Evaluation of quality of life in children with heart disease
Introduction
Ultimately, the goals of pediatric research and clinical care are to maximize health and minimize symptomatology, disability, and dysfunction that may impact the lives of children with acute and chronic disease processes. Over the last several decades, new surgical techniques and advances in cardiopulmonary bypass, intensive care, interventional cardiac catheterization, non-invasive imaging, and medical therapies have significantly lowered neonatal mortality rates for children with the most complex congenital heart disease (CHD) (e.g. hypoplastic left heart disease) to less than 10% [1]. In addition, cardiac-related mortality in patients with congenital and acquired heart disease (HD) has diminished significantly during the first two decades of life [2], [3]. Although survival rates vary by disease complexity, long-term survival (> 20 years) rates for children with HD are estimated to be 95% for simple CHD, 90% for moderate CHD, and 80% for complex CHD in the current era [3].
Despite these advances, however, survivors suffer from morbidity resulting from their circulatory abnormalities and the medical and surgical therapies they have received. These morbidities significantly impact the child's neurodevelopmental [4], [5], [6], psychosocial [7], [8], [9], and physical [10], [11], [12] functioning and diminish their QOL (Fig. 1). Given the high incidence of functional impairment in the pediatric cardiac population, there has been a paradigm shift in clinical research from short-term mortality prevention to long-term morbidity assessment. As a result, outcome assessment focusing on QOL has become increasingly important in this high risk population.
QOL may be described as a child's ability to function in situational contexts (family, school, peer) and derive personal satisfaction from doing so [13], [14], [15]. The construct of QOL is thought to include three essential domains: physical health status and physical functioning; psychological status, and social functioning (Fig. 2) [13], [14], [15]. QOL measurement provides a comprehensive description of an individual's health, may result in the identification of physical, functional, and psychosocial dysfunction, and is a critical component of the evaluation of long-term outcomes of chronic conditions and disease-specific therapies.
This paper will delineate health measurement definitions including QOL and health-related QOL (HRQOL), identify inherent difficulties in HRQOL measurement in the pediatric HD population, discuss salient aspects of HRQOL instrument evaluation In addition, this manuscript will describe existing generic and disease-specific HRQOL measures that may be used to assess HRQOL in the pediatric HD population, what research on HRQOL in the pediatric HD population has shown, and the extent to which HRQOL evaluations are being fully utilized in clinical practice. Finally, a research and clinical agenda are proposed to harness the potential applications of HRQOL assessment.
Section snippets
Health measurement definitions: QOL and HRQOL
There are many ways to define “health”. While the differences in meaning between various attributes of health may be subtle, the differences are important and have significant implications as to how research is interpreted and findings incorporated into how we care for children with chronic disease. Health has been defined by the World Health Organization as “a state of complete physical, mental, and social well-being and not merely the absence of disease or infirmity” [16]. Indeed it was this
Inherent difficulties of HRQOL evaluation in the pediatric HD population
HRQOL assessment in the pediatric population is challenging due to the wide age range and the changing developmental capabilities of the patients as they age. HRQOL assessment in the pediatric cardiac population is further complicated by the variety of congenital and acquired diseases, varying levels of severity, the array of therapeutic modalities that may be utilized to treat the patient (medical, surgical, and interventional), and the spectrum of outcomes. Similar to other pediatric chronic
HRQOL instrument evaluation
When measuring HRQOL it is important to be clear on whether the goal of the application is to assess HRQOL or functional status or both. These measures of health are distinct constructs and are often confused with each other; note prior definitions. When assessing either HRQOL or functional status for research and clinical application, specific aspects of instrument selection (Table 1, Table 2), validation (Table 3), and availability (Table 4) must be considered.
When selecting an instrument it
Existing generic and disease-specific HRQOL measures that may be used to assess HRQOL in the pediatric HD population
Both the PedsQL 4.0 Cores scales, a generic HRQOL measure, and the Child Health Questionnaire (CHQ), a generic functional status measure, have patient and proxy reporting, wide age range, and may be administered in a reasonable time-frame. In addition, these commercially available tools have been shown to be reliable and internally and externally valid, and responsive in the United States and many other countries after language translation (Table 1, Table 2, Table 3, Table 4) [27], [28]. Five
HRQOL studies in the general CHD population
Most early outcome studies in patients with CHD described mortality and morbidity or health status, including anatomic or hemodynamic outcome, electrophysiologic sequelae, and/or exercise capacity following surgical interventions or included “quality of life” parameters such as marital status, number of offspring, employment status, or educational attainment in adults with CHD. More recent studies have recognized the multidimensional nature of HRQOL and have included not only physical health
Clinical implementation (Is HRQOL evaluation utilized in clinical practice?)
The importance and utility of HRQOL assessment with reliable, valid, generalizable, and responsive instruments in the pediatric HD population are centered on the fact that they provide a means to improve patient HRQOL outcomes through: 1) The improvement of comprehensive follow-up of the pediatric HD population (surveillance and screening); 2) The identification of at-risk patients (risk stratification of HD sub-populations); 3) The identification of modifiable risk factors to prevent adverse
Research agenda (What is needed in HRQOL research in pediatric HD patients?)
- 1.
Future HRQOL research should focus on the associations of specific morbidities/phenotypes in the pediatric HD population and HRQOL to determine candidate factors for interventional studies for prevention and treatment. Specifically, further research is needed to discover links between neurodevelopmental, psychosocial, and physical morbidity factors and HRQOL to identify specific functional deficits that may be prevented or mitigated through intervention.
- 2.
Both patient and parent respondents need
Clinical agenda (What is needed to harness the potential of HRQOL assessment for clinical use in pediatric HD patients?)
- 1.
Begin incorporating HRQOL evaluation into the clinic visit to take full advantage of the current advances in HRQOL measurement.
- 2.
Strive to make all HRQOL evaluations conducted in clinical settings “research quality” (or at least create standard evaluation protocols) so that the field may benefit from reliable, valid, and potentially generalizable clinical information. There is so much variability in the way evaluations are presently conducted that it makes it difficult to generalize any data
Conclusion
Over the last several decades mortality rates for children with HD have fallen. However, survivors may have neurodevelopmental, psychosocial, and physical morbidities that lower HRQOL. Although HRQOL assessment in this high risk population has been lacking due to inherent issues in HRQOL assessment in the pediatric HD population, advances have been made in HRQOL measurement with new reliable, valid, and generalizable measures. These questionnaires may be utilized to rapidly improve HRQOL
Financial disclosure/conflicts of interest
There are no financial relationships or conflicts of interest to disclose relevant to this manuscript.
Acknowledgement
We would like to thank Shawna Hottinger, MS for her contributions to the initial outline and tables for this manuscript.
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