[High-dose treatment of systemic AL-amyloidosis with autologous stem cell support].
BACKGROUND
AL-amyloidosis is a serious disease with a short median survival without treatment. Treatment with high-dose melphalan with autologous stem cell support (HMAS) has a potential to increase survival, but is associated with toxicity and mortality. In this paper we report the Norwegian results retrospectively.


METHODS
We used questionnaires and had personal contact with a local physician from each hospital with HMAS experience. Diagnosis and treatment were evaluated according to the guidelines at the time of treatment, and the results were compared to internationally published reports.


RESULTS
Stem cell harvesting was attempted in 18 patients from 1997 to 2006. 15 of these received HMAS treatment. Treatment-related mortality was 20%, and 5 of 11 (45%) had an organ response. Median survival was not reached within the 55-month median observation time. The course of the disease was more complicated when known risk factors for HMAS treatment were present, such as reduced kidney function, advanced heart involvement, reduced performance status, and multiorgan disease. Three of 18 patients were not diagnosed according to relevant guidelines. In seven of 12 patients the response to treatment was not evaluated adequately with respect to haematology.


CONCLUSIONS
AL-amyloidosis is a difficult diagnosis and the condition is probably under-diagnosed in Norway. The results of HMAS treatment in Norway are comparable with those in published reports from centres abroad. The follow-up of patients should be improved.
Medisinsk avdeling Seksjon for blodsykdommer Rikshospitalet 0027 Oslo. fredrikschjesvold@gmail.com
Fredrik Hellem
FH
Schjesvold
Malvin
M
Sjo
Jon-Magnus
JM
Tangen
Jens
J
Hammerstrøm
Lorentz
L
Brinch
D000368:Aged; D000686:Amyloidosis / Q000175:diagnosis / Q000188:drug therapy / Q000401:mortality / Q000628:therapy*; D018906:Antineoplastic Agents, Alkylating / Q000009:adverse effects / Q000627:therapeutic use; D003131:Combined Modality Therapy; D006801:Humans; D008558:Melphalan / Q000009:adverse effects / Q000627:therapeutic use; D008875:Middle Aged; D010818:Practice Patterns, Physicians'; D012189:Retrospective Studies; D033581:Stem Cell Transplantation / Q000009:adverse effects; D011795:Surveys and Questionnaires; D015996:Survival Rate; D014182:Transplantation, Autologous
D018906:Antineoplastic Agents, Alkylating; D008558:Melphalan