Staged heart transplantation and chemotherapy as a treatment option in patients with severe cardiac light-chain amyloidosis.
OBJECTIVE
The prognosis of advanced cardiac light-chain amyloidosis is poor. Heart transplantation might enable causative therapy and ultimately improve prognosis.


RESULTS
Nineteen patients with cardiac amyloidosis but no obvious involvement of other organs were scheduled for heart transplantation. Four to 6 months later, high-dose melphalan chemotherapy and autologous stem cell transplantation (HDM-ASCT) was planned in patients not in complete remission. Seven of nineteen patients died while waiting for heart transplantation. The remaining 12 patients (complete remission, n = 4) underwent surgery. Chemotherapy in patients not in complete remission consisted of HDM-ASCT (n = 5/12; subsequent complete remission, n = 2; partial remission, n = 3) or melphalan-prednisolone (partial remission, n = 1). Two of twelve patients were ineligible for any chemotherapy. Three of twelve patients died [423.5 (105-2131) days] from progressive disease, relapse, or sepsis. The 1- and 3-year survival rates were 83 and 83%, respectively, similar to those of patients undergoing heart transplantation for standard indications. Corresponding survival rates stratified by haematological response were 100 and 100% for complete remission (partial remission, 100 and 100%; progressive disease, 0 and 0%).


CONCLUSIONS
Heart transplantation in advanced cardiac amyloidosis is a promising approach to interrupting the vicious circle of ineligibility for potential curative chemotherapeutic treatment and extremely poor prognosis of cardiac amyloidosis without chemotherapy. Highly urgent heart transplantation combined with subsequent HDM-ASCT appears to offer a successful treatment option to improve the poor outcome of cardiac amyloidosis. However, it should be restricted to highly selected patients in specialized centres.
Kristen
Arnt V
AV
Department of Cardiology, Angiology, and Respiratory Medicine, University Hospital Heidelberg, Im Neuenheimer Feld 410, Heidelberg D-69120, Germany. arnt_kristen@med.uni-heidelberg.de
Sack
Falk-Udo
FU
Schonland
Stefan O
SO
Hegenbart
Ute
U
Helmke
Burkhard M
BM
Koch
Achim
A
Schnabel
Philipp A
PA
Röcken
Christoph
C
Hardt
Stefan
S
Remppis
Andrew
A
Goldschmidt
Hartmut
H
Karck
Matthias
M
Ho
Anthony D
AD
Katus
Hugo A
HA
Dengler
Thomas J
TJ
D000686:Amyloidosis / Q000175:diagnosis / Q000188:drug therapy* / Q000401:mortality / Q000601:surgery*; D015331:Cohort Studies; D003131:Combined Modality Therapy; D018572:Disease-Free Survival; D005260:Female; D006084:Graft Rejection; D006085:Graft Survival; D006331:Heart Diseases / Q000175:diagnosis / Q000188:drug therapy* / Q000401:mortality / Q000601:surgery*; D016027:Heart Transplantation / Q000009:adverse effects / Q000379:methods*; D018380:Hematopoietic Stem Cell Transplantation / Q000379:methods; D006801:Humans; D053208:Kaplan-Meier Estimate; D008297:Male; D008558:Melphalan / Q000627:therapeutic use; D011336:Probability; D011379:Prognosis; D012189:Retrospective Studies; D018570:Risk Assessment; D012720:Severity of Illness Index; D015996:Survival Rate; D014182:Transplantation, Autologous; D016896:Treatment Outcome; D014850:Waiting Lists
D008558:Melphalan