Children's Hospital Colorado

The study goal was to evaluate the outcomes associated with albumin use in children with sepsis and shock compared with those without shock, using causal inference analysis in a multicenter cohort. This was a secondary analysis of electronic health record data collected from 13 U.S. PICUs between 2012 and 2018, consisting of children younger than 18 years who met Phoenix sepsis criteria within the first 24 hours of PICU admission. Covariate-balancing propensity score weighting was applied to adjust for indication bias in the albumin use. Patients receiving at least 0.5 g/kg albumin within 24 hours of PICU admission were assigned to the albumin group; others to the control. Only 24-hour survivors were included to address immortal time bias. Overall, 17,307 children with sepsis survived at least 24 hours. Of these, 1,344 patients (7.8%) who received albumin within the first 24 hours, and 9,678 (55.9%) met the criteria for septic shock. A significant interaction between albumin use and shock status was observed (interaction: -0.353, p = 0.007), with albumin administration in pediatric septic shock patients associated with lower in-hospital mortality: odds ratio equals to 0.698 (95% CI, 0.629-0.774), risk ratio equals to 0.746 (95% CI, 0.625-0.891), and hazard ratio equals to 0.688 (95% CI, 0.558-0.848). In contrast, there was no difference in outcomes between the albumin and control groups in the non-shock group. Early albumin administration was associated with improved outcomes in children with septic shock, but not in those without shock. These results highlight the importance of considering clinical heterogeneity, such as the presence of shock, in identifying treatment-responsive subgroups and enabling more targeted interventions in pediatric sepsis. Further prospective validation is warranted.

Outcomes for pediatric relapsed/refractory (R/R) acute myeloid leukemia (AML) remain dismal. CPX-351, a liposomal formulation of cytarabine and daunorubicin, may have less off-target toxicities than traditional chemotherapies and has shown improved outcomes for adults with newly diagnosed therapy-related AML. In this first-in-pediatric, single center phase 1 study (NCT01943682), 27 patients with R/R acute leukemia (acute lymphoblastic leukemia [ALL, n = 3], AML [n = 23], or mixed phenotype acute leukemia [MPAL, n = 1]) received one cycle of CPX-351 (on days 1, 3, 5) at either 44mg/m2 daunorubicin/dose or 59mg/m2 daunorubicin/dose. The primary objectives were to determine the CPX-351 recommended phase 2 dose (RP2D) and to evaluate its tolerability; secondary objectives were to assess marrow overall response rate (ORR) and cardiotoxicity. The RP2D was determined to be 44mg/m2 daunorubicin/dose. Grade ≥3 non-hematologic toxicities occurred in 89% of patients including febrile neutropenia (85%), infection (52%), and maculo-papular rash (37%). No grade ≥3 acute cardiac toxicities or significant changes in cardiac biomarkers were detected. The marrow ORR for AML patients was 48% (10/21 evaluable patients), despite high-risk genetic findings in most and nearly half of patients having undergone prior hematopoietic stem cell transplant. No patients with ALL or MPAL responded. CPX-351 at 44mg/m2 daunorubicin/dose was safe and tolerable in children with R/R acute leukemia, showing promising activity in heavily pretreated, high-risk AML and is now the FDA-approved pediatric dose for therapy-related AML or AML with myelodysplastic changes.

This cross-sectional study uses a mystery caller approach to identify the level of acceptance of new pediatric patients and the length of waiting for a first appointment at general and pediatric dermatology practices.

Allergy and dermatology societies do not recommend food avoidance for pediatric atopic dermatitis (AD) management, as it is ineffective and increases the risk of food allergy. However, we hypothesized that many caregivers restrict their child's intake of allergenic foods to treat AD and aimed to characterize diet modifications and motivating factors in this cross-sectional observational study. Caregivers of young children with AD were asked to complete a survey in this single-center study assessing AD severity, allergic comorbidities, diet modifications, and sources of recommendations. Our results indicated that many caregivers hold the belief that diet impacts AD (42% of study population) and implemented food avoidance (38% of study population), often at the recommendation of primary care providers (50% of study population), highlighting the need for improved counseling on AD management as it relates to diet, particularly given the known risk of food allergy development with food avoidance.