Liver transplantation for end-stage liver disease associated with alpha-1-antitrypsin deficiency in children: pretransplant natural history, timing and results of transplantation

Filipponi, Franco; Soubrane, Olivier; Labrousse, F; Devictor, Denis; Bernard, Olivier; Valayer, J; Houssin, D

doi:10.1016/s0168-8278(05)80469-6

Cited by 71 publications

(35 citation statements)

References 28 publications

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“…Although reported series are small, the long-term outcome of these patients after liver transplantation appears to be excellent. 75,76 Recommendations Selected patients with severe liver disease secondary to alpha 1 -antitrypsin deficiency should be considered for liver transplantation (rating, III ACD).…”

Section: Metabolic Diseasesmentioning

confidence: 99%

Liver transplantation

Carithers

2000

Liver Transpl

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Liver transplantation has revolutionized the care of patients with end-stage liver disease. Liver transplantation is indicated for acute or chronic liver failure from any cause. Because there are no randomized controlled trials of liver transplantation versus no therapy, the efficacy of this surgery is best assessed by carefully comparing postoperative survival with the known natural history of the disease in question. The best examples of this are in primary biliary cirrhosis and primary sclerosing cholangitis, for which well-validated disease-specific models of natural history are available. There are currently relatively few absolute contraindications to liver transplantation. These include severe cardiopulmonary disease, uncontrolled systemic infection, extrahepatic malignancy, severe psychiatric or neurological disorders, and absence of a viable splanchnic venous inflow system. One of the most frequently encountered contraindications to transplantation is ongoing destructive behavior caused by drug and alcohol addiction. The timing of the surgery can have a profound impact on the mortality and morbidity of patients undergoing liver transplantation. Because of the long waiting lists for donor organs, the need to project far in advance when transplantation might be required has proven to be one of the greatest challenges to those treating patients with end-stage liver disease. Three important questions must be addressed in a patient being considered for liver transplantation: (1) when should the patient be referred for possible transplantation? (2) when should the patient be listed for transplantation? and (3) when is the patient too sick to have a reasonable chance of surviving the perioperative period?

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Section: Metabolic Diseasesmentioning

confidence: 99%

Liver transplantation

Carithers

2000

Liver Transpl

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“…Indeed, restrictive changes have been shown to regress within the first months after OLT [28]. This phenomenon has been observed even in children with structural pulmonary changes after OLT for cystic fibrosis [30] or alpha-l-antitrypsin deficiency [16]. Our data from long-term survivors eliminate potential confounding variables of the postoperative period, such as impaired motility of the right hemidiaphragm [33] or pleural effusions [23,28,371.…”

Section: Discussionmentioning

confidence: 58%

High prevalence of pulmonary diffusion abnormalities without interstitial changes in long-term survivors of liver transplantation

Ewert¹,

Mutze²,

Schachschal³

et al. 1999

Transplant International

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Abnormalities in lung function are frequent findings in patients with terminal stage chronic liver disease. While spirometric parameters improve early after liver transplantation, a reduction in diffusion capacity has been reported up to 15 months after transplantation. It is unknown to what extent this disturbance in gas exchange occurs among long term survivors after liver transplantation. We assessed lung function in terms of spirometry, and gas exchange as well as pulmonary morphology by high resolution computed tomography (HRCT) in 40 patients 38 months (median, range 20-147 months) after liver transplantation. The prevalence of restrictive or obstructive changes was not different from predicted values. For the whole group of longterm survivors the carbon monoxide transfer coefficient (KCO) was reduced to 71.3 + 12.0 % predicted ( P < 0.05). HRCT revealed interstitial changes in only 2/40 (5.0 %), emphysematous bullae in 2/40 (5.0 %) and pleural thickening in 9/40 (22,s Yo). Diffusion abnormalities are prevalent in the majority of patients after liver transplantation, whereas spirometric abnormalities are absent also in the long term. The high prevalence of impaired gas exchange and the absence of interstitial lesions imply that changes in pulmonary blood vessels are the most likely cause.

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“…Replacement of the cirrhotic liver results in acquisition of the donor phenotype, a rise in serum levels of al-AT and significant improvement in the quality of life [3,20]. In this series, all children are currently alive at a median follow-up of 40 months with a graft survival of 84 YO.…”

Section: Discussionmentioning

confidence: 93%

“…It has been proposed that the appropriate time is when signs of hepatic failure or decompensation begin to appear. Thus, persistent or recurrent jaundice has been associated with poor prognosis, while paucity of the interlobular bile ducts on liver biopsy has been considered both as a good [12] and a poor prognostic feature [3,4]. The timing of transplantation remains di€ficult.…”

Section: Discussionmentioning

confidence: 99%

Liver transplantation for alpha-1-antitrypsin deficiency in children

Prachalias¹,

Kalife²,

Francavilla³

et al. 2000

Transplant Int

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Alpha-1-antitrypsin (al-AT) deficiency is an inborn error of metabolism, which can cause liver disease. The condition is one of the most common genetic disorders in the Caucasian population. Here we review our experience with 21 children suffering from end-stage liver disease due to al-AT deficiency. All children are PIZZ homozygotes. Nineteen of them initially presented with neonatal jaundice and two with hepatosplenomegaly in childhood. Twenty-five liver transplantions were performed. All children are currently alive at a median followup of 40 months. Liver replacement provides the only definite treatment for children with end-stage liver disease associated with al-AT deficiency. Excellent results can be achieved by reducing waiting time for transplantation and by early referral to a liver transplant centre.

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Liver transplantation for end-stage liver disease associated with alpha-1-antitrypsin deficiency in children: pretransplant natural history, timing and results of transplantation

Cited by 71 publications

References 28 publications

Liver transplantation

Liver transplantation

High prevalence of pulmonary diffusion abnormalities without interstitial changes in long-term survivors of liver transplantation

Liver transplantation for alpha-1-antitrypsin deficiency in children

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