The Minneapolis-Manchester Quality of Life instrument: Reliability and validity of the Youth Form

OBJECTIVE: Improvements in survival after childhood cancer have increased emphasis on health-related quality of life (HRQoL) of survivors. We developed the Minneapolis-Manchester Quality of Life-Youth Form (MMQL-YF) as a standardized patient self-report instrument designed to assess HRQoL in childhood cancer survivors between the ages of 8 and 12 years. STUDY DESIGN: To validate the instrument, the MMQL-YF was administered to 643 children (481 healthy, 162 with cancer). Factor analysis was conducted to refine the instrument, and Cronbach's alpha coefficient was used to measure its internal reliability. Known-groups validity was determined by comparing healthy children with those with cancer. Construct validity was studied by a comparison of similar domains in the MMQL-YF and the Child Health Questionnaire (CHQ). Stability was tested by re-administration of the MMQL-YF 2 weeks later. RESULTS: Internal consistency reliability was in the acceptable range for this instrument. The MMQL-YF was able to distinguish between known groups, and its scales correlated highly with similar CHQ domains. Test-retest reliability showed that the instrument was extremely stable in all scales tested. CONCLUSION: Data provide evidence for the validity and reliability of the MMQL-YF as a comprehensive, multidimensional, self-report instrument for measuring HRQoL among childhood cancer survivors.     

Quality of life after growth hormone therapy and induced puberty in women with Turner syndrome

OBJECTIVE: To evaluate health-related quality of life (HRQoL) in young women with Turner syndrome (TS) after long-term growth hormone (GH) therapy and induced puberty and to analyze whether HRQoL was influenced by auxologic parameters, pubertal development, or subjective parameters. STUDY DESIGN: The study group comprised 49 women with TS, mean (standard deviation) age 19.6 (+/-3.0) years, all former participants of 2 GH studies, > or =6 months after GH discontinuation. Puberty was induced by estrogen treatment, at mean age 12.9 (+/-1.1) years. HRQoL was measured by self-reports of the 2 generic questionnaires, SF36 and TAAQOL. As an additional source of information on HRQoL, we applied parental proxy reports. RESULTS: HRQoL of the women with TS was normal. Remarkably, the women with TS had higher HRQoL scores on some of the scales, including "social functioning" and "role-emotional." Satisfaction with height and breast development had a positive influence on several HRQoL scales. CONCLUSIONS: The young women with TS who reached normal height and had age-appropriate pubertal development reported normal HRQoL. The relatively high scores on some of the HRQoL scales can be explained by an estrogen effect or by a possible response shift, indicating a different internal reference in women with TS. We hypothesize that GH and estrogen treatment positively influenced HRQoL in young women with TS.     

Clinical and cost implications of target joints in Canadian boys with severe hemophilia A

OBJECTIVE: To compare target joint-associated costs incurred by boys with severe hemophilia A 1 year before and 1 year after development of a target joint (pre-TJ, post-TJ). STUDY DESIGN: Resource utilization data were extracted retrospectively from medical and hemophilia clinic charts and patient diaries for 16 boys attending the Hospital for Sick Children (HSC)'s comprehensive care hemophilia program. Resources examined included drugs, medical care, hospitalization, laboratory tests, therapies, and transfusions received. All costs were figured using standard price lists and were discounted using an annual rate of 3%. RESULTS: Fifteen of the 16 boys developed at least one target joint, defined as three bleeds into any single joint within a consecutive 3-month period, at an average age of 54 months (range, 15-94 months), with ankles being most often affected, followed by elbows and knees (46% vs 28% and 23%, respectively). The total cost of treating a boy with on-demand Factor VIII (FVIII) increased by 119% after development of a target joint, from $20,091 (in 2002 Canadian dollars [$CDN]) in the year before to $43,890 in the year after target joint development. Factor VIII use accounted for 87% of the total cost in the year before target joint development and 93% in the year after. CONCLUSIONS: This study identified substantial increased costs of care associated with target joint development. This finding provides further support for more aggressive treatment aimed at reducing target joints-either more aggressive treatment of joint bleeds or institution of primary prophylactic therapy at an early age.     

Quality of life and psychological adjustment in children and adolescents with neurofibromatosis type 1

OBJECTIVE: To assess quality of life (QoL) and psychological adjustment in children and adolescents with neurofibromatosis type 1 (NF1). STUDY DESIGN: Forty-six patients with NF1 were investigated between the ages of 7 and 16 years (mean, 11.6 years), with children and parents used as informants. TNO-AZL Questionnaire for Children's Health-Related Quality of Life and Child Behavior Checklist scores were compared with healthy reference groups. Predictive values of sociodemographic variables, illness-related variables, and family-related variables for quality of life and psychological adjustment were assessed. RESULTS: Most dimensions of QoL in NF1 children and adolescents were different from reference values. Deviations in the NF1 group were an impairment of motor, cognitive, and social functioning and a reduction of positive and negative emotions. Also, psychological adjustment in patients with NF1 was significantly impaired compared with normal subjects. Illness-related variables had a negative impact on the emotional domain of QoL. Good family relationships positively affected both QoL and psychological adjustment. CONCLUSIONS: QoL and psychological adjustment are impaired in children and adolescents with NF1. Illness-related variables and the quality of family relationships are important predictors.     

Health-related quality of life and psychosocial adjustment in steroid-sensitive nephrotic syndrome

OBJECTIVES: To evaluate quality of life (QOL) and psychosocial adjustment by standardized tests in well-characterized patients with steroid-sensitive nephrotic syndrome. STUDY DESIGN: Forty-five patients were investigated at a median age of 9.8 years (range, 3.4 to 19.8) and 5.9 years (range, 0.1 to 16.3) since diagnosis. Child- and parent-rated QOL was evaluated by the The Netherlands Organization for Applied Scientific Research Academical Medical Center (TNO-AZL) Child Quality of life Questionnaire. Psychosocial adjustment was assessed by the Child Behavior Checklist and the Teacher Report Form. RESULTS: In patients, only the QOL subscale "social functioning" was impaired compared with that in control subjects, whereas parents rated 4 of a total of 7 subscales as abnormal. Psychosocial adjustment was impaired at home and at school. Steroid dependency and cytotoxic treatment had a significant negative impact on QOL only, whereas family climate, especially maternal distress, negatively affected both QOL and psychosocial adjustment. CONCLUSIONS: QOL and psychosocial adjustment are impaired in steroid-sensitive nephrotic syndrome. Both illness-related variables and family climate play an important role.     

The impact of realized access to care on health-related quality of life: a two-year prospective cohort study of children in the California State Children's Health Insurance Program

OBJECTIVE: To examine the effect of realized access to care (problems getting care, access to needed care) on health-related quality of life (HRQOL) in the California State Children's Health Insurance Program. STUDY DESIGN: This was a prospective cohort study (n = 4,925; 70.5% [3438] had complete data). Surveys were taken at enrollment and after 1 and 2 years in the program. Parents and children reported HRQOL (PedsQL 4.0 Generic Core Scales). Repeated-measures analysis accounted for within-person correlation and adjusted for baseline PedsQL, baseline realized access, race/ethnicity, language, chronic health condition, and having a regular physician. RESULTS: Realized access to care during the prior year was related to HRQOL for each subsequent year. Foregone care and problems getting care were associated with decrements of 3.5 (P < .001) and 4.5 (P < .001) points for parent proxy-report PedsQL and with decrements of 3.2 (P < .001) and 4.4 (P < .001) points for child self-report PedsQL. Improved realized access resulted in higher PedsQL scores, continued realized access resulted in sustained PedsQL scores, and foregone care resulted in cumulative declines in PedsQL scores. CONCLUSIONS: Realized access to care is associated with statistically significant and clinically meaningful changes in HRQOL in children enrolled in the California State Children's Health Insurance Program.     

Child and environmental factors associated with leisure participation in adolescents born extremely preterm

Background

Developmental impairments persist among adolescents born extremely preterm, and these individuals are at an increased risk for chronic disease later in life. Participating in active and positive leisure activities may act as a buffer against negative outcomes, but involvement in active-physical and skill-based activities is low in youth born preterm.

Aims

To explore the child and environmental determinants of leisure participation among adolescents born extremely preterm.

Study design

Cross-sectional study.

Subjects

Participants were recruited from the hospital's Neonatal Follow-Up Program and included 128 adolescents born preterm (mean gestational age: 26.5 weeks).

Outcome measures

Leisure participation was assessed using the Children's Assessment of Participation and Enjoyment. Potential determinants were assessed using standardized tests and questionnaires. Selected factors were entered into five separate multivariable regression models.

Results

Child and environmental factors contributed between 21% (skill-based) and 52% (active physical) of the adjusted variance for participation intensity. Lower gestational age was associated with greater participation in recreational activities. Male sex, higher maternal education and better motor competence were associated with involvement in active-physical activities. Being older and feeling socially accepted were associated with participation in social activities. Families oriented to hobbies and higher maternal education were associated with participation in skill-based activities. Preference was the strongest determinant of participation in all five leisure activities.

Conclusions

Activities should be adapted to individual skill level, include family and peers, foster social acceptance and be driven by the adolescent's preferences. Although certain factors cannot be modified, they can be used to identify adolescents at risk for low participation.     

The relationship of dystonia and choreoathetosis with activity,participation and quality of life in children and youth with dyskinetic cerebral palsy

Aim

To relate dystonia and choreoathetosis with activity, participation and quality of life (QOL) in children and youth with dyskinetic Cerebral Palsy (CP).