Long-term Outcomes

Primary biliary cholangitis (PBC) is an autoimmune liver disease predominantly affecting middle-aged women. While historically it was deemed rare, advancements in specific auto-antibody tests have led to increased recognition of PBC. The long-term survival of PBC patients in China is not yet fully understood. Several studies have investigated the prognosis of PBC in China. While these studies provide valuable insights into the disease characteristics and prognostic factors of PBC in China, they are all single-center studies with limitations. They lack consideration of the impact of symptoms, varying disease stages, and second-line treatments on prognosis. Therefore, there is a pressing need for multicenter and large-scale studies to further elucidate the characteristics and long-term survival of PBC patients in China. Biochemical response to ursodeoxycholic acid (UDCA) is an independent factor associated with long-term survival. Unfortunately, approximately 30-40% of PBC patients demonstrate insufficient biochemical response to UDCA. In our cohort, we reported a higher nonresponse rate, with about 44% according to the Paris I criteria. These patients remain at risk for disease progression to advanced stages and may benefit from additional second-line drug therapies. With significant advancements in the development of new drugs for PBC, a comprehensive understanding of patients with suboptimal responses to UDCA-including long-term prognosis, the distribution of different disease stages, and the prevalence of pruritus-will provide a basis for individualized treatment strategies. Additionally, fatigue and pruritus are prevalent symptoms for PBC patients and fluctuates independently of disease activity or stage, which significantly diminishing health-related quality of life. However, the incidence and impact of fatigue and pruritus on long-term outcomes for PBC patients in China need further clarification. Therefore, we would like to conduct this multicenter study to estimate the prevalence of PBC in China and to evaluate the symptomatic burden, treatment, and long-term outcomes among PBC patients in China.

Status epilepticus (SE) is a common life-threatening neurological emergency in which prolonged or multiple closely spaced seizures can result in long-term impairments. SE remains associated with considerable mortality and morbidity, with little progress over the last three decades. The proportion of patients who die in the hospital is about 20% overall and 40% in patients with refractory SE. Morbidity is more difficult to evaluate, as adverse effects of SE are often difficult to differentiate from those attributed to the cause of SE. Our experience suggests that nearly 50% of patients may experience long-term functional impairments. The precise description of the consequences of these functional impairments and their impact on quality of life after SE requiring intensive care management has been little studied. Indeed, if cognitive, physical and mental impairments are now identified in the populations of patients who required intensive care under the term postresuscitation syndrome (PICS), neuronal lesions consecutive to the SE itselfor to its cause could be responsible for these different functional alterations. Thus, the following have been described: (i) cognitive disorders in the areas of attention, executive functions and verbal fluency, visual and working memory disorders, but also spatio-temporal disorders; (ii) physical disorders such as the so-called post-resuscitation polyneuromyopathy; and (iii) mental disorders such as anxiety disorders, depressive states or those related to post-traumatic stress. Assessment and characterization of patient-reported outcomes is essential to complement the holistic assessment of clinically relevant outcomes from the patient's perspective. The POSEIDON study was a cross-sectional collection of PROs and HR-QOL components, and associated with patient functional outcomes, in those who required ICU management for status epilepticus. We propose here to continue the description of potential alterations after a subsequent ME, namely a longitudinal study (POSEIDON 2) which will also include the evaluation of patient-reported experience (PREMS) and the measurement of family burden.