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Rare Diseases Drug Development
Published in Wei Zhang, Fangrong Yan, Feng Chen, Shein-Chung Chow, Advanced Statistics in Regulatory Critical Clinical Initiatives, 2022
Shein-Chung Chow, Shutian Zhang, Wei Zhang
Fast track designation is designed to aid in the development and expedite the review of drugs which show promise in treating a serious or life-threatening disease and address an unmet medical need. Serious condition is referred to as the determination of the seriousness of a disease. The determination is a matter of judgment, but generally is based on whether the drug will affect the factors such as survival, day-to-day functioning, or the likelihood that the disease (if left untreated) will progress from a less severe condition to a more serious one. For a drug to address an unmet medical need, the drug must be developed as a treatment or preventative measure for a disease that exists no current therapy. If there are existing therapies, a fast track eligible drug must show some advantage over available treatment. For example, (i) showing superior effectiveness, (ii) avoiding serious side effects of an available treatment, (iii) improving the diagnosis of a serious disease where early diagnosis results in an improved outcome, (iv) decreasing a clinically significant toxicity of an available treatment, and (v) addressing an expected public health need.
Biochemistry of Exercise Training and Type 1 Diabetes
Published in Peter M. Tiidus, Rebecca E. K. MacPherson, Paul J. LeBlanc, Andrea R. Josse, The Routledge Handbook on Biochemistry of Exercise, 2020
Sam N. Scott, Matt Cocks, Anton J. M. Wagenmakers, Sam O. Shepherd, Michael C. Riddell
Mild hypoglycaemia is defined as a blood glucose concentration of 3.0–3.9 mmol/L; serious or clinically important hypoglycaemia can be defined as a blood glucose concentration ≤2.9 mmol/L; while severe hypoglycaemia is defined as the patient requiring assistance from another for recovery (90). Interestingly, symptoms of hypoglycaemia (i.e., shakiness, hunger, confusion) can occur at blood glucose levels above 3.9 mmol/L, particularly in individuals recently diagnosed with T1D and who have been in a state of chronic hyperglycaemia (156). However, it is also important to note that in individuals with hypoglycaemia unawareness, symptoms are not triggered until blood glucose levels are very low, often after cognitive function is impaired (78). The symptoms of hypoglycaemia can range in seriousness from mild tremor, loss of coordination, and mental confusion to convulsions, unconsciousness, brain damage, and even death (15, 46). Reports estimate that people with T1D are exposed to, on average, 3.5–7.2 episodes of symptomatic hypoglycaemia per month (24, 70, 135), although studies using continuous glucose monitors show higher unnoticed (often nocturnal) incidents (93, 105). On average, around 12% of adults living with T1D experience at least one severe temporarily disabling episode of hypoglycaemia per year (79, 116, 190, 198).
Eating disorders
Published in Quentin Spender, Judith Barnsley, Alison Davies, Jenny Murphy, Primary Child and Adolescent Mental Health, 2019
Quentin Spender, Judith Barnsley, Alison Davies, Jenny Murphy
A young person who has not yet seen her general practitioner should do so, as she is in the best position to consider the medical seriousness and the possibility of any other medical conditions. Some parents or young people may not immediately accept referral to a mental health service; for these families, initial referral could be to a paediatrician or paediatric dietician.
The attributable mortality of sepsis for acute kidney injury: a propensity-matched analysis based on multicenter prospective cohort study
Published in Renal Failure, 2023
Hui-Miao Jia, Yi-Jia Jiang, Xi Zheng, Wen Li, Mei-Ping Wang, Xiu-Ming Xi, Wen-Xiong Li
The various mortalities of septic AKI were reported in different studies. Our study showed a hospital mortality of 33.1% in matched AKI patients with sepsis compared with 24.0% of their matched AKI controls without sepsis. Angus et al. examined 192,980 patients with severe sepsis from seven US states [33]. They found AKI occurred in 22% and was associated with a mortality of 38.2%. The SOAP cohort study recruited 3147 patients [34]. Of them, AKI occurred in 51% of sepsis cases and was related to an ICU mortality of 41%. Different from studies above, this study highlights the attributable mortality of sepsis for AKI, and a close link was observed between increased mortality and sepsis severity. There are a lot of studies focused on septic AKI, we should also be concerned for the AKI patients who developed sepsis after AKI. Whether sepsis developed before, simultaneously or after AKI, it would deteriorate organ function and contribute excess mortality for AKI. Furthermore, septic shock contributed to major excess mortality for AKI than sepsis. The seriousness of this condition emphasized the need for prompt and appropriate intervention. Prevention of sepsis development and progress to septic shock may represent a potential key therapeutic target for AKI and decrease mortality.
Pulmonary rehabilitation assessment in COPD based on the ICF brief core set: a latent profile analysis
Published in Annals of Medicine, 2023
Xinyu Wang, Xiaoxuan Meng, ZhenJie Yu, Yongmei Zhang, Yane Li, Xi Yu, Jingchun He, Jingling Zhang, Lan Wang
The low-dysfunction group of patients had the mildest grade of functional impairment, with a better ability to perform daily activities and exercise function. Patients in this group showed poorer respiratory function and cardio-respiratory endurance compared to their activity participation ability, suggesting that patients’ body dysfunction had less impact on activity and participation. In terms of clinical characteristics, this group had a younger mean age (67.58 ± 9.08) and less severe disease. More patients in the low dysfunction group did not use inhalation medication compared to those in the other groups (19.2%), and most patients did not participate in oxygen therapy (72.6%), which might be related to the lower symptom burden in this group. Previous studies have noted that PR programs improved exercise tolerance and reduced exacerbations in patients with mild COPD, including warm-up training, endurance training, psychosocial support, and education [54]. However, high dropout rates are a common challenge in conducting PR programs for patients with mild COPD, and patient compliance with PR programs is poor [45]. It might be associated with patients’ less seriousness towards their disease symptoms and management. Studies showed that low adherence in patients to PR programs was associated with current smoking. Most patients with mild COPD have problems with quitting smoking, affecting patient adherence to PR programs and reducing PR efficacy [55]. To address these issues, the PR program for such patients needs to pay particular attention to adherence behaviours based on maintaining cardiopulmonary endurance.
Building the foundation for a community-generated national research blueprint for inherited bleeding disorders: research priorities to transform the care of people with hemophilia
Published in Expert Review of Hematology, 2023
Duc Q. Tran, Craig C. Benson, Judith A. Boice, Meera Chitlur, Amy L. Dunn, Miguel A. Escobar, Kalpna Gupta, Jill M. Johnsen, James Jorgenson, Scott D. Martin, Suzanne Martin, Shannon L. Meeks, Alfredo A. Narvaez, Doris V. Quon, Mark T. Reding, Ulrike M. Reiss, Brittany Savage, Kim Schafer, Bruno Steiner, Courtney Thornburg, Lena M. Volland, Annette von Drygalski
The management of hypertension in PWH exemplifies the need for collaborative care that extends beyond the traditionally defined HTC. PWH tend to have higher blood pressure than the general male population. The etiology remains poorly understood and the phenomenon is underrecognized [19,57–59]. Primary care physicians must be made aware of the urgency of controlling high blood pressure in, even young, PWH since it increases the risk for hemorrhagic stroke, a major cause of death in this population [60,61]. The increased incidence of certain risk factors in some populations (e.g. African American PWH), obstacles to accessing or paying for the required medications (e.g. type or lack of insurance), and lack of comprehension of the seriousness of the condition (e.g. younger PWH) necessitate a collaborative management plan between the primary care physician, the HTC multidisciplinary care team, and the PWH. Improving education and awareness with primary care physicians and PWH is even more important for individuals not cared for at an HTC.