This study mainly evaluated the efficacy and safety of autologous stem cell transplantation for the treatment of AL amyloidosis, the role of induction and maintenance therapy in autologous stem cell transplantation, and the long-term efficacy and prognosis risk factors of autologous stem cell transplantation for the treatment of AL amyloidosis.
This study is divided into two parts. In the first part, the investigators retrospectively analyze the data of patients with AL amyloidosis who treated with autologous stem cell transplantation from July 2010 to December 2019. All patients had a biopsy-proven disease by positive Congo red stain with a concomitant demonstration of plasma cell dyscrasia. Organ involvement was established according to the criteria established at the 10th International Symposium on Amyloid and Amyloidosis. The protocol of ASCT included mobilization with colony-stimulating factor alone and conditioning with high-dose melphalan 140 or 200 mg/m2. In addition to analyzing the efficacy and safety of all the patients, the investigators also analyzed the difference in efficacy between patients in different subgroups. For example, subgroups divided according to different induction treatment regimens; subgroups divided according to different plasma cell FISH data and FCM data; subgroups divided according to the degree of organ involvement, and subgroups divided according to different maintenance treatment regimens. In the second part of the study, the investigators will conduct a prospective study to explore the best autologous stem cell transplantation treatment protocol for AL amyloidosis. The protocol includes the induction therapy with bortezomib and daratumumab before ASCT, the maintenance treatment with lenalidomide after ASCT, and the treatment options for recurrence after transplantation.
Study Type
OBSERVATIONAL
Enrollment
500
National Clinical Research Center of Kidney Diseases, Jinling Hospital
Nanjing, Jiangsu, China
overall survival
the 5 years overall survival after autologous stem cell transplantation.
Time frame: 5 years
progression-free survival
the 5 years progression-free survival after autologous stem cell transplantation.
Time frame: 5 years
hematological complete response rate
the 1 year hematologic complete response rate after autologous stem cell transplantation.
Time frame: 1 year
organ response rate
the 1 years organ response rate after autologous stem cell transplantation.
Time frame: 1 year
hematological overall response rate
the 1 years hematologic overall response rate after autologous stem cell transplantation.
Time frame: 1 year
the relapse rate of complete remission participants
the relapse rate of complete remission participants after autologous stem cell transplantation.
Time frame: 5 years
the rate of Minimal Residual Disease-negative participants
the rate of Minimal Residual Disease-negative participants after autologous stem cell transplantation.
Time frame: 5 years
the median time form stem cell transplantation to next treatment
the median time form stem cell transplantation to next chemotherapy or antibody treatment.
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Time frame: 5 years