Skip to main content

Advertisement

SpringerLink
Log in

Patient reported outcome measures (PROMs) in amyotrophic lateral sclerosis

  • Original Communication
  • Published:
Journal of Neurology Aims and scope Submit manuscript

Abstract

Objective

Patient reported outcome measures (PROMs) can provide researchers with a direct view of patients’ experiences. They are becoming increasingly important tools for evaluating clinical care and research outcomes. There has been little data on the application of PROMs to amyotrophic lateral sclerosis (ALS) care. The objective of this study was to examine the feasibility of PROM collection in an academic ALS clinic and to measure correlations between PROMs and standard ALS clinical outcome measures.

Methods

PROMs were gathered from tablet-based surveys offered to adult patients in the waiting room, prior to ALS outpatient clinic visits. They included a demographic section and two validated surveys: the patient reported outcome measurement information system (PROMIS-10), which generates physical health and mental health subscores, and the quality of life in neurological disorders-fatigue subscale (NeuroQoL-fatigue). The ALS functional rating scale-revised (ALSFRS-R) and other ALS measures were collected by clinic staff as part of routine clinical care.

Results

PROMIS-10 physical and mental health scores correlated positively with the ALSFRS-R score (physical: R = 0.85, p < 0.001; mental: R = 0.58, p = 0.02). NeuroQoL-fatigue scores were inversely correlated with the ALSFRS-R scores—higher fatigue correlated with lower function (R = − 0.72, p = 0.004).

Conclusion

Collection of PROMs is feasible in the context of routine ALS care. PROM scores are highly correlated with validated ALS outcome measures.

This is a preview of subscription content, log in via an institution to check access.

Access this article

Log in via an institution

Price excludes VAT (USA)
Tax calculation will be finalised during checkout.

Instant access to the full article PDF.

Institutional subscriptions

Fig. 1
Fig. 2
Fig. 3

Similar content being viewed by others

Data availability

The datasets generated during analysis of this study are available from the corresponding author on reasonable request.

References

  1. Cella D, Yount S, Rothrock N et al (2007) The Patient-Reported Outcomes Measurement Information System (PROMIS): progress of an NIH Roadmap cooperative group during its first two years. Med Care 45:S3–S11

    Article  Google Scholar 

  2. Cella D, Riley W, Stone A et al (2010) The Patient-Reported Outcomes Measurement Information System (PROMIS) developed and tested its first wave of adult self-reported health outcome item banks: 2005–2008. J Clin Epidemiol 63:1179–1194

    Article  Google Scholar 

  3. Moura LM, Schwamm E, Moura Junior V et al (2016) Feasibility of the collection of patient-reported outcomes in an ambulatory neurology clinic. Neurology 87:2435–2442

    Article  Google Scholar 

  4. Schaffler N, Schonberg P, Stephan J, Stellmann JP, Gold SM, Heesen C (2013) Comparison of patient-reported outcome measures in multiple sclerosis. Acta Neurol Scand 128:114–121

    Article  CAS  Google Scholar 

  5. Heatwole C, Bode R, Johnson N et al (2012) Patient-reported impact of symptoms in myotonic dystrophy type 1 (PRISM-1). Neurology 79:348–357

    Article  Google Scholar 

  6. Heatwole C, Johnson N, Bode R et al (2015) Patient-reported impact of symptoms in myotonic dystrophy type 2 (PRISM-2). Neurology 85:2136–2146

    Article  CAS  Google Scholar 

  7. Jacobi H, du Montcel ST, Bauer P et al (2018) Long-term evolution of patient-reported outcome measures in spinocerebellar ataxias. J Neurol 265:2040–2051

    Article  Google Scholar 

  8. Mongiovi P, Dilek N, Garland C et al (2018) Patient reported impact of symptoms in spinal muscular atrophy (PRISM-SMA). Neurology 91:e1206–e1214

    Article  Google Scholar 

  9. Abdulla S, Vielhaber S, Korner S et al (2013) Validation of the German version of the extended ALS functional rating scale as a patient-reported outcome measure. J Neurol 260:2242–2255

    Article  Google Scholar 

  10. Smith RA, Macklin EA, Myers KJ et al (2018) Assessment of bulbar function in amyotrophic lateral sclerosis: validation of a self-report scale (Center for Neurologic Study Bulbar Function Scale). Eur J Neurol 25:907

    Article  CAS  Google Scholar 

  11. Stephens HE, Joyce NC, Oskarsson B (2017) National study of muscle cramps in ALS in the USA. Amyotroph Lateral Scler Frontotemporal Degener 18:32–36

    Article  Google Scholar 

  12. Vogt S, Petri S, Dengler R, Heinze HJ, Vielhaber S (2018) Dyspnea in amyotrophic lateral sclerosis (ALS): Rasch-based development and validation of a patient-reported outcome (DALS-15). J Pain Symptom Manage 56:736–745

    Article  Google Scholar 

  13. Cedarbaum JM, Stambler N, Malta E et al (1999) The ALSFRS-R: a revised ALS functional rating scale that incorporates assessments of respiratory function. BDNF ALS Study Group (Phase III). J Neurol Sci 169:13–21

    Article  CAS  Google Scholar 

  14. Gordon PH, Miller RG, Moore DH (2004) Alsfrs-R. Amyotroph Lateral Scler Other Motor Neuron Disord 5(Suppl 1):90–93

    Article  Google Scholar 

  15. Andrews JA, Meng L, Kulke SF et al (2018) Association between decline in slow vital capacity and respiratory insufficiency, use of assisted ventilation, tracheostomy, or death in patients with amyotrophic lateral sclerosis. JAMA Neurol 75:58–64

    Article  Google Scholar 

  16. Shefner JM, Liu D, Leitner ML et al (2016) Quantitative strength testing in ALS clinical trials. Neurology 87:617–624

    PubMed  PubMed Central  Google Scholar 

  17. Ludolph A, Drory V, Hardiman O et al (2015) A revision of the El Escorial criteria—2015. Amyotroph Lateral Scler Frontotemporal Degener 16(5–6):291–292

    Article  Google Scholar 

  18. Broderick JE, DeWitt EM, Rothrock N, Crane PK, Forrest CB (2013) Advances in patient-reported outcomes: the NIH PROMIS((R)) measures. EGEMS (Wash DC) 1:1015

    Google Scholar 

  19. Hays RD, Bjorner JB, Revicki DA, Spritzer KL, Cella D (2009) Development of physical and mental health summary scores from the patient-reported outcomes measurement information system (PROMIS) global items. Qual Life Res 18:873–880

    Article  Google Scholar 

  20. Cella D, Lai JS, Nowinski CJ et al (2012) Neuro-QOL: brief measures of health-related quality of life for clinical research in neurology. Neurology 78:1860–1867

    Article  CAS  Google Scholar 

  21. Gershon RC, Lai JS, Bode R et al (2012) Neuro-QOL: quality of life item banks for adults with neurological disorders: item development and calibrations based upon clinical and general population testing. Qual Life Res 21:475–486

    Article  Google Scholar 

  22. Knudson RJ, Lebowitz MD, Holberg CJ, Burrows B (1983) Changes in the normal maximal expiratory flow-volume curve with growth and aging. Am Rev Respir Dis 127:725–734

    CAS  PubMed  Google Scholar 

  23. Gibbons C, Pagnini F, Friede T, Young CA (2018) Treatment of fatigue in amyotrophic lateral sclerosis/motor neuron disease. Cochrane Database Syst Rev 1:CD011005

    PubMed  Google Scholar 

  24. Krupp LB, LaRocca NG, Muir-Nash J, Steinberg AD (1989) The fatigue severity scale. Application to patients with multiple sclerosis and systemic lupus erythematosus. Arch Neurol 46:1121–1123

    Article  CAS  Google Scholar 

  25. Paganoni S, Cudkowicz M, Berry JD (2014) Outcome measures in amyotrophic lateral sclerosis clinical trials. Clin Investig (Lond) 4:605–618

    Article  CAS  Google Scholar 

  26. Kelly L, Jenkinson C, Morley D (2015) Outcome measurement in neurodegenerative disease: attributes, applications & interpretation. Neurodegener Dis Manag 5:305–316

    Article  Google Scholar 

  27. Dirven L, Taphoorn MJ, Reijneveld JC et al (2014) The level of patient-reported outcome reporting in randomised controlled trials of brain tumour patients: a systematic review. Eur J Cancer 50:2432–2448

    Article  Google Scholar 

  28. Jonsson AC, Delavaran H, Iwarsson S, Stahl A, Norrving B, Lindgren A (2014) Functional status and patient-reported outcome 10 years after stroke: the Lund Stroke Register. Stroke 45:1784–1790

    Article  Google Scholar 

  29. Markman JD, Gewandter JS, Frazer ME et al (2015) Evaluation of outcome measures for neurogenic claudication: a patient-centered approach. Neurology 85:1250–1256

    Article  Google Scholar 

Download references

Acknowledgements

We would like to thank all study participants and their families and caregivers for their dedication and contribution to ALS research.

Funding

No targeted funding.

Author information

Author notes

  1. Fabiola De Marchi and James D. Berry contributed equally to the manuscript.

Authors and Affiliations

  1. Healey Center for ALS, Department of Neurology, Massachusetts General Hospital, Harvard Medical School, 165 Cambridge St, Suite 600, Boston, MA, USA

    Fabiola De Marchi, James D. Berry, Sarah Caldwell, Amy Ellrodt, Jennifer Scalia, Katherine Burke, Ton Fang & Sabrina Paganoni

  2. Massachusetts General Hospital, Biostatistics Center, Harvard Medical School, Boston, MA, USA

    James Chan

  3. Massachusetts General Physicians Organization, Harvard Medical School, MGH Partners Healthcare, Somerville, MA, USA

    Rachel Clark Sisodia

  4. Department of Neurology, Massachusetts General Hospital, Harvard Medical School, Boston, MA, USA

    Lee H. Schwamm & Lidia M. V. R. Moura

  5. Department of Physical Medicine and Rehabilitation, Spaulding Rehabilitation Hospital, Harvard Medical School, Boston, MA, USA

    Sabrina Paganoni

Authors
  1. Fabiola De Marchi
    View author publications

    You can also search for this author in PubMed Google Scholar

  2. James D. Berry
    View author publications

    You can also search for this author in PubMed Google Scholar

  3. James Chan
    View author publications

    You can also search for this author in PubMed Google Scholar

  4. Sarah Caldwell
    View author publications

    You can also search for this author in PubMed Google Scholar

  5. Amy Ellrodt
    View author publications

    You can also search for this author in PubMed Google Scholar

  6. Jennifer Scalia
    View author publications

    You can also search for this author in PubMed Google Scholar

  7. Katherine Burke
    View author publications

    You can also search for this author in PubMed Google Scholar

  8. Ton Fang
    View author publications

    You can also search for this author in PubMed Google Scholar

  9. Rachel Clark Sisodia
    View author publications

    You can also search for this author in PubMed Google Scholar

  10. Lee H. Schwamm
    View author publications

    You can also search for this author in PubMed Google Scholar

  11. Lidia M. V. R. Moura
    View author publications

    You can also search for this author in PubMed Google Scholar

  12. Sabrina Paganoni
    View author publications

    You can also search for this author in PubMed Google Scholar

Corresponding author

Correspondence to Sabrina Paganoni.

Ethics declarations

Conflicts of interest

Fabiola De Marchi: reports no disclosure. James D. Berry has been a consultant to Denali Therapeutics and has research funding from Amylyx Pharmaceuticals, Anelixis Pharmaceuticals, Biogen, Brainstorm Cell Therapeutics, Genentech, MT Pharma of America, ALS Association, ALS Finding A Cure, ALS One, EGL Foundation, and Muscular Dystrophy Association. James Chan: reports no disclosure. Sarah Caldwell: reports no disclosure. Amy Ellrodt: reports no disclosure. Jennifer Scalia: reports no disclosure. Katherine Burke: reports no disclosure. Ton Fang: reports no disclosure. Rachel Clark Sisodia: reports no disclosure. Lidia M.V.R. Moura: reports no disclosure. Lee Schwamm: reports no disclosure. Sabrina Paganoni has received research funding from the Salah Foundation, the Spastic Paraplegia Foundation, the ALS Association, ALS Finding a Cure, the American Academy of Neurology and Amylyx.

Ethical statement

This study has been approved by the appropriate ethics committee and has, therefore, been performed in accordance with the ethical standards (Declaration of Helsinki). In particular, this retrospective chart review and analysis was approved by the Partners Healthcare Human Research Committee.

Rights and permissions

Reprints and permissions

About this article

Check for updates. Verify currency and authenticity via CrossMark

Cite this article

De Marchi, F., Berry, J.D., Chan, J. et al. Patient reported outcome measures (PROMs) in amyotrophic lateral sclerosis. J Neurol 267, 1754–1759 (2020). https://doi.org/10.1007/s00415-020-09774-8

Download citation

  • Received:

  • Revised:

  • Accepted:

  • Published:

  • Issue Date:

  • DOI: https://doi.org/10.1007/s00415-020-09774-8

Keywords

Search

Navigation