| Title |
Quality of life and participation in daily life of adults with Pompe disease receiving enzyme replacement therapy: 10 years of international follow‐up
|
|---|---|
| Published in |
Journal of Inherited Metabolic Disease, November 2015
|
| DOI | 10.1007/s10545-015-9889-6 |
| Pubmed ID | |
| Authors |
Deniz Güngör, Michelle E. Kruijshaar, Iris Plug, Dimitris Rizopoulos, Tim A. Kanters, Stephan C. A. Wens, Arnold J. J. Reuser, Pieter A. van Doorn, Ans T. van der Ploeg |
| Abstract |
Pompe disease is an inheritable metabolic disorder for which enzyme replacement therapy (ERT) has been available since 2006. Effects of ERT have been shown on distance walked, pulmonary function and survival. We investigated whether it also improves quality of life and participation in daily life in adult patients with the disease. In an international survey, we assessed quality of life (Short Form 36, SF-36) and participation (Rotterdam Handicap Scale, RHS) annually between 2002 and 2012. Repeated measurements mixed effects models were used to describe the data over time. Responses were available for 174 adult patients. In the periods before and after start of ERT, the median follow-up times were 4 years each (range 0.5-8). The SF-36 Physical Component Summary measure (PCS) deteriorated before ERT (-0.73 score points per year (sp/y); CI 95 % -1.07 to -0.39), while it improved in the first 2 years of ERT (1.49 sp/y; CI 0.76 to 2.21), and remained stable thereafter. The Mental Component Summary measure (MCS) remained stable before and during ERT. After declining beforehand (-0.49 sp/year; CI -0.64 to-0.34), the RHS stabilized under ERT. In adult patients with Pompe disease, ERT positively affects quality of life and participation in daily life. Our results reinforce previous findings regarding the effect of ERT on muscle strength, pulmonary function and survival. |
X Demographics
The data shown below were collected from the profiles of 5 X users who shared this research output. Click here to find out more about how the information was compiled.
Geographical breakdown
| Country | Count | As % |
|---|---|---|
| Canada | 1 | 20% |
| United States | 1 | 20% |
| Unknown | 3 | 60% |
Demographic breakdown
| Type | Count | As % |
|---|---|---|
| Members of the public | 3 | 60% |
| Science communicators (journalists, bloggers, editors) | 1 | 20% |
| Practitioners (doctors, other healthcare professionals) | 1 | 20% |
Mendeley readers
The data shown below were compiled from readership statistics for 104 Mendeley readers of this research output. Click here to see the associated Mendeley record.
Geographical breakdown
| Country | Count | As % |
|---|---|---|
| Unknown | 104 | 100% |
Demographic breakdown
| Readers by professional status | Count | As % |
|---|---|---|
| Student > Master | 17 | 16% |
| Researcher | 16 | 15% |
| Student > Ph. D. Student | 13 | 13% |
| Other | 6 | 6% |
| Student > Postgraduate | 6 | 6% |
| Other | 12 | 12% |
| Unknown | 34 | 33% |
| Readers by discipline | Count | As % |
|---|---|---|
| Medicine and Dentistry | 22 | 21% |
| Nursing and Health Professions | 11 | 11% |
| Biochemistry, Genetics and Molecular Biology | 7 | 7% |
| Agricultural and Biological Sciences | 6 | 6% |
| Sports and Recreations | 6 | 6% |
| Other | 17 | 16% |
| Unknown | 35 | 34% |
Attention Score in Context
This research output has an Altmetric Attention Score of 11. This is our high-level measure of the quality and quantity of online attention that it has received. This Attention Score, as well as the ranking and number of research outputs shown below, was calculated when the research output was last mentioned on 28 January 2020.
All research outputs
#3,034,965
of 24,164,942 outputs
Outputs from Journal of Inherited Metabolic Disease
#151
of 1,936 outputs
Outputs of similar age
#42,917
of 289,944 outputs
Outputs of similar age from Journal of Inherited Metabolic Disease
#2
of 10 outputs
Altmetric has tracked 24,164,942 research outputs across all sources so far. Compared to these this one has done well and is in the 87th percentile: it's in the top 25% of all research outputs ever tracked by Altmetric.
So far Altmetric has tracked 1,936 research outputs from this source. They receive a mean Attention Score of 4.8. This one has done particularly well, scoring higher than 92% of its peers.
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We're also able to compare this research output to 10 others from the same source and published within six weeks on either side of this one. This one has scored higher than 8 of them.