Impact of the COVID-19 pandemic in childhood and adolescent cancer care in northern Tanzania: a cross-sectional study.

INTRODUCTION: The SARS-CoV-2 (COVID-19) pandemic has strained healthcare systems and presented unique challenges for children requiring cancer care, particularly in low- and middle-income countries. This study aimed to assess the impact of the COVID-19 pandemic on access to cancer care for children and adolescents in Northern Tanzania. METHODS: In this cross-sectional study, we assessed the demographic and clinical characteristics of 547 pediatric and adolescent cancer patients (ages 0-19 years old) between 2016 and 2022 using the population-based Kilimanjaro Cancer Registry (KCR). We categorized data into pre-COVID-19 (2016-2019) and COVID-19 (2020-2022) eras, and performed descriptive analyses of diagnostic, treatment, and demographic information. A secondary analysis was conducted on a subset of 167 patients with stage of diagnosis at presentation. RESULTS: Overall admissions nearly doubled during the pandemic (n = 190 versus 357). The variety of diagnoses attended at KCMC increased during the pandemic, with only five groups of diseases reported in 2016 to twelve groups of diseases in 2021. Most patients were diagnosed at a late stage (stage III or IV) across eras, with the proportion of under-five years old patients increasing late-diagnoses from 29.4% (before the pandemic), 52.8% (during the pandemic), when compared to the overall cohort. Around 95% of children in this age category reported late-stage diagnosis during the pandemic. Six out of the twelve cancer site groups also reported an increase in late-stage diagnosis. During the pandemic, the proportion of children receiving surgery increased from 15.8 to 30.8% (p < 0.001). CONCLUSION: Childhood and adolescent cancer care changed in Northern Tanzania during the COVID-19 pandemic, with increased late-stage diagnoses presentations among younger patients and the increased use of surgical therapies in the context of a growing practice. Understanding the impact of the COVID-19 pandemic on pediatric and adolescent cancer care can help us better adapt healthcare systems and interventions to the emerging needs of children and adolescents with cancer in the midst of a health crisis.

Contemporary trends in choledochal cyst excision: An analysis of the pediatric national surgical quality improvement program.

BACKGROUND: Choledochal cysts are rare congenital anomalies of the biliary tree that may lead to obstruction, chronic inflammation, infection, and malignancy. There is wide variation in the timing of resection, operative approach, and reconstructive techniques. Outcomes have rarely been compared on a national level. METHODS: We queried the Pediatric National Surgical Quality Improvement Program (NSQIP) to identify patients who underwent choledochal cyst excision from 2015 to 2020. Patients were stratified by hepaticoduodenostomy (HD) versus Roux-en-Y hepaticojejunostomy (RNYHJ), use of minimally invasive surgery (MIS), and age at surgery. We collected several outcomes, including length of stay (LOS), reoperation, complications, blood transfusions, and readmission rate. We compared outcomes between cohorts using nonparametric tests and multivariate regression. RESULTS: Altogether, 407 patients met the study criteria, 150 (36.8%) underwent RNYHJ reconstruction, 100 (24.6%) underwent MIS only, and 111 (27.3%) were less than one year old. Patients who underwent open surgery were younger (median age 2.31 vs. 4.25 years, p = 0.002) and more likely underwent RNYHJ reconstruction (42.7% vs. 19%, p = 0.001). On adjusted analysis, the outcomes of LOS, reoperation, transfusion, and complications were similar between the type of reconstruction, operative approach, and age. Patients undergoing RNYHJ had lower rates of readmission than patients undergoing HD (4.0% vs. 10.5%, OR 0.34, CI [0.12, 0.79], p = 0.02). CONCLUSIONS: In children with choledochal cysts, most short-term outcomes were similar between reconstructive techniques, operative approach, and age at resection, although HD reconstruction was associated with a higher readmission rate in this study. Clinical decision-making should be driven by long-term and biliary-specific outcomes.

Determinants of delayed childhood cancer care in low- and middle-income countries: A systematic review.

Early access to care is essential to improve survival rates for childhood cancer. This study evaluates the determinants of delays in childhood cancer care in low- and middle-income countries (LMICs) through a systematic review of the literature. We proposed a novel Three-Delay framework specific to childhood cancer in LMICs by summarizing 43 determinants and 24 risk factors of delayed cancer care from 95 studies. Traditional medicine, household income, lack of transportation, rural population, parental education, and travel distance influenced most domains of our framework. Our novel framework can be used as a policy tool toward improving cancer care and outcomes for children in LMICs.

Defining Feasibility as a Criterion for Essential Surgery: A Qualitative Study with Global Children's Surgery Experts.

BACKGROUND: The Disease Control Priorities (DCP-3) group defines surgery as essential if it addresses a significant burden, is cost-effective, and is feasible-yet the feasibility component remains largely unexplored. The aim of this study was to develop a precise definition of feasibility for essential surgical procedures for children. METHODS: Four online focus group discussions (FGDs) were organized among 19 global children's surgery providers with experience of working in low- and lower-middle-income countries (LMICs), representing 10 countries. FGDs were transcribed verbatim, and qualitative data analysis was performed. Codes, categories, themes, and subthemes were identified. RESULTS: Six determinants of feasibility were identified, including: adequate human resources; adequate material resources; procedure and disease complexity; team commitment and understanding of their setting; timely access to care; and the ability to monitor and achieve good outcomes. Factors unique to feasibility of children's surgery included children's right to health and their reliance on adults for accessing safe and timely care; the need for specialist workforce; and children's unique perioperative care needs. FGD participants reported a greater need for task-sharing and shifting, creativity, and adaptability in resource-limited settings. Resource availability was seen to have a direct impact on decision-making and prioritization, e.g., saving a life versus achieving the best outcome. CONCLUSIONS: The identification of a precise definition of feasibility serves as a pivotal step in identifying a list of essential surgical procedures for children, which would serve as indicators of institutional surgical capacity for this age group.

Factors contributing to delayed childhood cancer care in low- and middle-income countries: A systematic review protocol.

BACKGROUND: Significant disparities exist for timely access to cancer care for children, with the highest disparities in low- and middle-income countries (LMICs). This study aims to conduct a systematic review that identifies the factors contributing to delayed care of childhood cancers in LMICs. METHODS: We will conduct a systematic review with search strings compliant with the PICO framework: (1) the Population-children (aged 0-18 years) from LMICs; (2) the Exposure-factors contributing to timely childhood cancer care; (3) the Outcome-delays in childhood cancer care. DISCUSSION: Our study is an essential step to guide strategic interventions to assess the myriad of factors that prevent children from accessing timely cancer care in LMICs. The results will be submitted for publication in a peer-reviewed journal and shared with institutions related to the field.

Modeling the Scale-up of Surgical Services for Children with Surgically Treatable Congenital Conditions in Somaliland.

BACKGROUND: Congenital conditions comprise a significant portion of the global burden of surgical conditions in children. In Somaliland, over 250,000 children do not receive required surgical care annually, although the estimated costs and benefits of scale-up of children's surgical services to address this disease burden is not known. METHODS: We developed a Markov model using a decision tree template to project the costs and benefits of scale-up of surgical care for children across Somaliland. We used a proxy set of congenital anomalies across Somaliland to estimate scale-up costs using three different scale-up rates. The cost-effectiveness ratio and net societal monetary benefit were estimated using these models, supported by disability weights in existing literature. RESULTS: Overall, we found that scale-up of surgical services at an aggressive rate (22.5%) over a 10-year time horizon is cost effective. Although the scale-up of surgical care for most conditions in the proxy set was cost effective, scale-up of hydrocephalus and spina bifida are not as cost effective as other conditions. CONCLUSIONS: Our analysis concludes that it is cost effective to scale-up surgical services for congenital anomalies for children in Somaliland.

Defining Surgical Workforce Density Targets to Meet Child and Neonatal Mortality Rate Targets in the Age of the Sustainable Development Goals: A Global Cross-Sectional Study.

OBJECTIVES: To reduce preventable deaths of newborns and children, the United Nations set a target rate per 1000 live births of 12 for neonatal mortality (NMR) and 25 for under-5 mortality (U5MR). The purpose of this paper is to define the minimum surgical workforce needed to meet these targets and evaluate the relative impact of increasing surgeon, anesthesia, and obstetrician (SAO) density on reducing child mortality. METHODS: We conducted a cross-sectional study of 192 countries to define the association between surgical workforce density and U5MR as well as NMR using unadjusted and adjusted B-spline regression, adjusting for common non-surgical causes of childhood mortality. We used these models to estimate the minimum surgical workforce to meet the sustainable development goals (SDGs) for U5MR and NMR and marginal effects plots to determine over which range of SAO densities the largest impact is seen as countries scale-up SAO workforce. RESULTS: We found that increased SAO density is associated with decreased U5MR and NMR (P < 0.05), adjusting for common non-surgical causes of child mortality. A minimum SAO density of 10 providers per 100,000 population (95% CI: 7-13) is associated with an U5MR of < 25 per 1000 live births. A minimum SAO density of 12 (95% CI: 9-20) is associated with an NMR of < 12 per 1000 live births. The maximum decrease in U5MR, on the basis of our adjusted B-spline model, occurs from 0 to 20 SAO per 100,000 population. The maximum decrease in NMR based on our adjusted B-spline model occurs up from 0 to 18 SAO, with additional decrease seen up to 80 SAO. CONCLUSIONS: Scale-up of the surgical workforce to 12 SAO per 100,000 population may help health systems meet the SDG goals for childhood mortality rates. Increases in up to 80 SAO/100,000 continue to offer mortality benefit for neonates and would help to achieve the SDGs for neonatal mortality reduction.

Potentially Avertable Child Mortality Associated with Surgical Workforce Scale-up in Low- and Middle-Income Countries: A Global Study.

BACKGROUND: Expansion of access to surgical care can improve health outcomes, although the impact that scale-up of the surgical workforce will have on child mortality is poorly defined. In this study, we estimate the number of child deaths potentially avertable by increasing the surgical workforce globally to meet targets proposed by the Lancet Commission on Global Surgery. METHODS: To estimate the number of deaths potentially avertable through increases in the surgical workforce, we used log-linear regression to model the association between surgeon, anesthetist and obstetrician workforce (SAO) density and surgically amenable under-5 mortality rate (U5MR), infant mortality rate (IMR), and neonatal mortality rate (NMR) for 192 countries adjusting for potential confounders of childhood mortality, including the non-surgical workforce (physicians, nurses/midwives, community health workers), gross national income per capita, poverty rate, female literacy rate, health expenditure per capita, percentage of urban population, number of surgical operations, and hospital bed density. Surgically amenable mortality was determined using mortality estimates from the UN Inter-agency Group for Child Mortality Estimation adjusted by the proportion of deaths in each country due to communicable causes unlikely to be amenable to surgical care. Estimates of mortality reduction due to upscaling surgical care to support the Lancet Commission on Global Surgery (LCoGS) minimum target of 20-40 SAO/100,000 were calculated accounting for potential increases in surgical volume associated with surgical workforce expansion. RESULTS: Increasing SAO workforce density was independently associated with lower surgically amenable U5MR as well as NMR (p < 0.01 for each model). When accounting for concomitant increases in surgical volume, scale-up of the surgical workforce to 20-40 SAO/100,000 could potentially prevent between 262,709 (95% CI 229,643-295,434) and 519,629 (465,046-573,919) under 5 deaths annually. The majority (61%) of deaths averted would be neonatal deaths. CONCLUSION: Scale up of surgical workforce may substantially decrease childhood mortality rates around the world. Our analysis suggests that scale-up of surgical delivery through increase in the SAO workforce could prevent over 500,000 children from dying before the age of 5 annually. This would represent significant progress toward meeting global child mortality reduction targets.

LONG-TERM HEMATOLOGIC AND CLINICAL OUTCOMES OF SPLENECTOMY IN CHILDREN WITH HEREDITARY SPHEROCYTOSIS AND SICKLE CELL DISEASE.

Background: Total splenectomy (TS) and partial splenectomy (PS) are used for children with congenital hemolytic anemia (CHA), although the long-term outcomes of these procedures are poorly defined. This report describes long-term outcomes of children with CHA requiring TS or PS. Procedure: We collected data from children ages 2-17 with hereditary spherocytosis (HS) or sickle cell disease (SCD) requiring TS or PS from 1996 to 2016 from 14 sites in the Splenectomy in Congenital Hemolytic Anemia (SICHA) consortium using a prospective, observational patient registry. We summarized hematologic outcomes, clinical outcomes, and adverse events to 5 years after surgery. Hematologic outcomes were compared using mixed effects modeling. Results: Over the study period, 110 children with HS and 97 children with SCD underwent TS or PS. From preoperatively compared to postoperatively, children with HS increased their mean hemoglobin level by 3.4 g/dL, decreased their mean reticulocyte percentage by 6.7%, and decreased their mean bilirubin by 2.4mg/dL. Hematologic improvements and improved clinical outcomes were sustained over 5 years of follow-up. For children with SCD, there was no change in hemoglobin after PS or TS following surgery, although all clinical outcomes were improved. Over 5 years, there was one child with HS and 5 children with SCD who developed post-splenectomy sepsis. Conclusions: For children with HS, there are excellent long-term hematologic and clinical outcomes following either PS or TS. Although hemoglobin levels do not change after TS or PS in SCD, the long-term clinical outcomes for children with SCD are favorable.

Pancreaticoduodenectomy for the treatment of pancreatic neoplasms in children: A Pediatric Surgical Oncology Research Collaborative study.

BACKGROUND: To better characterize short-term and long-term outcomes in children with pancreatic tumors treated with pancreaticoduodenectomy (PD). METHODS: Patients 21 years of age or younger who underwent PD at Pediatric Surgical Oncology Collaborative (PSORC) hospitals between 1990 and 2017 were identified. Demographic, clinical information, and outcomes (operative complications, long-term pancreatic function, recurrence, and survival) were collected. RESULTS: Sixty-five patients from 18 institutions with a median age of 13 years (4 months-22 years) and a median (IQR) follow-up of 2.8 (4.3) years were analyzed. Solid pseudopapillary tumor of the pancreas (SPN) was the most common histology. Postoperative complications included pancreatic leak in 14% (n = 9), delayed gastric emptying in 9% (n = 6), marginal ulcer in one patient, and perioperative (30-day) death due to hepatic failure in one patient. Pancreatic insufficiency was observed in 32% (n = 21) of patients, with 23%, 3%, and 6% with exocrine, or endocrine insufficiencies, or both, respectively. Children with SPN and benign neoplasms all survived. Overall, there were 14 (22%) recurrences and 11 deaths (17%). Univariate analysis revealed non-SPN malignant tumor diagnosis, preoperative vascular involvement, intraoperative transfusion requirement, pathologic vascular invasion, positive margins, and need for neoadjuvant chemotherapy as risk factors for recurrence and poor survival. Multivariate analysis only revealed pathologic vascular invasion as a risk factor for recurrence and poor survival. CONCLUSION: This is the largest series of pediatric PD patients. PD is curative for SPN and benign neoplasms. Pancreatic insufficiency is the most common postoperative complication. Outcome is primarily associated with histology.

Barriers to Surgical Care Among Children in Somaliland: An Application of the Three Delays Framework.

BACKGROUND: There are complex barriers that increase delays to surgical care in low- and middle-income countries, particularly among the vulnerable population of children. Understanding these barriers to surgical care can result in targeted and strategic intervention efforts to improve care for children. The three-delay model is a widely used framework in global health for evaluating barriers associated with seeking (D1), reaching (D2), and receiving health care (D3). The goal of our study is to evaluate reasons for delays in the surgical care for children in Somaliland using the three-delay framework. METHODS: Data were collected in a cross-sectional study in Somaliland from 1503 children through a household survey. Among children with a surgical need, we quantified the number of children seeking, reaching, and receiving care along the surgical care continuum, according to the three-delay framework. We evaluated predictors of the three delays through a multivariate logistic regression model, including the child's age, gender, village type, household income level, region, and household size. RESULTS: Of the 196 children identified with a surgical condition, 50 (27.3%) children had a delay in seeking care (D1), 28 (20.6%) children had a delay in reaching care (D2), and 84 (71.2%) children had a delay in receiving care (D3), including 10 children who also experienced D1 and D2. The main reasons cited for D1 included seeking a traditional healthcare provider, while lack of money and availability of care were main reasons cited for D2. Significant predictors for delays included household size for D1 and D3 and condition type and region for D2. CONCLUSION: Children in Somaliland experience several barriers to surgical care along the entire continuum of care, allowing for policy guidance tailored to specific local challenges and resources. Since delays in surgical care for children can substantially impact the effectiveness of surgical interventions, viewing delays in surgical care under the lens of the three-delay framework can inform strategic interventions along the pediatric surgical care continuum, thereby reducing delays and improving the quality of surgical care for children.

Implementation Analysis of a Perioperative Patient Safety Program in Guatemala.

BACKGROUND: The implementation of programs to improve patient safety remains challenging in low- and middle-income countries. The goal of our study was to define the barriers and facilitators to implementation of a perioperative patient safety program in Guatemala. MATERIAL AND METHODS: We conducted semi-structured interviews with 16 staff pre-intervention and a follow-up focus group discussion 1 year later in the perioperative department at the Roosevelt Hospital in Guatemala. We performed qualitative thematic analysis to identify barriers and facilitators to the implementation process, with analysis guided by the Consolidated Framework for Implementation Research. RESULTS: We found several dominant themes affecting implementation of a patient safety program. Implementation facilitators included strong prioritization of patient needs, program compatibility with existing workflow, and staff attributes. Barriers included a lack of knowledge about patient safety, limited resources, limited leadership engagement, and lack of formal implementation leaders. Several program modifications were made to enhance successful implementation iteratively during the implementation process. DISCUSSION: Our analysis highlights several dominant themes which affect the implementation of a perioperative safety program in Guatemala. Understanding the barriers and facilitators to implementation during program deployment allows for program modification and improvement of the implementation process itself.

Waiting Too Long: The Contribution of Delayed Surgical Access to Pediatric Disease Burden in Somaliland.

BACKGROUND: Delayed access to surgical care for congenital conditions in low- and middle-income countries is associated with increased risk of death and life-long disabilities, although the actual burden of delayed access to care is unknown. Our goal was to quantify the burden of disease related to delays to surgical care for children with congenital surgical conditions in Somaliland. METHODS: We collected data from medical records on all children (n = 280) receiving surgery for a proxy set of congenital conditions over a 12-month time period across all 15 surgically equipped hospitals in Somaliland. We defined delay to surgical care for each condition as the difference between the ideal and the actual ages at the time of surgery. Disability-adjusted life years (DALYs) attributable to these delays were calculated and compared by the type of condition, travel distance to care, and demographic characteristics. RESULTS: We found long delays in surgical care for these 280 children with congenital conditions, translating to a total of 2970 attributable delayed DALYs, or 8.4 avertable delayed DALYs per child, with the greatest burden among children with neurosurgical and anorectal conditions. Over half of the families seeking surgical care had to travel over 2 h to a surgically equipped hospital in the capital city of Hargeisa. CONCLUSIONS: Children with congenital conditions in Somaliland experience substantial delays to surgical care and travel long distances to obtain care. Estimating the burden of delayed surgical care with avertable delayed DALYs offers a powerful tool for estimating the costs and benefits of interventions to improve the quality of surgical care.

Provision of Surgical Care for Children Across Somaliland: Challenges and Policy Guidance.

BACKGROUND: Existing data suggest a large burden of surgical conditions in low- and middle-income countries (LMICs). However, surgical care for children in LMICs remains poorly understood. Our goal was to define the hospital infrastructure, workforce, and delivery of surgical care for children across Somaliland and provide policy guidance to improve care. METHODS: We used two established hospital assessment tools to assess infrastructure, workforce, and capacity at all hospitals providing surgical care for children across Somaliland. We collected data on all surgical procedures performed in children in Somaliland between August 2016 and July 2017 using operative logbooks. RESULTS: Data were collected from 15 hospitals, including eight government, five for-profit, and two not-for-profit hospitals. Children represented 15.9% of all admitted patients, and pediatric surgical interventions comprised 8.8% of total operations. There were 0.6 surgical providers and 1.2 anesthesia providers per 100,000 population. A total of 1255 surgical procedures were performed in children in all hospitals in Somaliland over 1 year, at a rate of 62.4 surgical procedures annually per 100,000 children. Care was concentrated at private hospitals within urban areas, with a limited number of procedures for many high-burden pediatric surgical conditions. CONCLUSIONS: We found a profound lack of surgical capacity for children in Somaliland. Hospital-level surgical infrastructure, workforce, and care delivery reflects a severely resource-constrained health system. Targeted policy to improved essential surgical care at local, regional, and national levels is essential to improve the health of children in Somaliland.

Developing a national patient safety plan in Guatemala.

OBJECTIVE: Patient safety is challenging for health systems around the world, particularly in low-and middleincome countries such as Guatemala. The goal of this report is to summarize a strategic planning process for a national patient safety plan in Guatemala. METHODS: This strategic planning process involved multiple stakeholders, including representatives of the Guatemala Ministry of Health and Social Assistance, medical leadership from across the public health system, and academic experts from Guatemala and the United States of America. We used mixed methods (quantitative and qualitative surveys) and a nominal group technique at a national symposium to prioritize patient safety challenges across Guatemala, and subsequent meetings to develop a national patient safety plan. RESULTS: This national patient safety plan outlines four domains to advance patient safety across the public hospital system over a five-year period in Guatemala: leadership and governance, training and awareness, safety culture, and outcome metrics. For each domain, we developed a set of goals, activities, outputs, and benchmarks to be overseen by the Ministry of Health. CONCLUSIONS: With this national patient safety plan, Guatemala has made a long-term commitment to improving patient safety across the public hospital system of Guatemala. Future efforts will require its extension to all levels of the Guatemalan health system.

Comparing oncologic outcomes after minimally invasive and open surgery for pediatric neuroblastoma and Wilms tumor.

BACKGROUND: Minimally invasive surgery (MIS) has been widely adopted for common operations in pediatric surgery; however, its role in childhood tumors is limited by concerns about oncologic outcomes. We compared open and MIS approaches for pediatric neuroblastoma and Wilms tumor (WT) using a national database. METHODS: The National Cancer Data Base from 2010 to 2012 was queried for cases of neuroblastoma and WT in children ≤21 years old. Children were classified as receiving open or MIS surgery for definitive resection, with clinical outcomes compared using a propensity matching methodology (two open:one MIS). RESULTS: For children with neuroblastoma, 17% (98 of 579) underwent MIS, while only 5% of children with WT (35 of 695) had an MIS approach for tumor resection. After propensity matching, there was no difference between open and MIS surgery for either tumor for 30-day mortality, readmissions, surgical margin status, and 1- and 3-year survival. However, in both tumors, open surgery more often evaluated lymph nodes and had larger lymph node harvest. CONCLUSION: Our retrospective review suggests that the use of MIS appears to be a safe method of oncologic resection for select children with neuroblastoma and WT. Further research should clarify which children are the optimal candidates for this approach.

Disability Weights for Pediatric Surgical Procedures: A Systematic Review and Analysis.

BACKGROUND: Metrics to measure the burden of surgical conditions, such as disability weights (DWs), are poorly defined, particularly for pediatric conditions. To summarize the literature on DWs of children's surgical conditions, we performed a systematic review of disability weights of pediatric surgical conditions in low- and middle-income countries (LMICs). METHOD: For this systematic review, we searched MEDLINE for pediatric surgery cost-effectiveness studies in LMICs, published between January 1, 1996, and April 1, 2017. We also included DWs found in the Global Burden of Disease studies, bibliographies of studies identified in PubMed, or through expert opinion of authors (ES and HR). RESULTS: Out of 1427 publications, 199 were selected for full-text analysis, and 30 met all eligibility criteria. We identified 194 discrete DWs published for 66 different pediatric surgical conditions. The DWs were primarily derived from the Global Burden of Disease studies (72%). Of the 194 conditions with reported DWs, only 12 reflected pre-surgical severity, and 12 included postsurgical severity. The methodological quality of included studies and DWs for specific conditions varied greatly. INTERPRETATION: It is essential to accurately measure the burden, cost-effectiveness, and impact of pediatric surgical disease in order to make informed policy decisions. Our results indicate that the existing DWs are inadequate to accurately quantify the burden of pediatric surgical conditions. A wider set of DWs for pediatric surgical conditions needs to be developed, taking into account factors specific to the range and severity of surgical conditions.

Gallbladder abnormalities in children with metachromatic leukodystrophy.

BACKGROUND: Metachromatic leukodystrophy (MLD) is a lysosomal storage disease that leads to neurological deterioration and visceral involvement, including sulphatide deposition in the gallbladder wall. Using our institution's extensive experience in treating MLD, we examined the incidence of gallbladder abnormalities in the largest cohort of children with MLD to date. METHODS: We conducted a retrospective review of all children with MLD, adrenoleukodystrophy (ALD), or Krabbe disease who underwent hematopoietic stem cell transplantation (HSCT) at our institution between 1994 and 2015. Baseline characteristics and unadjusted outcomes were compared using the Kruskal-Wallis test for continuous variables and Pearson χ2 test for categorical variables, with significance defined as P < 0.05. RESULTS: In total, 87 children met study criteria: 29 children with MLD and 58 children with ALD or Krabbe disease. Children with MLD were more likely to demonstrate gallbladder abnormalities on imaging, both before HSCT (41.4% versus 5.2%, P < 0.001) and after HSCT (75.9% versus 41.4%, P = 0.002). Consequently, a larger proportion of children with MLD underwent surgical or interventional management of biliary disease (10.3% versus 3.4%, P = 0.03). CONCLUSIONS: Children with MLD have a significantly greater incidence of gallbladder abnormalities than children with other lysosomal storage diseases. Biliary disease should be considered in children with MLD who develop abdominal pain, and cholecystectomy should be considered for persistent, symptomatic gallbladder abnormalities.

Renal medullary carcinoma: A national analysis of 159 patients.

BACKGROUND: Renal medullary carcinoma (RMC) is an aggressive malignancy seen predominantly in young males with sickle cell trait. RMC is poorly understood, with fewer than 220 cases described in the medical literature to date. We used a large national registry to define the typical presentation, treatments, and outcomes of this rare tumor. METHODS: The National Cancer Database was queried for patients under 40 years of age diagnosed with RMC from 1998 to 2011. An analysis of patient and tumor characteristics, treatment details, and overall survival (OS) was undertaken, and factors associated with mortality were identified using multivariable regression analysis. RESULTS: In total, 159 patients with RMC were identified, of whom a majority were male (71%), African American (87%), and had metastatic disease (71%). Median tumor size was 6 cm and median survival was 7.7 months. Most patients underwent surgery (60%) and chemotherapy (65%). Few patients received radiation (12%). Patients with metastatic disease had a significantly worse median survival (4.7 vs. 17.8 months, P < 0.001) and were less likely to receive surgery (42% vs. 91%, P < 0.001). Age and tumor size did not appear to impact OS. CONCLUSION: In the largest cohort to date of patients with RMC, we found a dismal median survival of less than 8 months. Age and tumor size were not associated with OS. Metastatic disease at presentation was the main negative prognostic indicator in RMC and was present in a majority of patients at the time of diagnosis.

Children Receiving Hematopoietic Stem Cell Transplantation are at Increased Risk of Onychocryptosis Requiring Surgical Management.

PURPOSE: At our institution, a high proportion of children with onychocryptosis (ingrown toenail) requiring surgical intervention were noted to have a history of hematopoietic stem cell transplantation (HSCT). We analyzed the characteristics of patients who underwent surgical intervention for onychocryptosis and examined our institutional HSCT database to determine if an association exists between onychocryptosis and HSCT. MATERIALS AND METHODS: Surgical cases for onychocryptosis performed from 2000 to 2012 were identified. Nine demographic, clinical, and perioperative variables for both patients with and without prior HSCT were assessed. In a separate analysis, the institutional HSCT database was then queried to identify the prevalence and clinical characteristics associated with onychocryptosis after HSCT. RESULTS: We identified 17 children who had undergone surgical management of onychocryptosis, of which 8 (47.1%) had previous HSCT. Children who had undergone HSCT had an aggressive form of onychocryptosis with 50.0% having bilateral great toe and nail edge involvement and 37.5% having a recurrence. In HSCT cohort analysis of 1069 children, 91 (8.5%) had onychocryptosis. Male sex, non-black race, acute graft versus host disease, and increasing age at transplantation were independently associated with onychocryptosis. CONCLUSIONS: HSCT is strongly associated with onychocryptosis requiring surgical intervention. Children with a history of HSCT may also have more aggressive toenail disease, with higher rates of surgical intervention, bilateral ingrown toenails, recurrence, and need for return to the operating room. Clinicians should perform careful screening and early treatment in these patients.