دورية أكاديمية
أعرض في EDS

Emerging therapy in light-chain and acquired transthyretin-related amyloidosis: an Italian single-centre experience in heart transplantation.

التفاصيل البيبلوغرافية
العنوان: Emerging therapy in light-chain and acquired transthyretin-related amyloidosis: an Italian single-centre experience in heart transplantation.
المؤلفون: Di Nora C; Department of Cardiothoracic Science, Azienda Sanitaria Universitaria Integrata di Udine., Sponga S; Department of Cardiothoracic Science, Azienda Sanitaria Universitaria Integrata di Udine., Ferrara V; Department of Cardiothoracic Science, Azienda Sanitaria Universitaria Integrata di Udine., Patriarca F; Haematology Department, Azienda sanitaria Universitaria Integrata, DAME., Fanin R; Haematology Department, Azienda sanitaria Universitaria Integrata, DAME., Nalli C; Department of Cardiothoracic Science, Azienda Sanitaria Universitaria Integrata di Udine., Lechiancole A; Department of Cardiothoracic Science, Azienda Sanitaria Universitaria Integrata di Udine., Vendramin I; Department of Cardiothoracic Science, Azienda Sanitaria Universitaria Integrata di Udine., Livi U; Department of Cardiothoracic Science.; Department of Medical Area DAME, University of Udine, Udine, Italy.
المصدر: Journal of cardiovascular medicine (Hagerstown, Md.) [J Cardiovasc Med (Hagerstown)] 2021 Apr 01; Vol. 22 (4), pp. 261-267.
نوع المنشور: Journal Article
اللغة: English
بيانات الدورية: Publisher: Lippincott Williams & Wilkins Country of Publication: United States NLM ID: 101259752 Publication Model: Print Cited Medium: Internet ISSN: 1558-2035 (Electronic) Linking ISSN: 15582027 NLM ISO Abbreviation: J Cardiovasc Med (Hagerstown) Subsets: MEDLINE
أسماء مطبوعة: Original Publication: Hagerstown, MD : Lippincott Williams & Wilkins, c2006-
مواضيع طبية MeSH: Amyloid Neuropathies, Familial*/complications , Amyloid Neuropathies, Familial*/diagnosis , Amyloid Neuropathies, Familial*/epidemiology , Amyloid Neuropathies, Familial*/therapy , Cardiomyopathies*/diagnosis , Cardiomyopathies*/epidemiology , Cardiomyopathies*/etiology , Cardiomyopathies*/surgery , Graft Rejection*/diagnosis , Graft Rejection*/etiology , Heart Transplantation*/adverse effects , Heart Transplantation*/methods , Immunoglobulin Light-chain Amyloidosis*/complications , Immunoglobulin Light-chain Amyloidosis*/diagnosis , Immunoglobulin Light-chain Amyloidosis*/epidemiology , Immunoglobulin Light-chain Amyloidosis*/therapy , Postoperative Complications*/diagnosis , Postoperative Complications*/mortality , Renal Insufficiency*/diagnosis , Renal Insufficiency*/etiology, Stem Cell Transplantation/*methods, Female ; Humans ; Italy/epidemiology ; Male ; Middle Aged ; Outcome and Process Assessment, Health Care ; Patient Selection ; Prognosis ; Secondary Prevention/methods ; Survival Analysis
مستخلص: Aims: The prognosis for patients affected by light-chain cardiac amyloidosis and acquired transthyretin-related (TTR) amyloidosis is poor. Heart transplantation (HTx) could improve prognosis also enabling autologous stem cell transplantation (ASCT) in the first group.
Methods and Results: A total of 36 patients affected by systemic amyloidosis have been referred to our centre from 2009 to 2019. Of these, nine had cardiac involvement: seven by light-chain amyloidosis and two by acquired TTR amyloidosis. None died while waiting for HTx. A specific internal protocol useful to select candidates and to monitor the organ involvement after HTx was developed. Median age at diagnosis was 54 years and 66% were male. The most common short-term complication after HTx was renal failure (44%), followed by acute cardiac rejection more than 2R (22%). ASCT was performed in six out of seven light-chain cardiac amyloidosis patients, with a median time of 6 months after HTx. Two patients affected by light-chain cardiac amyloidosis died due to amyloidosis relapse: one before undergoing ASCT. After a median follow-up of 31 (7-124) months, 1- and 5-year survival was 88 and 66% in the cardiac light-chain amyloidosis group. Conversely, 1- and 5-year survival was 100% in the acquired TTR amyloidosis group.
Conclusion: HTx may represent a valuable option in carefully selected patients. ASCT after HTx is an effective treatment that could decrease amyloidosis relapse in light-chain cardiac amyloidosis patients. A multidisciplinary approach is mandatory to select the best candidates and to obtain the most effective results with a specific surveillance follow-up protocol.
(Copyright © 2020 Italian Federation of Cardiology - I.F.C. All rights reserved.)
References: Sipe JD, Benson MD, Buxbaum JN, et al. Amyloid fibril proteins and amyloidosis: chemical identification and clinical classification International Society of Amyloidosis 2016 Nomenclature Guidelines. Amyloid 2016; 4:209–213.
Rapezzi C, Milandri A, Lorenxini M. The complex interplay between systolic and diastolic function at rest and during exercise in heart failure: the case of cardiac amyloidosis. Eur J Heart Fail 2017; 11:1466–1467.
Rapezzi C, Merlini G, Quarta CC, et al. Systemic cardiac amyloidoses: disease profiles and clinical courses of the 3 main types. Circulation 2009; 120:1203–1212.
Seferović PM, Polovina M, Bauersachs J, et al. Heart failure in cardiomyopathies: a position paper from the Heart Failure Association of the European Society of Cardiology. Eur J Heart Fail 2019; 5:553–576.
Kristen AV, Perz JB, Schonland SO, et al. Noninvasive predictors of survival in cardiac amyloidosis. Eur J Heart Fail 2007; 9:617–624.
Maurizi N, Rella V, Fumagalli C, et al. Prevalence of cardiac amyloidosis among adult patients referred to tertiary centres with an initial diagnosis of hypertrophic cardiomyopathy. Int J Cardiol 2020; 300:191–195.
Nitsche C, Aschauer S, Kammerlander AA, et al. Light-chain and transthyretin cardiac amyloidosis in severe aortic stenosis: prevalence, screening possibilities, and outcome. Eur J Heart Fail 2020; doi:10.1002/ejhf.1756. (PMID: 10.1002/ejhf.1756)
Muchtar E, Buadi FK, Dispenzieri A, Gertz MA. Immunoglobulin light-chain amyloidosis: from basics to new developments in diagnosis, prognosis and therapy. Acta Haematol 2016; 135:172–190.
Ladefoged B, Dybro A, Povlsen JA, Vase H, Clemmensen TS, Poulsen SH. Diagnostic delay in wild type transthyretin cardiac amyloidosis – a clinical challenge. Int J Cardiol 2020; 304:138–143.
Siegismund CS, Escher F, Lassner D, Kühl U, Gross U, Fruhwald F. Intramyocardial inflammation predicts adverse outcome in patients with cardiac AL amyloidosis. Eur J Heart Fail 2018; 4:751–757.
Maurer MS, Schwartz JH, Gundapaneni B, et al. Tafamidis treatment for patients with transthyretin amyloid cardiomyopathy. N Engl J Med 2018; 11:1007–1016.
Sponga S, Deroma L, Sappa R, et al. Recipient age impact on outcome after cardiac transplantation: should it still be considered in organ allocation? Interact Cardiovasc Thorac Surg 2016; 4:573–579.
Lund LH, Edwards LB, Kucheryavaya AY, et al. The registry of International Society of Heart and Lung Transplantation: thirtieth official adult heart transplant report – 2013. J Heart Lung Transplant 2013; 32:951–964.
Gertz MA, Comenzo R, Falk RH, et al. 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 2005; 79:319–328.
Hosenpud JD, Uretsky BF, Griffith BP, O’Connell JB, Olivari MT, Valantine HA. Successful intermediate-term outcome for patients with cardiac amyloidosis undergoing heart transplantation: results of a multicentre survey. J Heart Transplant 1990; 9:346–350.
Hall R, Hawkins PN. Cardiac transplantation for AL amyloidosis. BMJ 1994; 309:1135–1137.
Hosenpud JD, DeMarco T, Frazier OH, et al. 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 1991; 84:338–343.
Kpodonu J, Massad MG, Caines A, Geha AS. Outcome of heart transplantation in patients with amyloid cardiomyopathy. J Heart Lung Transplant 2005; 24:1763–1765.
Dubrey SW, Burke MM, Hawkins PN, Banner NR. Cardiac transplantation for amyloid heart disease: the United Kingdom experience. J Heart Lung Transplant 2004; 23:1142–1153.
Sattianayagam PT, Gibbs SD, Pinney JH, et al. Solid organ transplantation in AL amyloidosis. Am J Transplant 2010; 10:2124–2131.
Lacy MQ, Dispenzieri A, Hayman SR, et al. Autologous stem cell transplant after heart transplant for light chain (AL) amyloid cardiomyopathy. J Heart Lung Transplant 2008; 27:823–829.
Roig E, Almenar L, Gonzalez-Vilchez F, et al. Outcomes of heart transplantation for cardiac amyloidosis: subanalysis of the Spanish Registry for heart transplantation. Am J Transplant 2009; 9:1414–1419.
Rosenbaum AN, AbouEzzeddine OF, Grogan M, et al. Outcomes after cardiac transplant for wild type transthyretin amyloidosis. Transplantation 2018; 11:1909–1913.
Mancini D, Lietz K. Selection of cardiac transplantation candidates in 2010. Circulation 2010; 122:173–183.
Davis MK, Lee PH, Witteles RM. Changing outcomes after heart transplantation in patients with amyloid cardiomyopathy. J Heart Lung Transplant 2015; 34:658–666.
Milandri A, Farioli A, Gagliardi C, et al. Carpal tunnel syndrome in cardiac amyloidosis: implications for early diagnosis and prognostic role across the spectrum of aetiologies. Eur J Heart Fail 2020; 3:507–515.
Aimo A, Buda G, Fontana M, et al. Therapies for cardiac light chain amyloidosis: an update. Int J Cardiol 2018; 271:152–160.
Kristen AV, Sack F, Schonland SO, et al. Staged heart transplantation and chemotherapy as a treatment option in patients with severe cardiac light-chain amyloidosis. Eur J Heart Fail 2009; 10:1014–1020.
Maurer MS, Raina A, Hesdorffer C, et al. Cardiac transplantation using extended-donor criteria organs for systemic amyloidosis complicated by heart failure. Transplantation 2007; 83:539–545.
Di Nora C, Livi U. Heart transplantation in cardiac storage diseases: data on Fabry disease and cardiac amyloidosis. Curr Opin Organ Transplant 2020; 25:211–217.
Di Nora C, Miani D, D’Elia AV, et al. Heart transplantation in Danon disease: long term single centre experience and review of the literature. Eur J Med Genet 2020; 2:1036–1045.
Patel J, Kobashigawa JA. Cardiac transplantation: the alternate list and expansion of the donor pool. Curr Opin Cardiol 2004; 19:162.
Di Nora C, Paldino A, Miani D, et al. Heart transplantation in Kearns–Sayre syndrome. Transplantation 2019; 12:e393–e394.
Müller ML, Butler J, Heidecker B. Emerging therapies in transthyretin amyloidosis – a new wave of hope after years of stagnancy? Eur J Heart Fail 2020; 22:39–45.
Canepa M, Tini G, Musumeci B, et al. Real-world versus trial patients with transthyretin amyloid cardiomyopathy. Eur J Heart Fail 2019; 11:1479–1481.
SCR Disease Name: Amyloidosis, Hereditary, Transthyretin-Related
تواريخ الأحداث: Date Created: 20210226 Date Completed: 20211215 Latest Revision: 20240229
رمز التحديث: 20240229
DOI: 10.2459/JCM.0000000000001094
PMID: 33633041
قاعدة البيانات: MEDLINE
الوصف
تدمد:1558-2035
DOI:10.2459/JCM.0000000000001094