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

• Published in: Bone marrow transplantation (Basingstoke) Vol. 51; no. 12; pp. 1569 - 1572
• Main Authors: Rosengren, S, Mellqvist, U-H, Nahi, H, Forsberg, K, Lenhoff, S, Strömberg, O, Ahlberg, L, Linder, O, Carlson, K
• Format: Journal Article
• Language: English
• Published: London Nature Publishing Group UK 01-12-2016; Nature Publishing Group
• Subjects: 692/699/1541; 692/700/565/2319; Adult; Amyloidosis; Autografts; Bone marrow; Care and treatment; Cell Biology; Dexamethasone; Disease-Free Survival; Dosage and administration; Female; Follow-Up Studies; Heart; Heart Diseases; Hematology; Hematopoietic Stem Cell Transplantation - methods; Humans; Immunoglobulin Light-chain Amyloidosis - mortality; Immunoglobulin Light-chain Amyloidosis - pathology; Immunoglobulin Light-chain Amyloidosis - therapy; Internal Medicine; Male; Medicin och hälsovetenskap; Medicine; Medicine & Public Health; Melphalan; Melphalan - administration & dosage; Middle Aged; Mortality; Multiple Organ Failure; original-article; Patient outcomes; Patients; Prognosis; Public Health; Stem cell transplantation; Stem Cells; Survival; Survival Rate; Sweden; Time-to-Treatment; Transplantation; Transplantation, Autologous; Treatment Outcome; Sweden
• ISSN: 0268-3369; 1476-5365; 1476-5365
• DOI: 10.1038/bmt.2016.249

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.