease, who we compared with an age-matched control group. METHODS Data were collected in a cross-sectional survey in Germany and The Netherlands. Pain was assessed using the short-form brief pain inventory (BPI). Patients also completed the Short Form-36 item (SF-36v2), the Hospital Anxiety and Depression Scale (HADS) and the Rotterdam Handicap Scale (RHS). RESULTS Forty-five percent of the 124 adult Pompe patients reported having had pain in the previous 24h, against 27% of the 111 controls (p=0.004). The median pain severity score in Pompe patients reporting pain was 3.1 (on a scale from 0 to 10), indicating mild pain; against 2.6 amongst controls (p=0.06). The median score of pain interference with daily activities in patients who reported pain was 3.3, against 1.3 in controls (p=0.001). Relative to patients without pain, those with pain had lower RHS scores (p=0.02), lower SF-36 Physical and Mental component summary scores (p<0.001 and p=0.049), and higher levels of depression and anxiety (p=0.005 and p=0.003). CONCLUSIONS To date, this is one of the largest studies on pain in a specific neuromuscular disorder. Nearly one in two Pompe patients had experienced pain in the previous 24h. Although pain severity and its interference with daily life were mild, pain was related to a reduced quality of life, less participation in daily life, and greater depression and anxiety. Its management should therefore be seen as part of clinical practice involving Pompe patients.

Innovations in health care and mortality trends from five cancers in seven European countries between 1970 and 2005

ObjectivesAlthough the contribution of health care to survival from cancer has been studied extensively, much less is known about its contribution to population health. We examine how medical innovations have influenced trends in cause-specific mortality at the national level.MethodsBased on literature reviews, we selected six innovations with proven effectiveness against cervical cancer, Hodgkin’s disease, breast cancer, testicular cancer, and leukaemia. With data on the timing of innovations and cause-specific mortality (1970–2005) from seven European countries we identified associations between innovations and favourable changes in mortality.ResultsFor none of the five specific cancers, sufficient evidence for an association between introduction of innovations and a positive change in mortality could be found. The highest association was found between the introduction of Tamoxifen and breast cancer mortality.ConclusionsThe lack of evidence of health care effectiveness may be due to gradual improvements in treatment, to effects limited to certain age groups or cancer subtypes, and to contemporaneous changes in cancer incidence. Research on the impact of health care innovations on population health is limited by unreliable data on their introduction.

Classic infantile Pompe patients approaching adulthood: a cohort study on consequences for the brain

To examine the long‐term consequences of glycogen storage in the central nervous system (CNS) for classic infantile Pompe disease using enzyme replacement therapy.

Innovations in medical care and mortality trends from four circulatory diseases between 1970 and 2005

BACKGROUND Governments have identified innovation in pharmaceuticals and medical technology as a priority for health policy. Although the contribution of medical care to health has been studied extensively in clinical settings, much less is known about its contribution to population health. We examine how innovations in the management of four circulatory disorders have influenced trends in cause-specific mortality at the population level. METHODS Based on literature reviews, we selected six medical innovations with proven effectiveness against hypertension, ischaemic heart disease, heart failure and cerebrovascular disease. We combined data on the timing of these innovations and cause-specific mortality trends (1970-2005) from seven European countries. We sought to identify associations between the introduction of innovations and favourable changes in mortality, using Joinpoint-models based on linear spline regression. RESULTS For both ischaemic heart disease and cerebrovascular disease, the timing of medical innovations was associated with improved mortality in four out of five countries and five out of seven countries, respectively, depending on the innovation. This suggests that innovation has impacted positively on mortality at the population level. For hypertension and heart failure, such associations could not be identified. CONCLUSION Although improvements in cause-specific mortality coincide with the introduction of some innovations, this is not invariably true. This is likely to reflect the incremental effects of many interventions, the time taken for them to be adopted fully and the presence of contemporaneous changes in disease incidence. Research on the impact of medical innovations on population health is limited by unreliable data on their introduction.

Antibody formation to enzyme therapy in classic infantile Pompe disease: implications of patient age

Enzyme-replacement therapy (ERT) with alglucosidase alfa has improved the lifespan of patients with classic infantile Pompe disease, although ERT is not effective in a subset of patients who mount an immune response to the exogenous enzyme. We studied the development of antibodies in response to ERT and its effect on clinical outcomes in 11 patients with classic infantile Pompe disease treated with ERT since 1999 for a median of 4.2 years (range 3 months to 12 years).

Impact of enzyme replacement therapy on survival in adults with Pompe disease

Background Since 2006, enzyme replacement therapy (ERT) has been available as a treatment for patients with Pompe disease. ERT has shown efficacy concerning muscle strength and pulmonary function in adult patients. However, no data on the effect of ERT on the survival of adult patients are currently available. Our objective was to assess the effect of ERT on survival in adult patients with Pompe disease.

The timing of introduction of pharmaceutical innovations in seven European countries

Rationale, aims and objectives Differences in the performance of medical care may be due to variation in the introduction and diffusion of medical innovations. The objective of this paper is to compare seven European countries (United Kingdom, the Netherlands, West Germany, France, Spain, Estonia and Sweden) with regard to the year of introduction of six specific pharmaceutical innovations (antiretroviral drugs, cimetidine, tamoxifen, cisplatin, oxalaplatin and cyclosporin) that may have had important population health impacts. Methods We collected information on introduction and further diffusion of drugs using searches in the national and international literature, and questionnaires to national informants. We combined various sources of information, both official years of registration and other indicators of introduction (clinical trials, guidelines, evaluation reports, sales statistics). Results and conclusions The total length of the period between first and last introduction varied between 8 years for antiretroviral drugs and 22 years for cisplatin. Introduction in Estonia was generally delayed until the 1990s. The average time lags were smallest in France (2.2 years), United Kingdom (2.8 years) and the Netherlands (3.5 years). Similar rank orders were seen for year of registration suggesting that introduction lags are not only explained by differences in the process of registration. We discuss possible reasons for these between-country differences and implications for the evaluation of medical care.