Outcomes Assessment of Fractures in Children

Frameworks of Health and Disease and the Evaluation of Outcomes

An evolution has occurred in the conceptualization of health since the traditional medical model, in which health was characterized merely by the absence of disease, to more complex models that take a more holistic account of the human experience. This evolution has been accompanied by changes in the way we measure health and disease, reflected by an array of outcome measures that quantify a wide range of health-related phenomena, including physical, mental, and social status and quality of life (QOL).

International Classification of Functioning, Disability and Health

The International Classification of Functioning, Disability and Health (ICF), developed by the World Health Organization (WHO), provides a unified, standard language that classifies health and health-related domains of individuals or populations, and provides a framework to measure health and disability associated with any health condition. The ICF complements the International Classification of Diseases (ICD) system, which codifies these conditions. The model has been adapted for children to develop the ICF for Children and Youth. The ICF list of domains includes a list of body structures and functions and a list of activities and participation, which can be influenced by contextual factors such as the environment and personal characteristics ( Fig. 9.1 ).

In the ICF framework, the term body structures refers to the anatomic parts of the body affected by the health condition of interest (e.g., effect of an injury on bones, muscles, and neurovascular structures), and the term body functions refers to physiological and psychological functions of various body systems (e.g., range of motion). Intact body structures and body function allow for activities . An activity refers to the completion of a specific task or action (e.g., throwing, running), which, when performed for a particular purpose or role, is referred to as participation (e.g., playing baseball). Participation implies doing things that one wants to do. Participation in life roles is a key component of QOL. Disruption of body structures and body functions, associated with a health condition, results in impairments . For instance, pediatric injury may be associated with impairments of the body structure, specifically the musculoskeletal system (e.g., femur fracture) and body function (e.g., joint range of motion). Impairments of body structures and body functioning may lead to limitation in activities. Limitation in an activity (e.g., inability to run) can result in restriction of participation (e.g., being dropped from the soccer team). The impact of a health condition and its treatment on activities and participation constitute what are often called “functional” outcomes. The ICF framework includes the consideration of contextual factors that can be strong determinants, either as facilitators or barriers, of functional outcomes. These include external environmental factors , such as home/school/community, socioeconomic status, access to health care, as well as personal factors , such as demographic characteristics, culture and upbringing, lifestyle preferences, motivation, and personality traits. Contextual factors are important to consider, as they can explain the gap between what one can do (capacity) and what one actually does do in daily life (performance).

Technical Versus Functional Outcomes

In the management of fractures, the immediate objective of interventions is to restore and maintain alignment and length until the fracture unites and consolidates. In the ICF framework, these are outcomes at the level of impairments of body structures and body functions. Goldberg refers to these as technical (or clinical) outcomes. These are important indicators of the success of the intervention in achieving its “technical” objectives (e.g., anatomic alignment and bony union after open reduction and internal fixation). However, there is an expectation that these will lead to the functional outcomes that patients and parents ultimately want, which is to return to full activities and participation without restrictions (e.g., return to playing soccer). It is important to measure functional outcomes separately because these are related to the ultimate goals of the patient. A technically successful outcome cannot always be assumed to result in a functionally successful outcome. A functionally successful outcome may not always require technical perfection. Indeed, more harm than good might arise from one’s pursuit, and even achievement, of technical perfection. For example, an open reduction of a radial neck fracture to restore anatomic alignment might lead to a worse functional outcome than accepting some magnitude of malalignment that is completely compatible with a perfect functional result. Functional outcomes are more meaningful indicators of effectiveness than technical/clinical outcomes.

The Priority Framework for Outcomes Evaluation

Outcomes are most meaningful when they are aligned with patient priorities . Living with a health condition is associated with a set of experiences that include current symptoms or “complaints” and/or potential future consequences related to the natural history of that condition. These experiences and the knowledge of future consequences can be associated with a set of concerns about the health condition and/or its treatment, resulting in certain desires (wishes) and expectations of these treatments and outcomes. Concerns, desires, and expectations can be collectively considered as patient priorities. Elicitation of these priorities enables a patient to define a set of goals related to their priorities, which can influence their choice (or preference) for specific treatment options. The elicitation of patients’ priorities might provide important insight into hitherto unknown patient preferences, which, in turn, might influence the process of informed choice, decision-making, and true informed consent, and facilitate the evaluation of outcomes that matter most to patients. PRO instruments will only be meaningful if the questions asked of patients reflect what is relevant and important to them.

These concepts have been incorporated in a priority framework for evaluation of outcomes. In the center of the framework is the health condition of interest (e.g., pediatric fractures). Living with the health condition leads to a set of priorities (concerns, needs, desires, and expectations), which are fundamental to defining the goals that are derived from these priorities. Different stakeholders might have different priorities (e.g., child, parent[s], family, surgeon, societal) which might overlap but may not be concordant. Understanding priorities and goals is crucial for making decisions about interventions that will best address these priorities and goals, or for developing new treatments/interventions where these do not exist or are insufficiently effective in addressing the priorities and goals. Interventions must be held accountable in terms of achievement of these goals. Their effectiveness is evaluated (e.g., in clinical trials or cohort studies or at the level of the individual patient) using outcome measures that specifically incorporate the goals and priorities of the patient population. In this framework, defining goals, choosing interventions, and evaluating outcomes or developing valid measures to do so all come back to and depend on an understanding of patient priorities and the goals that arise from these priorities ( Fig. 9.2 ).

In the management of fractures, achieving anatomic alignment and bony union are a means to an end. The ultimate goal is to ensure that the injured patient is restored to the preinjured state. If the desired goals include that the injured limb should look, feel, and work as well as it did before the injury, these goals should be embedded in any outcome measure that purports to evaluate effectiveness in terms that are meaningful for patients.

Whose Perspective Prevails?

Outcome measures are tools used to assess a change in particular attributes that are deemed meaningful to a person’s life over time. To the extent that the patient’s perspective is recognized to be preeminent in making judgments about effectiveness, the use of PRO measures is now considered the standard when the effectiveness of interventions is evaluated. PROs should be derived from patients themselves, particularly for outcomes that pertain to personal experience (e.g., pain, body image, and self-esteem). This is challenging in the context of pediatric conditions. When children are too young or too cognitively immature to respond, one has to rely on the report of the child’s parent(s). A parent(s)’ report must be recognized to be a proxy for what the child might report. The views of older children can and must be taken into consideration, but their perspective might differ from those of their parents. Parents may or may not recognize that their priorities might be different from those of their children, and parents may not agree with each other. The level of agreement between parents and children is usually good for domains reflecting physical activity, functioning, and symptoms, but poorer for domains reflecting more social or emotional issues. Proxies and children may not agree about many issues, but both perspectives may be valid and should be considered during decision-making, as well as in measuring outcomes.

Some outcomes are more important than others, and different stakeholders will have different perspectives on the relative importance of different outcomes. Clinical outcomes are more relevant to patients; nonclinical outcomes (e.g., length of stay, cost) are of interest to hospital administrators, payers, and health policy makers.

Generic Versus Condition-Specific Measures

Outcome measures can be generic or disease- or condition-specific. Generic outcome measures include those that measure general physical function, health status, and well-being. These have the advantage of comparing outcomes across different clinical conditions and interventions and are particularly useful to policy makers who might be interested in understanding the relative value of some types of interventions over others for purposes of health care utilization, planning, and resource allocation. However, generic outcome measures may assess some things that are not relevant to the condition and may neglect to include other issues of crucial importance. Generic measures are usually less sensitive to change than condition-specific measures that are designed to focus on issues relevant to the condition of interest. Condition-specific measures have more limited applicability. For instance, an outcome measure designed to evaluate outcomes of lower extremity (LE) fractures in children may not be relevant for other musculoskeletal conditions in children, let alone nonmusculoskeletal pediatric conditions.