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2016 ; 39
(5
): 705-712
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International Paediatric Mitochondrial Disease Scale
#MMPMID27277220
Koene S
; Hendriks JCM
; Dirks I
; de Boer L
; de Vries MC
; Janssen MCH
; Smuts I
; Fung CW
; Wong VCN
; de Coo IRFM
; Vill K
; Stendel C
; Klopstock T
; Falk MJ
; McCormick EM
; McFarland R
; de Groot IJM
; Smeitink JAM
J Inherit Metab Dis
2016[Sep]; 39
(5
): 705-712
PMID27277220
show ga
OBJECTIVE: There is an urgent need for reliable and universally applicable
outcome measures for children with mitochondrial diseases. In this study, we
aimed to adapt the currently available Newcastle Paediatric Mitochondrial Disease
Scale (NPMDS) to the International Paediatric Mitochondrial Disease Scale (IPMDS)
during a Delphi-based process with input from international collaborators,
patients and caretakers, as well as a pilot reliability study in eight patients.
Subsequently, we aimed to test the feasibility, construct validity and
reliability of the IPMDS in a multicentre study. METHODS: A clinically,
biochemically and genetically heterogeneous group of 17 patients (age
1.6-16 years) from five different expert centres from four different continents
were evaluated in this study. RESULTS: The feasibility of the IPMDS was good, as
indicated by a low number of missing items (4 %) and the positive evaluation of
patients, parents and users. Principal component analysis of our small sample
identified three factors, which explained 57.9 % of the variance. Good construct
validity was found using hypothesis testing. The overall interrater reliability
was good [median intraclass correlation coefficient for agreement between raters
(ICCagreement) 0.85; range 0.23-0.99). CONCLUSION: In conclusion, we suggest
using the IPMDS for assessing natural history in children with mitochondrial
diseases. These data should be used to further explore construct validity of the
IPMDS and to set age limits. In parallel, responsiveness and the minimal
clinically important difference should be studied to facilitate sample size
calculations in future clinical trials.
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