Stem cell transplantation in patients with autonomic neuropathy due to primary (AL) amyloidosis.
OBJECTIVE
Patients with AL amyloidosis can benefit from high-dose chemotherapy and autologous stem cell transplantation (ASCT). Transplantation can be challenging due to fluid shifts, sepsis, and cardiac dysrhythmias. Amyloidosis may present with autonomic neuropathy (AN) that renders peritransplant care problematic. The purpose of this study was to determine the outcome of patients with AN during and after ASCT.


METHODS
We performed a case-control study of patients with AL amyloidosis with associated AN and compared them to a large matched cohort without AN who also underwent ASCT.


RESULTS
We identified 13 patients with AN who underwent ASCT and a matched control group of 95 patients without AN. Patients with AN had more organs involved (median 2.5 vs 1, p < 0.001) and the conditioning dose of melphalan was often reduced by 30% compared to controls without AN (p = 0.0015). Median duration of hospitalization was similar for both cohorts, as were engraftment kinetics. Atrial fibrillation occurred in all patients with AN but in only 1 control patient (p < 0.0001). Median overall survival (OS) for patients with AN was 29 months but >60 months for controls (p < 0.0001). On univariate analysis, cardiac involvement (p = 0.0132), AN (p = 0.0011), glomerular filtration rate (p = 0.038), number of organs involved (p = 0.0064), and NT-pro-BNP (p = 0.039) all had an impact on OS. On multivariate analysis, AN retained an independent adverse impact on OS.


CONCLUSIONS
Patients with autonomic neuropathy secondary to AL amyloidosis can undergo autologous stem cell transplantation with relative safety. Autonomic neuropathy is an independent, adverse determinant of survival in these patients.
Division of Hematology, Department of Internal Medicine, Mayo Clinic, Rochester, MN 55905, USA. dingli.david@mayo.edu
D
D
Dingli
T S
TS
Tan
S K
SK
Kumar
F K
FK
Buadi
A
A
Dispenzieri
S R
SR
Hayman
M Q
MQ
Lacy
D A
DA
Gastineau
W J
WJ
Hogan
M A
MA
Gertz
D000328:Adult; D000368:Aged; D017772:Amyloid Neuropathies / Q000503:physiopathology / Q000601:surgery*; D018906:Antineoplastic Agents, Alkylating / Q000627:therapeutic use; D001342:Autonomic Nervous System Diseases / Q000503:physiopathology / Q000601:surgery*; D016022:Case-Control Studies; D016009:Chi-Square Distribution; D003131:Combined Modality Therapy; D016208:Databases, Factual; D005260:Female; D006321:Heart / Q000294:innervation / Q000503:physiopathology*; D018380:Hematopoietic Stem Cell Transplantation; D006801:Humans; D007668:Kidney / Q000294:innervation / Q000503:physiopathology*; D008297:Male; D008558:Melphalan / Q000627:therapeutic use; D008875:Middle Aged; D011379:Prognosis; D018709:Statistics, Nonparametric; D019172:Transplantation Conditioning; D016896:Treatment Outcome
D018906:Antineoplastic Agents, Alkylating; D008558:Melphalan