Haema 2013; 4(3):228-237
by Marie-Christine Kyrtsonis1, Ioanna Vardounioti1, Panagiotis Repousis2
1Haematology Section, 1st Department of Propaedeutic Internal Medicine, National and Kapodistrian University of Athens Medical School, Laikon General Hospital
2Department of Hematology, Metaxa Cancer Hospital, Piraeus, Greece
AL amyloidosis, POEMS syndrome and Waldenström′s macroglobulinemia (Wμ) are three rare and distinct disease entities that share the presence of a serum monoclonal immunoglobulin. All three disorders present a wide spectrum of clinical manifestations, as well as varying outcomes among patients; their diagnosis is often hard to establish. Considerable improvements have been made in their management over the past years but they remain incurable. High dose chemotherapy followed by autologous stem cell transplantation (ASCT) proved to be a valuable option for patients able to tolerate it. In both AL amyloidosis and POEMS syndrome, ASCT can eventually be applied up-front without previous chemotherapy, since disease burden is usually low. In AL amyloidosis, ASCT produces sustained remissions and tissue amyloid clearance with subsequent clinical improvement in more than half of the patients, even in the presence of cardiac involvement that is associated with considerable morbidity and poor outcome. In POEMS syndrome, the results of ASCT are very encouraging; almost all patients respond and survival is considerably prolonged, as reported in the few series available in the medical literature. As compared to chemotherapy alone, response rates, duration of response and overall survival are improved in patients treated with ASCT. Relapses are however eventually observed. In patients with Wμ, experience with ASCT is limited because of the rarity of the disease, its indolent nature and the advanced age of the majority of patients. Reported results are unclear and the superiority of ASCT over chemotherapy is not established yet.