Faculty Bibliography

INTRODUCTION/UNASSIGNED:Physicians in the United States play an essential role guiding patients through single patient pre-approval access (PAA) to investigational medical products via either the Food and Drug Administration (FDA)'s Expanded Access (EA) or the federal Right To Try (RTT) pathways. In this study, we sought to better understand pediatric hematologist/oncologists' attitudes about seeking PAA, on behalf of single patients, to investigational drugs outside of clinical trials. METHODS/UNASSIGNED:A cross-sectional survey was developed and sent to pediatric hematologist/oncologists via St. Baldrick's Foundation's email distribution list. RESULTS/UNASSIGNED: = 46) had prior experience with single patient PAA. Respondents were most concerned about the unknown risks and benefits of investigational drugs and financial implications of PAA for patients. One hundred percent and 91.1% of respondents indicated a willingness to support patients through EA and RTT pathways, respectively. When asked about their most recent experience with PAA, 40 out of 46 indicated that they used the FDA's EA pathway to seek PAA and 4 out of 46 indicated that they used the RTT pathway. Of 44 respondents who had used the EA or RTT pathway, 43 indicated that the biotechnology or pharmaceutical company they solicited granted access to the requested product. CONCLUSION/UNASSIGNED:Survey results support other findings suggesting a need for additional physician support and education about PAA and that physicians may have unequal access to information about investigational drugs and concerns about financial implications of PAA for their patients.

BACKGROUND: Covid-19 disease, resulting from the virus SARS-CoV-2, has caused significant morbidity and mortality across the globe. In the acute setting, Covid-19 is characterized by its inflammatory impact, notably leading to acute respiratory distress syndrome, increased risk of blood clots, cardiomyopathy, and acute kidney injury. Long-term complications known as longhaul Covid, chronic Covid, or post-Covid syndrome include fatigue, depression, persistent respiratory complaints, and decreased quality of life. However, little research exists to elucidate the primary care needs of those who have recovered from Covid-19. This longitudinal observational study describes healthcare usage patterns and new medical diagnoses after acute Covid-19 infection.
METHOD(S): We queried the NYU Langone COVID Deidentified Dataset for adults 18+ years old with a positive SARS-CoV-2 PCR test. Patients had at least one visit in the NYU Langone Health system >2 weeks before and >2 weeks after infection (n = 2940). We further narrowed this cohort to patients with a primary care encounter where a Covid-19 related concern was documented at follow-up (n = 454; 57% female; 22%=18-42 years, 51%=43-67 years, 27%=68+ years old). The median length of follow-up was 6 weeks (IQR=3.6-10.1 weeks, max=38 weeks). ICD-10 codes and the Clinical Classification Software Refined (CCSR) categories were used to identify diagnoses newly developed after Covid-19 infection or symptoms that persisted beyond the initial 14-day infection period.
RESULT(S): Of 2,940 patients with pre and post-infection visits, only 454 (15%) sought primary care for a Covid-19 related concern. Respiratory signs and symptoms were the most common complaint. Prevalent diagnoses included cough (8%), hypoxia or respiratory failure (8%), and shortness of breath (7%). Malaise and fatigue (7%), musculoskeletal pain (6%), and generalized weakness and deconditioning (5%) were also common. Nutritional deficiencies were documented among 28 patients (6%), most often for vitamin D deficiency (5%). Palpitations (5%) and nonspecific chest pain (such as chest tightness or discomfort, 3%) as well as deep vein thrombosis and pulmonary emboli (4%) were also reported.
CONCLUSION(S): Most patients with a Covid-19 diagnosis did not seek follow-up, consistent with reports that Covid-19 predominantly causes acute illness. For patients who developed new or persistent symptoms, the most common complaints were respiratory concerns, malaise and fatigue, and musculoskeletal pain. The persistence of these symptoms in our cohort suggests that patients may indeed present a constellation of symptoms after acute Covid-19 infection. Updated longitudinal queries and further research may continue to highlight the importance of tailoring longer term primary care for those who have recovered from acute Covid infection. LEARNING OBJECTIVE #1: Identify common symptoms of patients returning to primary care more than 2 weeks after Covid-19 infection LEARNING OBJECTIVE #2: Determine need for primary care follow-up after Covid-19 recovery

Automatic emotion regulation (AER) plays a vital role in the neuropathology underlying both suicide and self-harm via modifying emotional impact effortlessly. However, both the effortless account and the neural mechanisms of AER are undetermined. To investigate the neural changes at AER, we collected functional MRI (fMRI) in 31 participants who attended to neutral and disgust pictures in three conditions: watching, goal intention (GI), and reappraisal by implementation intention (RII). Results showed that RII (but not GI) decreased negative feelings and bilateral amygdala activity without increasing cognitive efforts, evidenced by the reduced effort rating and less prefrontal engagement during RII compared with during watching and GI. These emotion-regulatory effects of RII cannot be explained by emotional habituation, as the supplementary experiment (N = 31) showed no emotional habituation effects when the same disgust pictures were presented repeatedly three times for each watching and GI condition. Task-based network analysis showed both RII and GI relative to watching increased functional connectivities (FCs) of the ventral anterior cingulate cortex to the left insula and right precuneus during conditions, two FCs subserving goal setup. However, RII relative to GI exhibited weaker FCs in brain networks subserving effortful control, memory retrieval, aversive anticipation, and motor planning. In these FCs, the FC intensity of putamen-operculum/lingual and paracentral-superior temporal gyri positively predicted regulatory difficulty ratings. These findings suggest that the setup of implementation intention automatizes emotion regulation by reducing the online mobilization of emotion-coping neural systems.

Background: Despite evidence-based national guidelines for ADHD in the United Kingdom (UK), ADHD is under-identified, under-diagnosed, and under-treated. Many seeking help for ADHD face prejudice, long waiting lists, and patchy or unavailable services, and are turning to service-user support groups and/or private healthcare for help. Methods: A group of UK experts representing clinical and healthcare providers from public and private healthcare, academia, ADHD patient groups, educational, and occupational specialists, met to discuss shortfalls in ADHD service provision in the UK. Discussions explored causes of under-diagnosis, examined biases operating across referral, diagnosis and treatment, together with recommendations for resolving these matters. Results: Cultural and structural barriers operate at all levels of the healthcare system, resulting in a de-prioritization of ADHD. Services for ADHD are insufficient in many regions, and problems with service provision have intensified as a result of the response to the COVID-19 pandemic. Research has established a range of adverse outcomes of untreated ADHD, and associated long-term personal, social, health and economic costs are high. The consensus group called for training of professionals who come into contact with people with ADHD, increased funding, commissioning and monitoring to improve service provision, and streamlined communication between health services to support better outcomes for people with ADHD. Conclusions: Evidence-based national clinical guidelines for ADHD are not being met. People with ADHD should have access to healthcare free from discrimination, and in line with their legal rights. UK Governments and clinical and regulatory bodies must act urgently on this important public health issue.

As more young people feel safe to outwardly identify as transgender or gender expansive (TGE), meaning that their gender identity does not align with the sex they were assigned at birth, an increasing number of youth who identify as TGE seek gender-affirming medical care (GAMC). GAMC raises a number of ethical questions, such as the capacity of a minor to assent or consent, the role of parents or legal guardians in decisions about treatment, and implications for equitable access to care when differing parental or custodial viewpoints are present. These questions are further complicated by the difficulties in explaining the limits of long-term research in GAMC, with regard to the preservation of fertility, for example. We present two de-identified composite case studies to highlight dilemmas that may arise and offer recommendations to better support patient- and family-centered decision making for GAMC. These include employing a multidisciplinary shared decision-making approach, disentangling informed consent and assent from chronological age, developing a consistent approach to the assessment of decisional capacity, and developing age-appropriate informational materials.