Der Einsatz von Patient-reported Outcome Measures (PROM) und die Perspektive digitaler Biomarker bei der Amyotrophen Lateralsklerose

Zusammenfassung

Die systematische Erfassung des klinischen Zustands sowie der Erfahrung mit Behandlung oder Versorgung durch einen strukturierten Bericht des Patienten wird als „Patient-reported Outcome Measures“ (PROM) bezeichnet. Bei der Amyotrophen Lateralsklerose (ALS) haben sich PROM insbesondere zur Dokumentation funktioneller Defizite, z. B. mit der ALS-Funktionsskala, und weiterer komplexer Symptome im Rahmen von klinischer Forschung etabliert. In der Behandlungspraxis werden PROM dazu genutzt, den Verlauf und die Prognose der Erkrankung einzuschätzen. Mit PROM werden neue biologische Biomarker (z. B. Neurofilamente) und digitale Biomarker (z. B. durch den Einsatz von Sensorik) auf ihre patientenzentrierte Relevanz evaluiert. Durch die digitale Anwendung von PROM und die Verknüpfung mit digitalen Biomarkern kann eine engmaschigere Erhebung von zu Hause aus erfolgen und damit die Datenqualität erhöht werden. Patienten können selbst den Gesundheitszustand monitorieren sowie Behandlungs- und Versorgungsergebnisse dokumentieren. Damit nehmen sie zunehmend eine aktive Rolle in der individuellen Behandlung und Versorgung ein.

Abstract

The systematic assessment of clinical conditions, experience with treatment or healthcare by a structured report of the patient is referred to as "Patient-reported Outcome Measures" (PROM). In amyotrophic lateral sclerosis (ALS), PROM are particularly well established for documenting functional deficits, e. g. with the ALS Functional Rating Scale, and additional complex symptoms in the context of clinical research. In clinical setting, PROM are used to assess the course and prognosis of the disease. Using PROM, new biological biomarkers (e. g., neurofilaments) and digital biomarkers (e. g., using sensor technology) are being evaluated regarding patient-centric relevance. The digital application of PROM and its linkage to digital biomarkers allows for a more densely collected data from home, enhancing the quality of the data. Patients are able to monitor their own health status and document the results of treatment and care. Thus, they take an increasingly active role in individual care management.

Schlüsselwörter

ALS - PROM - Selbstbewertung - digitale Biomarker - Amyotrophe Lateralsklerose

Key words

Patient-reported outcome measures - amyotrophic lateral sclerosis - digital biomarker - PROM - ALSFRS-R

• Literatur

• 1 Kühnlein P, Kübler A, Raubold S. et al. Palliative care and circumstances of dying in German ALS patients using non-invasive ventilation. Amyotrophic Lateral Sclerosis 2009; 9: 91-98
• 2 Doward LC, Gnanasakthy A, Baker MG.. Patient reported outcomes: looking beyond the label claim. Health and Quality of Life Outcomes 2010; 8
• 3 De Marchi F, Berry JD, Chan J. et al. Patient reported outcome measures (PROMs) in amyotrophic lateral sclerosis. Journal of Neurology 2020; 267: 1754-1759
• 4 Steinbeck V, Ernst S-C, Pross C.. Patient-Reported Outcome Measures (PROMs): ein internationaler Vergleich. Bertellsmann Stiftung: Gütersloh; 2021
• 5 Deshpande P, Sudeepthi B, Rajan S. et al. Patient-reported outcomes: A new era in clinical research. Perspectives in Clinical Research 2011; 2
• 6 Cedarbaum JM, Stambler N, Malta E. et al. The ALSFRS-R: a revised ALS functional rating scale that incorporates assessments of respiratory function. Journal of the Neurological Sciences 1999; 169: 13-21
• 7 Kaufmann P, Levy G, Montes J. et al. Excellent inter-rater, intra-rater, and telephone-administered reliability of the ALSFRS-R in a multicenter clinical trial. Amyotrophic Lateral Sclerosis 2009; 8: 42-46
• 8 McElhiney M, Rabkin JG, Goetz R. et al. Seeking a measure of clinically meaningful change in ALS. Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration 2014; 15: 398-405
• 9 Montes J, Levy G, Albert S. et al. Development and evaluation of a self-administered version of the ALSFRS-R. Neurology 2006; 67: 1294-1296
• 10 Maier A, Holm T, Wicks P. et al. Online assessment of ALS functional rating scale compares well to in-clinic evaluation: A prospective trial. Amyotrophic Lateral Sclerosis 2012; 13: 210-216
• 11 Maier A, Boentert M, Reilich P et al. ALSFRS-R-SE: An Adapted, Annotated, and Self-Explanatory Version of the Revised Amyotrophic Lateral Sclerosis Functional Rating Scale. Neurological Research and Practice 2022; 4: 60. https://doi.org/10.1186/s42466-022-00224-6
• 12 Plowman EK, Tabor LC, Robison R. et al. Discriminant ability of the Eating Assessment Tool-10 to detect aspiration in individuals with amyotrophic lateral sclerosis. Neurogastroenterology & Motility 2016; 28: 85-90
• 13 Vogt S, Petri S, Dengler R. et al. Dyspnea in Amyotrophic Lateral Sclerosis: Rasch-Based Development and Validation of a Patient-Reported Outcome (DALS-15. Journal of Pain and Symptom Management 2018; 56: 736-745.e732
• 14 Meyer R, Spittel S, Steinfurth L. et al. Patient-Reported Outcome of Physical Therapy in Amyotrophic Lateral Sclerosis: Observational Online Study. JMIR Rehabilitation and Assistive Technologies 2018; 5
• 15 Pongratz D, Zierz S, Fischer W. et al. Validity and reliability of the German language SF-36 health survey in patients with amyotrophic lateral sclerosis. NERVENHEILKUNDE 2002; 21: 432-+
• 16 Peters N, Dal Bello-Haas V, Packham T. et al. Do Generic Preference-Based Measures Accurately Capture Areas of Health-Related Quality of Life Important to Individuals with Amyotrophic Lateral Sclerosis: A Content Validation Study. Patient Related Outcome Measures 2021; Volume 12: 191-203
• 17 Jenkinson RFCBC. Evidence for the validity and reliability of the ALS assessment questionnaire: The ALSAQ-40. Amyotrophic Lateral Sclerosis and Other Motor Neuron Disorders 2009; 1: 33-40
• 18 Vogt S, Schlichte I, Schreiber S. et al. A Multi-Center Cohort Study on Characteristics of Pain, Its Impact and Pharmacotherapeutic Management in Patients with ALS. Journal of Clinical Medicine 2021; 10
• 19 Chiò A, Mora G, Lauria G.. Pain in amyotrophic lateral sclerosis. The Lancet Neurology 2017; 16: 144-157
• 20 Meyer T, Funke A, Münch C. et al. Real world experience of patients with amyotrophic lateral sclerosis (ALS) in the treatment of spasticity using tetrahydrocannabinol:cannabidiol (THC:CBD. BMC Neurology 2019; 19
• 21 Holm T, Maier A, Wicks P. et al. Severe Loss of Appetite in Amyotrophic Lateral Sclerosis Patients: Online Self-Assessment Study. interactive Journal of Medical Research 2013; 2
• 22 Beswick E, Park E, Wong C. et al. A systematic review of neuropsychiatric and cognitive assessments used in clinical trials for amyotrophic lateral sclerosis. Journal of Neurology 2020; 268: 4510-4521
• 23 Hammer EM, Häcker S, Hautzinger M. et al. Validity of the ALS-Depression-Inventory (ADI-12)— A new screening instrument for depressive disorders in patients with amyotrophic lateral sclerosis. Journal of Affective Disorders 2008; 109: 213-219
• 24 Thakore NJ, Pioro EP.. Depression in ALS in a large self-reporting cohort. Neurology 2016; 86: 1031-1038
• 25 Maier A, Gaudlitz M, Grehl T. et al. Use and subjective experience of the impact of motor-assisted movement exercisers in people with amyotrophic lateral sclerosis: a multicenter observational study. Scientific Reports 2022; 12
• 26 Kongsved SM, Basnov M, Holm-Christensen K. et al. Response Rate and Completeness of Questionnaires: A Randomized Study of Internet Versus Paper-and-Pencil Versions. Journal of Medical Internet Research 2007; 9
• 27 Chew S, Burke KM, Collins E. et al. Patient reported outcomes in ALS: characteristics of the self-entry ALS Functional Rating Scale-revised and the Activities-specific Balance Confidence Scale. Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration 2021; 22: 467-477
• 28 Meyer T, Spittel S, Grehl T. et al. Remote digital assessment of amyotrophic lateral sclerosis functional rating scale – a multicenter observational study. Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration 2022; 1-10
• 29 Kiernan MC, Vucic S, Talbot K. et al. Improving clinical trial outcomes in amyotrophic lateral sclerosis. Nature Reviews Neurology 2020; 17: 104-118
• 30 van Eijk RPA, Beelen A, Kruitwagen ET. et al. A Road Map for Remote Digital Health Technology for Motor Neuron Disease. Journal of Medical Internet Research 2021; 23
• 31 Meyer T, Grosskreutz J, Münch C. et al. AmbulanzPartner – multiprofessionelles und internet-unterstütztes Versorgungsmanagement bei der ALS. Klinische Neurophysiologie 2013; 44: 159-166
• 32 Coravos A, Khozin S, Mandl KD.. Developing and adopting safe and effective digital biomarkers to improve patient outcomes. npj Digital Medicine 2019; 2
• 33 Youn B-Y, Ko Y, Moon S. et al. Digital Biomarkers for Neuromuscular Disorders: A Systematic Scoping Review. Diagnostics 2021; 11
• 34 Vieira FG, Venugopalan S, Premasiri AS. et al. A machine-learning based objective measure for ALS disease severity. npj Digital Medicine 2022; 5
• 35 Törmä S, Ihalainen T, Palovuori K. et al. Recognizing swallowing movements using a textile-based device. Textile Research Journal 2022;
• 36 Helleman J, Bakers JNE, Pirard E. et al. Home-monitoring of vital capacity in people with a motor neuron disease. Journal of Neurology 2022; 269: 3713-3722
• 37 Quigg KH, Wilson MW, Choi PJ.. Transcutaneous CO2 monitoring as indication for inpatient non-invasive ventilation initiation in patients with amyotrophic lateral sclerosis. Muscle & Nerve 2021; 65: 444-447
• 38 Dorst J, Behrendt G, Ludolph AC.. Non-invasive ventilation and hypercapnia-associated symptoms in amyotrophic lateral sclerosis. Acta Neurologica Scandinavica 2019; 139: 128-134
• 39 Maier A, Eicher C, Kiselev J. et al. Acceptance of Enhanced Robotic Assistance Systems in People With Amyotrophic Lateral Sclerosis–Associated Motor Impairment: Observational Online Study. JMIR Rehabilitation and Assistive Technologies 2021; 8
• 40 Greenhalgh J, Gooding K, Gibbons E. et al. How do patient reported outcome measures (PROMs) support clinician-patient communication and patient care? A realist synthesis. Journal of Patient-Reported Outcomes 2018; 2
• 41 Hyland CJ, Guo R, Dhawan R. et al. Implementing patient-reported outcomes in routine clinical care for diverse and underrepresented patients in the United States. Journal of Patient-Reported Outcomes 2022; 6
• 42 Claessen FMAP MellemaJJ, Stoop N. et al. Influence of Priming on Patient-Reported Outcome Measures: A Randomized Controlled Trial. Psychosomatics 2016; 57: 47-56
• 43 Johnson S, Alonso B, Faulkner K. et al. Quality of Life Perspectives of People With Amyotrophic Lateral Sclerosis and Their Caregivers. The American Journal of Occupational Therapy 2017; 71 7103190010p7103190011 7103190010p7103190017
• 44 Burke L.. History of Patient-Reported Outcome Measurement at FDA. In: Mapi Research Trust; 2014: Im Internet: https://www.mapi-trust.org/resources/pro-newsletter/history-of-patient-report/ (Stand: 03.10.2022)
• 45 Young CA, Ealing J, McDermott C. et al. The relationships between symptoms, disability, perceived health and quality of life in amyotrophic lateral sclerosis/motor neuron disease. Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration 2019; 20: 317-327
• 46 Ambulanzpartner Soziotechnologie. Orientierung, Versorgung und Forschung mit Deutschlands einziger ALS-App 2022. Im Internet: https://www.ambulanzpartner.de/apps/als-app; Stand: 28.10.2022