Very few studies examined trend over time of the revised Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS-R) and factors influencing it; previous studies, then, included only patients attending tertiary ALS Centres. We studied ALSFRS-R decline, factors influencing this trend and survival in a population-based setting. From 2009 onwards, a prospective registry records all incident ALS cases among residents in Emilia Romagna (population: 4.4 million). For each patient, demographic and clinical details (including ALSFRS-R) are collected by caring physicians at each follow-up. Analysis was performed on 402 incident cases (1279 ALSFRS-R assessments). The average decline of the ALSFRS-R was 0.60 points/month during the first year after diagnosis and 0.34 points/month in the second year. ALSFRS-R decline was heterogeneous among subgroups. Repeated measures mixed model showed that ALSFRS-R score decline was influenced by age at onset (p < 0.01), phenotype (p = 0.01), body mass index (BMI) (p < 0.01), progression rate at diagnosis (ΔFS) (p < 0.01), El Escorial Criteria—Revised (p < 0.01), and FVC% at diagnosis (p < 0.01). Among these factors, at multivariate analysis, only age, site of onset and ΔFS independently influenced survival. In this first population-based study on ALSFRS-R trend, we confirm that ALSFRS-R decline is not homogeneous among ALS patients and during the disease. Factors influencing ALSFRS-R decline may not match with those affecting survival. These disease modifiers should be taken into consideration for trials design and in clinical practice during discussions with patients on prognosis.

Heterogeneity in ALSFRS-R decline and survival: a population-based study in Italy / Mandrioli, J.; Biguzzi, S.; Guidi, C.; Sette, E.; Terlizzi, E.; Ravasio, A.; Casmiro, M.; Salvi, F.; Liguori, R.; Rizzi, R.; Pietrini, Vladimiro; Borghi, A.; Rinaldi, R.; Fini, N.; Chierici, E.; Santangelo, M.; Granieri, E.; Mussuto, V.; De Pasqua, S.; Georgoulopoulou, E.; Fasano, A.; Errals, Group; Ferro, S.; D’Alessandro, R.; Mandrioli, J.; Fini, N.; Georgoulopoulou, E.; Fasano, A.; Nichelli, P.; Biguzzi, S.; Venturini, E.; Passarin, M. G.; Guidi, C.; Neri, W.; Sette, E.; Tugnoli, V.; Tola, M. R.; Terlizzi, E.; Guidetti, D.; Currò Dossi, M.; Pasquinelli, M.; Andruccioli, J.; Ravasio, A.; Casmiro, M.; Rasi, F.; Salvi, F.; Bartolomei, I.; Michelucci, R.; Avoni, P.; De Pasqua, S.; Liguori, R.; Rizzi, R.; Canali, E.; Marcello, N.; Grassi, A.; Delay, L.; Pietrini, V.; Aiello, Marina; Chierici, E.; Montanari, E.; Santangelo, M.; Amidei, S.; Greco, G.; Casetta, I.; Groppo, E.; Granieri, E.; De Massis, P.; Mussuto, V.; Borghi, A.; Gabellini, A.; Sacquegna, T.; Rinaldi, R.; Cirignotta, F.; Ferro, S.; D'Alessandro, R.. - In: NEUROLOGICAL SCIENCES. - ISSN 1590-1874. - 36:12(2015), pp. 2243-2252. [10.1007/s10072-015-2343-6]

Heterogeneity in ALSFRS-R decline and survival: a population-based study in Italy

PIETRINI, Vladimiro;AIELLO, Marina;
2015-01-01

Abstract

Very few studies examined trend over time of the revised Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS-R) and factors influencing it; previous studies, then, included only patients attending tertiary ALS Centres. We studied ALSFRS-R decline, factors influencing this trend and survival in a population-based setting. From 2009 onwards, a prospective registry records all incident ALS cases among residents in Emilia Romagna (population: 4.4 million). For each patient, demographic and clinical details (including ALSFRS-R) are collected by caring physicians at each follow-up. Analysis was performed on 402 incident cases (1279 ALSFRS-R assessments). The average decline of the ALSFRS-R was 0.60 points/month during the first year after diagnosis and 0.34 points/month in the second year. ALSFRS-R decline was heterogeneous among subgroups. Repeated measures mixed model showed that ALSFRS-R score decline was influenced by age at onset (p < 0.01), phenotype (p = 0.01), body mass index (BMI) (p < 0.01), progression rate at diagnosis (ΔFS) (p < 0.01), El Escorial Criteria—Revised (p < 0.01), and FVC% at diagnosis (p < 0.01). Among these factors, at multivariate analysis, only age, site of onset and ΔFS independently influenced survival. In this first population-based study on ALSFRS-R trend, we confirm that ALSFRS-R decline is not homogeneous among ALS patients and during the disease. Factors influencing ALSFRS-R decline may not match with those affecting survival. These disease modifiers should be taken into consideration for trials design and in clinical practice during discussions with patients on prognosis.
2015
Heterogeneity in ALSFRS-R decline and survival: a population-based study in Italy / Mandrioli, J.; Biguzzi, S.; Guidi, C.; Sette, E.; Terlizzi, E.; Ravasio, A.; Casmiro, M.; Salvi, F.; Liguori, R.; Rizzi, R.; Pietrini, Vladimiro; Borghi, A.; Rinaldi, R.; Fini, N.; Chierici, E.; Santangelo, M.; Granieri, E.; Mussuto, V.; De Pasqua, S.; Georgoulopoulou, E.; Fasano, A.; Errals, Group; Ferro, S.; D’Alessandro, R.; Mandrioli, J.; Fini, N.; Georgoulopoulou, E.; Fasano, A.; Nichelli, P.; Biguzzi, S.; Venturini, E.; Passarin, M. G.; Guidi, C.; Neri, W.; Sette, E.; Tugnoli, V.; Tola, M. R.; Terlizzi, E.; Guidetti, D.; Currò Dossi, M.; Pasquinelli, M.; Andruccioli, J.; Ravasio, A.; Casmiro, M.; Rasi, F.; Salvi, F.; Bartolomei, I.; Michelucci, R.; Avoni, P.; De Pasqua, S.; Liguori, R.; Rizzi, R.; Canali, E.; Marcello, N.; Grassi, A.; Delay, L.; Pietrini, V.; Aiello, Marina; Chierici, E.; Montanari, E.; Santangelo, M.; Amidei, S.; Greco, G.; Casetta, I.; Groppo, E.; Granieri, E.; De Massis, P.; Mussuto, V.; Borghi, A.; Gabellini, A.; Sacquegna, T.; Rinaldi, R.; Cirignotta, F.; Ferro, S.; D'Alessandro, R.. - In: NEUROLOGICAL SCIENCES. - ISSN 1590-1874. - 36:12(2015), pp. 2243-2252. [10.1007/s10072-015-2343-6]
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Utilizza questo identificativo per citare o creare un link a questo documento: https://hdl.handle.net/11381/2816672
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