Old measures and new scores in spinal muscular atrophy patients

Elena Mazzone, Jacqueline Montes, Marion Main, Anna Mayhew, Danielle Ramsey, Allan M Glanzman, Sally Dunaway, Rachel Salazar, Amy Pasternak, Janet Quigley, Marika Pane, Maria C Pera, Mariacristina Scoto, Sonia Messina, Maria Sframeli, Adele D'amico, Marleen Van Den Hauwe, Serena Sivo, Nathalie Goemans, Basil T DarrasPetra Kaufmann, Enrico Bertini, Darryl C De Vivo, Francesco Muntoni, Richard Finkel, Eugenio Mercuri

Research output: Contribution to journalArticlepeer-review

3 Citations (Scopus)


INTRODUCTION: A recent Rasch analysis performed on the Hammersmith Functional Motor Scale-Expanded (HFMSE) in patients with spinal muscular atrophy (SMA) identified issues impacting scale validity, redundant items, and disordered thresholds on some items.

METHODS: We modified the HMFSE scoring based on the Rasch analysis and on expert consensus to establish whether the traditional scoring overestimated the number of patients with changes within 2 points from baseline. Data were collected retrospectively from multicenter data sets in 255 type 2 and 3 SMA patients.

RESULTS: The mean 12-month changes using the new and the traditional scoring system did not differ significantly (P > 0.05). The numbers of patients who improved or decreased by >2 points were also similar.

CONCLUSIONS: The presence of outliers using the traditional scoring system was not due to overestimation of changes in activities that were tested bilaterally or to discrepancies in the scoring hierarchy of individual items.

Original languageEnglish
Pages (from-to)435-7
Number of pages3
JournalMuscle & Nerve
Issue number3
Early online date24 Jul 2015
Publication statusPublished - 12 Aug 2015


  • Adolescent
  • Adult
  • Child
  • Child, Preschool
  • Female
  • Humans
  • Male
  • Middle Aged
  • Muscular Atrophy, Spinal
  • Psychometrics
  • Retrospective Studies
  • Severity of Illness Index
  • Spinal Muscular Atrophies of Childhood
  • Young Adult
  • Journal Article
  • Research Support, Non-U.S. Gov't


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