Salvage therapy with thalidomide in multiple myeloma patients relapsing after autologous peripheral blood stem cell transplantation

Background and Objectives. The introduction of high-dose therapy with stem cell support has significantly improved the outcome of patients with multip le myeloma (MM) in terms of increased complete remission ICR) rate and exte nded survival, both disease-free and overall. Few options, however, are pre sently available for patients who relapse after single or double autologous stem cell transplantation (SCT). Thalidomide, a glutamic acid derivative w ith anti-angiogenetic properties, has been recently proposed as salvage tre atment for such patients. The present study was aimed at evaluating thalido mide as single agent therapy for patients who had previously received autol ogous peripheral blood stem cell transplantation, Design and Methods. From October 1999 to August 2000, 11 patients (7 males/ 4 females) who had relapsed after single (n=4) or double (n=7) autologous p eripheral blood SCT were enrolled in the trial, Thalidomide, always employe d as a single agent, was initially administered at a dose of 100 mg/day; if well tolerated, the dose was increased serially by 200 mg every other week to a maximum of 800 mg/day. Results. The median administered dose was 600 mg/day, WHO grade > II toxic effects were constipation, lethargy, and leukopenia. Four patients (36%) sh owed > 50% reduction in serum M protein concentration and 4 showed > 25% re duction, for a total response rate averaging 72%. After a median follow-up of 5 months, 3 out of 8 responding patients are alive and progression-free and 5 patients have relapsed. Interpretation and Conclusions. These data confirm that thalidomide is acti ve in poor-prognosis MM patients such as those relapsing after autologous S CT, and could thus deserve further testing in combination therapy, (C) 2001 , Ferrata Storti Foundation.