Recent outcomes of liver transplantation for Budd-Chiari syndrome: A study of the European Liver Transplant Registry (ELTR) and affiliated centers.

BACKGROUND AND AIMS: Management of Budd-Chiari syndrome (BCS) has improved over the last decades. The main aim was to evaluate the contemporary post-liver transplant (post-LT) outcomes in Europe. APPROACH AND RESULTS: Data from all patients who underwent transplantation from 1976 to 2020 was obtained from the European Liver Transplant Registry (ELTR). Patients < 16 years, with secondary BCS or HCC were excluded. Patient survival (PS) and graft survival (GS) before and after 2000 were compared. Multivariate Cox regression analysis identified predictors of PS and GS after 2000. Supplemental data was requested from all ELTR-affiliated centers and received from 44. In all, 808 patients underwent transplantation between 2000 and 2020. One-, 5- and 10-year PS was 84%, 77%, and 68%, and GS was 79%, 70%, and 62%, respectively. Both significantly improved compared to outcomes before 2000 ( p < 0.001). Median follow-up was 50 months and retransplantation rate was 12%. Recipient age (aHR:1.04,95%CI:1.02-1.06) and MELD score (aHR:1.04,95%CI:1.01-1.06), especially above 30, were associated with worse PS, while male sex had better outcomes (aHR:0.63,95%CI:0.41-0.96). Donor age was associated with worse PS (aHR:1.01,95%CI:1.00-1.03) and GS (aHR:1.02,95%CI:1.01-1.03). In 353 patients (44%) with supplemental data, 33% had myeloproliferative neoplasm, 20% underwent TIPS pre-LT, and 85% used anticoagulation post-LT. Post-LT anticoagulation was associated with improved PS (aHR:0.29,95%CI:0.16-0.54) and GS (aHR:0.48,95%CI:0.29-0.81). Hepatic artery thrombosis and portal vein thrombosis (PVT) occurred in 9% and 7%, while recurrent BCS was rare (3%). CONCLUSIONS: LT for BCS results in excellent patient- and graft-survival. Older recipient or donor age and higher MELD are associated with poorer outcomes, while long-term anticoagulation improves both patient and graft outcomes.

Normothermic regional perfusion in controlled DCD liver procurement: Outcomes of the Swedish national implementation protocol.

Liver transplantation (LTX) using donors after controlled circulatory death (cDCD) is associated with poorer graft survival and increased incidence of nonanastomotic biliary strictures (NASs) compared to livers procured from brain-dead donors (DBD). The use of normothermic regional perfusion (NRP) during cDCD procurement may improve posttransplant outcomes and reduce the incidence of NAS. In Sweden, cDCD LTX was introduced through a national pilot protocol with mandatory NRP. This study aims to evaluate the outcome of cDCD LTX during the pilot period. Donor and recipient data were collected on all cDCD liver transplants during the pilot period between January 2020 to December 2022. Outcome on NAS, patient and graft survival, early allograft dysfunction, acute kidney injury, and comprehensive complication index was compared to a matched cohort of 28 patients transplanted with a DBD liver between 2018 and 2022. Eighteen patients were transplanted with a liver from a cDCD donor after using NRP. The mean functional warm ischemia time was 29 ± 6 minutes. The mean lactate reduction during NRP was 8.7 ± 2.4 mmol/L, and the end NRP perfusate alanine aminotransferase was 1.4 ± 1 µkat/L. When comparing recipients of cDCD liver transplant to DBD, no significant differences were observed in the incidence of NAS, patient and graft survival, comprehensive complication index, early allograft dysfunction, or acute kidney injury. Study protocol magnetic resonance cholangiopancreatography in cDCD patients showed no signs of subclinical biliary strictures. Evaluation of the Swedish national pilot of cDCD LTX with mandatory NRP shows comparable outcomes to a matched DBD cohort with 94.4% 1-year patient and graft survival and no incidence of NAS within the first year.

Usability testing of the Set Brave Goals app, a continence goal-selection app for children with spina bifida.

INTRODUCTION: The Set Brave Goals app is the first digital health app (DHA) aimed at helping children with spina bifida (SB) aged 8-17 years old to select and track their urinary and fecal continence goals. Developed by children, parents, providers and researchers, its usability, or appropriateness to a purpose ("user-friendliness"), remains unknown. In an alpha testing stage, our aim was to determine the usability of the app prior to clinical use. METHODS: We recruited children with SB and their parents in clinic and via social media. A recruitment goal of 10 children exceeded industry standard of 5 participants (10 participants are expected to identify approximately 96% of usability problems). Participants downloaded and used the app for a week. They completed a questionnaire, including the System Usability Scale (SUS) and closed and open-ended questions. The SUS is a 10-item validated usability questionnaire most widely used in DHA usability testing (scores range from 0 to 100, 100 representing greatest usability). Median/mean SUS scores for DHAs are 68. Mann-Whitney-U and t-tests were used. RESULTS: Ten children with SB participated (median age: 14 years old, 6 female, 8 shunted, 8 using bladder catheterizations). Twenty parents participated (17 mothers, median age: 42 years old). Median SUS score was 77.5 for children and 73.8 for parents, corresponding to "good" to "excellent" usability (Summary Figure). There were no significant differences between child/parent scores (p = 0.69) or those for other DHAs (p = 0.11). It took a median 5-10 min to go through the app (2 parents felt it took too much time). All participants felt the app was easy to understand and use (100%). Most participants would recommend it to children and parents (children: 89%, parents: 80%), and believed it would be valuable to children (90%, 75%) and urologists (80%, 80%). Free text responses related to more varied color schemes, keeping free-text notes within the app and more flexible goal/alarm setting. These changes were incorporated into the app. COMMENT: Usability testing prior to clinical launch identified areas for app improvement. Although this study sample met industry standards, findings are limited by a small group of participants. The app will undergo further refinement during prospective beta testing. CONCLUSIONS: The Set Brave Goals app for children with SB has acceptable usability parameters. This justifies proceeding with wider use to formalize children's continence goal setting/tracking and testing its value to children with SB and their healthcare providers.