High-dose intravenous melphalan with autologous stem cell transplantation in AL amyloidosis-associated end-stage renal disease.
BACKGROUND
The development of end-stage renal disease (ESRD) is common among patients with amyloid light-chain AL amyloidosis-associated renal disease and survival of these patients is poor. High-dose intravenous melphalan and autologous stem cell transplantation induce remission of the plasma cell dyscrasia in a significant proportion of patients with AL amyloidosis. The efficacy and tolerability of such treatment for patients with AL amyloidosis-associated ESRD are unknown.


METHODS
Between June 1994 and June 2000, 15 patients with AL amyloidosis-associated ESRD were treated with intravenous melphalan (70 to 200 mg/m2) and autologous peripheral blood stem cell transplantation. Clinical and laboratory data were prospectively collected prior to treatment, during the peritransplant period, and at 3 months, 12 months, and annually thereafter. Treatment outcomes and toxicities were compared with 180 non-ESRD patients treated during the study period.


RESULTS
Eight of 15 patients (53%) had a hematologic complete response following treatment. Two patients (13%) died during the peritransplant period. Transfusion requirements were greater and there was a trend toward increased severity of mucositis in the ESRD patients compared with the non-ESRD patients. Median survival for the ESRD patients with a hematologic complete response was 4.5 years. Five patients with hematologic complete response have either undergone or are awaiting renal transplantation.


CONCLUSIONS
High-dose intravenous melphalan with stem cell transplantation is an effective treatment in selected patients with AL amyloidosis-associated ESRD. Although the toxicity profile is greater in ESRD patients, the treatment offers the possibility of successful renal transplantation if hematologic remission is achieved. This treatment should be considered for patients with AL amyloidosis-associated ESRD.
Casserly
Liam F
LF
Department of Medicine, Boston University School of Medicine, Boston, Massachusetts 02118, USA.
Fadia
Amit
A
Sanchorawala
Vaishali
V
Seldin
David C
DC
Wright
Daniel G
DG
Skinner
Martha
M
Dember
Laura M
LM
D000328:Adult; D000368:Aged; D000686:Amyloidosis / Q000150:complications / Q000188:drug therapy* / Q000401:mortality; D018906:Antineoplastic Agents, Alkylating / Q000008:administration & dosage* / Q000009:adverse effects; D005260:Female; D006801:Humans; D007275:Injections, Intravenous; D007676:Kidney Failure, Chronic / Q000188:drug therapy* / Q000209:etiology / Q000401:mortality; D008297:Male; D008558:Melphalan / Q000008:administration & dosage* / Q000009:adverse effects; D008875:Middle Aged; D011446:Prospective Studies; D012074:Remission Induction; D033581:Stem Cell Transplantation; D016019:Survival Analysis; D014182:Transplantation, Autologous
D018906:Antineoplastic Agents, Alkylating; D008558:Melphalan