Outcome of AL amyloidosis after high-dose melphalan and autologous stem cell transplantation in Sweden, long-term results from all patients treated in 1994-2009.
High-dose melphalan and autologous stem cell transplantation (HDM/ASCT) is widely used in immunoglobulin light chain (AL) amyloidosis, but the benefit is debated mainly because of the high treatment-related mortality (24% in a randomised study comparing HDM/ASCT with oral melphalan/dexamethasone). We report here on the long-term outcome of all patients treated with HDM/ASCT for AL amyloidosis in Sweden between 1994 and 2009. Seventy-two patients were treated at eight Swedish centres. Median follow-up was 67.5 months. At least partial response (organ or haematological) was seen in 64% of the patients. Median overall survival was 98 months or 8.2 years, with 5-year survival 63.9% and 10-year survival 43.4%. In patients with cardiac involvement or multiple organ involvement, survival was significantly shorter, median overall survival 49 and 56 months, respectively. All mortality within 100 days from ASCT was 12.5% for all patients and 17.2% in the patients with cardiac involvement. For patients treated in the earlier time period (1994-2001), 100-day mortality was 23.8% compared with 7.8% in the later period (2002-2009). In conclusion, long survival times can be achieved in patients with AL amyloidosis treated with HDM/ASCT, also in smaller centres. Early mortality is high, but with a decreasing trend over time.
Department of Medical Sciences, Division of Hematology, Uppsala University Hospital, Uppsala, Sweden.
S
S
Rosengren
Department of Hematology, South Elvsborg Hospital, Borås, Sweden.
U-H
UH
Mellqvist
Department of Hematology, Karolinska Institute, Stockholm, Sweden.
H
H
Nahi
Department of Hematology, Norrlands University Hospital, Umeå, Sweden.
K
K
Forsberg
Department of Hematology, Skåne University Hospital, Lund, Sweden.
S
S
Lenhoff
Department of Hematology, Karolinska Institute, Stockholm, Sweden.
O
O
Strömberg
Department of Hematology, Linköping University Hospital, Linköping, Sweden.
L
L
Ahlberg
Department of Hematology, Örebro University Hospital, Örebro, Sweden.
O
O
Linder
Department of Medical Sciences, Division of Hematology, Uppsala University Hospital, Uppsala, Sweden.
K
K
Carlson
D000328:Adult; D018572:Disease-Free Survival; D005260:Female; D005500:Follow-Up Studies; D006331:Heart Diseases; D018380:Hematopoietic Stem Cell Transplantation / Q000379:methods*; D006801:Humans; D000075363:Immunoglobulin Light-chain Amyloidosis / Q000401:mortality / Q000473:pathology / Q000628:therapy*; D008297:Male; D008558:Melphalan / Q000008:administration & dosage*; D008875:Middle Aged; D009102:Multiple Organ Failure; D015996:Survival Rate; D013548:Sweden; D061665:Time-to-Treatment; D014182:Transplantation, Autologous; D016896:Treatment Outcome
D008558:Melphalan
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