Heart Failure Reviews

, Volume 22, Issue 3, pp 317–327 | Cite as

Heart transplantation in cardiac amyloidosis

  • Matthew Sousa
  • Gregory Monohan
  • Navin Rajagopalan
  • Alla Grigorian
  • Maya Guglin
Article

Abstract

“Cardiac amyloidosis” is the term commonly used to reflect the deposition of abnormal protein amyloid in the heart. This process can result from several different forms, most commonly from light-chain (AL) amyloidosis and transthyretin (ATTR) amyloidosis, which in turn can represent wild-type (ATTRwt) or genetic form. Regardless of the origin, cardiac involvement is usually associated with poor prognosis, especially in AL amyloidosis. Although several treatment options, including chemotherapy, exist for different forms of the disease, cardiac transplantation is increasingly considered. However, high mortality on the transplantation list, typical for patients with amyloidosis, and suboptimal post-transplant outcomes are major issues. We are reviewing the literature and summarizing pros and cons of listing patients with amyloidosis for cardiac or combine organ transplant, appropriate work-up, and intermediate and long-term outcomes. Both AL and ATTR amyloidosis are included in this review.

Keywords

Heart transplantation Heart failure Cardiac amyloidosis 

References

  1. 1.
    Falk RH, Alexander KM, Liao R, Dorbala S (2016) AL (light-chain) cardiac amyloidosis: a review of diagnosis and therapy. J Am Coll Cardiol 68:1323–1341CrossRefPubMedGoogle Scholar
  2. 2.
    Coelho T, Adams D, Silva A et al (2013) Safety and efficacy of RNAi therapy for transthyretin amyloidosis. N Engl J Med 369:819–829CrossRefPubMedGoogle Scholar
  3. 3.
    Merlini G, Bellotti V (2003) Molecular mechanisms of amyloidosis. N Engl J Med 349:583–596CrossRefPubMedGoogle Scholar
  4. 4.
    Franklin EC, Lowenstein J, Bigelow B, Meltzer M (1964) Heavy chain disease—a new disorder of serum gamma-globulins : report of the first case. Am J Med 37:332–350CrossRefPubMedGoogle Scholar
  5. 5.
    Guan J, Mishra S, Shi J et al (2013) Stanniocalcin1 is a key mediator of amyloidogenic light chain induced cardiotoxicity. Basic Res Cardiol 108:378CrossRefPubMedPubMedCentralGoogle Scholar
  6. 6.
    Maleszewski JJ (2015) Cardiac amyloidosis: pathology, nomenclature, and typing. Cardiovascular pathology : the official journal of the Society for Cardiovascular Pathology 24:343–350CrossRefGoogle Scholar
  7. 7.
    Yunokawa K, Hagiyama Y, Mochizuki Y, Tanaka N, Ochi M (2007) Hypertrophic spinal pachymeningitis associated with heavy-chain disease. Case report. Journal of neurosurgery Spine 7:459–462PubMedGoogle Scholar
  8. 8.
    Desport E, Bridoux F, Sirac C et al (2012) Al amyloidosis. Orphanet journal of rare diseases 7:54CrossRefPubMedPubMedCentralGoogle Scholar
  9. 9.
    Lee MT, Parwani A, Humphrey R, Hamilton RG, Myers DI, Detrick B (2008) Gamma heavy chain disease in a patient with diabetes and chronic renal insufficiency: diagnostic assessment of the heavy chain fragment. J Clin Lab Anal 22:146–150CrossRefPubMedGoogle Scholar
  10. 10.
    Gertz MA, Comenzo R, Falk RH et al (2005) Definition of organ involvement and treatment response in immunoglobulin light chain amyloidosis (AL): a consensus opinion from the 10th International Symposium on Amyloid and Amyloidosis, Tours, France, 18-22 April 2004. Am J Hematol 79:319–328CrossRefPubMedGoogle Scholar
  11. 11.
    Patel KS, Hawkins PN (2015) Cardiac amyloidosis: where are we today? J Intern Med 278:126–144CrossRefPubMedGoogle Scholar
  12. 12.
    Kim SK, Park IK, Park BH et al (2005) A case report: isolated a heavy chain monoclonal gammopathy in a patient with polyneuropathy, organomegaly, endocrinopathy, monoclonal gammopathy and skin change syndrome. International journal of clinical practice Supplement 26-30Google Scholar
  13. 13.
    Merlini G, Palladini G (2013) Light chain amyloidosis: the heart of the problem. Haematologica 98:1492–1495CrossRefPubMedPubMedCentralGoogle Scholar
  14. 14.
    Vrana JA, Gamez JD, Madden BJ, Theis JD, Bergen HR 3rd, Dogan A (2009) Classification of amyloidosis by laser microdissection and mass spectrometry-based proteomic analysis in clinical biopsy specimens. Blood 114:4957–4959CrossRefPubMedGoogle Scholar
  15. 15.
    Brambilla F, Lavatelli F, Di Silvestre D et al (2012) Reliable typing of systemic amyloidoses through proteomic analysis of subcutaneous adipose tissue. Blood 119:1844–1847CrossRefPubMedGoogle Scholar
  16. 16.
    Wester SM, Banks PM, Li CY (1982) The histopathology of gamma heavy-chain disease. Am J Clin Pathol 78:427–436CrossRefPubMedGoogle Scholar
  17. 17.
    Wechalekar AD, Schonland SO, Kastritis E et al (2013) A European collaborative study of treatment outcomes in 346 patients with cardiac stage III AL amyloidosis. Blood 121:3420–3427CrossRefPubMedGoogle Scholar
  18. 18.
    Kumar SK, Dispenzieri A, Lacy MQ et al (2011) Changes in serum-free light chain rather than intact monoclonal immunoglobulin levels predicts outcome following therapy in primary amyloidosis. Am J Hematol 86:251–255CrossRefPubMedPubMedCentralGoogle Scholar
  19. 19.
    Dispenzieri A, Kyle RA, Lacy MQ et al (2004) Superior survival in primary systemic amyloidosis patients undergoing peripheral blood stem cell transplantation: a case-control study. Blood 103:3960–3963CrossRefPubMedGoogle Scholar
  20. 20.
    Dubrey SW, Cha K, Anderson J et al (1998) The clinical features of immunoglobulin light-chain (AL) amyloidosis with heart involvement. QJM : monthly journal of the Association of Physicians 91:141–157CrossRefPubMedGoogle Scholar
  21. 21.
    Gray Gilstrap L, Niehaus E, Malhotra R et al (2014) Predictors of survival to orthotopic heart transplant in patients with light chain amyloidosis. The Journal of heart and lung transplantation : the official publication of the International Society for Heart Transplantation 33:149–156CrossRefGoogle Scholar
  22. 22.
    Sperry BW, Ikram A, Hachamovitch R et al (2016) Efficacy of chemotherapy for light-chain amyloidosis in patients presenting with symptomatic heart failure. J Am Coll Cardiol 67:2941–2948CrossRefPubMedGoogle Scholar
  23. 23.
    Potena L, Quarta CC, Grigioni F, Rapezzi C (2014) Prognostic stratification and treatment of cardiac light chain amyloidosis: a narrow path in the jungle. The Journal of heart and lung transplantation : the official publication of the International Society for Heart Transplantation 33:136–138CrossRefGoogle Scholar
  24. 24.
    Cordes S, Dispenzieri A, Lacy MQ et al (2012) Ten-year survival after autologous stem cell transplantation for immunoglobulin light chain amyloidosis. Cancer 118:6105–6109CrossRefPubMedGoogle Scholar
  25. 25.
    Gertz MA, Lacy MQ, Dispenzieri A et al (2002) Stem cell transplantation for the management of primary systemic amyloidosis. Am J Med 113:549–555CrossRefPubMedGoogle Scholar
  26. 26.
    Sanchorawala V (2012) Role of high-dose melphalan and autologous peripheral blood stem cell transplantation in AL amyloidosis. American journal of blood research 2:9–17PubMedPubMedCentralGoogle Scholar
  27. 27.
    Estep JD, Bhimaraj A, Cordero-Reyes AM, Bruckner B, Loebe M, Torre-Amione G (2012) Heart transplantation and end-stage cardiac amyloidosis: a review and approach to evaluation and management. Methodist DeBakey cardiovascular journal 8:8–16CrossRefPubMedPubMedCentralGoogle Scholar
  28. 28.
    Bradshaw SH, Veinot JP (2012) Cardiac amyloidosis: what are the indications for transplant? Curr Opin Cardiol 27:143–147PubMedGoogle Scholar
  29. 29.
    Maurer MS, Hanna M, Grogan M et al (2016) Genotype and phenotype of transthyretin cardiac amyloidosis: THAOS (Transthyretin Amyloid Outcome Survey). J Am Coll Cardiol 68:161–172CrossRefPubMedGoogle Scholar
  30. 30.
    Benson MD (2000) Amyloidosis. The metabolic and molecular bases of inherited disease. McGraw-Hill, New York, pp 5345–5378Google Scholar
  31. 31.
    Ando Y, Coelho T, Berk JL et al (2013) Guideline of transthyretin-related hereditary amyloidosis for clinicians. Orphanet journal of rare diseases 8:31CrossRefPubMedPubMedCentralGoogle Scholar
  32. 32.
    Yamashita T, Hamidi Asl K, Yazaki M, Benson MD (2005) A prospective evaluation of the transthyretin Ile122 allele frequency in an African-American population. Amyloid : the international journal of experimental and clinical investigation : the official journal of the International Society of Amyloidosis 12:127–130CrossRefGoogle Scholar
  33. 33.
    Buxbaum J, Alexander A, Koziol J, Tagoe C, Fox E, Kitzman D (2010) Significance of the amyloidogenic transthyretin Val 122 Ile allele in African Americans in the Arteriosclerosis Risk in Communities (ARIC) and Cardiovascular Health (CHS) Studies. Am Heart J 159:864–870CrossRefPubMedPubMedCentralGoogle Scholar
  34. 34.
    Rapezzi C, Quarta CC, Riva L et al (2010) Transthyretin-related amyloidoses and the heart: a clinical overview. Nat Rev Cardiol 7:398–408CrossRefPubMedGoogle Scholar
  35. 35.
    Suhr OB, Larsson M, Ericzon BG, Wilczek HE (2016) Survival after transplantation in patients with mutations other than Val30Met: extracts from the FAP world transplant registry. Transplantation 100:373–381CrossRefPubMedPubMedCentralGoogle Scholar
  36. 36.
    Arruda-Olson AM, Zeldenrust SR, Dispenzieri A et al (2013) Genotype, echocardiography, and survival in familial transthyretin amyloidosis. Amyloid : the international journal of experimental and clinical investigation : the official journal of the International Society of Amyloidosis 20:263–268CrossRefGoogle Scholar
  37. 37.
    Grogan M, Scott CG, Kyle RA et al (2016) Natural history of wild-type transthyretin cardiac amyloidosis and risk stratification using a novel staging system. J Am Coll Cardiol 68:1014–1020CrossRefPubMedGoogle Scholar
  38. 38.
    Castano A, Drachman BM, Judge D, Maurer MS (2015) Natural history and therapy of TTR-cardiac amyloidosis: emerging disease-modifying therapies from organ transplantation to stabilizer and silencer drugs. Heart Fail Rev 20:163–178CrossRefPubMedPubMedCentralGoogle Scholar
  39. 39.
    Rowczenio D, Dogan A, Theis JD et al (2011) Amyloidogenicity and clinical phenotype associated with five novel mutations in apolipoprotein A-I. Am J Pathol 179:1978–1987CrossRefPubMedPubMedCentralGoogle Scholar
  40. 40.
    Selvanayagam JB, Hawkins PN, Paul B, Myerson SG, Neubauer S (2007) Evaluation and management of the cardiac amyloidosis. J Am Coll Cardiol 50:2101–2110CrossRefPubMedGoogle Scholar
  41. 41.
    Mehra MR, Canter CE, Hannan MM et al (2016) The 2016 International Society for Heart Lung Transplantation listing criteria for heart transplantation: a 10-year update. The Journal of heart and lung transplantation : the official publication of the International Society for Heart Transplantation 35:1–23CrossRefGoogle Scholar
  42. 42.
    DePasquale EC, Nasir K, Jacoby DL (2012) Outcomes of adults with restrictive cardiomyopathy after heart transplantation. The Journal of heart and lung transplantation : the official publication of the International Society for Heart Transplantation 31:1269–1275CrossRefGoogle Scholar
  43. 43.
    Dubrey SW, Burke MM, Hawkins PN, Banner NR (2004) Cardiac transplantation for amyloid heart disease: the United Kingdom experience. The Journal of heart and lung transplantation : the official publication of the International Society for Heart Transplantation 23:1142–1153CrossRefGoogle Scholar
  44. 44.
    Hall R, Hawkins PN (1994) Cardiac transplantation for AL amyloidosis. BMJ (Clinical research ed) 309:1135–1137CrossRefGoogle Scholar
  45. 45.
    Conner R, Hosenpud JD, Norman DJ, Pantely GA, Cobanoglu A, Starr A (1988) Heart transplantation for cardiac amyloidosis: successful one-year outcome despite recurrence of the disease. The Journal of heart transplantation 7:165–167PubMedGoogle Scholar
  46. 46.
    Kpodonu J, Massad MG, Caines A, Geha AS (2005) Outcome of heart transplantation in patients with amyloid cardiomyopathy. The Journal of heart and lung transplantation : the official publication of the International Society for Heart Transplantation 24:1763–1765CrossRefGoogle Scholar
  47. 47.
    Kristen AV, Brokbals E, Aus dem Siepen F et al (2016) Cardiac amyloid load: a prognostic and predictive biomarker in patients with light-chain amyloidosis. J Am Coll Cardiol 68:13–24CrossRefPubMedGoogle Scholar
  48. 48.
    Dey BR, Chung SS, Spitzer TR et al (2010) Cardiac transplantation followed by dose-intensive melphalan and autologous stem-cell transplantation for light chain amyloidosis and heart failure. Transplantation 90:905–911CrossRefPubMedPubMedCentralGoogle Scholar
  49. 49.
    Lacy MQ, Dispenzieri A, Hayman SR et al (2008) Autologous stem cell transplant after heart transplant for light chain (Al) amyloid cardiomyopathy. The Journal of heart and lung transplantation : the official publication of the International Society for Heart Transplantation 27:823–829CrossRefGoogle Scholar
  50. 50.
    Hsich EM, Rogers JG, McNamara DM et al. ( 2016) Does survival on the heart transplant waiting list depend on the underlying heart disease? JACC Heart failureGoogle Scholar
  51. 51.
    Alloni A, Pellegrini C, Ragni T et al (2004) Heart transplantation in patients with amyloidosis: single-center experience. Transplant Proc 36:643–644CrossRefPubMedGoogle Scholar
  52. 52.
    Hosenpud JD, DeMarco T, Frazier OH et al (1991) Progression of systemic disease and reduced long-term survival in patients with cardiac amyloidosis undergoing heart transplantation. Follow-up results of a multicenter survey. Circulation 84:III338–III343PubMedGoogle Scholar
  53. 53.
    Gillmore JD, Goodman HJ, Lachmann HJ et al (2006) Sequential heart and autologous stem cell transplantation for systemic AL amyloidosis. Blood 107:1227–1229CrossRefPubMedGoogle Scholar
  54. 54.
    Rosenbaum AN, Edwards BS (2015) Current indications, strategies, and outcomes with cardiac transplantation for cardiac amyloidosis and sarcoidosis. Curr Opin Organ Transplant 20:584–592CrossRefPubMedGoogle Scholar
  55. 55.
    Colvin M, Smith JM, Skeans MA et al (2016) Heart. Am J Transplant Off J Am Soc Transplant Am Soc Transplant Surg 16(Suppl 2):115–140CrossRefGoogle Scholar
  56. 56.
    Davis MK, Kale P, Liedtke M et al (2015) Outcomes after heart transplantation for amyloid cardiomyopathy in the modern era. Am J Transplant Off J Am Soc Transplant Am Soc Transplant Surg 15:650–658CrossRefGoogle Scholar
  57. 57.
    Kristen AV, Sack FU, Schonland SO et al (2009) Staged heart transplantation and chemotherapy as a treatment option in patients with severe cardiac light-chain amyloidosis. Eur J Heart Fail 11:1014–1020CrossRefPubMedGoogle Scholar
  58. 58.
    Grogan M, Gertz M, McCurdy A et al (2016) Long term outcomes of cardiac transplant for immunoglobulin light chain amyloidosis: the Mayo Clinic experience. World J Transplant 6:380–388CrossRefPubMedPubMedCentralGoogle Scholar
  59. 59.
    Varr BC, Liedtke M, Arai S, Lafayette RA, Schrier SL, Witteles RM (2012) Heart transplantation and cardiac amyloidosis: approach to screening and novel management strategies. The Journal of heart and lung transplantation : the official publication of the International Society for Heart Transplantation 31:325–331CrossRefGoogle Scholar
  60. 60.
    Davis MK, Lee PH, Witteles RM (2015) Changing outcomes after heart transplantation in patients with amyloid cardiomyopathy. The Journal of heart and lung transplantation : the official publication of the International Society for Heart Transplantation 34:658–666CrossRefGoogle Scholar
  61. 61.
    Baumgratz JF, Vila JH, Guilhen CJ et al (2009) Heart transplantation in primary amyloidosis. Rev Bras Cir Cardiovasc 24:409–412CrossRefPubMedGoogle Scholar
  62. 62.
    Perz JB, Kristen AV, Rahemtulla A et al (2006) Long-term survival in a patient with AL amyloidosis after cardiac transplantation followed by autologous stem cell transplantation. Clinical research in cardiology : official journal of the German Cardiac Society 95:671–674CrossRefGoogle Scholar
  63. 63.
    Maurer MS, Raina A, Hesdorffer C et al (2007) Cardiac transplantation using extended-donor criteria organs for systemic amyloidosis complicated by heart failure. Transplantation 83:539–545CrossRefPubMedGoogle Scholar
  64. 64.
    Sack FU, Kristen A, Goldschmidt H et al (2008) Treatment options for severe cardiac amyloidosis: heart transplantation combined with chemotherapy and stem cell transplantation for patients with AL-amyloidosis and heart and liver transplantation for patients with ATTR-amyloidosis. European journal of cardio-thoracic surgery : official journal of the European Association for Cardio-thoracic Surgery 33:257–262CrossRefGoogle Scholar
  65. 65.
    Gibbs SD, Sattianayagam PT, Hawkins PN, Gillmore JD (2009) Cardiac transplantation should be considered in selected patients with either AL or hereditary forms of amyloidosis: the UK National Amyloidosis Centre experience. Intern Med J 39:786–787 author reply 787-8 CrossRefPubMedGoogle Scholar
  66. 66.
    Luo JM, Chou NK, Chi NH et al (2010) Heart transplantation in patients with amyloidosis. Transplant Proc 42:927–929CrossRefPubMedGoogle Scholar
  67. 67.
    Mignot A, Varnous S, Redonnet M et al (2008) Heart transplantation in systemic (AL) amyloidosis: a retrospective study of eight French patients. Archives of cardiovascular diseases 101:523–532CrossRefPubMedGoogle Scholar
  68. 68.
    Roig E, Almenar L, Gonzalez-Vilchez F et al (2009) Outcomes of heart transplantation for cardiac amyloidosis: subanalysis of the Spanish registry for heart transplantation. Am J Transplant Off J Am Soc Transplant Am Soc Transplant Surg 9:1414–1419CrossRefGoogle Scholar
  69. 69.
    Sattianayagam PT, Gibbs SD, Pinney JH et al (2010) Solid organ transplantation in AL amyloidosis. Am J Transplant Off J Am Soc Transplant Am Soc Transplant Surg 10:2124–2131CrossRefGoogle Scholar
  70. 70.
    Te HS, Anderson AS, Millis JM, Jeevanandam V, Jensen DM (2008) Current state of combined heart-liver transplantation in the United States. The Journal of heart and lung transplantation : the official publication of the International Society for Heart Transplantation 27:753–759CrossRefGoogle Scholar
  71. 71.
    Holmgren G, Steen L, Ekstedt J et al (1991) Biochemical effect of liver transplantation in two Swedish patients with familial amyloidotic polyneuropathy (FAP-met30). Clin Genet 40:242–246CrossRefPubMedGoogle Scholar
  72. 72.
    Rela M, Muiesan P, Heaton ND et al (1995) Orthotopic liver transplantation for hepatic-based metabolic disorders. Transplant international : official journal of the European Society for Organ Transplantation 8:41–44CrossRefGoogle Scholar
  73. 73.
    Wilczek HE, Larsson M, Ericzon BG (2011) Long-term data from the Familial Amyloidotic Polyneuropathy World Transplant Registry (FAPWTR). Amyloid : the international journal of experimental and clinical investigation : the official journal of the International Society of Amyloidosis 18(Suppl 1):193–195CrossRefGoogle Scholar
  74. 74.
    Ericzon BG, Wilczek HE, Larsson M et al (2015) Liver transplantation for hereditary transthyretin amyloidosis: after 20 years still the best therapeutic alternative? Transplantation 99:1847–1854CrossRefPubMedGoogle Scholar
  75. 75.
    Cannon RM, Hughes MG, Jones CM, Eng M, Marvin MR (2012) A review of the United States experience with combined heart-liver transplantation. Transplant international : official journal of the European Society for Organ Transplantation 25:1223–1228CrossRefGoogle Scholar
  76. 76.
    Careddu L, Zanfi C, Pantaleo A et al (2015) Combined heart-liver transplantation: a single-center experience. Transplant international : official journal of the European Society for Organ Transplantation 28:828–834CrossRefGoogle Scholar
  77. 77.
    Coelho T, Maia LF, da Silva AM et al (2013) Long-term effects of tafamidis for the treatment of transthyretin familial amyloid polyneuropathy. J Neurol 260:2802–2814CrossRefPubMedPubMedCentralGoogle Scholar
  78. 78.
    Ammirati E, Marziliano N, Vittori C et al (2012) The first Caucasian patient with p.Val122Ile mutated-transthyretin cardiac amyloidosis treated with isolated heart transplantation. Amyloid : the international journal of experimental and clinical investigation : the official journal of the International Society of Amyloidosis 19:113–117CrossRefGoogle Scholar
  79. 79.
    Thenappan T, Fedson S, Rich J et al (2014) Isolated heart transplantation for familial transthyretin (TTR) V122I cardiac amyloidosis. Amyloid : the international journal of experimental and clinical investigation : the official journal of the International Society of Amyloidosis 21:120–123CrossRefGoogle Scholar
  80. 80.
    Hamour IM, Lachmann HJ, Goodman HJ et al (2008) Heart transplantation for homozygous familial transthyretin (TTR) V122I cardiac amyloidosis. Am J Transplant Off J Am Soc Transplant Am Soc Transplant Surg 8:1056–1059CrossRefGoogle Scholar
  81. 81.
    Arpesella G, Chiappini B, Marinelli G et al (2003) Combined heart and liver transplantation for familial amyloidotic polyneuropathy. J Thorac Cardiovasc Surg 125:1165–1166CrossRefPubMedGoogle Scholar
  82. 82.
    Grazi GL, Cescon M, Salvi F et al (2003) Combined heart and liver transplantation for familial amyloidotic neuropathy: considerations from the hepatic point of view. Liver transplantation : official publication of the American Association for the Study of Liver Diseases and the International Liver Transplantation Society 9:986–992CrossRefGoogle Scholar
  83. 83.
    Nardo B, Beltempo P, Bertelli R et al (2004) Combined heart and liver transplantation in four adults with familial amyloidosis: experience of a single center. Transplant Proc 36:645–647CrossRefPubMedGoogle Scholar
  84. 84.
    Raichlin E, Daly RC, Rosen CB et al (2009) Combined heart and liver transplantation: a single-center experience. Transplantation 88:219–225CrossRefPubMedGoogle Scholar
  85. 85.
    Barreiros AP, Post F, Hoppe-Lotichius M et al (2010) Liver transplantation and combined liver-heart transplantation in patients with familial amyloid polyneuropathy: a single-center experience. Liver transplantation : official publication of the American Association for the Study of Liver Diseases and the International Liver Transplantation Society 16:314–323CrossRefGoogle Scholar
  86. 86.
    Nelson LM, Penninga L, Sander K et al (2013) Long-term outcome in patients treated with combined heart and liver transplantation for familial amyloidotic cardiomyopathy. Clin Transpl 27:203–209CrossRefGoogle Scholar

Copyright information

© Springer Science+Business Media New York 2017

Authors and Affiliations

  1. 1.University of KentuckyLexingtonUSA

Personalised recommendations