TY  - JOUR
A1  - Wohnrade, Camilla
A1  - Velling, Ann-Kathrin
A1  - Mix, Lucas
A1  - Wurster, Claudia D.
A1  - Cordts, Isabell
A1  - Stolte, Benjamin
A1  - Zeller, Daniel
A1  - Uzelac, Zeljko
A1  - Platen, Sophia
A1  - Hagenacker, Tim
A1  - Deschauer, Marcus
A1  - Lingor, Paul
A1  - Ludolph, Albert C.
A1  - Lulé, Dorothée
A1  - Petri, Susanne
A1  - Osmanovic, Alma
A1  - Schreiber-Katz, Olivia
T1  - Health-related quality of life in spinal muscular atrophy patients and their caregivers — a prospective, cross-sectional, multi-center analysis
JF  - Brain Sciences
N2  - Spinal muscular atrophy (SMA) is a disabling disease that affects not only the patient’s health-related quality of life (HRQoL), but also causes a high caregiver burden (CGB). The aim of this study was to evaluate HRQoL, CGB, and their predictors in SMA. In two prospective, cross-sectional, and multi-center studies, SMA patients (n = 39) and SMA patient/caregiver couples (n = 49) filled in the EuroQoL Five Dimension Five Level Scale (EQ-5D-5L) and the Short Form Health Survey 36 (SF-36). Caregivers (CGs) additionally answered the Zarit Burden Interview (ZBI) and the Hospital Anxiety and Depression Scale (HADS). Patients were clustered into two groups with either low or high HRQoL (EQ-5D-5L index value <0.259 or >0.679). The latter group was mostly composed of ambulatory type III patients with higher motor/functional scores. More severely affected patients reported low physical functioning but good mental health and vitality. The CGB (mean ZBI = 22/88) correlated negatively with patients’ motor/functional scores and age. Higher CGB was associated with a lower HRQoL, higher depression and anxiety, and more health impairments of the CGs. We conclude that patient and CG well-being levels interact closely, which highlights the need to consider the health of both parties while evaluating novel treatments.
KW  - caregiver
KW  - caregiver burden
KW  - mental health
KW  - quality of life
KW  - spinal muscular atrophy
KW  - patient reported outcome measures
Y1  - 2023
U6  - http://nbn-resolving.de/urn/resolver.pl?urn:nbn:de:bvb:20-opus-305048
SN  - 2076-3425
VL  - 13
IS  - 1
ER  - 
TY  - JOUR
A1  - Peseschkian, Tara
A1  - Cordts, Isabell
A1  - Günther, René
A1  - Stolte, Benjamin
A1  - Zeller, Daniel
A1  - Schröter, Carsten
A1  - Weyen, Ute
A1  - Regensburger, Martin
A1  - Wolf, Joachim
A1  - Schneider, Ilka
A1  - Hermann, Andreas
A1  - Metelmann, Moritz
A1  - Kohl, Zacharias
A1  - Linker, Ralf A.
A1  - Koch, Jan Christoph
A1  - Büchner, Boriana
A1  - Weiland, Ulrike
A1  - Schönfelder, Erik
A1  - Heinrich, Felix
A1  - Osmanovic, Alma
A1  - Klopstock, Thomas
A1  - Dorst, Johannes
A1  - Ludolph, Albert C.
A1  - Boentert, Matthias
A1  - Hagenacker, Tim
A1  - Deschauer, Marcus
A1  - Lingor, Paul
A1  - Petri, Susanne
A1  - Schreiber-Katz, Olivia
T1  - A nation-wide, multi-center study on the quality of life of ALS patients in Germany
JF  - Brain Sciences
N2  - Improving quality of life (QoL) is central to amyotrophic lateral sclerosis (ALS) treatment. This Germany-wide, multicenter cross-sectional study analyses the impact of different symptom-specific treatments and ALS variants on QoL. Health-related QoL (HRQoL) in 325 ALS patients was assessed using the Amyotrophic Lateral Sclerosis Assessment Questionnaire 5 (ALSAQ-5) and EuroQol Five Dimension Five Level Scale (EQ-5D-5L), together with disease severity (captured by the revised ALS Functional Rating Scale (ALSFRS-R)) and the current care and therapies used by our cohort. At inclusion, the mean ALSAQ-5 total score was 56.93 (max. 100, best = 0) with a better QoL associated with a less severe disease status (β = −1.96 per increase of one point in the ALSFRS-R score, p < 0.001). “Limb-onset” ALS (lALS) was associated with a better QoL than “bulbar-onset” ALS (bALS) (mean ALSAQ-5 total score 55.46 versus 60.99, p = 0.040). Moreover, with the ALSFRS-R as a covariate, using a mobility aid (β = −7.60, p = 0.001), being tracheostomized (β = −14.80, p = 0.004) and using non-invasive ventilation (β = −5.71, p = 0.030) were associated with an improved QoL, compared to those at the same disease stage who did not use these aids. In contrast, antidepressant intake (β = 5.95, p = 0.007), and increasing age (β = 0.18, p = 0.023) were predictors of worse QoL. Our results showed that the ALSAQ-5 was better-suited for ALS patients than the EQ-5D-5L. Further, the early and symptom-specific clinical management and supply of assistive devices can significantly improve the individual HRQoL of ALS patients. Appropriate QoL questionnaires are needed to monitor the impact of treatment to provide the best possible and individualized care.
KW  - Amyotrophic Lateral Sclerosis (ALS)
KW  - Amyotrophic Lateral Sclerosis Assessment Questionnaire 5 (ALSAQ-5)
KW  - ALS treatment
KW  - “bulbar-onset” ALS (bALS)
KW  - “limb-onset” ALS (lALS)
KW  - EuroQol Five Dimension Five Level Scale (EQ-5D-5L)
KW  - health-related quality of life (HRQoL)
KW  - quality of life (QoL)
KW  - symptom-specific treatment
KW  - assistive devices
Y1  - 2021
U6  - http://nbn-resolving.de/urn/resolver.pl?urn:nbn:de:bvb:20-opus-234147
SN  - 2076-3425
VL  - 11
IS  - 3
ER  -