Hepatopulmonary Syndrome in Children: A 20-Year Review of Presenting Symptoms, Clinical Progression, and Transplant Outcome.

Hepatopulmonary syndrome (HPS) in stable patients with cirrhosis can easily be overlooked. We report on the presenting symptoms, disease progression, and outcomes after liver transplantation (LT) in children with HPS. Twenty patients were diagnosed with HPS between 1996 and 2016. The etiologies were as follows: biliary atresia (n = 9); alpha-1-antitrypsin deficiency (n = 2); cryptogenic liver disease (n = 3); and others (n = 6). HPS presentations were as follows; dyspnea (n = 17) and pneumonia (n = 3). For diagnostic confirmation, the following techniques were used: technetium-99m-labeled macroaggregated albumin lung perfusion scan (n = 13) or contrast echocardiogram (n = 7). There were 16 patients listed for LT, with a median age at HPS diagnosis of 10 years and an average wait from listing to LT of 9 weeks. A marked rise in hemoglobin (Hb; median, 125-143.5 g/L) and modest decrease in oxygen saturation (SpO2 ; median 91% to 88% room air) were evident over this time. Patients' need for assisted ventilation (1 day), pediatric intensive care unit (PICU) stay (3 days), and total hospital stay (20 days) were similar to our general LT recipients-the key difference in the postoperative period was the duration of supplementary O2 requirement. Hb of ≥130 g/L on the day of LT correlated with a longer PICU stay (P value = 0.02), duration of supplementary O2 (P value = 0.005), and the need for the latter beyond 7 days after LT (P value = 0.01). Fifteen patients had resolution of their HPS after LT. The 5-, 10-, and 20-year survival rates were unchanged at 87.5%. None had a recurrence of HPS. In conclusion, HPS is a life-threatening complication of cirrhosis which usually develops insidiously. This combined with the often-stable nature of the liver disease leads to delays in diagnosis and listing for LT. Progressive polycythemia extends the need for supplementary O2 and PICU stay. We advocate screening for HPS with a combination of SpO2 and Hb monitoring to facilitate earlier recognition, timely LT, and shortened recovery periods.

Paediatric Hepatology Dependency score (PHD score): an audit tool.

BACKGROUND: The aim was to develop a tool that measures patient dependency and disease severity in children with mild to severe liver disease. METHODS: The initial pilot score was based on known markers of disease severity in acute and chronic liver disease. Between 1997 and 2001, the score was modified 4 times and reduced to comprise 10 key parameters: aspartate transaminase, prothrombin time, albumin, bilirubin, ascites, nutritional support, organ dysfunction, blood product support, sepsis and intravenous access. The score's face validity, internal consistency, interobserver agreement and construct validity were evaluated statistically and by the use of endpoints such as survival and transplant after 6 months. RESULTS: The final 10 variable score was tested on 71 children admitted over two 3-month periods. The Cronbach's alpha score (a test for internal consistency) for the total score was 0.72. Serial data paralleled clinical deterioration and response to interventions. A Paediatric Hepatology Dependency score >15 had an odds ratio of 7 (P = 0.0125) for death or transplant at 6 months. The score was also found to agree with the paediatric end-stage liver disease score for the 12 patients being listed for liver transplantation (r = 0.660, P < 0.05). CONCLUSION: The Paediatric Hepatology Dependency score is valid and internally consistent and is a convenient measure of dependency and disease severity in a heterogeneous group of patients with liver disease. It also allows admissions to be audited for comparison between eras and for monitoring the progress of patients while on the ward.

Visual loss and idiopathic intracranial hypertension in children with Alagille syndrome.

BACKGROUND: Alagille syndrome (AGS) is an autosomal dominant, multisystem disorder defined by developmental abnormalities of the liver, heart, eye and skeleton. Although visual problems are recognised, the severity of visual loss and its link with idiopathic intracranial hypertension (IIH) has not been reported. AIM: To review the incidence of visual loss and IIH in children with AGS managed at a National Paediatric Liver Unit between 1989 and 2004. SUBJECTS AND METHODS: Retrospective case note review of children who fulfilled criteria for diagnosis of AGS and had an ophthalmic examination by a paediatric ophthalmologist. RESULTS: Fifty-five children with AGS were evaluated. Of these, 41 children fulfilled diagnostic criteria and had a documented ophthalmic examination. Six children had undergone liver transplantation. Three children had a definite diagnosis of IIH, 2 of whom developed postliver transplant. All 3 were treated medically, but 1 child with IIH required lumboperitoneal shunting. All 3 children with definite IIH have normal vision after treatment. Another child with probable undiagnosed IIH has bilateral optic atrophy and is registered blind. Two children with AGS are registered partially sighted, one with rod cone dystrophy and the other with pigmentary retinopathy and right disc atrophy. SUMMARY: Although visual abnormalities are well described in children with AGS, a minority of children have significant progressive visual loss. Idiopathic intracranial hypertension has been identified as a potentially treatable precipitating factor. CONCLUSIONS: We recommend annual fundoscopy in the follow-up of children with AGS to facilitate early detection and appropriate management of IIH to prevent visual loss.

A comparison of two validated scores for estimating risk of mortality of children with intestinal failure associated liver disease and those with liver disease awaiting transplantation.

BACKGROUND AND AIMS: To evaluate risk of mortality in children with intestinal failure associated liver disease (IFALD) compared with other liver disease using two validated scores. METHODS: Sixty-seven children listed for transplant were studied: cholestatic liver disease (CLDn23); liver disease secondary to other processes (LDsec n11); (IFALDn22), acute liver failure (ALFn11). Paediatric Hepatology Score (PHD) score and Pediatric end-stage liver disease score (PELD) were evaluated by Receiver Operating Curves (ROC), proportional hazards regression. RESULTS: The highest PHD and PELD scores were found in ALF; the lowest in LDsec. Both scores correlated well in identifying waiting list (WL) mortality in patients with CLD and ALF, but not in those with IFALD where PELD scores were lower. Cox proportional hazard regression of time spent on the waiting list prior to death or transplant/delisting showed significant associations with PHD (P=0.006) and PELD (P=0.008). WL mortality was strongly predicted by disease group (6/8 deaths in IFALD). ROC analysis of all data showed that a PHD score greater than 15.5 had sensitivity of 87.5% and specificity of 81% for waiting list mortality (P<0.001); PELD greater than 8 had a sensitivity of 87.5% and specificity of 40%. Neither PHD nor PELD scores correlated with post-transplant mortality. CONCLUSION: PHD and PELD scores had the same sensitivity for identifying risk of WL mortality in all patients, but PELD failed to identify the sickest children with IFALD, lowering its specificity. The PHD score has the added advantage for European centres of being in SI units, not requiring a computer application to calculate and was simpler to use at bedside.