Long-term results of single-agent thalidomide as initial therapy for asymptomatic (smoldering or indolent) myeloma.
We report the long-term follow-up results of a phase II trial of thalidomide for early-stage multiple myeloma (MM). Patients were eligible if they had smoldering multiple myeloma (SMM) or indolent MM without the need for immediate therapy. Thalidomide was initiated at a dose of 200 mg/day and adjusted as tolerated. Disease progression was defined using modified American Society of Hematology/Food and Drug Administration consensus panel criteria for SMM. Thirty-one patients were enrolled; 29 (19 SMM and 10 indolent MM) were eligible. The median age was 61 years. Median follow-up of living patients was 10.2 years (range, 7.5-11.0 years). Ten patients (34%) had a partial response (PR) and nine had minimal response (MR) for an MR plus PR rate of 66%. The median time to progression (TTP) to symptomatic myeloma was 35 months. Median TTP was 61 months in those achieving PR, 39 months with MR, and 9 months among those failing to achieve either MR or PR, P = 0.005. Median overall survival from diagnosis was 86 months; median survival from onset of symptomatic myeloma was 49 months. Grade 3-4 nonhematologic adverse events were noted in 55% of patients. Randomized trials are needed to determine the role of early therapy in SMM.
Detweiler-Short
Kristen
K
Division of Hematology, Mayo Clinic, 200 First Street SW, Rochester, MN 55905, USA.
Hayman
Suzanne
S
Gertz
Morie A
MA
Lacy
Martha Q
MQ
Dispenzieri
Angela
A
Kumar
Shaji
S
Zeldenrust
Steven R
SR
Russell
Stephen J
SJ
Lust
John A
JA
Kyle
Robert A
RA
Greipp
Philip R
PR
Witzig
Thomas E
TE
Vincent Rajkumar
S
S
D000368:Aged; D000970:Antineoplastic Agents / Q000008:administration & dosage / Q000009:adverse effects / Q000627:therapeutic use*; D003248:Constipation / Q000139:chemically induced; D018450:Disease Progression; D018572:Disease-Free Survival; D004334:Drug Administration Schedule; D005221:Fatigue / Q000139:chemically induced; D005260:Female; D005500:Follow-Up Studies; D006801:Humans; D053208:Kaplan-Meier Estimate; D008297:Male; D008875:Middle Aged; D009101:Multiple Myeloma / Q000188:drug therapy*; D010523:Peripheral Nervous System Diseases / Q000139:chemically induced; D012074:Remission Induction; D013792:Thalidomide / Q000008:administration & dosage / Q000009:adverse effects / Q000627:therapeutic use*; D016896:Treatment Outcome
D000970:Antineoplastic Agents; D013792:Thalidomide