[Clinical investigation of primary amyloidosis with autologous hematopoietic stem cell transplantation].
OBJECTIVE
To investigate the treatment of primary amyloidosis with high-dose melphalan and autologous hematopoietic stem cell transplantation to further examine the survival, hematologic response, and improvement of amyloid-related organ dysfunction.


METHODS
Retrospective analysis of 20 patients with primary amyloidosis treated with autologous hematopoietic stem cell transplantation. The status of major organ function before transplantation, mobilization programs and conditioning regimen as possible risk factors for survival were also investigated.


RESULTS
Of 20 cases, 11 out of 15 evaluable cases achieved hematologic response, among them, 6 got complete remission (CR, 40%) and 5 partial remission (PR, 33%). The median onset time was 3.0 months (1.5 - 4.0 months) and 4 months (3 - 5 months), respectively after transplantation. The overall hematologic response was 73%. The 11 hematologic responders also had kidney responses. The median time to achieve kidney response was 3 months (2 - 6 months). The 3-year overall survival of the cohort of cases was 71.4%. The major causes of death were heart failure, renal dysfunction and gastrointestinal bleeding. G-CSF alone could obtain satisfactory mobilization results and most of patients well tolerated to the conditioning regimen of melphalan doses from 140 mg/m(2) to 200 mg/m(2).


CONCLUSIONS
Treatment of primary amyloidosis with high-dose melphalan followed by autologous peripheral blood stem cell transplantation produced high efficacy. The cardiovascular system involvement, renal dysfunction and the abnormality of coagulation function before transplantation may be the risk factors for survival.
Department of Hematology, Peking University First Hospital, Beijing, China.
Zhi-xiang
ZX
Qiu
Mang-ju
MJ
Wang
Li-hong
LH
Wang
Yu-hua
YH
Sun
Wei-lin
WL
Xu
Wei
W
Liu
Jin-ping
JP
Ou
Yu-jun
YJ
Dong
Wen-sheng
WS
Wang
Yuan
Y
Li
Yue
Y
Yin
Ze-yin
ZY
Liang
Xi-nan
XN
Cen
Han-yun
HY
Ren
D000328:Adult; D000368:Aged; D000686:Amyloidosis / Q000188:drug therapy / Q000401:mortality / Q000601:surgery*; D002319:Cardiovascular System / Q000503:physiopathology; D005260:Female; D006471:Gastrointestinal Hemorrhage / Q000503:physiopathology; D018380:Hematopoietic Stem Cell Transplantation; D006801:Humans; D000075363:Immunoglobulin Light-chain Amyloidosis; D007668:Kidney / Q000503:physiopathology; D008297:Male; D008558:Melphalan / Q000627:therapeutic use; D008875:Middle Aged; D012189:Retrospective Studies; D012307:Risk Factors; D015996:Survival Rate; D014182:Transplantation, Autologous; D016896:Treatment Outcome
D008558:Melphalan