Evaluation of quality of life in children with heart disease

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Abstract

Due to recent advances in pediatric cardiovascular therapy, mortality rates for children with heart disease (HD) have decreased dramatically. 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, psychosocial, and physical functioning and diminish their quality of life (QOL). 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 and derive personal satisfaction from doing so. 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.

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.

References (56)

  • D.R. DeMaso et al.

    The longitudinal impact of psychological functioning, medical severity, and family functioning in pediatric heart transplantation

    J Heart Lung Transplant

    (2004)
  • J. Wray et al.

    Longitudinal assessment of psychological functioning in children after heart or heart–lung transplantation

    Journal of Heart and Lung Transplantation

    (2006)
  • J.S. Tweddell et al.

    Improved survival of patients undergoing palliation of hypoplastic left heart syndrome: lessons learned from 115 consecutive patients

    Circulation

    (2002)
  • W.T. Mahle et al.

    Survival after reconstructive surgery for hypoplastic left heart syndrome: a 15-year experience from a single institution

    Circulation

    (2000)
  • W.T. Mahle et al.

    Neurodevelopmental outcome and lifestyle assessment in school-aged and adolescent children with hypoplastic left heart syndrome

    Pediatrics

    (2000)
  • G. Wernovsky et al.

    Cognitive development after the Fontan operation

    Circulation

    (2000)
  • J. Wray et al.

    How does the intervention of cardiac surgery affect the self-perception of children with congenital heart disease?

    Child Care Health Dev

    (1998)
  • F.A. Casey et al.

    Behavioral adjustment of children with surgically palliated complex congenital heart disease

    J Pediatr Psychol

    (1996)
  • C.C. Davis et al.

    Psychological adaptation and adjustment of mothers of children with congenital heart disease: stress, coping, and family functioning

    J Pediatr Psychol

    (1998)
  • C. Limperopoulos et al.

    Functional limitations in young children with congenital heart defects after cardiac surgery

    Pediatrics

    (2001)
  • J.E. Ware

    Methodology in behavioral and psychosocial cancer research Conceptualizing disease impact and treatment outcomes

    Cancer

    (1984)
  • Aaronson NK. Quality of life: what is it? How should it be measured? Oncology (Williston Park) 1988; 1988 May;2:...
  • World Health Organization. Preamble to the Constitution of the World Health Organization as adopted by the...
  • Stein RE, MD, Jessop DJ, Phd

    Functional status II(R): a measure of child health status

    Med Care

    (1990)
  • D. Drotar

    Measuring health-related quality of life in children and adolescents

    (1998)
  • I.J. Higginson et al.

    Measuring quality of life: using quality of life measures in the clinical setting

    BMJ

    (2001)
  • B.S. Marino et al.

    Quality-of-life concerns differ among patients, parents, and medical providers in children and adolescents with congenital and acquired heart disease

    Pediatrics

    (2008)
  • P. Moons et al.

    Caliber of quality-of-life assessments in congenital heart disease: a plea for more conceptual and methodological rigor

    Arch Pediatr Adolesc Med

    (2004)
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