future analyses, such as determination of value of services, which in turn could support the justification of HSCT pharmacy services.

SMX/TMP is the current gold standard for prophylaxis against PCP in immunocompromised pediatric patients. Currently, there are several second-line options for prophylaxis but many, including intravenous (IV) pentamidine, have not been reported to be as effective or as safe as SMX/TMP in the pediatric transplant population. This study is to determine the efficacy and safety of IV pentamidine in preventing PCP in pediatric transplant patients. A retrospective chart review was conducted to evaluate all transplant patients that received at least one dose of IV pentamidine from January 2010 to July 2013. The primary outcome, IV pentamidine efficacy, was evaluated by the incidence of PCP diagnosis for 28 days after the last dose of IV pentamidine if patient was transitioned to another agent for PCP prophylaxis. Patients on IV pentamidine for entire course of PCP prophylaxis were followed at least six months after discontinuation of IV pentamidine. The safety of IV pentamidine was assessed by the incidence of adverse events leading to pentamidine discontinuation. All data were analyzed using descriptive statistics. All transplant patients at CCHMC who had received IV pentamidine were reviewed, and 333 patients met inclusion criteria. The overall incidence of PCP was found to be 0.3% for pediatric transplant patients on pentamidine. Pentamidine was found to be safe, and the incidence of adverse events leading to discontinuation was 6% with the most common reason being tachycardia 2.1%. IV pentamidine is safe and effective as PCP prophylaxis in pediatric transplant patients with a PCP breakthrough rate of 0.3% (1 of 333 patients), and only 20 adverse events led to discontinuation. We recommend that IV pentamidine be considered as a second-line option in pediatric transplant patients who cannot tolerate SMX/TMP.

Cardiac rhabdomyoma is the most common neonatal cardiac tumor and is typically associated with tuberous sclerosis complex (TSC). Although these tumors may naturally regress, some patients require surgical resection because of cardiac instability. If not fully resected, patients may also require medical therapy to improve their hemodynamics. Everolimus, a mammalian target of rapamycin inhibitor, has shown promise in reducing rhabdomyoma in patients with TSC, but the drug’s impact in patients without TSC has not been reported. Monitoring of tumor response has typically been limited to echocardiograms, which is not ideal given inherent difficulties in three-dimensional measurements. We report a case of sporadic cardiac rhabdomyoma in a neonate treated with everolimus resulting in tumor regression as documented by cardiac MRI. While on everolimus, our patient had an increased incidence of a preexisting arrhythmia, which resolved with planned cessation of therapy, suggesting that close monitoring is imperative in patients with arrhythmia.

Electronic medical records of 171 consecutive pediatric allogeneic HCT recipients (<21 years) from 2008-2013 were reviewed for BSI, transplant characteristics during the first year post HCT. Outcomes reported included relapse, acute graft versus host disease (aGVHD), chronic GVHD, OS, EFS, transplant cost and hospital stay. All patients had a central line during part of HCT utilizing standardized line care protocols. Five year OS, EFS for cohort with BSI was 45% (95%CI 32-56), 40% (95%CI 27-53) respectively; and 78% (95%CI 66-89),73% (95%CI 59-83) respectively for non BSI cohort. Median age was 8.9y, 61% males with 61% receiving HCT for a malignancy, 56% receiving myeloablative conditioning and 58% receiving peripheral blood stem cells. Median follow up was 4.5y (Table 1). There were 196 BSI episodes reported in 49% of 171 total HCT recipients. 50% had a single BSI episode and the remaining multiple. BSI occurred in 49, 26 and 31 patients from days 0-30, D+ 31-100 and D+ 101-365 post HCT respectively. There was no difference in baseline characteristics between the BSI and non BSI group. Intriguingly, a statistically significant increase in the incidence of >Gr2 aGVHD, GI aGVHD and death seen in BSI group compared to non-BSI group (p¼0.018, 0.014, 0.0002) respectively. To determine causality, we focused on early post HCT BSI (D0-30), before most patients developed aGVHD and found an increase in >Gr 2 aGVHD and death (p¼0.05 and 0.02, respectively), suggesting that BSI predating aGVHD could have contributed independently to poor outcomes. Inpatient hospital stay was median of 11 days extra and cost of HCT was 20% higher in BSI cohort in the first year post HCT. In conclusion, BSI remains a significant factor in HCT related outcomes. Although causality cannot be determined from this analysis there was increased incidence of severe aGVHD and death with early BSI, possibly suggesting that a BSI induced inflammatory state could adversely affect subsequent clinical outcomes. Standardized susceptibility based protocols need to be instituted to decrease the incidence of BSI in HCT.