Pediatric acute liver failure: Etiology,outcomes, and the role of serial pediatric end‐stage liver disease scores

To describe etiology, short‐term outcomes and prognostic accuracy of serial PELD scores in PALF. Retrospective analysis of children aged ≤16 yr, admitted with PALF under the QLTS, Brisbane, Australia, between 1991 and 2011. PELD‐MELD scores were ascertained at three time points (i) admission (ii), meeting PALF criteria, and (iii) peak value. Fifty‐four children met criteria for PALF, median age 17 months (1 day–15.6 yr) and median weight 10.2 kg (1.9–57 kg). Etiology was known in 69%: 26% metabolic, 15% infective, 13% drug‐induced, 6% autoimmune, and 9% hemophagocytic lymphohistiocytosis. Age <3 months and weight <4.7 kg predicted poor survival in non‐transplanted children. Significant independent predictors of poor outcome (death or LT) were peak bilirubin > 220 μm /L and peak INR > 4. Serial PELD‐MELD scores were higher in the 17 (32%) transplant recipients (mean: [i] 26.8, [ii] 31.8, [iii] 42.6); highest in the 12 (22%) non‐transplanted non‐survivors (mean: [i] 31.6, [ii] 37.2, [iii] 45.7) compared with the 25 (46%) transplant‐free survivors (mean: [i] 25.3, [ii] 26.0, [iii] 30.3). PELD‐MELD thresholds of ≥27 and ≥42 at (ii) meeting PALF criteria and (iii) peak predicted poor outcome (p < 0.001). High peak bilirubin and peak INR predict poor outcome and serial PELD‐MELD is superior to single admission PELD‐MELD score for predicting poor outcome.     

Orthotopic liver transplantation for children with Alagille syndrome

Arnon R, Annunziato R, Miloh T, Suchy F, Sakworawich A, Hiroshi S, Kishore I, Kerkar N. Orthotopic liver transplantation for children with Alagille syndrome.
Pediatr Transplantation 2010: 14:622–628. © 2010 John Wiley & Sons A/S. Abstract: AGS is an inherited disorder involving the liver, heart, eyes, face, and skeleton. Aim: To determine the outcome of LT in children with AGS compared to those with BA. Methods: Children with AGS and BA who had a LT between 10/1987 and 5/2008 were identified from the UNOS database. Results: Of 11 467 children who received a liver transplant, 461 (4.0%) had AGS and 3056 (26.7%) had BA. One‐ and five‐yr patient survival was significantly lower in patients with AGS in comparison with patients with BA (AGS; 82.9%, 78.4%, BA; 89.9%, 84%, respectively). Early death (<30 days from transplant) was significantly higher in AGS than in BA. One‐ and five‐yr graft survival was significantly lower in AGS than in BA (AGS; 74.7%, 61.5%, BA; 81.6%, 70.0%, respectively). Death from graft failure, neurological, and cardiac complications was significantly higher in patients with AGS than in patients with BA. Serum creatinine at transplant, prior LT, and cold ischemic time >12 h were identified as risk factors for death. Conclusion: Children with AGS were older at the time of LT and their one‐ and five‐yr patient and graft survival were significantly lower compared to BA. Risk factors for poor outcome in AGS after LT were identified.     

Results after the adoption of a MELD/PELD‐based liver allocation policy in Argentina

In July 2005, Argentina switched from a categorical liver allocation system to a MELD/PELD‐based policy for patients with CLD. To analyze WL outcomes and survival after LT in children. From January 2000 to December 2010, 923 children were registered. Two consecutive five‐yr periods were analyzed and compared: Era I (January 2000–July 2005) (n = 379) and Era II (July 2005–December 31, 2010) (n = 544). All data were prospectively collected and analyzed using the Kaplan–Meier method. After adopting the MELD/PELD system, WL registrations increased by 44% (from 379 to 544) and the number of LT increased by only 24% (from 278 to 365). However, three‐month WL mortality rate (32% to 18%, p < 0.0001, HR 2.002 CI 95% 1.5–2.8) decreased significantly. No significant differences were observed between Era 1 and II in one‐yr post‐LT survival (77.5% vs. 84.1%, p = 0.3053) and in acute re‐LT rate (9% vs. 5%, p = 0.1746). Under the MELD/PELD‐based allocation system in Argentina, mortality on the WL significantly decreased in children with CLD without affecting post‐LT survival, although reduced access to LT was observed.     

Prognostic factors for event‐free survival in liver transplantation for hepatoblastoma: A single‐center experience

Hepatoblastoma (HB) is the most common malignant liver tumor in children. Twenty percent of the cases may remain unresectable after neoadjuvant chemotherapy and, for these patients, liver transplant (LT) is an accepted therapeutic option. To analyze the risk factors to event‐free survival (EFS) that influence the clinical outcome of patients with HB receiving LT, we retrospectively analyzed 21 patients with HB who underwent LT between January 1, 2005, and May 1, 2018. Overall survival (OS) was 90%. The univariate analysis shows that the AFP level at the time of LT was associated with a higher risk of EFS. With a ROC curve analysis, we established a cutoff point value of AFP levels at 16 000 ng/dL, with a sensitivity of 71.43% and a specificity of 85.71%. Multivariate analysis showed that patients with higher values of pretransplant AFP (>16 000 ng/dL) had a significantly higher risk of EFS than those transplanted with lower levels (HR: 10.180; 95% CI: 1.54‐66.97; P = .02). Efforts should be made to improve the selection of candidates for LT for unresectable HB, aiming at a better definition of chemoresistance as a risk factor of poor outcomes.     

Tacrolimus‐related seizure after pediatric liver transplantation – A single‐center experience

To identify the risk factors for new‐onset seizures after pediatric LT and to assess their clinical implications and long‐term prognosis. The clinical and laboratory data of 27 consecutive children who underwent LT from January 2007 to December 2010 in our center were analyzed retrospectively. Patients were divided into seizures group and a non‐seizures group. Pre‐operative, intra‐operative, and post‐operative data were collected. Seizures occurred in four children, an incidence of 14.8%. All exhibited generalized tonic–clonic seizures within the first two wk after LT. Univariate analysis showed that the risk factors associated with seizures after pediatric LT included gender, pediatric end‐stage liver disease score before surgery, Child–Pugh score before surgery, serum total bilirubin after surgery, and trough TAC level. Multivariate analysis showed that trough TAC level was the only independent risk factor associated with the seizures. All children who experienced seizures survived with good graft function and remained seizure‐free without anti‐epileptic drugs over a mean follow‐up period of 33.7 ± 14.6 months. High trough TAC level was the predominant factor that contributed to seizures in the early post‐operative period after pediatric LT. High PELD and Child‐Pugh scores before LT and high post‐operative serum Tbil may be contributory risk factors for TAC‐related seizures.     

Waiting list outcome of Peld/Meld exceptions: A single‐center experience in Argentina

As PELD/MELD‐based allocation policy was adopted in Argentina in 2005, a system of exception points has been in place in order to award increased waitlist priority to those patients whose severity of illness is not captured by the PELD/MELD score. We aimed to investigate the WL outcome of patients with granted PELD/MELD exceptions. A retrospective cohort study was conducted in children under 18 years old. WL outcomes were evaluated using univariable analysis. From 07/2005 to 01/2014, 408 children were listed for LT. There were 304 classified by calculated PELD/MELD. During this time, 85 (30%) PELD/MELD exceptions were granted. In this cohort, 89.4% (76 of 85) were transplanted and 7.1% (6 of 85) died while on the WL. The remaining 3 pts (3.5%) were removed from the WL due to other causes. We compared the impact of PELD/MELD exceptions in those 85 patients to outcomes in 87 non‐exception patients with PELD/MELD ≥19 points. Patients with the exception had significantly better access to WL and lower WL mortality. Our data suggest that children listed by PELD/MELD exceptions had an advantage compared to children with CLD with equivalent PELD/MELD listing priorities.     

Living donor liver transplantation for biliary atresia – An Indian experience

LT has played a significant role in improving the outcome of children with BA. We review our five‐yr experience of LDLT for children with BA. Records of all children who underwent LDLT in our institution over a five‐yr period (August 2010–June 2015) were reviewed and those with a primary diagnosis of BA were selected for our study. Data were extracted from a prospectively maintained database. Additional data were collected by review of case notes and imaging studies. Analysis was carried out using standard statistical means. One hundred and thirty‐two children underwent LDLT at our center over the study period, of which 58 children (31 females) had a primary diagnosis of BA. Thirty‐three (56.9%) children had undergone a prior KPE and 25 (43.1%) had a primary LT. Thirty‐four children had at least one post‐op complication, of which 13 had minor complications (Clavien grades I and II) and 21 had major complications (Clavien grade >II). Thirty‐day survival was 96.6% and one‐yr survival was 91.4%. Univariate analysis of variables comparing children who did and did not have a KPE prior to LT showed that age at LT, weight at LT, PELD, and GRWR were significantly different. LDLT provides excellent outcomes in children with BA. Primary LDLT and LT after KPE provide equivalent results, although the former is technically more challenging as the child is younger.     

The efficacy of serum brain natriuretic peptide for the early detection of portopulmonary hypertension in biliary atresia patients before liver transplantation

Severe portopulmonary hypertension (POPH) is a contraindication for liver transplantation (LT) because of the high risk of postoperative heart failure. The early detection of POPH is important for patients with biliary atresia (BA). Brain natriuretic peptide (BNP) is known to be correlated with liver fibrosis in patients with liver cirrhosis. The aim of this study was to elucidate the efficacy of BNP measurement for the follow‐up of patients with BA. Thirty‐two patients with BA were identified from September 2011 to December 2016. As indices of liver fibrosis/cirrhosis, APRI (P < .0001), FIB‐4 (P < .0001), Child‐Pugh score (P < .0001), IV collagen (P = .0005), and hyaluronic acid (P = .0291) had high or moderate correlations with BNP. Patients with splenomegaly, esophageal varices, liver fibrosis, and collateral veins had significantly higher BNP levels than those without. Patients diagnosed with POPH had significantly higher BNP levels in comparison with those patients without (P = .0068). In contrast, PELD/MELD scores showed an almost negligible correlation with the BNP level. LT was successful in 3 asymptomatic BA patients with POPH who had high BNP levels despite the low PELD/MELD scores. In conclusion, routine serum BNP surveillance can be easy to predict asymptomatic POPH. This may help to identify POPH before it reaches a stage that would contraindicate LT.     

Survival outcomes scores (SOFT,BAR, and Pedi‐SOFT) are accurate in predicting post‐liver transplant survival in adolescents

SOFT and BAR scores utilize recipient, donor, and graft factors to predict the 3‐month survival after LT in adults (≥18 years). Recently, Pedi‐SOFT score was developed to predict 3‐month survival after LT in young children (≤12 years). These scoring systems have not been studied in adolescent patients (13–17 years). We evaluated the accuracy of these scoring systems in predicting the 3‐month post‐LT survival in adolescents through a retrospective analysis of data from UNOS of patients aged 13–17 years who received LT between 03/01/2002 and 12/31/2012. Recipients of combined organ transplants, donation after cardiac death, or living donor graft were excluded. A total of 711 adolescent LT recipients were included with a mean age of 15.2±1.4 years. A total of 100 patients died post‐LT including 33 within 3 months. SOFT, BAR, and Pedi‐SOFT scores were all found to be good predictors of 3‐month post‐transplant survival outcome with areas under the ROC curve of 0.81, 0.80, and 0.81, respectively. All three scores provided good accuracy for predicting 3‐month survival post‐LT in adolescents and may help clinical decision making to optimize survival rate and organ utilization.     

Pulmonary complications after liver transplantation in children: risk factors and impact on early post‐operative morbidity

Liver transplantation (LT) is associated with high post‐operative morbidity, despite excellent survival rates. With this retrospective study, we report the incidence of early and late pulmonary complications (PC) after LT, identify modifiable risk factors for PC and analyzed the role of PC in post‐operative ventilation duration and hospital length of stay. In a series of 79 children (0‐16 years) with LT over a 12 years period, early (<3 months post‐LT) and/or late (>3 months post‐LT) PC occurred in 68 patients (86%). Sixty‐four percent (64%) developed early major complications such as pulmonary edema, atelectasis, or pleural effusion. Atelectasis requiring an intervention (P ≤ .02), pulmonary edema (P ≤ .02), or elevated PELD/MELD scores (P = .05) were associated with an increase in total ventilation duration and length of stay in the ICU. Risk factors for early PC included preoperative hypoxemia (P = .005), low serum albumin at LT admission (P = .003), or early rejection (P = .002). About 20% of patients experienced late PC of which 81% were infections. Risk factor assessment prior to LT may ultimately help reduce early PC thereby possibly minimizing post‐operative morbidity and ICU length of stay.     

Hyponatremia increases mortality in pediatric patients listed for liver transplantation

Carey RG, Bucuvalas JC, Balistreri WF, Nick TG, Ryckman FR, Yazigi N. Hyponatremia increases mortality in pediatric patients listed for liver transplantation.
Pediatr Transplantation 2010: 14: 115–120. © 2009 John Wiley & Sons A/S.
Abstract:  To evaluate hyponatremia as an independent predictor of mortality in pediatric patients with end-stage liver disease listed for transplantation. We performed a single-center retrospective study of children listed for liver transplantation. We defined hyponatremia as a serum sodium concentration <130 mEq/L that persisted for at least seven days. The primary outcome was death on the waiting list. Ninety-four patients were eligible for the study. The prevalence of hyponatremia was 26%. Kaplan–Meier survival analysis demonstrated that patients with hyponatremia had decreased pretransplant survival compared with patients who maintained a serum sodium >130 mEq/L (p < 0.001). Univariable association analyses demonstrated death on the waiting list was also associated with higher median PELD scores at listing (p = 0.01), non-white race (p = 0.02), and age <1 yr (p = 0.001). Logistic regression analysis identified hyponatremia and non-white race as independently associated with pretransplant mortality [OR = 8.0 (95% CI: 1.4–45.7), p = 0.02 and OR = 6.3 (95% CI: 1.25–33.3), p = 0.03]. When hyponatremia was added to the PELD score, it was significantly better in predicting mortality than the PELD score alone ( c -statistic = 0.79, p = 0.03). Hyponatremia identifies a subset of pediatric patients with increased risk of pretransplant mortality and improves the predictive ability of the current PELD score.     

First liver transplantation for biliary atresia in children: The hidden effects of non‐centralization

The aim of our study was to determine the impact of initial orientation for medical and surgical care of children with BA on procedures and outcomes of the first LT. We retrospectively analyzed charts of children with BA who underwent first LT between 2006 and 2015. Patients were divided into two groups for comparison: a single‐center management group (from diagnosis to transplantation) and a secondarily referred group (children referred after failure of KP). We focused analysis on disease severity at transplantation, blood transfusion, and overall survival. One hundred and eighty‐five children were included. The median delay between pretransplant check‐up and transplantation was shorter in patients secondarily referred. A severe undernutrition was observed in 23.7% of children secondarily referred compared to 11.1% in children with a single‐center management (P = .024). At transplantation, INR and factor V level were higher in single‐center group patients (respectively, 67% vs 55%, P < .001 and 61% vs 49%, P = .002). The total of red blood cell and fresh frozen plasma administrated during procedure was two times higher in patients secondarily referred. Finally, patients with a single‐center management had a higher overall 12 months of survival rate (92.1% vs 83.1%, P = .033). In a country without low‐density population issues, the authors advocate an early referring to transplant center to further improving LT outcomes in children with BA.     

Artificial neural network is highly predictive of outcome in paediatric acute liver failure

Current prognostic models in PALF are unreliable, failing to account for complex, non‐linear relationships existing between multiple prognostic factors. A computational approach using ANN should provide superior modelling to PELD‐MELD scores. We assessed the prognostic accuracy of PELD‐MELD scores and ANN in PALF in children presenting to the QLTS, Australia. A comprehensive registry‐based data set was evaluated in 54 children (32M, 22F, median age 17 month) with PALF. PELD‐MELD scores calculated at (i) meeting PALF criteria and (ii) peak. ANN was evaluated using stratified 10‐fold cross‐validation. Outcomes were classified as good (transplant‐free survival) or poor (death or LT) and predictive accuracy compared using AUROC curves. Mean PELD‐MELD scores were significantly higher in non‐transplanted non‐survivors (i) 37 and (ii) 46 and transplant recipients (i) 32 and (ii) 43 compared to transplant‐free survivors (i) 26 and (ii) 30. Threshold PELD‐MELD scores ≥27 and ≥42, at meeting PALF criteria and peak, gave AUROC 0.71 and 0.86, respectively, for poor outcome. ANN showed superior prediction for poor outcome with AUROC 0.96, sensitivity 82.6%, specificity 96%, PPV 96.2% and NPV 85.7% (cut‐off 0.5). ANN is superior to PELD‐MELD for predicting poor outcome in PALF.     

Association of post‐transplant donor‐specific HLA antibody with liver graft fibrosis during long‐term follow‐up after pediatric liver transplantation

The aim of this study was to evaluate the significance of post‐transplant DSA as a predictor of liver fibrosis during long‐term follow‐up after pediatric LT. We evaluated the histological findings in 18 LT recipients who underwent liver biopsy after DSA screening. Liver fibrosis was scored based on the METAVIR fibrosis staging. Patients were divided into 2 groups based on histological findings, and clinical characteristics among patients with liver fibrosis were assessed. Of 18 patients, 7 were included in the fibrosis group. No significant between‐group differences were found regarding peritransplant characteristics, including age, sex, primary disease, ABO incompatibility, and immunosuppressive regimen. Episodes of acute rejection and non‐adherence to immunosuppressive drugs were comparable between both groups. The MFI for anti‐DR DSA and positive rate were significantly higher in the fibrosis group (1655 vs 216; P = .019, 86% vs 27%; P = .012, respectively). MFI for anti‐DQ DSA was higher in the fibrosis group, but non‐significantly (2052 vs 384; P = .46). Post‐transplant anti‐DR DSA is associated with graft fibrosis during long‐term follow‐up. This finding seems useful for the implementation of valid histological examinations of liver grafts for patients with higher MFI, especially for anti‐DR DSA, after pediatric LT.     

Outcome of liver re‐transplantation in children—Impact and special analysis of early re‐transplantation

In case of graft failure, re‐LTX is the only life‐saving option but it has been associated with inferior results. This study analyzes the outcome following pediatric re‐LTX with a main focus on the timely relation between initial transplant and re‐LTX. All pediatric LTX at our institution between 2000 and 2010 divided into patients with primary LTX and patients undergoing re‐LTX early (≤30 days) or late (>30 days) after previous LTX were analyzed. Two hundred and ninety‐eight primary LTX(79%), 33 early (9%), and 46 late (12%) re‐LTX were performed. Patient/graft survival was significantly worse for children undergoing early re‐LTX compared to primary LTX and late re‐LTX (p = 0.024/0.001 and p = 0.015/0.03). One‐/five‐yr graft survival rates were 66%/49% for early re‐LTX compared to 86%/76% for late re‐LTX and 90%/74% for primary LTX. The inferior results in children undergoing early re‐LTX were due to events occurring in the first six months with similar survival thereafter. No difference in outcome was evident after adjustment of the groups for high‐urgency status. Outcome was excellent for primary LTX and late re‐LTX, supporting late re‐LTX in children. Early re‐LTX takes an elevated risk of early graft loss and patient death; however, beyond the early postoperative period, the outcome was comparable.     

Kidney and liver transplantation in children with fibrocystic liver–kidney disease: Data from the US Scientific Registry of Transplant Recipients: 1990–2010

The natural history and survival of children with fibrocystic liver–kidney disease undergoing solid organ transplantation have infrequently been described. We report outcomes in a cohort of US children with fibrocystic liver–kidney disease receiving solid organ transplants over 20 yr. Retrospective cohort study of pediatric transplant recipients with diagnoses of fibrocystic liver–kidney disease from 1/1990 to 3/2010, using data from the SRTR. Subjects were categorized by the first transplanted organ: LT, KT, or SLK. Primary outcomes were death, re‐transplant, transplant of the alternate organ, or initiation of dialysis. Seven hundred and sixteen subjects were transplanted in this period. Median age at first transplant was 9.7 yr. Of the LT, 14 (19%) required a second liver transplant at median of 0.2 yr, and five (7%) required kidney transplant or dialysis at a median of 9.0 yr. Of the KT, 188 (31%) required a second kidney transplant or dialysis at a median of 5.9 yr. Twenty‐nine (5%) subsequently received liver transplant at a median of 6.0 yr. Among patients in this registry, far more children underwent kidney than liver transplants. The risk of subsequently needing transplantation of an alternate organ was low.     

Outcomes in pediatric hepatitis C transplant recipients: Analysis of the UNOS database

HCV may lead to the development of ESLD in late childhood and, consequently, contributes to the need for liver transplantation. The aim of this study was to examine post‐transplant outcomes in HCV‐positive pediatric patients with ESLD from any cause and to determine the impact of the PELD scoring system, introduced in February 2002, on post‐transplant patient and graft survival. A retrospective analysis of the UNOS database from 1994 to 2010 was performed to assess graft and patient survival in pediatric HCV‐seropositive liver transplant recipients. Graft survival and patient survival comparing subjects in the pre‐PELD era and post‐PELD era were analyzed using Kaplan–Meier statistics. Factors associated with survival were identified using Cox regression analysis. Of 120 pediatric HCV transplant recipients, 80 were transplanted in the pre‐PELD era and 40 were transplanted post‐PELD. Median serum total bilirubin, INR, and creatinine were 4.8 mg/dL, 1.6, and 0.7 mg/dL in the pre‐PELD era vs. 5.5 mg/dL, 1.7, and 0.6 mg/mL, respectively, in the post‐PELD era (p NS). One‐yr graft survival in the pre‐PELD vs. post‐PELD era was 65.0% and 89.7%, respectively (p < 0.01); corresponding three‐yr graft survival was 57.3% vs. 76.2% (p = 0.04). One‐yr patient survival in the pre‐PELD vs. post‐PELD era was 79.0% and 97.5%, respectively (p < 0.01); corresponding three‐yr survival was 79.0% vs. 89.4% (p = 0.17). Twenty‐eight patients (23.3%) were retransplanted: 24 (30%) in the pre‐PELD era (median time to retransplant 272 days) and four (10%) in the post‐PELD era (median time to retransplant 586 days). Early follow‐up demonstrates a trend toward improved pediatric HCV liver transplant graft and patient survival in the post‐PELD era. Superior outcomes may be attributed to pretransplant factors, improved surgical technique and better treatment options for HCV infection.     

Impact of liver transplantation on rate-corrected QT interval and myocardial function in children with chronic liver disease*

Prolonged QTc interval (>440 ms) is a common abnormality in adult patients with CLD and has been reported to predict patient survival. In this study, 88 children who underwent evaluation for LT, including a 12-lead electrocardiogram and echocardiogram included to determine the frequency of QTc prolongation and related factors in children with CLD and the effect of LT on these factors. Sixty-nine healthy, age- and sex-matched children served as controls. QTc interval was prolonged in 40 CLD patients (45.4%). It was found to be related to PELD score and presence of portal hypertension. Mean QTc was higher in patients who died prior to LT than in the survivors without LT. Mortality risk was increased 3.66-fold in patients with prolonged QTc (p = 0.001, 95% CI: 2-7.2). Cox regression analysis showed that only PELD score was an independent predictor of survival (p = 0.001, beta = -0.41, 95% CI: 5.58-1.82). Five of 48 transplanted children died within three months post-transplant; QTc was not related to post-transplant survival (p = 0.27). QTc normalized in 63.8% patients after LT. After LT, LAD, LVEF, and LVPWT decreased. In conclusion, QTc prolongation is common in children with CLD and associated with high mortality. It may be useful for assessment of the severity of CLD and for the timing for transplantation.     

Pediatric liver transplantation for neonatal‐onset Niemann‐Pick disease type C: Japanese multicenter experience

Niemann‐Pick disease type C (NPC) is a rare autosomal recessive inherited disease characterized by lysosomal accumulation of free cholesterol in macrophages within multiple organs. Infantile‐onset NPC often presents with jaundice and hepatosplenomegaly from birth, but these symptoms usually improve during early childhood, and it rarely progresses to liver failure. We report three cases from different hospitals in Japan; the patients developed neonatal‐onset NPC, and liver transplantation (LT) was performed as a life‐saving procedure. LT was performed at 19 days, 59 days, and 4 months of age, respectively. The last patient was diagnosed with NPC before LT, while the first two patients were diagnosed with neonatal hemochromatosis at LT. In these two patients, the diagnosis of NPC was made more than a year after LT. Even though oral administration of miglustat was started soon after the diagnosis of NPC, all patients showed neurological regression and required artificial respiratory support. All patients survived more than one year after LT; however, one patient died due to tracheal hemorrhage at 4.5 years of age, and another one patient was suspected as recurrence of NPC in liver graft. In conclusion, while LT may be a temporary life‐saving measure in patients with neonatal‐onset NPC leading to liver failure, the outcome is poor especially due to neurological symptoms. A preoperative diagnosis is thus critical.