TY - JOUR
T1 - International Survey of ALS Experts about Critical Questions for Assessing Patients with ALS
AU - De Carvalho, Mamede
AU - Ryczkowski, Adam
AU - Andersen, Peter
AU - Gromicho, Marta
AU - Grosskreutz, Julian
AU - Kuźma-Kozakiewicz, Magdalena
AU - Petri, Susanne
AU - Piotrkiewicz, Maria
AU - Miltenberger Miltenyi, Gabriel
N1 - Publisher Copyright:
© 2017 World Federation of Neurology on behalf of the Research Group on Motor Neuron Diseases.
PY - 2017/10/2
Y1 - 2017/10/2
N2 - Objective: To define an applicable dataset for ALS patient registries we weighted specific clinical items as scored by worldwide ALS experts. Methods: Sixty participants were invited based on relevant clinical work, publications and personal acquaintance. They rated 160 clinical items consensually agreed by the members of our project, incorporating specialists from five European Centres. Scoring scheme was defined as: 1–essential; 2–important; 3–not very important. A mixed effect model was applied to rank items and to find possible correlations with geographical region (Europe vs. outside Europe). Results: We received 40 responses, 20 from Europe and 20 from outside; 42/160 data were scored as essential by >50% of the respondents, including: date of birth, gender, date of disease onset, date of diagnosis, ethnicity, region of onset, predominant upper neuron (UMN) or lower motor neuron (LMN) impairment, proximal versus distal weakness, respiratory symptoms, dysarthria, weight loss, signs of LMN/UMN involvement, emotional incontinence, cognitive changes, respiratory signs, neck weakness, body mass index, ALSFRS-R at entry, ALSFRS-R subscores at entry, timing and pattern of spreading and staging, electromyography, spirometry, MRI, CK level, riluzole intake, genetic background, history of physical exercise and previous and current main occupation. Four components were scored as non-relevant, including place of birth, blood pressure and pain at onset. There was no significant difference between regions (European vs. non-European countries). Conclusions: Our study identified a consensual set of clinical data with 42 specific items that can be used as a minimal data set for patient registers and for clinical trials.
AB - Objective: To define an applicable dataset for ALS patient registries we weighted specific clinical items as scored by worldwide ALS experts. Methods: Sixty participants were invited based on relevant clinical work, publications and personal acquaintance. They rated 160 clinical items consensually agreed by the members of our project, incorporating specialists from five European Centres. Scoring scheme was defined as: 1–essential; 2–important; 3–not very important. A mixed effect model was applied to rank items and to find possible correlations with geographical region (Europe vs. outside Europe). Results: We received 40 responses, 20 from Europe and 20 from outside; 42/160 data were scored as essential by >50% of the respondents, including: date of birth, gender, date of disease onset, date of diagnosis, ethnicity, region of onset, predominant upper neuron (UMN) or lower motor neuron (LMN) impairment, proximal versus distal weakness, respiratory symptoms, dysarthria, weight loss, signs of LMN/UMN involvement, emotional incontinence, cognitive changes, respiratory signs, neck weakness, body mass index, ALSFRS-R at entry, ALSFRS-R subscores at entry, timing and pattern of spreading and staging, electromyography, spirometry, MRI, CK level, riluzole intake, genetic background, history of physical exercise and previous and current main occupation. Four components were scored as non-relevant, including place of birth, blood pressure and pain at onset. There was no significant difference between regions (European vs. non-European countries). Conclusions: Our study identified a consensual set of clinical data with 42 specific items that can be used as a minimal data set for patient registers and for clinical trials.
UR - http://www.scopus.com/inward/record.url?scp=85023772469&partnerID=8YFLogxK
U2 - 10.1080/21678421.2017.1349150
DO - 10.1080/21678421.2017.1349150
M3 - Journal articles
C2 - 28705085
AN - SCOPUS:85023772469
SN - 2167-8421
VL - 18
SP - 505
EP - 510
JO - Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration
JF - Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration
IS - 7-8
ER -