An Ethical and Policy Analysis of Elective Transplantation for Metabolic Conditions Diagnosed by Newborn Screening

Journal of Pediatrics

Volumn 156, Issue 1, 2010, Pages 139-144

  Ross, Lainie Friedman ^a  

^a UNIVERSITY OF CHICAGO   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

ANTIBIOTIC AGENT; CARNITINE; HYDROXOCOBALAMIN;

ARTICLE; CLINICAL FEATURE; DECISION MAKING; DIET RESTRICTION; DOCTOR PATIENT RELATION; ETHICS; HEALTH CARE POLICY; HEMODIALYSIS; HEMOFILTRATION; HUMAN; LIVER TRANSPLANTATION; MAPLE SYRUP URINE DISEASE; METHYLMALONIC ACIDEMIA; NEWBORN SCREENING; PRACTICE GUIDELINE; PRIORITY JOURNAL; PROTEIN RESTRICTION;

ETHICS, CLINICAL; HEALTH POLICY; HUMANS; INFANT, NEWBORN; LIVER TRANSPLANTATION; MAPLE SYRUP URINE DISEASE; METABOLISM, INBORN ERRORS; METHYLMALONIC ACID; NEONATAL SCREENING; SURGICAL PROCEDURES, ELECTIVE;

EID: 72149100197     PISSN: 00223476     EISSN: None     Source Type: Journal    
DOI: 10.1016/j.jpeds.2009.06.067     Document Type: Article

References (46)

1. Strauss K.A., Mazariegos G.V., Sindhi R., Squires R., Finegold D.N., Vockley G., et al. Elective liver transplantation for the treatment of classical maple syrup urine disease. Am J Transplant 6 (2006) 557-564
2. Data from the United Network of Organ Sharing/Organ Procurement and Transplantation Network (UNOS/OPTN). Based on OPTN data as of Jan 9, 2009. Available at: http://www.optn.transplant.hrsa.gov/latestData/viewDataReports.asp. Accessed Aug 1, 2009.
3. American College of Medical Genetics. Newborn screening: toward a uniform screening panel and system. Genet Med 8 Suppl (2006) 1-252 S
4. National Newborn Screening Report. Updated 7/7/2009. Available at: http://genes-r-us.uthscsa.edu/nbsdisorders.pdf. Accessed Aug 1, 2009.
5. Strauss KA, Puffenberger EG, Morton DH. Maple syrup urine disease. Initial posting Jan 30, 2006 on GeneReviews. Available at: http://www.ncbi.nlm.nih.gov/bookshelf/br.fcgi?book=gene&part=msud. Accessed Aug 1, 2009.
6. Morton D.H., Strauss K.A., Robinson D.L., Puffenberger E.G., and Kelley R.I. Diagnosis and treatment of maple syrup disease: a study of 36 patients. Pediatrics 109 (2002) 999-1008
7. Venditti CP. Methylmalonic acidemia. Initial posting Aug 16, 2005; revised Jan 18, 2007. Available at: http://www.ncbi.nlm.nih.gov/bookshelf/br.fcgi?book=gene&part=mma. Accessed Aug 1, 2009.
8. Zwickler T., Lindner M., Aydin H.I., Baumgartner M.R., Bodamer O.A., Burlina A.B., et al. Diagnostic work-up and management of patients with isolated methylmalonic acidurias in European metabolic centres. J Inher Metab Dis 31 (2008) 361-367
9. McGuire P.J., Lim-Melia E., Diaz G.A., Raymond K., Larkin A., Wasserstein M.P., et al. Combined liver-kidney transplant for the management of methylmalonic aciduria: a case report and review of the literature. Molec Genet Metab 93 (2008) 22-29
10. van't Hoff W., McKiermana P.J., Surtees R.A.H., and Leonard J.V. Liver transplantation for methlmalonic acidaemia. Eur J Pediatrics 158 Suppl 2 (1999) S70-S74
11. Horster F., and Hoffmann G.F. Pathophysiology, diagnosis, and treatment of methlmalonic aciduria-recent advances and new challenges. Pediatric Nephr 19 (2004) 1071-1074
12. Kaplan P., Ficicioglu C., Mazur A.T., Palmieri M.J., and Berry G.T. Liver transplantation is not curative for methylmalonic acidopathy caused by methylmalonyl-coA mutase deficiency. Molec Genet Metab 88 (2006) 322-326
13. Leonard J.V., Walter J.H., and McKiernan P.J. The management of organic acidaemias: the role of transplantation. J Inher Metab Dis 24 (2001) 309-311
14. Nyhan W.L., Gargus J.J., Boyle K., Selby R., and Koch R. Progressive neurologic disability in methylmalonic academia despite transplantation of the liver. Eur J Pediatr 161 (2002) 377-379
15. Morioka D., Kasahara M., Horkawa R., Yokoyama S., Fukuda A., and Nakagawa A. Efficacy of living donor liver transplantation for patients with methylmalonic acidemia. Am J Transplant 7 (2007) 2782-2787
16. Beauchamp T.L., and Childress J.F. Principles of biomedical ethics. 5th ed. (2001), Oxford University Press, New York
17. Veatch R.M. Transplantation ethics (2000), Georgetown University Press, Washington, DC
18. Rawls J. A theory of justice (1971), Belknap Press of Harvard University Press, Cambridge, MA
19. Lee K.W., Cameron A.M., Maley W.R., Segev D.L., and Montgomery R.A. Factors affecting graft survival after adult/child split-liver transplantation: analysis of the UUNOS/OPTN data base. Am J Transplant 8 (2008) 1186-1196
20. Gaicomoni A., Lauterio A., Donadon M., de Gasperi A., Belli L., Slim A., et al. Should we still offer split-liver transplantation for two adult recipients? A retrospective study of our experience. Liver Transplant 14 (2008) 999-1006
21. Aronsohn A., Thistlethwaite Jr. J.R., Jensen D., and Ross L.F. Is there a justification for the attitudes and practices of hepatologists and transplant surgeons regarding transplantation in patients with alcoholic liver disease? Poster presented at Digestive Disease Week 2008. San Diego CA (May 2008)
22. Morioka D., Takada Y., Kasahara M., Ito T., Uryuhara K., Ogawa K., et al. Living donor liver transplantation for noncirrhotic inheritable metabolic liver diseases: impact of the use of heterozygous donors. Transplant 80 (2005) 623-628
23. Kawagishi N., Satoh K., Akamatsu Y., Sekiguchi S., Ishigaki Y., Okawa S., et al. Long-term outcome after living donor liver transplantation for two cases of homozygous familial hypercholesterolemia from a heterozygous donor. J Atherosclerosis Thrombosis 14 (2006) 94-98
24. Ban K., Sugiyama N., Sugiyama K., Wada Y., Suzuki T., Hashimoto T., et al. A pediatric patient with classical citrullinemia who underwent living-related partial liver transplantation. Transplantation 71 (2001) 1495-1497
25. Nagasaka H., Yorifuji T., Egawa H., Kikuta H., Tanaka K., and Kobayashi K. Successful living-donor liver transplantation from an asymptomatic carrier mother in ornithine transcarbamylase deficiency. J Pediatr 138 (2001) 432-434
26. Barshop B.A., and Khanna A. Domino hepatic transplantation in maple syrup urine disease. N Eng J Med 353 22 (2005) 2410-2411
27. Khanna A., Hart M., Nyhan W.L., Hassanein T., Panyard-Davis J., and Barshop B.A. Domino liver transplantation in maple syrup urine disease. Liver Transplant 12 (2006) 876-882
28. Ericzon B- G. Domino transplantation using livers from patients with familial amyloidotic polyneuropathy: should we halt?. Liver Transplant 13 (2007) 185-187
29. Children's Hospital of Pittsburgh. Domino transplant allows child with rare liver disease to receive new organ, pass his on to another patient waiting for liver donor. Jul 18, 2007, Press Release. Available at: http://www.chp.edu/CHP/071807. Accessed Aug 1, 2009.
30. Farese S., Trost N., Candinas D., and Huynh-Do U. Early renal failure after domino hepatic transplantation using the liver from a compound heterozygous patient with primary hyperoxaluria. Nephrol Dial Transplant 20 (2005) 2557-2560
31. Donckier V., Nakadi I.E., Closset J., Ickx B., Louis H., Le Moine O., et al. Domino hepatic transplantation using the liver from a patient with primary hyperoxaluria. Transplantation 71 (2001) 1346-1348
32. Wilczek H.E., Larsson M., Yamamoto S., and Ericzon B- G. Domino liver transplantation. J Hepatobiliary Pancreatic Surg 15 (2008) 139-148
33. Advisory Committee on Heritable Disorders in Newborns and Children. Minutes of the second meeting, September 2004. Available at: http://www.hrsa.gov/heritabledisorderscommittee/meetings/2004september/2 ndmeeting.htm. Accessed Aug 1, 2009.
34. Keith D., Ashby V.B., Port F.K., and Leichtman A.B. Insurance type and minority status associated with large disparities in prelisting dialysis among candidates for kidney transplantation. Clin J Am Soc Nephr CJASN 3 (2008) 463-470
35. Axelrod D.A., Guidinger M.K., Finlayson S., Schaubel D.E., Goodman D.C., Chobanian M., et al. Rates of solid-organ wait-listing, transplantation, and survival among residents of rural and urban areas. JAMA 299 (2008) 202-207
36. Reid A.E., Resnick M., Chang Y., Buerstatte N., and Weissman J.S. Disparity in use of orthotopic liver transplantation among blacks and whites. Liver Transplant 10 (2004) 834-841
37. Garg P.P., Diener-West M., and Powe N.R. Reducing racial disparities in transplant activation: whom should we target?. Am J Kidney Dis 37 (2001) 921-931
38. Segev D.L., Kucirka L.M., Oberai P.C., Parekh R.S., Boulware L.E., Powe N.R., et al. Age and comorbidities are effect modifiers of gender disparities in renal transplantation. J Amer Soc Nephrol (2009) In press
39. Kotlyar D.S., Burke A., Campbell M.S., and Weinrieb R.M. A critical review of candidacy for orthotopic liver transplantation in alcoholic liver disease. Am J Gastroenterol 103 (2008) 734-743
40. Wiesner R., Lake J.R., Freeman R.B., and Gish R.G. Model for End-Stage Liver Disease (MELD) exception guidelines. Liver Transplant 12 Suppl 3 (2006) S85-S87
41. Olthoff K.M., Brown Jr. R.S., Delmonico F.L., Freeman R.B., McDiarmid S.V., Merion R.M., et al. Summary report of a national conference: evolving concepts in liver allocation in the MELD and PELD era. Liver Transplant 10 (2004) A6-22
42. Rodriguez-Luna H., Vargas H.E., Moss A., Reddy K.S., Freeman R.B., and Mulligan D. Regional variations in peer reviewed liver allocation under the MELD system. Am J Transplant 5 (2005) 2244-2247
43. Voigt M.D., Zimmerman B., Katz D.A., and Rayhill S.C. New neatonal liver transplant allocation policy: is the regional review board process fair?. Liver Transplant 10 (2004) 666-674
44. Freeman R.B. Mortatlity risk versus other endpoints: who should come first on the liver transplant waiting list?. Liver Transplant 10 (2004) 675-677
45. McDiarmid S., Gish R.G., Horslen S., and Mazariegos G.V. Model for End-stage Liver Disease (MELD) exception for unusual metabolic liver diseases. Liver Transplant 12 Suppl 3 (2006) S124-S127
46. Freeman Jr. R.B., Gish R.G., Harper A., Davis G.L., Vierling J., Lieblein L., et al. Model for End-Stage Liver Disease (MELD) exception guidelines: results and recommendations from the MELD Exception Study Group and Conference (MESSAGE) for the approval of patients who need liver transplantation with diseases not considered by the standard MELD formula. Liver Transplant 12 Suppl 3 (2006) S128-S136