Integrated care of muscular dystrophies in Italy. Part 2. Psychological treatments, social and welfare support, and financial costs.

This paper describes the psycho-social treatments received by 502 patients with MDs and their relatives, and the costs for care sustained by the families in the previous six month period. Data were collected by the MD-Care Schedule (MD-CS) and the Family Problems Questionnaire (FPQ). Psycho-educational interventions were provided to 72 patients (14.3%), and social/welfare support to 331 patients (65.9%). Social/welfare support was higher in patients with DMD or LGMD, in those showing more severe disability, and in patients who were in contact with centres located in Northern Italy. Psycho-educational interventions were received by 156 (31%) relatives, and social/welfare support by 55 (10.9%) and mainly provided by Family/Patients Associations (83.6%). Relatives with higher educational levels, who spent more daily hours in the assistance of patients with DMD, and in contact with centres in Central Italy more frequently benefited from psycho-educational interventions. In the previous year, costs for care were sustained by 314 (63.9%) relatives. Financial difficulties related to patient's condition, were higher in families of patients who needed more intensive rehabilitation and daily hours of caregiving, and in families who lived further away from the reference's centre. These results showed that psycho-social aspects of MDs care are only partially met in Italy, and that ad hoc supportive interventions for these patients and their families should be potentiated.

Burden, professional support, and social network in families of children and young adults with muscular dystrophies.

INTRODUCTION: This study explores burden and social and professional support in families of young patients with muscular dystrophies (MDs) in Italy. METHODS: The study was carried out on 502 key relatives of 4- to 25-year-old patients suffering from Duchenne, Becker, or Limb-Girdle MD who were living with at least 1 adult relative. RESULTS: A total of 77.1% of relatives reported feelings of loss, 74.0% had feelings of sadness, and 59.1% had constraints in leisure activities. Burden was higher among relatives of patients with higher disability and who spent more daily hours in caregiving. Practical difficulties were higher among relatives who perceived lower help in patient emergencies and less practical support by their social network. Psychological burden was higher in those relatives who were unemployed, those with poorer support in emergencies, and those with lower social contacts. CONCLUSIONS: Caring for patients with MDs may be demanding for relatives even in the early stages of these disorders, especially when social support is poor and the patient's disability increases.

A national profile of health care and family impacts of children with muscular dystrophy and special health care needs in the United States.

We used the 2005-2006 National Survey of Children with Special Health Care Needs to compare 3 types of outcomes between children with and those without parental reported muscular dystrophy: (1) functional limitations; (2) health care experiences in terms of the 5 components of a medical home; and (3) family impacts, including financial or out-of-pocket costs and parental employment and time use. We used weighted logistic regression to examine their associations with muscular dystrophy after adjustment for socio-demographic characteristics. Among children with special health care needs, children with reported muscular dystrophy were much more likely to have difficulties with ambulation and self-care. They were more likely to have family members who reported financial problems, reduced or stopped employment, and spent more than 10 hours weekly providing or coordinating care. Muscular dystrophy was not associated with the likelihood of having a medical home after adjustment for socioeconomic status and other socio-demographic characteristics.

The struggle against neuromuscular diseases in France and the emergence of the "partnership model" of patient organisation.

The past few decades have witnessed the increasingly active participation of patient organisations in research activities concerning them. They contribute substantially to the funding of scientific and clinical research. More importantly, certain patient organisations take strategic decisions concerning that research, and contribute to the production of knowledge on their diseases. In France, the AFM (Association Française contre les Myopathies-French Muscular Dystrophy Organisation), is a striking illustration. The paper argues that the model of the AFM's engagement in research-the "partnership model"-is original insofar as it renews the power relations between patients and professionals found in two classic models: the "auxiliary model" and the "emancipatory model". Based on a long-term study of the French Muscular Dystrophy Organisation, this "partnership model" is characterised and its implications discussed in three respects: the possible generalisation of the mode of relations it establishes between patients and professionals; its effects on the steering of research; and its consequences for the dynamics of patient organisations movements.

Expenditures for care of children with chronic illnesses enrolled in the Washington State Medicaid program, fiscal year 1993.

OBJECTIVE: We calculated expenditures for children with one of eight selected chronic health conditions who were enrolled in the Washington State Medicaid program and compared them with payments for all Medicaid-enrolled children. We examined variation in mean, median, and total expenditures and identified expenditure sources. METHODS: This study analyzed Medicaid claims data for 310 977 children aged 0 to 18 who were enrolled at any time in fiscal year 1993. Tracer conditions were used to examine expenditure variation within and between diagnostic groupings. A total of 18 233 children (5.9%) had at least one of the conditions. Expenditures were calculated based on payments made by the Medicaid program. RESULTS: Children with one of the eight selected conditions incurred mean expenditures of $3800, compared with $955 for all Medicaid-enrolled children. Mean payments associated with the selected conditions ranged from 2.5 times to 20 times more than payments to all children. Approximately 10% of children accounted for approximately 70% of the payments in general and in each diagnostic grouping. Variation in mean, median, and total expenditures was extensive among the conditions. For most conditions, inpatient stays accounted for the greatest proportion of expenditures; for some conditions, durable equipment, home nursing, and medication-related services accounted for substantial proportions of total expenditures. CONCLUSIONS: Medical care for children with selected chronic health conditions is 2.5 to 20 times more expensive than children in general, depending on the condition. A relatively few children account for the majority of expenditures. Extensive variation in mean, median, and total expenditures suggests that different conditions will need to be kept distinct for purposes of establishing payment rates. Children with certain conditions are vulnerable to restrictions in specific services, depending on what restrictions are imposed by a financing program. Further analyses are needed to identify risk-adjustment strategies to support delivery of high-quality services to this population of children as they migrate into managed-care environments.

Cost-effectiveness of neonatal screening for Duchenne muscular dystrophy--how does this compare to existing neonatal screening for metabolic disorders?

Costs of screening a series of 18,152 newborn males for Duchenne muscular dystrophy (DMD) in Canada were evaluated. The final aim of neonatal screening for DMD is the avoidance of additional cases in the families identified. Total costs to avoid one case of DMD were estimated at Cdn. $172,000, while the incremental costs were found to be $83,000. Reagent costs, test sensitivity, efficacy of screening and compliance with genetic advice were identified as factors crucial for cost-effectiveness. Costs of neonatal screening for DMD are compared with costs of neonatal screening for inborn metabolic disorders. It is found that the two programmes are similar in costs. Earlier predictions of inordinate costs of screening for DMD are refuted.