Pediatric neurosurgical conditions can dramatically affect development, functional status, and therefore quality of life—these changes can have life lasting consequences or benefits. There are very few disease-specific quality outcome scales, which have been validated in a prospective fashion, to assess children treated by pediatric neurosurgeons. We will review the current metrics that have been used in children with hydrocephalus, epilepsy, brain tumors, Chiari I malformation, or spina bifida. There is growing interest to develop and validate new quality-of-life metrics, which capture the perspective of both the parent and child.
KeywordsQuality, Pediatric neurosurgery, Child health
Since its origin, neurosurgery, as a field, has relied on expert opinion. After all, its development and advancement have been intertwined with names such as Harvey Cushing. In pediatric neurosurgery, there are relatively low disease incidences compared to conditions such as heart disease or prostate cancer. Neurosurgical care is generally expensive, and focused around academic or tertiary centers rather than widespread throughout communities. Culturally, neurosurgery has always had a fascination with rare or extreme findings. Many neurosurgeons have made their name by developing novel approaches or have been called “master surgeons,” such that other surgeons have been unable to replicate their outcomes.
With the exception of spine surgery, neurosurgery has generally been slow to adopt an evidence-based approach to practice. However, in an era of information availability for our patients, data sharing across institutions, and cost-consciousness in our healthcare system, neurosurgeons are also prompted to ask, “Are we providing good quality care?.” We will focus this chapter on existing quality-of-life outcome measures in pediatric neurosurgery and how these have allowed us to assess quality of care and, most importantly, to promote effective treatments.
Quality of Care
How we define quality of care in neurosurgery is similar to other, especially surgical, subspecialties of medicine. Though not the focus of this chapter, neurosurgeons must first ask what are the indications for surgery, and how does the procedure compare to other surgical or nonsurgical interventions?
Three basic tenets of any intervention are
Is there a survival benefit or alternatively an increase in mortality following an intervention relative to the natural history of the disease?
If there is a survival benefit, what is the child’s quality of life or functional status?
What is the cost of the intervention and is the perceived benefit “worth” the cost?
Outcome studies in pediatric neurosurgery have largely focused on reporting clinical data including surgical complications. Importantly, pediatric neurosurgical conditions can dramatically affect development, functional status, and therefore quality of life. However, pediatric neurosurgery has less readily measured health status or health-related quality-of-life outcomes (HRQOL) using validated assessment tools. Health status outcomes differ slightly from HRQOL in that the former is a more objective measure of functioning, whereas the latter incorporates the patient’s perception of their own well-being. Both are relevant for neurosurgical diseases, with HRQOL outcomes being especially important in conditions that involve pain symptoms. Though far from perfect, these standardized tools provide a metric to compare different treatments and providers and can further inform practice guidelines.
As reimbursement structures change, neurosurgeons are increasingly expected to justify expensive procedures for relatively rare disease processes, as well as differences in practice patterns to treat those diseases. Differences in reimbursement, even between the neurosurgical subspecialties, put pediatric neurosurgery at a disadvantage. Longitudinal care and the transferability of electronic medical records mean that capturing long-term outcomes is now easier than ever before. Focusing on the quality of care allows hospitals to make widespread improvement measures. Best practice implementation and an improvement in the quality of care can decrease costs by decreasing variations in practice and improving health care efficiency. Making cost decisions based on quality measures in children will benefit the health care system as a whole.
Validated Outcome Measures
Assessing the quality of care necessitates valid and reliable outcome measures that can be applied across institutions. Pediatric neurosurgery has lagged behind in generating outcome studies, despite a growing emphasis across health care on measurable outcomes. In all, 31 articles using HRQOL tools have been published over the past 10 years across three major pediatric neurosurgical journals compared to 55 general pediatric articles in Pediatrics . In pediatric neurosurgery, many tools remain unvalidated and the same tools have not been used consistently between studies. Furthermore, many studies assessing quality of life or functional outcomes in pediatric neurosurgery have relied on outcome measures validated primarily in the adult population and then applied to children. For example, most series assessing pediatric arteriovenous malformations have used the modified Rankin Scale to measure disability, even though this scale has not been validated in the pediatric population. Measures such as independence and the ability to work are less relevant in children. Standardized time points to assess prospectively quality-of-life outcomes are crucial.
Two types of HRQOL outcomes tools exist: those that are generic across health conditions, and those that are disease specific. Neurosurgeons who are developing HRQOL tools must also consider whether to subcategorize health factors and whether to take into account age-specific considerations.
The Pediatric Quality-of-Life Inventory
The Pediatric Quality-of-Life Inventory (PedsQL 4.0) is one example of a generic HRQOL tool that measures health outcomes in children. This tool includes 23 general questions as well as different disease-specific variables. Questions are scored using a five-point Likert scale and subscored into psychosocial and physical scores. If more than 50% of the items are missing, the results are null. Cognitive outcomes can be measured either by the child themselves or by the parents as proxy. For pediatric brain tumors, there are 24 items that fall into six dimensions: cognitive problems, pain and hurt, movement and balance, procedural anxiety, nausea, and worry. These variables can give valuable insight into a child’s physical, psychological, and emotional well-being, as well as how he or she is functioning socially and in school. The PedsQL has high reported reliability (> 0.88). Within neurosurgery, the Ped QL 4.0 has already been used to assess a number of different conditions.
Health Utilities Index Mark 3 (HUI3)
The HUI3 is a 15-item questionnaire used in children that evaluates eight basic variables that affect quality of life: vision, hearing, speech, cognition, emotion, pain, ambulation, and dexterity. Participants have a choice of 4–5 responses. Normative scores are established based on population level preferences for optimal health and can range from death (0.0) to perfect health (1.00). Using this scoring algorithm, individual variables can be used to determine a multiattribute score. Aggregate scores have a theoretical minimum of − 0.341 and a maximum of 1.0 (perfect health). Data are nonparametric (values tend to cluster near the maximum of 1.0) and do not follow a normal distribution. The HUI3 has been used to assess patient-reported disease outcomes for a number of neurosurgical conditions including Chiari I malformation and spina bifida. The utility of the HUI3 in evaluating these conditions will be discussed below.
Disease-Specific Outcome Measures
Childhood disease-specific quality-of-life outcome measures are important to capture disease nuances that may vary subtly after different treatments. “It is also crucial to identify” disease-specific symptoms that may overlap with chronic comorbid conditions. Several of the disease-specific tools to assess pediatric neurologic disorders originate from pediatric neurology. In the era of cost-conscious health care decision-making, it is especially important that pediatric neurosurgeons partake in the conversation about outcome measures related to potentially surgical diseases.
Hydrocephalus Outcome Questionnaire
The Hydrocephalus Outcome Questionnaire (HOQ) is a highly reliable (> 0.88), multidimensional questionnaire to assess different physical, social, emotional, and cognitive factors affecting the lives of children with hydrocephalus. These subsections are then added to give an overall health score. Importantly, the HOQ aims to capture the multifactorial ways in which hydrocephalus (not just shunting and shunt failure) can affect quality of life. The HOQ consists of 51 items and takes approximately 10–15 min to administer. Parents answer the questions as a proxy for their children, as many children with hydrocephalus may be too young or unable to answer the questions on their own. The questionnaire was developed by experts in the field as well as focus groups consisting of parents of children with hydrocephalus. A list of 165 items was then narrowed down by a subgroup of parents, who were asked to identify what they deemed as the most important health items. Another cohort of 90 parents then tested the construct validity and reliability of the HOQ against other general HRQOL outcome questionnaires. The authors intended the HOQ as a discriminative rather than an evaluative instrument, meaning that it should be used to differentiate patients at one point in time rather than assess patient progress over multiple points in time. Data obtained using HOQ have shown greater cognitive impairments in children who have had multiple shunt revisions, as well as worse overall health status in children who also have epilepsy.
The Chicago Chiari Outcomes Scale
The Chicago Chiari Outcomes Scale (CCOS) is a health-related quality outcomes scale that relies on clinician assessment of patient’s pain, nonpain symptoms, functioning, and complications. A flowchart set of questions for each category guides clinicians to a score ranging from 1 to 4. The purpose of the scoring system is to quantify postoperative outcomes . Scores were found to correlate with the more traditional gestalt assessments of “improved,” “unchanged,” and “worse.” However, the lack of patient input into the outcome measures could be seen as one disadvantage. The scoring system is also limited in its ability to compare across patients as scores are based on relative outcomes for that individual.
Chiari Health Index for Pediatrics
The Chiari Health Index for Pediatrics (CHIP) is a 45-item multidimensional questionnaire that addresses both the physical and psychosocial aspects of Chiari I malformation. Within the realm of physical symptoms, questions fall into pain frequency, pain severity, and nonpain symptoms. HRQOL indices are particularly important for assessing postoperative outcomes in Chiari I because pain is an important component of the disease symptomatology and significant variation exists in the current practice patterns. The CHIP was designed to have discriminative, evaluative, and predictive capabilities. CHIP questions are consistent across age groups and can be completed by the child alone, with a parent, or by the parent alone. Scoring is based on a five-point scale and is normalized to account for missing responses. The physical and psychosocial components are each also weighed to account for the variance in the instrument and the correlation between each factor.
The CHIP was validated in a group of 55 patients with Chiari I aged 5–18-years in comparison to a group of 25 healthy children. HRQOL outcomes were also verified across the same groups using the Health Utilities Index Mark 3 (HUI3), an established general tool for pediatric health outcomes. The CHIP physical and psychosocial subscores were significantly lower for patients with Chiari I compared to healthy controls ( P < .001). CHIP scores also correlated quite well with HUI3, making an additional argument for its validity as a measurement tool. Only 25% of surveys were missing one or more items, and therefore the CHIP was deemed relatively feasible to administer. Longitudinal data are needed to assess its ongoing validity. Although usable as a preoperative assessment tool, it has yet to be shown which variables correlate with favorable postoperative outcomes.
Early medical and surgical intervention is particularly important in spina bifida and can have lifelong implications on quality of life. Given that patients with spina bifida deal with many chronic issues, the effect of psychosocial well-being on quality of life cannot be understated. The HRQOL-SB scale is a 47-item parent-reported scale or a 50-item adolescent self-reported scale developed after a literature review with refinement by focus groups of parents and patients. Of patients with spina bifida, those with a diagnosis of myelomeningocele have a lower overall quality of life than those with closed spinal dysraphism since the former often have impaired neurologic function. An assessment of HRQOL using the HUI3 demonstrates that both overall quality of life and ambulatory scores are affected by bowel and bladder incontinence, though capturing this variable was limited by its effect on one of the 8 HUI3 variables. A subgroup analysis of patients with open spinal dysraphism showed a significant negative impact of shunts on quality of life. The presence of Chiari II malformation also contributed to lower overall quality-of-life scores. For spina bifida, like many other complex chronic medical conditions, multidisciplinary care leads to better psychosocial outcomes.
The Quality of Life in Childhood Epilepsy Questionnaire
The American Quality of Life in Childhood Epilepsy Questionnaire (QOLCE) was developed from the Australian QOLCE as a 76-item questionnaire aimed at assessing quality of life in 4- to 18-year-old children with epilepsy. The questionnaire contains 16 subscales covering five domains: physical function, social function, cognition, and emotional and behavioral well-being. The QOLCE relies on parent-reported responses and is highly reliable and correlates with other generic health outcome measures. A shortened QOLCE—the QOLCE-55—was also created that demonstrated reliability and validity consistent with the original version.
Pediatric Functional Assessment of Cancer Therapy Questionnaire in Brain Tumor Survivor (pedsFACT-BrS)
Pediatric brain tumor survivors can face a number of ongoing health issues depending on the aggressiveness of their disease and the necessity of treatment. Posttreatment sequelae including neurocognitive impairment can significantly impact quality of life. The pedsFACT-BrS is a 34-item questionnaire that addresses the physical well-being, emotional well-being and illness experiences, social well-being, and brain tumor-specific concerns of pediatric brain tumor patients. It was developed by interviewing survivors, caregivers as well as clinicians and teachers and found to have good validity. The questionnaire was later stratified into age-specific modules, as well as parent and patient versions.
Challenges and Solutions
In pediatric neurosurgery, the power of prospective studies is limited by the relative rarity of diseases and randomized control trials are generally lacking. The level of evidence for outcome studies in pediatric neurosurgical journals is typically level 4. When validated outcome measures are lacking, consensus building can help determine which variables are important to measure with regard to certain disease processes. However, it is still not entirely clear how much a quantitative quality-of-life score might need to change for a child or parent to perceive an impactful improvement after a neurosurgical procedure.
To assess quality of life for children, “self-reported” outcomes often rely on parental responses, which are not a perfect proxy for determining the child’s quality of life. Parental reporting may further vary by which parent is surveyed, and at which time points in postoperative recovery children are assessed. Nevertheless, parents, as caregivers, are also intimately tied to the child’s well-being and quality of life, and child or adolescent responses alone may lack this dimension.