PMID- 38375606
OWN - NLM
STAT- MEDLINE
DCOM- 20240409
LR  - 20240409
IS  - 1468-1331 (Electronic)
IS  - 1351-5101 (Linking)
VI  - 31
IP  - 5
DP  - 2024 May
TI  - Establishing how much improvement in lung function and distance walked is 
      clinically important for adult patients with Pompe disease.
PG  - e16223
LID - 10.1111/ene.16223 [doi]
AB  - BACKGROUND AND PURPOSE: Pompe disease is a rare, inheritable, progressive 
      metabolic myopathy. This study aimed to estimate the minimal clinically important 
      difference (MCID) for an improvement in forced vital capacity in the upright 
      seated position (FVC(up)) and the 6-min walk test (6MWT) after a year of 
      treatment with enzyme replacement therapy. METHODS: Data were obtained from two 
      prospective follow-up studies. Between-group and within-group MCIDs were 
      estimated using anchor-based methods. Additionally, a distribution-based method 
      was used to generate supportive evidence. As anchors, self-reported change in 
      health and in physical functioning, shortness of breath and a categorization of 
      the Short-Form 36 Physical Component Summary score were used. Anchor 
      appropriateness was assessed using Spearman correlations (absolute values >/=0.29) 
      and a sufficient number of observations in each category. RESULTS: In all, 102 
      patients had at least one FVC(up) or 6MWT measurement during enzyme replacement 
      therapy. Based on the anchors assessed as appropriate, the between-group MCID for 
      an improvement in FVC(up) ranged from 2.47% to 4.83% points. For the 6MWT, it 
      ranged from 0.35% to 7.47% points which is equivalent to a distance of 
      2.18-46.61 m and 1.97-42.13 m for, respectively, a man and a woman of age 50, 
      height 1.75 m and weight 80 kg. The results of the distribution-based method were 
      within these ranges when applied to change in the outcome values. CONCLUSION: The 
      MCIDs for FVC(up) and 6MWT derived in this study can be used to interpret 
      differences between and within groups of patients with Pompe disease in clinical 
      trials and cohort studies.
CI  - (c) 2024 The Authors. European Journal of Neurology published by John Wiley & Sons 
      Ltd on behalf of European Academy of Neurology.
FAU - Lika, Aglina
AU  - Lika A
AUID- ORCID: 0009-0004-7474-3568
AD  - Department of Pediatrics, Centre for Lysosomal and Metabolic Diseases, Erasmus MC 
      University Medical Centre, Rotterdam, The Netherlands.
AD  - Department of Biostatistics, Erasmus MC University Medical Centre, Rotterdam, The 
      Netherlands.
AD  - Department of Epidemiology, Erasmus MC University Medical Centre, Rotterdam, The 
      Netherlands.
FAU - Andrinopoulou, Eleni-Rosalina
AU  - Andrinopoulou ER
AD  - Department of Biostatistics, Erasmus MC University Medical Centre, Rotterdam, The 
      Netherlands.
AD  - Department of Epidemiology, Erasmus MC University Medical Centre, Rotterdam, The 
      Netherlands.
FAU - van der Beek, Nadine A M E
AU  - van der Beek NAME
AUID- ORCID: 0000-0001-9161-3301
AD  - Department of Neurology, Centre for Lysosomal and Metabolic Diseases, Erasmus MC 
      University Medical Centre, Rotterdam, The Netherlands.
FAU - Rizopoulos, Dimitris
AU  - Rizopoulos D
AD  - Department of Biostatistics, Erasmus MC University Medical Centre, Rotterdam, The 
      Netherlands.
AD  - Department of Epidemiology, Erasmus MC University Medical Centre, Rotterdam, The 
      Netherlands.
FAU - van der Ploeg, Ans T
AU  - van der Ploeg AT
AUID- ORCID: 0000-0002-3359-1324
AD  - Department of Pediatrics, Centre for Lysosomal and Metabolic Diseases, Erasmus MC 
      University Medical Centre, Rotterdam, The Netherlands.
FAU - Kruijshaar, Michelle E
AU  - Kruijshaar ME
AUID- ORCID: 0009-0009-8700-0591
AD  - Department of Pediatrics, Centre for Lysosomal and Metabolic Diseases, Erasmus MC 
      University Medical Centre, Rotterdam, The Netherlands.
LA  - eng
GR  - 05-09-2007/ZONMW_/ZonMw/Netherlands
GR  - 152001005/ZONMW_/ZonMw/Netherlands
GR  - 80-83600-98-13007/ZONMW_/ZonMw/Netherlands
PT  - Journal Article
DEP - 20240220
PL  - England
TA  - Eur J Neurol
JT  - European journal of neurology
JID - 9506311
SB  - IM
MH  - Male
MH  - Adult
MH  - Female
MH  - Humans
MH  - Middle Aged
MH  - *Glycogen Storage Disease Type II/drug therapy
MH  - Prospective Studies
MH  - Walk Test
MH  - Follow-Up Studies
MH  - Lung
MH  - Treatment Outcome
OTO - NOTNLM
OT  - 6-minute walk test
OT  - Pompe disease
OT  - anchor-based method
OT  - clinically meaningful threshold
OT  - forced vital capacity
OT  - minimal clinically important difference
OT  - minimal important change
EDAT- 2024/02/20 11:50
MHDA- 2024/04/09 06:45
CRDT- 2024/02/20 04:23
PHST- 2023/12/21 00:00 [revised]
PHST- 2023/06/29 00:00 [received]
PHST- 2024/01/10 00:00 [accepted]
PHST- 2024/04/09 06:45 [medline]
PHST- 2024/02/20 11:50 [pubmed]
PHST- 2024/02/20 04:23 [entrez]
AID - 10.1111/ene.16223 [doi]
PST - ppublish
SO  - Eur J Neurol. 2024 May;31(5):e16223. doi: 10.1111/ene.16223. Epub 2024 Feb 20.