A quality improvement initiative standardizing premedication for blood product transfusions.

For children with cancer, blood product transfusions are crucial, but can be complicated by transfusion reactions. To prevent these complications, premedication is often given, although not always evidence-based. Herein, we describe a significant decrease in the use of premedication (72%-28%) at our institution after the implementation of standardized guidelines, without an increase in transfusion reactions (3.2% prior vs. 1.5% after standardization). Importantly, there were no severe transfusion reactions leading to hospitalization or death. Our results provide evidence in favor of more judicious use of premedication prior to transfusions in patients 21 years and younger being treated for cancer.

Brentuximab vedotin after autologous transplantation in pediatric patients with relapsed/refractory Hodgkin lymphoma.

Outcomes for children and adolescents with relapsed and refractory Hodgkin lymphoma (HL) are poor, with ∼50% of patients experiencing a subsequent relapse. The anti-CD30 antibody-drug conjugate brentuximab vedotin improved progression-free survival (PFS) when used as consolidation after autologous stem cell transplantation (ASCT) in adults with high-risk relapsed/refractory HL. Data on brentuximab vedotin as consolidative therapy after ASCT in pediatric patients with HL are extremely limited, with data of only 11 patients reported in the literature. We performed a retrospective analysis of 67 pediatric patients who received brentuximab vedotin as consolidation therapy after ASCT for the treatment of relapsed/refractory HL to describe the experience of this regimen in the pediatric population. This is the largest cohort reported to date. We found that brentuximab vedotin was well tolerated with a safety profile similar to that of adult patients. With a median follow-up of 37 months, the 3-year PFS was 85%. These data suggest a potential role for the use of brentuximab vedotin as consolidation therapy after ASCT for children with relapsed/refractory HL.

Acute myeloid leukemia with an MN1-ETV6 fusion in a young child with Down syndrome.

Myeloid leukemia of Down syndrome (ML-DS) in young children is associated with distinct clinical and biological features and is typically initiated with oncogenic mutations in the X-linked megakaryocytic transcription factor GATA1. Here we present a 3-yr-old child with DS diagnosed with acute myeloid leukemia (AML), which lacks typical immunophenotypic and molecular characteristics of ML-DS, including GATA1 mutations. The leukemic blasts were found to have an MN1-ETV6 gene fusion, a high-risk oncofusion not previously described in DS patients. This report highlights the importance of immunophenotypic, cytogenetic, and molecular characterization of ML-DS for identification of rare cases with unique features that may benefit from treatment protocols that are more intensive than those developed for patients with typical GATA1 mutant ML-DS.

A five year retrospective study of short term respiratory support outcomes for infants who received tracheostomy before one year of age.

OBJECTIVES: This study aims to describe respiratory support requirements at the time of hospital discharge for infants who undergo tracheostomy, and to determine whether certain indications for tracheostomy are significantly associated with ventilator or oxygen dependence at the time of discharge. METHODS: Retrospective chart review identified 150 patients who underwent tracheostomy before 1 year of age at a single center from 2007 to 2012 and were discharged alive. Patients were divided into groups based on primary indication for tracheostomy: chronic lung disease (CLD); cardiac; airway anomalies (e.g., tracheomalacia, subglottic stenosis); anatomic anomalies of head, neck and chest; neuro/muscular; mixed group (>1 primary indication). Chi-squared tests were used to compare respiratory support requirements at time of discharge, as well as need for supplemental oxygen. RESULTS: Of the 150 patients included in the study, three were discharged on room air alone. Of those 147 who did require some form of support at discharge, significant differences were found between groups when comparing CPAP to ventilator support. For example, of the patients with CLD, 82% were discharged on ventilator support whereas of those with a primary airway indication nearly 54% were discharged on CPAP. Significant differences were also found among groups when comparing patients discharged on room air vs. supplemental oxygen. Patients with CLD were more likely to be discharged on supplemental oxygen (p=0.001) whereas of the patients with anatomic indication 77% required no supplemental oxygen at the time of discharge. CONCLUSION: Respiratory support needs at the time of discharge for neonates who underwent tracheostomy varied significantly depending on the initial indication for tracheostomy. Information about respiratory requirements of infants who undergo tracheostomy can help clinicians counsel families and anticipate post-discharge needs.