A randomized controlled trial to examine the effect of the Pediatric Opioid Analgesia Self-Instruction System (PedOASIS) tool on pediatric hematology/oncology trainee education.

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.

Prolonged Survival Using Outpatient Palliative Chemotherapy in Two Children With Refractory Acute Myelogenous Leukemia.

BACKGROUND: Therapy options for relapsed/refractory acute myelogenous leukemia (AML) are limited. Palliative chemotherapy options have been explored in adult patients, but little evidence exists in children. OBJECTIVES: Describe the clinical course of 2 pediatric patients with refractory AML who transitioned to outpatient palliative chemotherapy with good disease control and quality of life on these regimens. PATIENTS AND METHODS: Patient 1 was a 2-year-old girl who received a total of 4 cycles of standard chemotherapy with multiple complications and 15% to 20% blasts on marrow subsequent evaluation. An outpatient regimen of decitabine and vorinostat was consequently chosen for her. Patient 2 was a 16-year-old boy with residual disease after induction 1 with arm A with cytarabine, daunorubicin, and etoposide. His induction 2 course was complicated by multiorgan failure secondary to multiple infections including Klebsiella pneumonia and radiographically identified pulmonary fungal disease. On recovery, the marrow showed no disease but after the toxicities of initial therapy, the patient pursued a palliative regimen with azacitidine and lenalidomide. RESULTS: Patient 1 tolerated her regimen for 14 months, requiring weekly blood products and only one hospitalization for a central-line infection. Her blast count then increased precipitously, the disease progressed, and she died comfortably while receiving hospital-based end-of-life care. Patient 2 tolerated 14 months of his regimen. On a surveillance marrow sample, he was found to have 0.02% minimal residual disease. He then elected to pursue marrow transplantation. He maintained remission until his 6-month posttransplant surveillance bone marrow biopsy, which revealed 0.04% minimal residual disease. CONCLUSION: We describe 2 pediatric patients with relapsed/refractory AML who achieved disease control and acceptable quality of life utilizing outpatient palliative chemotherapy for over 12 months. These regimens should be considered in patients who no longer desire cytotoxic chemotherapy or are ineligible for further aggressive approaches.

Development and Validation of Pediatric Opioid Analgesia Self-Instruction System (PedOASIS): An Opioid Knowledge Tool for Pediatric Clinicians.

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.

Evaluation of knowledge and comfort with opioid prescribing among pediatric hematology/oncology fellows.

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.

Cost-effectiveness Analysis of Screening Extremely Low Birth Weight Children for Hepatoblastoma Using Serum Alpha-fetoprotein.

OBJECTIVES: To evaluate the cost-effectiveness of screening children born at extremely low birth weight (ELBW) for hepatoblastoma using serial serum alpha-fetoprotein measurements. STUDY DESIGN: We created a decision tree to evaluate the cost effectiveness of screening children born at ELBW between 3 and 48 months of age compared with current standard of care (no screening). Our model used discounted lifetime costs and monetary benefits in 2018 US dollars, based on estimates in the published literature. The effects of uncertainty in model parameters were also assessed using univariate sensitivity analyses, in which we changed the values for one parameter at a time to assess the effect on the estimated incremental cost-effectiveness ratio. RESULTS: For the estimated 55 699 children born at ELBW in the US each year, this screening is associated with 77.7 additional quality-adjusted life-years (QALYs) at a cost of $8.7 million. This results in an incremental cost-effectiveness ratio of about $112 000/QALY, which is considered cost effective from a US societal perspective. For children diagnosed with hepatoblastoma, our model finds that the screening regimen is associated with a 10.1% increase in survival, a 4.18% increase in expected QALYs, and a $245 184 decrease in expected cost. CONCLUSIONS: Screening ELBW children for hepatoblastoma between 3 and 48 months of age dominates the alternative and is cost effective from a societal perspective.

Counting Backward by Seven.

I delayed my neurology rotation in medical school because I was trying to ignore the symptoms of dementia developing in my mother. They became painfully apparent when I saw my parents less frequently after I moved away for residency, and finally had to be addressed when I had a child of my own and felt unsafe having her care for him. Through my experiences with her and in my training in palliative care and oncology, I have learned that tools like the Mini Mental State Examination (MMSE) and Functional Assessment Staging Test (FAST) can only tell us so much; the true measure of a patient's decline can be found in the patient's and family's own story.

Acute Myocardial Dysfunction and Hypereosinophilic Infiltrative Myocarditis Secondary to New-Onset Pediatric Acute Lymphoblastic Leukemia.

Myocardial infiltration by eosinophils leads to myocardial inflammation and fibrosis, resulting in restrictive hemodynamics. We describe an uncommon presentation of eosinophilic predominant acute lymphoblastic leukemia that manifested with hypereosinophilic infiltrative myocarditis. (Level of Difficulty: Advanced.).

Not Quite Child's Play: Using Beanbags to Teach Opioid Dosing and Rotation.

Pain management education is lacking in medical education, especially pediatrics. Using beanbags constructed to represent commonly used opioids with areas proportional to the drugs' potencies, this hands-on approach is used in small-group sessions to introduce opioid dosing, potencies, and rotation through case-based exercises. Next steps will include analysis of impact using pre- and post-intervention surveys.

Mutations within the Activation Loop Domain of FLT3 in Two Pediatric Patients with Refractory Infant Acute Myeloid Leukemia.

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.