Gait Problems in Cerebral Palsy: Identification, Patient Goals, and Surgical Treatment

Age 4–7 years

CLINICAL ENCOUNTERS

By the age of 4 to 6 years, most gross motor development has occurred and the vast majority of children with cerebral palsy can be classified according to the Gross Motor Function Classification System (GMFCS). Children are mature enough to undergo assessment and rehabilitation.

Gait deviations in cerebral palsy can be grouped into primary (neuropathic causes), secondary (musculoskeletal deformity), and compensation. Detailed data are required to drive clinical decisions for complex gait to make the right decisions and avoid the wrong decisions more often. Computerized 3D gait analysis is gathered before and after surgical interventions and increasingly at routine intervals as children grow (Video A.1).

An integrated, multidisciplinary approach with physical therapy, orthotics, and promotion of physical activities is facilitated by appropriately timed surgical intervention. For most children, tone management is one of the primary concerns at these ages. Lower extremity surgery should be delayed until the child is older.

Patient and Family Priorities: Principles, Tools, and Gait Outcomes Assessment List (GOAL) Results in Children 4 to 7 years old at Gillette Children’s

Elizabeth R. Boyer, Jean L. Stout, Katie Walt, and Steven E. Koop

COMMENTARY

Erich Rutz

How appropriate that the first chapter in this excellent and authoritative book should address the concept of ‘patient goals’. On a superficial level, everyone understands what a goal is. The first definition in most dictionaries relates to sports such as soccer or hockey. The second is along the lines of ‘the aim, to which effort is directed,’ a concise summary of our strivings in healthcare. Once we move to the concept of patient goals, things can rapidly become more complex and sometimes ambiguous. In 2020, Secunda and colleagues defined goals of care as: ‘the overarching aims of medical care for a patient that are informed by patients’ underlying values and priorities, established within the existing clinical context, and used to guide decisions about the use of or limitation(s) on specific medical interventions’.

This encapsulates and summarizes much of the thinking in this chapter and sets the scene for the rest of the book, as patient- and family-centered care.

The chapter includes a description of the evolution of our thinking about patient goals, the centrality of the World Health Organization’s International Classification of Functioning, Disability and Health (ICF) and the important concept of shared decision making. This is followed by a discussion on tools designed to help us capture ‘patient priorities and goals’. This section flows naturally from what we used to do, the unstructured interview (aka taking the clinical history) through a series of legacy approaches (Goal Attainment Scaling, Canadian Occupational Performance Measure) to where we are now, with a description of the Gait Outcomes Assessment List (GOAL) questionnaire. The GOAL is rapidly becoming an integral part of patient evaluation in motion analysis laboratories around the world. Early adopters were Gillette and the Hugh Williamson Gait Analysis Laboratory, Royal Children’s Hospital in Melbourne. The GOAL is the first patient-reported outcome measure for ambulant children with cerebral palsy. Crucially, it was designed by dialogue with children with cerebral palsy and their families. It has enormous potential and utility both for clinical practice and as a research outcomes tool.

The ‘Relevant Literature: What We Have Learned’ section is a useful summary of recent literature, including studies from Gillette and elsewhere. A standout section is on caregiver priorities as derived from the GOAL questionnaire at Gillette Children’s.

The final section is a series of thoughtful reflections on the process of identifying and establishing priorities and goals. This section gives a voice to former patients and family members, who were invited to discuss their experiences with identifying priorities and establishing goals, in a series of structured interviews. These are insightful, sometimes poignant, and worthy of prolonged reflection.

This chapter is both historical and contemporary. It leads the reader from the past to better ways for the future. In summary, ‘We shall not cease from exploration, and the end of all our exploring, will be to arrive where we started, and know the place for the first time’ (Eliot 1942).

REFERENCE

Eliot TS (1942) Little Gidding. London: Faber & Faber.

The science and art of medicine are perpetually changing, influenced by experiential knowledge, scientific and technological advances, cultural dynamics, patient preferences, environmental changes, government regulations, business pressures, and global pandemics. Navigating this plethora of forces, which often conflict, can be daunting. But at the center of this Gordian knot are people – the patient and the clinician. They need to align for favorable outcomes to be possible. This first chapter explores the patient–clinician relationship, resulting patient goals, tools to measure these goals, and conceptual medical models. We include a short discussion on how they have changed over time, especially in the context of disability. This introductory information is unique to Chapter 1, but it is essential to laying the groundwork on patient and family priorities that appear throughout this book for the three main age ranges, 4 to 7 years, 8 to 12 years, and adolescence.

EVOLUTION OF THINKING ABOUT PATIENT GOALS

Individuals with cerebral palsy (CP) are surrounded by a group of people who work together to identify and prioritize medical and functional concerns and establish goals for change when change is thought to be necessary. It is a collaboration that evolves as the individual with CP ages. During infancy and early childhood, parents act on behalf of their child. As the child with CP gets older, they slowly gain agency over their own medical goals, until they successfully transition to independence in adulthood (given the decision-making capacity to do so). The concerns and goals of the medical team will be specific to the clinician’s specialty (e.g. pediatrics, physical, occupational and speech therapy, neurology, physiatry, neurosurgery, orthopedics). One of the main responsibilities of the individual with CP (or parents) is to advocate for their own priorities and goals alongside those of the medical specialists. Then, the individual (or parent) must take inventory of all competing goals and make choices. This is hard work that involves education, dialogue, and intentional inclusion of the individual with CP and family members.

By whom and how medical concerns and goals are identified, measured, and set has undoubtedly changed over time for individuals with disabilities, and it may still differ today by patient preference, medical institution, specialty, country, and culture. Historically, medical concerns and goals were primarily clinician driven, falling under what is generally considered the biological or medical model of disability (World Health Organization 2001; Haegele and Hodge 2016; Lawson and Beckett 2021).

The medical model views disability as a deficiency or abnormality in structure (anatomy) or function (physiology) with the goal of medical treatment to change or ‘fix’ structure or function such that it is more ‘normal’ or typical (Haegele and Hodge 2016). Deference to the medical model was by virtue of the medical professional’s training and certification, which recognizes them as experts in their respective specialties. Adapting the medical model has been attributed to physicians and scientists supplanting religious leaders as the societal ‘cognitive authority’ to define, diagnose, and heal injuries or sicknesses (Haegele and Hodge 2016). Culturally, this was accepted as laypersons had little access to medical knowledge and relied on those with medical training to act in their best interest.

International Classification of Functioning, Disability and Health

The purely biological or medical model of disability is flawed, and other social/human rights models of disability have been proposed (World Health Organization 2001; Haegele and Hodge 2016; Lawson and Beckett 2021). In the societal model disability is viewed predominantly as a social problem, external to the person with impairments (Haegele and Hodge 2016; Lawson and Beckett 2021). As such, medical concerns, goals, and methods to address them have evolved. The World Health Organization’s International Classification of Functioning, Disability and Health (ICF) framework is one of the most widely accepted models of disability which integrate biological, psychological, and social models (World Health Organization 2001). The ICF framework defines key constructs and organizes them into a multidimensional model to describe health and disability on a continuum (Figure 1.1). The following definitions are taken verbatim from the ICF:

Functioning is an umbrella term for body functions, body structures, activities, and participation. It denotes the positive aspects of the interaction between an individual (with a health condition) and that individual’s contextual factors (environmental and personal factors).

Disability is an umbrella term for impairments, activity limitations, and participation restrictions. It denotes the negative aspects of the interaction between an individual (with a health condition) and that individual’s contextual factors (environmental and personal factors).

Body functions are the physiological functions of body systems (including psychological functions).

Body structures are anatomical parts of the body such as organs, limbs, and their components.

Impairments are problems in body function and structure such as significant deviation or loss.

Activity is the execution of a task or action by an individual.

Participation is involvement in a life situation.

Activity limitations are difficulties an individual may have in executing activities.

Participation restrictions are problems an individual may experience in involvement in life situations.

Environmental factors are the physical, social, and attitudinal environment in which people live and conduct their lives. These are either barriers to or facilitators of the person’s functioning.

Figure 1.1 The World Health Organization International Classification of Functioning, Disability and Health (ICF) model with relationships between ICF constructs indicated with arrows. Reproduced with kind permission of WHO.

Because disability was historically viewed with a medical model lens, the medical approach was to identify and treat a person’s structural or functional impairments (e.g. atypically shaped bones [structure], abnormally muscle tone [function]), since the ‘cause’ of a disability was ascribed to biology (Haegele and Hodge 2016). Structural and functional outcomes are usually more readily measured with objective metrics and directly influenced through medical intervention. Alternatively, outcomes in the activity and participation domains are influenced much more by society and personal preferences and are not as straightforward to measure.

Various tools can be used to measure gait-related body functions and structure in the context of identifying impairments. Within the neuromusculoskeletal and movement-related functions of the ICF, common tools and examinations include goniometers or radiographs to measure passive joint range of motion, manual muscle testing to measure voluntary muscle strength, the Modified Ashworth Scale or Hypertonia Assessment Tool to measure muscle spasticity or hypertonia, or instrumented gait analysis to measure gait pattern. Tools used to measure lower extremity structures may include various medical imaging to measure bones, joints, muscles, tendons, and ligaments. Clinical history includes the patient’s medical history in addition to patient symptoms or complaints (e.g. pain, toe dragging, falls). This model relies on a knowledgeable clinician to interpret information from all sources, investigate conflicting information (if any), and decide on the appropriate management. Together, the myriad of tools can inform orthopedic surgery planning for individuals with CP and was proposed by Davids et al. in 2003 as the ‘Diagnostic Matrix’ (Davids et al. 2003). They proposed various sources of information to quantify impairments and inform treatment, including clinical history, physical examination, medical imaging, quantitative gait analysis, and physical examination under anesthesia.

Shared Decision Making

Complementary to the adoption of the biopsychosocial model of disability is the adoption of a shared decision-making (SDM) medical model in most of the Western world (Bomhof-Roordink et al. 2019). SDM is generally defined as a process in which information is shared in an understandable manner so that medical decisions are a consensus between the patient (parent) and the clinician (Bomhof-Roordink et al. 2019). SDM acknowledges the clinician as the expert in their respective medical area who is knowledgeable of the quality of the scientific evidence, and the patient (parent/caregiver) as the expert in living with their condition who has their own values and priorities (Bomhof-Roordink et al. 2019). These principles are central to an evidence-based definition of ‘goals of care’: ‘overarching aims of medical care for a patient that are informed by patients’ underlying values and priorities, established within the existing clinical context, and used to guide decisions about the use of or limitation on specific medical interventions’, which emulates the SDM model (Secunda et al. 2020) One process to facilitate SDM endorsed by the US Agency for Healthcare Research and Quality is the SHARE Approach (Agency for Healthcare Research and Quality 2020). The five steps include:

1. Seek your patient’s participation.

2. Help your patient explore and compare treatment options.

3. Assess your patient’s values and preferences.

4. Reach a decision with your patient.

5. Evaluate your patient’s decision.

SDM has been widely embraced for many reasons. When patients (parents/caregivers) are involved in the medical decision-making process, positive outcomes may be noted across several domains, including affective–cognitive, behavioral, and health (Shay and Lafata 2015). The patient stakeholder group reported more benefits than clinical stakeholders, and the greatest effect was in the affective–cognitive domain. Specific benefits of an SDM include (Hauser et al. 2015; Shay and Lafata 2015; Agency for Healthcare Research and Quality, 2020):

1. Patients are more likely to adhere to treatment and reach goals.

2. Patients report greater satisfaction with their medical care.

3. Improved care delivery quality.

4. Improved satisfaction and experience of care by the patient.

5. Possible improvement in health outcomes.

Embracing SDM for pediatric care may have long-term effects. For instance, a study of adults with CP showed that satisfaction with orthopedic surgery during childhood was significantly associated with the level of the patient’s involvement in the decision-making process (Gannotti et al. 2021).

By involving the patient (parent/caregiver) in the goal-setting conversation with SDM, it is common for more activity or participation goals within their personal or environmental contexts to be included, thereby including more domains on the ICF (Vargus-Adams and Martin 2011). A natural byproduct of these conversations is the recognition and acceptance by all stakeholders that not all patient goals are amenable to surgery. Similarly, it allows for incongruencies in goals between clinician and patient and/or parent/caregiver and child to be discovered. This is quite common (Missiuna et al. 2006; Varni et al. 2006; Dunn et al. 2009; Maggs et al. 2011; Pritchard et al. 2022). Parents/caregivers and/or clinicians may underestimate the ability of young children to report their concerns or goals (Pritchard et al. 2022). When comparing parent-proxy-reported to child-reported outcomes, parents often systematically rate their child’s ability and/or happiness lower than the child rates themselves (Varni et al. 2006; Khanna et al. 2022). This systematic difference is especially evident in psychological, social, and pain domains while it is less evident in function domains. Therefore, when the child is old enough to self-report, child-report should be measured and included in the discussion with parent(s) and clinician (Khanna et al. 2022).

CAPTURING PATIENT PRIORITIES AND GOALS

There are both unstructured and structured methods and tools to measure patient (parent) concerns and goals (goal-setting theories or frameworks guide these methods) (Pritchard-Wiart and Phelan 2018). All methods or tools cover the generation or listing of concerns or goals, while some help to weight or prioritize goals, and still others expand on how goals will be achieved (Bovend’Eerdt, Botell, and Wade 2009). The following is a summary of some common methods and tools used for goal setting among individuals with CP. Customized surveys may also be created (e.g. Vargus-Adams and Martin 2011) but will not be summarized here. First, the method or tool’s purpose and structure are summarized, then Table 1.1 provides further details on key psychometric properties and utility.

Unstructured or Semi-structured Interview

Unstructured interviews are unscripted questions or discussions between the patient (parent) and clinician that can include patient (parent) concerns or goals. Questions from the clinician might include, ‘What are your biggest concerns for your child?’ and ‘What are you hoping to achieve with treatment?’ The patient (parent/caregiver) may also interject their concerns or goals, for example, ‘My child trips and falls a lot’, or ‘I’d like to help them walk more upright, so they don’t have as much pain now or in the future’. Semi-structured interviews do have some scripts or planned open-ended questions that cover certain topics. However, it is allowable and common for the interviewer (the clinician) to go off-script and probe further on topics, depending on the response of the patient (parent/caregiver). This helps the conversation to evolve organically, while the semi-structured component still leads to gathering key information for reporting or comparison purposes. Patient (parent/caregiver) goals acquired this way may be documented in the patient’s medical record and may be rephrased as a SMART (specific, measurable, achievable, realistic/relevant, timed) goal.

SMART Goals

Developing SMART goals is a method of goal setting that avoids ambiguity so that achievement can be assessed (Bovend’Eerdt, Botell, and Wade 2009; Ogbeiwi 2021). Specific means the goal usually addresses the who, what, and how – who does what and how they will achieve the goal. Measurable means some quantifiable scale can be used to determine if a goal was achieved and/or progress being made toward that goal. Achievable means that the goal is realistic for the patient, given their characteristics and resources/environment. Relevant means the goals are relevant or important to the patient and their values. Timed means specific deadlines are named in which the goal should be achieved. An example of a SMART goal would be:

‘In 3 months, I will be able to walk for 30 minutes at 2.5 mph on the treadmill without stopping.’

The SMART goal method, when used in the medical field, often involves codevelopment of goals with the patient (parent) and medical professional. SMART goals are often incorporated into the other methods and tools listed here.

Goal Attainment Scaling/Scale

The Goal Attainment Scaling/Scale (GAS) is a method and scoring system in which patients (parents) identify goals for themselves before an intervention and rate their achievement of those goals after intervention (Turner-Stokes 2009; Gaasterland et al. 2016; Shirley Ryan Ability Lab 2020). It is unstructured in gathering goals, in that a standard list of domains or items is not used. However, it is structured in rating goal achievement, which is rated by the patient (parent) on a 5-point Likert scale of success, ranging from ‘−2 most unfavorable outcome’ to ‘+2 best anticipated outcome.’ The overall score is the summation across goals and turned into a standardized T-score. The patient (parent) may optionally weight goals by importance, which factors into the score. Reliability/minimal change: Rating one’s goal achievement is a measurement of change; furthermore, the GAS overall score is considered responsive to change while measures of reliability are variable (Gaasterland et al. 2016).

Abbreviations: approx, approximately; COPM, Canadian Occupational Performance Measure; GAS, Goal Attainment Scale; GOAL, Gait Outcomes Assessment List; N/A, not applicable; PEGS, Perceived Efficacy and Goal Setting; pt: point; SMART: specific, measurable, achievable, realistic/relevant, timed.

Perceived Efficacy and Goal Setting System

The Perceived Efficacy and Goal Setting (PEGS) system is a dual-component goal setting tool that includes a child (patient) and caregiver component, usually facilitated by a therapist (Missiuna and Pollock 2000; Missiuna, Pollock, and Law 2004; Missiuna et al. 2006; Shirley Ryan Ability Lab 2017; CanChild 2023). The child-reported tool uses illustrated cards and descriptions so they can rate their performance on 27 daily activities and set goals (2nd edition). Domains include self-care, school, and leisure. The child is presented with 27 pairs of cards – one that shows difficulty with an activity and another that shows no difficulty with that same activity – and selects which one best describes themself. Using the pile of more difficult tasks, the therapist asks the child which four cards are their priority. Relatedly, the caregiver component includes a questionnaire of the same list of activities. The caregiver rates performance on a 4-point Likert scale, which is summed for an overall performance score. The caregiver lists four skills they would like their child to improve as their goals. The child, caregiver, and therapist work together to set goals based on responses. Any skill not included in the original list can be identified by the caregiver. Reliability/minimal change: Children may be unreliable in selecting their top four goals, as only 32% selected 4/4 of the same cards/goals and 37% selected 3/4 of the same goals over a 2-week period (Missiuna et al. 2006). Agreement on top four goals between parent and child may also be low, with the same study finding only 22% of parent–child pairs agreed on two or more goals.

Canadian Occupational Performance Measure

The Canadian Occupational Performance Measure (COPM) is a standard list of nine daily living items or areas that fall within three domains: self-care, productivity, and leisure topics that a patient (parent) can identify problem areas and rate their performance and satisfaction with that performance. It is administered within a semi-structured interview, often by occupational therapists (Law et al. 2019; Shirley Ryan Ability Lab 2019; The Canadian Occupational Performance Measure 2023). In Step 1, the therapist probes about tasks in patients’ daily life they want, need, or are expected to perform but struggle to do or are not satisfied with how they perform. Each problem identified is rated on a 10-point importance scale in Step 2. The patient and therapist select up to five problems identified in Step 3. The therapist may guide the patient in re-writing the problems as SMART goals. In Step 4, the patient rates both their performance level and satisfaction with performance for each problem on a 10-point scale. Total scores for performance and satisfaction are calculated as the average score across the number of problems. Finally, in Step 5, patients score themselves again after an intervention to assess for change. Reliability/minimal change: Though a change of two or more points on either performance or satisfaction were often considered the minimal clinically important difference, a 2023 review found no empirical evidence for this (McColl et al. 2023).

GOAL Questionnaire

The GOAL questionnaire is a patient-reported outcome tool (adolescent and parent versions) that evaluates gait-related function and happiness on 49 items or skills across seven domains for ambulatory children with CP, as well as rate the importance of each item as a goal to improve (Narayanan, Davidson, and Weir 2011; Narayanan et al. 2015; Thomason et al. 2018; Boyer et al. 2022; The Hospital for Sick Children [SickKids] 2023; Stout et al. 2024). Importance is rated on a 3-point Likert scale (0: not a goal, 1: somewhat important, 2: very important). In practice, multiple items may be listed as very important, but there is no guidance from the developers on goal prioritization. The seven domains include Activities of Daily Living & Independence; Gait Function & Mobility; Pain, Discomfort, & Fatigue; Physical Activities, Sports, & Recreation; Gait Pattern & Appearance; Use of Braces & Mobility Aids; and Body Image & Self-Esteem (Table 1.2). Any item or skills that are of importance to the patient (parent) but not included in the original list can be named and rated. A major strength of this tool is the comprehensive nature of gait-related concerns presented to the patient (parent), which was informed by family advisory groups during tool development (Narayanan, Davidson, and Weir 2011). This allows common concerns to not be overlooked and helps communicate relative importance to clinicians. Reliability/minimal change: Across all 49 items, agreement on goal importance was 73% among parents, so a change in 1-point is likely a true change (Stout et al. 2024). Agreement in goal importance between the adolescent and parent versions has not been evaluated.

Other Tools

There are many tools that objectively assess a patient’s ability to perform mobility (e.g. Gross Motor Function Measure) (Russell et al. 1989, 2021) or self- or proxy-report ability (e.g. Gillette Functional Assessment Questionnaire) (Novacheck, Stout, and Tervo 2000; Stout et al. 2012), Functional Independence Measure for Children (WeeFIM™) (Msall et al. 1994; Ottenbacher et al. 1996), Pediatric Outcomes Data Collection Instrument (PODCI) (Daltroy et al. 1998), Pediatric Quality of Life (PedsQL) CP Module (Varni et al. 2006). In the PedsQL CP Module, for instance, a patient (parent) is asked to rate to what extent performing 35 activities is a problem. Because responses most likely measure the difficulty of performance and not necessarily if that activity is a goal for the patient to improve, these types of tools were not discussed, though they may prompt further discussion with the patient (parent) to help identify patient (parent) goals. Furthermore, newer patient-centric tools are being developed that can also inform mobility-related priorities of children with disabilities, such as the Children’s Meaningful Activities and Participation in Rehabilitation (CMAP) (Vänskä et al. 2022).

RELEVANT LITERATURE: WHAT WE HAVE LEARNED

Development and Life Situations that Might Influence Concerns and Priorities

Children and their families are influenced by a multitude of factors when they describe concerns, establish priorities, and set goals. A critical factor is growth in knowledge about CP and acceptance of the diagnosis (Knox 2008). This process starts early in childhood and continues into adolescence and young adulthood. Other factors include, but are not limited to the child’s age, Gross Motor Function Classification System (GMFCS) level (Palisano et al. 2008), experience with medical, surgical, and therapeutic treatments, the degree of social isolation of parents/caregivers, and social determinants of health.

The evolving nature of family/child understanding and acceptance of the consequences of CP will cause goals and priorities to change across time (Pritchard-Wiart, Thompson-Hodgetts, and McKillop, 2019; Forber-Pratt et al. 2021; Munger et al. 2023a, 2023b). Recent work has shown that the odds of activities of daily living or mobility on the GOAL questionnaire being a priority decreased 12% and 14% respectively, with each additional year of a child’s age (Munger et al. 2023b).

GMFCS level (and caregiver knowledge of GMFCS) has a substantial impact on goals and priorities (Knox 2008; Boyer et al. 2022). These will be discussed in detail specific to age in subsequent sections and chapters when available.

Participation in rehabilitation therapies also influences priorities across age, including the toddler and preschool years (Ostensjo, Oien, and Fallang 2008; Kang et al. 2020). The influence of other treatments on priorities, such as botulinum toxin, has not been well studied.

The impact of social factors, including social determinants of health, on goals and priorities should not be underestimated. In the simplest of terms, social determinants of health (SDH) are ‘the non-medical factors that influence health outcomes’ (World Health Organization 2024). The US Office of Disease Prevention and Health Promotion expands the definition as ‘the conditions in the environments where people are born, live, work, play, worship, and age that affect a wide range of health, functioning, and quality-of-life outcomes and risks’ (US Department of Health and Human Services) (Figure 1.2). In the context of the ICF, SDH can be considered the ‘personal and environmental factors that interact to influence function’ (Rosenbaum and Stewart 2004). The expanding attention to SDH over the past decade includes their impact on the priorities of families/children with CP (Munger et al. 2023a, 2023b; Ostojic et al. 2024). Factors in the SDH categories of healthcare access (including care coordination), neighborhood and built environment, social and community context, and economic stability (Figure 1.2) have been identified as important for families of children with CP (Oskoui et al. 2016; Casseus and Cheng 2021; Munger et al. 2023b; Shetty et al. 2023; Ostojic et al. 2024). The rehabilitation priorities of families living in low-resource settings (areas that lack the necessary resources to provide adequate healthcare service delivery) are often driven by societal expectations for independent ambulation, rather than achievable functional goals (Shetty et al. 2023).