About the Quality of Life of Children with Spinal Muscular Atrophy Type 1


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The aim of our SMA I Project was to measure HRQOL in SMA I Italian children using the PEDSQLTM 4.0 Generic

Core Infant Scales Parents Report1and the PEDSQLTM 3.0 Neuromuscular Module Parents and Infants Report2.

Materials and methods

Subjects of our study: We sent our questionnaires to 171 families that had a child with SMA I. To date June 30, 2012, 73 families had responded, resulting in a response rate of 42.69%. Method: The research was conducted providing the questionnaires by mail to these families. In a single questionnaire we included 46 questions about the motivations for the choice of treatments. These questions were identical for parents of a child with SMA I that still was alive and for those whose child had died. Additionally, the parents whose son was alive responded to a PEDSQLTM 4.0 General Core Parents Report or a PEDSQLTM 3.0 Neuro-muscular Module Parents Report. If the child was older than five years we included a PEDSQLTM 3.0 Neuromuscular Module Infants Report according to their age.


The HRQOL assigned by parents to their children was 51.2 out of 100. The 30% of parents assigned a HRQOL around 50 points and almost another 30% who scored above 65 points. The QL self assigned by SMA I children was 48.5 points. There is a strong correlation between HRQOL scores of parents and children (r 0,77 and p = 0.003). The results showed no significant differences between the HRQOL that parents gave to their SMA I children with invasive ventilation (51,58) and the HRQOL attached by parents of SMA I children with noninvasive ventilation (50,46). There was a difference in the HRQOL self-assigned by SMA I children with tracheostomy (50.31) on regarding the HRQOL of SMA I children with noninvasive ventilation (45,00). The small sample size did not allow us to establish the significance of this difference.


The HRQOL of SMA I children assigned by their parents (51,2) is higher than the HRQOL assigned by physicians3(28,5). HRQOL self assigned by children with SMA I (48,5) is higher than the HRQOL assigned to them by physicians (28,5). No significant differences were detected when we analyzed HRQOL assigned by parents of SMAI children in relation to type of nutrition, ventilation or religion. The arguments against the use of invasive ventilation (tracheostomy) for SMA I children can`t be based on the lower HRQOL...

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