Abstract:

Objective: To evaluate the short time efficiency and safety of autologous hematopoietic stem cell transplantation (AHSCT) in primary systemic amyloidosis (AL).  Methodology: A total of thirteen patients with biopsy-proven amyloidosis shown to be immunoglobulin light-chain type were enrolled on this study. The method of mobilization was granulocyte-colony-stimulating factor (G-CSF) alone. The patients received AHSCT in 2~6 weeks after collection. The condition regimen was high dose melphalan, the dose adjusted to 100, 140 and 200mg/m2 according to patient strati?cation. The assessment of organ involvement and treatment response was according to the consensus opinion from the 10th international symposium on amyloid and amyloidosis.  Results: The primary clinical symptoms of these patients was nephritic syndrome. The clonal light chain subtype, classi?ed by immunohistochemical staining, was κ in 2 patients and λ in 11 patients. The involved organ number was 1~3 (2.0±0.58), which in the kidneys (100%), heart (76.9%), liver (15.4%), and peripheral nerves (7.7%). 4 patients were untreated before AHSCT, while others were received induction therapy. All patients were collected enough stem cells. The main complications of collections were hypocalcemia, hypopotassaemia and thrombocytopenia. The number of CD34+ cells was 2.0~8.36×106/kg [(4.02±2.01) ×106/kg]. All patients had successful engraftment, and achieved a granulocyte count of 500/L between day 9 and day 13(median 9d), and an untransfused platelet count of 20 000/L from day 10 to day 21(13.69±2.56). The major complications of peri-transplantation period were nausea/vomiting (84.6%), mucositis (76.9%), fever (53.8%), arrhythmia (53.8%), acute renal injury (46.2%) and diarrhea (30.8%). The uncommon complications were acute liver injury, hepatic rupture, pleural effusion, sepsis and so on. One patient died of transplant-related hepatic rupture at the day 21. The treatment related mortality was 7.7% in day 100. After follow up for 4~10 months, 8 patients achieved hematologic response, 5 had complete response and 3 had partial response. 7 patients achieved organ response, 3 cases had renal and heart response, and 4 had renal response only.  Conclusion: The AHSCT was an efficient and safe treatment for AL in selected patients, the hematologic and organ response rate was high. Though the complications of peri-transplantation were common, the fatal complication was rare. The long-term outcomes need future observation.

Key words: autologous hematopoietic stem cell transplantation, primary systemic amyloidosis, , efficiency, complication