The aim of this study was to assess the need for tube feeding in a cohort of treated infants with type I SMA and to identify predictive factors. All patients were classified at baseline, when treatment started, and at follow-up according to their functional level and the need for tube feeding. Fisher’s exact test was used to examine the associations between the outcome at the last follow-up and SMA type, SMN2 copy number, and baseline nutritional status. ANOVA was performed to compare CHOP INTEND scores and age at treatment initiation with outcomes. The cohort includes 75 type I SMA infants treated between 0.1 and 5 years of age. At the last follow-up, 34 had no need for tube feeding, 9 had tube feeding but were also able to be fed by mouth, and 32 had tube feeding and were unable to be fed by mouth. Thirty of the 41 infants with tube feeding at follow-up already had feeding difficulties when treatment was started. The need for tube feeding at follow-up was associated with the level of feeding involvement at baseline and with CHOP INTEND scores [p < 0.001] but not with SMN2 copy number, SMA type 1 subtypes or age at treatment. The results of this study suggest that the need for tube feeding is not frequent in treated infants with type I SMA and, when occurring, can be predicted by the level of feeding involvement and low CHOP INTEND scores at baseline. (Table presented.)
Pane, M., Stanca, G., Coratti, G., D' Amico, A., Sansone, V. A., Berti, B., Fanelli, L., Albamonte, E., Ausili Cefaro, C., Cerchiari, A., Catteruccia, M., De Sanctis, R., Leone, D., Palermo, C., Buchignani, B., Onesimo, R., Kuczynska, E. M., Tosi, M., Pera, M. C., Bravetti, C., Tiziano, F. D., Bertini, E., Mercuri, E. M., Prognostic factors for tube feeding in type I SMA patients treated with disease-modifying therapies: a cohort study, <<EUROPEAN JOURNAL OF PEDIATRICS>>, 2024; (11): 4735-4745. [doi:10.1007/s00431-024-05735-9] [https://hdl.handle.net/10807/296197]
Prognostic factors for tube feeding in type I SMA patients treated with disease-modifying therapies: a cohort study
Pane, Marika;Coratti, Giorgia;Fanelli, Lavinia;Ausili Cefaro, Carolina;De Sanctis, Roberto;Onesimo, Roberta;Pera, Maria Carmela;Bravetti, Chiara;Tiziano, Francesco Danilo;Mercuri, Eugenio Maria
2024
Abstract
The aim of this study was to assess the need for tube feeding in a cohort of treated infants with type I SMA and to identify predictive factors. All patients were classified at baseline, when treatment started, and at follow-up according to their functional level and the need for tube feeding. Fisher’s exact test was used to examine the associations between the outcome at the last follow-up and SMA type, SMN2 copy number, and baseline nutritional status. ANOVA was performed to compare CHOP INTEND scores and age at treatment initiation with outcomes. The cohort includes 75 type I SMA infants treated between 0.1 and 5 years of age. At the last follow-up, 34 had no need for tube feeding, 9 had tube feeding but were also able to be fed by mouth, and 32 had tube feeding and were unable to be fed by mouth. Thirty of the 41 infants with tube feeding at follow-up already had feeding difficulties when treatment was started. The need for tube feeding at follow-up was associated with the level of feeding involvement at baseline and with CHOP INTEND scores [p < 0.001] but not with SMN2 copy number, SMA type 1 subtypes or age at treatment. The results of this study suggest that the need for tube feeding is not frequent in treated infants with type I SMA and, when occurring, can be predicted by the level of feeding involvement and low CHOP INTEND scores at baseline. (Table presented.)
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