Validation of Polish version of Gastrointestinal Dysfunction Scale for Parkinson's Disease.

AIM OF STUDY: The Gastrointestinal Dysfunction Scale for Parkinson's Disease (GIDS-PD) is a novel, disease-specific self-report questionnaire used to quantitatively assess features of gastrointestinal dysfunction symptoms in patients with Parkinson's Disease. The aim of this paper was to validate the Polish translation of the scale, to summarise its consistency with the English language version, and to assess its clinimetric properties. CLINICAL RATIONALE FOR STUDY: Gastrointestinal dysfunction is a common and often debilitating manifestation of Parkinson's Disease (PD). Gastrointestinal symptoms are also considered to be prodromal features of this disease. To date, there has been no scale in Polish that has precisely assessed gastrointestinal symptoms in patients with PD. MATERIAL AND METHODS: The GIDS-PD was translated into Polish by two investigators (M.K. and J.N.). A back-translation was completed by two separate investigators (M.F. and A.A.) who were not involved in the original translation. Afterwards, 10 Polish PD patients underwent cognitive pre-testing. After the final translation was officially approved by the Movement Disorder Society, it was tested on 64 individuals with PD during field testing. For the purpose of testing scale reliability, 20 of the patients recruited for field testing underwent the GIDS-PD for a second time after 8-12 weeks. RESULTS: The GIDS-PD demonstrated overall good consistency (Cronbach's alpha of 0.74, ICC of 0.74). Regarding the individual domains, the constipation subscore demonstrated good reliability, the bowel irritability subscore demonstrated moderate reliability, and the upper GI subscore demonstrated poor reliability. Upper GI symptoms seem to be less pronounced, and also more varied, in the Polish PD population than in its English language counterpart. CONCLUSIONS AND CLINICAL IMPLICATIONS: This paper provides a validated Polish translation of the GIDS-PD questionnaire. We highly recommend using the GIDS-PD for research purposes, as well as everyday clinical practice in the Polish PD population.

Protective Effect of Glucocorticoids against Symptomatic Disease in CSF1R Variant Carriers.

BACKGROUND: There is an unmet need for the treatment of colony-stimulating factor-1 receptor (CSF1R)-related leukoencephalopathy. OBJECTIVES: To evaluate the association of glucocorticoids (GCs) with disease onset and progression in CSF1R variant carriers. METHODS: Retrospective cohort study on CSF1R variants carriers (n = 41) whose medical records were collected at Mayo Clinic Florida from 2003 to 2023. We retrieved information on sex, ethnicity, family history, medications, disease onset, course and duration, neuroimaging features, and activities of daily living (ADL). RESULTS: Risk of developing symptoms was significantly lower for individuals who used GCs (n = 8) compared to individuals who did not (n = 33) (12.5% vs. 81.8%, hazard ratio [HR] = 0.10, P = 0.036). The risk of becoming dependent in ADL was markedly lower for the GCs group (0.0% vs. 43.8%, P = 0.006). White matter lesions and corpus callosum involvement were less common in the GCs group (62.5% vs. 96.6%, P = 0.026; 37.5% vs. 84.6%, P = 0.017; respectively). CONCLUSIONS: We found a protective association of GCs in CSF1R variant carriers against developing CSF1R-related leukoencephalopathy. We call for further studies to validate our findings and investigate the potential application of GCs in CSF1R-related leukoencephalopathy. © 2023 International Parkinson and Movement Disorder Society.