PMID- 37147862
OWN - NLM
STAT- MEDLINE
DCOM- 20230724
LR  - 20230725
IS  - 1531-8257 (Electronic)
IS  - 0885-3185 (Linking)
VI  - 38
IP  - 6
DP  - 2023 Jun
TI  - Defining Clinical Meaningfulness in Huntington's Disease.
PG  - 1036-1043
LID - 10.1002/mds.29394 [doi]
AB  - BACKGROUND: Minimal clinically important difference (MCID) represents the 
      smallest within-person change on an outcome measure considered meaningful to the 
      patient. Anchor-based MCID methods evaluate the relationship between changes in 
      an outcome measure and the patient-reported clinical importance of that change. 
      OBJECTIVE: This study aims to estimate longitudinal MCID for clinically relevant 
      outcome measures for individuals who have Stages 2 or 3 disease as measured by 
      the Huntington's Disease Integrated Staging System (HD-ISS). METHODS: Data were 
      drawn from Enroll-HD, a large global longitudinal, observational study and 
      clinical research platform for HD family members. We analyzed HD participants 
      (N = 11,070) by staging group using time frames ranging from 12 to 36 months. The 
      anchor was the physical component summary score of the 12-item short-form health 
      survey. HD-relevant motor, cognitive, and functional outcome measures were 
      independent, external criterion outcomes. Complex analysis was conducted using 
      multiple, independent, linear mixed effect regression models with decomposition 
      to calculate MCID for each external criterion by group. RESULTS: MCID estimates 
      varied by progression stage. MCID estimates increased as stage progression 
      increased and as the time frame increased. MCID values for key HD measures are 
      provided. For example, starting in HD-ISS stage 2, meaningful group change over 
      24 months equals an average increase of 3.6 or more points on the Unified 
      Huntington's Disease Rating Scale Total Motor Score. CONCLUSIONS: This is the 
      first study to examine MCID estimation thresholds for HD. The results can be used 
      to improve clinical interpretation of study outcomes and enable treatment 
      recommendations to support clinical decision-making and clinical trial 
      methodology. (c) 2023 International Parkinson and Movement Disorder Society.
CI  - (c) 2023 International Parkinson and Movement Disorder Society.
FAU - Hamilton, Jamie L
AU  - Hamilton JL
AUID- ORCID: 0000-0002-8522-610X
AD  - Clinical Department, CHDI Management/CHDI Foundation, Princeton, New Jersey, USA.
FAU - Mills, James A
AU  - Mills JA
AD  - Department of Psychiatry, University of Iowa, Iowa City, Iowa, USA.
FAU - Stebbins, Glenn T
AU  - Stebbins GT
AD  - Neurological Sciences, Rush University Medical Center, Chicago, Illinois, USA.
FAU - Long, Jeffrey D
AU  - Long JD
AD  - Department of Psychiatry, University of Iowa, Iowa City, Iowa, USA.
FAU - Fuller, Rebecca L M
AU  - Fuller RLM
AD  - Clinical Department, CHDI Management/CHDI Foundation, Princeton, New Jersey, USA.
FAU - Sathe, Swati
AU  - Sathe S
AD  - Clinical Department, CHDI Management/CHDI Foundation, Princeton, New Jersey, USA.
FAU - Roche, Matt
AU  - Roche M
AD  - Clinical Department, CHDI Management/CHDI Foundation, Princeton, New Jersey, USA.
FAU - Sampaio, Cristina
AU  - Sampaio C
AUID- ORCID: 0000-0002-7052-9079
AD  - Clinical Department, CHDI Management/CHDI Foundation, Princeton, New Jersey, USA.
LA  - eng
PT  - Journal Article
PT  - Observational Study
PT  - Research Support, Non-U.S. Gov't
DEP - 20230505
PL  - United States
TA  - Mov Disord
JT  - Movement disorders : official journal of the Movement Disorder Society
JID - 8610688
SB  - IM
MH  - Humans
MH  - *Huntington Disease
MH  - Longitudinal Studies
OTO - NOTNLM
OT  - Huntington's disease
OT  - minimal clinically important difference
OT  - outcome measures
OT  - patient experience data
OT  - quality of life
EDAT- 2023/05/06 09:42
MHDA- 2023/07/24 06:42
CRDT- 2023/05/06 03:39
PHST- 2023/02/10 00:00 [revised]
PHST- 2022/09/12 00:00 [received]
PHST- 2023/03/15 00:00 [accepted]
PHST- 2023/07/24 06:42 [medline]
PHST- 2023/05/06 09:42 [pubmed]
PHST- 2023/05/06 03:39 [entrez]
AID - 10.1002/mds.29394 [doi]
PST - ppublish
SO  - Mov Disord. 2023 Jun;38(6):1036-1043. doi: 10.1002/mds.29394. Epub 2023 May 5.