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خرید پکیج
تعداد آیتم قابل مشاهده باقیمانده : 3 مورد
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Transplantation of allogeneic hepatocytes in patients with inherited metabolic diseases

Transplantation of allogeneic hepatocytes in patients with inherited metabolic diseases
Diagnosis [reference] Age Procedure and outcome
Alpha-1 antitrypsin deficiency
[1,2] 4.5 months Orthotopic liver transplantation (OLT) 2 days after hepatocyte transplantation. Explanted liver showed cirrhosis.
Crigler-Najjar syndrome type 1
[3] 10 years Total bilirubin reduced by 50%. Bilirubin glucuronide demonstrated in bile up to 3.5 years. Auxiliary liver transplantation (ALTx) after 4 years.
[4] 3.5 years Nine infusions were given. Serum bilirubin was reduced by 40% for at least 9 months. Ultimate outcome not reported.
[5] 9 years Serum bilirubin was reduced by 32%. OLT after 4.6 months.
[6] 8 years Serum bilirubin reduced by 30%. OLT after 11 months.
[7] 8 years Hepatocyte transplantation by one percutaneous infusion and eight infusions through an implanted mesenteric Port-a-Cath (PAC) over 5 months. Serum bilirubin was reduced by 40%. OLT after 20 months.
[8] 1.5 years Serum bilirubin was reduced by 40%. OLT after 8 months.
[8] 3.5 years No clear benefit.
[9] 2 years Hepatic progenitor cells were infused through the hepatic artery. Serum bilirubin was reduced by 50%. Ultimate outcome was not reported.
[10] 11 years Serum bilirubin was reduced by 20%. On waiting list for OLT.
[11] 7 months Serum bilirubin was reduced by 50%, bilirubin levels stable after 1 year. Improvement of psychomotor parameters.
Factor VII deficiency
[12] 3 months Requirement of rFVII was reduced by 70%. OLT after 7 months.
[12] 35 months Requirement of rFVII was reduced by 70%. OLT after 8 months.
[13] 4 months Requirement of rFVII was reduced. Ultimate outcome not reported.
Glycogen storage disease type 1a
[11] 6 years Hypoglycemic episodes were reduced. No admission for hypoglycemia at 1-year follow up after hepatocyte transplantation.
[14] 47 years No hypoglycemia on a normal diet on a 9-month follow up. Long-term outcome not reported.
Glycogen storage disease type 1b
[15] 18 years Normal glucose-6-phosphatase activity for 7 months. Ultimate outcome not reported.
Infantile Refsum's disease
[16] 4 years Pipecholic acid decreased by 40% and c26:c22 fatty acid ratio by 36% after 18 months. Ultimate outcome not reported.
Phenylketonuria
[17] 6 years Patient received hepatocytes from the explanted liver of a patient with glucogen storage disease 1a, and a second transplantation from a deceased donor 7.5 months later. Plasma phenylalanine levels were normalized for 3 months after the second infusion. The therapeutic effect waned after that and patient had to be reinstated on phenylalanine-restricted diet.
[18] 27 years Radiation pretreatment followed by hepatocyte transplantation with rejection risk monitoring was associated with lower post-transplant peripheral blood phenylalanine levels.
Primary hyperoxaluria type 1
[19] 33 months Reduction of plasma oxalate levels by 25 to 33%. OLT and kidney transplantation after 12 months.
Progressive familial intrahepatic cholestasis type 2
[4,8] 32 months Patient had cirrhosis of the liver. No benefit was demonstrated by hepatocyte transplantation. OLT after 5 months.
[4,8] 16 months Patient had cirrhosis of the liver. No benefit was demonstrated by hepatocyte transplantation. OLT after 14 months.
Tyrosinemia type 1
[11] 45 days Serum bilirubin levels decreased and there was improvement of coagulopathy. OLT after 45 days. Explanted liver had established cirrhosis.
Urea cycle disorders
Ornithine transcarbamylase (OTC) deficiency
[20] 5 years Blood ammonia level decreased. Biopsy on day 28 showed 0.5% of the OTC activity in normal liver. Patient died of sepsis 42 days after hepatocyte transplantation.
[21] 10 hours Blood ammonia level decreased. Protein tolerance improved. OLT after 6 months.
[22] Newborn Hepatocytes were infused through the umbilical vein. Blood ammonia level was normal on normal diet. No neurological abnormality developed. OLT after 7 months.
[7] 14 months Catheter was malpositioned. Effect of hepatocyte transplantation was not reported. OLT after 6 months.
[23] 14 months Blood ammonia was reduced and urea production was increased. There was psychomotor improvement. OLT after 6 months.
[24] 1 day Blood ammonia was reduced and urea production was increased. Protein tolerance was improved. ALTx after 7 months.
[25] 6 hours Blood ammonia was reduced, urea production was increased, and urinary orotic acid excretion became normal. Patient died 4 months after hepatocyte transplantation.
[25] 9 days Blood ammonia was reduced, protein tolerance improved, and urinary orotic acid excretion became normal. On waiting list for OLT 6 months after hepatocyte transplantation.
[26] 5 years Blood ammonia was reduced and glutamine levels were normal. Patient died 45 days after hepatocyte transplantation.
[22] 1 day Blood ammonia was reduced, urea production increased, and protein tolerance improved. ALTx after 7 months. Patient remained neurologically normal.
[11] 12 years Blood ammonia was reduced, urea production increased, and glutamine levels were normal. Patient died of septicemia 30 days after hepatocyte transplantation.
[27] 11 days Blood ammonia was reduced, and the patient remained neurologically normal 3 months after hepatocyte transplantation.
[18] 7 months Radiation pretreatment followed by hepatocyte transplantation was associated with improved protein tolerance post-transplant. However, evaluation of allospecific CD154+ T-cytotoxic memory cells and their subsets indicated allograft rejection. Patient received liver transplantation at age 11 months.
Arginosuccinate lyase deficiency
[28] 42 months Blood ammonia was reduced and there was psychomotor improvement. OLT after 18 months.
[7] 3 years Blood ammonia was reduced. Ultimate outcome was not reported.
Carbomoyl phosphate synthase I deficiency
[29] 2.5 months Blood ammonia was reduced and urea production increased. Patient was on waiting list for OLT 11 months after hepatocyte transplantation.
Citrulinemia
[29] 3 years Blood ammonia was reduced, urea production was increased, and protein tolerance was improved. Ultimate outcome was not reported.
[18] 4 months Radiation pretreatment followed by hepatocyte transplantation and immunosuppression resulted in no therapeutic benefit. Evaluation of allospecific CD154+ T-cytotoxic memory cells and their subsets indicated allograft rejection, and the patient received liver transplantation 3.5 months after hepatocyte transplantation.
References:
  1. Fisher RA, Strom SC. Human hepatocyte transplantation: Worldwide results. Transplantation 2006; 82:441.
  2. Strom SC, Roy-Chowdhury J, Fox IJ. Hepatocyte transplantation for the treatment of human diseases. Semin Liver Dis 1999; 19:39.
  3. Fox IJ, Roy-Chowdhury J, Kaufman SS, et al. Treatment of Crigler-Najjar syndrome type 1 with hepatocyte transplantation. N Eng J Med 1998; 338:1422.
  4. Hughes RS, Mitry RR, Dhawan A. Hepatocyte transplantation for metabolic liver disease: UK experience. J Royal Soc Med 2005; 98:341.
  5. Ambrosino G, Varotto S, Strom SC, et al. Isolated hepatocyte transplantation for Crigler-Najjar syndrome type 1. Cell Transplant 2005; 14:151.
  6. Allen KJ, Mifsud NA, Williamson R, et al. Cell-mediated rejection results in allograft loss after liver cell transplantation. Liver Transpl 2008; 14:688.
  7. Darwish AA, Sokal E, Stephenne X, et al. Permanent access to the portal system for cellular transplantation using an implantable port device. Liver Transpl 2004; 10:1213.
  8. Dhawan A, Mitry RR, Hughes RD. Hepatocyte transplantation for liver-based metabolic disorders. J Inherit Metab Dis 2006; 29:431.
  9. Khan AA, Parveen N, Mahaboob VS, et al. Treatment of Crigler-Najjar syndrome type 1 by hepatic progenitor cell transplantation: A simple procedure for management of hyperbilirubinemia. Transplant Proc 2008; 40:1148.
  10. Meyburg J, Hoerster F, Schmidt J, et al. Monitoring of intraportal liver cell application in children. Cell Transplant 2010; 19:629.
  11. Ribes-Koninckx C, Ibars EP, Calzado Agrasot MA, et al. Clinical outcome of hepatocyte transplantation in four pediatric patients with inherited metabolic diseases. Cell Transplant 2012; 21:2267.
  12. Dhawan A, Mitry RR, Hughes RD, et al. Hepatocyte transplantation for inherited Factor VII deficiency. Transplantation 2004; 78:1812.
  13. Hughes RD, Mitry RR, Dhawan A, et al. Isolation of hepatocytes from livers from non-heart-beating donors for cell transplantation. Cell Transpl 2006; 12:713.
  14. Muraca M, Gerunda G, Neri D, et al. Hepatocyte transplantation as a treatment of glycogen storage disease type 1a. Lancet 2002; 359:317.
  15. Lee KW, Lee JH, Shin SW, et al. Hepatocyte transplantation for glycogen storate disease type Ib. Cell Transplant 2007; 16:629.
  16. Sokal EM, Smets F, Bourgois A, et al. Hepatocyte transplantation in a 4-year-old girl with peroxisomal biogenesis disease: Technique, safety and metabolic follow-up. Transplantation 2003; 76:735.
  17. Stephenne X, Debray FG, Smets F, et al. Hepatocyte Transplantation Using the Domino Concept in a Child With Tetrabiopterin Nonresponsive Phenylketonuria. Cell Transplant 2012; 21:2765.
  18. Soltys KA, Setoyama K, Tafaleng EN, et al. Host conditioning and rejection monitoring in hepatocyte transplantation in humans. J Hepatol 2017; 66:987.
  19. Beck BB, Habbig S, Dittrich K, et al. Liver cell transplantation in severe infantile oxalosis - a potential bridging procedure to orthotopic liver transplantation? Nephrol Dial Transplant 2012; 27:2984.
  20. Strom SC, Fisher RA, Rubinstein WS, et al. Transplantation of human hepatocytes. Transplant Proc 1997; 29:2101.
  21. Horslen SP, McCowan TC, Goertzen TC, et al. Isolated hepatocyte transplantation in an infant with a severe urea cycle disorder. Pediatrics 2003; 111:1262.
  22. Mitry RR, Dhawan A, Hughes RD, et al. One liver, three recipients - Segment 4 from split liver procedure as a source of hepatocytes for cell transplantation. Transplantation 2004; 77:1624.
  23. Stephenne X, Najimi M, Smets F, et al. Cryopreserved liver cell transplantation controls ornithine transcarbamylase deficient patient while awaiting liver transplantation. Am J Transplant 2005; 5:2058.
  24. Puppi J, Tan N, Mitry RR, et al. Hepatocyte transplantation followed by auxiliary liver transplantation - a novel treatment for ornithine transcarbamylase deficiency. Am J Transplant 2008; 8:452.
  25. Meyburg J, Das AM, Hoerster F, et al. One liver for four children: First clinical series of liver cell transplantation for severe neonatal urea cycle defects. Transplantation 2009; 87:636.
  26. Bohnen NI, Carron M, Reyes J, et al. Use of Indium-111-labeled hepatocytes to determine the biodistribution of transplanted hepatocytes through portal ven infusion. Clin Nucl Med 2000; 25:447.
  27. Ensawa S, Horikwa R, Yamamoto A, et al. Hepatocyte transplantation using a living donor reduced graft in a baby with ornithine transcarbamylase deficiency: a novel source of hepatocytes. Liver Transpl 2014; 20:391.
  28. Stephenne X, Najimi M, Sibille EM. Sustained engraftment and tissue enzyme activity after liver cell transplantation for arginosuccinate lyase deficiency. Gastroenterology 2006; 130:1317.
  29. Meyburg K. Alexandrova K, Barthold M, et al. Liver cell transplantation: Basic investigations for safe application in infants and small children. Cell Transplant 2009; 18:777.
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