Prognostic factors for tube feeding in type I SMA patients treated with disease-modifying therapies: a cohort study

Author:

Pane Marika,Stanca Giulia,Coratti Giorgia,D’ Amico Adele,Sansone Valeria Ada,Berti Beatrice,Fanelli Lavinia,Albamonte Emilio,Ausili Cefaro Carolina,Cerchiari Antonella,Catteruccia Michela,De Sanctis Roberto,Leone Daniela,Palermo Concetta,Buchignani Bianca,Onesimo Roberta,Kuczynska Eliza Maria,Tosi Michele,Pera Maria Carmela,Bravetti Chiara,Tiziano Francesco Danilo,Bertini Enrico,Mercuri Eugenio

Abstract

AbstractThe 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. What is Known:• The advent of disease-modifying therapies is increasingly changing the approach to swallowing and nutritional management in type I SMA.Clinical trials and real-world data using all three disease-modifying therapies report a rather wide variability of feeding outcome and need for tube feeding that is often related to different cohorts that makes comparison between studies very difficult. What is New:• The real-world findings of this study, including all the children treated since treatments became available, confirmed that the need for tube feeding is not an invariable finding.The level of feeding involvement at baseline appears to be a reliable prognostic indicator of bulbar outcome.The results highlight the need for interventional studies with structured Speech and Language Therapist protocols that will help to better understand the extent to which bulbar function can be maintained or regained even in children requiring tube feeding.

Funder

Ministero della Salute

Next Generation EU

Università Cattolica del Sacro Cuore

Publisher

Springer Science and Business Media LLC

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