Observational Study for the Epidemiology of Cerebral Palsy in Italy

Официальное название

DataNetCP: Establishing a Hospital-based Cerebral Palsy Registry in Italy

Обзор

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.

Вмешательства

• Другое 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

Первичные конечные точки

• Participants demographic data [Срок оценки: Baseline]
• Cerebral palsy (CP) classification [Срок оценки: Baseline]
• Anamnestic data related to the etiopathogenesis of the disorder [Срок оценки: Baseline]
• Level of Gross Motor Function Classification System (GMFCS) [Срок оценки: Baseline]
• Level of Manual Ability Classification System (MACS) [Срок оценки: Baseline]
• Level of Bimanual Fine Motor Function (BFMF) [Срок оценки: Baseline]
• Level of Visual Function Classification System (VFCS) [Срок оценки: Baseline]
• Level of Communication Function Classification System (CFCS) [Срок оценки: Baseline]
• Level of Viking Speech Scale (VSS) [Срок оценки: Baseline]
• Level of Eating and Drinking Classification System (EDACS) [Срок оценки: Baseline]

Критерии участия

Критерии включения

• Diagnosis of cerebral palsy (CP) confirmed clinically in accordance with recent international guidelines (SCPE Guidelines, 2019);
• Age between 4 and 18 years.

Критерии исключения

• Progressive neuromotor disorder (defined as a condition involving the loss of previously acquired abilities);
• Patients with a neurological condition defined by isolated hypotonia;
• Patients with neurological deficits secondary to spinal injury;

Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.

Здоровые добровольцы: Нет

Дизайн исследования

Модель наблюдения

Когортное

Центры проведения

Италия · 3 центра

• Associazione La Nostra Famiglia - IRCCS Eugenio Medea — Brindisi
• Fondazione I.R.C.C.S. Istituto Neurologico Carlo Besta — Milan
• IRCCS Fondazione Stella Maris — Calambrone

Публикации

• Pennington L, Virella D, Mjoen T, da Graca Andrada M, Murray J, Colver A, Himmelmann K, Rackauskaite G, Greitane A, Prasauskiene A, Andersen G, de la Cruz J. Development of The Viking Speech Scale to classify the speech of children with cerebral palsy. Res Dev Disabil. 2013 Oct;34(10):3202-10. doi: 10.1016/j.ridd.2013.06.035. Epub 2013 Jul 24. PMID 23891732
• Bax M, Goldstein M, Rosenbaum P, Leviton A, Paneth N, Dan B, Jacobsson B, Damiano D; Executive Committee for the Definition of Cerebral Palsy. Proposed definition and classification of cerebral palsy, April 2005. Dev Med Child Neurol. 2005 Aug;47(8):571-6. doi: 10.1017/s001216220500112x. PMID 16108461
• Baranello G, Signorini S, Tinelli F, Guzzetta A, Pagliano E, Rossi A, Foscan M, Tramacere I, Romeo DMM, Ricci D; VFCS Study Group. Visual Function Classification System for children with cerebral palsy: development and validation. Dev Med Child Neurol. 2020 Jan;62(1):104-110. doi: 10.1111/dmcn.14270. Epub 2019 Jun 10. PMID 31180136
• Himmelmann K, Horber V, De La Cruz J, Horridge K, Mejaski-Bosnjak V, Hollody K, Krageloh-Mann I; SCPE Working Group. MRI classification system (MRICS) for children with cerebral palsy: development, reliability, and recommendations. Dev Med Child Neurol. 2017 Jan;59(1):57-64. doi: 10.1111/dmcn.13166. Epub 2016 Jun 21. PMID 27325153
• Sellers D, Mandy A, Pennington L, Hankins M, Morris C. Development and reliability of a system to classify the eating and drinking ability of people with cerebral palsy. Dev Med Child Neurol. 2014 Mar;56(3):245-51. doi: 10.1111/dmcn.12352. Epub 2013 Dec 18. PMID 24344767
• Fiori S, Cioni G, Klingels K, Ortibus E, Van Gestel L, Rose S, Boyd RN, Feys H, Guzzetta A. Reliability of a novel, semi-quantitative scale for classification of structural brain magnetic resonance imaging in children with cerebral palsy. Dev Med Child Neurol. 2014 Sep;56(9):839-45. doi: 10.1111/dmcn.12457. Epub 2014 Apr 19. PMID 24750109
• Elvrum AK, Andersen GL, Himmelmann K, Beckung E, Ohrvall AM, Lydersen S, Vik T. Bimanual Fine Motor Function (BFMF) Classification in Children with Cerebral Palsy: Aspects of Construct and Content Validity. Phys Occup Ther Pediatr. 2016;36(1):1-16. doi: 10.3109/01942638.2014.975314. Epub 2014 Nov 6. PMID 25374154
• Eliasson AC, Krumlinde-Sundholm L, Rosblad B, Beckung E, Arner M, Ohrvall AM, Rosenbaum P. The Manual Ability Classification System (MACS) for children with cerebral palsy: scale development and evidence of validity and reliability. Dev Med Child Neurol. 2006 Jul;48(7):549-54. doi: 10.1017/S0012162206001162. PMID 16780622

Идентификаторы

NCT: NCT06813586 · DataNetCP

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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