Thalidomide therapy in adult patients with myelodysplastic syndrome. A North Central Cancer Treatment Group phase II trial.
BACKGROUND
Thalidomide has shown promise for the treatment of patients with myelodysplastic syndrome. The current prospective multicenter study examined the efficacy and toxicity of thalidomide in adult patients with myelodysplastic syndrome.


METHODS
Using the International Prognostic Scoring System (IPSS), patients were stratified into 2 groups: favorable (IPSS score, 0-1.0) or unfavorable (IPSS score, 1.5-3.5). Seventy-two patients (42 of whom were favorable and 30 of whom were unfavorable) received a starting dose of oral thalidomide of 200 mg daily. The dose was increased by 50 mg per week to a targeted maximum daily dose of 1000 mg.


RESULTS
According to the International Working Group response criteria for myelodysplastic syndrome, 1 patient in the unfavorable group achieved a partial remission with a complete cytogenetic response. Overall, 2 patients (5%) in the favorable group and 4 patients (14%) in the unfavorable group experienced either a hematologic improvement or a partial response. The most frequent Grade 3 or 4 (grading was based on the National Cancer Institute's Common Toxicity Criteria [version 2.0]) nonhematologic adverse events were fatigue (24%), infection (19%), neuropathy (13%), dyspnea (8%), and constipation (7%).


CONCLUSIONS
Thalidomide alone, at the schedule and dose levels used in the current study, is not a safe and viable therapeutic option for patients with myelodysplastic syndrome. Limited efficacy and increased toxicity were observed in the current Phase II trial.
Moreno-Aspitia
Alvaro
A
Division of Hematology and Oncology, Mayo Clinic, Jacksonville, Florida, USA.
Colon-Otero
Gerardo
G
Hoering
Antje
A
Tefferi
Ayalew
A
Niedringhaus
Robert D
RD
Vukov
Allen
A
Li
Chin-Yang
CY
Menke
David M
DM
Geyer
Susan M
SM
Alberts
Steven R
SR
D000368:Aged; D000369:Aged, 80 and over; D020533:Angiogenesis Inhibitors / Q000627:therapeutic use*; D018450:Disease Progression; D004305:Dose-Response Relationship, Drug; D005260:Female; D006801:Humans; D008297:Male; D020714:Maximum Tolerated Dose; D008875:Middle Aged; D009190:Myelodysplastic Syndromes / Q000188:drug therapy* / Q000473:pathology; D011446:Prospective Studies; D012307:Risk Factors; D015996:Survival Rate; D013792:Thalidomide / Q000627:therapeutic use*; D013997:Time Factors; D016896:Treatment Outcome
D020533:Angiogenesis Inhibitors; D013792:Thalidomide