Pediatric Oncology Research Research Articles

Interhospital variability in localization techniques for small pulmonary nodules in children: A pediatric surgical oncology research collaborative study

BackgroundPulmonary nodules that are deep within lung parenchyma and/or small in size can be challenging to localize for biopsy. This study describes current trends in performance of image-guided localization techniques for pulmonary nodules in pediatric patients. MethodsA retrospective review was performed on patients < 21 years of age undergoing localization of pulmonary nodules at 15 institutions. Localization and resection success, time in interventional radiology (IR), operating room (OR) and total anesthesia time, complications, and technical problems were compared between techniques. Results225 patients were included with an average of 1.3 lesions (range 1–5). Median nodule size and depth were 4 mm (range 0–30) and 5.4 mm (0–61), respectively. The most common localization techniques were: wire + methylene blue dye (MBD) (28%), MBD only (25%), wire only (14%), technetium-99 only (11%), coil + MBD (7%) and coil only (5%). Localization technique was associated with institution (p < 0.01); technique and institution were significantly associated with mean IR, OR, and anesthesia time (all p < 0.05). Comparing techniques, there was no difference in successful IR localization (range 92–100%, p = 0.75), successful resection (94–100%, p = 0.98), IR technical problems (p = 0.22), or operative complications (p = 0.16). ConclusionsMany IR localization techniques for small pulmonary nodules in children can be successful, but there is wide variability in application by institution and in procedure time. Level of evidenceRetrospective review, Level 3.

Malnutrition identification and management variability: An administrative database study of children with solid tumors.

BackgroundMalnutrition during cancer treatment increases treatment‐related morbidity and mortality. Our study better characterizes variability in malnutrition identification and treatment by examining nutrition‐related diagnoses and support for children with central nervous system (CNS) and non‐CNS solid tumors during therapy. We examined diagnosis of malnutrition, use of tube feeding or parenteral nutrition (PN), and appetite stimulants.MethodsWe retrospectively reviewed 0 to 21‐year‐old patients in the Pediatric Health Information System from 2015 to 2019. Patients were classified as having (1) billed malnutrition diagnosis, (2) malnutrition diagnosis or using PN and enteral nutrition (“functional malnutrition”), and (3) any previous criteria or prescribed appetite stimulants (“possible malnutrition”), as well as associated risk factors.ResultsAmong 13,375 unique patients, CNS tumors were most common (24.4%). Overall, 26.5% of patients had malnutrition diagnoses, 45.4% met functional malnutrition criteria, and 56.0% had possible malnutrition. Patients with adrenal tumors had highest billed, functional, and possible malnutrition (36.6%, 64.1%, and 69.4%, respectively) followed by CNS tumors (29.1%, 52.4%, and 64.1%). Patients with adrenal tumors had highest rates of PN use (47.4%) and those with CNS tumors had the highest tube feeding use (26.8%). Hospital admissions with malnutrition had a longer hospital length of stay (LOS) (6 vs 3 days, P < 0.0001), more emergency department admissions (24.4% vs 21.8%, P < 0.0001), and more opioid use (58.6% vs 41.4%, P < 0.0001).ConclusionsVariability in malnutrition diagnoses hinders clinical care and nutrition research in pediatric oncology. Improving disease‐specific recognition and treatment of malnutrition can target nutrition support, ensure appropriate reimbursement, and potentially improve outcomes for children with solid tumors.

Education for Children With Chronic Illness

This Viewpoint provides an overview of online learning trends and highlights findings from pediatric oncology research, providing insight regarding ensuring optimal learning for all children with chronic medical needs.

Recruitment principles and strategies for supportive care research in pediatric oncology

BackgroundVariations in clinical practice contribute to negative outcomes for children with cancer. Research in this area is imperative to standardise practice, yet such research is challenging to undertake, and a significant proportion of studies fail. A common reason for failure is poor recruitment, yet little information is available to support researchers and clinicians planning such research.MethodsOur primary aim was to describe the recruitment strategies and outcomes in a tertiary children’s hospital across multiple observational supportive care studies. Secondary aims were to establish principles to improve both recruitment strategies and the reporting of recruitment. We undertook a retrospective descriptive analysis of the recruitment logs and data from three studies in pediatric oncology. The mean time to recruit one participant was calculated. Common reasons for not approaching eligible participants and reasons potential participants declined are described.ResultsOf the 235 potential candidates across all studies, 186 (79%) were approached and of these 125 (67%) provided consent, with 117 (63%) completing baseline measures. We estimated recruitment per participant required an average 98 min of experienced research nurse time. Four factors are described that influence recruitment and six principles are outlined to maximise recruitment and the generalisability of research findings.ConclusionsWe highlight the recruitment experiences across three different projects in children’s cancer supportive care research and provide a roadmap for other researchers planning to undertake clinical research in pediatrics.

Setting international standards for patient and parent involvement and engagement in childhood, adolescent and young adult cancer research: A report from a European Collaborative Workshop.

BackgroundPatient and Public Involvement and Engagement (PPIE) in research, advocates for research conducted ‘with’ not ‘for’ the affected population. In paediatric oncology research, the parents of children, adolescents and young adults affected by cancer are represented by the term ‘public’ in the acronym PPIE. Patients (those with cancer and cancer survivors) are also passionate advocates who drive forward the research priorities of children, adolescents and young adults throughout the entire research process.AimsA workshop was held at an international professional meeting in 2019 with the aim to define Patient and Parent Involvement and Engagement (PPIE); capture PPIE activities on a European level; and to explore the role of PPIE in non‐interventional research. A proposed framework for a European PPIE strategy for childhood, adolescent and young adult cancers was also discussed.MethodsThe 60‐minute workshop was attended by health care professionals, researchers, scientists, parents, survivors and charity/support organisations. A presentation to define PPIE, including the difference in terminology for PPIE in the context of childhood, adolescent, and young adult cancers was discussed. Best practice examples from the United Kingdom (UK) helped to demonstrate the positive impact of PPIE in paediatric oncology research. Three breakout groups then explored themes relating to PPIE, namely PPIE priorities, PPIE mapping for Europe, and PPIE in non‐interventional research and data‐linkage.ResultsDisparity in PPIE activities across Europe was evident, with ambiguity surrounding terminology and expected roles for PPIE representatives in paediatric oncology research. A lack of PPIE activity in Eastern Europe correlated with a lack of availability for clinical trials and poorer survival rates for paediatric oncology patients. There was unanimous support for PPIE embedded research in all areas, including in non‐interventional studies.ConclusionA European‐level definition of PPIE for paediatric oncology research is needed. Further exploration into the role and responsibilities of patients, parents, and professionals when undertaking PPIE related activities is also recommended. Best practice examples from the UK, France, Germany, The Netherlands and Belgium demonstrated a preliminary evidence base from which a European PPIE strategy framework can be designed, inclusive of the patient and parent voice.

Abstract 2289: Empowering point-and-click genomic analysis with large pediatric genomic reference data on St. Jude Cloud

Abstract Next-generation sequencing-based genomic profiling is now a mainstay of pediatric oncology research and clinical testing. Correlating genomic features of patient cancer genomes with curated data extracted from large reference cohorts is critical for identifying molecular subtypes and underlying mutagenesis processes. To facilitate such investigation, we developed two user-friendly workflows on St. Jude Cloud, a data sharing ecosystem hosting genomic data for &amp;gt;10,000 pediatric cancer patients and survivors. These workflows leverage St. Jude Cloud comprehensive pediatric cancer genomic data, including 1,616 RNA-seq of 135 cancer subtypes and 958 whole genome sequencing (WGS) of 35 subtypes, to enable user analysis of their data in the context of St. Jude Cloud cohorts without a need to download large datasets. The RNA-Seq Expression Classification workflow enables a user to compare their patient RNA-Seq gene expression data with blood (832), brain (456), and solid tumor (319) pediatric cancer reference cohorts and PDX models (45), enabling subtype classification using t-Distributed Stochastic Neighbor Embedding (t-SNE). Reference cohorts include curated subtype-defining somatic alterations integrating genomic variant data with expression profile. Resulting interactive t-SNE plots can be explored and annotated - with options to highlight cancer subtypes or samples and display sample information (age of onset, clinical diagnosis, molecular driver). To demonstrate, we analyze PAWNXH, a Children's Oncology Group AML sample with a novel ZBTB7A-NUTM1 fusion and find it clusters with AML samples harboring KMT2A re-arrangements suggesting a potential mechanism of pathogenesis. Integrating PDX samples enables model selection for functional experiments by connecting patient subtypes with mouse models. The Mutational Signatures workflow identifies and quantifies COSMIC mutational signatures in user-uploaded somatic VCF files for comparison to reference pediatric cancer cohorts. The interactive interface enables rapid identification of signatures within the query cohort and facilitates comparison to the reference using a cohort-level summary view. Identified signatures may also be explored at the sample-level for both query and reference cohorts, enabling the user to identify samples with signatures of interest for further analysis. We show an example comparison of mutational signatures identified in pediatric and adult AML samples. These workflows enable users to leverage curated pediatric cancer data to make discoveries in their own samples. Enabling point-and-click analysis in St. Jude Cloud removes the barrier for non-computational researchers and eliminates the need to download large reference datasets for local analysis. Both workflows utilize post-processed rather than raw genomic data, reducing transfer costs for uploading user data to the cloud. Citation Format: Andrew Thrasher, Michael Macias, Alexander M. Gout, Delaram Rahbarinia, Xin Zhou, Samuel W. Brady, Clay McLeod, Michael C. Rusch, Xiaolong Chen, Soheil Meshinchi, Michael A. Dyer, Suzanne J. Baker, Martine F. Roussel, Jinghui Zhang. Empowering point-and-click genomic analysis with large pediatric genomic reference data on St. Jude Cloud [abstract]. In: Proceedings of the American Association for Cancer Research Annual Meeting 2021; 2021 Apr 10-15 and May 17-21. Philadelphia (PA): AACR; Cancer Res 2021;81(13_Suppl):Abstract nr 2289.

Risk and Benefit for Targeted Therapy Agents in Pediatric Phase II Trials in Oncology: A Systematic Review with a Meta-Analysis.

BackgroundFor research with human participants to be ethical, risk must be in a favorable balance with potential benefits. Little is known about the risk/benefit ratio for pediatric cancer phase II trials testing targeted therapies.ObjectiveOur aim was to conduct a systematic review of preliminary efficacy and safety profiles of phase II targeted therapy clinical trials in pediatric oncology.MethodsOur protocol was prospectively registered in PROSPERO (CRD42020146491). We searched EMBASE and PubMed for phase II pediatric cancer trials testing targeted agents. We included solid and hematological malignancy studies published between 1 January, 2015 and 27 February, 2020. We measured risk using drug-related grade 3 or higher adverse events, and benefit by response rates. When possible, data were meta-analyzed. All statistical tests were two-sided.ResultsWe identified 34 clinical trials (1202 patients) that met our eligibility criteria. The pooled overall response rate was 24.4% (95% confidence interval [CI] 14.5–34.2) and was lower in solid tumors, 6.4% (95% CI 3.2–9.6), compared with hematological malignancies, 55.1% (95% CI 35.9–74.3); p < 0.001. The overall fatal drug-related (grade 5) adverse event rate was 1.6% (95% CI 0.6–2.5), and the average drug-related grade 3/4 adverse event rate per person was 0.66 (95% CI 0.55–0.78).ConclusionsWe provide an estimate for the risks and benefits of participation in pediatric phase II cancer trials. These data may be used as an empirical basis for informed communication about benefits and burdens in pediatric oncology research.Supplementary InformationThe online version contains supplementary material available at 10.1007/s11523-021-00822-5.

Inflammatory myofibroblastic tumor: A multi-institutional study from the pediatric surgical oncology research collaborative.

10024 Background: Inflammatory myofibroblastic tumor (IMT) is a mesenchymal neoplasm of intermediate malignancy, predominantly seen in children and young adults. Given its rarity, data are limited. We describe the largest cohort of patients with IMT to date, with an aim to further characterize this poorly understood tumor. Methods: A multi-institutional review was performed at 18 North American hospitals participating in the Pediatric Surgical Oncology Research Collaborative to identify IMT patients ≤39 years, diagnosed from 2000-2018. Descriptive statistics are described as median and interquartile range. Multivariable analysis was used to identify predictors of event free survival (EFS). Results: 182 patients were identified with a median age of 11 years (5-17); 52% were female. 33% of tumors were thoracic, 26% abdominal/pelvic, 20% head/neck, and 14% genitourinary. Common presenting symptoms included pain (29%), respiratory symptoms (24%), weight loss (12%), and a palpable mass (10%). Median tumor size was 3.9 cm (1.9-6.5). Anaplastic lymphoma kinase (ALK) overexpression was identified via immunohistochemistry in 53% of patients tested. One third of patients had abnormal cytogenetics, with 12% of the entire cohort having an ALK mutation. 7% of patients had distant disease at diagnosis. 13% of patients received neoadjuvant therapy including chemotherapy (3%), ALK inhibitor (4%), radiation (0.5%), non-steroidal anti-inflammatories (NSAIDs) (7%), or steroids (2%). Of those who underwent resection with known margin status (n = 158), 66% had complete resection, 20% had microscopic positive margins, and 14% had gross residual disease. Just over 40% of patients had an en bloc resection of involved organs, most commonly lung (26%). 21% of patients received adjuvant therapy, including chemotherapy (3%), ALK inhibitor (9%), radiation (0.5%), NSAIDs (8%), or steroids (5%). 12% of all patients received an ALK inhibitor: 24% neoadjuvant, 62% adjuvant, and 14% without surgery. Median follow-up time was 36 months (14-69). Overall 5-year survival (OS) was 95% and 5-year EFS was 80%. Predictors of recurrence included respiratory symptoms, larger tumor size, or distant disease at diagnosis. Gender, race, age and primary site were not predictive of EFS. Likewise, there was no association of ALK overexpression or ALK mutation with EFS. The presence of gross or microscopic margins following resection was not associated with recurrence. Conclusions: IMT is a rare tumor with favorable OS. Five year recurrence rate was 20%. Presenting with respiratory symptoms, larger tumor size, or metastatic disease was associated with recurrence, while ALK positivity was not. Notably, a positive margin after resection was not associated with increased recurrence, indicating that aggressive attempts at surgical resection that would compromise form or function may not be warranted.

Ethical Issues at the Interface of Physiotherapy Care and Research Practice in Pediatric Oncology-Descriptive Study

Pediatric oncology has a major role in research. This paper discuss about the ethical consequences ofphysiotherapy care and research in Pediatric Oncology. Empirical ethics is a broad division,grasping,difference in indicatingethics and empirical research. A major Australian tertiary teaching hospital revealsthat 65 % of patient presents with specific indication for physiotherapy, in that only12.8% are receivingphysiotherapy. Indications includes in physiotherapy care are pain, lymph- oedema, incontinence, respiratoryand musculoskeletal Problems also it includes hard to move. Physiotherapy has extensive role inpalliativeoncology, it also has large preventative ,educative and supportive roles to play and provides independentand complementary therapies for physical debility and pain. Pain from reflex spasm and contracture aredealt with Physiotherapists. Founded that no reference based on standard of life and quality-adjusted lifeyears in terms of preventing dependance and avoid hospitalisation. Treatment of pediatric oncology oftenhas physically, socially and psychologically demands. Always there must be some ethical reason for allthe decision on treatment and care for a patient with cancer. It is a fact that ethics needs not only all thestakeholders to do the right things but also it needs the one who do it in the right way. Research practice hasled us to great advances in pediatric oncology and the proportion is greater in children than adults. Obtainingthe informed consent and assent was the most important ethical challenges faced during the research.

Leveraging a qualitative data repository to integrate patient and caregiver perspectives into clinical research.

Understanding patient and caregiver experiences is a critical component of the conception, design, and implementation of clinical research studies. The "Database of Individual Patient Experiences" (DIPEx) is an innovative, evidence-based approach for eliciting rich information about health experiences. We conducted a formative evaluation with 14 pediatric oncology researchers to assess the value of using data from a DIPEx study on patient and caregiver experiences with childhood cancer to inform patient-centered research in pediatric oncology. Participants identified barriers to incorporating patient perspectives and experiences into their research and how the DIPEx approach could be leveraged to facilitate this practice.

Analysis of Pediatric Oncology Nursing Research in Mainland China, 2008-2018.

Up-to-date research from low- and middle-income countries is needed to inform local pediatric cancer nursing care and share best practices from these settings. Access to all current Chinese-language publications on pediatric oncology nursing research is limited because of a language barrier. The aim of this study was to document the volume, type of research study, and yearly and geographical distribution of published pediatric oncology nursing research in Mainland China and evaluate their content and quality. A systematic search was performed for published pediatric oncology research conducted by nurses in Mainland China (2008-2018), using 3 English databases and 3 Chinese databases. Included articles were evaluated using the Johns Hopkins Nursing Evidence-Based Practice Research Evidence Appraisal for strength and quality of evidence. A total of 133 articles were included: 106 in Chinese and 27 in English. Most studies showed level III evidence (88/133) and were rated as good quality (81/133). The most frequently researched topics were psychosocial care, clinical nursing practice, and psychometric testing, which accounted for 63.2% of all publications. Progress in pediatric oncology nursing research capacity among Mainland China is promising. To gain higher-quality evidence and make existing evidence transferable for nursing practice, optimization of specific research topics is still needed. In Mainland China, developing interventions to address the symptoms of children with cancer and caregivers' psychosocial issues based on local nursing research should be prioritized. Some synthesized findings of this review may serve as guidance for the future of pediatric oncology nursing science in similar settings.

Implications of Tumor Characteristics and Treatment Modality on Local Recurrence and Functional Outcomes in Children With Chest Wall Sarcoma: A Pediatric Surgical Oncology Research Collaborative Study.

To determine the impact of tumor characteristics and treatment approach on (1) local recurrence, (2) scoliosis development, and (3) patient-reported quality of life in children with sarcoma of the chest wall. Children with chest wall sarcoma require multimodal therapy including chemotherapy, surgery, and/or radiation. Despite aggressive therapy which places them at risk for functional impairment and scoliosis, these patients are also at significant risk for local recurrence. A multi-institutional review of 175 children (median age 13 years) with chest wall sarcoma treated at seventeen Pediatric Surgical Oncology Research Collaborative institutions between 2008 and 2017 was performed. Patient-reported quality of life was assessed prospectively using PROMIS surveys. The most common diagnoses were Ewing sarcoma (67%) and osteosarcoma (9%). Surgical resection was performed in 85% and radiation in 55%. A median of 2 ribs were resected (interquartile range = 1-3), and number of ribs resected did not correlate with margin status ( P = 0.36). Local recurrence occurred in 23% and margin status was the only predictive factor(HR 2.24, P = 0.039). With a median follow-up of 5 years, 13% developed scoliosis (median Cobb angle 26) and 5% required corrective spine surgery. Scoliosis was associated with posteriorrib resection (HR 8.43; P= 0.003) and increased number of ribs resected (HR 1.78; P = 0.02). Overall, patient-reported quality of life is not impaired after chest wall tumor resection. Local recurrence occurs in one-quarter of children with chest wall sarcoma and is independent of tumor type. Scoliosis occurs in 13% of patients, but patient-reported quality of life is excellent.

First person profile: Leslie L. Robison, PhD: A world-renowned epidemiologist, Dr. Robison played key roles in developing both the national Childhood Cancer Survivor Study and the St. Jude Lifetime Cohort Study.

First person profile: Leslie L. Robison, PhD: A world-renowned epidemiologist, Dr. Robison played key roles in developing both the national Childhood Cancer Survivor Study and the St. Jude Lifetime Cohort Study.

Informed consent for a paediatric oncology research trial

Informed consent for a paediatric oncology research trial

Pneumonectomy for Pediatric Tumors-a Pediatric Surgical Oncology Research Collaborative Study.

To describe utilization and long-term outcomes of pneumonectomy in children and adolescents with cancer. Pneumonectomy in adults is associated with significant morbidity and mortality. Little is known about the indications and outcomes of pneumonectomy for pediatric tumors. The Pediatric Surgical Oncology Research Collaborative (PSORC) identified pediatric patients <21 years of age who underwent pneumonectomy from 1990 to 2017 for primary or metastatic tumors at 12 institutions. Clinical information was collected; outcomes included operative complications, long-term function, recurrence, and survival. Univariate log rank, and multivariable Cox analyses determined factors associated with survival. Thirty-eight patients (mean 12 ± 6 yrs) were identified; median (IQR) follow-up was 19 (5-38) months. Twenty-six patients (68%) underwent pneumonectomy for primary tumors and 12 (32%) for metastases. The most frequent histologies were osteosarcoma (n = 6), inflammatory myofibroblastic tumors (IMT; n = 6), and pleuropulmonary blastoma (n = 5). Median postoperative ventilator days were 0 (0-1), intensive care 2 (1-3), and hospital 8 (5-16). Early postoperative complications occurred in 10 patients including 1 death. Of 25 (66%) patients alive at 1 year, 15 reported return to preoperative pulmonary status. All IMT patients survived while all osteosarcoma patients died during follow-up. On multivariable analysis, metastatic indications were associated with nonsurvival (HR = 3.37, P = 0.045). This is the largest review of children who underwent pneumonectomy for cancer. There is decreased procedure-related morbidity and mortality than reported for adults. Survival is worse with preoperative metastatic disease, especially osteosarcoma.

A multi-platform approach to promote clinical and research activities in nutrition and pediatric oncology

The increased risk of mortality and treatment-related toxicities among children with cancer who are also malnourished has prompted several efforts aimed at improving access to clinical care and promoting high-quality research in nutrition and pediatric oncology. The presence of strong nutrition programs in pediatric cancer units located in low- and middle-income countries where endemic rates of malnutrition, nutrient deficiencies and food insecurity are high makes this an even more crucial component of pediatric cancer care. This is especially true in Southeast Asia where nearly 30% of children in the region experience severe acute malnutrition at some point in childhood. Several global initiatives have been put forth to support pediatric cancer units in closing this disparity in nutritional care by increasing the availability of nutritionists knowledgeable in the provision of nutrition care to children with cancer. This report provides highlights of several collaborative, global initiatives aimed at further improving the quality of care, increasing education opportunities, and fostering multi-disciplinary collaborations in nutrition and research in pediatric oncology.

The Pediatric Oncology East and Mediterranean (POEM) group – A regional collaborative platform for childhood cancer healthcare professionals

The Pediatric Oncology East and Mediterranean (POEM) group was established in 2013 as a cooperative platform for physicians, scientists and healthcare professionals from institutions in the East and Mediterranean area with the goal of sharing experiences, initiating cooperative trials and establishing common strategies to achieve optimization of the care of pediatric oncology patients in the region. The mission of POEM group is to continuously improve pediatric oncology care, research, training and advocacy in the East and Mediterranean region by means of integrated teamwork. Our Vision is to be the leading pediatric oncology collaborative group in the East and Mediterranean Region. POEM group is currently at more than 550 registrants from 119 centers in 28 countries regionally. This review summarizes the structure and activities of POEM group since its inception, and highlights identified challenges and future plans.

The relevance of nutrition to pediatric oncology: A cancer control perspective

It is indisputable that adequate and appropriate nutrition is fundamental to the health, growth, and development of infants, children, and adolescents, including those with cancer. Nutrition has a role in most of the accepted components of the cancer control spectrum, from prevention through to palliation. The science of nutrigenomics, nutrigenetics, and bioactive foods (phytochemicals), and how nutrition affects cancer biology and cancer treatment, is growing. Nutritional epigenetics is giving us an understanding that there are possible primary prevention strategies for pediatric cancers, especially during conception and pregnancy, which need to be studied. Primary prevention of cancer in adults, such as colorectal cancer, should commence early in childhood, given the long gestation of nutritionally related cancers. Obesity avoidance is definitely a target for both pediatric and adult cancer prevention, commencing in childhood. There is now compelling evidence that the nutritional status of children with cancer, both overweight and underweight, does affect cancer outcomes. This is a potentially modifiable prognostic factor. Consistent longitudinal nutritional assessment of patients from diagnosis through treatment and long-term follow-up is required so that interventions can be implemented and evaluated. While improving, there remains a dearth of basic and clinical nutritional research in pediatric oncology. The perspective of evaluating nutrition as a cancer control factor is discussed in this article.

Optimization of percutaneous biopsy for diagnosis and pretreatment risk assessment of neuroblastoma.

Image-guided percutaneous core needle biopsy (PCNB) is increasingly utilized to diagnose solid tumors. The objective of this study is to determine whether PCNB is adequate for modern biologic characterization of neuroblastoma. A multi-institutional retrospective study was performed by the Pediatric Surgical Oncology Research Collaborative on children with neuroblastoma at 12 institutions over a 3-year period. Data collected included demographics, clinical details, biopsy technique, complications, and adequacy of biopsies for cytogenetic markers utilized by the Children's Oncology Group for risk stratification. A total of 243 children were identified with a diagnosis of neuroblastoma: 79 (32.5%) tumor excision at diagnosis, 94 (38.7%) open incisional biopsy (IB), and 70 (28.8%) PCNB. Compared to IB, there was no significant difference in ability to accurately obtain a primary diagnosis by PCNB (95.7%vs 98.9%, P=.314) or determine MYCN copy number (92.4%vs 97.8%, P=.111). The yield for loss of heterozygosity and tumor ploidy was lower with PCNB versus IB (56.1%vs 90.9%, P<.05; and 58.0%vs. 88.5%, P<.05). Complications did not differ between groups (2.9 % vs 3.3%, P=1.000), though the PCNB group had fewer blood transfusions and lower opioid usage. Efficacy of PCNB was improved for loss of heterozygosity when a pediatric pathologist evaluated the fresh specimen for adequacy. PCNB is a less invasive alternative to open biopsy for primary diagnosis and MYCN oncogene status in patients with neuroblastoma. Our data suggest that PCNB could be optimized for complete genetic analysis by standardized protocols and real-time pathology assessment of specimen quality.

Thirty-Day Outcomes following Pediatric Bone and Soft Tissue Sarcoma Surgery: A NSQIP Pediatrics Analysis.

Background Pediatric bone and soft tissue sarcomas are rare; therefore, national registries are essential tools for orthopedic oncology research. Past studies provide excellent data on long-term prognosis and survival trends but fail to examine treatment-specific morbidity. The aim of this study is to use a national registry to describe patient demographics, comorbidities, and adverse events in the first thirty days following surgical management of pediatric bone and soft tissue sarcomas. Methods A retrospective review of patients in the American College of Surgeons National Surgical Quality Improvement Program—Pediatrics database (NSQIP-P) was performed. The cohort was partitioned by tumor origin (bone versus soft tissue) and tumor location (axial versus appendicular). Results One-hundred ninety-two patients were identified. Bone sarcomas were more common (71.9%) and predominately appendicular (62.3%), while soft tissue sarcomas were predominately axial (77.8%). The overall complication rate was 8.9%. The most frequent etiologies were wound dehiscence (3.6%) and infectious complications such as surgical site infections (2.6%), pneumonia (1.6%), urinary tract infections (1.6%), and C. diff colitis (1.0%). Twenty-four percent of patients experienced bleeding requiring transfusion. The unplanned readmission rate was 12.5% (3.6% related to principle procedure), and the unplanned reoperation rate was 4.7% (4.2% related to principle procedure). The mortality rate was 1.0%. Neoadjuvant chemotherapy was associated with higher rates of wound dehiscence and infectious complications. There were no differences in adverse events with respect to tumor origin or location. Conclusion Approximately 1 in 11 pediatric patients will experience a complication in the first thirty days following surgery. However, perioperative mortality remains low. This study represents the first comprehensive review of pediatric bone and soft tissue sarcoma surgery in the NSQIP-P database. As the case volume of NSQIP-P continues to grow, NSQIP-P has the potential to become a powerful tool for pediatric orthopedic oncology research.