Briefly describe a specific question, or set of related questions, about a health care test or treatment that this program should consider.

QUESTION: For children and adults with cerebral palsy what is the comparative effectiveness of outcomes for each GMFCS level?

BACKGROUND: The Gross Motor Function Classification System for Cerebral Palsy (GMFCS) (Palisano et al. 1997, 2008) is the first purpose-designed classification system for people with CP that has been shown to be both reliable (when used by different professionals, and between professionals and parents), and valid as a discriminative way to categorize importantly different levels of gross motor levels, and a predictive way to consider later motor status (into young adulthood). First published in 1997, the GMFCS is used all over the world in at least 25 languages, and has been cited in the literature on CP several hundred times. Virtually all research reports on CP now categorize/stratify their population with the GMFCS. ISSUE FOR SYSTEMATIC REVIEW: We believe that there is a valuable opportunity to undertake a systematic review of all the published literature in CP that has used the GMFCS, to determine what has been learned about people with CP as categorized by this effective classification variable.

EXPECTED OUTCOME: We believe that the outcome of this process will be a comprehensive ‘modern’ overview of what is, and is not yet, known about a host of aspects of the lives and functional status of children and youth (and to some extent adults) with CP.

Does your question include a comparison of different health care approaches? (If no, your topic will still be considered.)

no

If yes, explain the specific technologies, devices, drugs, or interventions you would like to see compared:

At the present time the answer is No. However, comparison of variations in any outcomes by GMFCS levels will allow us to explore health care approaches and develop goals and expectations of interventions, and hence of expected outcomes, by GMFCS level (where these are known from the literature to vary). This would provide what would be evidence-based data from the extensive literature review we are proposing. As but a few simple examples we could address the everyday questions: “How do children in different GMFCS levels benefit from physical therapy?” “Is there a dose-response relationship?” “Are there qualitative and/or quantitative differences in outcomes by GMFCS level?”

By virtue of the GMFCS stratification that is reported in the research papers, there will usually, if not always, be implicit comparisons of whatever ‘outcomes’ are being reported by levels of function. Thus, for example, it will be possible (a) to know whether specific outcomes do or do not vary by GMFCS level, and (b) to identify those areas of the ‘CP population’ that have not been studied with whatever interventions are being reported.

What patients or group(s) of patients does your question apply to? (Please include specific details such as age range, gender, coexisting diagnoses, and indications for therapy.)

All published CP research that has described their population by GMFCS levels, or used the GMFCS as a stratification element, will have addressed some/all of the population, though of course this review will enable us to learn who has/has not been studied, what outcomes are/are not being reported, what the gaps are in our understanding of CP, and what opportunities there are to fill those gaps with the next generation of primary research studies.

Are there subgroups of patients that your question might apply to? (For example, an ethnic group, stage or severity of a disease.)

The main ‘subgroups’ within the CP population are, of course, the GMFCS categories! The GMFCS makes it possible, for the first time, to explore a host of outcomes using a valid and reliable stratification system. It is hard to be certain, but there is likely to be only limited research on adults with CP whose gross motor functional status has rarely been reported systematically. Insofar as there is a gaping hole in our knowledge about adults with CP, this kind of ‘subgroup’ information will be invaluable. (Note that although this question about ‘adults with a developmental disability’ is specific to CP, we propose to ask it because we can start to answer it in a way that is difficult if not impossible in other areas of neurodisability for which we do not have valid and reliable functional classification systems.) Another possible ‘subgroup’ perspective that could come out of the proposed systematic review would be the ability to explore whether the ‘outcomes’ of people with CP vary by putative causes (e.g., antenatally-acquired disabilities, perinatally-acquired disabilities, impairment acquired after birth [perhaps even subdivided by the nature of the acquired injuries], etc.)

Describe the health-related benefits you are interested in. (For example, improvements in patient symptoms or problems from treatment or diagnosis.)

The benefits we expect to result from this proposed systematic review include the following ideas:

• We strongly recommend categorizing ALL the research findings according to the WHO’s International Classification of Functioning, Health and Disability (2001) [ICF] framework. By so doing we will be able to identify what areas of our understanding of CP need much more research. This information will be evidence-based rather than ‘eminence-based’.
• This recommendation is based on the reality that traditional interventions in CP have addressed some (but by no means all) ‘impairments’ (at the level of body structure and function), but have paid relatively less attention to functional outcomes (at the ‘activity’ level, such as fitness) and even less attention to ‘participation’ (engagement in life). By looking systematically at the literature in the ways proposed here we will gain perspectives about issues for which we have, at best, some vague impressions by no systematic understanding.
• We will learn what is known about a wide variety of ‘outcomes’ in CP – stratified by functional levels – be these outcomes reported in descriptive studies (clinical or epidemiological) or in intervention studies of any type.
• We will learn what aspects of function do, and do not, vary by ‘severity’ as described by GMFCS levels. This has both direct clinical and research implications.

Describe any health-related risks, side effects, or harms that you are concerned about.

There are no concerns about the studies that are being proposed in this systematic review.

Note, however, that many treatments are administered in childhood – surgery, intensive therapy, orthotics, medical therapies, etc. – however the impact of these treatments may include adverse effects that are not recognized until later. For example, an over-lengthening of the Achilles tendons during the early growth years may decreases the extension of the knee as the child grows and contribute to crouching; some of the adverse effects of Botulinum toxin are apparent early and appear to be related to severity. The burden of treatments as well as the potential outcome benefits, need to analyzed by GMFCS level.

Appropriateness for EHC Program

Does your question include a health care drug, intervention, device, or technology available (or likely to be available) in the U.S.?

yes

Which priority area(s) and population(s) does this topic apply to? (check all that apply)

EHC Priority Conditions (updated in 2008)

• Developmental delays, attention-deficit hyperactivity disorder, and autism
• Functional limitations and disability

AHRQ Priority Populations

• Low income groups
• Minority groups
• Children
• Individuals with special health care needs, including individuals with disabilities or who need chronic care or end-of-life health care

Federal Health Care Program

• Medicaid
• Medicare
• State Children's Health Insurance Program (SCHIP)
• Other

Importance

Describe why this topic is important.

If this study is completed as proposed, using the ICF as the framework to categorize all research in CP that has used the GMFCS, the world of childhood disability will be enormously enriched, as follows:

1. For the first time, we will have a ‘map’ of the research territory in CP, identifying what we know, what we need to start to explore, and ‘where’ in the ICF framework there are important opportunities for research (i.e., what aspects of health and function have been the focus of prior research and which have been less well studied).
2. The GMFCS is a powerfully useful way to describe a population of people with a common functional impairment (in this case, problems in gross motor development). Other fields (e.g., the clinical field of autism spectrum disorders) may find this approach to clinical categorization useful because of the way that all research in the field can be described through this common language.
3. By looking at several hundred research reports the systematic review will provide a considerable amount of information about what ‘works’ and doesn’t ‘work’ in CP, as studied and reported by GMFCS level. There will almost certainly be several scientific papers to be ‘extracted’ and published from this systematic review, reporting specific outcomes.

What specifically motivated you to ask this question? (For example, you are developing a clinical guideline, working with a policy with large uncertainty about the appropriate approach, costly intervention, new research you have read, items in the media you may have seen, a clinical practice dilemma you know of, etc.)

As leaders in the field of CP research and service (through our active engagement with the American Academy for Cerebral Palsy and Developmental Medicine [AACPDM]) we are aware of the need for a systematic overview of the research ‘territory’ in the field of CP. We believe that this proposal will challenge people to look at the literature that has appeared in the past decade and provide all of us with extremely valuable perspectives on what we know and what we need to know. We know that not every intervention we have available for people with CP is appropriate for every child, but in the absence of clear and systematic evidence from the literature it can be very challenging to either provide, or ‘withhold’, what is available. One consequence is that we see a lot of waste of resources, as well as poor outcomes that may well be associated with the inappropriate allocation of a ‘treatment’ to the ‘wrong’ kind of problem. For example, does a child with GMFCS level 5 CP (the most functionally limited group) benefit from physical therapy 3/week, as may be recommended in personal injury settlements (or by very strong parent advocates), when more specific and targeted interventions – using evidence extracted from a systematic review of the intervention literature – might prove more effective, even if they are provided with less ‘therapy’? These kinds of critical questions, if well explicated with the kind of systematic review we are proposing, could lead to evidence-based policy for decision-making at the clinical, educational and policy levels, as well as related social and resource utilization.

Does your question represent uncertainty for clinicians and/or policy-makers? (For example, variations in clinical care, controversy in what constitutes appropriate clinical care, or a policy decision.)

yes

If yes, please explain:

There is huge variation in care for children with CP across communities and countries. Policy concerning basic rehabilitation and medical services is often based on non-existent or, at best, inadequate research and the opinions of people with the loudest voices (be they clinicians, researchers or parents). As but a couple of examples of how research using the GMFCS already helps us clinically:

(i) We know from at least two important studies in different countries that the risk of hip dislocation in people with CP varies linearly across the five GMFCS levels, from 0% in level I [the most functional people with CP] to over 90% in people with level V CP. That information is powerfully important for clinicians assessing and following children and young adults with CP, for example with respect to the need (or not) for X-ray surveillance of the hip. This of course has considerable health services and economic implications for families and health care systems.

(ii) A second example concerns the lack of variation in self-reported quality of life by GMFCS level! This has implications at the clinical level and clearly is important for research that seeks to understand what factors do, and do not, appear to moderate people’s quality of life. It is also important for both parents and clinicians, who can too easily assume a ‘proportional’ relationship between increased ‘severity’ and lower quality of life.

Potential Impact

How will an answer to your research question be used or help inform decisions for you or your group?

AACPDM is the largest multidisciplinary international organization in the world that strives to improve the lives of children and adults with CP and other developmental disabilities through educational and research outcomes. We have a long history of developing and promoting the use of clinical guidelines. Our members constitute a major network of professionals that will provide credence and action for this ‘product’. We would anticipate a keynote presentation of the findings at an AACPDM Annual Meeting, a report to members in the AACPDM Newsletter, and likely several review articles reporting the findings in the AACPDM’s official journal Developmental Medicine and Child Neurology, the world’s leading childhood disability journal. As described throughout this proposal, this systematic review will provide a platform for the next generation of research in CP around the world! This serves the needs of researchers, but equally will guide how researchers develop their questions and the issues they explore in collaboration with families, to whom we believe the results of the systematic review should also (in our opinion) be shared. Needless to say the findings will be of considerable interest to granting agencies that are or might be interested to fund research concerning people with CP.

There are, as illustrated above, important clinical lessons to be learned and applied from the systematic review.

Describe the timeframe in which an answer to your question is needed.

The sooner the systematic review is completed the sooner the many stakeholders with an interest in CP will be able to act on its findings. Note as well that whatever is found will be useful, as long as the systematic review has been well designed and executed along the lines being suggested in this submission.

Describe any health disparities, inequities, or impact on vulnerable populations your question applies to.

There is considerable inequity in the availability and delivery of health care services for families of children in low socioeconomic (SES) circumstance in many parts of the US. Low birthweight (LBW) infants are disproportionately represented in this stratum of the community, and are many more times likely to have CP associated with the LBW status. The care that is available and provided to children with CP in low SES circumstances has considerable impact for these infants and families even before birth as well as after. Many children with CP in the disadvantaged neighborhoods are less likely to have access to therapy and other resources. It is therefore critical to understand the basic needs of people with CP by GMFCS levels as a basis for advocating for appropriate resources to promote their development and prevent secondary impairments.

Nominator Information

Other Information About You: (optional)

Please choose a description that best describes your role or perspective: (you may select more than one category if appropriate)

AACPDM is the largest multidisciplinary international organization in the world that strives to improve the lives of children and adults with CP and other developmental disabilities through educational and research outcomes. We have a long history of developing and promoting the use of clinical guidelines. Our members constitute a major network of professionals that will provide credence and action for this ‘product’. We would anticipate a keynote presentation of the findings at an AACPDM Annual Meeting, a report to members in the AACPDM Newsletter, and likely several review articles reporting the findings in the AACPDM’s official journal Developmental Medicine and Child Neurology, the world’s leading childhood disability journal. As described throughout this proposal, this systematic review will provide a platform for the next generation of research in CP around the world! This serves the needs of researchers, but equally will guide how researchers develop their questions and the issues they explore in collaboration with families, to whom we believe the results of the systematic review should also (in our opinion) be shared. Needless to say the findings will be of considerable interest to granting agencies that are or might be interested to fund research concerning people with CP.

There are, as illustrated above, important clinical lessons to be learned and applied from the systematic review.

Are you making a suggestion as an individual or on behalf of an organization?

Organization

Please tell us how you heard about the Effective Health Care Program

Many of us attended the session hosted by AHRQ October 26 2010 in Bethesda, MD, where we were introduced to the possibilities of systematic reviews and the opportunities for partnerships between AHRQ and people/groups like us.