Comparison of toxicity following single versus tandem autologous transplant regimens for pediatric medulloblastoma.

Background: Medulloblastoma outcomes have improved with craniospinal irradiation and chemotherapy, but such therapy has resulted in poor neurocognitive outcomes for young patients. Chemotherapy-only regimens with autologous transplant have been implemented with the intention of avoiding radiation. It is not yet known whether single or tandem transplantation is superior with respect to efficacy and/or safety.

Thinking ahead: Parents' worries about late effects of childhood cancer treatment.

Background: Many childhood cancersurvivors experience at least one late effect of treatment, and both late effects and persistent cancer-related worry can negatively impact quality of life in survivorship. Little is known about the prevalence or impact of parental worry about late effects early in treatment. This study evaluated parental perceived likelihood, impact, and worry about late effects of childhood cancer.

Disparities in pediatric psychosocial oncology utilization.

Background: Integratedbehavioral health models have been proposed as care delivery approaches to mitigate mental health disparities in primary care settings. However, these models have not yet been widely adopted or evaluated in pediatric oncology medical homes.

Methods: We conducted a retrospective cohort study of 394 children with newly diagnosed cancer at Dana-Farber/Boston Children's Cancer and Blood Disorders Center (DF/BCH) from April 2013 to January 2017.

Pyruvate kinase deficiency in children.

Background: Pyruvate kinase deficiency (PKD) is a rare, autosomal recessive red blood cell enzyme disorder, which leads to lifelong hemolytic anemia and associated complications from the disease and its management.

Methods: An international, multicenter registry enrolled 124 individuals younger than 18 years old with molecularly confirmed PKD from 29 centers. Retrospective and prospective clinical data were collected.

Retrospective evaluation of single patient investigational new drug (IND) requests in pediatric oncology.

Background: Single patient Investigational New Drug (IND) applications are one mechanism through which experimental therapies are accessed for children with cancer. The landscape of use, outcomes, and toxicity from single patient INDs remains unknown in pediatric oncology.

Methods: We performed a retrospective analysis of all single patient INDs requested and prescribed at a single institution between 1/1/2007 and 5/1/2019.

Building a Harmonized Datamart by Integrating Cross-Institutional Systems of Clinical, Outcome, and Genomic Data: The Pediatric Patient Informatics Platform ().

Purpose: Siloed electronic medical data limits utility and accessibility. At the Dana-Farber/Boston Children's Cancer and Blood Disorders Center, cross-institutional data were inconsistent and difficult to access. To unify data for clinical operations, administration, and research, we developed the Pediatric Patient Informatics Platform (), an integrated datamart harmonizing multiple source systems across two institutions into a common technology.

Feasibility and acceptability of the "Day 100 Talk": An interdisciplinary communication intervention during the first six months of childhood cancer treatment.

Background: Communication gaps arise early in the childhood cancer trajectory and may persist. The authors conducted a pilot study of the feasibility and acceptability of a communication intervention, the Day 100 Talk (D100). D100 involves an interprofessional family conference during initial months of treatment between oncologists, psychosocial clinicians, and parents, facilitated by a 3-part conversation tool.

Engaging Parents of Children Who Died From Cancer in Research on the Early Grief Experience.

Context: Bereaved parents provide an important perspective for improving care for patients and families throughout the illness and after a child's death. However, involvement of bereaved parents in research studies is fraught with concerns over inflicting psychological distress and issues with study recruitment. Data on research strategies to engage parents early in their bereavement are limited.

Off-label prescribing of targeted anticancer therapy at a large pediatric cancer center.

Background: Off-label drug prescribing is common in pediatric clinical medicine, though the extent and impact of this practice in pediatric oncology has not yet been characterized.

Methods: We completed a retrospective single-institution cohort study evaluating prevalence, characteristics, and clinical outcomes of off-label prescribing of 108 FDA-approved targeted anticancer drugs in patients < 30 years old treated for cancer from 2007 to 2017. Dosing strategies were adjusted for body size and compared to FDA-approved adult dosing regimen.

The pyruvate kinase (PK) to hexokinase enzyme activity ratio and erythrocyte PK protein level in the diagnosis and phenotype of PK deficiency.

Diagnosis of pyruvate kinase deficiency (PKD), the most common cause of hereditary non-spherocytic haemolytic anaemia, remains challenging in routine practice and no biomarkers for clinical severity have been characterised. This prospective study enrolled 41 patients with molecularly confirmed PKD from nine North American centres to evaluate the diagnostic sensitivity of pyruvate kinase (PK) enzyme activity and PK:hexokinase (HK) enzyme activity ratio, and evaluate the erythrocyte PK (PK-R) protein level and erythrocyte metabolites as biomarkers for clinical severity. In this population not transfused for ≥90 days before sampling, the diagnostic sensitivity of the PK enzyme assay was 90% [95% confidence interval (CI) 77-97%], whereas the PK:HK ratio sensitivity was 98% (95% CI 87-100%).

Parent Perceptions of Team-Delivered Care for Children With Advanced Cancer: A Report From the PediQUEST Study.

Context: Childhood cancer care is delivered by interprofessional health care teams; however, little is known about how parents perceive overall team-delivered care (TDC).

Objectives: We sought to describe parent perceptions of TDC and associated factors, including care rendered by individual clinicians, teamwork, information consistency, and patient and parent characteristics.

Methods: Cross-sectional surveys were distributed to parents of 104 children with recurrent/refractory cancer enrolled in a multisite symptom management trial.

An Anatomical Site and Genetic-Based Prognostic Model for Patients With Nuclear Protein in Testis (NUT) Midline Carcinoma: Analysis of 124 Patients.

Background: NUT midline carcinoma, renamed NUT carcinoma (NC), is an aggressive squamous cancer defined by rearrangement of the gene. Although a subset of patients can be cured, for the majority of patients the prognosis is grim. We sought to classify patients into risk groups based on molecular and clinicopathologic factors at the time of diagnosis.

Parental Considerations Regarding Cure and Late Effects for Children With Cancer.

Background: More than 80% of children with cancer become long-term survivors, yet most survivors experience late effects of treatment. Little is known about how parents and physicians consider late-effects risks against a potential survival benefit when making treatment decisions.

Methods: We used a discrete choice experiment to assess the importance of late effects on treatment decision-making and acceptable trade-offs between late-effects risks and survival benefit.

Comparison of Outcomes of Myeloablative Allogeneic Stem Cell Transplantation for Pediatric Patients with Bone Marrow Failure, Myelodysplastic Syndrome and Acute Myeloid Leukemia with and without Germline GATA2 Mutations.

Germline mutations in GATA2 are associated with an inherited predisposition to bone marrow failure (BMF), myelodysplastic syndromes (MDS), and acute myeloid leukemia (AML). Hematopoietic stem cell transplantation (HSCT) remains the only curative therapy. However, patients may be at an increased risk for transplant-related toxicity (TRT) and transplant-related mortality (TRM) due to their underlying disease biology.

Genotype-phenotype correlation and molecular heterogeneity in pyruvate kinase deficiency.

Pyruvate kinase (PK) deficiency is a rare recessive congenital hemolytic anemia caused by mutations in the PKLR gene. This study reports the molecular features of 257 patients enrolled in the PKD Natural History Study. Of the 127 different pathogenic variants detected, 84 were missense and 43 non-missense, including 20 stop-gain, 11 affecting splicing, five large deletions, four in-frame indels, and three promoter variants.

Risk Factors and Outcomes of Nonmelanoma Skin Cancer in Children and Young Adults.

Objective: To identify risk factors associated with nonmelanoma skin cancer (NMSC) occurrence and survival in children.

Study Design: This was a multicenter, retrospective, case-control study of patients <20 years of age diagnosed with NMSC between 1995 and 2015 from 11 academic medical centers. The primary outcome measure was frequency of cases and controls with predisposing genetic conditions and/or iatrogenic exposures, including chemotherapy, radiation, systemic immunosuppression, and voriconazole.

The relationship between household income and patient-reported symptom distress and quality of life in children with advanced cancer: A report from the PediQUEST study.

Background: Children with advanced cancer experience high symptom distress, which negatively impacts their health-related quality of life (HRQOL). To the authors' knowledge, the relationship between income and symptom distress and HRQOL is not well described.

Methods: The Pediatric Quality of Life and Symptoms Technology (PediQUEST) multisite clinical trial evaluated an electronic patient-reported outcome system to describe symptom distress and HRQOL in children with advanced cancer via repeated surveys.

The Impact of Intraoperative Electrocorticography on Seizure Outcome After Resection of Pediatric Brain Tumors: A Cohort Study.

Background: Intraoperative electrocorticography (ECoG) has been utilized in patients with tumor-associated seizures; however, its effectiveness for seizure control remains controversial.

Objective: To evaluate clinical outcomes in pediatric patients undergoing lesionectomy with or without ECoG.

Methods: Patients undergoing brain tumor resection at Boston Children's Hospital were examined retrospectively (2005-2014).

Clinical spectrum of pyruvate kinase deficiency: data from the Pyruvate Kinase Deficiency Natural History Study.

An international, multicenter registry was established to collect retrospective and prospective clinical data on patients with pyruvate kinase (PK) deficiency, the most common glycolytic defect causing congenital nonspherocytic hemolytic anemia. Medical history and laboratory and radiologic data were retrospectively collected at enrollment for 254 patients with molecularly confirmed PK deficiency. Perinatal complications were common, including anemia that required transfusions, hyperbilirubinemia, hydrops, and prematurity.

Provider Perspectives on Use of Medical Marijuana in Children With Cancer.

Background: Although medical marijuana (MM) may have utility in the supportive care of children with serious illness, it remains controversial. We investigated interdisciplinary provider perspectives on legal MM use in children with cancer.

Methods: We sent a 32-item, cross-sectional survey to 654 pediatric oncology providers in Illinois, Massachusetts, and Washington characterizing MM practices, knowledge, attitudes, and barriers.

Household material hardship in families of children post-chemotherapy.

Poverty is an important patient-reported outcome of therapy and a potential predictor of outcome disparities in pediatric cancer. We previously identified that nearly 30% of pediatric cancer families experience household material hardship (HMH), a concrete measure of poverty including food, energy, or housing insecurity, during the first 6 months of chemotherapy. We conducted a follow-up survey in a subcohort of these families at least 1 year off-therapy and found that 32% reported HMH in early survivorship.

Patterns and Outcomes of Care in Children With Advanced Heart Disease Receiving Palliative Care Consultation.

Context: Although access to subspecialty pediatric palliative care (PPC) is increasing, little is known about the role of PPC for children with advanced heart disease (AHD).

Objectives: The objective of this study was to examine features of subspecialty PPC involvement for children with AHD.

Methods: This is a retrospective single-institution medical record review of patients with a primary diagnosis of AHD for whom the PPC team was initially consulted between 2011 and 2016.

Influence of early phase clinical trial enrollment on patterns of end-of-life care for children with advanced cancer.

We conducted a retrospective cohort study of 125 pediatric oncology patients who died in 2010-2014 to explore how healthcare utilization, pediatric palliative care (PPC) receipt, and end-of-life care (EOLC) differed between patients enrolled in early phase clinical trials (EP) and those not enrolled (NEP). Baseline characteristics and healthcare utilization did not significantly differ between groups. EP patients received PPC consultation closer to death than NEP patients (median days before death = 58 [interquartile range = 16-84] vs.