Feasibility and impact of a school-based intervention for families of urban adolescents with asthma: results from a randomized pilot trial

The purpose of this study was to test the feasibility and short-term outcomes of Asthma: It's a Family Affair!, a school-based intervention for adolescents with asthma and their caregivers. Twenty-four ethnic minority families with a middle school student with asthma were randomized to immediate intervention or no-treatment control. Intervention students received six group sessions on prevention and management of asthma. Caregivers received five group sessions teaching child-rearing skills to support the youth's autonomy and asthma self-management. All students attended all sessions; caregivers attended an average of three. Two months post-intervention, relative to controls, intervention caregivers reported better problem-solving with children. Intervention students were more responsible for carrying medication, took more prevention steps, and woke fewer nights from asthma. The intervention resulted in positive short-term changes in family relations, asthma management by students, and health statu

Cognitive-Behavioral Therapy and Eye Movement Desensitization and Reprocessing in an Adolescent with Difficult-to-Control Asthma

Stress and distress have been suggested to prolong difficult-to-control asthma (DTCA), that is, asthma that is not under control despite optimal medical treatment. A pediatric pulmonologist referred a 16-year-old girl with DTCA in whom asthma-specific fear induced by disturbed memories and distorted cognitions following frightening asthma attacks were driving asthma exacerbations. We examined whether cognitive-behavioral therapy (CBT) and eye movement desensitization and reprocessing (EMDR) focusing on asthma-specific fear and disturbed memories could reduce asthma symptoms and its burden. The single-case experimental design included 48 weekly assessments of primary outcomes during all phases, and 4 assessments of secondary outcomes at intake, pretherapy, posttherapy, and follow-up. Analysis of the time series data with a piecewise regression model demonstrated that the level or slope (trend) showed an improvement during the intervention and a sustained improvement during follow-up on all primary outcomes: burden of asthma exacerbations, physical activities, social activities, physical complaints, and worrying. Analyses using the Reliable Change Index showed significant pretherapy to posttherapy changes on most domains of questionnaires measuring secondary outcomes: Asthma Control Test, Pediatric Asthma Quality of Life Questionnaire, Child Behavior Checklist, and Youth Self-Report. Moreover, use of rescue medication went down and lung function (FEV1) came just into the normal range at follow-up. The study showed that asthma symptoms and the burden of asthma were reduced after CBT and EMDR. This proof-of-principle study suggests that DTCA may improve by psychological interventions in pediatric patients with psychological stress or distres

Cognitive development in patients with Mucopolysaccharidosis type III (Sanfilippo syndrome)

Mucopolysaccharidosis type III (MPS III, Sanfilippo syndrome) is a lysosomal storage disorder caused by a deficiency of one of the enzymes involved in the degradation of heparan sulfate. MPS III is characterized by progressive mental deterioration resulting in severe dementia. A number of potentially disease-modifying therapies are studied. As preservation of cognitive function is the ultimate goal of treatment, assessment of cognitive development will be essential in order to evaluate treatment efficacy. However, no large scale studies on cognitive levels in MPS III patients, using formal psychometric tests, have been reported. We aimed to assess cognitive development in all 73 living patients with MPS III in the Netherlands. Cognitive development could be assessed in 69 patients. In 39 of them developmental level was estimated > 3 months and formal psychometric testing was attempted. A remarkable variation in the intellectual disability was detected. Despite special challenges encountered, testing failed in only three patients. The observed broad variation in intellectual disability, should be taken into account when designing therapeutic trial

Cognitive development in patients with Mucopolysaccharidosis type III (Sanfilippo syndrome)

Mucopolysaccharidosis type III (MPS III, Sanfilippo syndrome) is a lysosomal storage disorder caused by a deficiency of one of the enzymes involved in the degradation of heparan sulfate. MPS III is characterized by progressive mental deterioration resulting in severe dementia. A number of potentially disease-modifying therapies are studied. As preservation of cognitive function is the ultimate goal of treatment, assessment of cognitive development will be essential in order to evaluate treatment efficacy. However, no large scale studies on cognitive levels in MPS III patients, using formal psychometric tests, have been reported. We aimed to assess cognitive development in all 73 living patients with MPS III in the Netherlands. Cognitive development could be assessed in 69 patients. In 39 of them developmental level was estimated > 3 months and formal psychometric testing was attempted. A remarkable variation in the intellectual disability was detected. Despite special challenges encountered, testing failed in only three patients. The observed broad variation in intellectual disability, should be taken into account when designing therapeutic trial

Effects of a short self-management intervention for patients with asthma and diabetes: Evaluating health-related quality of life using then-test methodology

Despite intensive outpatient care and optimized medical treatment, some patients with chronic illnesses experience prolonged difficulties in the self-management of their illness and fail to attain optimal disease control. The present study describes the effects of an intervention programme aimed at improving self-management and quality of life among patients with asthma and diabetes. The intervention was based on insights from self-regulation theory and proactive coping theory. Patients with asthma ( N = 70) and diabetes ( N = 55) were randomly assigned to an intervention group or control group ( standard care). In addition to a conventional pre-test-post-test-follow-up design, the then-test methodology was employed to examine the effects on quality of life. The present study showed no intervention effects on self-efficacy, self-care activities or proactive coping. The same was true for quality of life when measured with the conventional design. However, positive intervention effects for asthma patients were found on global quality of life and physical health when measured with the then-test procedure. These results accentuate the value of including then-tests when measuring intervention outcomes. Additional subgroup analyses showed that patients who scored relatively high o