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Is orthotopic liver transplant (OLTx) a good therapy for familial amyloid polineuropathy? Lisbon experience
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IS ORTHOTOPIC LIVER TRANSPLANT (OLTx) A GOOD THERAPY FOR F A M I L I A L AMYLOID POLINEUROPATHY? LISBON EXPERIENCE E. Monteiro*, A. Morbey*, A. Freire*, J. Veloso*, A. Martins*, J. Rebelo-Andrade*, E. Barroso*, M. L. Salles Luis**, P. Pinho e Costa***, J. Pena* Liver Transplant Unit, Curry Cabral Hospital, Lisbon*, Dept of Neurology, Santa Maria University Hospital**, Study Center of Paramiloidosis, St* Antonio Hospital, Oporto***. FAP is a fatal autosomal dominant inherited disorder. In type I FAP of Portuguese, Swedish aod Japanese origin, amyloid fibrils contain a variant transthyretin molecule: TTR-Met 30. Over 90% of its main production takes place in the liver. So far, some reports ~'2 suggest that OLTx can stop the progression of the disease. Aim of the study: 1. To confirm these preliminary findings. 2. To find out if there is any regression of the disease. Patients and methods: Thirteen FAP pts were submitted to OLTx from Sept 92 to July 95 with a follow-up of 10 days to 30 months.; 8 men and 5 women, age range 26-31 yrs, mean 39,2 yrs were submitted to OLTx. Duration of disease from 21 to 96 months. All pts had high levels of TTR-Met 30, polyneuropathy, autonomic disorders (diarrhoea and/or constipation) erectile sexual disfunetion, malnutrition; some had neurogenic bladder. Mild proteinfda was de~ected in four. Serum creatinin levels were normal in all. Results: Five pts (39%) had acute celular rejection and 9 (70%) had infections. Three pts developed chronic renal failure: one had a kidney Tx, 2 required hemodialysis. Overall mortality was 23%; 3 pts - one from. multiple organ failure at 3 days and two from sepsis at 3 and 30 months. TTR-MCt 30, cleared from the serum after OLTx. Diarrhoea and erectile sexual dysfunction improved at 6 to 12 months in 31% and 43% respectively. Weight gain after 1 yr in 4 pts. Subjective regression of polyneuropathy in 30% pts. Conclusions: I.OLTx seems to stop disease progression in FAP pts. 2. TTR-Met 30 clears from the serum. 3. Regression of autonomic complaints and weight gain after 1 year. 4. Regression of subjective signs of polyneuropathy after 36 months. 5. A latent renal insufficiency must be carefully traced before OLTx. 1. G. Holmgren et al. Lancet, 1993; 341:1115-6 2. E. Monteiro et al. Hepatology, 1994; 20:120,4
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LIVING-RELATED LIVER TRANSPLANTATION FOR THE PATIENTS WITH FAMILIAL AMYLOID POLYNEUROPATHY Yo-ichi Takeil), Shu-ichi Ikedal), Nobuo Yanagisawal) Hidetoshi Matsunami2), Seiji Kawasaki2), and Masatoshi Maku-uchi3) 1) Department of Medicine (Neurology), Shinshu University School of Medicine, Matsumoto, 390 Japan 2) First Department of Surgery, Shinshu University School of Medicine 3) Second Department of Surgery, Tokyo University Faculty of Medicine Objectives a n d Method: In Japan, brain death has not been commonly accepted and therefore, we evaluated the indication and efficacy of living-related liver transplantation for six patients of familial amyioid polyneuropathy (FAP) due to the transthyretin Met30 variant (ranging 3 to 10 years after the onset of symptoms of FAP). Result: Four FAP patients (Case 1,2,3,4) underwent living-related liver transplantation from November 1993 to May 1995. In the first patient (Case 1: 31y.o. female), her neurological symptoms rapidly improved and she returned to her previous social life. Case 2 (47y.o. female) was an advanced stage of FAP, and after operation she developed complete atrioventrieular block due to the side effect of immunosuppressive agent (FK506). She got a permanent pacemaker, and immunosuppressive agent was changed to eyelosporin A. She discharged 10months after transplantation. Case 3 (46y.o. female) suffered from severe FAP symptoms which started 10years ago. She died three months after transplantation ~_.cause of multifocal infections. Case 4 (43y.o. male), 5 years after onset of FAP, underwent living-related liver tmasplantation from his elder sister (47y.o.) on May 1995, resulting in good recovery. The other two patients (Case 5,6) could not find the appropriate donors. They strongly wanted to get liver transplantation and had gone to Australia, and one patient underwent orthotopic cadaveric liver transplantation at Princess Alexandra Hospital in Australia. Conclusion: Living-related liver transplantation is promising for the treatment of FAP but we need to examine the recipients and donors carefully.
IS ORTHOTOPIC LIVER TRANSPLANT (OLTx) A GOOD THERAPY FOR F A M I L I A L AMYLOID POLINEUROPATHY? LISBON EXPERIENCE E. Monteiro*, A. Morbey*, A. Freire*, J. Veloso*, A. Martins*, J. Rebelo-Andrade*, E. Barroso*, M. L. Salles Luis**, P. Pinho e Costa***, J. Pena* Liver Transplant Unit, Curry Cabral Hospital, Lisbon*, Dept of Neurology, Santa Maria University Hospital**, Study Center of Paramiloidosis, St* Antonio Hospital, Oporto***. FAP is a fatal autosomal dominant inherited disorder. In type I FAP of Portuguese, Swedish aod Japanese origin, amyloid fibrils contain a variant transthyretin molecule: TTR-Met 30. Over 90% of its main production takes place in the liver. So far, some reports ~'2 suggest that OLTx can stop the progression of the disease. Aim of the study: 1. To confirm these preliminary findings. 2. To find out if there is any regression of the disease. Patients and methods: Thirteen FAP pts were submitted to OLTx from Sept 92 to July 95 with a follow-up of 10 days to 30 months.; 8 men and 5 women, age range 26-31 yrs, mean 39,2 yrs were submitted to OLTx. Duration of disease from 21 to 96 months. All pts had high levels of TTR-Met 30, polyneuropathy, autonomic disorders (diarrhoea and/or constipation) erectile sexual disfunetion, malnutrition; some had neurogenic bladder. Mild proteinfda was de~ected in four. Serum creatinin levels were normal in all. Results: Five pts (39%) had acute celular rejection and 9 (70%) had infections. Three pts developed chronic renal failure: one had a kidney Tx, 2 required hemodialysis. Overall mortality was 23%; 3 pts - one from. multiple organ failure at 3 days and two from sepsis at 3 and 30 months. TTR-MCt 30, cleared from the serum after OLTx. Diarrhoea and erectile sexual dysfunction improved at 6 to 12 months in 31% and 43% respectively. Weight gain after 1 yr in 4 pts. Subjective regression of polyneuropathy in 30% pts. Conclusions: I.OLTx seems to stop disease progression in FAP pts. 2. TTR-Met 30 clears from the serum. 3. Regression of autonomic complaints and weight gain after 1 year. 4. Regression of subjective signs of polyneuropathy after 36 months. 5. A latent renal insufficiency must be carefully traced before OLTx. 1. G. Holmgren et al. Lancet, 1993; 341:1115-6 2. E. Monteiro et al. Hepatology, 1994; 20:120,4
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LIVING-RELATED LIVER TRANSPLANTATION FOR THE PATIENTS WITH FAMILIAL AMYLOID POLYNEUROPATHY Yo-ichi Takeil), Shu-ichi Ikedal), Nobuo Yanagisawal) Hidetoshi Matsunami2), Seiji Kawasaki2), and Masatoshi Maku-uchi3) 1) Department of Medicine (Neurology), Shinshu University School of Medicine, Matsumoto, 390 Japan 2) First Department of Surgery, Shinshu University School of Medicine 3) Second Department of Surgery, Tokyo University Faculty of Medicine Objectives a n d Method: In Japan, brain death has not been commonly accepted and therefore, we evaluated the indication and efficacy of living-related liver transplantation for six patients of familial amyioid polyneuropathy (FAP) due to the transthyretin Met30 variant (ranging 3 to 10 years after the onset of symptoms of FAP). Result: Four FAP patients (Case 1,2,3,4) underwent living-related liver transplantation from November 1993 to May 1995. In the first patient (Case 1: 31y.o. female), her neurological symptoms rapidly improved and she returned to her previous social life. Case 2 (47y.o. female) was an advanced stage of FAP, and after operation she developed complete atrioventrieular block due to the side effect of immunosuppressive agent (FK506). She got a permanent pacemaker, and immunosuppressive agent was changed to eyelosporin A. She discharged 10months after transplantation. Case 3 (46y.o. female) suffered from severe FAP symptoms which started 10years ago. She died three months after transplantation ~_.cause of multifocal infections. Case 4 (43y.o. male), 5 years after onset of FAP, underwent living-related liver tmasplantation from his elder sister (47y.o.) on May 1995, resulting in good recovery. The other two patients (Case 5,6) could not find the appropriate donors. They strongly wanted to get liver transplantation and had gone to Australia, and one patient underwent orthotopic cadaveric liver transplantation at Princess Alexandra Hospital in Australia. Conclusion: Living-related liver transplantation is promising for the treatment of FAP but we need to examine the recipients and donors carefully.