Primary therapy of Waldenström's macroglobulinemia with 2-chlorodeoxyadenosine.
OBJECTIVE
To assess the activity of 2-chlorodeoxyadenosine (2CdA) as primary therapy for patients with Waldenström's macroglobulinemia.


METHODS
2CdA was given to 26 consecutive, previously untreated and symptomatic patients with Waldenström's macroglobulinemia. Two courses were administered to outpatients at a dose of 0.1 mg/kg body weight per day for a 7-day continuous infusion using a portable pump through a central venous catheter. Responding patients were followed up without further therapy and were scheduled to receive two additional treatments with 2CdA on disease relapse.


RESULTS
Twenty-two of 26 patients responded to the 2CdA therapy (85%; 95% confidence interval [CI], 65% to 96%), including three patients who achieved a complete response and 19 patients who had a partial response. Treatment was well tolerated, with no acute hematologic toxicity. A marked and sustained reduction of CD4+ lymphocytes occurred in all patients and may have contributed to a fatal infection with disseminated herpes simplex in one patient. With a median follow-up of 13 months, five patients have relapsed and all re-treated patients have responded again to 2CdA.


CONCLUSIONS
2CdA is highly active in previously untreated patients with Waldenström's macroglobulinemia. A limited program of treatment induced responses of good quality and long duration in more than 80% of patients.
Dimopoulos
M A
MA
Department of Hematology, University of Texas M.D. Anderson Cancer Center, Houston.
Kantarjian
H
H
Weber
D
D
O'Brien
S
S
Estey
E
E
Delasalle
K
K
Rose
E
E
Cabanillas
F
F
Keating
M
M
Alexanian
R
R
D000328:Adult; D000368:Aged; D000369:Aged, 80 and over; D018791:CD4 Lymphocyte Count; D017338:Cladribine / Q000009:adverse effects / Q000627:therapeutic use*; D018572:Disease-Free Survival; D005260:Female; D005500:Follow-Up Studies; D006801:Humans; D007262:Infusions, Intravenous; D008297:Male; D008875:Middle Aged; D012008:Recurrence; D012074:Remission Induction / Q000379:methods; D016019:Survival Analysis; D016896:Treatment Outcome; D008258:Waldenstrom Macroglobulinemia / Q000188:drug therapy* / Q000401:mortality
D017338:Cladribine