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Although rare in adults, Wilms tumor is the most common pediatric renal tumor. Treatment typically involves radical nephrectomy followed by adjuvant chemotherapy or radiation, although outcomes differ between children and adults which may be due to challenges in accurately diagnosing these patients. In this article, we present a case report of an adult patient with Jeune syndrome and multiple urologic abnormalities who underwent radical nephrectomy for a large renal mass and was subsequently diagnosed with an epithelial predominant Wilms tumor. Epithelial predominant Wilms tumor may have distinct origins from other Wilms tumor histological subtypes and may incur better outcomes. Herein, we discuss the literature surrounding this rare entity as well as the anticipated treatment course., Competing Interests: The authors declared no potential conflicts of interest with respect to research, authorship, and/or publication of this article., (Copyright: Chapman S, et al.)

Purpose: Sepsis is the leading cause of mortality in patients with childhood cancer receiving cytotoxic chemotherapy. Pediatric hematology/oncology and transplant (PHOT) providers must counsel their patients on the safety of public activities and weigh the risk of infection exposure with the social and developmental benefits of in-person school and social outings. We hypothesize that there is significant variability in recommendations given by PHOT providers., Methods: An electronic anonymous survey was developed and piloted by a group of PHOT providers to assess current methods for educating patients and families on limiting infectious exposures. Five clinical vignettes were created by the study team to explore how providers balance the competing priorities of safety and health-related quality of life (HRQoL). The electronic survey was institutional review board-approved and disseminated via email to all PHOT providers affiliated with the Children's Oncology Group across the United States., Results: In total, 545 clinicians completed the survey. Most respondents were attending physicians (393, 72%), followed by fellows (61, 11%), advanced practice providers (APPs; 38, 7%), and nurses (37, 7%). On average, nurses and fellows made more conservative recommendations for avoiding infectious exposures compared with the recommendations from attending physicians and APPs ( P < .0001). On average, providers with more years of clinical experience expressed less cautious recommendations, whereas those with less years of experience provided more cautious recommendations for avoiding infectious exposures ( P = .0072)., Conclusion: This survey demonstrates the importance of collaboration between all members of the care team in defining priorities for balancing safety risk and HRQoL to provide consistent messaging to patients. The variations in survey responses highlight the need for universal guidelines to standardize physician recommendations for limiting infectious exposures in pediatric patients on chemotherapy.

Pediatric hematology-oncology (PHO) is 1 of the oldest recognized pediatric subspecialities. PHO physicians care for infants, children, adolescents, and young adults with all types of cancer and nonmalignant blood conditions, in many cases temporarily assuming the role of a primary care physician because of the complexity and intensity of treatment. However, the number of clinically active PHO subspecialists needed to care for children in the United States remains unknown. Recent papers suggest a potential oversaturation of PHO physicians in some geographic areas. This article is part of a Pediatrics supplement focused on projecting the future supply of the pediatric subspecialty workforce. It draws on information available in the literature, data from the American Board of Pediatrics, and findings from a new microsimulation model estimating the future supply of pediatric subspecialists through 2040. The model predicts a workforce growth in PHO subspecialists of 66% by 2040. Alternative scenarios, including changes in clinical time and fellowship size, resulted in a difference in growth of ±18% from baseline. The model also forecasts significant geographic maldistribution. For example, the current workforce is concentrated in the Northeast Census region and the model predicts the New England Census division will have a 2.9-fold higher clinical workforce equivalent per 100 000 children aged 0 to 18 years than the Mountain Census division by 2040. These findings suggest potential opportunities to improve the PHO subspecialty workforce and the outcomes and experiences of its patient population through educational changes, practice initiatives, policy interventions, and dedicated research., (Copyright © 2024 by the American Academy of Pediatrics.)

Purpose: Adolescents and young adults (AYAs, 15-39 years) with cancer experience disparities in care and outcomes compared with older/younger patients. AYAs receive care from medical and pediatric oncologists, however, little is known about the extent of training fellows receive. This needs assessment evaluating current AYA oncology (AYA-O) education in pediatric and medical oncology fellowship programs to identify knowledge gaps for curricular development. Methods: An anonymous, cross-sectional, web-based survey developed by pediatric and medical oncologists was sent to medical ( n  = 178) and pediatric ( n  = 119) hematology/oncology program directors (PDs) at 251 sites in the United States. PDs were asked to participate and distribute the survey to their fellows. Survey questions addressed current AYA curriculum, provider comfort, and priorities for future AYA educational content. Results: Participants from 69/251 programs responded (program response rate = 27%), including 51 PDs (32 pediatric, 19 medical oncology) and 58 fellows (33 pediatric, 25 medical oncology). Eighty-five percent of PDs (44/51) reported lacking formal AYA curricula. Of these, 80% (35/44) offer some topic-specific lectures, while 20% (9/44) provide little/no education for any topics. For nearly all topics, at least 45% of combined respondents reported little/no education. Respondents believe AYA topics are important for inclusion in future curricula. The most important topics for inclusion reported were oncofertility (82%), survivorship (78%), and communication (77%). Conclusions: There are large and actionable gaps in AYA-O education during fellowship training. Efforts are underway to develop AYA-O curriculum to provide both medical and pediatric oncology fellows with the knowledge and skills required to provide optimal AYA care.

Abstract: Commitment to clinical education often requires significant forethought and attention to provide a comprehensive learning experience for trainees. In these settings, teaching is typically time-limited, prompted by a clinical scenario, and requires preparation. However, it is not uncommon for teachers to have insufficient time to prepare or to encounter a clinical scenario in which they have not yet developed a teaching script.In this article, the authors share 5 categories of teaching techniques that instructors can pull from regardless of the prompt or busyness of the clinical setting and that are ideal for using when the teaching script is "blank." They call this approach of having scenario-independent teaching techniques ready to be applied with minimal preparation, "pseudo-improvised teaching."Drawing from the literature, their own experience, and borrowing from improvisational theater, the authors share a toolkit of pseudo-improvised teaching techniques spanning from pathophysiology to clinical skills to work-life integration. In addition to highlighting several techniques, they describe models of meta-structure for teaching in which the use of themes for the day (i.e., longitudinal themes) and routines can ease some of the cognitive load felt by both learners and educators., (Copyright © 2023 the Association of American Medical Colleges.)

The pediatric hematology and oncology (PHO) workforce landscape has been evolving over the past decades, with concern for waning interest in the subspecialty. We aimed to evaluate the impact of the initial PHO job search on fellow stress and anxiety, in addition to perceptions of the job search experience and potential areas of improvement. An anonymous survey consisting of demographics, emotional health impacts such as stress and anxiety, and job search experiences was developed and distributed to program leadership at all 74 PHO fellowship programs. At least one representative from 49 (66%) programs responded. Faculty were less likely than fellows to perceive that fellows are struggling to find jobs (p = .0198). However, faculty were more likely than fellows to perceive that fellows are either "extremely stressed" or "stressed" due to job search (p = .0003). By June 2021, 30 of 44 (68%) candidates had been offered a position, and 80% of those had accepted. There were no significant associations between fellow stress level and job offering, proximity to their ideal goals, or change in career type. Common barriers to the job search included geographic constraints and partner employment. Respondents identified centralized job listings, formal training on career development strategies, introduction to various career paths, and more transparency about the search as needed interventions. The perception of difficulty and stress regarding the job search is endorsed by most, identifying a need for more interventions to improve the experience. These data also highlight unmet mentorship and educational needs among PHO fellows in preparing for the job search., (© 2023 Wiley Periodicals LLC.)

The American Society of Pediatric Hematology/Oncology (ASPHO) conducted a workshop "Can you hear me now? Cultivating a culture of respect, value, and appreciation within pediatric hematology/oncology" at their annual meeting in May 2022 in hopes of exploring how the members can enhance wellness in a climate of increasing diversity. Initiatives in the past have focused on personal care, but it has been widely shown that administrative and institutional driven initiatives are essential to create an environment of wellness. In this interactive workshop, we discovered that 22% of participants felt their institution does not instill a culture of respect. We offered tools to the audience on multiple levels: graceful self-promotion, diversity and inclusion, and leadership perspective on creating a culture of respect to address the individual, local community, and top-down leadership approaches. Here, we offer a summary on the content of the workshop, and expand upon many of the discussion points that were raised during the workshop. We bring forth novel information on each topic individually from diverse points of view, specific to the field of pediatric hematology/oncology (PHO). We aim to highlight the importance of creating a diverse and respectful work environment in PHO in hopes of ensuring motivated, satisfied, and fulfilled healthcare providers who feel appreciated and valued., (© 2022 Wiley Periodicals LLC.)

Background: A recent survey of pediatric hematology oncology (PHO) physicians identified that a majority believe fellows are struggling to find jobs that align with their goals. Career development for trainees has historically been home institution-specific, limiting fellows' exposures to career path possibilities. The "virtual-Symposium of Pediatric Hematology/Oncology of New York (v-SYMPHONY)" instituted a tristate Career Development Series for PHO trainees to better address their needs and increase awareness of the variety of PHO career opportunities., Procedure: The v-SYMPHONY Career Development Series incorporated three sessions: (a) institutional perspective, (b) individual perspectives, and (c) nuts and bolts of job search. Pre- and post-series surveys were administered to participants to measure impact., Results: Forty-one fellows registered for the series and completed a pre-survey. Over half (54%) were in their third or later year of fellowship. Careers with a clinical focus were the most commonly desired career path (59%). Most had received career development advice only from faculty within their institutions (90%). Post-surveys were completed by 11 PHO fellows. Overall, 100% of respondents reported benefiting from the career sessions and recommended the series should be repeated annually. Over 90% learned new information to prepare for the job search., Conclusions: The v-SYMPHONY Career Development Series for PHO fellows across multiple institutions was established and was extremely well received by its participants. PHO fellows agreed that these sessions were beneficial in helping prepare them for the job search process. An annual regional Career Development Series is feasible and is strongly suggested to support PHO fellows., (© 2022 Wiley Periodicals LLC.)

We present a case of chemotherapy refractory spindle cell rhabdomyosarcoma of the lower urinary tract in a 15-month-old female that ultimately required consolidative surgery with cystectomy, urethrectomy, ovarian-sparing hysterectomy, bilateral salpingectomy, anterior vaginal wall resection, and bilateral pelvic lymph node dissection. Genitourinary reconstruction was performed by ileal conduit creation and vaginoplasty. After completion of her maintenance postoperative chemotherapy regimen, the patient has remained disease-free for approximately 27 months., (Copyright © 2022 Elsevier Inc. All rights reserved.)

The use of social media continues to increase in health care and academia. Health care practice, particularly the oncologic field, is constantly changing because of new knowledge, evidence-based research, clinical trials, and government policies. Therefore, oncology trainees and professionals continue to strive to stay up-to-date with practice guidelines, research, and skills. Although social media as an educational and professional development tool is no longer completely new to medicine and has been embraced, it is still under-researched in terms of various outcomes. Social media plays several key roles in professional development and academic advancement. We reviewed the literature to evaluate how social media can be used for professional development and academic promotion of oncology professionals.

Background: The impact of the coronavirus 2019 (COVID-19) pandemic on the emotional health of health care workers continues to be an area of active research. However, few studies have focused on those working in pediatrics and its subspecialties, as well as ancillary and non-patient-facing staff. The purpose of this study was to determine the prevalence and associated predictors of burnout and emotional well-being of providers and staff., Methods: An anonymous electronic survey was developed evaluating demographics, pandemic experiences, possible predictor variables, and three main outcomes of burnout, psychological distress, and perceived stress. Pediatric hematology oncology (PHO) chiefs and program directors across the country were invited to participate and disseminate the survey to their programs., Results: A total of 682/1950 (35% of invited) individuals responded to all predictor and outcome variables. Over half reported high levels of burnout and some reported moderate/high levels of distress. Prepandemic burnout and decreased trust in leadership were associated with all three outcomes. Additional predictors included having a child ≤18 years at home, hospital role, and worrying about patient care or relationship with their patients. The majority (n = 444/682, 65.5%) reported that their institution had made COVID-19-related mental health resources available. However, only 6.5% (n = 44/682) reported utilizing these resources., Conclusions: While the majority of PHO providers and staff were resilient during the early stages of the COVID-19 pandemic, many reported high levels of burnout, yet few are utilizing institutional resources. This study has highlighted several actionable areas to help identify and address factors that are wearing down the emotional well-being of providers and staff., (© 2021 Wiley Periodicals LLC.)

Purpose: Medical student mistreatment is pervasive, yet whether all physicians have a shared understanding of the problem is unclear. The authors presented professionally designed trigger videos to physicians from 6 different specialties to determine if they perceive mistreatment and its severity similarly., Method: From October 2016 to August 2018, resident and attending physicians from 10 U.S. medical schools viewed 5 trigger videos showing behaviors that could be perceived as mistreatment. They completed a survey exploring their perceptions. The authors compared perceptions of mistreatment across specialties and, for each scenario, evaluated the relationship between specialty and perception of mistreatment., Results: Six-hundred fifty resident and attending physicians participated. There were statistically significant differences in perception of mistreatment across specialties for 3 of the 5 scenarios: aggressive questioning (range, 74.1%-91.2%), negative feedback (range, 25.4%-63.7%), and assignment of inappropriate tasks (range, 5.5%-25.5%) (P ≤ .001, for all). After adjusting for gender, race, professional role, and prior mistreatment, physicians in surgery viewed 3 scenarios (aggressive questioning, negative feedback, and inappropriate tasks) as less likely to represent mistreatment compared with internal medicine physicians. Physicians from obstetrics-gynecology and "other" specialties perceived less mistreatment in 2 scenarios (aggressive questioning and negative feedback), while family physicians perceived more mistreatment in 1 scenario (negative feedback) compared with internal medicine physicians. The mean severity of perceived mistreatment on a 1 to 7 scale (7 most serious) also varied statistically significantly across the specialties for 3 scenarios: aggressive questioning (range, 4.4-5.4; P < .001), ethnic insensitivity (range, 5.1-6.1; P = .001), and sexual harassment (range, 5.5-6.3; P = .004)., Conclusions: Specialty was associated with differences in the perception of mistreatment and rating of its severity. Further investigation is needed to understand why these perceptions of mistreatment vary among specialties and how to address these differences., (Copyright © 2021 by the Association of American Medical Colleges.)

Objective: Frontline health-care workers and patients with COVID-19 have been identified as high-risk groups for psychological problems. Experience of working or staying in quarantine wards generated psychological stressors for health-care workers and patients with COVID-19. The present study aimed to investigate the psychological symptoms of hospitalized patients with COVID-19 and the health-care workers treating them during the outbreak period, examine the effects of psychological stressors on mental health in both populations and perceived coping resources for both sides. Methods: Three hundred and eleven health-care workers working in a COVID-19 designated hospital in Wuhan, China, and 148 hospitalized patients with COVID-19 in the same hospital participated in this cross-sectional survey conducted in February 2020. Psychological symptoms, psychological stressors, and perceived coping resources were reported by both groups. Results: Thirty-three percent of health-care workers and 35.2% of patients with COVID-19 had significant psychological symptoms that were indicative of a high risk for psychological disorders. Pandemic-related psychological stressors contributed to psychological symptoms for both populations. Concern about patients was one aspect of psychological stressors of frontline health-care workers and both groups perceived support from the opposite side as an important external coping resource. Conclusion: The results shed light on the need to provide psychological support to both frontline health-care workers and patients with COVID-19 and suggest enhancing the treatment alliance might be effective to improve mental health for both populations during the crisis., Competing Interests: The authors declare that the research was conducted in the absence of any commercial or financial relationships that could be construed as a potential conflict of interest., (Copyright © 2021 Zhou, Guan, Rosenthal, Moerdler, Guan and Sun.)

COVID-19 has upended medical practice and education, but has also catalyzed enhancements in the field. Early on, a local group of researchers united to investigate the impact of the pandemic on pediatric hematology oncology (PHO). From this group, a regional educational series was established, "virtual-Symposium of Pediatric Hematology/Oncology of New York" (v-SYMPHONY). The implementation of these endeavors while PHO fellowship applications are declining has highlighted our perceptions that education, mentoring, and career expectations are not keeping up with the needs of current trainees. We describe our regional experience joining together to further education and research, and reflect on the current landscape of PHO training and workforce., (© 2021 Wiley Periodicals LLC.)

The B7 family ligand HERV-H LTR-associating protein 2 (HHLA2) is an attractive target for cancer immunotherapy because of its coinhibitory function, overexpression in human cancers, and association with poor prognoses. However, the knowledge of the HHLA2 pathway is incomplete. HHLA2 has an established positive receptor transmembrane and immunoglobulin (Ig) domain containing 2 (TMIGD2) but a poorly characterized negative receptor human killer cell Ig-like receptor, three Ig domains, and long cytoplasmic tail (KIR3DL3). Here, KIR3DL3 and TMIGD2 simultaneously bound to different sites of HHLA2. KIR3DL3 was mainly expressed on CD56 dim NK and terminally differentiated effector memory CD8 + T (CD8 + T EMRA ) cells. KIR3DL3 + CD8 + T EMRA acquired an NK-like phenotype and function. HHLA2 engagement recruited KIR3DL3 to the immunological synapse and coinhibited CD8 + T and NK cell function and killing, inducing immune-evasive HHLA2 + tumors. KIR3DL3 recruited SHP-1 and SHP-2 to attenuate Vav1, ERK1/2, AKT, and NF-κB signaling. HHLA2 + tumors from human kidney, lung, gallbladder, and stomach were infiltrated by KIR3DL3 + immune cells. KIR3DL3 blockade inhibited tumor growth in multiple humanized mouse models. Thus, our findings elucidated the molecular and cellular basis for the inhibitory function of KIR3DL3, demonstrating that the KIR3DL3-HHLA2 pathway is a potential immunotherapeutic target for cancer., (Copyright © 2021 The Authors, some rights reserved; exclusive licensee American Association for the Advancement of Science. No claim to original U.S. Government Works.)

Purpose: The COVID-19 pandemic has significantly affected the well-being of healthcare workers to varying degrees. The aim of the current study was to investigate how the pandemic has affected the burnout, stress, and emotional well-being of pediatric hematology oncology (PHO) providers and staff in the New York and New Jersey epicenter., Methods: The study was conducted in June 2020 during the pandemic through an electronic survey. The survey contained questions surrounding demographics, pandemic experiences, and validated burnout, stress, and emotional well-being measures., Results: Two hundred fifty-two PHO providers and staff responded to the survey. Overall, half of the participants reported high levels of burnout, average stress scores were in the mild-moderate range, and the majority scored in the none-to-mild symptomatology range for their well-being. Self-reported burnout levels before the pandemic and geographic work location were statistically significant risk factors for all outcomes. Additional predictors for some outcomes included hospital role, lack of trust in leadership, and deployment. The majority of participants (87.0%) reported that their hospitals had made mental health resources related to COVID-19 available to them but only 8.4% reported having used them., Conclusion: PHO providers and staff in the NYC/NJ area are experiencing a range of emotional experiences during the COVID-19 pandemic, but the majority are not using current resources. With the continuation of the pandemic, we must continue efforts to improve provider and staff distress to mitigate the degree of potential negative short-term and long-term impact., Competing Interests: Adam S. LevyConsulting or Advisory Role: AstraZenecaNo other potential conflicts of interest were reported.

LAG-3, through interaction with a variety of ligands, regulates T cell function via inhibition of T cell proliferation and activation. It has been demonstrated to be overexpressed on tumor infiltrating lymphocytes (TILs) of a variety of cancers with associated poor outcomes. The purpose of this study is to characterize the expression pattern and clinical significance of LAG-3 in pediatric Hodgkin lymphoma (HL). Patient tumor samples from Children's Oncology Group clinical trial AHOD0031 with matched patient outcome data were analyzed for the expression of LAG-3 and PD-L1 using immunohistochemistry. 73/115 patients (63%) demonstrated positive LAG-3 staining. No demographic or survival outcome data were significantly associated with LAG-3 expression. Interestingly, patients with the lowest density of expression were found to have the worst EFS, and those with highest density of expression demonstrated the best EFS. There was a positive statistically significant relationship between presence of LAG-3 and PD-L1 expression. This project is innovative in its characterization of LAG-3 as an immune checkpoint target in pediatric HL.

Burnout is a significant problem in the medical community, including among pediatric hematology/oncology (PHO) faculty. However, the prevalence of burnout, its associated risk factors, and outcomes within PHO fellows are unknown. We present the results of a cross-sectional study of PHO fellows from 21 training programs. A total of 45/115 fellows (39.1%) met criteria for high level of burnout. Fellows who met criteria for high burnout also demonstrated poor outcomes including decreased empowerment, increased doctor-centered care, decreased self-assessed humanism, and decreased satisfaction with training. Further longitudinal investigation is needed to better understand burnout and the causative factors affecting PHO fellows., (© 2020 Wiley Periodicals, Inc.)

Purpose: Although humanism and professionalism are central tenets to the practice of medicine, few formal curricula exist for medical trainees. Following a national needs assessment among pediatric hematology-oncology (PHO) fellows, we created a novel curriculum entitled "Humanism and Professionalism for Pediatric Hematology-Oncology" (HP-PHO). In this study, we measure outcomes of this curricular intervention., Method: We cluster-randomized 20 PHO fellowship programs to deliver usual training in humanism and professionalism (UT) or the novel curriculum (intervention) during the 2016-2017 academic year. The primary outcome measure was the Pediatric Hematology-Oncology Self-Assessment in Humanism (PHOSAH). Secondary measures included the Maslach Burnout Inventory, Patient-Provider Orientation Scale, Empowerment at Work Scale, and a 5-point satisfaction scale. Participating fellows completed pre- and posttests at the beginning and end of the academic year, respectively, and we calculated change scores for each study instrument., Results: Cluster randomization yielded 59 intervention and 41 UT fellows. The nine intervention sites administered 33 of 36 modules. Change scores on the PHOSAH were not significantly different between the UT and intervention arms. However, fellows on the intervention arm gave significantly higher ratings on several items within the satisfaction scale related to physician burnout, physician depression, balancing professional duties and personal life, and humanism overall., Conclusions: Exposure to the HP-PHO curriculum did not alter fellows' self-assessed humanism and professionalism skills. However, intervention fellows expressed significantly higher levels of satisfaction in their humanism training, indicating the curriculum's potential for positive impact on the fellows' perceived learning environment., (© 2020 Wiley Periodicals, Inc.)

Neuroblastoma, the most common extracranial solid tumor of childhood, can present in multiple primary sites, but the extent of genetic heterogeneity among tumor foci, as well as the presence or absence of common oncogenic drivers, remains unknown. Although PHOX2B genetic aberrations can cause familial neuroblastoma, they demonstrate incomplete penetrance with respect to neuroblastoma pathogenesis, suggesting that additional undescribed oncogenic drivers are necessary for tumor development. We performed comprehensive molecular characterization of neuroblastoma tumors from two siblings affected by familial multifocal neuroblastoma, including whole exome sequencing and single-nucleotide polymorphism (SNP) arrays of tumor and matched blood samples. Data were processed and analyzed using established bioinformatics algorithms to evaluate for germline and somatic mutations and copy number variations (CNVs). We confirmed the presence of a PHOX2B deletion and NF1 mutation across all tumor samples and the germline genome. Matched tumor-blood whole exome sequencing also identified 365 genes that contained nonsilent coding mutations across all tumor samples, with no recurrent mutations across all tumors. SNP arrays also showed significant heterogeneity with respect to CNVs. The only common CNV across all tumors was 17q gain, with differing chromosomal coordinates across samples but a common region of overlap distal to 17q21.31, suggesting this adverse prognostic biomarker may offer insight about additional drivers for multifocal neuroblastoma in patients with germline PHOX2B or NF1 aberrations. Molecular characterization of all tumors from patients with multifocal primary neuroblastoma has potential to yield novel insights on neuroblastoma pathogenesis., (© 2019 Wiley Periodicals, Inc.)

Background: Targeted cancer treatments are almost always first studied in adults, even when there is a biologically plausible potential for efficacy in children. Through compassionate use programs, children who are not eligible for a clinical trial and for whom there are no known effective therapies may obtain access to investigational agents, including drugs under development for adults. However, little is known about pediatric oncologists' experiences with applying for and obtaining compassionate use agents., Methods: This study surveyed 132 pediatric oncologists to assess awareness and utilization of compassionate use programs, to identify barriers to their use, and to evaluate available institutional support and resources., Results: We found that the process of applying for access to drugs in development is poorly understood, which presents a barrier to obtaining investigational drugs. Fifty-seven percent of the pediatric oncologists applied for compassionate use. Providers from larger institutions or with more than 15 years of clinical experience were more likely to complete an application and obtain investigational agents for their patients., Conclusion: Identified perceived and actual barriers to compassionate use application submission suggest pediatric oncologists may benefit from educational resources and support to ensure children with cancer equal access to investigational agents and care., (© 2018 Wiley Periodicals, Inc.)

Introduction: Medical students have limited preclinical exposure to pediatrics. We created an optional preclinical curriculum for first-year medical students called "Preseason Pediatrics" (PSP). This 6-month curriculum teaches pediatric-specific knowledge and clinical skills, consisting of monthly resident-led didactic sessions followed by complementary resident-mentored clinical experiences., Methods: Participants completed a survey before and after completion of PSP. Knowledge was assessed with multiple-choice questions pertaining to each topic covered in PSP and perceived skills, with a 5-point Likert scale ranging from not at all (1) to extremely (5) for skills taught. Skill maintenance was assessed with a newborn objective structured clinical exam (OSCE) 6 months after PSP completion in 2016. Students beginning their pediatric clerkship also completed a survey, comparing students who did and did not complete PSP., Results: From 2014 to 2017, 135 first-year medical students participated. Percent correct scores on pediatric knowledge increased in 4/5 topics covered, and students perceived increases in their pediatric skills in all course domains. 86.8% ( n  = 92/106) of students reported feeling more prepared for the pediatric clerkship. 94.3% ( n  = 100/106) would recommend the PSP experience to other students. Third-year students who participated in PSP reported higher comfort with pediatric patients prior to their clerkship., Conclusions: PSP is a successful novel preclinical program introducing students to pediatrics. We demonstrated that didactics paired with resident-mentored clinical experiences improved pediatric knowledge, clinical-based skills, and perceived clerkship preparedness. Students may academically and professionally benefit from such earlier exposure to pediatrics., Competing Interests: Conflict of InterestThe authors declare that they have no conflict of interest., (© International Association of Medical Science Educators 2019.)

Although the seminal event in sickle cell disease is the polymerization of abnormal hemoglobin, the downstream pathophysiology of vasoocclusion results from heterotypic interactions between the altered, adhesive sickle cell red blood cells, neutrophils, endothelium, and platelets. Ischemia reperfusion injury, hemolysis, and oxidant damage all contribute to heightened inflammation and activation of the hemostatic system. These various pathways are the focus of emerging treatments with potential to ameliorate disease manifestations. This review summarizes the considerable progress in development of these agents despite challenges in selection of study end points and complex pathophysiology., Competing Interests: Conflict-of-interest disclosure: The authors declare no competing financial interests., (© 2018 by The American Society of Hematology. All rights reserved.)