AbstractPegcetacoplan is a complement component 3 inhibitor approved to treat adults with paroxysmal nocturnal hemoglobinuria, a rare disease characterized by life-threatening complement-mediated hemolysis (often leading to anemia) and thrombosis. Pegcetacoplan is self-administered as a subcutaneous (SC) injection with an at-home infusion pump. A newly approved, more convenient, wearable, single-use automatic injector was evaluated in a human factors study for the safe and effective SC delivery of pegcetacoplan in the abdominal area. Adult patients with anemia (representative of PNH) and caregivers (n=15 each) received formal injector use training (2-hour session followed by 1-hour decay) and reviewed the instructions for use (IFU), whereas health care providers (HCPs; n=15) only received the IFU. Participants completed a use test, knowledge-based assessment (KBA), and interviews. Forty-four of 45 participants (98%) passed the use test. Residual risks from observed errors (17 critical use and 6 KBA errors) were deemed acceptable. This study validated the injector for ease of use and safe and effective SC delivery of pegcetacoplan at home, which may potentially reduce treatment burden for patients with PNH.

AbstractRationale: There are limited real-world data regarding the proportion of patients with severe asthma (SA) who achieve clinical remission with biologic treatment. Methods: CHRONICLE is an observational study of subspecialist-treated adults with SA. Sites report exacerbations and biologic use from 12 months prior to enrollment forward. Monthly Asthma Control Test (ACT) scores and 6-monthly specialist assessments of asthma control are collected. Clinical remission was evaluated among patients enrolled from February 2018 to February 2021. Remission was defined as absence of systemic corticosteroid (SCS) use for 12 months, majority of recent ACT scores ≥20, and recent asthma control per specialist and patient report. Results: Among evaluable patients with biologic use for ≥12 months (N=908), 68% had no SCS use within the most recent 12 months. A further 35% were SCS-free with most monthly ACT scores ≥20 and patient- and specialist-reported asthma control. Conclusions: In a real-world population, nearly 70% of patients with SA who had ≥12 months of biologic use had no exacerbations or SCS use. Approximately 1 in 3 achieved potential clinical remission.

AbstractNarcolepsy is a chronic neurological disorder characterized by excessive daytime sleepiness, cataplexy, and other REM-dissociative features. Assessment of treatment response requires evaluating multiple domains that include narcolepsy symptoms, comorbid psychiatric conditions (e.g., anxiety, depression, ADHD), quality of life, and medication side effects. Existing scales may be insufficient to capture the dynamic and personalized changes that occur visit by visit during treatment. Instead, individualized, patient-centered assessment entails the identification of specific patient goals and progress over the course of treatment. Optimal assessment of treatment response involves a process in which each patient’s symptoms/functioning are evaluated relative to their previous visits, followed by any necessary adjustments to medication(s) and adjunctive therapies. Input from family members may provide a more complete understanding of the patient’s symptoms and functioning. There is an unmet need for a brief instrument that can be used efficiently in the clinic setting to improve the assessment of response to treatment in patients with narcolepsy. This publication identifies challenges confronted by clinicians and discusses approaches to consider in the ongoing evaluation and treatment of narcolepsy.

AbstractThe modernization of healthcare is critical to all clinicians, especially those who treat patients with chronic pain. Preliminary data suggests that mHealth technology can improve treatment plan adherence, medication protocol compliance and lead to improved patient outcomes. The mHealth platform described bridges a treatment gap that occurs between visits and has been demonstrated to improve clinically important outcomes. Improving physician-patient communication in conjunction with increased patient compliance and monitoring may reduce the risk of opioid misuse and addiction. Importantly, mHealth provides clinicians with information that can help differentiate addiction from tolerance and physical dependence. It may also provide the practice a means to be compensated by the Centers for Medicare & Medicaid Services and many other payors for the time and expertise of providing daily contact with patients. Lastly, this mHealth platform is designed with attributes that serve as a safeguard against legal liability due to enhanced communication and affirmative clinical documentation.

AbstractTheResearchPost is a start-up, open-access, peer-reviewed, medical journal built on a data visualization platform. The journal is designed for medical research, including clinical and preclinical research, real-world evidence, health economics and outcomes research, observational studies, and clinical case studies. The journal's format features slides instead of manuscripts, which streamlines the writing and review process, and better matches how people read and share information. The journal's open-access format makes high-quality, medical research freely available to all.