Tag: PROM

Patient-Reported Treatment Outcomes in ME/CFS and Long COVID

Abstract:

Myalgic encephalomyelitis/chronic fatigue syndrome (ME/CFS) and Long COVID are persistent multi-system illnesses affecting many patients. With no known effective FDA-approved treatments for either condition, patient-reported outcomes of treatments are invaluable for guiding management strategies in patient care and generating new avenues for research. Here, we present the results of an ME/CFS and Long COVID treatment survey with responses from 3,925 patients.

We assessed the experiences of these patients with more than 150 treatments, as well as their demographics, symptoms, and comorbidities. Patients with each condition who participated in the study shared similar symptom profiles, including all the core symptoms of ME/CFS, e.g., 89.7% of ME/CFS and 79.4% of Long COVID reported post-exertional malaise (PEM). Treatments with the greatest perceived benefits were identified, which had varied effects on different core symptoms.

In addition, treatment responses were significantly correlated (R² = 0.68) between the two patient groups. Patient subgroups with distinct profiles of symptoms and comorbidities showed varied responses to treatments, e.g., a POTS-dominant cluster benefiting from autonomic modulators and a cognitive-dysfunction cluster from CNS stimulants.

This study underscores the symptomatic and therapeutic similarities between ME/CFS and Long COVID and highlights the commonalities and nuanced complexities of infection-associated chronic diseases and related conditions. Insights from patient-reported experiences, in the absence of approved treatments, provide urgently needed real-world evidence for targeted therapies in patient care and for developing future clinical trials.

Source: Martha Eckey, Peng Li, Braxton Morrison, Ronald W Davis, Wenzhong Xiao. Patient-Reported Treatment Outcomes in ME/CFS and Long COVID. medRxiv 2024.11.27.24317656; doi: https://doi.org/10.1101/2024.11.27.24317656 https://www.medrxiv.org/content/10.1101/2024.11.27.24317656v1.full-text (Full text)

A pilot cross-sectional investigation of symptom clusters and associations with patient-reported outcomes in Myalgic Encephalomyelitis/Chronic Fatigue Syndrome and Post COVID-19 Condition

Abstract:

Background: Myalgic Encephalomyelitis/Chronic Fatigue Syndrome (ME/CFS) is associated with long-term disability and poor quality of life (QoL). Cardinal ME/CFS symptoms (including post-exertional malaise, cognitive dysfunction and sleep disturbances) have been observed in Post COVID-19 Condition (PCC). To gain further insight into the potential role of ME/CFS as a post-COVID-19 sequela, this study investigates associations between symptoms and patient-reported outcomes, as well as symptom clusters.
Methods: Participants included Australian residents aged between 18 and 65 years formally diagnosed with ME/CFS fulfilling the Canadian or International Consensus Criteria or PCC meeting the World Health Organization case definition. Validated, self-administered questionnaires collected participants’ sociodemographic and illness characteristics, symptoms, QoL and functional capacity. Associations between symptoms and patient-reported outcomes were investigated with multivariate linear regression models. Hierarchical cluster analysis was performed to identify symptom clusters.
Results: Most people with ME/CFS (pwME/CFS) and people with PCC (pwPCC) were female (n = 48/60, 80.0% and n = 19/30, 63.3%, respectively; p = 0.12). PwME/CFS were significantly younger (x̄=41.75, s = 12.91 years) than pwPCC (x̄=48.13, s =10.05 years; p =0.017). Autonomic symptoms (notably dyspnoea) were associated with poorer scores in most patient-reported outcome domains for both cohorts. None of the four symptom clusters identified were unique to ME/CFS or PCC. Clusters were largely delineated by the presence of gastrointestinal and neurosensory symptoms, illness duration, ME/CFS criteria met and total symptoms.
Conclusions: Illness duration may explain differences in symptom burden between pwME/CFS and pwPCC. PCC diagnostic criteria must be refined to distinguish pwPCC at risk of long-term ME/CFS-like illness and subsequently deliver necessary care and support.
Source: Weigel B, Eaton-Fitch N, Thapaliya K, Marshall-Gradisnik S. A pilot cross-sectional investigation of symptom clusters and associations with patient-reported outcomes in Myalgic Encephalomyelitis/Chronic Fatigue Syndrome and Post COVID-19 Condition. Qual Life Res. 2024 Oct 3. doi: 10.1007/s11136-024-03794-x. Epub ahead of print. PMID: 39361124. https://link.springer.com/article/10.1007/s11136-024-03794-x (Full text)

 

A National Evaluation of Outcomes in Long COVID Services using Digital PROM Data from the ELAROS Platform

Summary:

Key findings of this service evaluation study:
• Patient characteristics: A sample of 5,318 patients from 14 participating NHS LC sites were analysed. The sample had a female:male ratio of 2.1:1. The average age was 48.4 yrs, with 87% (of those whose ethnicity was recorded) of white ethnicity and 9% of Black or Asian ethnicity.
• Comorbidities: This sample of patients had a low prevalence of co-morbidities (7%) with a clear onset of new LC symptoms after their COVID-19 infection supporting the onset of a new condition in this cohort of previously healthy individuals.
• Duration of LC: The average duration of LC in this sample was 384 days (>12 months) at first assessment in an LC site, with symptoms still ongoing at presentation, with more than 90% of the sample being non-hospitalised patients.
• Digital platform: A total of 17,471 PROMs (C19-YRS and EQ-5D-5L) were completed by this sample of patients with at least 1,532 participants completing multiple assessments on the same PROM on the digital PROM platform. The completion of PROMs around the 3-month mark was low for both measures (11.7% for C19-YRS and 14.6% for EQ-5D-5L). The ones who completed PROMs both around the 3-month mark and the 6-month mark were 4.3% for C19-YRS and 5% for EQ-5D-5L. This limits the generalisability of the findings in this evaluation to all the LC population, but the findings remain valid for this cohort of individuals.
• New-onset disability: 3,395 patients who completed at least one C19-YRS questionnaire at first assessment showed significant new-onset symptom burden, functional disability, and deterioration of overall health since the COVID-19 infection.
• Comparison between LC and other chronic conditions: The cross-sectional EQ-5D-5L Index value of 3,438 patients suggests the burden and disability in LC are worse than that reported in the literature for Diabetes Mellitus, COPD, Heart Failure, and Multiple Sclerosis.
• 3-month follow-up: Among those who completed an initial C19-YRS assessment and another at 3 months, there was a statistically significant improvement in symptom burden, functional disability and overall health. Patients at 3 months however still had significant LC symptom
burden and disability compared to their pre-COVID-19 health status, i.e., their condition had improved, but they were far off from a complete recovery. Among those who completed EQ-5D- National Evaluation of Long COVID Service Outcomes using ELAROS Data (09 Oct 23) Page 3 of 31
5L, at first assessment and at 3 months, their EQ-5D-5L Index score did not show any statistically significant improvement, but the EQ-5D-5L VAS showed a statistically significant improvement.
• 6-month follow-up: Among those who completed measures at the first assessment, 3 months, and 6 months, C19-YRS and EQ-5D-5L VAS showed statistically significant improvement whereas EQ-5D-5L Index Value showed statistically significant deterioration. Patients at 6 months still had
significant LC symptom burden and disability compared to their pre-COVID-19 health status, i.e., their condition had improved but had not fully recovered. The follow-up changes in scores support the efficacy of interventions provided by LC services and suggest that continued specialist input is needed to manage these patients with persistent symptoms.
• C19-YRS (condition-specific measure) vs EQ-5D-5L (generic measure): The 3-month month follow-up changes in scores and responsiveness of PROMs highlight that C19-YRS is a more sensitive measure than EQ-5D-5L in this cohort of individuals with LC. This is in keeping with the literature recommending the use of condition-specific measures in addition to EQ-5D-5L.
• Vocational problems: 62% of this sample had their work role affected with them having to either be on sick leave, reduce hours, change roles, or quit roles. Only 21% were able to maintain their previous roles held prior to their COVID-19 infection. This is suggestive of considerable productivity loss and financial implications to the country.
• Fluctuating condition: In patients who completed multiple assessments, it was evident that LC is a fluctuant condition with no necessary linear trend of improvement or deterioration between the domains of symptom burden, functional disability, and overall health. This highlights the need to understand the triggers for the condition and invest in self-management and ongoing support from community healthcare services.
• Long-Term Condition: In most patients in this sample, LC has evidently become a Long-Term Condition (LTC) with fluctuations in their condition causing disability and significant deterioration of their overall health status seen even after 18 months of LC with no complete resolution or full recovery. There needs to be a national investment in managing this new LTC along with other LTCs.

Source: Dr Manoj Sivan, et al.  A National Evaluation of Outcomes in Long COVID Services using Digital PROM Data from the ELAROS Platform. National Evaluation of Long COVID Service Outcomes using ELAROS Data (09 Oct 23) https://locomotion.leeds.ac.uk/wp-content/uploads/sites/74/2023/10/National-Evaluation-of-LC-Service-Outcomes-using-ELAROS-Data-09-10-23.pdf (Full text)

Myalgic Encephalomyelitis-Chronic Fatigue Syndrome Common Data Element item content analysis

Abstract:

Introduction: Myalgic Encephalomyelitis/Chronic Fatigue Syndrome (ME/CFS) is a multisystem chronic disease estimated to affect 836,000-2.5 million individuals in the United States. Persons with ME/CFS have a substantial reduction in their ability to engage in pre-illness levels of activity. Multiple symptoms include profound fatigue, post-exertional malaise, unrefreshing sleep, cognitive impairment, orthostatic intolerance, pain, and other symptoms persisting for more than 6 months. Diagnosis is challenging due to fluctuating and complex symptoms. ME/CFS Common Data Elements (CDEs) were identified in the National Institutes of Health (NIH) National Institute of Neurological Disorders and Stroke (NINDS) Common Data Element Repository. This study reviewed ME/CFS CDEs item content.

Methods: Inclusion criteria for CDEs (measures recommended for ME/CFS) analysis: 1) assesses symptoms; 2) developed for adults; 3) appropriate for patient reported outcome measure (PROM); 4) does not use visual or pictographic responses. Team members independently reviewed CDEs item content using the World Health Organization International Classification of Functioning, Disability and Health (ICF) framework to link meaningful concepts.

Results: 119 ME/CFS CDEs (measures) were reviewed and 38 met inclusion criteria, yielding 944 items linked to 1503 ICF meaningful concepts. Most concepts linked to ICF Body Functions component (b-codes; n = 1107, 73.65%) as follows: Fatiguability (n = 220, 14.64%), Energy Level (n = 166, 11.04%), Sleep Functions (n = 137, 9.12%), Emotional Functions (n = 131, 8.72%) and Pain (n = 120, 7.98%). Activities and Participation concepts (d codes) accounted for a smaller percentage of codes (n = 385, 25.62%). Most d codes were linked to the Mobility category (n = 69, 4.59%) and few items linked to Environmental Factors (e codes; n = 11, 0.73%).

Discussion: Relatively few items assess the impact of ME/CFS symptoms on Activities and Participation. Findings support development of ME/CFS-specific PROMs, including items that assess activity limitations and participation restrictions. Development of psychometrically-sound, symptom-based item banks administered as computerized adaptive tests can provide robust assessments to assist primary care providers in the diagnosis and care of patients with ME/CFS.

Source: Slavin MD, Bailey HM, Hickey EJ, Vasudevan A, Ledingham A, Tannenbaum L, Bateman L, Kaufman DL, Peterson DL, Ruhoy IS, Systrom DM, Felsenstein D, Kazis LE. Myalgic Encephalomyelitis-Chronic Fatigue Syndrome Common Data Element item content analysis. PLoS One. 2023 Sep 12;18(9):e0291364. doi: 10.1371/journal.pone.0291364. PMID: 37698999. https://journals.plos.org/plosone/article?id=10.1371/journal.pone.0291364 (Full text)

Developing and pretesting a new patient reported outcome measure for paediatric Chronic Fatigue Syndrome/ Myalgic Encephalopathy (CFS/ME): cognitive interviews with children.

Abstract:

BACKGROUND: There is a lack of patient derived, child specific outcome measures to capture what health outcomes are important to children with Chronic Fatigue Syndrome/ Myalgic Encephalopathy (CFS/ME). We developed a new Patient Reported Outcome Measure (PROM) for paediatric CFS/ME through qualitative research with children. This study aimed to pre-test the new measure through cognitive interviews with children with CFS/ME.

METHODS: Cognitive interviews were undertaken in children’s homes or over Skype. The Three-Step Test-Interview (TSTI) method was used to assess the quality of the draft PROM with children with CFS/ME to identify problems with initial content and design and test modifications over subsequent interview rounds. Children were purposively sampled from a single specialist paediatric CFS/ME service in England.

RESULTS: Twenty-four children and their parents took part. They felt the new measure captured issues relevant to their condition and preferred it to the generic measures they completed in clinical assessment. Changes were made to item content and phrasing, timeframe and response options and tested through three rounds of interviews.

CONCLUSIONS: Cognitive interviews identified problems with the draft PROM, enabling us to make changes and then confirm acceptability in children aged 11-18. Further cognitive interviews are required with children 8-10 years old to examine the acceptability and content validity and provide evidence for age related cut offs of the new PROM to meet FDA standards. This study demonstrates the content validity of the new measure as relevant and acceptable for children with CFS/ME. The next stage is to undertake a psychometric evaluation to support the reduction of items, confirm the structure of the PROM and provide evidence of the data quality, reliability and validity.

Source: Parslow RM, Shaw A, Haywood KL, Crawley E. Developing and pretesting a new patient reported outcome measure for paediatric Chronic Fatigue Syndrome/ Myalgic Encephalopathy (CFS/ME): cognitive interviews with children. J Patient Rep Outcomes. 2019 Nov 9;3(1):67. doi: 10.1186/s41687-019-0156-8. https://www.ncbi.nlm.nih.gov/pubmed/31707635

Chronic fatigue syndrome and quality of life

Abstract:

Chronic fatigue syndrome (CFS), also known as myalgic encephalomyelitis (ME), is a challenging long-term condition (LTC) with complex and fluctuating symptoms. It is heterogeneous in presentation without diagnostic indicators; therefore, in health care encounters, insight must be gained from the patient’s perspective. One indicator of impact can be gained by measuring quality of life (QoL). By applying a patient-reported outcome measure (PROM), professionals can gather insights with direct relevance to the patient questioned. Such a tool can act therapeutically tool to promote holistic and individualized professional interventions and interval measurement can inform commissioning of specialist services.

Standard practice appears not fully to capture the experience of CFS, while a search of the literature turned up QoL patient-reported outcome tools, but failed to reveal a CFS/ME-specific measure. The author explores a valid and reliable PROM that can monitor change and evaluate the UK National Institute of Clinical Excellence rehabilitation program, as delivered by specialist National Health Service units. An alternative, the World Health Organization’s quality-of life instrument (WHOQoL)-Bref26, is reviewed for relevance to the condition, measuring treatment outcomes and the wider debate of measuring QoL in LTCs.

Source: Roberts D. Chronic fatigue syndrome and quality of life. Patient Relat Outcome Meas. 2018 Aug 1;9:253-262. doi: 10.2147/PROM.S155642. eCollection 2018. https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6078083/ (Full article)

What matters to children with CFS/ME? A conceptual model as the first stage in developing a PROM

Abstract:

BACKGROUND: Paediatric chronic fatigue syndrome (CFS)/myalgic encephalomyelitis (ME) is relatively common and disabling. Research is hampered because current patient-reported outcome measures (PROMs) do not capture outcomes that are important to children with CFS/ME.

AIM: The aim of this study was to explore the aspects of life and health outcomes that matter to children with CFS/ME.

METHODS: Twenty-five children with CFS/ME were interviewed (11 males, 14 females; mean age 12.9 years (SD 2.2), range 8-17). Twelve were trial participants interviewed during the trial and 13 were recruited as part of a follow-up qualitative study. Parents were present in 19 interviews with their children. Three mothers participated in a focus group. All the interviews and the focus group were audio-recorded and transcribed. Data were analysed thematically using techniques of constant comparison. NVivo was used to structure and categorise data in a systematic way.

RESULTS: Children identified four key themes (health outcome domains): ‘symptoms’ that fluctuated, which caused an unpredictable reduction in both ‘physical activity’ and ‘social participation’ all of which impacted on ’emotional well-being’. These domains were influenced by both ‘management’ and ‘contextual factors’, which could be positive and negative. The relationship between healthcare and school was considered pivotal.

CONCLUSIONS: Children’s descriptions helped to inform a conceptual model that is necessary to develop a new paediatric CFS/ME PROM. Doctors need to be aware of how children conceptualise CFS/ME; the relationship between healthcare and school is fundamental to ameliorate the impact of CFS/ME.

TRIAL REGISTRATION NUMBER: ISRCTN81456207.

Published by the BMJ Publishing Group Limited. For permission to use (where not already granted under a licence) please go to http://www.bmj.com/company/products-services/rights-and-licensing/

 

Source: Parslow R, Patel A, Beasant L, Haywood K, Johnson D, Crawley E. What matters to children with CFS/ME? A conceptual model as the first stage in developing a PROM. Arch Dis Child. 2015 Dec;100(12):1141-7. doi: 10.1136/archdischild-2015-308831. Epub 2015 Oct 9. https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4680202/ (Full article)

 

Quality and acceptability of patient-reported outcome measures used in chronic fatigue syndrome/myalgic encephalomyelitis (CFS/ME): a systematic review

Abstract:

PURPOSE: To review the quality and acceptability of condition-specific, domain-specific and generic multi-item patient-reported outcome measures (PROMs) used in the assessment of adults with chronic fatigue syndrome/myalgic encephalomyelitis (CFS/ME).

METHODS: Systematic literature searches were made to identify PROMs. Quality and acceptability was assessed against an appraisal framework, which captured evidence of both the thoroughness and results of evaluations: evidence of measurement (reliability, validity, responsiveness, interpretability, data quality/precision) and practical properties (feasibility, patient acceptability), and the extent of active patient involvement was sought.

RESULTS: A total of 11 CFS/ME-specific, 55 domain-specific and 11 generic measures were reviewed. With the exception of the generic SF-36, all measures had mostly limited evidence of measurement and/or practical properties. Patient involvement was poorly reported and often cursory.

CONCLUSIONS: The quality and acceptability of reviewed PROMs is limited, and recommendations for patient-reported assessment are difficult. Significant methodological and quality issues in PROM development/evaluation were identified by the appraisal framework, which must be addressed in future research. Clear discrepancies exist between what is measured in research and how patients define their experience of CFS/ME. Future PROM development/evaluation must seek to involve patients more collaboratively to measure outcomes of importance using relevant and credible methods of assessment.

 

Source: Haywood KL, Staniszewska S, Chapman S. Quality and acceptability of patient-reported outcome measures used in chronic fatigue syndrome/myalgic encephalomyelitis (CFS/ME): a systematic review. Qual Life Res. 2012 Feb;21(1):35-52. doi: 10.1007/s11136-011-9921-8. Epub 2011 May 18. https://www.ncbi.nlm.nih.gov/pubmed/21590511