Selumetinib for symptomatic, inoperable plexiform neurofibromas in children with neurofibromatosis type 1: A national real-world case series.

Neurofibromatosis type 1-associated plexiform neurofibromas can cause debilitating symptoms and be life threatening. Treatment options are limited, given their tendency to regrow following surgery and their propensity to transform into malignant tumours following radiation. Selumetinib is an oral selective inhibitor of RAS-mitogen-activated protein kinase (MAPK) 1 and 2, which has shown efficacy for tumour shrinkage/stabilisation and symptom improvement. We report a national case series of 19 children treated with selumetinib. All patients experienced symptom improvement or stabilisation with an acceptable toxicity profile, including those patients previously treated with trametinib. This real-world experience confirms previous trials showing significant clinical benefit for this patient population.

Indirect costs associated with out-of-country referral for proton therapy: a survey of adult and pediatric patients in Alberta, Canada.

BACKGROUND: Patients in Alberta, Canada are referred to the United States (US) for proton treatment. The Alberta Ministry of Health pays for the proton treatment and the cost of flights to and from the United States. This study aimed to determine the out-of-pocket expenses incurred by patients or patients' families. METHODS: An electronic survey was sent to 59 patients treated with proton therapy between January 2008 and September 2019. Survey questions asked about expenses related to travel to the US and those incurred while staying in the US, reimbursement of expenses, and whether any time away from work was paid or unpaid leave. RESULTS: Seventeen respondents (response rate, 29%) reported expenses of flights for family members (mean, CAD 1886; range CAD 0-5627), passports/visas and other travel costs (mean, CAD 124; range CAD 0-546), accommodation during travel to the US (mean, CAD 50; range CAD 0-563), food during travel to the US (mean, CAD 89; range CAD 0-338), accommodation in the US (rented home/apartment mean, CAD 7394; range CAD 3075-13,305; hotel mean, CAD 4730; range CAD 3564-5895; other accommodation mean CAD 2660; range CAD 0-13,842), transportation in the US (car mean, CAD 2760; range CAD 0-7649; bus/subway mean, CAD 413; range CAD 246-580), and food in the US (mean, CAD 2443; range 0-6921). Expenses were partially reimbursed or covered by not-for-profit organizations or government agencies for some patients (35%). Patients missed a mean of 59 days of work; accompanying family members missed an average of 34 days. For 29% this time away from work was paid, but unpaid for 71% of respondents. CONCLUSIONS: Multiple factors contributed to the expenses incurred including age of the patient, number of accompanying individuals, available accommodation, mode of transportation within the US, and whether the patient qualified for financial support. Added to this burden is the potential loss of wages for time away from work. The study showed a large variation in indirect costs for each family and supports actively seeking more opportunities for financial support for families with children with cancer.

Evaluation of enoxaparin dosing requirements in infants and children. Better dosing to achieve therapeutic levels.

Increasing the starting dose of enoxaparin results in the early achievement of therapeutic anti-factor Xa levels in children receiving enoxaparin which is critical for effective therapy and the reduction of venipunctures. The aim of this study was: i) to determine the enoxaparin dose required to achieve therapeutic anti-factor Xa levels in infants and children, and ii) to establish whether increasing the starting dose of enoxaparin influenced the time required to reach the therapeutic range and the number of venipunctures required for dose-adjustment, and iii) the radiographic outcome of the thrombosis, where applicable. A retrospective chart review of children who received enoxaparin was carried out at the Stollery Children's Hospital, Edmonton, Alberta, Canada. Patients treated with standard-dose enoxaparin (1.5 mg/kg for children < or =3 months of age, 1.0 mg/kg for children > or =3 months of age), were compared with children who received a higher initial starting dose of enoxaparin (1.7 mg/kg for children > or =3 months of age, 1.2 mg/kg for children > or =3 months of age). Infants <3 months required an enoxaparin dose of 1.83 mg/kg, and those who received an increased initial enoxaparin dose resulted in faster attainment of therapeutic anti-factor Xa levels requiring significantly fewer venipunctures. Similarly, infants > or =3-12 months, 1-5 years, and 6-18 years, require enoxaparin 1.48 mg/kg, 1.23 mg/kg and 1.13 mg/kg, respectively, in order to achieve a therapeutic anti-factor Xa level. In conclusion, increasing the starting dose of enoxaparin may result in more rapid attainment of therapeutic range with fewer venipunctures, dose adjustments, and without an increase in adverse events.

Accuracy of the CoaguChek XS for point-of-care international normalized ratio (INR) measurement in children requiring warfarin.

Point-of-care INR (POC INR) meters can provide a safe and effective method for monitoring oral vitamin K antagonists (VKAs) in children. Stollery Children's Hospital has a large POC INR meter loan program for children requiring oral VKAs. Our protocol requires that POC INR results be compared to the standard laboratory INR for each child on several consecutive tests to ensure accuracy of CoaguChek XS (Roche Diagnostics, Basel Switzerland) meter. It was the objective of the study to determine the accuracy of the CoaguChek XS by comparing whole blood INR results from the CoaguChek XS to plasma INR results from the standard laboratory in children. POC INR meter validations were performed on plasma samples from two time points from 62 children receiving warfarin by drawing a venous blood sample for laboratory prothrombin (PT)-INR measurements and simultaneous INR determinations using the POC-INR meter. Agreement between CoaguChek XS INR and laboratory INR was assessed using Bland-Altman plots. Bland-Altman's 95% limits of agreement were 0.11 (-0.20; 0.42) and 0.13 (-0.22; 0.48) at the two time points, respectively. In conclusion, the CoaguChek XS meter appraisal generates an accurate and precise INR measure in children when compared to laboratory INR test results.

Cerebral venous thrombosis in newborns, infants and children.

Cerebral venous thrombosis is currently thought to be a relatively rare and benign entity in childhood. Recent studies however have shown that cerebral venous thrombosis is more common than previously believed, and carries significant mortality and neurologic morbidity. Neonates are the most commonly affected age group, compared to children >1 month of age. Magnetic resonance imaging venography is the gold standard by which this diagnosis should be made. Clinical trials are currently necessary to determine the most efficacious,safe, and age-specific approach for anticoagulation for this childhood disorder.

Standardization of telephone triage in pediatric oncology.

Nurses in pediatric oncology are often the main resource for overwhelmed parents and deal with complex patient issues over the telephone but often not without concerns about best patient care, liability, and accountability for the advice given. The question is whether using standardization of telephone triage practices can provide opportunities for improvement in the care of pediatric oncology patients. A review of the literature pertaining to telephone triage, standardization of practice, and the practice in outpatient oncology was conducted. The utilization of easy-to-use, accessible yet nonrestrictive resources and a well-designed documentation tool can help guide the decision-making process while addressing legal concerns and ensuring best possible patient care. An advantage that nurses in outpatient oncology settings have in performing telephone triage is the knowledge they have of their patient population and the disease process and treatments. Using a balanced approach to standardization of telephone triage practices can provide opportunities for improvements in care while still capitalizing on the intuitive knowledge and experience of the nurses involved.