PMID- 32781490
OWN - NLM
STAT- MEDLINE
DCOM- 20210610
LR  - 20210811
IS  - 1468-1331 (Electronic)
IS  - 1351-5101 (Linking)
VI  - 27
IP  - 12
DP  - 2020 Dec
TI  - Minimal clinically important differences in functional motor scores in adults 
      with spinal muscular atrophy.
PG  - 2586-2594
LID - 10.1111/ene.14472 [doi]
AB  - BACKGROUND AND PURPOSE: In patients with spinal muscular atrophy (SMA), 
      functional disease scores are frequently used to evaluate the course of the 
      disease and the efficacy of treatment. The aim of the present study was to 
      propose minimal clinically important difference (MCID) values for motor scores in 
      order to estimate the degree of change within a functional score that can be 
      considered clinically meaningful. METHODS: To estimate the MCID, 
      distribution-based approaches were used. For each assessment [Revised Upper Limb 
      Module (RULM), Hammersmith Functional Motor Scale Expanded (HFMSE) and 6-min walk 
      test (6MWT)] and subgroup (SMA type 2, SMA type 3, ambulatory and 
      non-ambulatory), the following MCID values based on a cohort of 51 adults with 
      SMA were calculated: standard error of measurement (SEm), one-half of standard 
      deviation (1/2 SD) and one-third of standard deviation (1/3 SD) of patients' 
      baseline scores. RESULTS: For the overall cohort, the SEm, 1/2 SD and 1/3 SD MCID 
      values were 2.9, 6.4 and 4.3 for the RULM and 4.3, 10.6 and 7.0 for the HFMSE, 
      respectively. Subgroup analysis led to generally lower standard deviations and 
      consecutively lower MCID values due to the significantly different motor 
      functions of the groups. The respective MCID values for the 6MWT were 55.5 m, 
      71.1 m and 47.8 m. CONCLUSIONS: Our data provide MCID values for functional motor 
      scores commonly used in adults with SMA in order to distinguish statistical 
      effects from 'real' changes. A complementary systematic consensus process could 
      help to further adjust the MCID values we propose.
CI  - (c) 2020 European Academy of Neurology.
FAU - Stolte, B
AU  - Stolte B
AUID- ORCID: 0000-0002-2774-067X
AD  - Department of Neurology, University Hospital Essen, Essen, Germany.
FAU - Bois, J-M
AU  - Bois JM
AD  - Institute of Medical Informatics, Biometrics and Epidemiology, University 
      Hospital Essen, Essen, Germany.
FAU - Bolz, S
AU  - Bolz S
AUID- ORCID: 0000-0003-1209-1715
AD  - Department of Neurology, University Hospital Essen, Essen, Germany.
FAU - Kizina, K
AU  - Kizina K
AD  - Department of Neurology, University Hospital Essen, Essen, Germany.
FAU - Totzeck, A
AU  - Totzeck A
AD  - Department of Neurology, University Hospital Essen, Essen, Germany.
FAU - Schlag, M
AU  - Schlag M
AD  - Department of Neurology, University Hospital Essen, Essen, Germany.
FAU - Kleinschnitz, C
AU  - Kleinschnitz C
AUID- ORCID: 0000-0002-1650-8875
AD  - Department of Neurology, University Hospital Essen, Essen, Germany.
FAU - Hagenacker, T
AU  - Hagenacker T
AUID- ORCID: 0000-0002-3631-3450
AD  - Department of Neurology, University Hospital Essen, Essen, Germany.
LA  - eng
PT  - Journal Article
PT  - Research Support, Non-U.S. Gov't
DEP - 20200906
PL  - England
TA  - Eur J Neurol
JT  - European journal of neurology
JID - 9506311
SB  - IM
CIN - Eur J Neurol. 2021 Jun;28(6):e40-e41. PMID: 33604967
CIN - Eur J Neurol. 2021 Jun;28(6):e42-e43. PMID: 33710742
MH  - Adult
MH  - Cohort Studies
MH  - Humans
MH  - *Muscular Atrophy, Spinal/diagnosis
MH  - *Spinal Muscular Atrophies of Childhood/diagnosis
MH  - Walk Test
OTO - NOTNLM
OT  - 6MWT
OT  - HFMSE
OT  - MCID
OT  - RULM
OT  - SMA
EDAT- 2020/08/12 06:00
MHDA- 2021/06/11 06:00
CRDT- 2020/08/12 06:00
PHST- 2020/04/24 00:00 [received]
PHST- 2020/06/26 00:00 [revised]
PHST- 2020/08/07 00:00 [accepted]
PHST- 2020/08/12 06:00 [pubmed]
PHST- 2021/06/11 06:00 [medline]
PHST- 2020/08/12 06:00 [entrez]
AID - 10.1111/ene.14472 [doi]
PST - ppublish
SO  - Eur J Neurol. 2020 Dec;27(12):2586-2594. doi: 10.1111/ene.14472. Epub 2020 Sep 6.