The National Institutes of Health have released the results of their 1.5 day workshop on Myalgic Encephalomyelitis/Chronic Fatigue Syndrome. The full P2P workshop abstract is available online but some of the most interesting and important points are excerpted below. Leave a comment and tell us, what do you like about the report? What don’t you like?
Incidence, Prevalence, and Manifestations
There are no agreed-on variables for defining ME/CFS and no accurate ways to identify and diagnose it; as 1 speaker pointed out, the syndrome has 163 possible combinations of symptoms.
Patients with ME/CFS have neurocognitive dysfunction with abnormalities on functional magnetic resonance imaging and positron emission tomography. Strong evidence indicates that immunologic and inflammatory pathologic conditions, neurotransmitter signaling disruption, microbiome perturbation, and metabolic or mitochondrial abnormalities are potentially important for the definition and treatment of ME/CFS.
Ways to Foster Research and Enhance Development of Treatments
The following questions need to be answered: What is the pathogenesis of ME/CFS? What are the roles of virologic mechanisms, especially herpesviruses? Does mononucleosis lead to ME/CFS in adolescents? What are the roles of other pathogenic agents? Is this a genetic disease? Is there a gene–environment interaction? Is it a spectrum disease? Are different pathways responsible for different symptoms?
Future Directions and Recommendations
Overall, we have not implemented what we already know for patients with ME/CFS while the disease steals their health and well-being.
The influence of health literacy and cognitive impairment on informed consent must be considered.
The dissemination of diagnostic and therapeutic recommendations should begin by focusing on primary care providers and expand to other areas, such as neurology, rheumatology, and infectious disease.
1. Define Disease Variables
A team of stakeholders (such as patients, clinicians, researchers, and federal agencies) should be assembled to reach a consensus on the definition and variables of ME/CFS.
A national and international research network should be developed to clarify the case definition and advance the field.
2. Create New Knowledge
Developing biomarkers and diagnostic tests should be a priority.
Specific activities should focus on the following:
- Valid prognostic tests that can guide treatment strategies using genomic, epigenomic, proteomic, and metabolomic strategies to identify critical biomarkers that will be clinically applicable
- Functional magnetic resonance imaging and imaging technologies should be further studied as diagnostic tools and methods to better understand the neurologic dysfunction of ME/CFS.
- Biological samples (such as serum and saliva, RNA, DNA, whole blood or peripheral blood mononuclear cells, and tissues) and deidentified survey data should be linked in a registry or repository to understand pathogenesis and prognosis and facilitate biomarker discovery.
- Further exploration is needed of the intestinal microbiome and the effect, if any, of the environment and microbiome on ME/CFS development.
- Epidemiologic studies of ME/CFS, including incidence and prevalence, persons who are at high risk, risk factors, geographic distribution, and the identification of potential health care disparities
- Previously collected research data should be analyzed to advance knowledge and inform trial development and design and facilitate necessary clinical studies. In particular, drug therapies used for fibromyalgia or other pain-related syndromes and disorders should be examined for effectiveness in ME/CFS.
- Studies that stratify by clinical characteristics should be used to develop diagnostic and prognostic algorithms to identify who will develop ME/CFS after infection or other triggers.
- Longitudinal studies to explore the possibility of a progressive immune exhaustion or dysfunction
- Studies of identical twins to identify gene expression biomarkers are needed. Both male and female models must be used to explore the role of sex, X-chromosome genes, and hormones
- How patients’ background medications (including psychiatric drugs) affect function and outcome
- Studies investigating homeopathy, nonpharmacologic, complementary and alternative medicine treatments, and biopsychosocial variables (including the mind–body connection), function, and QoL
3. Improve Methods and Measures
The need for improved measures to identify ME/CFS while including the patient’s voice through patient-reported outcomes is critical.
Without a diagnostic test, stratification must occur to reduce and comprehend variability (such as onset, time course, and comorbid conditions) and to identify clearly defined end points for treatment trials and interventions.
Although ME/CFS is not a psychiatric disease, exploring psychiatric comorbid conditions, such as depression, anxiety, and fear, is critical to improve QoL.
Telemedicine or home visits for patients who cannot participate in clinical trials or treatment in person as well as outreach to underserved communities are needed.
4. Provide Training and Education
Many clinicians do not fully understand ME/CFS. We believe that it is a distinct disease that requires a multidisciplinary care team (such as physicians, nurses, case managers, social workers, and psychologists).
5. Finding New Funding Resources
With a relatively small number of researchers in the field and finite resources, partnerships across institutions are needed to advance the research and develop new scientists.
A network of collaborative centers working across institutions and disciplines, including clinical, biological, and social sciences, should be created.
A central archive of deidentified data and tissue samples from previous and ongoing studies to enable sharing of data and samples should be established.
6. Conduct Clinical Trials
The NIH should work with ME/CFS partners and stakeholders to create a Web site for patient and clinician educational materials as well as information about clinical trials.
Opportunities to use the NIH Clinical Center for clinical trials and fast-track testing of new therapies should also be explored.
7. Improve Treatment
Patients should be active participants in care and decision making.
The modest benefit from cognitive behavioral therapy should be studied as an adjunct to other methods.
Future treatment studies should evaluate multifaceted therapies focusing on biomedical and supportive care. Comparative effectiveness research is also needed.
We recommend that the NIH and U.S. Food and Drug Administration convene a meeting on the state of ME/CFS treatment.
Patients with ME/CFS want their stories to be heard, and the ME/CFS community may benefit from education on how to effectively communicate their concerns to clinicians. Clinicians could benefit from enhanced active listening skills and increased education.
Furthermore, the multiple case definitions for ME/CFS have hindered progress. In particular, continuing to use the Oxford definition may impair progress and cause harm. Therefore, for progress to occur, we recommend that this definition be retired; the ME/CFS community concur on a single case definition (even if it is not perfect); and patients, clinicians, and researchers agree on a definition for meaningful recovery.
The NIH should work with the Centers for Medicare & Medicaid Services and the Patient-Centered Outcomes Research Institute to develop demonstration projects of patient-centered medical homes for patients with ME/CFS.
We recommend that the NIH Office of Disease Prevention convene another expert panel in 5 years to monitor progress. We hope our work has dignified ME/CFS and affected persons while providing expert guidance to the NIH and the broader research community.
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