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PURPOSEThis study aimed to describe and assess the regional experience of a pediatric hematology/oncology fellowship program based in Guatemala.METHODSThe Unidad Nacional de Oncología Pediátrica (UNOP) in Guatemala City, Guatemala, is the only hospital in Central America dedicated exclusively to childhood and adolescent cancer. To address the regional need for specialists, a fellowship program in pediatric hematology/oncology was launched in 2003. The UNOP fellowship program comprises 3 years of training. Although the program is based at UNOP, it also includes rotations locally and internationally to enhance clinical exposure. The curriculum is based on international standards to cover clinical expertise, research, professionalism, communication, and health advocacy. Trainees are selected according to country or facility-level need for pediatric hematologists/oncologists, with a plan for them to be hired immediately after completing their training.RESULTSForty physicians from 10 countries in Latin America have completed training. In addition, there are currently 13 fellows from five countries in training. Of the graduates, 39 (98%) are now practicing in pediatric hematology/oncology in Latin America. Moreover, many of them have leadership positions within their institutions and participate in research, advocacy, and policy making. Graduates from the UNOP program contribute to institutions by providing care for an increasing number of patients with pediatric cancer. The UNOP program is the first pediatric hematology/oncology fellowship program in the world to be accredited by Accreditation Council for Graduate Medical Education-International, an international body accrediting clinical training programs.CONCLUSIONThe UNOP program has trained specialists to increase the available care for children with cancer in Latin America. This regional approach to specialist training can maximize resources and serve as a model for other programs and regions.

PURPOSEThere is an urgent need to improve access to cancer therapy globally. Several independent initiatives have been undertaken to improve access to cancer medicines, and additional new initiatives are in development. Improved sharing of experiences and increased collaboration are needed to achieve substantial improvements in global access to essential oncology medicines.METHODSThe inaugural Access to Essential Cancer Medicines Stakeholder Meeting was organized by ASCO and convened at the June 2022 ASCO Annual Meeting in Chicago, IL, with two subsequent meetings, Union for International Cancer Control World Cancer Congress held in Geneva, Switzerland, in October 2022 and at the ASCO Annual Meeting in June of 2023. Invited stakeholders included representatives from cancer institutes, physicians, researchers, professional societies, the pharmaceutical industry, patient advocacy organizations, funders, cancer organizations and foundations, policy makers, and regulatory bodies. The session was moderated by ASCO. Past efforts and current and upcoming initiatives were initially discussed (2022), updates on progress were provided (2023), and broad agreement on resulting action steps was achieved with participants.RESULTSSummit participants recognized that while much work was ongoing to enhance access to cancer therapeutics globally, communication and synergy across projects and organizations could be enhanced by providing a platform for collaboration and shared expertise.CONCLUSIONThe summit resulted in new cross-stakeholder insights and planned collaboration addressing barriers to accessing cancer medications. Specific actions and timelines for implementation and reporting were established.

Summary: Background: Paediatric hepatocellular carcinomas (HCC) traditionally arise in the context of a normal structural and functional liver and carry a dismal prognosis. While chemotherapy is the frontline standard, there is emerging interest in the study of immunotherapies for paediatric patients with relapsed/refractory disease. There is limited data to support whether immunotherapies will be of utility in this patient population. Methods: Six paediatric patients (median age:16 years, range: 12–17 at the time of treatment) with advanced hepatocellular neosplams, either conventional hepatocellular or fibrolamellar carcinoma, were treated with immunotherapy. Patients were consented to institutional genomic profiling and biobanking protocols. Baseline samples and serial tissue samples, when available, were evaluated for somatic mutation rate, actionable gene mutations, and pan-immune bulk RNA expression profiling. Results were correlated with clinical course. Findings: Three patients responded to checkpoint inhibition: one achieved a complete, durable response and the other two, prolonged stable disease. Three additional patients progressed. Diagnostic tissue from the complete responder demonstrated a higher relative mutational burden and robust immune infiltrate. Pre-treatment samples from the three responders demonstrated decreased expression of genes associated with T-cell dysfunction. Interpretation: A subset of patients with primary paediatric hepatocellular tumours will respond to immunotherapy. Immunotherapies are currently under prospective study for relapsed/refractory liver tumours in paediatric patients. Results from this report support the prospective collection of serial serum and tissue samples which may further identify genomic and immunophenotypic patterns predictive of response. Funding: This work was supported by Philanthropic funds (Pan Mass Challenge, Team Angus and Team Perspective).

Abstract Background Histiocytoses are rare disorders manifested by increased proliferation of pathogenic myeloid cells sharing histological features with macrophages or dendritic cells and accumulating in various organs, i.a., bone and skin. Pre-clinical in vitro models that could be used to determine molecular pathways of the disease are limited, hence research on histiocytoses is challenging. The current study compares cytophysiological features of progenitor, stromal-like cells derived from histiocytic lesions (sl-pHCs) of three pediatric patients with different histiocytoses types and outcomes. The characterized cells may find potential applications in drug testing. Methods Molecular phenotype of the cells, i.e. expression of CD1a and CD207 (langerin), was determined using flow cytometry. Cytogenetic analysis included GTG-banded metaphases and microarray (aCGH) evaluation. Furthermore, the morphology and ultrastructure of cells were evaluated using a confocal and scanning electron microscope. The microphotographs from the confocal imaging were used to reconstruct the mitochondrial network and its morphology. Basic cytophysiological parameters, such as viability, mitochondrial activity, and proliferation, were analyzed using multiple cellular assays, including Annexin V/7-AAD staining, mitopotential analysis, BrdU test, clonogenicity analysis, and distribution of cells within the cell cycle. Biomarkers potentially associated with histiocytoses progression were determined using RT-qPCR at mRNA, miRNA and lncRNA levels. Intracellular accumulation of histiocytosis-specific proteins was detected with Western blot. Cytotoxicyty and IC50 of vemurafenib and trametinib were determined with MTS assay. Results Obtained cellular models, i.e. RAB-1, HAN-1, and CHR-1, are heterogenic in terms of molecular phenotype and morphology. The cells express CD1a/CD207 markers characteristic for dendritic cells, but also show intracellular accumulation of markers characteristic for cells of mesenchymal origin, i.e. vimentin (VIM) and osteopontin (OPN). In subsequent cultures, cells remain viable and metabolically active, and the mitochondrial network is well developed, with some distinctive morphotypes noted in each cell line. Cell-specific transcriptome profile was noted, providing information on potential new biomarkers (non-coding RNAs) with diagnostic and prognostic features. The cells showed different sensitivity to vemurafenib and trametinib. Conclusion Obtained and characterized cellular models of stromal-like cells derived from histiocytic lesions can be used for studies on histiocytosis biology and drug testing.

Abstract Background Pediatric interventional oncology (PIO) is a growing field intended to provide additional or alternative treatment options for pediatric patients with benign or malignant tumors. Large series of patients treated uniformly and subjected to rigorous endpoints for efficacy are not available. Methods We designed a collaborative initiative to capture data from pediatric patients with benign and malignant tumors who underwent a therapeutic interventional radiology procedure. Modified Response Evaluation Criteria in Solid Tumors (mRECIST) was utilized as a measure of radiologic response and data were collected regarding improvement in pain and functional endpoints. Cumulative incidence of progressive disease was calculated using both the treated site and the patient as the analytic unit. Findings Forty patients, 16 with malignant tumors and 24 with benign tumors, underwent a total of 88 procedures. Cryo‐ and radiofrequency ablation were the most frequently utilized techniques for both cohorts of patients. A complete or partial response, or prolonged disease stability, were achieved in approximately 40% of patients with malignant tumors and 60% of patients with benign tumors. No patients had progressive disease as their best response. Resolution of pain and improved mobility with return‐to‐baseline activity were demonstrated across patients from both cohorts. Only minor complications were experienced. Interpretation Interventional radiology‐guided interventions can serve as an alternative or complementary approach to the treatment of benign and malignant tumors in pediatric patients. Prospective, multi‐institutional trials are required to adequately study utility, treatment endpoints, and durability of response.

Summary: The germline TP53 p.R337H mutation is reported as the most common germline TP53 variant. It exists at a remarkably high frequency in the population of southeast Brazil as founder mutation in two distinct haplotypes with the most frequent co-segregating with the p.E134∗ variant of the XAF1 tumor suppressor and an increased cancer risk. Founder mutations demonstrate linkage disequilibrium with neighboring genetic polymorphic markers that can be used to identify the founder variant in different geographic regions and diverse populations. We report here a shared haplotype among Brazilian, Portuguese, and Spanish families and the existence of three additional distinct TP53 p.R337H alleles. Mitochondrial DNA sequencing and Y-STR profiling of Brazilian carriers of the founder TP53 p.R337H allele reveal an excess of Native American haplogroups in maternal lineages and exclusively European haplogroups in paternal lineages, consistent with communities established through male European settlers with extensive intermarriage with Indigenous women. The identification of founder and independent TP53 p.R337H alleles underlines the importance for considering the haplotype as a functional unit and the additive effects of constitutive polymorphisms and associated variants in modifier genes that can influence the cancer phenotype.

While all childhood cancers are rare, tumors that are particularly infrequent or underrepresented within pediatrics are studied under the umbrella of the Children’s Oncology Group Rare Tumor committee, divided into the Retinoblastoma and Infrequent Tumor subcommittees. The Infrequent Tumor subcommittee has traditionally included an emphasis on globally rare tumors such as adrenocortical carcinoma, nasopharyngeal carcinoma, or those tumors that are rare in young children, despite being common in adolescents and young adults, such as colorectal carcinoma, thyroid carcinoma, and melanoma. Pleuropulmonary blastoma, gonadal stromal tumors, pancreatic tumors including pancreatoblastoma, gastrointestinal stromal tumor, nonmelanoma skin cancers, neuroendocrine tumors, and desmoplastic small round cell tumors, as well as other carcinomas are also included under the heading of the Children’s Oncology Group Rare Tumor committee. While substantial challenges exist in rare cancers, inclusion and global collaboration remain key priorities to ensure high quality research to advance care.

Abstract Background In high‐income countries, hope facilitates parental coping and builds the clinical relationship between families of children with cancer and their clinicians. However, the manifestation of hope in low‐ and middle‐income countries (LMICs) remains poorly understood. Our study explores Guatemalan parents' experiences with hope during the pediatric oncology diagnostic process and aims to identify discrete actions clinicians take to support hope. Methods This qualitative study utilized audio‐recordings of the diagnostic process and an additional semi‐structured interview for 20 families of children with cancer at Unidad Nacional de Oncología Pediátrica in Guatemala. Spanish audio‐recordings were translated into English, transcribed, and coded using a priori and novel codes. Thematic content analysis using constant comparative methods explored parents' hopes and concerns. Results At diagnosis, Guatemalan parents expressed both hopes and concerns related to the entire cancer continuum. Throughout the diagnostic process, hope grew as concerns were alleviated. Clinicians supported hope by creating a supportive environment, providing information, affirming religious beliefs, and empowering parents. These strategies helped parents shift their focus from fear and uncertainty toward hope for their child's future. Parents expressed that establishing hope improved mood, promoted acceptance, and enabled them to care for themselves and their children. Conclusion These results confirm the relevance of supporting hope in pediatric oncology settings in LMICs and suggest that culture informs hope‐related needs. Supporting hope is critical across cultures and can be integrated into clinical conversation using the four processes identified by our results.

Abstract Background Communication between providers and patients' families is an integral part of clinical care. Family concern is a validated component of Pediatric Early Warning Systems (PEWS); however, little is known about the impact of PEWS on provider‐family communication. Methods Semi‐structured interviews were conducted with 83 ward and Pediatric Intensive Care Unit (PICU) providers involved in the care of patients with deterioration at two pediatric oncology hospitals of different resource levels: St. Jude Children's Research Hospital (n = 42) in the United States and Unidad Nacional de Oncología Pediátrica (UNOP, n = 41) in Guatemala. Interviews were conducted in the participants' native language (English or Spanish), transcribed, and translated into English. Transcripts were coded by two researchers and analyzed for thematic content surrounding family communication and concern. Results All participants recognized patients' families as a valuable part of the care team, particularly during events requiring escalation of care. Perceived barriers to communication included limited time spent at the bedside, and, at UNOP, language and literacy challenges which occasionally limited providers' ability to assess family concern and involve families in patient care. Despite these barriers, providers perceived PEWS improved communication by facilitating more interaction with families, allowing for relationship‐building, anticipatory guidance, and destigmatization of the PICU. PEWS assessments also allowed families to contribute to identification of deterioration. Conclusions PEWS improve the quality of communication between providers and families by providing more opportunities for interaction, building relationships, and trust. These findings further support the use of PEWS in the care of children with cancer in hospitals of all resource‐levels.

Abstract Background The recent implementation of novel therapies has accelerated progress in pediatric cancer care. Despite the significantly poorer survival of patients in low- and middle-income countries (LMICs), administation complexities and other significant resource barriers have limited the translation of these novel therapies in these regions. This study aims to develop a model that can be used to support the implementation of novel therapies, such as blinatumomab (bispecific antibody therapy for B-cell acute lymphoblastic leukemia [B-ALL]) in LMIC centers, with the long-term goal of developing an implementation framework for similar future efforts. Methods In this study, mixed methods will be applied to understand the key contextual considerations that can be accounted for through a training program and prospectively designed implementation activities. The Consolidated Framework for Implementation Research will guide the activities related to implementation evaluation in parallel with a drug donation program. A multidisciplinary research team comprising high- and low-middle income healthcare professionals, industry, and implementation scientists has been assembled with the common goal of improving safe access to blinatumomab. To assess the factors affecting blinatumomab administration, semi-structured interviews with diverse collaborators and quantitative assessments of organizational characteristics will be conducted, together with quantitative and qualitative assessments of feasibility, acceptability, appropriateness, and cost of blinatumomab implementation. A quantitative assessment of stakeholder perceptions of different implementation strategies used as part of the multifaceted approach will also be performed. Finally, we will examine the key domains and processes used and construct the implementation roadmap for translation of novel therapies. Discussion This study will rigorously develop an implementation roadmap for translation of novel therapies in low-resource settings. The knowledge gained in the formative assessment will reveal the priority areas and key implementation strategies. Thereby, the resultant roadmap will facilitate future scale-out strategies for novel therapies in LMICs, thus increasing access, building capacity for management, and ultimately improving the care for children in LMICs.

IntroductionJordan hosts one of the highest numbers of refugees per capita in the world, with the Syrian crisis leading to an influx of displaced persons to the already vulnerable population. However, limited resources and a lack of cancer-care strategies have made it difficult for refugees in Jordan to access quality cancer care. The King Hussein Cancer Center (KHCC) and Foundation (KHCF) have played a pivotal role in providing financial and medical support for displaced children with cancer, treating 968 non-Jordanian children with cancer between 2011-2022, with a median age of 6 years. Of these, 84% were fully funded by KHCF, and nationalities included Syrians (29%), Palestinians (26%), Iraqis (23%), and Yemenis (17%). Cancer diagnoses included solid tumors (44%), leukemia (23%), lymphoma (13%), bone sarcomas (9.5%), and retinoblastoma (9.1%). The median cost of treatment was JOD 18,000 (USD 25,352), with a total estimated cost of JOD 23.8 million (USD 33.5 million). More recently, in partnership with St. Jude Children’s Research Hospital (SJCRH), two successive humanitarian funds (HF) were established to optimize cancer care for displaced children in Jordan.ResultsBetween February 2018 and September 2022, 51 children were fully treated on KHCC-SJCRH-HF, with a median age of 6 years and nationalities including Syrians (80%), Iraqis (6%), and Yemenis (8%). The most common cancer diagnoses were leukemia (41%), lymphoma (25%), solid tumors (24%), retinoblastoma (6%), and brain tumors (4%). Of these, 94% are alive and 51% are still receiving coverage. The median coverage for patients was JOD 21,808 (USD 30,715), and the total cost of treatment on KHCC/KHCF-SJCRH/American Lebanese Syrian-Associated Charities HF1 and HF2 was JOD 1.44 million (USD 1.97 million) and JOD 1.18 million (USD 1.67 million), respectively.ConclusionThis experience highlights the high burden of displaced children with cancer in Jordan, and the importance of local foundations like KHCC/KHCF and partnerships with international partners like SJCRH in providing lifesaving humanitarian initiatives and quality cancer care. Innovative cancer-care delivery models and sustainable financing are essential to ensure continuous coverage and access to cancer care for displaced persons in Jordan.

Background: In recent years, modifications of treatment protocols introduced in pediatric oncology have resulted in a significant improvement in treatment outcomes. Unfortunately, the probability of subsequent malignant neoplasm (SMN) in this group of patients is 3 to 6 times higher than the general age-matched population. In this study, we sought to evaluate the treatment options for patients with secondary bone tumors after prior anti-cancer therapy. Materials and Methods: Twenty-four patients (median age 12.9 years) with subsequent malignant bone tumors were treated according to oncological guidelines for bone sarcoma during the period 1991–2020. All patients had a standard tumor imaging and laboratory evaluation. All toxicities were documented. Results: The median time from the first neoplasm to SMN was 7.6 years (range 2.4 to 16.3 years). All patients received chemotherapy and underwent surgery as a local control procedure. Two patients with Ewing sarcoma had additional radiation on the tumor bed. A complete response was achieved in 20 patients. With a median follow-up of 18.3 years (range 5.7 to 40.3 years), 18 patients (75%) are alive. The estimated 5-year post-subsequent bone malignant neoplasm survival was 74.5% (95% CI 55–95%). Fourteen patients required chemotherapy dose modification, and doxorubicin was discontinued in seven patients. One patient required a renal transplant two years after treatment. There were no other significant toxicities. Conclusions: The treatment of bone SMNs can be effective, although in many patients it is necessary to reduce the doses of drugs. Early detection and aggressive treatment can improve the outcome.

PURPOSEAlthough > 90% of children with cancer live in low- and middle-income countries, little is known about communication priorities and experiences of families in these settings. We examined communication priorities and the quality of information exchange for Guatemalan caregivers of children with cancer during diagnostic communication.METHODSA cross-sectional survey including items used in pediatric communication studies from high-income countries and novel questions was verbally administered to 100 caregivers of children with cancer in Guatemala.RESULTSGuatemalan caregivers prioritized communication functions of exchanging information (99%), fostering healing relationships (98%), decision making (97%), enabling self-management (96%), and managing uncertainty (94%) over responding to emotions (66%) and cultural awareness (48%). Almost all caregivers wanted as many details as possible about their child's diagnosis and treatment (96%), likelihood of cure (99%), and late effects (97%). Only 67% were always given the information they needed without asking for it, and most caregivers sometimes (56%) or always (18%) had questions they wanted to discuss but did not. Approximately half of the caregivers (54%) correctly identified their child's diagnosis, primary site, disease extent (localized v metastatic), proposed treatment length, and treatment intent (curative v palliative). Caregivers of children with leukemia were more likely to correctly identify all attributes than those whose children had solid tumors (P < .001).CONCLUSIONCaregivers in Guatemala prioritize many of the same aspects of diagnostic communication as parents in the United States, and experience similar challenges. Shared communication values offer potential for adaptation of communication interventions across settings with varying resources and diverse cultures.

PURPOSEPediatric CNS tumors are increasingly a priority, particularly with the WHO designation of low-grade glioma (LGG) as one of six index childhood cancers. There are currently limited data on outcomes of pediatric patients with LGGs in low- and middle-income countries (LMICs).METHODSTo better understand the outcomes of LGGs in LMICs, this systematic review interrogated nine literature databases.RESULTSThe search identified 14,977 publications. Sixteen studies from 19 countries met the selection criteria and were included for data abstraction and analysis. Eleven studies (69%) were retrospective reviews from single institutions, and one (6%) captured institutional data prospectively. The studies captured a total of 957 patients with a median of 49 patients per study. Seven (44%) of the studies described the treatment modalities used. Of 373 patients for whom there was information, 173 (46%) had a gross total or near total resection, 109 (29%) had a subtotal resection, and 91 (24%) had only a biopsy performed. Seven studies, with a total of 476 patients, described the frequency of use of radiotherapy and/or chemotherapy in the cohorts: 83 of these patients received radiotherapy and 76 received chemotherapy. The 5-year overall survival ranged from 69.2% to 93.5%, although lower survival rates were reported at earlier time points. We identified limitations in the published studies with respect to the cohort sizes and methodologies.CONCLUSIONThe included studies reported survival rates frequently exceeding 80%, although the ultimate number of studies was limited, pointing to the paucity of studies describing the outcomes of children with LGGs in LMICs. This study underscores the need for more robust data on outcomes in pediatric LGG.

PURPOSESurveys to assess patient and family experiences of pediatric cancer care have been primarily developed and validated in high-income Western settings with English-speaking participants. However, 90% of children with cancer live in low- and middle-income countries. We sought to develop a survey focused on pediatric cancer communication for use in a low-literacy population in Guatemala, including adaptation of many previously validated items.METHODSA multidisciplinary team developed a quantitative survey on the basis of a theoretical model of important components and influences on pediatric cancer communication. The original survey included established items previously used in high-income settings and novel questions designed for this study. The survey was translated into Spanish and pilot tested with parents of children receiving treatment at Unidad Nacional de Oncologia Pediatrica in Guatemala City, Guatemala, from April-June 2019. Cognitive interviews were used during pilot testing, and the survey was iteratively revised throughout this process.RESULTSEarly in testing, Guatemalan parents tended to choose answers at the extreme ends of response categories and socially desirable responses. Ultimately, a visual aid was developed to accompany three-item Likert scale response options. This allowed for successful administration of the survey instrument, resulting in moderate variation of response options and similar proportions to those generated when the original five-item responses were used in parent populations from the United States.CONCLUSIONAppropriately adapted surveys are necessary to understand patient-centered communication among pediatric oncology populations in low- and middle-income countries. Eventual validation of such tools will enable cross-cultural studies and comparative analysis of results.

Objectives To examine treatment decision-making priorities and experiences among parents of children with cancer in Guatemala.Setting This study was conducted at Guatemala’s National Pediatric Cancer Center in Guatemala City.Participants Spanish-speaking parents of paediatric patients (≤18 years of age) diagnosed with any form of cancer within the 8 weeks prior to study enrolment. The quantitative portion of this study included 100 parent participants; the qualitative component included 20 parents. Most participants were Catholic or Evangelical Spanish-speaking mothers.Outcomes Priorities and experiences of cancer treatment decision-making including decision-making role and experienced regret.Results A range of paediatric ages and cancer diagnoses were included. Most Guatemalan parents surveyed (70%) made decisions about their child’s cancer together and almost all (94%) without input from their community. Surveyed parents predominately preferred shared decision-making with their child’s oncologist (76%), however 69% agreed it was best not to be provided with many options. Two-thirds of surveyed parents (65%) held their preferred role in decision-making, with fathers more likely to hold their preferred role than mothers (p=0.02). A small number of parents (11%) experienced heightened decisional regret, which did not correlate with socio-demographic characteristics or preferred decision-making role. Qualitative results supported quantitative findings, demonstrating a decision-making process that emphasised trust and honesty.Conclusions Guatemalan parents preferred to make decisions with their medical team and appreciated providers who were honest and inclusive, but directive about decisions. This study reinforces the importance of the provider–parent relationship and encourages clinicians in all settings to ask about and honour each parent’s desired role in decision-making.

PURPOSEThe global pediatric oncology clinical research landscape, particularly in Central and South America, Africa, and Asia, which bear the highest burden of global childhood cancer cases, is less characterized in the literature. Review of how existing pediatric cancer clinical trial groups internationally have been formed and how their research goals have been pursued is critical for building global collaborative research and data-sharing efforts, in line with the WHO Global Initiative for Childhood Cancer.METHODSA narrative literature review of collaborative groups performing pediatric cancer clinical research in each continent was conducted. An inventory of research groups was assembled and reviewed by current pediatric cancer regional and continental leaders. Each group was narratively described with identification of common structural and research themes among consortia.RESULTSThere is wide variability in the structure, history, and goals of pediatric cancer clinical trial collaborative groups internationally. Several continental regions have longstanding endogenously-formed clinical trial groups that have developed and published numerous adapted treatment regimens to improve outcomes, whereas other regions have consortia focused on developing foundational database registry infrastructure supported by large multinational organizations or twinning relationships.CONCLUSIONThere cannot be a one-size-fits-all approach to increasing collaboration between international pediatric cancer clinical trial groups, as this requires a nuanced understanding of local stakeholders and resources necessary to form partnerships. Needs assessments, performed either by local consortia or in conjunction with international partners, have generated productive clinical trial infrastructure. To achieve the goals of the Global Initiative for Childhood Cancer, global partnerships must be sufficiently granular to account for the distinct needs of each collaborating group and should incorporate grassroots approaches, robust twinning relationships, and implementation science.

PURPOSEWe aimed to evaluate the capacity to treat retinoblastoma in the Middle East, North Africa, and West Asia region.METHODSA Web-based assessment that investigated retinoblastoma-related pediatric oncology and ophthalmology infrastructure and associated capacity at member institutions of the Pediatric Oncology East and Mediterranean group was distributed. Data were analyzed in terms of availability, location, and confidence of use for each resource needed for the management of retinoblastoma. Resources were categorized by diagnostics, focal therapy, chemotherapy, advanced treatment, and supportive care. Responding institutions were further divided into an asset-based tiered system.RESULTSIn total, responses from 23 institutions were obtained. Fifteen institutions reported the availability of an ophthalmologist, 12 of which held primary off-site appointments. All institutions reported the availability of a pediatric oncologist and systemic chemotherapy A significant portion of available resources was located off site. Green laser was available on site at seven institutions, diode laser at six institutions, cryotherapy at 12 institutions, and brachytherapy at nine institutions. There existed marked disparity between the availability of some specific ophthalmic resources and oncologic resources.CONCLUSIONThe assessment revealed common themes related to the treatment of retinoblastoma in low- and- middle-income countries, including decentralization of care, limited resources, and lack of multidisciplinary care. Resource disparities warrant targeted intervention in the Middle East, North Africa, and West Asia region to advance the management of retinoblastoma in the region.

PURPOSE Hospitalized pediatric oncology patients are at high risk of deterioration and require frequent interdisciplinary communication to deliver high-quality care. Pediatric early warning systems (PEWS) are used by hospitals to reduce deterioration, but it is unknown how these systems affect communication about patient care in high- and limited-resource pediatric oncology settings. METHODS This qualitative study included semistructured interviews describing PEWS and subsequent team communication at 2 pediatric cancer centers, 1 in the United States and 1 in Guatemala. Participants included nurses, and frontline and intensive care providers who experienced recent deterioration events. Transcripts were coded and analyzed inductively using MAXQDA software. RESULTS The study included 41 providers in Guatemala and 42 providers in the United States (33 nurses, 30 ward providers, and 20 pediatric intensive care providers). Major themes identified include “hierarchy,” “empowerment,” “quality and method of communication,” and “trigger.” All providers described underlying medical hierarchies affecting the quality of communication regarding patient deterioration events and identified PEWS as empowering. Participants from the United States described the algorithmic approach to care and technology associated with PEWS contributing to impaired clinical judgement and a lack of communication. In both settings, PEWS sparked interdisciplinary communication and inspired action. CONCLUSION PEWS enhance interdisciplinary communication in high- and limited-resource study settings by empowering bedside providers. Traditional hierarchies contributed to negative communication and, in well-resourced settings, technology and automation resulted in lack of communication. Understanding contextual elements is integral to optimizing PEWS and improving pediatric oncology outcomes in hospitals of all resource levels.

Pediatric adrenocortical tumors (ACTs) are rare and heterogeneous. Approximately 50% of children with ACT carry a germline TP53 variant; however, the genetic underpinning of remaining cases has not been elucidated. In patients having germline TP53 variants, loss of maternal chromosome 11 and duplication of the paternal copy [paternal uniparental disomy, (UPD)] occurs early in tumorigenesis and explains the overexpression of IGF2, the hallmark of pediatric ACT. Beckwith-Wiedemann syndrome (BWS) is also associated with overexpression of IGF2 due to disruption of the 11p15 loci, including segmental UPD. Here, we report six children with ACT with wild type TP53 and germline paternal 11p15 UPD. Median age of five girls and one boy was 3.2 years (range 0.5-11 years). Two patients met the criteria for BWS before diagnosis of ACT. However, ACT was the first and only manifestation of paternal 11p15 UPD in four children. Tumor weight ranged from 21.5 g to 550 g. Despite poor prognostic features at presentation, such as pulmonary metastasis, bilateral adrenal involvement, and large tumors, all patients are alive 8-21 years after cancer diagnosis. Our observations suggest that children with ACT and wild type TP53, irrespective of their age, should be screened for germline abnormalities in chromosome 11p15.

BackgroundChildren with cancer are at high risk for clinical deterioration and subsequent mortality. Pediatric Early Warning Systems (PEWS) have proven to reduce the frequency of clinical deterioration in hospitalized patients. This qualitative study evaluates provider perspectives on the impact of PEWS on quality of care during deterioration events in a high-resource and a resource-limited setting.MethodsWe conducted semi-structured interviews with 83 healthcare staff (nurses, pediatricians, oncology fellows, and intensivists) involved in recent deterioration events at two pediatric oncology hospitals of different resource levels: St. Jude Children’s Research Hospital (SJCRH; n = 42) and Unidad Nacional de Oncología Pediátrica (UNOP; n = 41). Interviews were conducted in the participant’s native language (English or Spanish), translated into English, and transcribed. Transcripts were coded and analyzed inductively.ResultsProviders discussed both positive and negative perspectives of clinical deterioration events. Content analysis revealed “teamwork,” “experience with deterioration,” “early awareness,” and “effective communication” as themes associated with positive perception of events, which contributed to patient safety. Negative themes included “lack of communication,” “inexperience with deterioration,” “challenges with technology”, “limited material resources,” “false positive score,” and “objective tool.” Participants representing all disciplines across both institutions shared similar positive opinions. Negative opinions, however, differed between the two institutions, with providers at UNOP highlighting limited resources while those at SJCRH expressing concerns about technology misuse.ConclusionProviders that care for children with cancer find PEWS valuable to improve the quality of hospital care, regardless of hospital resource-level. Identified challenges, including inadequate critical care resources and challenges with technology, differ by hospital resource-level. These findings build on growing data demonstrating the positive impact of PEWS on quality of care and encourage wide dissemination of PEWS in clinical practice.

PURPOSE: The most significant adverse risk factor for neuroblastoma (NB) is MYCN gene amplification, which strongly associates with high-risk disease. Fluorescent in situ hybridization (FISH) is considered the best method to evaluate MYCN gene status. However, it requires a laboratory that can perform highly complex testing, specialized personnel, and costly reagents. Herein, we aimed to investigate the feasibility of using immunohistochemistry (IHC) to detect MYCN protein expression in lieu of FISH, a strategy potentially useful in areas with limited resources. METHODS: A pilot cohort of 78 patients with NB, including 34 of Middle Eastern descent (MED) who had a higher prevalence of MYCN gene amplification (44.11%) and 44 of North American descent (NAD), nine (20.45%) of whom had MYCN amplification, was evaluated with IHC for MYCN protein. Correlations of FISH results and protein expression are presented. RESULTS: A positive correlation between MYCN gene amplification and protein expression by IHC was seen in 22 (91.66%) of the 24 MYCN-amplified NB cases—14 (93.33%) of 15 patients of MED and eight (88.88%) of nine patients of NAD. Agreement between negative FISH and negative IHC results was noted in 18 (94.73%) patients of MED and 34 (97.14%) patients of NAD. Two cases had weak protein expression but no gene amplification (MED: n = 1; 5.0%; NAD: n = 1; 2.9%). CONCLUSION: An excellent overall correlation between MYCN gene status by FISH and MYCN protein expression by IHC was confirmed. MYCN IHC in NB with reflexing to FISH in equivocal cases is potentially useful in a limited-resource setting. Evaluation of effectiveness using a larger cohort and optimization to perform MYCN IHC manually is needed.

Accessibility to immunohistochemistry (IHC) is invaluable to proper diagnosis and treatment of pediatric patients with malignant neoplasms. Whereas IHC is widely available in anatomic pathology laboratories in high-income countries, access to it in anatomic pathology laboratories of low- and middle-income countries remains a struggle, with many limitations. To advance the quality of the pathology service offered to children with cancer in areas with limited resources, a 5-day pathology training workshop was offered to pathologists and histotechnologists from various countries of the Central American and Caribbean region. An initial assessment of the workshop participants’ current laboratory capacities was performed, and a regional training center was selected. Didactic and hands-on activities were offered, and review and evaluation of the IHC slides produced during the training course were compared with original slides from the participants’ sites. This model of intensive 5-day training appears to be effective and can potentially be used in other budget-constrained regions. Moreover, it can serve as a continuing education activity for pathologists and histotechnologists, and as part of validations and quality improvement projects to build capacity and develop IHC assay proficiency in low- and middle-income countries.

Background: Skilled healthcare professionals are critical for providing quality healthcare for children with cancer globally. Training curricula addressing the knowledge needs in infection care and prevention (ICP) in cancer are scarce. Program description: We implemented a 10-week blended course in ICP. The distance learning had four 2-week modules: Infectious Complications, Quality in Infection Care, Quality in Infection Prevention, and Sustainability, Research, and Dissemination. Each module had pre- and post-tests and weekly webinars. The 2-week in-person learning had lectures, group exercises, clinical observations, hospital and laboratory tours, and ended in an annual conference. An individual project developed during the distance learning was presented in the in-person workshop. Course attendance criteria were English language proficiency and participants’ role in ICP at their institutions. Program evaluation and results: Twenty-two students from 17 hospitals in 10 countries completed the course, developed a project, and answered surveys covering knowledge assessments and satisfaction, and 6-month course and 1-year project follow-ups. Pretest and post-test scores revealed knowledge improvement ( P

Introduction Fatalistic cancer beliefs may contribute to delayed diagnosis and poor outcomes, including treatment abandonment, for children with cancer. This study explored Guatemalan parents’ cancer beliefs during initial paediatric cancer communication, and the sociocultural and contextual factors that influence these beliefs.Methods Twenty families of children with cancer were included in this study. We audio-recorded psychosocial conversations with psychologists and diagnostic conversations with oncologists, then conducted semi-structured interviews with parents to explore the evolution of their cancer beliefs. Audio-recordings were transcribed and translated from Spanish into English, with additional review in both languages by bilingual team members. All 60 transcripts were thematically analysed using a priori and novel codes.Results Guatemalan parents’ beliefs evolve as they learn about cancer through various sources. Sources of information external to the cancer centre, including prior experiences with cancer, media exposure, community discussion and clinical encounters, contribute to pre-existing beliefs. Many parents’ pre-existing cancer beliefs are fatalistic; some are influenced by Mayan spirituality. Sources internal to the cancer centre include psychologists and oncologists, other providers, other patients and families. Psychologists acknowledge pre-existing beliefs and deliver cancer education using verbal explanations and hand-drawings. Oncologists provide diagnostic information and outline treatment plans. Both support hope by providing a path toward cure. Parents’ lived experience is a culmination of sources and simultaneously independent. Ultimately most parents arrive at an understanding of cancer that is consistent with an allopathic medical model and offers optimism about outcomes.Conclusion An interdisciplinary communication process that includes cancer education, is attentive to pre-existing beliefs, and supports hope may encourage acceptance of the allopathic medical model and need for treatment. Providers in settings of all resource levels may be able to use these techniques to support cross-cultural cancer communication, reduce treatment abandonment and improve therapy adherence.

BackgroundPediatric oncology patients have a high rate of clinical deterioration frequently requiring critical care. Patient deterioration events are distressing for clinicians, but little is known about how Pediatric Early Warning Systems (PEWS) impact clinicians’ emotional responses to deterioration events.MethodsSemi-structured interviews were conducted with 83 nurses, pediatricians, oncologists, and intensive care clinicians who had recently participated in a patient deterioration event at two pediatric oncology hospitals of different resource-levels: St. Jude Children’s Research Hospital (N = 42 participants) in Memphis, Tennessee or Unidad Nacional de Oncología Pediátrica (N = 41 participants) in Guatemala City, Guatemala. Interviews were conducted in the participants’ native language (English or Spanish), transcribed, and translated into English. Each transcript was coded by two researchers and analyzed for thematic content.ResultsEmotions around patient deterioration including concern, fear, and frustration were reported across all disciplines at both hospitals. Concern was often triggered by an elevated PEWS score and usually resulted in increased attention, which reassured bedside clinicians that patients were receiving necessary interventions. However, persistently elevated PEWS scores, particularly at St. Jude Children’s Research Hospital, occasionally resulted in a false sense of relief, diminishing clinician attention and negatively impacting patient care. Nurses at both institutions described how PEWS amplified their voices, engendering confidence and empowerment, two of the only positive emotions described in the study.ConclusionClinicians experienced a range of emotions while caring for high-risk patients in the setting of clinical deterioration. These emotions have the potential to contribute to compassion fatigue and burnout, or to resilience. Acknowledgment and further investigation of the complex interplay between PEWS and clinician emotions are necessary to maximize the impact of PEWS on patient safety while simultaneously supporting staff wellbeing.

Purpose: Global data mapping access to essential chemotherapeutics for pediatric cancer are scarce. We report a survey of international pediatric cancer care providers’ access to these medicines. Methods: A Web-based survey was sent to pediatric oncologists registered on the Cure4Kids Web portal. We queried chemotherapeutics in the WHO Essential Medicines List for Children, from which the average proportional availability was summarized as each country’s access score. In addition, we examined availability of drug packages defined by the WHO-sanctioned Expert Committee for eight pediatric cancers. We undertook a sensitivity analysis investigating how regimen access would change if the cytotoxics specified in recent agreements between the Clinton Health Access Initiative, American Cancer Society, and pharmaceutical companies were universally available. Results: There were significant (P < .001) differences in the median access scores between World Bank income groups, and 42.9% of respondents from low-income and lower middle–income countries reported suboptimal access scores. Our disease-based analysis revealed that 42.1% of patients in low-income and lower middle–income countries lacked full access to chemotherapy packages. Guaranteed availability of the cytotoxics specified in the Clinton Health Access Initiative/American Cancer Society agreements was projected to increase this regimen-based access by 1.6%, although including four additional chemotherapeutics would further increase coverage by 13.9%. Conclusion: This study is the first, to our knowledge, to assess worldwide variation in practical access to pediatric chemotherapy. Although mapping the proportion of available chemotherapeutics is informative, we also developed a meaningful estimate of access using disease-specific drug packages. These data provide an important baseline for continued monitoring and can aid in planning adaptive treatment guidelines that consider the trade-offs between access and outcomes.

Table of contents O1 Tumour heterogeneity: what does it mean? Dow-Mu Koh O2 Skeletal sequelae in adult survivors of childhood cancer Sue Creviston Kaste O3 Locoregional effects of breast cancer treatment Sarah J Vinnicombe O4 Imaging of cancer therapy-induced CNS toxicity Giovanni Morana, Andrea Rossi O5 Screening for lung cancer Christian J. Herold O6Risk stratification of lung nodules Theresa C. McLoud O7 PET imaging of pulmonary nodules Kirk A Frey O8 Transarterial tumour therapy Bernhard Gebauer O9 Interventional radiology in paediatric oncology Derek Roebuck O10 Image guided prostate interventions Jurgen J. Fütterer O11 Imaging cancer predisposition syndromes Alexander J. Towbin O12Chest and chest wall masses Thierry AG Huisman O13 Abdominal masses: good or bad? Anne MJB Smets O14 Hepatobiliary MR contrast: enhanced liver MRI for HCC diagnosis and management Giovanni Morana O15 Role of US elastography and multimodality fusion for managing patients with chronic liver disease and HCC Jeong Min Lee O16 Opportunities and challenges in imaging metastatic disease Hersh Chandarana O17 Diagnosis, treatment monitoring, and follow-up of lymphoma Marius E. Mayerhoefer, Markus Raderer, Alexander Haug O18 Managing high-risk and advanced prostate cancer Matthias Eiber O19 Immunotherapy: imaging challenges Bernhard Gebauer O20 RECIST and RECIST 1.1 Andrea Rockall O21 Challenges of RECIST in oncology imaging basics for the trainee and novice Aslam Sohaib O22 Lymphoma: PET for interim and end of treatment response assessment: a users’ guide to the Deauville Score Victoria S Warbey O23 Available resources Hebert Alberto Vargas O24 ICIS e-portal and the online learning community Dow-Mu Koh O25 Benign lesions that mimic pancreatic cancer Jay P Heiken O26 Staging and reporting pancreatic malignancies Isaac R Francis, Mahmoud, M Al-Hawary, Ravi K Kaza O27 Intraductal papillary mucinous neoplasm Giovanni Morana O28 Cystic pancreatic tumours Mirko D’Onofrio O29 Diffusion-weighted imaging of head and neck tumours Harriet C. Thoeny O30 Radiation injury in the head and neck Ann D King O31 PET/MR of paediatric brain tumours Giovanni Morana, Arnoldo Piccardo, Maria Luisa Garrè, Andrea Rossi O32 Structured reporting and beyond Hebert Alberto Vargas O33 Massachusetts General Hospital experience with structured reporting Theresa C. McLoud O34 The oncologist’s perspective: what the oncologist needs to know Nick Reed O35 Towards the cure of all children with cancer: global initiatives in pediatric oncology Carlos Rodriguez-Galindo O36 Multiparametric imaging of renal cancers Hersh Chandarana O37 Linking imaging features of renal disease and their impact on management strategies Hebert Alberto Vargas O38 Adrenals, retroperitoneum and peritoneum Isaac R Francis, Ashish P Wasnik O39 Lung and pleura Stefan Diederich O40 Advances in MRI Jurgen J. Fütterer O41 Advances in molecular imaging Wim J.G. Oyen O42 Incorporating advanced imaging, impact on treatment selection and patient outcome Cheng Lee Chaw, Nicholas van As S1 Combining ADC-histogram features improves performance of MR diffusion-weighted imaging for Lymph node characterisation in cervical cancer Igor Vieira, Frederik De Keyzer, Elleke Dresen, Sileny Han, Ignace Vergote, Philippe Moerman, Frederic Amant, Michel Koole, Vincent Vandecaveye S2 Whole-body diffusion-weighted MRI for surgical planning in patients with colorectal cancer and peritoneal metastases R Dresen, S De Vuysere, F De Keyzer, E Van Cutsem, A D’Hoore, A Wolthuis, V Vandecaveye S3 Role of apparent diffusion coefficient (ADC) diffusion-weighted MRI for predicting extra capsular extension of prostate cancer. P. Pricolo (paola.pricolo@ieo.it), S. Alessi, P. Summers, E. Tagliabue, G. Petralia S4 Generating evidence for clinical benefit of PET/CT – are management studies sufficient as surrogate for patient outcome? C. Pfannenberg, B. Gückel, SC Schüle, AC Müller, S. Kaufmann, N. Schwenzer, M. Reimold,C. la Fougere, K. Nikolaou, P. Martus S5 Heterogeneity of treatment response in skeletal metastases from breast cancer with 18F-fluoride and 18F-FDG PET GJ Cook, GK Azad, BP Taylor, M Siddique, J John, J Mansi, M Harries, V Goh S6 Accuracy of suspicious breast imaging—can we tell the patient? S Seth, R Burgul, A Seth S7 Measurement method of tumour volume changes during neoadjuvant chemotherapy affects ability to predict pathological response S Waugh, N Muhammad Gowdh, C Purdie, A Evans, E Crowe, A Thompson, S Vinnicombe S8 Diagnostic yield of CT IVU in haematuria screening F. Arfeen, T. Campion, E. Goldstraw S9 Percutaneous radiofrequency ablation of unresectable locally advanced pancreatic cancer: preliminary results D’Onofrio M, Ciaravino V, Crosara S, De Robertis R, Pozzi Mucelli R S10 Iodine maps from dual energy CT improve detection of metastases in staging examinations of melanoma patients M. Uhrig, D. Simons, H. Schlemmer S11Can contrast enhanced CT predict pelvic nodal status in malignant melanoma of the lower limb? Kate Downey S12 Current practice in the investigation for suspected Paraneoplastic Neurological Syndromes (PNS) and positive malignancy yield. S Murdoch, AS Al-adhami, S Viswanathan P1 Technical success and efficacy of Pulmonary Radiofrequency ablation: an analysis of 207 ablations S Smith, P Jennings, D Bowers, R Soomal P2 Lesion control and patient outcome: prospective analysis of radiofrequency abaltion in pulmonary colorectal cancer metastatic disease S Smith, P Jennings, D Bowers, R Soomal P3 Hepatocellular carcinoma in a post-TB patient: case of tropical infections and oncologic imaging challenges TM Mutala, AO Odhiambo, N Harish P4 Role of apparent diffusion coefficient (ADC) diffusion-weighted MRI for predicting extracapsular extension of prostate cancer P. Pricolo, S. Alessi, P. Summers, E. Tagliabue, G. Petralia P5 What a difference a decade makes; comparison of lung biopsies in Glasgow 2005 and 2015 M. Hall, M. Sproule, S. Sheridan P6 Solid pseudopapillary tumour of pancreas: imaging features of a rare neoplasm KY Thein, CH Tan, YL Thian, CM Ho P7 MDCT - pathological correlation in colon adenocarcinoma staging: preliminary experience S De Luca, C Carrera, V Blanchet, L Alarcón, E Eyheremnedy P8 Image guided biopsy of thoracic masses and reduction of pneumothorax risk: 25 years experience B K Choudhury, K Bujarbarua, G Barman P9 Tumour heterogeneity analysis of 18F-FDG-PET for characterisation of malignant peripheral nerve sheath tumours in neurofibromatosis-1 GJ Cook, E Lovat, M Siddique, V Goh, R Ferner, VS Warbey P10 Impact of introduction of vacuum assisted excision (VAE) on screen detected high risk breast lesions L Potti, B Kaye, A Beattie, K Dutton P11 Can we reduce prevalent recall rate in breast screening? AA Seth, F Constantinidis, H Dobson P12 How to reduce prevalent recall rate? Identifying mammographic lesions with low Positive Predictive Value (PPV) AA Seth (archana.seth@nhs.net), F Constantinidis, H Dobson P13 Behaviour of untreated pulmonary thrombus in oncology patients diagnosed with incidental pulmonary embolism on CT R. Bradley, G. Bozas, G. Avery, A. Stephens, A. Maraveyas P14 A one-stop lymphoma biopsy service – is it possible? S Bhuva, CA Johnson, M Subesinghe, N Taylor P15 Changes in the new TNM classification for lung cancer (8th edition, effective January 2017) LE Quint, RM Reddy, GP Kalemkerian P16 Cancer immunotherapy: a review of adequate imaging assessment G González Zapico, E Gainza Jauregui, R Álvarez Francisco, S Ibáñez Alonso, I Tavera Bahillo, L Múgica Álvarez P17 Succinate dehydrogenase mutations and their associated tumours O Francies, R Wheeler, L Childs, A Adams, A Sahdev P18 Initial experience in the usefulness of dual energy technique in the abdomen SE De Luca, ME Casalini Vañek, MD Pascuzzi, T Gillanders, PM Ramos, EP Eyheremendy P19 Recognising the serious complication of Richter’s transformation in CLL patients C Stove, M Digby P20 Body diffusion-weighted MRI in oncologic practice: truths, tricks and tips M. Nazar, M. Wirtz, MD. Pascuzzi, F. Troncoso, F. Saguier, EP. Eyheremendy P21 Methotrexate-induced leukoencephalopathy in paediatric ALL Patients D.J. Quint, L. Dang, M. Carlson, S. Leber, F. Silverstein P22 Pitfalls in oncology CT reporting. A pictorial review R Rueben, S Viswanathan P23 Imaging of perineural extension in head and neck tumours B Nazir, TH Teo, JB Khoo P24 MRI findings of molecular subtypes of breast cancer: a pictorial primer K Sharma, N Gupta, B Mathew, T Jeyakumar, K Harkins P25 When cancer can’t wait! A pictorial review of oncological emergencies K Sharma, B Mathew, N Gupta, T Jeyakumar, S Joshua P26 MRI of pancreatic neuroendocrine tumours: an approach to interpretation D Christodoulou, S Gourtsoyianni, A Jacques, N Griffin, V Goh P27 Gynaecological cancers in pregnancy: a review of imaging CA Johnson, J Lee P28 Suspected paraneoplastic neurological syndromes - review of published recommendations to date, with proposed guideline/flowchart JA Goodfellow, AS Al-adhami, S Viswanathan P29 Multi-parametric MRI of the pelvis for suspected local recurrence of prostate cancer after radical prostatectomy R Bradley P30 Utilisation of PI-RADS version 2 in multi-parametric MRI of the prostate; 12-months experience R Bradley P31 Radiological assessment of the post-chemotherapy liver A Yong, S Jenkins, G Joseph P32 Skeletal staging with MRI in breast cancer – what the radiologist needs to know S Bhuva, K Partington P33 Perineural spread of lympoma: an educational review of an unusual distribution of disease CA Johnson, S Bhuva, M Subesinghe, N Taylor P34 Visually isoattenuating pancreatic adenocarcinoma. Diagnostic imaging tools. C Carrera, A Zanfardini, S De Luca, L Alarcón, V Blanchet, EP Eyheremendy P35 Imaging of larynx cancer: when is CT, MRI or FDG PET/CT the best test? K Cavanagh, E Lau

Advances in multidisciplinary care for pediatric cancer have resulted in significant improvement in cure rates over the last decades; however, these advances have not been uniform across all age groups. Cancer is an important cause of perinatal mortality, yet the full spectrum of malignant neoplasms in newborns is not well defined. Methods: The authors have reviewed the clinical features and outcomes of 37 newborns with congenital malignant tumors treated at three referral centers in North, Central, and South Poland between 1980 and 2014. Event-free survival (EFS) and overall survival (OS) rates were estimated by Kaplan–Meier methods and compared using long-rank test and Cox models. Results: Twenty-two patients were diagnosed prenatally. The most common diagnoses were neuroblastoma (48.7%), followed by malignant germ-cell tumor (16.2%), and Wilms' tumor (8.1%). Neuroblastoma was the most common malignancy among full-term infants, and malignant sacrococcygeal teratoma was the most common malignancy in premature infants. Thirty patients (81%) are alive with a median follow-up of 4.8 years from diagnosis. Patients with Wilms' tumor and malignant germ-cell tumors had the best outcomes (5-year OS 100% for both), whereas the worst prognosis was observed for sarcoma patients (5-year OS 72.92%). Premature infants had better outcome than full-term infants (5-year OS 92.8% vs. 72.58%, respectively). Conclusion: Although rare, neonatal cancers can present with an aggressive clinical behavior, but they have a generally good outcome. Early diagnosis and management by expert multidisciplinary teams that integrate perinatal medicine experts with pediatric and surgical oncologists are critical. Centralized care with clear referral pathways that facilitate early initiation of specialized treatment should be prioritized.

Identifying therapeutic targets in rare cancers is challenging due to the lack of relevant pre-clinical models. Here, the authors generate a cancer cell line from a paediatric patient with a rare undifferentiated sarcoma and through functional genomics and chemical screens identified CDK4 and XPO1 as potential therapeutic targets in this cancer.

Retinoblastoma is the most common primary intraocular tumor in children. The first sign that is often reported by parents is the appearance of recurrent leukocoria (i.e., "white eye") in recreational photographs. A quantitative definition or scale of leukocoria--as it appears during recreational photography--has not been established, and the amount of clinical information contained in a leukocoric image (collected by a parent) remains unknown. Moreover, the hypothesis that photographic leukocoria can be a sign of early stage retinoblastoma has not been tested for even a single patient. This study used commercially available software (Adobe Photoshop®) and standard color space conversion algorithms (operable in Microsoft Excel®) to quantify leukocoria in actual "baby pictures" of 9 children with retinoblastoma (that were collected by parents during recreational activities i.e., in nonclinical settings). One particular patient with bilateral retinoblastoma ("Patient Zero") was photographed >7, 000 times by his parents (who are authors of this study) over three years: from birth, through diagnosis, treatment, and remission. This large set of photographs allowed us to determine the longitudinal and lateral frequency of leukocoria throughout the patient's life. This study establishes: (i) that leukocoria can emerge at a low frequency in early-stage retinoblastoma and increase in frequency during disease progression, but decrease upon disease regression, (ii) that Hue, Saturation and Value (i.e., HSV color space) are suitable metrics for quantifying the intensity of retinoblastoma-linked leukocoria; (iii) that different sets of intraocular retinoblastoma tumors can produce distinct leukocoric reflections; and (iv) the Saturation-Value plane of HSV color space represents a convenient scale for quantifying and classifying pupillary reflections as they appear during recreational photography.