Thalidomide in patients with advanced multiple myeloma: a study of 83 patients--report of the Intergroupe Francophone du Myélome (IFM).
BACKGROUND
To evaluate treatment by thalidomide and identify predictive factors of survival, event free survival and response among patients with advanced multiple myeloma treated with thalidomide as single agent therapy.


METHODS
Patients with advanced multiple myeloma (n=83) were treated with an oral dose of thalidomide (median 400 mg/day). At start of treatment, all patients had active disease and 58 (69%) had received at least one autologous transplantation.


RESULTS
With a median follow-up of 338 days (range, 247-629 days), 52 patients are alive, whereas 31 died between 8 and 150 days after the first administration of thalidomide. The response to thalidomide was considered as major in 11 patients (13%), partial in 29 patients (35%) and minor in 15 patients (18%), giving a total response rate of 66% (54 out of 83 patients). Thirteen patients had stable disease and 15 patients progressed. In multivariable analysis, age greater than 60 years, short interval between diagnosis and onset of thalidomide, requirement for red blood cell transfusion, IgA isotype, platelets' count <80 x 10(9)/l and serum albumin level <30 g/l at the start of thalidomide were associated with poor outcome. These three last factors produced a simplified prognostic model for patients with advanced myeloma and treated with thalidomide. Thus, among the 38 patients without any of these unfavorable risk features, one-year overall survival and event free survival were 87% and 78%. By contrast, the 43 patients with at least one unfavorable feature had one-year overall survival and event free survival of 40% and 32%, respectively (Log-Rank, P=0.0002 for both). Patients who received > or =34.4 g of thalidomide in the first 90 days of treatment had a better outcome than those who received <34.4 g. However, the mean received daily dose of thalidomide in the first 90 days has not been found to influence survival, event free survival or response. Short-term side effects of thalidomide were generally moderate.


CONCLUSIONS
Thalidomide is an effective treatment for patients with advanced myeloma, in particular, who have no poor-risk features. The poor results achieved by the other patients emphasize the need for prospective protocols using thalidomide in combination, especially with dexamethasone. In addition, further studies are needed to determine the optimal thalidomide dose and duration.
Service des Maladies du Sang, and the laboratoire de biostatistique CERIM, CHRU, Lille, France.
Ibrahim
I
Yakoub-Agha
Michel
M
Attal
Charles
C
Dumontet
Válerie
V
Delannoy
Philippe
P
Moreau
Christian
C
Berthou
Thierney
T
Lamy
Bernard
B
Grosbois
Charly
C
Dauriac
Véronique
V
Dorvaux
Jacques-Olivier
JO
Bay
Matthieu
M
Monconduit
Jean Luc
JL
Harousseau
Corinne
C
Duguet
Alain
A
Duhamel
Thierry
T
Facon
D000328:Adult; D000367:Age Factors; D000368:Aged; D000970:Antineoplastic Agents / Q000009:adverse effects / Q000627:therapeutic use*; D001772:Blood Cell Count; D003131:Combined Modality Therapy; D018572:Disease-Free Survival; D004341:Drug Evaluation; D017707:Erythrocyte Transfusion; D005260:Female; D005500:Follow-Up Studies; D018380:Hematopoietic Stem Cell Transplantation; D006801:Humans; D007070:Immunoglobulin A / Q000097:blood; D015993:Life Tables; D008297:Male; D008875:Middle Aged; D009101:Multiple Myeloma / Q000097:blood / Q000188:drug therapy* / Q000401:mortality / Q000473:pathology; D009194:Myeloma Proteins / Q000032:analysis; D011379:Prognosis; D012074:Remission Induction; D012307:Risk Factors; D016879:Salvage Therapy; D012709:Serum Albumin / Q000032:analysis; D016019:Survival Analysis; D013792:Thalidomide / Q000009:adverse effects / Q000627:therapeutic use*; D014182:Transplantation, Autologous; D016896:Treatment Outcome
D000970:Antineoplastic Agents; D007070:Immunoglobulin A; D009194:Myeloma Proteins; D012709:Serum Albumin; D013792:Thalidomide