Targeted-Agent Continual Reassessment Method: A Novel Bayesian Enrichment Design for Phase I Trials of Molecularly Targeted Therapies.

Purpose: Novel therapies targeting specific genomic alterations are a promising treatment approach for relapsed/refractory cancer. Patients with specific alterations may be more likely to respond. Trial designs should maximize opportunities for such patients to enroll on these trials.

NOVEL USE OF NILE TILAPIA FISH () SKIN AS XENOGRAFT IN ZOOLOGICAL SPECIES.

Nile tilapia fish skin () has been increasingly used in human and veterinary medicine in xenografts for treatment of burn wounds. The objective of this study was to self-prepare the graft using a Brazilian patent (WO2017/035615 A1) as a guide and apply it to full-thickness wounds in zoological species ( = 4). The wounds, which were all characterized as contaminated wounds in which the entire dermal layer was missing, had all previously been surgically debrided and treated with traditional wound-healing techniques without success.

Predominance of in candidemia among patients with solid tumor cancer in Oman: A retrospective study.

Objectives: species frequently cause bloodstream infections; however, there is a lack of epidemiological studies on candidemia in Oman.

Methods: To address this, we conducted a retrospective study at Sultan Qaboos Comprehensive Cancer and Research Center from October 2021 to October 2023.

Results: Our study identified 27 episodes of candidemia among 26 patients with cancer, with an incidence of 4.

The Manchester Scoring System for predicting BRCA1/2 mutations underperforms in Arabic Omani breast cancer patients.

Risk assessment models that are applied to assess the lifetime risk of cancer and pathogenic variant risk are more commonly used in Western populations. Using these models, without validation, for non-Western populations has been questioned. This study aimed to evaluate the use and consistency of the Manchester Scoring System as a risk assessment model for the Omani population.

Clinicopathological Features and Outcomes of Endometrial Cancer: A single institution experience.

Objectives: This study aimed to report the demographic features, clinical presentation, pathological types and long-term outcomes of patients diagnosed with endometrial cancer (EC) in Oman. EC is the sixth most common cancer in women worldwide and the fifth most common cancer in women in Oman. Survival outcomes of EC have not been reported previously from Oman.

Analyzing Cancer Incidence Trends in Oman From 1996 to 2019: A Comprehensive Study of the National Cancer Annual Reports.

Purpose: Previous studies have reported that cancer incidence trends in Oman varied by tumor site and sex. No comprehensive analysis of all cancer sites had been reported. The objective of this study is to analyze cancer incidence trends in Oman and calculate the annual percent change (APC) in age-standardized rates (ASRs) for all-cancer and 61 individual cancer sites in Omani men and women from 1996 to 2019.

Deciphering Urogenital Cancers through Proteomic Biomarkers: A Systematic Review and Meta-Analysis.

Urogenital cancers, which include prostate, bladder, and kidney malignancies, exert a substantial impact on global cancer-related morbidity and mortality. Proteomic biomarkers, emerging as valuable tools, aim to enhance early detection, prognostic accuracy, and the development of personalized therapeutic strategies. This study undertook a comprehensive systematic review and meta-analysis of the existing literature investigating the role and potential of proteomic biomarkers in plasma, tissue, and urine samples in urogenital cancers.

The Prevalence and Patterns of Toxicity With Immune Checkpoint Inhibitors in Solid Tumors: A Real-World Experience From a Tertiary Care Center in Oman.

Introduction Immune checkpoint inhibitors (ICIs) have revolutionized the management of multiple cancers over the last decade. They work by employing the immune system and exhibiting activity over T cells resulting in immune upregulation. Despite their widespread use, they produce side effects that can limit their use.

A case of horizontal gaze palsy with progressive scoliosis.

Horizontal gaze palsy with progressive scoliosis is a rare entity with few cases in the literature. Despite the fact the patient will not present with typical symptoms of this syndrome, clinical suspicion should be raised particularly in terms of imaging findings. Imaging findings are characteristic to flag the possibility of this syndrome.

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.

Testicular leiomyosarcoma: A case report and literature review.

Introduction And Importance: Leiomyosarcoma is a malignant mesenchymal tumor derived from the smooth muscle, it represents approximately 7% of all soft tissue sarcomas. Male genitourinary leiomyosarcomas are rare (Abdullazade et al., 2013 [1]).

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.