Effects of sirolimus in the treatment of unresectable infantile hemangioma and vascular malformations in children: A single-center experience

Objective: Recently, sirolimus has emerged as a safe and effective treatment modality for unresectable vascular lesions. In the present study, we investigated the effectiveness and safety of sirolimus from our early experience with patients with unresectable vascular anomalies. Methods: The medical records and radiologic images of all patients with unresectable vascular anomalies treated with sirolimus at our center from January 2018 to November 2019 were retrospectively reviewed. All patients were administered oral doses of sirolimus 0.8 mg/m(2) every 12 hours as the initial dose, followed by maintenance of a target serum concentration (5-10 ng/mL) with therapeutic drug monitoring. Results: Six patients with unresectable vascular anomalies were treated with sirolimus for >= 10 months. Their median age at the initiation of sirolimus treatment was 17 months (range, 8-67 months). The median duration of treatment was 13 months (range, 10-16 months). One patient had a good response, four had an intermediate response, and one had no response to sirolimus therapy. None of the patients had discontinued sirolimus therapy because of adverse effects. Conclusions: Sirolimus can be used effectively and safely for patients with unresectable vascular anomalies. However, further prospective studies are warranted to evaluate the long-term effects of sirolimus and clarify the indications for early intervention

Assessment of native liver fibrosis using ultrasound elastography and serological fibrosis indices in children with biliary atresia after the Kasai procedure

Background: Validated non-invasive examinations are necessary to monitor liver fibrosis in children with biliary atresia (BA) after the Kasai procedure. Purpose: To evaluate the diagnostic accuracy of two-dimensional shear wave elastography (2D-SWE), transient elastography (TE), and the serologic biomarkers of aspartate transaminase-to-platelet ratio index (APRI) and Fibrosis-4 (FIB-4) score for evaluating native liver fibrosis in children with BA. Material and Methods: We retrospectively reviewed same-day 2D-SWE and TE liver stiffness (LS) measurements of 63 patients with BA who underwent the Kasai procedure. The APRI and FIB-4 score were computed. Hepatic fibrosis was categorized into three clinical categories based on the ultrasound (US) hepatic morphology and clinical manifestations of liver cirrhosis: I, pre-cirrhotic liver state (n ¼ 15); II, US and/or clinical signs of liver cirrhosis with compensated liver function (n ¼ 27); and III, liver cirrhosis with decompensated liver function (n ¼ 21). We compared area under the receiver operating characteristic curve (AUC) data among 2D-SWE, TE, APRI, and FIB-4 score. Combined evaluation of serologic fibrosis indices and US elastography was conducted and AUCs of combinations were analyzed. Results: 2D-SWE, TE, APRI, and FIB-4 score showed good to excellent diagnostic accuracy for differentiating clinical categories (AUCs 0.779?0.955). AUC values were significantly increased after adding TE to FIB-4 score for detecting liver cirrhosis (P ¼ 0.02). Conclusion: 2D-SWE, TE, APRI, and FIB-4 score are accurate non-invasive markers for monitoring native liver fibrosis in patients with BA. Combined use of serologic markers and US elastography could yield more accurate diagnoses of liver fibrosis than serologic markers alone

Long-Term Safety and Efficacy of Pure Laparoscopic Donor Hepatectomy in Pediatric Living Donor Liver Transplantation

Laparoscopic living donor hepatectomy for transplantation has been well established over the past decade. This study aimed to assess its safety and feasibility in pediatric living donor liver transplantation (LDLT) by comparing the surgical and long-term survival outcomes on both the donor and recipient sides between open and laparoscopic groups. The medical records of 100 patients (<= 17 years old) who underwent ABO-compatible LDLT using a left lateral liver graft between May 2008 and June 2016 were analyzed. A total of 31 donors who underwent pure laparoscopic hepatectomy and their corresponding recipients were included in the study; 69 patients who underwent open living donor hepatectomy during the same period were included as a comparison group. To overcome bias from the different distributions of covariables among the patients in the 2 study groups, a 1:1 propensity score matching analysis was performed. The mean follow-up periods were 92.9 and 92.7 months in the open and laparoscopic groups, respectively. The mean postoperative hospital stay of the donors was significantly shorter in the laparoscopic group (8.1 days) than in the open group (10.6 days; P < 0.001). Overall, the surgical complications in the donors and overall survival rate of recipients did not differ between the groups. Our data suggest that the laparoscopic environment was not associated with long-term graft survival during pediatric LDLT. In addition, the laparoscopic approach for the donors did not adversely affect the corresponding recipient's outcome. Laparoscopic left lateral sectionectomy for living donors is a safe, feasible, and reproducible procedure for pediatric liver transplantation

Outflow vein venoplasty of left lateral section graft for living donor liver transplantation in infant recipients

The orifice size of the LHV trunk in LLS grafts is often too small for direct anastomosis. Several methods were developed to enlarge the graft and recipient hepatic vein orifices. This study described our surgical techniques to secure hepatic vein reconstruction in infant recipients and analyzed the patency outcomes. Twelve infants undergoing pediatric LDLT were selected during 2-year study period between January 2018 and December 2019. Surgical techniques and vascular complications of graft hepatic vein outflow were analyzed. The mean recipient age was 12.5 +/- 4.5 months; mean body weight was 9.4 +/- 1.0 Kg; and mean graft-recipient weight ratio was 2.8 +/- 0.6%. Primary diseases were biliary atresia in six patients, metabolic diseases in two, hepatoblastoma in two, and acute liver failure in two. Eight LLS grafts were recovered through an open method, and four LLS grafts were recovered through a laparoscopic method. A small superficial LHV branch was present in five of 12 LLS grafts, which was opened to widen the graft hepatic vein orifice. Incision-and-patch venoplasty was performed in 10, unification venoplasty in 1 and no venoplasty in 1. All four LLS grafts recovered through a laparoscopic approach required circumferential vein patch because of very short hepatic vein stump. No patient experienced graft hepatic vein-associated vascular complications during the follow-up period of 19.3 +/- 9.3 months. Our surgical techniques with incision-and-patch venoplasty for LLS grafts is beneficial to reduce the risk of hepatic vein outflow obstruction in recipients receiving LLS grafts

Recent improvement in survival outcomes and reappraisal of prognostic factors in hepatoblastoma

Background Prognostic factors in hepatoblastoma need to be reevaluated considering the advances in treatment modalities. The study aimed to evaluate current outcomes of hepatoblastoma and reappraise the association of prognostic factors, including pre-treatment extent of tumor (PRETEXT) stage with annotation factors and Children's Hepatic tumors International Collaboration-Hepatoblastoma Stratification (CHIC-HS) system, with survival outcomes. Methods We evaluated 103 consecutive patients with hepatoblastoma retrospectively according to the treatment period based on the introduction of a liver transplantation program. Results The 5-year overall survival (OS), event-free survival (EFS), and transplant-free survival rates were 80.2%, 74.2%, and 61.8%, respectively. EFS and OS were improved significantly from 58.6% to 81.6% (P = 0.024) and from 58.6% to 90.8% (P < 0.001), respectively, in the late period (N = 74) compared with the early period (N = 29). The PRETEXT stage was significant or marginally significant for EFS and OS in the early period but not in the late period. The P, F, R, and C factors were significant for OS and EFS in the early period. However, in the late period, only the P factor was significant for OS, and the F and M factors were significant for EFS. The CHIC-HS system was significant or marginally significant for EFS in both the early and late periods; however, it was significant for OS only in the early period. Conclusion Survival rates were significantly improved in children with hepatoblastoma, especially in those with advanced PRETEXT stages with positive annotation factors and in a high-risk CHIC-HS group. Prognostic factors had different clinical implications with evolved treatment modalities