Background Deficits in knowledge and comfort related to pain management have been demonstrated in adult hematology/oncology fellows. No such evaluation has been undertaken in pediatric hematology/oncology (PHO) trainees. Procedure An IRB‐approved survey was administered to PHO fellows throughout the United States (US) to assess comfort with opioid dosing, attitudes related to the use of opioids, and knowledge of basic concepts including weight‐based dosing, incomplete cross‐tolerance, and management of side effects. Results Email addresses were obtained for 132 fellows from 37 programs. Seventy‐eight (59%) fellows participated. No significant difference was demonstrated between training level and comfort with dosing opioids in an opioid‐naive patient, though a smaller proportion of first‐year fellows (65%) reported comfort compared to more senior fellows (85.2% of second‐year fellows, 80.6% of third‐ and fourth‐year fellows). First‐year fellows correctly answered a mean of 5.05 ± 0.43 out of 10 objective knowledge questions; second‐year fellows answered 5.74 ± 0.35 correctly, and third‐ and fourth‐year fellows 5.58 ± 0.30. The majority of respondents chose an appropriate dose of intravenous morphine based on weight (92%), and identified a low‐dose naloxone drip as an appropriate intervention for opioid‐induced pruritis (91%). However, the remainder of the questions had a correct response rate of 15‐68%. Conclusion This study characterizes PHO fellows’ knowledge and comfort with prescribing opioids. Despite high levels of reported comfort, PHO fellows in all levels of training demonstrated knowledge gaps. PHO fellows may benefit from further education in pain management.

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Approximately 24% of all pediatric acute myeloid leukemia (AML) cases have mutations in the FMS-like tyrosine kinase 3 (FLT3) receptor gene. FLT3-TKD point mutations are rare in pediatrics and often occur in younger patients and in combination with 11q23 abnormalities. There is a paucity of data related to their prognostic implications in children. We describe 2 pediatric patients with FLT3-activating mutations as a feature of their AML. Both were diagnosed in infancy. The first experienced induction failure and had refractory disease without expression of FLT3-TKD mutation on subsequent bone marrow evaluations. His disease also harbored a KMT2A-PICALM gene rearrangement. He died of invasive fungal disease nine months after diagnosis. The second had a post-induction remission but developed swelling of the left calcaneus shown on biopsy to be a myeloid sarcoma positive for a new BRAF V600E mutation in addition to his known KMT2A rearrangement but without FLT3-TKD mutation. Despite multiple courses of therapy including BRAF/MEK-inhibition, he died of progressive disease nine months after diagnosis. FLT3 inhibition was not utilized in either patient as studies have largely focused on its role in internal tandem duplication (ITD) mutations and because the mutation was no longer detectable in either patient on subsequent evaluation. However, these cases add to the suggestion that these mutations confer a worse prognosis in pediatric AML patients.

Background: Acute pain is common in children and young adults with cancer and sickle cell disease. Current training curricula fail to adequately impart skills for pain management. We sought to develop and validate an education and assessment tool to address the safe effective use of opioids for pain management by pediatrics trainees. Methods:The first version of the tool contained 10 case-based, multiple-choice questions. It was pilot tested within a medium-sized pediatric residency program using preintervention and postintervention surveys to assess residents' knowledge and comfort related to prescribing opioids. Content validation was performed through an expert panel of physicians. Internal reliability was tested by administering the tool to learners and practitioners with varying levels of training.Results: Comfort with choosing and converting between opioids increased significantly in pilot testing (P = 0.005). Mean objective knowledge scores increased from 51% to 85.9% (P < 0.001). The revised tool showed internal reliability within each group (Cronbach alpha 0.71 to 0.78) and significant differences in mean scores between groups (F ratio = 9.45, P = 0.0002).Conclusions: This tool demonstrates validity and internal reliability. Its use was associated with short-term educational gains and it garnered overall favorable feedback from users. Further testing is needed to assess the duration of these gains.

Background: Many children with hematologic and oncologic diagnoses require opioids for management of pain, yet knowledge gaps persist among pediatric hematology/oncology (PHO) fellows.Objective: Pediatric Opioid Analgesia Self-Instruction System (PedOASIS) is an interactive, case-based education tool designed for independent learning. The goal of this study was to evaluate its efficacy in increasing PHO fellows' knowledge and comfort with using opioids to manage pain.Design/method: PHO fellows were recruited from 74 American College of Graduate Medical Education-accredited US programs during the 2019-2020 academic year and randomized to receive access to PedOASIS (intervention) or usual PHO training (control). Surveys at baseline, immediately after accessing the tool, and 6 months later assessed knowledge and comfort related to prescribing opioids.Results: A total of 64 PHO fellows completed the study, with 32 in the intervention group and 32 controls. At baseline, mean scores on the 10-question knowledge assessment were similar between groups (intervention: 5, control: 6; p = .8). Following intervention, mean score was significantly higher in the intervention group (9) versus controls (5; p < .0001). Six months later, scores in both groups decreased but remained significantly higher in the intervention group (7) compared to controls (5, p < .0001) and compared to baseline (p = .0002). Fellows in the intervention group reported significant increases in comfort dosing opioids after exposure to the tool (p = .02). Conclusion:PHO fellows exposed to the tool had improved scores on validated knowledge questions and greater comfort using opioids for pain management compared to controls. We therefore suggest that PedOASIS warrants further evaluation as a potential tool for PHO fellows.

Objectives: 36,360 new cases of CRC are expected for 2018/19 in Brazil. 5-year survival rate for patients with mCRC diagnosed in stage IV is as low as 12% mainly due to currently limited treatment options. Regorafenib was approved for the treatment of adult patients with mCRC previously treated with, or not eligible for, available therapies in Brazil in late 2018. The objective is to determine the budget impact of the incorporation of regorafenib in SHS for 3 rd -line mCRC in comparison to best supportive care (BSC). Methods: Budget impact analysis (BIA) considered a 5-year time horizon. 22.7% of Brazilian population is covered by SHS. The number of 3 rd -line mCRC eligible patients was calculated based on expected rates of metastatic disease according to stage of diagnosis and progression free and overall survival rates of standard 1 st and 2 nd -line treatments applied to the number of estimated new Brazilian CRC cases. Initial market-share for regorafenib was assumed to be 40%, reaching 80% by the 5 th year in the proposed scenario. Current scenario considers all patients receive BSC. The analysis considered direct costs related to treatment and adverse events management obtained from local public sources and clinical parameters from the CORRECT study. A deterministic sensitivity analysis was done to account for parameters' uncertainties. Results: The number of new mCRC patients eligible for 3 rd -line was estimated to be 2141 in the 1 st year, reaching 2546 in the 5 th . BIA showed overall impact of BRL 326 million for SHS in 5 years. The parameter with highest impact on the results was regorafenib's market-share in the proposed scenario. Conclusions: The budget impact of regorafenib's incorporation in comparison to BSC in SHS is related to the current unmet need of mCRC treatment in Brazil for patients who previously had no other effective therapies available.