Patient reported outcome measures (PROMs) in amyotrophic lateral sclerosis

Deutsche Zeitschrift für Nervenheilkunde - Tập 267 Số 6 - Trang 1754-1759 - 2020
Fabiola De Marchi1, James Berry1, James Chan2, Sarah Caldwell1, Amy Swartz Ellrodt1, Jennifer Scalia1, Katherine Burke1, Ton Fang1, Rachel Sisodia3, Lee H Schwamm4, Lidia M.V.R. Moura4, Sabrina Paganoni5
1Healey Center for ALS, Department of Neurology, Massachusetts General Hospital, Harvard Medical School, 165 Cambridge St, Suite 600, Boston, MA, USA.
2Massachusetts General Hospital, Biostatistics Center, Harvard Medical School, Boston, USA
3Massachusetts General Physicians Organization, Harvard Medical School, MGH Partners Healthcare, Somerville, MA, USA.
4Department of Neurology, Massachusetts General Hospital, Harvard Medical School, Boston, MA, USA
5Department of Physical Medicine and Rehabilitation, Spaulding Rehabilitation Hospital, Harvard Medical School, Boston, MA, USA

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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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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