Quality of life in home-ventilated children and their families

González, Rafael; Bustinza, Amaya; Fernández, Sarah N.; García, Miriam; Rodríguez, Silvia González; Teresa, María; Gáboli, Mirella; García, S. Beltrán; Sardón, Olaia; García, D. López; Salcedo, Antonio; Rodríguez, Antonio Galán; Luna, Ma Carmen; Hernández, Arturo; González, Catalina; Medina, Alberto; Pérez, Estela; Callejón, Alicia Callejón; Toledo, Juan Diego; Herranz, Mercedes; López‐Herce, Jesús

doi:10.1007/s00431-017-2983-z

Cited by 52 publications

(113 citation statements)

References 41 publications

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“…This study found that children on HMV had lower HRQoL compared to healthy controls, in most domains. These findings are consistent with a recent Spanish study of 41 children (mean age 8.2 years) whose global scores in the PedsQL questionnaire were below those of published normative data for healthy children 8 . In a Canadian study, which also used the HUI2/3 questionnaires, HRQoL of children on HMV was lower than published literature for healthy children 27 …”

Section: Discussionsupporting

confidence: 89%

Health‐related quality of life and developmental outcome of children on home mechanical ventilation in a developing country: A cross‐sectional study

Tan

Nathan

Jayanath

et al. 2020

Pediatric Pulmonology

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Background Provision of home mechanical ventilation (HMV) to children with chronic respiratory insufficiency enhances growth and quality of life. The hypothesis was that health‐related quality of life (HRQoL) and the development of these children were poorer than in healthy children. Objectives To determine the HRQoL and developmental outcome of children on HMV. Methods This cross‐sectional study used the TNO‐AZL Preschool children's Quality Of Life (TAPQOL; <5 years old) and Health Utilities Index (HUI) 2/3 (≥5 years old) to assess the quality of life and the Schedule of Growing Skills‐II to assess development. Instruments were used on children currently or previously on HMV (≥3 months) and compared with age and sex‐matched controls. Results Sixty‐five patients and 130 controls were recruited. Patients' median (interquartile range) age was 3.12 (1.65, 5.81) years. Patients had significantly lower TAPQOL scores in the domains of lung, liveliness, positive mood, social functioning, motor functioning, and communication, and lower HUI 2/3 scores in hearing, sensation, pain, speech, mobility, ambulatory, dexterity, and self‐care domains. The developmental outcome of patients was poorer in all domains. However, patients had fewer behavioral problems. Those with respiratory tract disease and without comorbidities had better HRQoL and developmental scores. Having a parent as the primary caregiver was associated with better speech and language skills. Conclusions HRQoL and the developmental outcome are lower in children on HMV compared to controls. Children with respiratory tract disease and without comorbidities have a better outcome. Parents play a crucial role in the acquisition of speech.

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Section: Discussionsupporting

confidence: 89%

Health‐related quality of life and developmental outcome of children on home mechanical ventilation in a developing country: A cross‐sectional study

Tan

Nathan

Jayanath

et al. 2020

Pediatric Pulmonology

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“…The high proportion of patients on ventilator support ≥16 hours per day (ie, permanent ventilation) further underscores the morbidity of this severe neuromuscular disease. Children requiring this level of support are unlikely to have intrinsic capacity to wean and would die without such interventions21; in addition, they and their families face substantial physical, emotional and financial demands associated with their need for mechanical ventilation 11–13. One cannot discount the possibility that some patients felt likely to do better by their managing care teams might have been more likely to be offered intervention.…”

Section: Discussionmentioning

confidence: 99%

“…Most who survive infancy require mechanical ventilation and supplementary feeding, rely on wheelchairs for mobility, undergo multiple surgeries in their first year of life and die in early childhood 9 10. Those who survive face substantial physical, emotional and financial demands related to their reliance on life-sustaining ventilatory and other forms of support 11–13…”

Section: Introductionmentioning

confidence: 99%

Mortality and respiratory support in X-linked myotubular myopathy: a RECENSUS retrospective analysis

et al. 2019

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Purpose Individuals with X-linked myotubular myopathy (XLMTM) who survive infancy require extensive supportive care, including ventilator assistance, wheelchairs and feeding tubes. Half die before 18 months of age. We explored respiratory support and associated mortality risk in RECENSUS, particularly among patients ≤5 years old who received respiratory support at birth; this subgroup closely matches patients in the ASPIRO trial of gene therapy for XLMTM. Design RECENSUS is an international, retrospective study of patients with XLMTM. Descriptive and timeto-event analyses examined survival on the basis of age, respiratory support, tracheostomy use, predicted mutational effects and life-sustaining care. Results Outcomes for 145 patients were evaluated. Among 126 patients with respiratory support at birth, mortality was 47% overall and 59% among those ≤5 years old. Median survival time was shorter for patients ≤5 years old than for those >5 years old (2.2 years (IQR 0.7-5.6) vs 30.2 years (IQR 19.4-30.2)). The most common cause of death was respiratory failure (66.7%). Median survival time was longer for patients with a tracheostomy than for those without (22.8 years (IQR 8.7-30.2) vs 1.8 years (IQR 0.2-not estimable)). The proportion of patients living without a tracheostomy was 50% at age 6 months and 28% at age 2 years. Median survival time was longer with provision of life-sustaining care than without (19.4 years (IQR 3.1-not estimable) vs 0.2 years (IQR 0.1-2.1)). Conclusions High mortality, principally due to respiratory failure, among patients with XLMTM ≤5 years old despite respiratory support underscores the need for early diagnosis, informed decision-making and diseasemodifying therapies. Trial registration number NCT02231697

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“…In total, 43 families of children with neuromuscular disease treated in the University medical center Hamburg-Eppendorf and the Children’s Hospital Altona were evaluated with standardized questionnaires and compared to a cohort of healthy children and children with other chronic diseases, respectively. In view of previous reports on health-related quality of life in ventilated patients [ 10 , 12 , 14 , 16 ] we propose that long-term ventilation is one factor that impairs quality of life of affected children and their families due to an increase in disease burden.…”

Section: Introductionmentioning

confidence: 84%

The impact of long-term ventilator-use on health-related quality of life and the mental health of children with neuromuscular diseases and their families: need for a revised perspective?

Johannsen

Fuhrmann

Grolle

et al. 2020

Health Qual Life Outcomes

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Background Life extension by medical interventions and health-related quality of life (HRQOL) are sometimes conflicting aspects of medical care. Long-term ventilation in children with neuromuscular disease is a well-established life-extending procedure and often at the center of this conflict. HRQOL and the mental health of affected children and their families become even more important in respect to emerging therapies in neuromuscular diseases with longer life-expectancy of treated patients and considerable costs of medical treatment. Methods We performed a questionnaire survey in a total of forty-three families of children with neuromuscular disease treated in the University Medical Center Hamburg-Eppendorf and the Children’s Hospital Altona. We evaluated self- and proxy-reported HRQOL and mental health outcomes of affected children and their parents using validated and age-appropriate instruments. Results Compared to normative data, children with neuromuscular diseases and their families experienced a lower HRQOL and mental health. However, there was no additional negative influence on the overall HRQOL by ventilator use. Conclusions As ventilator use was not responsible for the reduction of HRQOL and mental health our data contributes an important aspect to the discussion about life-prolonging procedures, in particular mechanical ventilation, in severly disabled patients.

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Quality of life in home-ventilated children and their families

Cited by 52 publications

References 41 publications

Health‐related quality of life and developmental outcome of children on home mechanical ventilation in a developing country: A cross‐sectional study

Health‐related quality of life and developmental outcome of children on home mechanical ventilation in a developing country: A cross‐sectional study

Mortality and respiratory support in X-linked myotubular myopathy: a RECENSUS retrospective analysis

The impact of long-term ventilator-use on health-related quality of life and the mental health of children with neuromuscular diseases and their families: need for a revised perspective?

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