title: Current therapeutic possibilities in primary and secondary amyloidosis and our experience with 31 patients.
abstract: Primary (AL) and secondary (AA) amyloidosis are systemic diseases characterized by a process of amyloid deposition in many organs with unsatisfactory survival of patients. Apart from surgical intervention in those patients with bronchiectasias or osteomyelitis, the possibilities of influencing the development of AA amyloidosis are limited. The milestone therapy in patients with rheumatic diseases includes early treatment with DMARDs (disease-modifying antirheumatic drugs). A new promising therapeutic alternative is represented by anti-tumour necrosis factor-alpha (TNF-alpha) drugs such as infliximab and etanercept. The last class of agents used in the treatment of AA interferes with fibril formation: iododoxorubicin and low molecular weight sulfates (fibrilex). In the group of patients with AL, in addition to the standard combination of melphalan and prednisone, other therapeutic approaches such as ASCT (autologous stem cell transplantation) and new drugs with different mechanisms of action have been added recently. For the future, we can expect the development of immunotherapy (both active vaccination and passive immunization). In our department, we have treated 17 patients with AL and 14 patients with AA amyloidosis since 1995. We used various treatment regimens in both groups of patients. The treatment stabilized the disease or achieved partial remission in only 36% of patients with AA amyloidosis despite the use of intensive therapeutic modalities, while in the AL group a response was achieved in 82% of patients. ASCT improves patients survival in AL amyloidosis, but strict selection criteria are necessary (less than two affected organs and no signs of myocardial dysfunction).
authors.affiliation: First Medical Department, First Medical Faculty, Charles University, Prague, Czech Republic. rysavar@ms.vfn.cz
authors.forename: R
authors.initials: R
authors.lastname: Rysavá
authors.forename: M
authors.initials: M
authors.lastname: Merta
authors.forename: I
authors.initials: I
authors.lastname: Spicka
authors.forename: V
authors.initials: V
authors.lastname: Tesar
mesh_terms: D000328:Adult; D000368:Aged; D000682:Amyloid / Q000378:metabolism*; D000686:Amyloidosis / Q000209:etiology* / Q000401:mortality / Q000473:pathology / Q000628:therapy*; D015331:Cohort Studies; D003131:Combined Modality Therapy; D003907:Dexamethasone / Q000008:administration & dosage*; D004305:Dose-Response Relationship, Drug; D004334:Drug Administration Schedule; D004359:Drug Therapy, Combination; D005260:Female; D005500:Follow-Up Studies; D006801:Humans; D008297:Male; D008558:Melphalan / Q000008:administration & dosage*; D008875:Middle Aged; D011241:Prednisone / Q000008:administration & dosage*; D012189:Retrospective Studies; D018570:Risk Assessment; D012720:Severity of Illness Index; D033581:Stem Cell Transplantation / Q000379:methods; D016019:Survival Analysis; D016896:Treatment Outcome
chemical_list: D000682:Amyloid; D003907:Dexamethasone; D008558:Melphalan; D011241:Prednisone