Researchers have proposed a core set of crucial outcome domains to serve as indicators of health performance for children with neurological and neurodevelopmental disorders or conditions.
Christopher Morris, DPhil, senior research fellow in Child Health at Peninsula Cerebra Childhood Disability Research Unit (PenCRU) at University of Exeter Medical School, and colleagues created the recommendations through a combination of qualitative research, surveys with health professionals and a review of outcome measures, as well as a meeting with key stakeholders who prioritized the outcomes.
Development of outcomes
Morris said the creation of key outcome domains is important for this group of patients because they usually need care from multidisciplinary teams whose members may have different goals. Morris and colleagues wanted to create a set of patient-centered outcomes that reflected a shared vision for all patients with pediatric neurological/neurodevelopmental disorders and conditions.
“To be meaningful, outcomes should be valued by patients and carers, be consistent with what health professionals seek to achieve and be robust in terms of measurement properties,” Morris told O&P News. Outcomes are used to quantify and aggregate patient health at any point in time, he said, adding, “This is useful for research, service evaluations, clinical audit and in some cases in individualized health care.”
The researchers focused on patients with “neurodisability,” which they define in the paper as “an umbrella term for conditions associated with the impairment of the nervous system.” The term refers to conditions such as cerebral palsy, autism and epilepsy.
Morris said although many individual neurological/developmental disorders and conditions are rare, when grouped together they share many attributes.
“Although it is reasonable to develop core outcome sets for more common conditions — such as have been proposed recently for cerebral palsy — it would also be efficient to devise core outcome sets that could be used across pediatric neurodisability,” Morris said. “This would avoid wasting resources to develop multiple core outcome sets for the vast number of different neurodisability conditions and syndromes.”
Morris said the results are relevant to “a wide audience of clinicians, managers and commissioners, and especially those responsible for ensuring health services meet their goals efficiently.”
The researchers emphasized public engagement throughout the study, with a goal of outcomes that included input from all stakeholders. Stakeholders included health professionals from a variety of fields associated with neurodevelopmental conditions, patients and their parent carers. Stakeholders provided different perspectives on the services that result in patient outcomes.
“Children/young people and their parents are service users or consumers, and health professionals are service providers,” Morris said.
Morris and colleagues combined multiple research methods to determine outcome priorities, beginning with a qualitative study of 54 children and 53 parents who participated in either a focus group or an interview. Families discussed communication, mobility, pain, self-care, temperament, interpersonal relationships and interactions, community and social life, emotional well-being, gaining independence and future aspirations. Parents also talked about sleep, behavior and safety.
The researchers also conducted a Delphi survey — an iterative series of surveys with a goal of reaching consensus — of more than 200 health professionals, who agreed on the following key outcome domains: mental health, confidence and emotional stability, anxiety and attention, sleep, pain, toileting, movement and manual ability, acquiring skills, communication, mobility, self-care, recreation and leisure.
“To engage a variety of health professionals from different specialties, we naturally ended up with a number too large and disparate to interview [individually], so we used several rounds of an online survey to gather and develop ideas and prioritize aspects of health with them,” Morris said.
In parallel, the researchers conducted a systematic review of patient-reported outcomes (PROMs), detailed in a separate study published in Value in Health. Morris and colleagues identified 41 multidimensional PROMs that assess various aspects of health in the International Classification of Functioning, Disability and Health for Children and Youth (ICF-CY) from the WHO. The review showed the domains most commonly described included: mental functions; interpersonal interactions and relationships; major life areas; and community, social and civic life.
Once these three sets of outcomes were gathered, Morris and colleagues conducted a meeting among stakeholders who had participated in earlier stages of the research to weigh in on prioritization. Stakeholders in the group included three patients – one with autism and two with a neuromuscular condition, five parent carers of children with various neurological disorders; and seven health professionals, including a pediatrician, physiotherapist, occupational therapist, nurse, pediatric surgeon and a child and adolescent psychologist.
While the group’s purpose was to sort and prioritize outcomes, the researchers wanted to observe the processes and discussions that led participants to their decisions. They created a list of health outcomes gathered from the qualitative research, Delphi survey and PROMs, removed duplicates and ended up with a final list of 33 “aspects of health” or health outcomes which they presented to the stakeholders. Participants were asked to share their personal views and discuss and negotiate the importance of each item among other aspects of health through group discussions and activities.
The researchers noted the difficulty for the group in reaching consensus, with debates about many aspects of health, such as which items were self-reportable and which items were more amenable to health care influence.
“Families value highly emotional well-being and social participation, and these were less prioritized by health professionals,” Morris said. “Although many professionals stated they targeted these aspects of health, they felt health services are less ‘accountable’ for them. Health care for people with long-term conditions could be improved by better integrating mental health support with chronic disease management.”
Results and next steps
“Agreement emerged regarding a core suit of health outcomes appropriate for children and young people with neurodisability: communication, emotional well-being, pain, sleep, mobility, self-care, independence, social activities, mental health, behavior, toileting and safety,” Morris said.
While the range of conditions was wide, Morris said the findings apply as much to children with cerebral palsy as to those with other neurological/neurodevelopmental conditions.
“The core outcome set we propose is entirely valid for children with cerebral palsy,” he said. “Interestingly, the condition-specific core outcome set for cerebral palsy developed in North America does not include key elements of communication – which was rated the highest priority outcome in our work – and does not include aspects of quality of life or well-being.”
The findings led the researchers to propose this group of outcomes as a suite of important health domains that should be assessed for children with neurological disorders and conditions. The identification of key domains also led to a comparison with current tools for assessing patient outcomes.
“We looked to see if any of the content assessed by patient-reported outcome measures covered the key domains, and whether there was any evidence of robust measurement properties for any patient-reported measure,” Morris said.
Morris noted a survey or questionnaire has yet to be created for patients that encapsulates all these outcomes.
“Unfortunately none of the patient-reported outcome measures we looked at assess enough of the key domains to enable us to recommend any existing questionnaires,” he said. “Also, there is a paucity of evidence for any of the patient-reported outcome measures being able to detect and quantify meaningful change from treatment.”
The researchers recommend further development of PROMs for children and young people with neurological and neurodevelopmental disorders and conditions. They recommended a greater role for “service users” – patients and their parent carers – in the development of PROMs, and the inclusion of all stakeholders as partners in the further development of PROMs.
“This study builds awareness of the priority health outcomes valued by families of children with a broad range of conditions that might be overlooked by health professionals,” Morris said. “Children’s sleep, toileting and safety are huge issues for parents of disabled children that can be neglected.” – by Amanda Alexander
- Morris C, et al. Health Qual Life Out. 2015;doi:10.1186/s12955-015-0284-7.
Disclosure: Morris reports no relevant financial disclosures.