title: Thalidomide in relapsed/refractory multiple myeloma: pivotal trials conducted outside the United States.
abstract: Recently completed phase II trials and retrospective analyses conducted outside the United States, primarily in Europe and Australia, have confirmed results of landmark US trials that established the efficacy of thalidomide in multiple myeloma. These trials evaluated thalidomide alone or in combination in patients with heavily pretreated relapsed/ refractory multiple myeloma. Single-agent thalidomide induced objective responses in approximately one third of patients and prolonged survival by approximately 1 year. These trials also indicated a possible dose-response relationship of thalidomide and identified several poor prognostic factors, including advanced age, high lactose dehydrogenase level, low platelet count, and low hemoglobin level. The combination of thalidomide with dexamethasone increased overall response by approximately 20% and reduced the time to response compared with thalidomide alone. In addition, lower doses of thalidomide used in the combination may increase patient tolerability. Finally, thalidomide in combination with chemotherapy yields response rates of approximately 60%, with median survival times of approximately 18 months. Randomized trials are needed to confirm these results and further explore the role of thalidomide in these treatment settings.
authors.affiliation: Department of Clinical Therapeutics, University of Athens School of Medicine, 227 Kifissias Ave, Kifissia, Athens 14651, Greece.
authors.forename: Meletios A
authors.initials: MA
authors.lastname: Dimopoulos
authors.forename: Athanasios
authors.initials: A
authors.lastname: Anagnostopoulos
mesh_terms: D000276:Adjuvants, Immunologic / Q000008:administration & dosage / Q000009:adverse effects / Q000627:therapeutic use; D020533:Angiogenesis Inhibitors / Q000008:administration & dosage / Q000009:adverse effects / Q000627:therapeutic use; D000971:Antineoplastic Combined Chemotherapy Protocols / Q000008:administration & dosage / Q000627:therapeutic use*; D001315:Australia; D017322:Clinical Trials, Phase II as Topic / Q000706:statistics & numerical data; D003520:Cyclophosphamide / Q000008:administration & dosage; D003907:Dexamethasone / Q000008:administration & dosage; D018572:Disease-Free Survival; D004305:Dose-Response Relationship, Drug; D019008:Drug Resistance, Neoplasm; D005060:Europe; D006801:Humans; D009101:Multiple Myeloma / Q000188:drug therapy* / Q000401:mortality / Q000473:pathology; D012307:Risk Factors; D016879:Salvage Therapy; D016019:Survival Analysis; D013792:Thalidomide / Q000008:administration & dosage / Q000009:adverse effects / Q000627:therapeutic use*; D016896:Treatment Outcome
chemical_list: D000276:Adjuvants, Immunologic; D020533:Angiogenesis Inhibitors; D013792:Thalidomide; D003907:Dexamethasone; D003520:Cyclophosphamide