Observational Study for the Epidemiology of Cerebral Palsy in Italy

Condition(s) studied

Investigational drug(s) / intervention(s)

hospital-based registry: The clinical data collected for each patient will reflect comprehensive multiprofessional clinical insights necessary for diagnostic definition and typically obtained during diagnostic evaluation and/or monitoring visits. In addition to these, instrumental data commonly acquired in clinical practice, such as neuroradiological and electrophysiological information, will also be collected. For each enrolled subject, a checklist (CRF) will be completed to gather study-related data. These data will be entered into a specific database constructed on the REDCap platform and managed in an anonymized form. Subject enrollment will be conducted progressively. This data collection will enable monitoring of study progress, including the number of enrolled subjects, completed assessments, and study adherence. Every three months, monitoring will be performed to assess study progress and the achievement of enrollment targets.

Study summary

BACKGROUND AND RATIONALE Cerebral Palsy (CP) is the leading cause of childhood disability, with an estimated prevalence of 2 to 2.5 per 1,000 births. This complex clinical condition encompasses motor development disorders as well as sensory, communication, cognitive, and emotional impairments. Despite the significant healthcare impact of CP, standardized guidelines for its study and treatment are lacking both nationally and internationally. Existing literature is limited by small sample sizes, inconsistent assessment tools, and insufficient etiopathogenetic and functional characterizations of different CP types. A national registry, which could offer comprehensive epidemiological data, does not currently exist.

This project aims to create a shared digital platform across clinical centers throughout Italy (three coordinative centers for northern, central, and southern Italy). This platform will allow systematic, standardized, retrospective, and prospective data collection, including clinical, instrumental, and genetic data (where available) for patients with CP. The study intends to complement ongoing national projects, such as the "Italian Network for Early Detection and Intervention in Developmental Disabilities (INEED)" and the "Italian Network for Cerebral Palsy (Ita-Net-CP)."

STUDY OBJECTIVES

This study aims to analyze epidemiological data, specifically:

* Evaluating the prevalence of various clinical types of CP
* Assessing the distribution of functional impairment severity and associated comorbidities.

STUDY DESIGN This is a non-profit, multicenter, observational study involving a cohort of CP patients, promoted within the Mariani Foundation Network. The study involves both retrospective and prospective data collection. It will enroll both patients already under care for follow-up and newly diagnosed patients.

A sample of at least 300 children and/or adolescents with CP of various etiologies, with 100 participants per clinical center, is targeted. Enrollment will be voluntary, either by invitation from the referring physician, who will explain the importance of the registry, or through project dissemination, allowing parents to request their children's inclusion. Basic registry items can be filled out by parents and, with permission, the primary physician for clinical fields. Parents can opt to be included in the registry to receive information on national clinical studies, approved by ethics committees.

Collected essential data includes:

* Identification and contact information for patient pseudonymization
* Socioeconomic status
* CP classification according to the European Surveillance Group
* Anamnesis regarding disorder etiology
* Neuroradiological classification based on SCPE (MRICS)
* Functional impairment level of each child
* Significant comorbidities

STUDY POPULATION At least 300 pediatric CP patients aged 4-18 years are expected to be recruited. Exclusion criteria only include lack of informed consent.

DRUG, MEDICAL DEVICE, NUTRACEUTICAL, BIOLOGICAL SAMPLES, OTHER ELEMENTS This observational study involves systematic, standardized retrospective and prospective data collection of clinical, instrumental, and genetic data (if available). No medical devices, pharmaceuticals, or nutraceuticals will be used. No human biological samples will be collected.

STUDY TIMELINE Study duration per patient: aligned with routine clinical visits. No additional evaluations are foreseen.

Recruitment duration: 12 months Overall study duration: 36 months

SAMPLE SIZE AND DATA ANALYSIS The study aims to recruit at least 300 pediatric CP patients. A control group is not planned.

Eligibility

Primary outcome measure(s)

• Participants demographic data — Baseline
  Age, gender, birth date, place of birth, nationality, address (country)
• Cerebral palsy (CP) classification — Baseline
  Classification of CP according to the European Surveillance Group (SCPE Guidelines, 2019): Spastic CP (bilateral or unilateral forms); Dyskinetic CP (dystonic form or choreoathetotic form); Ataxic CP.
• Anamnestic data related to the etiopathogenesis of the disorder — Baseline
  This refers to the medical history and background information that help in understanding the cause and development of a disorder i.e. visual and auditory impairments, birth weight, cognitive level, epilepsy, Apgar score and comorbidities.
• Level of Gross Motor Function Classification System (GMFCS) — Baseline
  The Gross Motor Function Classification System (GMFCS), is a five-level classification that differentiates children with cerebral palsy based on the child's current gross motor abilities, limitations in gross motor function, and need for assistive technology and wheeled mobility (Rosenbaum et al., 2008).
• Level of Manual Ability Classification System (MACS) — Baseline
  Manual Ability Classification System (MACS) classifies the child's ability from 4 to 18 years old to handle objects in daily activities on one of 5 levels (level I: greater ability, level V: less ability). MACS level I (able to handle objects easily and successfully), level II (able to handle most objects but with somewhat reduced quality and/or speed of achievement so that alternate ways of performance might be used), level III (handles objects with difficulty; needs help to prepare and/or modify activities), level IV (needs continuous support, handles a limited selection of objects), level V (does not handle objects, needs total assistance) (Eliasson et al., 2006).
• Level of Bimanual Fine Motor Function (BFMF) — Baseline
  Bimanual Fine Motor Function (BFMF) classifies fine motor function according to the child's best ability (capacity) to grasp, hold and manipulate objects for each hand separately. BFMF can be used in children from 3-18 years of age (Elvrum et al., 2016).
• Level of Visual Function Classification System (VFCS) — Baseline
  The Visual Function Classification System (VFCS) is a tool used to classify the level of visual function in individuals with disabilities, particularly in the context of cerebral palsy. It is divided into five levels, each describing the degree of visual ability and the impact on daily activities (Baranello et al., 2020).
• Level of Communication Function Classification System (CFCS) — Baseline
  The Communication Function Classification System (CFCS) is a classification tool used to assess and categorize the communication abilities of individuals with cerebral palsy or similar disabilities. It is divided into five levels, each describing the individual's ability to communicate in different environments with both familiar and unfamiliar partners.
• Level of Viking Speech Scale (VSS) — Baseline
  The Viking Speech Scale (VSS) is a classification system designed to assess and categorize the speech abilities of individuals with cerebral palsy or other neurological conditions that affect communication. It focuses on the clarity and intelligibility of speech, as well as the individual's ability to communicate effectively with others. The scale is divided into four levels (Pennington et al., 2013).
• Level of Eating and Drinking Classification System (EDACS) — Baseline
  The Eating and Drinking Classification System (EDACS) is a tool used to classify the eating and drinking abilities of individuals with disabilities, particularly in the context of cerebral palsy. It categorizes individuals into different levels based on their ability to safely and efficiently eat and drink, with a focus on both the safety and efficiency of these activities. The system is divided into five levels (Sellers et al., 2014).
• Neuroradiological classification according to the classification promoted by SCPE (MRICS) — Baseline
  Magnetic Resonance Imaging Classification System is used in the context of neuroradiological assessment in cerebral palsy. A. Maldevelopments A.1. Disorders of cortical formation (proliferation and/or migration and/or organization A.2. Other maldevelopments (examples: holoprosencephaly Dandy-Walker malformation, corpus callosum agenesis, cerebellar hypoplasia) B. Predominant white matter injury B.1. PVL (mild/severe) B.2. Sequelae of IVH or periventricular haemorrhagic infarction B.3. Combination of PVL and IVH sequelae C. Predominant grey matter injury C.1. Basal ganglia/thalamus lesions (mild/moderate/severe) C.2. Cortico-subcortical lesions only (watershed lesions in parasagittal distribution/multicystic encephalomalacia) not covered under C3 C.3. Arterial infarctions (middle cerebral artery/other) D. Miscellaneous (examples: cerebellar atrophy, cerebral atrophy, delayed myelination, ventriculomegaly not covered under B, haemorrhage not covered under B, brainstem lesions, calcific

Trial sites (3)

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