Literature DB >> 16011989

Multicentre versus single centre approach to rare diseases: the model of systemic light chain amyloidosis.

Giovanni Palladini1, Robert A Kyle, Dirk R Larson, Terry M Therneau, Giampaolo Merlini, Morie A Gertz.   

Abstract

BACKGROUND: Early diagnosis and supportive therapy are important in primary systemic amyloidosis (AL). In 1986, a national network was started in Italy to increase the awareness of medical professionals, achieve early diagnoses, and provide locally qualified care. We compared the AL patient population observed at a referral center, Mayo Clinic, with that recruited through the Italian network.
METHODS: All the patients diagnosed with AL between 1988 and 1998 at Mayo Clinic or in any of the centers of the Italian Amyloidosis Study Group were included.
FINDINGS: The median survival of Italian patients was 30 months versus 12 months in the Mayo cohort (P<0.001). Mayo Clinic patients were older (66.4 vs. 60.1 y, P<0.001). In the Italian cohort, dominant kidney involvement was more frequent (49.3% vs. 27.8%, P<0.001), while in the Mayo group more patients had dominant cardiac amyloidosis (37.4% vs. 27.8%, P=0.03). Italian patients were more likely to have kidney involvement without heart involvement (43.7% vs. 19.6%, P<0.001). Among Italian patients, 78.9% received alkylating agents versus 60.9% in the Mayo cohort (P<0.001). The multivariate analysis showed that age, performance status, dominant heart involvement, >or=2 organs involved, and treatment with alkylating agents independently predicted survival. This study shows that AL patient populations may differ significantly between centers according to their accrual systems, with possible impact on treatment trials.

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Year:  2005        PMID: 16011989     DOI: 10.1080/13506120500107055

Source DB:  PubMed          Journal:  Amyloid        ISSN: 1350-6129            Impact factor:   7.141


  12 in total

1.  [Unusual skin alterations in a 72-year-old patient with multiple myeloma].

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2.  Amyloidosis 2008 BMT Tandem Meetings (February 13-17, San Diego).

Authors:  Angela Dispenzieri; Giampaolo Merlini; Raymond L Comenzo
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3.  Improved outcomes for newly diagnosed AL amyloidosis between 2000 and 2014: cracking the glass ceiling of early death.

Authors:  Eli Muchtar; Morie A Gertz; Shaji K Kumar; Martha Q Lacy; David Dingli; Francis K Buadi; Martha Grogan; Suzanne R Hayman; Prashant Kapoor; Nelson Leung; Amie Fonder; Miriam Hobbs; Yi Lisa Hwa; Wilson Gonsalves; Rahma Warsame; Taxiarchis V Kourelis; Stephen Russell; John A Lust; Yi Lin; Ronald S Go; Steven Zeldenrust; Robert A Kyle; S Vincent Rajkumar; Angela Dispenzieri
Journal:  Blood       Date:  2017-01-26       Impact factor: 22.113

Review 4.  New developments in diagnosis, risk assessment and management in systemic amyloidosis.

Authors:  Iuliana Vaxman; Angela Dispenzieri; Eli Muchtar; Morie Gertz
Journal:  Blood Rev       Date:  2019-11-02       Impact factor: 8.250

Review 5.  The Clinical Presentation and Management of Systemic Light-Chain Amyloidosis in China.

Authors:  Xiang-Hua Huang; Zhi-Hong Liu
Journal:  Kidney Dis (Basel)       Date:  2016-02-25

6.  Clinical features and treatment response of light chain (AL) amyloidosis diagnosed in patients with previous diagnosis of multiple myeloma.

Authors:  Sumit Madan; Angela Dispenzieri; Martha Q Lacy; Francis Buadi; Suzanne R Hayman; Steven R Zeldenrust; S Vincent Rajkumar; Morie A Gertz; Shaji K Kumar
Journal:  Mayo Clin Proc       Date:  2010-03       Impact factor: 7.616

Review 7.  Deposition-associated diseases related with a monoclonal compound.

Authors:  M J Molina-Garrido; C Guillén-Ponce; A Mora; M Guirado-Risueño; M A Molina; M J Molina; A Carrato
Journal:  Clin Transl Oncol       Date:  2007-12       Impact factor: 3.405

8.  CD32B is highly expressed on clonal plasma cells from patients with systemic light-chain amyloidosis and provides a target for monoclonal antibody-based therapy.

Authors:  Ping Zhou; Raymond L Comenzo; Adam B Olshen; Ezio Bonvini; Scott Koenig; Peter G Maslak; Martin Fleisher; James Hoffman; Suresh Jhanwar; James W Young; Stephen D Nimer; Adam M Boruchov
Journal:  Blood       Date:  2008-01-23       Impact factor: 22.113

9.  The coming of age of artificial intelligence in medicine.

Authors:  Vimla L Patel; Edward H Shortliffe; Mario Stefanelli; Peter Szolovits; Michael R Berthold; Riccardo Bellazzi; Ameen Abu-Hanna
Journal:  Artif Intell Med       Date:  2008-09-13       Impact factor: 5.326

10.  Renal tubular P-glycoprotein expression is reduced in plasma cell disorders.

Authors:  Cihan Heybeli; Mehmet Asi Oktan; Hayri Ustun Arda; Serkan Yildiz; Mehtat Unlu; Caner Cavdar; Aykut Sifil; Ali Celik; Sulen Sarioglu; Taner Camsari
Journal:  Kidney Res Clin Pract       Date:  2019-06-30
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