Kidney and liver transplantation in patients with autosomal recessive polycystic kidney disease: a multicentric study

Chapal, Marion; Debout, A.; Salomon, Rémi; Roussey, G.; Burtey, Stéphane; Launay, Emma Allain; Vigneau, Cécile; Blancho, Gilles; Loirat, Chantal; Hourmant, Maryvonne; Fakhouri, Fádi

doi:10.1093/ndt/gfr588

Cited by 34 publications

(35 citation statements)

References 17 publications

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“…Among 78 patients in the USA, the 1-, 3-, and 5-year survival rates were 79.5, 75.5, and 75.5 %, respectively [1]. Similar percentages have been reported in single-center reports [50]. In our center, four of the 14 transplanted ARPKD patients had CLKT as the first operation, and five first underwent KT followed by CLKT at a later date.…”

Section: Long-term Outcomesupporting

confidence: 85%

“…On the other hand, refractory complications of portal hypertension and recurrent cholangitis suggest CLKT as the first option (Table 3). Severe pathological findings of the liver (cirrhosis, large and infected biliary dilatations) and genotype (truncating mutations) also affect the treatment strategy [50]. Many of the children need KT later on, and simultaneous CLKT from the same donor probably provides better long-term kidney function than a sequential transplantation.…”

Section: Autosomal Recessive Polycystic Kidney Diseasementioning

confidence: 99%

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Combined liver and kidney transplantation in children

Jalanko

Pakarinen

2013

Pediatr Nephrol

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Simultaneous combined liver-kidney transplantation (CLKT) is a rare operation in pediatric patients so that annually only 10-30 operations are performed worldwide. The main indications for CLKT are primary hyperoxaluria type 1 and autosomal recessive polycystic kidney disease. In addition, CLKT is indicated in individual patients with metabolic or cirrhotic liver diseases and end-stage kidney disease. The surgery and immediate post-operative management of CLKT remain challenging in infants and small children. The patients should be operated on before they become severely ill or develop major systemic manifestations of their metabolic disorder. The liver allograft is immunologically protective of the kidney graft in simultaneous CLKT, often resulting in well-preserved kidney function. The long-term outcome after CLKT is nowadays comparable to that of isolated liver and kidney transplantations.

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Section: Long-term Outcomesupporting

confidence: 85%

Section: Autosomal Recessive Polycystic Kidney Diseasementioning

confidence: 99%

Combined liver and kidney transplantation in children

Jalanko

Pakarinen

2013

Pediatr Nephrol

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“…97,98 Others have suggested earlier consideration of liver transplantation (LT) in ARPKD patients with severe liver disease who are being evaluated for kidney transplantation, even if they may not otherwise meet criteria for LT-based liver disease severity alone. 85,[98][99][100] In patients with liver-predominant disease who first undergo LT, acceleration of kidney disease progression is also a concern. 101 Currently, the only guidelines regarding listing for combined kidney-liver transplantation (CKLT) are in the context of end-stage liver disease due to hepatocellular dysfunction, for which listing for CKLT is recommended when estimated glomerular filtration rate is #30 mL/min/1.73m 2 .…”

Section: Dialysis and Transplantationmentioning

confidence: 99%

“…Studies in patients with ARPKD who underwent CKLT have reported patient survival rates of 70% to 100%. 99,103 …”

Section: Dialysis and Transplantationmentioning

confidence: 99%

Autosomal Recessive Polycystic Kidney Disease: A Hepatorenal Fibrocystic Disorder With Pleiotropic Effects

2014

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Autosomal recessive polycystic kidney disease (ARPKD) is an important cause of chronic kidney disease in children. The care of ARPKD patients has traditionally been the realm of pediatric nephrologists; however, the disease has multisystem effects, and a comprehensive care strategy often requires a multidisciplinary team. Most notably, ARPKD patients have congenital hepatic fibrosis, which can lead to portal hypertension, requiring close follow-up by pediatric gastroenterologists. In severely affected infants, the diagnosis is often first suspected by obstetricians detecting enlarged, echogenic kidneys and oligohydramnios on prenatal ultrasounds. Neonatologists are central to the care of these infants, who may have respiratory compromise due to pulmonary hypoplasia and massively enlarged kidneys. Surgical considerations can include the possibility of nephrectomy to relieve mass effect, placement of dialysis access, and kidney and/or liver transplantation. Families of patients with ARPKD also face decisions regarding genetic testing of affected children, testing of asymptomatic siblings, or consideration of preimplantation genetic diagnosis for future pregnancies. They may therefore interface with genetic counselors, geneticists, and reproductive endocrinologists. Children with ARPKD may also be at risk for neurocognitive dysfunction and may require neuropsychological referral. The care of patients and families affected by ARPKD is therefore a multidisciplinary effort, and the general pediatrician can play a central role in this complex web of care. In this review, we outline the spectrum of clinical manifestations of ARPKD and review genetics of the disease, clinical and genetic diagnosis, perinatal management, management of organ-specific complications, and future directions for disease monitoring and potential therapies.

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“…The authors reason that the complications of hepatic involvement (predominantly the risk of ascending cholangitis) (19) coupled with improved outcomes of liver transplantation mandate a careful assessment of risk-benefit ratio in all ARPKD patients with severe dual organ involvement. The authors assert that lower Pediatric End Stage Liver Disease/Model for End Stage Liver Disease (PELD/MELD) scores can be used to justify the benefit of combined liver–kidney transplant compared with isolated kidney transplants.…”

Section: From Pathophysiology To Current and Future Therapies Pgd/ivfmentioning

confidence: 99%

Pathophysiology of childhood polycystic kidney diseases: new insights into disease-specific therapy

Sweeney

Avner

2013

Pediatr Res

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ADPKD and ARPKD are a significant cause of morbidity and mortality in children and young adults. ADPKD with an incidence of 1:400–1,000 affects over 13 million individuals worldwide, and is a major cause of end-stage renal disease (ESRD) in adults. However, symptomatic disease is increasingly recognized in children. ARPKD is a dual organ hepatorenal disease with an incidence of 1:20,000 to 1:40,000 and a heterozygote carrier rate of 1 in 70. Currently, no clinically significant disease-specific therapy exists for ADPKD or ARPKD. The genetic basis of both ADPKD and ARPKD have been identified, and delineation of the basic molecular and cellular pathophysiology have led to the discovery that abnormal ADPKD and ARPKD gene products interact to create “polycystin" complexes” located at multiple sites within affected cells. The integrated signaling of such complexes leads to abnormal cellular proliferation, altered cellular transport, and abnormal matrix-vascular biology. This review will focus on the molecular and cellular basis of the abnormal cystic phenotype, and review the clinical translation of such basic data into new therapies, which promise to alter the natural history of disease for children with genetic PKDs.

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Kidney and liver transplantation in patients with autosomal recessive polycystic kidney disease: a multicentric study

Cited by 34 publications

References 17 publications

Combined liver and kidney transplantation in children

Combined liver and kidney transplantation in children

Autosomal Recessive Polycystic Kidney Disease: A Hepatorenal Fibrocystic Disorder With Pleiotropic Effects

Pathophysiology of childhood polycystic kidney diseases: new insights into disease-specific therapy

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