Development and Preliminary Validation of a Parkinsonism-Dystonia Scale for Infants and Young Children

Roser Pons; Toni S Pearson; Belen Perez-Dueñas; Angels Garcia-Cazorla; Manju A Kurian; Zoi Dalivigka; Vasiliki Zouvelou; Chrysa Outsika; Eleftheria Kokkinou; Maria Sigatullina-Bondarenko; Alejandra Darling; Maria Del Mar O'Callaghan; Robert Spaull; Dora B D Steel; Evdokia Salamou; Maria João Forjaz; Carmen Rodriguez-Blazquez

doi:10.1002/mds.30219

Development and Preliminary Validation of a Parkinsonism-Dystonia Scale for Infants and Young Children

Roser Pons^*
, Toni S Pearson
, Belen Perez-Dueñas
, Angels Garcia-Cazorla
, Manju A Kurian
, Zoi Dalivigka
, Vasiliki Zouvelou
, Chrysa Outsika
, Eleftheria Kokkinou
, Maria Sigatullina-Bondarenko
, Alejandra Darling
, Maria Del Mar O'Callaghan
, Robert Spaull
, Dora B D Steel
, Evdokia Salamou
, Maria João Forjaz
, Carmen Rodriguez-Blazquez

^*Corresponding author for this work

Bristol Medical School (THS)

Research output: Contribution to journal › Article (Academic Journal) › peer-review

3 Citations (Scopus)

Abstract

Background

Parkinsonism in infancy is rare and is highly correlated with the presence of dystonia. Advances in treating and characterizing developmental and infantile degenerative parkinsonism have highlighted the need for a specialized assessment scale.

Objective

The aim of this study was to design and validate a scale that effectively assesses parkinsonism-dystonia in early life.

Methods

The Infantile Parkinsonism-Dystonia Rating Scale (IPDRS) was designed to capture the key clinical features of parkinsonism-dystonia in early life. It consists of 28 items across three subscales: Non-motor symptoms, Motor symptoms, and Dyskinesias. Thirty-two patients with hypokinetic movement disorder were scored following a standardized protocol. Filmed motor examinations were analyzed independently by three pediatric movement disorders specialists to evaluate interrater reliability. Twenty additional patients with primary neurotransmitter disorders were scored, and nine of them were evaluated at baseline and after treatment. Psychometric validation was conducted.

Results

A total of 52 patients were scored using the IPDRS. Mean age was 3.1 years (standard deviation [SD]: 2.0), and the mean IPDRS score was 40.8 (SD: 13.17). Internal consistency analysis demonstrated a Cronbach's α of 0.21 for Non-motor symptoms subscale, 0.84 for Motor symptoms subscale, and 0.95 for Dyskinesia subscale. Kappa indexes exceeded 0.70 in seven items. Correlation coefficients for dystonia items with the Barry-Albright-Dystonia Scale ranged from 0.46 to 0.64. After treatment, all IPDRS scores changed significantly, with an effect size of 2.42.

Conclusions

The IPDRS appears to be a reliable and valid tool for assessing parkinsonism in early life. Further validation studies with a larger sample size are needed to confirm these findings and complete the validation process. © 2025 The Author(s). Movement Disorders published by Wiley Periodicals LLC on behalf of International Parkinson and Movement Disorder Society.

Original language	English
Pages (from-to)	1669-1679
Number of pages	11
Journal	Movement Disorders
Volume	40
Issue number	8
Early online date	13 May 2025
DOIs	https://doi.org/10.1002/mds.30219
Publication status	E-pub ahead of print - 13 May 2025

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Pons, R., Pearson, T. S., Perez-Dueñas, B., Garcia-Cazorla, A., Kurian, M. A., Dalivigka, Z., Zouvelou, V., Outsika, C., Kokkinou, E., Sigatullina-Bondarenko, M., Darling, A., O'Callaghan, M. D. M., Spaull, R., Steel, D. B. D., Salamou, E., Forjaz, M. J., & Rodriguez-Blazquez, C. (2025). Development and Preliminary Validation of a Parkinsonism-Dystonia Scale for Infants and Young Children. Movement Disorders, 40(8), 1669-1679. Advance online publication. https://doi.org/10.1002/mds.30219

@article{a976c5ff81794d69bfcff96597239991,

title = "Development and Preliminary Validation of a Parkinsonism-Dystonia Scale for Infants and Young Children",

abstract = "BackgroundParkinsonism in infancy is rare and is highly correlated with the presence of dystonia. Advances in treating and characterizing developmental and infantile degenerative parkinsonism have highlighted the need for a specialized assessment scale.ObjectiveThe aim of this study was to design and validate a scale that effectively assesses parkinsonism-dystonia in early life.MethodsThe Infantile Parkinsonism-Dystonia Rating Scale (IPDRS) was designed to capture the key clinical features of parkinsonism-dystonia in early life. It consists of 28 items across three subscales: Non-motor symptoms, Motor symptoms, and Dyskinesias. Thirty-two patients with hypokinetic movement disorder were scored following a standardized protocol. Filmed motor examinations were analyzed independently by three pediatric movement disorders specialists to evaluate interrater reliability. Twenty additional patients with primary neurotransmitter disorders were scored, and nine of them were evaluated at baseline and after treatment. Psychometric validation was conducted.ResultsA total of 52 patients were scored using the IPDRS. Mean age was 3.1 years (standard deviation [SD]: 2.0), and the mean IPDRS score was 40.8 (SD: 13.17). Internal consistency analysis demonstrated a Cronbach's α of 0.21 for Non-motor symptoms subscale, 0.84 for Motor symptoms subscale, and 0.95 for Dyskinesia subscale. Kappa indexes exceeded 0.70 in seven items. Correlation coefficients for dystonia items with the Barry-Albright-Dystonia Scale ranged from 0.46 to 0.64. After treatment, all IPDRS scores changed significantly, with an effect size of 2.42.ConclusionsThe IPDRS appears to be a reliable and valid tool for assessing parkinsonism in early life. Further validation studies with a larger sample size are needed to confirm these findings and complete the validation process. {\textcopyright} 2025 The Author(s). Movement Disorders published by Wiley Periodicals LLC on behalf of International Parkinson and Movement Disorder Society.",

author = "Roser Pons and Pearson, \{Toni S\} and Belen Perez-Due{\~n}as and Angels Garcia-Cazorla and Kurian, \{Manju A\} and Zoi Dalivigka and Vasiliki Zouvelou and Chrysa Outsika and Eleftheria Kokkinou and Maria Sigatullina-Bondarenko and Alejandra Darling and O'Callaghan, \{Maria Del Mar\} and Robert Spaull and Steel, \{Dora B D\} and Evdokia Salamou and Forjaz, \{Maria Jo{\~a}o\} and Carmen Rodriguez-Blazquez",

note = "{\textcopyright} 2025 The Author(s). Movement Disorders published by Wiley Periodicals LLC on behalf of International Parkinson and Movement Disorder Society.",

year = "2025",

month = may,

day = "13",

doi = "10.1002/mds.30219",

language = "English",

volume = "40",

pages = "1669--1679",

journal = "Movement Disorders",

issn = "0885-3185",

publisher = "John Wiley \& Sons, Inc",

number = "8",

}

Pons, R, Pearson, TS, Perez-Dueñas, B, Garcia-Cazorla, A, Kurian, MA, Dalivigka, Z, Zouvelou, V, Outsika, C, Kokkinou, E, Sigatullina-Bondarenko, M, Darling, A, O'Callaghan, MDM, Spaull, R, Steel, DBD, Salamou, E, Forjaz, MJ & Rodriguez-Blazquez, C 2025, 'Development and Preliminary Validation of a Parkinsonism-Dystonia Scale for Infants and Young Children', Movement Disorders, vol. 40, no. 8, pp. 1669-1679. https://doi.org/10.1002/mds.30219

TY - JOUR

T1 - Development and Preliminary Validation of a Parkinsonism-Dystonia Scale for Infants and Young Children

AU - Pons, Roser

AU - Pearson, Toni S

AU - Perez-Dueñas, Belen

AU - Garcia-Cazorla, Angels

AU - Kurian, Manju A

AU - Dalivigka, Zoi

AU - Zouvelou, Vasiliki

AU - Outsika, Chrysa

AU - Kokkinou, Eleftheria

AU - Sigatullina-Bondarenko, Maria

AU - Darling, Alejandra

AU - O'Callaghan, Maria Del Mar

AU - Spaull, Robert

AU - Steel, Dora B D

AU - Salamou, Evdokia

AU - Forjaz, Maria João

AU - Rodriguez-Blazquez, Carmen

PY - 2025/5/13

Y1 - 2025/5/13

N2 - BackgroundParkinsonism in infancy is rare and is highly correlated with the presence of dystonia. Advances in treating and characterizing developmental and infantile degenerative parkinsonism have highlighted the need for a specialized assessment scale.ObjectiveThe aim of this study was to design and validate a scale that effectively assesses parkinsonism-dystonia in early life.MethodsThe Infantile Parkinsonism-Dystonia Rating Scale (IPDRS) was designed to capture the key clinical features of parkinsonism-dystonia in early life. It consists of 28 items across three subscales: Non-motor symptoms, Motor symptoms, and Dyskinesias. Thirty-two patients with hypokinetic movement disorder were scored following a standardized protocol. Filmed motor examinations were analyzed independently by three pediatric movement disorders specialists to evaluate interrater reliability. Twenty additional patients with primary neurotransmitter disorders were scored, and nine of them were evaluated at baseline and after treatment. Psychometric validation was conducted.ResultsA total of 52 patients were scored using the IPDRS. Mean age was 3.1 years (standard deviation [SD]: 2.0), and the mean IPDRS score was 40.8 (SD: 13.17). Internal consistency analysis demonstrated a Cronbach's α of 0.21 for Non-motor symptoms subscale, 0.84 for Motor symptoms subscale, and 0.95 for Dyskinesia subscale. Kappa indexes exceeded 0.70 in seven items. Correlation coefficients for dystonia items with the Barry-Albright-Dystonia Scale ranged from 0.46 to 0.64. After treatment, all IPDRS scores changed significantly, with an effect size of 2.42.ConclusionsThe IPDRS appears to be a reliable and valid tool for assessing parkinsonism in early life. Further validation studies with a larger sample size are needed to confirm these findings and complete the validation process. © 2025 The Author(s). Movement Disorders published by Wiley Periodicals LLC on behalf of International Parkinson and Movement Disorder Society.

AB - BackgroundParkinsonism in infancy is rare and is highly correlated with the presence of dystonia. Advances in treating and characterizing developmental and infantile degenerative parkinsonism have highlighted the need for a specialized assessment scale.ObjectiveThe aim of this study was to design and validate a scale that effectively assesses parkinsonism-dystonia in early life.MethodsThe Infantile Parkinsonism-Dystonia Rating Scale (IPDRS) was designed to capture the key clinical features of parkinsonism-dystonia in early life. It consists of 28 items across three subscales: Non-motor symptoms, Motor symptoms, and Dyskinesias. Thirty-two patients with hypokinetic movement disorder were scored following a standardized protocol. Filmed motor examinations were analyzed independently by three pediatric movement disorders specialists to evaluate interrater reliability. Twenty additional patients with primary neurotransmitter disorders were scored, and nine of them were evaluated at baseline and after treatment. Psychometric validation was conducted.ResultsA total of 52 patients were scored using the IPDRS. Mean age was 3.1 years (standard deviation [SD]: 2.0), and the mean IPDRS score was 40.8 (SD: 13.17). Internal consistency analysis demonstrated a Cronbach's α of 0.21 for Non-motor symptoms subscale, 0.84 for Motor symptoms subscale, and 0.95 for Dyskinesia subscale. Kappa indexes exceeded 0.70 in seven items. Correlation coefficients for dystonia items with the Barry-Albright-Dystonia Scale ranged from 0.46 to 0.64. After treatment, all IPDRS scores changed significantly, with an effect size of 2.42.ConclusionsThe IPDRS appears to be a reliable and valid tool for assessing parkinsonism in early life. Further validation studies with a larger sample size are needed to confirm these findings and complete the validation process. © 2025 The Author(s). Movement Disorders published by Wiley Periodicals LLC on behalf of International Parkinson and Movement Disorder Society.

U2 - 10.1002/mds.30219

DO - 10.1002/mds.30219

M3 - Article (Academic Journal)

C2 - 40364572

SN - 0885-3185

VL - 40

SP - 1669

EP - 1679

JO - Movement Disorders

JF - Movement Disorders

IS - 8

ER -

Development and Preliminary Validation of a Parkinsonism-Dystonia Scale for Infants and Young Children

Abstract

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