RESUMO
Parkinson's disease (PD) is the second most common neurodegenerative disorder, and the condition is complicated by the emergence of wearing off/motor fluctuations with levodopa treatment after a variable period. COMT inhibitors when used as adjunct therapy to levodopa tend to smoothen out these wearing off fluctuations by enhancing delivery of levodopa and increasing its bioavailability to the brain. The study was conducted to investigate the motor and nonmotor effect, safety and tolerability of the third generation once-daily COMT inhibitor (opicapone), as add-on, adjuvant therapy to levodopa and at 6 and 12 months follow-up in a real-life cohort of consecutive Emirati and non-White PD patients. A real-life observational analysis using tolerability parameters as used previously by Rizos et al. and Shulman et al. based on clinical database of cases rat Kings College Hospital Dubai Parkinson care database. This was a prospective, single-arm follow-up clinical evaluation study that evaluated the effectiveness of opicapone 50 mg once-daily regime in 50 patients diagnosed with idiopathic neurodegenerative disorder. All patients were assessed with scales used in clinical pathway and include motor Unified Parkinson's Disease Rating Scale (UPDRS), nonmotor symptom scale (NMSS), quality of life (PDQ8) Parkinson's fatigue scale (PFS16) and King's Parkinson's Pain Scale (KIPS). Out of 50 patients treated with opicapone (72% male, mean age 66.9 years (SD 9.9, range 41-82 years) and mean duration of disease 5.7 years (SD 2.5 range (2-11), there was significant statistical improvements shown in motor function-UPDRS part 3: baseline 40.64 ± 2.7, at 6 months 32.12 ± 3.14 and after 12 months 33.72 ± 3.76. Nonmotor burden NMSS: 107.00 ± 21.86, at 6 months 100.78 ± 17.28 and 12 months 96.88 ± 16.11. Reduction in dyskinesias (UPDRS part 4): baseline 8.78 ± 1.07, at 6 months 7.4 ± 0.81 and 12 months 6.82 ± 0.75. Opicapone provides beneficial motor and nonmotor effects in Emirati and other non-White Parkinson's patients, resident in UAE, proving its efficacy across different racial groups as COMT activity may vary between races.
Assuntos
Doença de Parkinson , Humanos , Masculino , Animais , Ratos , Adulto , Pessoa de Meia-Idade , Idoso , Idoso de 80 Anos ou mais , Feminino , Doença de Parkinson/tratamento farmacológico , Levodopa/efeitos adversos , Antiparkinsonianos/efeitos adversos , Emirados Árabes Unidos , Estudos Prospectivos , Qualidade de Vida , Inibidores de Catecol O-Metiltransferase/farmacologia , Inibidores de Catecol O-Metiltransferase/uso terapêuticoRESUMO
Leptomeningeal carcinomatosis (LC) is a rare but serious complication of cancer in which cancer cells spread to the leptomeninges, the membranes that surround the brain and spinal cord. The diagnosis and treatment of LC can be challenging due to the non-specific symptoms and the difficulty of accessing the leptomeninges for a biopsy. In this case report, we describe a patient with advanced breast cancer who was diagnosed with LC and underwent treatment with chemotherapy. Despite aggressive treatment, the patient's condition worsened over time, and she was referred to palliative care, where adequate symptom control was achieved, and she was discharged to her home country as per her wish. Our case highlights the difficulties associated with the diagnosis and treatment of LC and the need for continued research to improve outcomes for patients with this condition. It specifically highlights the approach a palliative care team can take for this condition.
RESUMO
BACKGROUND: Phenotypic differences in Parkinson's Disease (PD) among locals (Emiratis) and Expatriates (Expats) living in United Arab Emirates have not been described and could be important to unravel local aspects of clinical heterogenicity of PD pointing towards genetic and epigenetic variations. OBJECTIVE: To investigate the range and nature of motor and nonmotor clinical presentations of PD and its impact on time to diagnosis, local service provisions, and quality of life in Emiratis and Expats in UAE, as well as address the presence of current unmet needs on relation to care and etiopathogenesis of PD related to possible genetic and epigenetic factors. METHODS: a cross-sectional one point in time prospective, observational real-life study of 171 patients recruited from PD and Neurology clinics across United Arab Emirates from 2019-2021. Primary outcomes were sociodemographic data, motor and nonmotor symptoms (NMS), including cognition and sleep, and quality of life (QOL) assessments, Results: A total of 171 PD patients (52 Emiratis 119 Expats) were included with mean age (Emiratis 48.5 (13.1) Expats 64.15 (13.1)) and mean disease duration (Emiratis 4.8 (3.2) Expats 6.1 (2.9)). In the Emiratis, there was a significant mean delay in initiating treatment after diagnosis (Emiratis 1.2 (0.9) Expats 1.6 (1.1)), while from a clinical phenotyping aspect, there is a high percentage of akinesia 25 (48.1) or tremor dominant (22 (42.3)) phenotypes as opposed to mixed subtype 67 (56.3) in Expat cohorts; double tremor dominant, especially Emirati females (25%), had a predominant lower limb onset PD. Both Emirati (27.9 (24.0)) and Expat 29.4 (15.6) showed moderate NMS burden and the NMS profile is dominated by Sleep, Fatigue, Mood, Emotional well-being 3.0 (1.1) and Social Stigma 3.5 (0.9) aspects of PDQ8 SI measurements are predicted worse QOL in Emiratis, while lack of social support 2.3 (1.3) impaired QOL in Expat population. Awareness for advanced therapies was low and only 25% of Emiratis were aware of deep brain surgery (DBS), compared to 69% Expats. Only 2% of Emiratis, compared to 32% of Expats, heard of Apomorphine infusion (CSAI), and no (0%) Emiratis were aware of intrajejunal levodopa infusion (IJLI), compared to 13% of expats. CONCLUSION: Our pilot data suggest clinical phenotypic differences in presentation of PD in Emiratis population of UAE compared to expats. Worryingly, the data also show delayed treatment initiation, as well as widespread lack of knowledge of advanced therapies in the Emirati population.
RESUMO
BACKGROUND: Pancreatic mucinous cystadenocarcinoma is a rare malignancy. Our aim was to investigate the demographic, pathological characteristics, treatment modalities and survival of patients with mucinous cystadenocarcinoma via the National Cancer Institute's Surveillance Epidemiology and End Results (SEER) database analysis. MATERIALS AND METHODS: This was a retrospective study of SEER database's records on patients with mucinous cystadenocarcinoma diagnosed from 1988 to 2012. Primary outcome measures were clinico-pathological characteristics, observed and disease-specific survival. RESULTS: A total of 507 patients were identified. Median age at diagnosis was 67 years and most patients were female (68.4%). The tumors were mainly low grade (82.9%, grade I-II) and frequently localized (42.8%) in the body/tail of the pancreas (45.6%). According to Kaplan-Meier curves observed survival was 111 months (95%CI: 82.5, 139.5) vs 14 months (95% CI: 10.9, 17.1) vs 4 months (95%CI: 2.9, 5.1) for patients with localized, regional and distant disease, respectively. One-year disease-specific survival for patients with localized disease was 90.1%, vs. 56.7% for those with regional and 18.7% with distant tumor spread. CONCLUSIONS: Mucinous cystadenocarcinomas tend to be low grade tumors, localized to the pancreatic body/tail. Surgery as the primary therapeutic intervention and tumor stage are independent predictors of disease-specific survival.