title: Outcome of Patients with Immunoglobulin Light-Chain Amyloidosis with Lung, Liver, Gastrointestinal, Neurologic, and Soft Tissue Involvement after Autologous Hematopoietic Stem Cell Transplantation.
abstract: There is limited information on the outcome when organs other than heart or kidneys are involved by immunoglobulin light-chain amyloidosis (AL). We report the outcome of 53 patients with AL with gastrointestinal (GI), peripheral nerve (PN), liver, lung, or soft-tissue involvement, who underwent high-dose chemotherapy and autologous hematopoietic stem cell transplantation (auto-HCT) at our institution between 1997 and 2013. The median age at auto-HCT was 56 years (range, 35 to 74). One, 2, 3, or 4 organs were involved in 43%, 22%, 28%, and 4% of patients, respectively. Concurrent cardiac, renal, or both were involved in 24 (45%) patients. Forty-six patients received induction therapy before auto-HCT. The 100-day and 1-year treatment-related mortality (TRM) were 3.8% (n = 2) and 7.5% (n = 4), respectively. Forty-one (80%) patients achieved a hematologic response. Organ response at 1 year after auto-HCT was seen in 23 (57%) of the 40 evaluable patients. With a median follow-up of 24 months, the median progression-free survival and overall survival (OS) were 36 and 73 months, respectively. Auto-HCT was associated with a low TRM, durable organ responses, and a median OS of > 6 years in selected patients with AL and GI, PN, liver, lung, or soft-tissue involvement.
authors.affiliation: Department of Stem Cell Transplantation and Cellular Therapy, The University of Texas MD Anderson Cancer Center, Houston, Texas.
authors.forename: Aimaz
authors.initials: A
authors.lastname: Afrough
authors.affiliation: Department of Stem Cell Transplantation and Cellular Therapy, The University of Texas MD Anderson Cancer Center, Houston, Texas.
authors.forename: Rima M
authors.initials: RM
authors.lastname: Saliba
authors.affiliation: Department of Stem Cell Transplantation and Cellular Therapy, The University of Texas MD Anderson Cancer Center, Houston, Texas.
authors.forename: Amir
authors.initials: A
authors.lastname: Hamdi
authors.affiliation: Department of Stem Cell Transplantation and Cellular Therapy, The University of Texas MD Anderson Cancer Center, Houston, Texas.
authors.forename: Riad
authors.initials: R
authors.lastname: El Fakih
authors.affiliation: Department of Internal Medicine, University of Texas Health Science Center, Houston, Texas.
authors.forename: Ankur
authors.initials: A
authors.lastname: Varma
authors.affiliation: Department of Stem Cell Transplantation and Cellular Therapy, The University of Texas MD Anderson Cancer Center, Houston, Texas.
authors.forename: Yvonne T
authors.initials: YT
authors.lastname: Dinh
authors.affiliation: Department of Stem Cell Transplantation and Cellular Therapy, The University of Texas MD Anderson Cancer Center, Houston, Texas.
authors.forename: Gabriela
authors.initials: G
authors.lastname: Rondon
authors.affiliation: Department of Stem Cell Transplantation and Cellular Therapy, The University of Texas MD Anderson Cancer Center, Houston, Texas.
authors.forename: A Megan
authors.initials: AM
authors.lastname: Cornelison
authors.affiliation: Department of Stem Cell Transplantation and Cellular Therapy, The University of Texas MD Anderson Cancer Center, Houston, Texas.
authors.forename: Nina D
authors.initials: ND
authors.lastname: Shah
authors.affiliation: Department of Stem Cell Transplantation and Cellular Therapy, The University of Texas MD Anderson Cancer Center, Houston, Texas.
authors.forename: Qaiser
authors.initials: Q
authors.lastname: Bashir
authors.affiliation: Department of Lymphoma and Myeloma, The University of Texas MD Anderson Cancer Center, Houston, Texas.
authors.forename: Jatin J
authors.initials: JJ
authors.lastname: Shah
authors.affiliation: Department of Stem Cell Transplantation and Cellular Therapy, The University of Texas MD Anderson Cancer Center, Houston, Texas.
authors.forename: Chitra
authors.initials: C
authors.lastname: Hosing
authors.affiliation: Department of Stem Cell Transplantation and Cellular Therapy, The University of Texas MD Anderson Cancer Center, Houston, Texas.
authors.forename: Uday
authors.initials: U
authors.lastname: Popat
authors.affiliation: Department of Lymphoma and Myeloma, The University of Texas MD Anderson Cancer Center, Houston, Texas.
authors.forename: Robert Z
authors.initials: RZ
authors.lastname: Orlowski
authors.affiliation: Department of Stem Cell Transplantation and Cellular Therapy, The University of Texas MD Anderson Cancer Center, Houston, Texas.
authors.forename: Richard E
authors.initials: RE
authors.lastname: Champlin
authors.affiliation: Department of Stem Cell Transplantation and Cellular Therapy, The University of Texas MD Anderson Cancer Center, Houston, Texas.
authors.forename: Simrit
authors.initials: S
authors.lastname: Parmar
authors.affiliation: Department of Stem Cell Transplantation and Cellular Therapy, The University of Texas MD Anderson Cancer Center, Houston, Texas. Electronic address: mqazilba@mdanderson.org.
authors.forename: Muzaffar H
authors.initials: MH
authors.lastname: Qazilbash
mesh_terms: D000328:Adult; D000368:Aged; D000686:Amyloidosis / Q000150:complications* / Q000401:mortality / Q000628:therapy*; D005260:Female; D018380:Hematopoietic Stem Cell Transplantation / Q000379:methods*; D006801:Humans; D007147:Immunoglobulin Light Chains / Q000378:metabolism*; D008297:Male; D008875:Middle Aged; D015996:Survival Rate; D019172:Transplantation Conditioning / Q000379:methods*; D014182:Transplantation, Autologous / Q000379:methods*; D016896:Treatment Outcome
chemical_list: D007147:Immunoglobulin Light Chains
keywords: Amyloidosis; Autologous hematopoietic stem cell transplantation; Response; Survival
references.citation: Blood. 2004 May 15;103(10):3960-3
references.pmid: 14739213
references.citation: Mayo Clin Proc. 2008 Oct;83(10):1131-8
references.pmid: 18828972
references.citation: Blood. 2013 Jun 27;121(26):5124-30
references.pmid: 23670179
references.citation: N Engl J Med. 2007 Sep 13;357(11):1083-93
references.pmid: 17855669
references.citation: Br J Haematol. 2003 Jun;121(5):749-57
references.pmid: 12780789
references.citation: Ann Intern Med. 2004 Jan 20;140(2):85-93
references.pmid: 14734330
references.citation: Am J Hematol. 2005 Aug;79(4):319-28
references.pmid: 16044444
references.citation: J Clin Oncol. 2001 Jul 15;19(14):3350-6
references.pmid: 11454882
references.citation: Swiss Med Wkly. 2014 Feb 19;144:w13922
references.pmid: 24554372
references.citation: Blood. 1998 May 15;91(10):3662-70
references.pmid: 9573002
references.citation: Clin J Am Soc Nephrol. 2006 Nov;1(6):1331-41
references.pmid: 17699366
references.citation: N Engl J Med. 2003 Aug 7;349(6):583-96
references.pmid: 12904524
references.citation: Haematologica. 2007 Oct;92(10):1415-8
references.pmid: 17768110
references.citation: Blood. 2011 Oct 20;118(16):4346-52
references.pmid: 21828140
references.citation: Bone Marrow Transplant. 2001 Oct;28(7):637-42
references.pmid: 11704785
references.citation: J Clin Oncol. 2012 Dec 20;30(36):4541-9
references.pmid: 23091105
references.citation: Bone Marrow Transplant. 2011 Jul;46(7):970-5
references.pmid: 20935685
references.citation: Blood. 2012 Feb 2;119(5):1117-22
references.pmid: 22147893
references.citation: Bone Marrow Transplant. 2004 Jul;34(2):149-54
references.pmid: 15156165
references.citation: Mayo Clin Proc. 2006 Jul;81(7):880-8
references.pmid: 16835967
references.citation: Blood. 2007 Jan 15;109(2):492-6
references.pmid: 16960148
references.citation: Bone Marrow Transplant. 2016 Feb;51(2):307-9
references.pmid: 26479980
references.citation: J Clin Oncol. 2011 May 10;29(14):1924-33
references.pmid: 21483018
references.citation: J Clin Oncol. 2010 Feb 20;28(6):1031-7
references.pmid: 20085941
references.citation: Blood. 2007 Jan 15;109(2):457-64
references.pmid: 16990593
references.citation: Blood. 2002 Jun 15;99(12):4276-82
references.pmid: 12036853
references.citation: Leuk Lymphoma. 2010 Dec;51(12):2181-7
references.pmid: 20958232