TY - JOUR
T1 - Healthcare needs, expectations, utilization, and experienced treatment effects in patients with hereditary spastic paraplegia
T2 - a web-based survey in the Netherlands
AU - Kerstens, Hans C. J. W.
AU - Van Lith, Bas J. H.
AU - Nijkrake, Maarten J.
AU - De Swart, Bert J. M.
AU - Van den Bemd, Laura A. C.
AU - Smeets, Rob J. E. M.
AU - Klemens, Fheodoroff
AU - Van de Warrenburg, Bart P. C.
AU - Van der Wees, Philip J.
AU - Geurts, Alexander C. H.
N1 - Funding Information:
This work was supported by a PhD Grant to the first author from the HAN University of Applied Sciences. This investigation was further supported by unrestricted grants from Merz Pharma and Ipsen Pharma. BvdW and AG are member of the European Reference Network for Rare Neurological Diseases—Project ID No. 739510.
Publisher Copyright:
© 2021, The Author(s).
PY - 2021/6/24
Y1 - 2021/6/24
N2 - Background We aimed to identify healthcare needs, expectations, utilization, and the experienced treatment effects in a population of Dutch patients with hereditary spastic paraplegia (HSP). Methods We distributed an online questionnaire among 194 adult persons with HSP in the Netherlands, of which 166 returned a fully completed version. After applying predefined exclusion criteria, 109 questionnaires from persons with pure HSP were analysed. Results Healthcare needs and expectations were primarily focused on the relief of muscle stiffness and reduction of balance and gait impairments (65-80%), but many participants also expressed needs regarding relief of non-motor symptoms (e.g. pain, fatigue), emotional problems, impaired sleep and self-care capacity, and participation problems (> 60%). Remarkably, despite these frequent needs, relatively few participants (< 33%) expected to be able to improve in these additional domains. Rehabilitation physicians and physiotherapists were more frequently consulted than neurologists and occupational therapists, respectively. Physiotherapy was the most often proposed non-pharmacological intervention (85%), followed by orthopedic footwear (55%) and splints (28%). Approximately one third of the participants was never offered any pharmacological (spasmolytic) treatment. Spasmolytic oral drugs, injections, and intrathecal baclofen were given to 41%, 26%, and 5% of the participants, respectively. Independent of the type of pharmacological intervention, 35-46% of these participants experienced decreased spastiticy and improved general fitness. Other experienced effects differed per type of intervention. Conclusions Based on this web-based survey in the Netherlands, there seems to be ample room for improvement to meet and attune the healthcare needs and expectations of people with HSP concerning both their motor and non-motor symptoms and functional limitations. In addition, the provision of adequate information about non-pharmacological and pharmacological interventions seems to be insufficient for many patients to allow shared decision making. These conclusions warrant a more pro-active attitude of healthcare providers as well as an interdisciplinary approach for a substantial proportion of the HSP population, also involving professionals with a primary occupational and/or psychosocial orientation.
AB - Background We aimed to identify healthcare needs, expectations, utilization, and the experienced treatment effects in a population of Dutch patients with hereditary spastic paraplegia (HSP). Methods We distributed an online questionnaire among 194 adult persons with HSP in the Netherlands, of which 166 returned a fully completed version. After applying predefined exclusion criteria, 109 questionnaires from persons with pure HSP were analysed. Results Healthcare needs and expectations were primarily focused on the relief of muscle stiffness and reduction of balance and gait impairments (65-80%), but many participants also expressed needs regarding relief of non-motor symptoms (e.g. pain, fatigue), emotional problems, impaired sleep and self-care capacity, and participation problems (> 60%). Remarkably, despite these frequent needs, relatively few participants (< 33%) expected to be able to improve in these additional domains. Rehabilitation physicians and physiotherapists were more frequently consulted than neurologists and occupational therapists, respectively. Physiotherapy was the most often proposed non-pharmacological intervention (85%), followed by orthopedic footwear (55%) and splints (28%). Approximately one third of the participants was never offered any pharmacological (spasmolytic) treatment. Spasmolytic oral drugs, injections, and intrathecal baclofen were given to 41%, 26%, and 5% of the participants, respectively. Independent of the type of pharmacological intervention, 35-46% of these participants experienced decreased spastiticy and improved general fitness. Other experienced effects differed per type of intervention. Conclusions Based on this web-based survey in the Netherlands, there seems to be ample room for improvement to meet and attune the healthcare needs and expectations of people with HSP concerning both their motor and non-motor symptoms and functional limitations. In addition, the provision of adequate information about non-pharmacological and pharmacological interventions seems to be insufficient for many patients to allow shared decision making. These conclusions warrant a more pro-active attitude of healthcare providers as well as an interdisciplinary approach for a substantial proportion of the HSP population, also involving professionals with a primary occupational and/or psychosocial orientation.
KW - Hereditary spastic paraplegia
KW - Spasticity
KW - Survey
KW - Patient-reported outcomes
KW - Needs assessment
KW - Healthcare utilization
KW - Experienced treatment effects
KW - OCCUPATIONAL-THERAPY
KW - PARAPARESIS
KW - FEATURES
KW - EFFICACY
UR - https://springernature.figshare.com/articles/dataset/Additional_file_1_of_Healthcare_needs_expectations_utilization_and_experienced_treatment_effects_in_patients_with_hereditary_spastic_paraplegia_a_web-based_survey_in_the_Netherlands/14843460/1
U2 - 10.1186/s13023-021-01915-0
DO - 10.1186/s13023-021-01915-0
M3 - Article
C2 - 34167574
SN - 1750-1172
VL - 16
JO - Orphanet Journal of Rare Diseases
JF - Orphanet Journal of Rare Diseases
IS - 1
M1 - 283
ER -