Abstract

Long-term survival remains poor for patients with cardiac amyloidosis. High-dose melphalan (MEL) with stem cell transplantation (HDM/SCT) is an effective treatment for AL amyloidosis, but patients with cardiac involvement are ineligible because of high therapy-related mortality. Here we report detailed HDM/SCT outcomes of 9 patients with cardiac failure. Their median age was 56 years (range, 45∼66). After a median follow-up of 15 months (range 9∼32), three died of multiorgan failure within the early phase after HDM/SCT, and the other six including poor risk patients are alive at present. Their symptoms of cardiac decompensation have improved. Decreases in interventricular septum thickness were confirmed in 4 patients 6∼12 months after HDM/SCT by echocardiography. One-year overall survival rate was 67%, longer than previously reported rates. HDM/SCT may lead to improvements in quality of life and extended survival in cardiac amyloidosis patients. Meanwhile, the median dosage of MEL in our procedure was 103 mg/m(2) (range 68∼180), less than the recommended dose, and patients were maintained on miscellaneous therapies. Further studies are required to clarify an effective MEL dose and to refine selection criteria for patients undergoing HDM/SCT.

Links

Publisher Full Text

Authors

Ogura M, Sekine R, Nishiyama S, Abe Y, Iizuka H, Kusaka S, Nakagawa Y, Suzuki K

Institution

Department of Hematology, Japanese Red Cross Medical Center.

Source

[Rinshō ketsueki] The Japanese journal of clinical hematology 53:7 2012 Jul pg 710-5

MeSH

Aged
Amyloidosis
Cardiomyopathies
Female
Heart Failure
Heart Function Tests
Humans
Male
Melphalan
Middle Aged
Peripheral Blood Stem Cell Transplantation
Retrospective Studies
Transplantation, Autologous
Treatment Outcome

Pub Type(s)

English Abstract
Journal Article

Language

jpn

PubMed ID

22975774