Faculty Bibliography

Importance/UNASSIGNED:The expanded access (EA) pathway permits patients to be treated with investigational medical products outside clinical trials. Because cancer care is a common indication for which EA is sought and these efforts require physician management, understanding oncologists' perspectives can help illuminate factors influencing patient access. Objective/UNASSIGNED:To learn how oncologists practicing at academic medical centers (AMCs) perceive EA and their role in offering it. Design, Setting, and Participants/UNASSIGNED:This qualitative study used data from semistructured interviews conducted from February 2020 to September 2021 with a purposive sample of oncologists recruited from large, urban AMCs in the northeast United States. Oncologists who had submitted at least 1 single-patient EA request to the institutional review boards at the University of Pennsylvania, Children's Hospital of Philadelphia, NYU Langone Health, and Dana-Farber Cancer Institute from January 1, 2014, through January 31, 2020, were eligible to participate. Data were analyzed from July 2021 to March 2022. Main Outcomes and Measures/UNASSIGNED:Interviews focused on oncologist practice demographics, experience with EA, factors relevant to decisions to pursue EA and comfort with those decisions, perspectives on oncologists' role in EA, perspectives on the FDA's role, and the Right to Try pathway to access investigational drugs. Results/UNASSIGNED:Eligible oncologists were interviewed until thematic saturation was reached, resulting in 25 interviews; most participants were women (15 participants [60%]), reported primarily treating adult patients (15 participants [60%]), had more than 10 years of clinical experience (16 participants [64%]), and had submitted at least 2 single-patient EA requests to their institutional review boards during the relevant period (14 participants [56%]). Oncologists viewed EA as an important tool for securing what they determined to be the best treatment option for their patients based on their own expert assessment of available data. Interviewees reported that they would rather access interventions as commercially available products or through clinical trials; however, if the preferred option was not available through these means, they viewed pursuit of EA as part of their obligation to patients, while often recognizing the potential for inequities in the broader patient population beyond their institutions. Participating oncologists felt confident pursuing investigational drugs for treatment use, despite the absence of FDA marketing approval, and did not necessarily view EA as a last resort. Conclusions and Relevance/UNASSIGNED:These findings indicate that oncologists practicing in large academic settings sought to treat patients with the interventions they deemed most likely to be beneficial, regardless of approval status. As such, they viewed EA as an unexceptional means to obtain promising products, although it remains unclear whether their confidence in evaluating investigational treatments was justified. Future research should examine whether oncologists outside large AMCs share this confidence, as differences may influence patient access to the EA treatment pathway.

UNLABELLED:Facial transplantation (FT) has advanced extensively over the past two decades, with over 40 transplants performed to date. Over this time, the FT literature has evolved as well, from early discussions on ethics and feasibility of FT to functional outcomes reports more recently. We aimed to evaluate the entire body of FT literature to identify trends in publications over time in addition to current existing gaps in the field. METHODS/UNASSIGNED:We conducted a comprehensive bibliometric analysis of the published FT literature from 1994, the first year FT was mentioned in the literature, through July 2020. Co-authorship and keyword information were analyzed using VOSviewer. Articles were manually categorized based on keywords and their aim to provide insight on trends. RESULTS/UNASSIGNED:A total of 2182 articles were identified. Analysis identified the top 50 publishing authors in the field and demonstrated co-authorship linkage between 84.8% of the top 1000 authors. Clinical surgical techniques, protocols, and experiments were the most frequently published category. Within clinical outcomes, immunologic outcomes were most frequent, while psychosocial were the lowest. Gaps were identified in long-term outcomes reporting and patient-reported outcomes, with physician-reported outcomes heavily outweighing patient-reported outcomes. CONCLUSIONS/UNASSIGNED:As the field continues to evolve, rigorous tracking of publication patterns over time will encourage development of a more robust evidence base, identify gaps in the published literature, and highlight opportunities to enhance collaboration in the field. This data will provide surgeons and research institutions with information to further improve this life-changing procedure.

The field of vascularized composite allotransplantation (VCA) is evolving, with some procedures poised to transition from highly experimental research toward standard of care. At present, the use of social support as an eligibility criterion for VCA candidacy is at the discretion of individual VCA programs, allowing VCA teams to consider the unique needs of each potential candidate. Yet this flexibility also creates potential for bias during the evaluation process which may disproportionately impact members of certain communities where social configurations may not resemble the model considered "optimal." We examine the extent to which ethical considerations for social support in solid organ transplantation (SOT) may be applied to or adapted for VCA, and the ethically meaningful ways in which VCA procedures differ from SOT. We conclude that VCA programs must retain some flexibility in determining criteria for candidacy at present; however, considerations of equity will become more pressing as VCA procedures evolve toward standard of care, and further empirical evidence will be needed to demonstrate the association between social support and post-operative success. The field of VCA has an opportunity to proactively address considerations of equity and justice and incorporate fair, inclusive practices into this innovative area of transplantation.

Purpose: Although the number of older adults who might benefit from kidney transplantation continues to increase in the United states, little is known about older adults' lived experience of transplantation and the impact of transplant on their sense of embodied selfhood.
Method(s): Employing a phenomenological research design, this study explored the lived experience of 10 deceased donor kidney transplant recipients aged 65 to 72 years old. Participants completed one to two in-depth phenomenological interviews lasting approximately one hour each, for a total of 15 interviews. The sample was drawn from a transplant center in an urban location in the United States. Interview transcripts and field notes were analyzed following the processes of phenomenological reduction, imaginative variation and synthesis.
Result(s): Analyses illuminated older adults' perceptions of the impact of kidney transplantation on their sense of physical, psychological and social selfhood. Participants reported experiencing physical and psychosocial challenges as they adjusted to their transplants, yet they also constructed powerful narratives of resilience and coping that were rooted in a deeply held sense of identity developed over the life course. This sense of continuity of the embodied self over time enabled most participants to better navigate the corporeal disruption and re-integration of transplantation. Participants expressed profound appreciation for their transplants and reported substantial improvement in quality of life as compared to their pre-transplant experience on dialysis. Most participants perceived their older age as playing a protective role in the recovery process.
Conclusion(s): These findings shed light on benefits of older age for kidney transplant recipients that may not be fully integrated into standard approaches to patient selection and assessment of transplant outcomes. As such, findings may help to address age-related bias in transplant policy and clinical practice, with the ultimate goal of improving wellbeing and quality of life for older adults

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.