Duchenne muscular dystrophy: quality of life among 95 patients evaluated using the Life Satisfaction Index for Adolescents

Abstract

The purpose of this study was to evaluate the quality of life (QoL) of patients with Duchenne muscular dystrophy (DMD) in different stages of the disease, by means of the Life Satisfaction Index for Adolescents (LSI-A). The practicality of this scale was also verified. The LSI-A was applied four times to 95 patients with DMD who were undergoing steroid therapy, at three-month intervals. The patients were divided into four groups according to age. The results from the four applications and the inter and intra-examiner concordance were treated statistically. Comparing the different age groups, patients with DMD did not lose QoL, even with disease progression. We concluded that, in spite of the progressive course of the disease, the QoL in patients with DMD does not get worse. The use of a scale that embraces a great diversity of circumstances in patients' lives, without considering clinical aspects excessively, is a good alternative for assessing the QoL of these patients. Key words: quality of life, Duchenne muscular dystrophy, steroid therapy, questionnaire, Life Satisfaction Index for Adolescents. Distrofia muscular de Duchenne: avaliação da qualidade de vida em 95 pacientes através do Life Satisfaction Index for AdolescentsRESUMO O objetivo deste estudo foi de quantificar a qualidade de vida (QV) em crianças com distrofia muscular de Duchenne (DMD) em diferentes idades através do uso do questionário Life Satisfaction Index for Adolescents (LSI-A). Foi também avaliada a praticidade do questionário. O LSI-A foi aplicado a 95 pacientes com distrofia muscular de Duchenne em corticoterapia, em diferentes idades, e por quatro vezes com intervalos de três meses. Os resultados concernentes às quatro avaliações e a concordância inter e intra-observador foram tratados estatisticamente. Comparando diferentes faixas etárias, mesmo ao longo da progressão da doença, não notamos perda da QV. Concluímos que por não valorizar excessivamente os aspectos clínicos e abranger uma diversidade de circunstâncias cotidianas, O LSI-A é útil na avaliação da QV das crianças com DMD, sendo também de fácil aplicação. Palavras-chave: qualidade de vida, distrofia muscular de Duchenne, corticoterapia, questionário, Life Satisfaction Index for Adolescents.

Quality of Life and Psychosocial Well-Being in Youth With Neuromuscular Disorders Who Are Wheelchair Users: A Systematic Review

“…One study was a survey conducted internationally 40 and three each were conducted in Australia [41][42][43] and the United States [44][45][46] . Nine studies recruited through tertiary clinics 43,44,[47][48][49][50][51][52][53] , five studies through multiple sources 40-42, 46, 54 and one study did not report their participant source 45 .…”

“…The majority were boys (n=376, 91%) and living with DMD (n=at least 339, 82%), with 9 of the 15 studies recruiting only youth with DMD. Four studies mentioned the exclusion of youth with cognitive impairment 41,47,51,53 , though only one of these indicated how intellect was assessed 41 and none specified numbers excluded. Youth with reading and writing difficulties were assisted in one study, though numbers were not specified 44 .…”

“…Two studies recruited by functional dependence on NIV 43,50 , one included only those with compromised sitting ability having undergone spinal fusion 52 and all three reported extremely low physical scores, hence they were assumed to include only non-ambulant participants. One study that did not specify wheelchair use recruited only 11-17 year old participants living with DMD 51 . As loss of loss of ambulation in DMD typically occurs around age 13 years old 4 , the study was included since the majority were likely using wheelchairs.…”

“…), one specific for children with chronic health conditions (DISABKIDS 47 ) (though these data were presented in figures only and could not be extracted), and one disease and age specific to adolescents with DMD [Life Satisfaction Index for Adolescents (LSI-A) 51 ].…”

“…Use of outcome measures with unverified validity and reliability for the population with NMD compromised the internal validity of four studies 40,51,52,54 (see Table 2 and Risk of interpretation bias appeared low in four quantitative 41,44,46,53 and two qualitative studies 42,45 who clearly reported the steps taken in their analysis process and which were assessed to be appropriate for their data.…”