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Cited by 6Amyloidosis: Diagnosis, Treatment, OutcomesMultiple Myeloma Research and TreatmentsLymphoma Diagnosis and Treatment

Outcome of patients with immunoglobulin light-chain amyloidosis with lung, liver, gastrointestinal, neurologic, and soft tissue involvement after autologous hematopoietic stem cell transplantation

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Afrough, Aimaz, Saliba, Rima M., Hamdi, Amir, El Fakih, Riad, Varma, Ankur, Dinh, Yvonne T., Rondon, Gabriela, Cornelison, A. Megan +9 more·2015·Biology of Blood and Marrow Transplantation
DOI 10.1016/j.bbmt.2015.03.025PMC PMC4825317
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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.

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  • Advanced therapies can improve GI symptoms in selected patients with amyloidosis, including stem cell transplantation with cytotoxic therapy in systemic amyloidosis and liver transplantation in transthyretin amyloidosis.

    “Advanced therapies can improve GI symptoms in selected patients, including stem cell transplantation with cytotoxic therapy in systemic amyloidosis and liver transplantation in transthyretin amyloidosis (Afrough et al., 2015; Wixner et al., 2015).”

    From Autonomic nervous system and gastrointestinal manifestations with an emphasis on gastric dysfunction (2026) · cites this paper as Afrough et al., 2015

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References 32 · Cited by 6

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Authors
Afrough, A., Saliba, R. M., Hamdi, A., El Fakih, R., Varma, A., Dinh, Y. T., Rondon, G., Cornelison, A. M., Shah, N. D., Bashir, Q., Shah, J. J., Hosing, C., Popat, U., Orlowski, R. Z., Champlin, R. E., Parmar, S., & Qazilbash, M. H.
Affiliations
  • The University of Texas MD Anderson Cancer Center (US)
  • The University of Texas Health Science Center at Houston (US)
Corresponding author
Qazilbash, Muzaffar H.
ORCID
Venue
Biology of Blood and Marrow Transplantation
Publisher
Elsevier BV
Published
2015-08
Volume
21
Issue
8
Pages
1413-1417
ISSN
1083-8791 (ISSN-L)
DOI
10.1016/j.bbmt.2015.03.025
PMC
PMC4825317
OpenAlex
W2119182043
Cited by
6
References
32
Citations by year
2026: 1 · 2021: 1 · 2020: 1 · 2016: 1 · 2015: 2
FWCI
0.43
Type
journal article
Language
en
Open access
Licence
Locations
  • Transplantation and Cellular Therapy (publishedVersion, cc-by-nc-nd)
  • PubMed (publishedVersion)
  • PubMed Central (acceptedVersion)
Funders
National Cancer Institute (P30CA016672)
Topics
  • Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › Amyloidosis: Diagnosis, Treatment, Outcomes
  • Health Sciences › Medicine › Hematology › Multiple Myeloma Research and Treatments
  • Health Sciences › Medicine › Pathology and Forensic Medicine › Lymphoma Diagnosis and Treatment
Keywords
immunoglobulin light chain amyloidosis, autologous stem cell transplantation, high-dose chemotherapy, gastrointestinal involvement, peripheral nerve involvement, liver involvement, pulmonary involvement, soft tissue involvement, non-relapse mortality, progression-free survival, overall survival, organ response
MeSH terms
Adult, Aged, Amyloidosis, Female, Humans, Immunoglobulin Light Chains, Male, Middle Aged, Transplantation, Autologous, Survival Rate, Treatment Outcome, Hematopoietic Stem Cell Transplantation, Transplantation Conditioning

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