P.131 Outcomes of Nusinersen in adult and pediatric cohorts of spinal
                        muscular amyotrophy after two years of usage at the CHU de Québec-Université
                        Laval

Background: Nusinersen is approved for spinal muscular amyotrophy type I,II,III and is available for adult and pediatric populations since January 2019 at the CHU de Québec-UL. Methods: Patients who received at least one dose of nusinersen between January 2019 and March 2021 were included. Required information was gathered from patients’ charts. Results: Ten adults (70 injections) and 23 infants and children (202 injections) were included. No child but two adults stopped the therapy for personal reasons. Tests were performed at 0, 6, 18, 24 months when possible to assess efficacy, tolerability and quality of life perception (Table 1). Preliminary data shows stabilization or improvement for many tests. Conclusions: Nusinersen is well tolerated. Favorable effects were observed in both populations. A monitoring is still imperative for an objective assessment and for evaluation of the most relevant tests. Long term benefits remain to be demonstrated but results are encouraging.

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Impact of type I Boston keratoprosthesis implantation on vision-related quality of life

British Journal of Ophthalmology ◽

10.1136/bjophthalmol-2017-310745 ◽

2017 ◽

Vol 102 (7) ◽

pp. 878-881 ◽

Cited By ~ 10

Author(s):

Marcus Ang ◽

Ryan Man ◽

Eva Fenwick ◽

Ecosse Lamoureux ◽

Mark Wilkins

Keyword(s):

Quality Of Life ◽

Effect Size ◽

Retention Rate ◽

Well Being ◽

Type I ◽

Boston Keratoprosthesis ◽

Related Quality ◽

The Impact

AimTo determine the impact of type I Boston keratoprosthesis (KPro) implantation on vision-related quality of life (VRQoL).MethodsProspective study in 33 patients (mean age 56±12 years, 67% male) with bilateral corneal blindness, who underwent a KPro implantation at a single tertiary eye hospital (June 2011–July 2015). VRQoL was evaluated using the Impact of Vision Impairment Questionnaire (IVI) at baseline and at 3–6 months postsurgery, after stabilisation of best-corrected visual acuity (BCVA). Rasch analysis was used to transform the IVI responses into interval-level measures comprising the ‘reading’, ‘mobility’ and ‘emotional’ subscales with effect sizes calculated for pre-post VRQoL scores.ResultsMean preoperative BCVA was counting-fingers at 2 feet in the operated eye (20/240 fellow eye). Preoperative VRQoL scores: −2.27, –2.91 and −3.06 logits for the reading, mobility and emotional subscales, respectively. Device retention rate was 90% over the follow-up period (mean 26±12 months). We observed large gains for reading and mobility of 1.92 logits (effect size 0.88), and 2.64 logits (effect size 0.89) respectively, with a moderate gain in the emotional subscale of 2.11 logits (effect size 0.59). These improvements did not vary significantly with BCVA on multivariate analysis (all p>0.05).ConclusionWe observed a differential short-term improvement to VRQoL after KPro implantation with a significant impact on emotional well-being, which may not be fully explained by visual improvement alone. Further studies are required to confirm if these improvements in VRQoL are sustained in the long-term and are generalisable to other populations.

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History and Perspective on DIY Closed Looping

Journal of Diabetes Science and Technology ◽

10.1177/1932296818808307 ◽

2018 ◽

Vol 13 (4) ◽

pp. 790-793 ◽

Cited By ~ 32

Author(s):

Dana Lewis

Keyword(s):

Quality Of Life ◽

Closed Loop ◽

Long Term Outcomes ◽

Closed Loop Systems ◽

Pediatric Populations ◽

Time In Range ◽

Basic Level

People with diabetes have been experimenting and self-modifying diabetes devices and technologies for many decades, in order to achieve the best possible quality of life and improving their long-term outcomes. There are now hundreds of individuals using DIY closed loop systems globally. They work similarly to commercial systems at a basic level, automatically adjusting and controlling insulin dosing, but are different in terms of transparency, access, customization, and usability. The potential downsides to DIY closed looping include varying responses from individual HCPs, who may be concerned about their own liability. However, initial outcomes from this self-selected community (including adult and pediatric populations globally) have been positive. There have now been several studies documenting improvements in A1c, time in range, and other outcomes such as quality-of-life benefits. More studies on quality-of-life improvements and more collaboration between companies and the community are recommended.

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Thinking Beyond Language: Intervention for Severe Aphasia

Perspectives on Neurophysiology and Neurogenic Speech and Language Disorders ◽

10.1044/nnsld19.1.15 ◽

2009 ◽

Vol 19 (1) ◽

pp. 15-22 ◽

Cited By ~ 3

Author(s):

Nina Simmons-Mackie

Keyword(s):

Quality Of Life ◽

Communication Training ◽

Language Intervention ◽

Specific Training ◽

Environmental Support ◽

Total Communication ◽

Communication Partners

Abstract Purpose: This article addresses several intervention approaches that aim to improve life for individuals with severe aphasia. Because severe aphasia significantly compromises language, often for the long term, recommended approaches focus on additional domains that affect quality of life. Treatments are discussed that involve increasing participation in personally relevant life situations, enhancing environmental support for communication and participation, and improving communicative confidence. Methods: Interventions that have been suggested in the aphasia literature as particularly appropriate for people with severe aphasia include training in total communication, training of communication partners, and activity specific training. Conclusion: Several intervention approaches can be implemented to enhance life with severe aphasia.

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