To conduct a cost-effectiveness analysis, from a hospital's perspective, of 4 procedural sedation and analgesia (PSA) regimens to facilitate forearm fracture manipulation in the pediatric emergency department (ED): deep sedation with ketamine/midazolam (K/M) administration, propofol/fentanyl administration, fentanyl/midazolam (F/M) administration, and axillary block.We constructed a decision analytic model using relevant probabilities from published studies of pediatric patients who underwent fracture manipulation in the EDs. Total costs were calculated by assessing ED resource utilization associated with uncomplicated PSA and with PSA complicated by adverse events. Costs of consumable equipment were considered to be fixed. Total sedation time, personnel time, and drug costs were considered variable. We assumed that all PSA regimens provided effective relief from procedural distress. Failure rates for axillary block were estimated based on reports in the literature. When patients experienced emesis, recovery agitation, respiratory depression, lidocaine toxicity, or regional block failure, we assumed that the patients would require 1 additional hour of ED stay. Sensitivity analyses of all key variables in the model were performed to identify those that may result in a change in the preferred option. Monte Carlo simulations were performed to assess model robustness.Under baseline assumptions, the propofol/fentanyl regimen was the most cost-effective choice (expected cost, 84.06 US dollars), followed by axillary block (88.18 US dollars), K/M (105.32 US dollars), and F/M (159.79 US dollars), respectively. Varying the fixed and variable costs by 50% to 200% of their baseline values did not alter the ranking. When ketamine and propofol were administered without adjunctive midazolam and fentanyl, respectively, propofol remained the optimum choice. With total PSA time as the outcome measure, the incremental cost-effectiveness ratios were 8.1 US dollars and 24.9 US dollars per hour of ED time saved, for propofol/fentanyl versus axillary block and for axillary block versus K/M, respectively.Among PSA regimens during forearm fracture manipulation in the pediatric ED, propofol/fentanyl is the most cost-effective regimen followed by axillary block, K/M, and F/M.
BCOR alterations are described in ultra-rare infantile soft tissue sarcomas including primitive myxoid mesenchymal tumor of infancy and undifferentiated round cell sarcoma (URCS). Previous reports often describe dismal outcomes. Thus, we undertook a retrospective, multi-institutional study of infants with BCOR -rearranged soft tissue sarcomas. Nine patients aged 6 weeks to 15 months were identified. One tumor carried a BCOR :: CCNB3 fusion, whereas 7 tumors harbored internal tandem duplication of BCOR , including 4 cases classified as primitive myxoid mesenchymal tumor of infancy, 1 case as URCS, and 2 cases characterized by a “hybrid morphology” in our evaluation. Four patients underwent upfront surgery with residual disease that progressed locally after a median of 2.5 months. Locoregional recurrences were observed in hybrid patients, and the URCS case recurred with brain metastases. Complete radiographic responses after chemotherapy were achieved in patients treated with vincristine/doxorubicin/cyclophosphamide alternating with ifosfamide/etoposide, vincristine/doxorubicin/cyclophosphamide alternating with cyclophosphamide/etoposide (regimen I), and ifosfamide/carboplatin/etoposide. Seven patients received radiotherapy. With a median of 23.5 months off therapy, 8 patients are with no evidence of disease. In our study, observation was inadequate for the management of untreated postsurgical residual disease. Tumors demonstrated chemosensitivity with anthracycline-based regimens and ifosfamide/carboplatin/etoposide. Radiotherapy was required to achieve durable response in most patients.
Abstract Infantile myofibroma is the most common fibrous tumor of infancy. Despite the frequency of these tumors, the natural history is incompletely understood. We present two cases with a unique pattern of disease: solitary myofibromas with subsequent progression to diffuse myofibromatosis. Given the variable spectrum of disease and the corresponding difference in morbidity and potential mortality based on the extent of disease, we propose surveillance recommendations.
Without early initiation of disease-modifying therapy, the acute and chronic complications of sickle cell anemia (SCA) begin early in childhood and progress throughout life. Hydroxyurea is a safe and effective medication that reduces or prevents most SCA-related complications. Despite recommendations to prescribe hydroxyurea for all children with SCA as young as 9 months, utilization remains low.We completed a retrospective review of hydroxyurea-prescribing practices and associated clinical outcomes at our institution over a 10-year period before and after the 2014 National Heart, Lung, and Blood Institute (NHLBI) recommendations to use hydroxyurea for all children with SCA.Hydroxyurea use more than doubled within our pediatric SCA population from 43% in 2010 to 95% in 2019. The age of hydroxyurea initiation was significantly younger during 2014-2019 compared to 2010-2013 (median 2 years vs. 6 years, p ≤ .001). With this change in clinical practice, nearly all (69/71 = 97%) children born after 2013 received disease-modifying therapy by the end of 2019, primarily hydroxyurea (93%). Concurrently, the number of SCA-related admissions significantly decreased from 67/100 patient-years in 2010 to 39/100 patient-years in 2019 (p < .001).The early and universal prescription of hydroxyurea for children with SCA is the standard of care. Here, we demonstrate that a careful and deliberate commitment to follow this guideline in clinical practice is feasible and results in measurable improvements in clinical outcomes. Our approach and improved outcomes can serve as a model for other programs to expand their hydroxyurea use for more children with SCA.
Thromboembolic disease rates are increasing in pediatric patients. Anticoagulation is prescribed for treatment and prevention of thromboembolic disease. While nonadherence to anticoagulation regimens predicts poor health outcomes in adults, data in anticoagulated pediatric patients are limited. We systematically reviewed the rates, outcomes, and predictors of anticoagulation nonadherence in the pediatric population. Out of a total of 3581 unique articles identified for review, 17 studies met inclusion criteria. These studies primarily evaluated patients with cardiac disease treated with vitamin K antagonists. Overall nonadherence rates varied from 3% to 42%, based upon population, definition of adherence, and measurement strategy. Patient age, goal international normalized ratio (INR), and number of concurrent potentially interacting medications correlated with nonadherence. Data examining the relationship between nonadherence and health outcomes were included in only two studies. Limitations of current literature, as well as critical knowledge gaps that require future study, are discussed.
