Biliary Atresia: Biliary‐Enteric Drainage or Primary Liver Transplant?

Kim, Heung Bae; Elisofon, Scott A.

doi:10.1002/hep.30935

Cited by 6 publications

(3 citation statements)

References 3 publications

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“…Because of the frequency of children ultimately requiring LT and 5-year graft survival >80% after LT in children, foregoing HPE for primary liver transplant (pLT) has been proposed as an alternative management strategy. (3)(4)(5) A recent observational study in California reported improved outcomes with pLT over HPE as the initial management. (6) However, LT is an expensive surgery with a recently estimated cost in children with BA of nearly $200,000 (7) compared with a cost of about $50,000 for HPE.…”

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confidence: 99%

Cost‐Effectiveness of Primary Liver Transplantation Versus Hepatoportoenterostomy in the Management of Biliary Atresia in the United States

et al. 2021

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Biliary atresia (BA) is the leading indication to perform a pediatric liver transplantation (LT). Timely hepatoportoenterostomy (HPE) attempts to interrupt the natural history and allow for enteric bile flow; however, most patients who are treated with HPE require LT by the age of 10 years. We determined the cost-effectiveness of foregoing HPE to perform primary LT (pLT) in children with BA compared with standard-of-care HPE management. A Markov model was developed to simulate BA treatment over 10 years. Costs were measured in 2018 US dollars and effectiveness in life-years (LYs). The primary outcome was incremental cost-effectiveness ratio (ICER) between treatments. Model parameters were derived from the literature. In the base model, we assumed similar LT outcomes after HPE and pLT. Sensitivity analyses on all model parameters were performed, including a scenario in which pLT led to 100% patient and graft survival after LT. Children undergoing HPE accumulated $316,692 in costs and 8.17 LYs per patient. Children undergoing pLT accumulated $458,059 in costs and 8.24 LYs per patient, costing $1,869,164 per LY gained compared with HPE. With parameter variation over plausible ranges, only post-HPE and post-LT costs reduced the ICER below a typical threshold of $100,000 per LY gained. On probabilistic sensitivity analysis, 93% of iterations favored HPE at that threshold. With 100% patient and graft survival after pLT, pLT cost $283,478 per LY gained. HPE is more economically favorable than pLT for BA. pLT is unfavorable even with no graft or patient loss. The ability to predict those patients who may experience high costs after HPE or low costs after LT may help identify those patients for whom pLT could be considered.

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confidence: 99%

Cost‐Effectiveness of Primary Liver Transplantation Versus Hepatoportoenterostomy in the Management of Biliary Atresia in the United States

et al. 2021

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“…Surprisingly, their rate of pLT was 50% which is much higher than any previously reported rates and calls into question the accuracy of the methods. Additionally, it does not take into account patients who underwent their KPE in another state [ 27 ]. While single-center retrospective studies lack the power to show significant association, large database studies lack granularity, accuracy and stringent data verification processes, and their results should, therefore, be interpreted with caution.…”

Section: Discussionmentioning

confidence: 99%

Primary Liver Transplantation vs. Transplant after Kasai Portoenterostomy for Infants with Biliary Atresia

Lemoine

LeShock

Brandt

et al. 2022

JCM

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Introduction: Primary liver transplants (pLT) in patients with biliary atresia (BA) are infrequent, since most babies with BA undergo a prior Kasai portoenterostomy (KPE). This study compared transplant outcomes in children with BA with or without a prior KPE. We hypothesized that pLT have less morbidity and better outcomes compared to those done after a failed KPE. Methods: A retrospective review of patients with BA transplanted at our institution was performed. Patients were included if they received a pLT or if they were transplanted less than 2 years from KPE. Outcomes were compared between those groups. Comparisons were also made based on era (early: 1997–2008 vs. modern: 2009–2020). p < 0.05 was considered significant. Results: Patients who received a pLT were older at diagnosis (141.5 ± 46.0 vs. KPE 67.1 ± 25.5 days, p < 0.001). The time between diagnosis and listing for transplant was shorter in the pLT group (44.5 ± 44.7 vs. KPE 140.8 ± 102.8 days, p < 0.001). In the modern era, the calculated PELD score for the pLT was significantly higher (23 ± 8 vs. KPE 16 ± 8, p = 0.022). Two waitlist deaths occurred in the KPE group (none in pLT, p = 0.14). Both the duration of transplant surgery and transfusion requirements were similar in both groups. There was a significant improvement in graft survival in transplants after KPE between eras (early era 84.3% vs. modern era 97.8%, p = 0.025). The 1-year patient and graft survival after pLT was 100%. Conclusions: Patient and graft survival after pLT are comparable to transplants after a failed KPE but pLT avoids a prior intervention. There was no significant difference in pre- or peri-transplant morbidity between groups other than wait list mortality. A multicenter collaboration with more patients may help demonstrate the potential benefits of pLT in patients predicted to have early failure of KPE.

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“…Schneider et al, for example, suggested that failure to normalize serum bilirubin within three months after Kasai should elicit prompt evaluation for LT as extended native liver survival is exceptional among these patients [50]. Moreover, though a "Kasai success predicting score" remains far from reality, other groups support more research in that direction as part of BA management-a strategy that would allow early primary transplantation [47,54,[57][58][59][60][61].…”

Section: Steadily Increasing Prioritization Requestsmentioning

confidence: 99%

Variability of Care and Access to Transplantation for Children with Biliary Atresia Who Need a Liver Replacement

Goyet

Illhardt

Chardot³

et al. 2022

JCM

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Background & Aims: Biliary atresia (BA) is the commonest single etiology indication for liver replacement in children. As timely access to liver transplantation (LT) remains challenging for small BA children (with prolonged waiting time being associated with clinical deterioration leading to both preventable pre- and post-transplant morbidity and mortality), the care pathway of BA children in need of LT was analyzed—from diagnosis to LT—with particular attention to referral patterns, timing of referral, waiting list dynamics and need for medical assistance before LT. Methods: International multicentric retrospective study. Intent-to-transplant study analyzing BA children who had indication for LT early in life (aged < 3 years at the time of assessment), over the last 5 years (2016–2020). Clinical and laboratory data of 219 BA children were collected from 8 transplant centers (6 in Europe and 2 in USA). Results: 39 patients underwent primary transplants. Children who underwent Kasai in a specialist -but not transplant- center were older at time of referral and at transplant. At assessment for LT, the vast majority of children already were experiencing complication of cirrhosis, and the majority of children needed medical assistance (nutritional support, hospitalization, transfusion of albumin or blood) while waiting for transplantation. Severe worsening of the clinical condition led to the need for requesting a priority status (i.e., Peld Score exception or similar) for timely graft allocation for 76 children, overall (35%). Conclusions: As LT currently results in BA patient survival exceeding 95% in many expert LT centers, the paradigm for BA management optimization and survival have currently shifted to the pre-LT management. The creation of networks dedicated to the timely referral to a pediatric transplant center and possibly centralization of care should be considered, in combination with implementing all different graft type surgeries in specialist centers (including split and living donor LTs) to achieve timely LT in this vulnerable population.

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Biliary Atresia: Biliary‐Enteric Drainage or Primary Liver Transplant?

Cited by 6 publications

References 3 publications

Cost‐Effectiveness of Primary Liver Transplantation Versus Hepatoportoenterostomy in the Management of Biliary Atresia in the United States

Cost‐Effectiveness of Primary Liver Transplantation Versus Hepatoportoenterostomy in the Management of Biliary Atresia in the United States

Primary Liver Transplantation vs. Transplant after Kasai Portoenterostomy for Infants with Biliary Atresia

Variability of Care and Access to Transplantation for Children with Biliary Atresia Who Need a Liver Replacement

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