title: [Analysis of clinical characteristics and outcome of patients with very high risk primary immunoglobulin light-chain amyloidosis].
abstract: Objective:To evaluate the clinical characteristics and outcomes of very high risk patients with primary immunoglobulin light-chain amyloidosis (pAL) at a single center in China.Method:Clinical data, treatment and outcome of 205 pAL patients in Peking Union Medical College Hospital from January 2009 to February 2016 were retrospectively analyzed. A 'very high risk' group includes patients with Mayo 2004 stage Ⅲb and Mayo 2012 stage 4.Results:Of 205 patients, 34 (16.6%) were defined as very high risk pAL patients. The median age at diagnosis was 57 (20-84) years, and 22 patients (64.7%) were male. All 34 patients were diagnosed with cardiac involvement, multi-organ involvement was observed in 15 patients (44.1%) , and 27 (81.8%) had New York Heart Association Class Ⅲ or Ⅳ. Median values of serum cTnI, NT-proBNP, and free light chains difference were 0.25 μg/L, 11 733 ng/L, and 403 mg/L, respectively. Eight (24.2%) had more than 10% plasma cell on the bone marrow aspirate. Sixteen (47.1%) patients received bortezomib based chemotherapy and overall hematologic response rate was 58.3%. Median overall survival (OS) was 4 months. The estimated OS at 3, 6, 12, and 24 months was 51.3%, 44.0%, 35.2%, and 29.6%, respectively. Fourteen (41.2%) patients died within 3 months after the diagnosis. The estimated 1-year survival rate for the patients who got hematologic response, without hematologic response, and palliative treatment was 90.9%, 11.1%, and 0, respectively (P<0.001) .Conclusion:Patients with very high risk pAL had very poor prognosis and the early death rate remained high. Those patients who obtained hematologic remission would have significantly better outcomes.
authors.forename: J
authors.initials: J
authors.lastname: Feng
authors.forename: X F
authors.initials: XF
authors.lastname: Huang
authors.forename: C L
authors.initials: CL
authors.lastname: Zhang
authors.forename: K N
authors.initials: KN
authors.lastname: Shen
authors.forename: C L
authors.initials: CL
authors.lastname: Zhang
authors.forename: J
authors.initials: J
authors.lastname: Sun
authors.forename: Z
authors.initials: Z
authors.lastname: Tian
authors.forename: X X
authors.initials: XX
authors.lastname: Cao
authors.forename: L
authors.initials: L
authors.lastname: Zhang
authors.forename: D B
authors.initials: DB
authors.lastname: Zhou
authors.affiliation: Department of Hematology, Peking Union Medical College Hospital, Chinese Academy of Medical Sciences, Beijing 100730, China.
authors.forename: J
authors.initials: J
authors.lastname: Li
mesh_terms: D000328:Adult; D000368:Aged; D000369:Aged, 80 and over; D000069286:Bortezomib; D005260:Female; D006801:Humans; D007147:Immunoglobulin Light Chains; D000075363:Immunoglobulin Light-chain Amyloidosis; D008297:Male; D008875:Middle Aged; D020097:Natriuretic Peptide, Brain; D010446:Peptide Fragments; D011379:Prognosis; D012189:Retrospective Studies; D012306:Risk; D055815:Young Adult
chemical_list: D007147:Immunoglobulin Light Chains; D010446:Peptide Fragments; C109794:pro-brain natriuretic peptide (1-76); D020097:Natriuretic Peptide, Brain; D000069286:Bortezomib
keywords: Bortezomib; Primary light chain amyloidosis; Very high risk