Outcomes of monthly video consultations as an add-on to regular care for children with type 1 diabetes: A 6-month quasi-randomized clinical trial followed by an extension phase.

OBJECTIVE: To explore the outcomes of monthly video consultations for children with diabetes. METHODS: The Virtual Outpatient Diabetes Clinic for Children and Youth (VIDIKI) was a 6-month multicenter controlled clinical trial followed by an extension phase. The 240 participants (1-16 years), all using a CGM, were quasi-randomized by residence location to the intervention group (IG) or the waitlist-control group (WG). The IG started immediately after enrollment with monthly video consultations as an add-on to regular care, while the WG received regular care for 6 months before starting the intervention. The extension phase lasted between 12 months and 2 years, depending on the enrollment date. Linear regression was applied to model the primary outcome of HbA1c after 6 months and other metabolic and psychosocial outcomes. RESULTS: After covariate adjustments, the HbA1c at 6 months-corresponding to the controlled treatment phase-was 0.11% lower in the IG than that in the WG (95% CI -0.31 to 0.09, P = .277). For the total study sample, a significant HbA1c improvement was found after 12 months of video consultations, which increased further until month 15. The diabetes burden of the main caregivers was lower, and parental treatment satisfaction was significantly higher in the IG than that in the WG. CONCLUSIONS: The VIDIKI study found no significant HbA1c difference between IG and WG after 6 months in the controlled phase, but there was a decreased diabetes burden and increased treatment satisfaction for the parents. In the longitudinal perspective, a significant HbA1c improvement was found after 12 and 15 months.

Psychosocial benefits of insulin pump therapy in children with diabetes type 1 and their families: The pumpkin multicenter randomized controlled trial.

OBJECTIVE: Continuous subcutaneous insulin infusion (CSII) is on the rise among pediatric patients with type 1 diabetes mellitus. Metabolic effects alone cannot explain this rising popularity. From the patient's perspective, the main benefits of CSII may be found in subjective psychosocial health outcomes (patient-reported outcomes [PRO]). SUBJECTS AND METHODS: In a multicenter open randomized controlled trial, children and adolescents aged 6 to16 years currently treated with multiple daily injections (MDI) were randomized 1:1, stratified by center, to either starting with CSII immediately after the baseline interview or to continuing MDI while waiting 6 months for transmission to CSII. The primary outcomes were patient-reported diabetes-specific health-related quality of life (DHRQOL) and diabetes burden of the main caregiver. Secondary outcomes were caregiver stress, fear of hypoglycemia, satisfaction with treatment, and HbA1c. RESULTS: Two-hundred and eleven patients were randomized between February 2011 and October 2014, and 186 caregivers and 170 patients were analyzed using the intention-to-treat principle for primary outcomes. Children 8 to 11 years in the CSII group reported improved DHRQOL at follow-up compared to MDI (median difference [MD] 9.5, 95% confidence interval [CI] 3.6-16.7, P = 0.004). There were no treatment differences in the adolescent age-group 12 to 16 years (MD 2.7; 95% CI -3.2-9.5; P = 0.353). The main caregivers of the CSII group reported a significant decline of overall diabetes burden at follow-up compared to the MDI group (MD 0; 95% CI -1-0; P = 0.029). Secondary PROs also were in favor of CSII. CONCLUSIONS: CSII has substantial psychosocial benefits. PROs demonstrate these benefits. Registered as NCT01338922 at clinicaltrials.gov.

Multicentre cross-sectional clinical evaluation study about quality of life in adults with disorders/differences of sex development (DSD) compared to country specific reference populations (dsd-LIFE).

BACKGROUND: Previous studies in quality of life (QOL) in individuals with disorders/differences of sex development (DSD) have been restricted to subpopulations of the condition. We describe QOL in adult persons with DSD compared to country specific references and assess the impact of diagnosis. METHODS: The multicentre cross-sectional clinical evaluation (dsd-LIFE) took place in 14 specialized clinics in six European countries. Adolescents (≥16 years) and adults having a DSD condition were included from 02/2014 to 09/2015. The main outcome QOL was measured by the WHOQOL-BREF (domains of physical health, psychological, social relationships, and environment). QOL was compared to country specific reference populations by using unpaired t-tests. Linear regression models explained the additional variance of the diagnosis on QOL. RESULTS: Three hundred one individuals with Turner Syndrome, 219 with Klinefelter Syndrome (including XYY), 226 with 46,XX CAH and 294 with rare DSD conditions (gonadal dysgenesis, androgen insensitivity syndrome, severe hypospadias, and androgen synthesis errors or other diagnosis) took part. Compared to healthy European populations, QOL was similar in psychological, slightly worse in physical health, and slightly better in environment. In social relationships, QOL was significantly poorer compared to healthy and non-healthy reference populations. In linear regression models health status was the most important predictor of QOL; additional variance was explained by feelings about household's income in all domains, and the relationship status in social relationships. Diagnosis explained nearly no additional variance. CONCLUSIONS: Except for social relationships, most people with DSD adapt well to their life circumstances and report a good QOL. Not diagnosis, but the individual's health status is much more important than previously thought. Therefore care for people with DSD should focus more on chronic physical or mental health problems both related and unrelated to the diagnosis itself. TRIAL REGISTRATION: German Clinical Trials Register DRKS00006072 .

Health-Related Quality of Life in Children and Adolescents with Cerebral Palsy: A Secondary Analysis of the DISABKIDS Questionnaire in the Field-Study Cerebral Palsy Subgroup.

INTRODUCTION: Health-related quality of life (HRQOL) instruments are increasingly being used to evaluate interventions and therapy outcomes in children and adolescents with cerebral palsy (CP). A variety of psychometrically sound and validated generic and disease-specific instruments are available. A third type of instrument, the chronic-generic instrument, pertains to features of HRQOL that are shared by various chronic conditions.The DISABKIDS family of questionnaires consists of a chronic-generic core measure (DCGM-37) and several condition-specific modules, among these, a CP module (CPM). The objective of this article was to describe the performance and, specifically, the validity of the DCGM-37 and CPM in children and adolescents with CP. METHODS: Psychometric properties of the DCGM-37 and the CPM are presented. The discriminant validity was assessed compared with generic measures of HRQOL regarding different levels of impairment (physical independence; developmental delay). RESULTS: A total of 86 patients with CP (mean age 13 years, range 7-19 years) and 78 main caretakers participated in this study. The DCGM-37 and CPM showed much better discriminative ability as compared with generic questionnaires. CONCLUSIONS: The DCGM-37 and CPM were able to differentiate between patients with different levels of impairment and can be recommended for treatment evaluation and group comparison in clinical studies of children and adolescents with CP.

Cross-cultural development of a child health care questionnaire on satisfaction, utilization, and needs.

OBJECTIVE: The instrument Child Health Care-Satisfaction, Utilization and Needs (CHC-SUN) has been developed cross-culturally to evaluate pediatric health care services for children with special health care needs (CSHCN) from the proxy perspective of parents. METHODS: The children of the participating parents received treatment in pediatric specialty clinics in 7 European countries for asthma, cystic fibrosis, diabetes, epilepsy, cerebral palsy, rheumatoid arthritis, and atopic dermatitis. The instrument was developed through a process including literature review, expert consensus, and item generation through focus groups. The pilot instrument was extensively tested to assess psychometric properties, support item reduction, and evaluate clinical validity. The final field version was tested in a new sample of 795 parents in 7 countries. RESULTS: Pilot testing and item reduction resulted in a practical 40-item instrument with 14 single items related to provision of services (module 1), and 26 items related to 6 scales on satisfaction with care (module 2), and 1 item on general satisfaction with care. Satisfaction with care scales showed very good psychometric properties, both initially and in the field version, with Cronbach's alpha ranging between .80 and .95 in the revised scales. Both modules discriminated well between functional status and diagnosis across countries. CONCLUSIONS: A new instrument is available to measure provision of services and satisfaction with care for children with chronic conditions from the perspective of parents. Cross-cultural analysis reveals that the measure is useful in multinational studies evaluating health services against the background of different health systems.