Loss of ambulation in SMA III at the time of disease-modifying treatments: an international study.
Обработка статьи · этапы, ошибки и управление
Ошибки и ограничения
История · было → стало
Состояние обновляется каждые 5 секунд, пока блок открыт. Процент показывается только там, где известен объём работы. Пауза останавливает продолжение этапа; текущий запрос к источнику может завершиться.
Coratti Giorgia, Bovis Francesca, Franchino Valentina, Montes Jacqueline, Sansone Valeria Ada, Dunaway Young Sally, Cutrì Chiara, Pasternak Amy, Pera Maria Carmela, Pane Marika, Glanzman Allan, Pegoraro Elena, Duong Tina, Sogus Elena, Sframeli Maria, Messina Sonia, Cavalcante Elzo, Scoto Maria Cristina, D'Amico Adele, Rodriguez-Torres Rafael, Mongini Tiziana, Maggi Lorenzo, Coccia Michela, Filosto Massimiliano, Zuccarino Riccardo, Masson Riccardo, Ricci Giulia, Bruno Claudio, Ruggiero Lucia, Vacchiano Veria, D'errico Eustachio, Verriello Lorenzo, Nigro Vincenzo, Scarpini Gaia, Garibaldi Matteo, Turri Mara, Ticci Chiara, Berardinelli Angela, Agosto Caterina, Ricci Federica, Zolkipli-Cunningham Zarazuela, Darras Basil T, Day John, Hirano Michio, Muntoni Francesco, Finkel Richard S, Mercuri Eugenio, international SMA consortium (ISMAC)
Аннотация
METHODS: This retrospective study analysed prospectively collected international data. Time-dependent Cox models assessed the association between treatment initiation and age at loss of ambulation, adjusting for age at onset, sex, SMN2 copies, birth year and country. Treatment was modelled as a time-dependent covariate to avoid immortal time bias. Descriptive analyses used Mann-Whitney U and χ² tests.
RESULTS: Among 555 individuals with type III, treatment halved the risk of ambulation loss (HR=0.50), with median loss at 44 vs 32 years in treated and untreated groups. Later onset, ≥4 SMN2 copies and female sex were also protective. The treatment effect was significant in type IIIA (HR=0.34) but not IIIB, with no significant interactions by sex, country or SMN2, though effects remained directionally protective.
CONCLUSIONS: Treatment in type III reduced the risk of ambulation loss by 50%, extending median ambulation by 12 years, with the greatest benefit in type IIIA. Later onset, female sex and higher SMN2 copy number were also protective but did not modify treatment effect. These findings underscore the value of early treatment and support its broad use to preserve ambulation across clinical subgroups.