Longitudinal use of patient reported outcomes in pediatric leukemia and lymphoma reveals clinically relevant symptomatic adverse events.

Background: Leukemia and lymphoma (LL) are the most common cancer diagnoses of childhood with high survival rates, but not without impact on the child's functioning and quality of life. This study aimed to use patient-reported data to describe the symptomatic adverse event (AE) experiences among children with LL diagnoses.

Methods: Two hundred and fifty seven children and adolescents aged 7-18 years with a first LL diagnosis completed the Pediatric Patient-Reported version of the Common Terminology Criteria for Adverse Events (Ped-PRO-CTCAE) and Patient-Reported Outcome Measurement Information System (PROMIS) Pediatric measures before starting a treatment course (T1) and after the treatment (T2).

Profiles of Symptom Suffering and Functioning in Children and Adolescents Receiving Chemotherapy.

Background: Some children and adolescents receiving chemotherapy experience few symptom-related adverse events, whereas others experience multiple adverse events. If oncology nurses could identify patients likely to have pronounced chemotherapy-related adverse events, tailored supportive care could be matched to these patients' symptom burdens.

Objective: The aim of this study was to identify symptom profiles in children and adolescents before and after chemotherapy, and the sociodemographic and psychological factors associated with profile classification and change.

Profile Comparison of Patient-Reported and Proxy-Reported Symptoms in Pediatric Patients With Cancer Receiving Chemotherapy.

Importance: The variability in individual symptom and adverse event reporting between pediatric patient-reports and proxy-reports is widely reported. However, the question of whether symptom profiles based on reports from children with cancer and their caregivers are similar or disparate have not yet been studied.

Objective: To compare proxy symptom reports with patient self-reports to assess alignment.

Lack of Concordance in Symptomatic Adverse Event Reporting by Children, Clinicians, and Caregivers: Implications for Cancer Clinical Trials.

Purpose: To examine concordance in symptomatic adverse event (AE) grading using the Common Terminology Criteria for Adverse Events (CTCAE 4.0) for clinicians and its patient-reported outcome (PRO) versions for children (Ped-PRO-CTCAE) and caregivers (Ped-PRO-CTCAE [Caregiver]).

Methods: Children age 7-18 years with a first cancer diagnosis, their clinicians, and caregivers completed CTCAE-based measures before starting a treatment course (T1) and after the treatment (T2).

Pharmacogenetic and clinical predictors of ondansetron failure in a diverse pediatric oncology population.

Purpose: Chemotherapy-induced nausea and vomiting (CINV) is a frequently seen burdensome adverse event of cancer therapy. The 5-HT3 receptor antagonist ondansetron has improved the rates of CINV but, unfortunately, up to 30% of patients do not obtain satisfactory control. This study examined whether genetic variations in a relevant drug-metabolizing enzyme (CYP2D6), transporter (ABCB1), or receptor (5-HT3) were associated with ondansetron failure.

Patients, caregivers, and clinicians differ in performance status ratings: Implications for pediatric cancer clinical trials.

Background: The Lansky Play-Performance Scale (LPPS) is often used to determine a child's performance status for cancer clinical trial eligibility. Differences between clinician and caregiver LPPS ratings and their associations with child-reported functioning have not been evaluated.

Methods: Children aged 7 to 18 years who were receiving cancer treatment and their caregivers were recruited from 9 pediatric cancer centers.

Validation of the caregiver Pediatric Patient-Reported Outcomes Version of the Common Terminology Criteria for Adverse Events measure.

Background: Despite improvements in survival rates, cancer treatments have significant side effects that affect the quality of life of children and their families. When an ill child cannot self-report symptoms (eg, he or she is too ill), caregiver (parent) reporting becomes critical. This study evaluates the validity and reliability of the caregiver-reported Pediatric Patient-Reported Outcomes version of the Common Terminology Criteria for Adverse Events (Ped-PRO-CTCAE [Caregiver]) measure.

Subjective Toxicity Profiles of Children in Treatment for Cancer: A New Guide to Supportive Care?

Context: Children and adolescents with cancer experience treatment-related, subjective adverse events (AEs). Identifying distinct groups of patients who predictably experience higher prevalence of AEs could guide patient care.

Objectives: Study aims were to 1) identify groups of children and adolescents reporting AEs using the Pediatric Patient-Reported Outcomes version of the Common Terminology Criteria for Adverse Events (Ped-PRO-CTCAE); 2) determine whether demographic and clinical characteristics predict AE group membership; and 3) examine whether AE group membership was related to the distal outcome of psychological stress.

Agreement Between Child Self-report and Caregiver-Proxy Report for Symptoms and Functioning of Children Undergoing Cancer Treatment.

Importance: Adult patients are considered the best reporters of their own health-related quality of life (HRQOL). Self-report in pediatrics has been challenged by a limited array of valid measures. Caregiver report is therefore often used as a proxy for child report.

Validity and Reliability of the Pediatric Patient-Reported Outcomes version of the Common Terminology Criteria for Adverse Events.

Background: Patient-reported outcome (PRO) measurements linked to Common Terminology Criteria for Adverse Events (CTCAE) grading may improve symptom adverse event (AE) reporting in pediatric oncology trials. We evaluated construct validity, responsiveness, and test-retest reliability of the Ped-PRO-CTCAE measurement system for children and adolescents undergoing cancer care.

Methods: A total of 482 children and adolescents (7-18 years, 41.

Expanding construct validity of established and new PROMIS Pediatric measures for children and adolescents receiving cancer treatment.

Background: The Patient-Reported Outcomes Measurement Information System (PROMIS) Pediatric measures were designed to assess symptoms and functioning in children and adolescents. The study goal was to evaluate the validity and responsiveness of the PROMIS Pediatric measures in a diverse cohort of children with cancer.

Methods: Children (7-18 years) from nine pediatric oncology hospitals completed surveys at 72 hours preceding treatment initiation (T1) and at follow-up (T2) approximately 7 to 17 days later for chemotherapy, and 4+ weeks later for radiation.

Mapping child and adolescent self-reported symptom data to clinician-reported adverse event grading to improve pediatric oncology care and research.

Background: Clinicians are the standard source for adverse event (AE) reporting in oncology trials, despite the subjective nature of symptomatic AEs. The authors designed a pediatric patient-reported outcome (PRO) instrument for symptomatic AEs to support the National Cancer Institute's Common Terminology Criteria for Adverse Events (CTCAE) (the Pediatric PRO-CTCAE). The current study developed a standardized algorithm that maps all possible Pediatric PRO-CTCAE response patterns to recommended CTCAE grades to improve the accuracy of AE reporting in pediatric oncology trials.

A Pediatric Case of Transformed Mycosis Fungoides in a BRCA2 Positive Patient.

Cutaneous T-cell lymphomas are very rare in children. Although mycosis fungoides is the most common of these rare cutaneous T-cell lymphomas in children, transformation to an aggressive malignancy remains extremely uncommon, and there are no clear guidelines for clinical management in the pediatric population. In addition, the increased usage of next-generation sequencing for pediatric patients with unusual malignancies may result in the discovery of pathogenic germline mutations, though the association between these mutations and the patient's cancer is not always clear.

PROMIS pediatric measures validated in a longitudinal study design in pediatric oncology.

Purpose: This study assessed the responsiveness to change over time and theorized associations of Patient-Reported Outcomes Measurement Information System (PROMIS) pediatric measures in children and adolescents in treatment for cancer to determine measure readiness for use in cancer clinical trials.

Methods: We administered eight PROMIS (three symptom, two psychological, and three performance) pediatric short-form measures and the Symptom Distress Scale (SDS) to 96 pediatric oncology patients at three time points during a course of chemotherapy. We assessed responsiveness using paired t tests and generalized estimating equation (GEE) models, calculated standardized response mean (SRM) values for PROMIS measures, and examined scores over three data points (T1-T3).

Integrative Therapy Use for Management of Side Effects and Toxicities Experienced by Pediatric Oncology Patients.

Integrative Therapies (IT), otherwise known as Complementary and Alternative Medicine, are widely used among pediatric oncology patients, despite a paucity of available evidence. This review summarizes surveys that describe the prevalence of IT use by pediatric oncology patients, both during therapy and in survivorship, as well as the modalities being used. Additionally, the evidence that exists for specific treatments that appear to be efficacious in controlling specific symptoms is described.

Feasibility and acceptability of the patient-reported outcomes measurement information system measures in children and adolescents in active cancer treatment and survivorship.

Background: Patient-reported outcomes related to symptoms, function, and quality of life during and following cancer treatment can guide care for pediatric cancer patients. To advance the science of patient-reported outcomes, the National Institutes of Health funded the Patient-Reported Outcomes Measurement Information System (PROMIS).

Objective: The objective of this study was to assess feasibility and acceptability of the PROMIS pediatric measures, as defined by enrollment and attrition rates as well as missingness by measure, item, participant, and assessment time point.

Substitution of oral and intravenous thioguanine for mercaptopurine in a treatment regimen for children with standard risk acute lymphoblastic leukemia: a collaborative Children's Oncology Group/National Cancer Institute pilot trial (CCG-1942).

Background: Although mercaptopurine (MP) is conventionally used to treat childhood acute lymphoblastic leukemia (ALL), thioguanine (TG) is a more potent thiopurine in vitro and, when administered orally to patients, achieves cytotoxic drug concentrations in the cerebrospinal fluid (CSF). We performed a pilot study incorporating oral and 24-hr continuous IV infusion (CIVI) TG in children with newly diagnosed standard-risk ALL.

Procedure: Children with newly diagnosed standard-risk ALL (age 1-10 years, WBC<50 k) were eligible.

Plasma and cerebrospinal fluid pharmacokinetics of intravenous oxaliplatin, cisplatin, and carboplatin in nonhuman primates.

Purpose: Describe and compare the central nervous system pharmacology of the platinum analogues, cisplatin, carboplatin, and oxaliplatin and develop a pharmacokinetic model to distinguish the disposition of active drug from inert platinum species.

Experimental Design: Oxaliplatin (7 or 5 mg/kg), cisplatin (2 mg/kg), or carboplatin (10 mg/kg) was given i.v.