Evidence map›Paper›PMID 39210071›Full record

ArticleEuropean journal of pediatrics2024

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

Marika Pane, Giulia Stanca, Giorgia Coratti, Adele D' Amico, Valeria Ada Sansone, Beatrice Berti, Lavinia Fanelli, Emilio Albamonte, Carolina Ausili Cefaro, Antonella Cerchiari and 13 more

Abstract read
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In one paragraph

Article in European journal of pediatrics, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 5 papers, 3 of them syntheses that pooled it.

0numbers the graph read from it
0cells of the map it votes in
5citing papers in PubMed, 3 pooled it
–field-weighted citation impact
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

5 citing papers in PubMed, 3 syntheses or guidelines pooled it.

  1. Pooled it
  2. Pooled it
  3. Pooled it
  4. Review
  5. Article
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

23 authors.

Marika PanePediatric Neurology, Università Cattolica del Sacro Cuore, Rome, Italy.
Giulia StancaCentro Clinico Nemo, Neuropsichiatria Infantile, Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy.
Giorgia CorattiPediatric Neurology, Università Cattolica del Sacro Cuore, Rome, Italy.
Adele D' AmicoUnit of Muscular and Neurodegenerative Disorders, Bambino Gesù Children's Hospital, IRCCS, Rome, Italy.
Valeria Ada SansoneThe NEMO Center in Milan, Neurorehabilitation Unit, University of Milan, ASST Niguarda Hospital, Milan, Italy.
Beatrice BertiCentro Clinico Nemo, Neuropsichiatria Infantile, Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy.
Lavinia FanelliPediatric Neurology, Università Cattolica del Sacro Cuore, Rome, Italy.
Emilio AlbamonteThe NEMO Center in Milan, Neurorehabilitation Unit, University of Milan, ASST Niguarda Hospital, Milan, Italy.
Carolina Ausili CefaroSpeech Language Pathology Unit, Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, Lazio, Italy.
Antonella CerchiariFeeding and Swallowing Services Unit, Bambino Gesù Children's Hospital IRCCS, Rome, Italy.
Michela CatterucciaUnit of Muscular and Neurodegenerative Disorders, Bambino Gesù Children's Hospital, IRCCS, Rome, Italy.
Roberto De SanctisCentro Clinico Nemo, Neuropsichiatria Infantile, Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy.
Daniela LeoneCentro Clinico Nemo, Neuropsichiatria Infantile, Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy.
Concetta PalermoCentro Clinico Nemo, Neuropsichiatria Infantile, Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy.
Bianca BuchignaniCentro Clinico Nemo, Neuropsichiatria Infantile, Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy.
Roberta OnesimoCenter for Rare Diseases and Birth Defects, Department of Woman and Child Health and Public Health, Fondazione Policlinico Universitario A. Gemelli IRCCS, Rome, Italy.
Eliza Maria KuczynskaCenter for Rare Diseases and Birth Defects, Department of Woman and Child Health and Public Health, Fondazione Policlinico Universitario A. Gemelli IRCCS, Rome, Italy.
Michele TosiUnit of Muscular and Neurodegenerative Disorders, Bambino Gesù Children's Hospital, IRCCS, Rome, Italy.
Maria Carmela PeraPediatric Neurology, Università Cattolica del Sacro Cuore, Rome, Italy.
Chiara BravettiPediatric Neurology, Università Cattolica del Sacro Cuore, Rome, Italy.
Francesco Danilo TizianoDepartment of Life Sciences and Public Health, Section of Genomic Medicine, Università Cattolica del Sacro Cuore, Rome, Italy.
Enrico BertiniUnit of Muscular and Neurodegenerative Disorders, Bambino Gesù Children's Hospital, IRCCS, Rome, Italy.
Eugenio MercuriPediatric Neurology, Università Cattolica del Sacro Cuore, Rome, Italy. eugeniomaria.mercuri@policlinicogemelli.it.

Funding

Ministero della Salute GR-2018-12365706Ministero della Salute GR-2021-12374579Ministero della Salute RF-2019-12370334Next Generation EU PNRR-MR1-2022-12376937
6 · The paper itself

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. 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.

Indexed as

Enteral NutritionSpinal Muscular Atrophies of ChildhoodChild, PreschoolCohort StudiesFemaleFollow-Up StudiesHumansInfantInfant, NewbornMaleNutritional StatusPrognosisSurvival of Motor Neuron 2 ProteinSurvival of Motor Neuron 2 ProteinDisease-modifying therapiesOro-bulbarSpinal muscular atrophySwallowing

Identifiers

PMID39210071
PMCPMC11473555

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Registered trials

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Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.