Introduction. Familial hypercholesterolemia (FH) is caused by nonfunctioning low-density lipoprotein (LDL) receptors, resulting in high serum cholesterol. Two types of FH are described: the heterozygous form is diagnosed in adults and responds well to medical therapy; the homozygous form is rare, diagnosed in children, and often requires multiple treatments to prevent complications. Cholesterol accumulation in tissues produces common clinical manifestations including cutaneous xanthomas, coronary artery disease, and aortic stenosis. Treatment options consist of lifestyle modifications, lipid-lowering medications, LDL aphaeresis, and orthotopic liver transplantation (OLT). Case Presentation. Two patients with FH presented at young ages due to characteristic cutaneous xanthomas. The patients underwent cardiac testing that revealed atherosclerotic changes. The patients received maximal medical therapy, but only experienced a small decrease in serum cholesterol and LDL levels. After several years of medical treatment without improvement of symptoms, the patients were listed for OLT. The transplantations were successful, and only one patient had a postoperative complication of acute rejection, treated successfully. Currently, both patients are doing well with regression of the cutaneous xanthomas and atherosclerotic changes. Conclusion. OLT is a safe and effective option for patients with homozygous FH refractory to maximal medical therapy and may represent the optimal treatment for these patients.

CF affects one of 2000 Caucasians, and approximately 25% are found to have CFLD for which OLT may be indicated. Timing of transplantation, contraindications, and survival are still widely debated. We report the outcomes of OLT for pediatric patients with CFLD from the largest children's hospital in the United States. Our records since September 1998 were analyzed for all patients undergoing OLT for CFLD. Nine patients were then compared to similar patients in the UNOS/OPTN database (n = 155). Survivals were calculated with the Kaplan-Meier method and compared using the log-rank test. All statistics were performed in SPSS 15.0. We performed OLT on nine pediatric patients with CFLD, with age ranging from nine to 17 yr at the time of transplant. Mean survival was 69.2 months; patient and allograft survivals at one and five yr were 88.9%, with one death at day 21 due to Aspergillus fumigatus sepsis. Two patients underwent concurrent multi-organ transplantation. One patient required double lung transplantation four yr after isolated OLT. Comparison to the UNOS/OPTN database revealed a trend toward improved survival. Patients with CF can achieve favorable outcomes after OLT, as we report excellent survivals for pediatric patients with CFLD.

Dosing IVIG to maintain an IgG level > 400 mg/dl is a cost-effective and safe way to prevent viral infections in pediatric patients undergoing HSCT.

BK virus hemorrhagic cystitis is a complication of HCST. Response to IV cidofovir is unpredictable, and treatment carries risk of toxicity. We report the largest series of pediatric patients with BKHC after HSCT successfully treated with intravesicular cidofovir. There was no significant decrease in urine or plasma BK PCR. There was significant decrease in pain score on days 3 and 7, with associated decrease in morphine use. No patients experienced toxicities associated with IV cidofovir. Intravesicular cidofovir appears to be safe and effective for symptomatic treatment of BKHC in pediatric patients after HSCT.

respiratory tract infections (LRI) in immunocompetent young children and older adults. Data on the impact of hMPV infections in HCT recipients has been scarce. In this study, we aimed to determine the morbidity and mortality of hMPV infections in HCT recipients. Methods: We reviewed the medical records of all 83 HCT recipients with positive hMPV testing from April 2012 to April 2015. We collected the demographics, clinical characteristics and outcomes of hMPV infections from medical records. Results: Most of patients were allogeneic HCT recipients (60%) including matched related and unrelated donor (27% each), haploidentical (5%) and mismatched (2%), and the rest were autologous HCT recipients (40%). The cell source of the donor was peripheral (72%), marrow (8%), and cord (5%). Majority of patients were Caucasians (63%), males (57%) with a median age of 58 years (range: 4 to 80 years). Corticosteroid use in the past 30 days was identified in 34%, lymphocytopenia in 8% and neutropenia in 5% of hMPV patients. Three patients (3%) had acute GvHD, 9 patients (11%) had chronic GvHD, and 1 patient had acute on chronic GvHD at the time of hMPV diagnosis. Majority of infections were community-acquired (88%), presented at the URI stage (72%) and overall LRI rate was 39% (28% presented with LRI). Rates of LRI were higher for patients undergoing mismatched/ haploidentical/cord blood HCT compared to autologous HCT (P ¼ 0.08) (Figure). Hospital admission secondary to infection (39%), mechanical ventilation (5%), and oxygen requirements (25%) were observed. At day 30, all-cause and hMPV associated mortality was low (2%). Conclusions: hMPV infections are common in HCT recipients with the potential for nosocomial transmission and can cause significant morbidity leading to high rates of hospital admissions.