Clinical investigation of primary amyloidosis with autologous hematopoietic stem cell transplantation.
- Author:
Zhi-xiang QIU
1
;
Mang-ju WANG
;
Li-hong WANG
;
Yu-hua SUN
;
Wei-lin XU
;
Wei LIU
;
Jin-ping OU
;
Yu-jun DONG
;
Wen-sheng WANG
;
Yuan LI
;
Yue YIN
;
Ze-yin LIANG
;
Xi-nan CEN
;
Han-yun REN
Author Information
- Publication Type:Journal Article
- MeSH: Adult; Aged; Amyloidosis; drug therapy; mortality; surgery; Cardiovascular System; physiopathology; Female; Gastrointestinal Hemorrhage; physiopathology; Hematopoietic Stem Cell Transplantation; Humans; Immunoglobulin Light-chain Amyloidosis; Kidney; physiopathology; Male; Melphalan; therapeutic use; Middle Aged; Retrospective Studies; Risk Factors; Survival Rate; Transplantation, Autologous; Treatment Outcome
- From: Chinese Journal of Hematology 2012;33(3):187-190
- CountryChina
- Language:Chinese
-
Abstract:
OBJECTIVETo 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.
METHODSRetrospective 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.
RESULTSOf 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).
CONCLUSIONTreatment 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.