Psychological Health in Adults with Morquio Syndrome

Ali, Nadia; Cagle, Stephanie

doi:10.1007/8904_2014_396

Cited by 15 publications

(19 citation statements)

References 20 publications

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“…The very poor quality of life scores in the MOR‐006 population seem to confirm these findings. The mean SF‐36 PCS in adult patients from MOR‐006 (17.3) was much lower than that recently reported for 20 adult Morquio A patients (36.5) [Ali and Cagle, ].…”

Section: Discussioncontrasting

confidence: 55%

Impact of elosulfase alfa in patients with morquio A syndrome who have limited ambulation: An open‐label, phase 2 study

Harmatz

Mengel

Geberhiwot

et al. 2016

American J of Med Genetics Pt A

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Efficacy and safety of elosulfase alfa enzyme replacement therapy (ERT) were assessed in an open‐label, phase 2, multi‐national study in Morquio A patients aged ≥5 years unable to walk ≥30 meters in the 6‐min walk test. Patients received elosulfase alfa 2.0 mg/kg/week intravenously for 48 weeks. Efficacy measures were functional dexterity, pinch/grip strength, mobility in a modified timed 25‐foot walk, pain, quality of life, respiratory function, and urine keratan sulfate (KS). Safety/tolerability was also assessed. Fifteen patients received elosulfase alfa, three patients discontinued ERT due to adverse events (two were grade 3 drug‐related adverse events, the other was not drug‐related), and two patients missed >20% of planned infusions; 10 completed treatment through 48 weeks and received ≥80% of planned infusions (Modified Per Protocol [MPP] population). The study population had more advanced disease than that enrolled in other trials. From baseline to week 48, MPP data showed biochemical efficacy (urine KS decreased 52.4%). The remaining efficacy results were highly variable due to challenges in test execution because of severe skeletal and joint abnormalities, small sample sizes, and clinical heterogeneity among patients. Eight patients showed improvements in one or more outcome measures; several patients indicated improvements not captured by the study assessments (e.g., increased energy, functional ability). The nature of adverse events was similar to other elosulfase alfa studies. This study illustrates the considerable challenges in objectively measuring impact of ERT in very disabled Morquio A patients and highlights the need to examine results on an individual basis. © 2016 The Authors. American Journal of Medical Genetics Part A Published by Wiley Periodicals, Inc.

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Section: Discussioncontrasting

confidence: 55%

Impact of elosulfase alfa in patients with morquio A syndrome who have limited ambulation: An open‐label, phase 2 study

Harmatz

Mengel

Geberhiwot

et al. 2016

American J of Med Genetics Pt A

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“…Scale scores are normed by gender and age and categorized as normal (<93rd percentile), borderline-clinical (93rd-97th percentiles), or clinical (>97th percentile). The ASEBA is used with a wide variety of medical conditions, including cystic fibrosis, Fabry, Morquio, Turner, Williams, Angelman, and Prader-Willi syndromes (Achenbach and Rescorla 2003;Ali and Cagle 2015;Laney et al 2010). For this study, the Depression scale and Mean Adaptive Functioning scale were used.…”

Section: Methodsmentioning

confidence: 99%

Treatment of Depression in Adults with Fabry Disease

2017

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Fabry disease (FD) is a genetic X-linked, multisystemic, progressive lysosomal storage disorder (LSD). Depression has emerged as a disease complication, with prevalence estimates ranging from 15 to 62%. This is a pilot study examining the effects of psychological counseling for depression in FD on depression, adaptive functioning (AF), quality of life (QOL), and subjective pain experience. Telecounseling was also piloted, as it has beneficial effects in other chronic diseases which make in-person counseling problematic. Subjects completed 6 months of in-person or telecounseling with the same health psychologist, followed by 6 months without counseling. Self-report measures of depression, AF, QOL, and subjective pain were completed every 3 months. All subjects experienced improvements in depression, which were sustained during the follow-up period. Improvements in depression were correlated with improvements in mental health QOL and subjective pain severity, while improvements in mental health QOL were correlated with improvements in AF. While statistical comparison between counseling modes was not possible with the given sample size, relevant observations were noted. Recommendations for future research include replication of results with a larger sample size and a longer counseling period. The use of video counseling may be beneficial. In conclusion, the present pilot study supports the efficacy of psychological treatment for depression in people with FD, highlighting the importance of having health psychologists housed in LSD treatment centers, rather than specialty psychology/psychiatry settings, to increase participation and decrease potential obstacles to access due to perceived stigma.

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“…The Older Adult Self-Report (OASR) is a psychopathological rating scale for persons aged over 60 years (Ali & Cagle, 2014) and is one of the scales of the Achenbach System of Empirically Based Assessment -ASEBA (Achenbach, 2006). The ASEBA is an empirically based research system most commonly used in clinical practice and research (Achenbach & Rescorla, 2007;Rocha, Araújo, & Silvares, 2008).…”

Section: Introductionmentioning

confidence: 99%

Older Adult Self Report Construct and Criterion Validity Evidence for the Brazilian Elderly

et al. 2017

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<p>In Brazil, the number of validated and recognized scientific instruments for clinical evaluation of elderly persons is limited. The Older Adult Self-Report (OASR) is a psychopathological rating scale for persons with more than 60 years, which evaluates adaptive functioning and psychopathology. The aim of this study was at investigating OASR construct and criterion validity evidence for Brazilian elderly. The sample included 345 individuals, between 60 and 94 years of age, which were recruited by convenience. Participants responded to a questionnaire containing questions about demographic and health information, the Mini Mental State Examination, the Geriatric Depression Scale short version and the OASR. Data were analyzed using descriptive statistics, Pearson's correlation and discriminant analysis. The results showed significant associations between the scores of OASR’s subscales, correlating with general cognitive ability and depressive symptoms. Furthermore, OASR was sensitive to identify elderly with significant symptoms of depression. In conclusion, the OASR showed adequate evidence of construct and criterion validity for a sample of Brazilian elderly.</p>

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Psychological Health in Adults with Morquio Syndrome

Cited by 15 publications

References 20 publications

Impact of elosulfase alfa in patients with morquio A syndrome who have limited ambulation: An open‐label, phase 2 study

Impact of elosulfase alfa in patients with morquio A syndrome who have limited ambulation: An open‐label, phase 2 study

Treatment of Depression in Adults with Fabry Disease

Older Adult Self Report Construct and Criterion Validity Evidence for the Brazilian Elderly

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