UNASSIGNED: Neuromyelitis optica spectrum disorder (NMOSD) is a rare and debilitating disease that has become more widely recognized in China. Legislative measures have been implemented by the government to improve treatment access for rare diseases.
UNASSIGNED: To investigate the diagnostic journey, treatment status, treatment accessibility, and treatment satisfaction of the NMOSD patients on disease-modifying therapies (DMTs) in China.
UNASSIGNED: A patient online survey.
UNASSIGNED: This cross-sectional online survey was conducted between November 2022 and January 2023. Patients over 18 years old and diagnosed with NMOSD were included. The questionnaire consisted of five sections covering demographics, diagnostic and treatment experiences, DMTs availability, cost and affordability, and treatment satisfaction using the Treatment Satisfaction Questionnaire for Medication (version II). Patient opinions and demands were also collected at the end of the survey.
UNASSIGNED: A total of 375 patients diagnosed with NMOSD were recruited, of which 321 patients used DMTs. It required 1.22 ± 3.22 years and 3.58 ± 4.24 hospital visits for a definitive diagnosis. One-third of the patients still needed to travel for over 2 h to access DMTs. The total treatment expenditure was estimated to be CNY 59,827.00 (USD 8315.95) a year. Drug expenses alone accounted for 52.22% of the average annual household income. The most common challenges perceived were the inability to afford treatment and a lack of effective options. No significant difference was found in treatment satisfaction among DMTs, except that rituximab scored lowest in convenience compared to other DMTs. Patients\' age and travel time required to obtain medications were negatively associated with global treatment satisfaction.
UNASSIGNED: In China, patients with NMOSD face challenges in obtaining proper treatment due to diagnostic difficulties, distant medication access, and high costs. Policies should prioritize improving disease education and alleviating financial burdens for the patients.

SCAN, an online survey, measured access to diagnosis, treatments and monitoring of neuroendocrine tumor (NET) patients globally. Between September and November 2019, NET patients and healthcare professionals (HCPs) completed an online, semi-standardized survey with 54 patient questions and 33 HCP questions. A total of 2359 patients with NETs and 436 HCPs responded. Misdiagnosis was common (44% [1043/2359]). Mean time to diagnosis was 4.8 years (standard deviation [SD], 6.2). Compared with global figures (60% [1407/2359]), the availability of 68 Ga-DOTA positron emission tomography (PET)/computed tomography (CT) was significantly lower in Asia (45% [126/280]) and higher in Oceania (86% [171/200]). HCPs reported that 68 Ga-DOTA PET/CT was free/affordable to fewer patients in Emerging and Developing Economies (EDE) than Advanced Economies (AE; 17% [26/150] and 59% [84/142], respectively). Compared with global data (52% [1234/2359]), patient-reported availability of peptide receptor radionuclide therapy (PRRT) was significantly lower in Asia (31% [88/280]) and higher in Oceania (61% [122/200]). Significant differences were observed in average annual NET specialist costs between AE and EDE ($1081 and $2915, respectively). Compared with AE, patients in EDE traveled further for NET specialists (1032 [SD, 1578] and 181 [SD, 496] km, respectively). Patients and HCPs both recommended referral to HCPs that were more knowledgeable in the field of NETs and had better access to NET experts/specialist centers. National care pathways, enhancing HCP NET knowledge and ensuring effective diagnostics and access to appropriate treatments are crucial to improving patient survival and NET care worldwide.

Uncontrolled epilepsy creates a constant source of worry for patients and puts them at a high risk of injury. Identifying recurrent \"premonitory\" symptoms of seizures and using them to recalibrate seizure prediction algorithms may improve prediction performances. This study aimed to investigate patients\' ability to predict oncoming seizures based on preictal symptoms.
Through an online survey, demographics and clinical characteristics (e.g., seizure frequency, epilepsy duration, and postictal symptom duration) were collected from people with epilepsy and caregivers across Canada. Respondents were asked to answer questions regarding their ability to predict seizures through warning symptoms. A total of 196 patients and 150 caregivers were included and were separated into three groups: those who reported warning symptoms within the 5 minutes preceding a seizure, prodromes (symptoms earlier than 5 minutes before seizure), and no warning symptoms.
Overall, 12.2% of patients and 12.0% of caregivers reported predictive prodromes ranging from 5 minutes to more than 24 hours before the seizures (median of 2 hours). The most common were dizziness/vertigo (28%), mood changes (26%), and cognitive changes (21%). Statistical testing showed that respondents who reported prodromes also reported significantly longer postictal recovery periods compared to those who did not report predictive prodromes (P < 0.05).
Findings suggest that patients who present predictive seizure prodromes may be characterized by longer patient-reported postictal recovery periods. Studying the correlation between seizure severity and predictability and investigating the electrical activity underlying prodromes may improve our understanding of preictal mechanisms and ability to predict seizures.

Lysosomal storage diseases (LSDs) are a group of rare diseases that cause progressive physical dysfunction and organ failure, which significantly affected patients\' quality of life. The objective of this study was to explore the characteristics and usage of Enzyme Replacement Treatments (ERTs), which is the only specific therapy for LSDs, of patients with the four different LSDs (Gaucher, Fabry, Pompe disease and Mucopolysaccharidosis) in Shanghai, and then evaluate the economic burden and quality of life of these patients. A total of 31patients, involving 5, 14, 4 and 8 patients with Gaucher, Fabry, Pompe disease and Mucopolysaccharidosis, respectively, were included in analysis. The result showed that only five Gaucher disease (GD) patients in Shanghai used Imiglucerase in 2019, while the other 26 patients with the other three LSDs did not receive ERTs. The total health expenditure of GD patients was 2,273,000CNY on average mainly resulted by the high cost of Imiglucerase. The total health expenditure of the other 26 patients was 37,765CNY on average. Though the cost-sharing mechanism between basic medical insurance, charity fund and patients had been explored for Gaucher disease in Shanghai, the out-of-pocket part, which was 164,301 CNY, still laid a heavy economic burden on the patients and their families. The mean EQ-VAS score of GD patients was 76.4 ± 15.5, which was higher than that of the other three LSDs. It is recommended that the scope of drug reimbursement list and the reimbursement level should be further expanded and raised to help improve the living conditions of patients with LSDs.

This review aims to summarize the progress of patient evaluation studies focusing on primary health care (PHC) in China, specifically in relation to survey instruments and implementation issues. Eligible studies published in English or Chinese were obtained through online searches of PubMed and China National Knowledge Infrastructure. A descriptive reporting approach was used due to variations in the measurements and administration methods between studies. A total of 471 articles were identified and of these articles; of those 91 full-text articles were included in the final analysis. Most studies used author-developed measurements with five-point Likert response scales and many used the Chinese translations of validated tools from other countries. Most instruments assessed the physical environment, medical equipment, clinical competency and convenience aspects of PHC using a satisfaction rating instead of care experience reporting. Many studies did not report the sampling approach, patient recruitment procedures and survey administration modes. The patient exit survey was the most commonly used survey implementation method. The focus on the structural dimensions of PHC, inconsistent wording, categories of response options that use satisfaction rating, and unclear survey implementation processes are common problems in patient evaluation studies of PHC in China. Further studies are necessary to identify population preferences of PHC in China in order to move towards developing Chinese value-based patient experience measurements.