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Fifty secondary progressive multiple sclerosis (SPMS) patients who had lost one or more EDSS points in the prior two years were selected to receive either cyclophosphamide (25 patients, 13 females, 12 males, F/M = 1.08; mean age: 42.4 years; mean disease duration: 13.3 years; mean EDSS at study entry: 5.7) or mitoxantrone (25 patients, 14 females, 11 males, F/M = 1.27; mean age: 38.2 years; mean disease duration: 11.5 years; mean EDSS at study entry: 5.5). SPMS patients were treated for two years with clinical evaluation (relapse rate, disability progression) every three months and radiological imaging (conventional magnetic resonance imaging) before therapy initiation and at the end of the first and second years of therapy. Safety profile and costs of the two therapeutic protocols were also analysed. In terms of clinical and radiological measures the drugs exerted a quite identical effect on both, and produced a significant reduction in both relapse rate (mitoxantrone Mito): p = 0.001, cyclophosphamide (Cy): p = 0.003) and disability progression (Mito: p = 0.01; Cy: p = 0.01). Subgroups of mitoxantrone- and cyclophosphamide-responding patients were identified (14/25 and 17/25, respectively) and were characterized by a significantly shorter duration of the secondary progressive phase of the disease. In these subgroups, the improvement in the EDSS score at the end of therapy was highly significant (p<0.0001 for Mito, p = 0.0004 for Cy). The safety profiles of both drugs were acceptable; however, the Cy-based therapy protocol was significantly less expensive. We conclude that Cy should be considered as a therapeutic option in rapidly deteriorating SPMS patients.

Background: Current treatment regimens for Waldenstrom macroglobulinemia (WM) are based on the use of oral alkylating agents. Recently, however, other more costly agents have been proposed for the treatment of WM. In the current study, the authors report on results obtained using oral melphalan, cyclophosphamide, and prednisone (MCP) to treat 72 patients with WM, and they compare these results (and the associated costs) with those observed using more aggressive protocols., Methods: Between July 1973 and April 2002, the authors documented overexpression of the immunoglobulin M paraprotein in 317 consecutive patients. Of these, 100 had newly diagnosed WM, and the 72 who were symptomatic were treated using the MCP protocol. Response rate, overall survival (OS), response duration, freedom from progression (FFP), event-free survival (EFS) duration, toxicity, and cost per course in Euro and U.S. dollars were evaluated for patients receiving this regimen., Results: Seventy-one of 72 patients (99%) were evaluable. Of these patients, 55 (77%) achieved a response; 7 others (10%) experienced disease stabilization, and the remaining 9 (13%) experienced disease progression. After a median follow-up of 72 months (range, 3-195 months), the median durations of EFS, FFP, response, and OS were 47, 55, 64, and 66 months, respectively. No World Health Organization Grade III or IV toxicities were observed, and side effects were limited to transient nausea, emesis, and mild neutropenia. The cost per course of the MCP regimen was $16, similar to that of standard protocols involving chlorambucil and much less than that of more aggressive protocols (price range, $91-11091) proposed for the treatment of WM., Conclusions: Like chlorambucil-based protocols, the MCP regimen is a cost-effective and safe option for the treatment of patients with WM. Furthermore, the results obtained do not appear to be inferior to those yielded by more expensive, aggressive, and toxic intravenous protocols., ((c) 2004 American Cancer Society)

Objectives: The purpose of this analysis was to assess the real-life direct costs of drug delivery for frequently used chemotherapeutic regimens in patients with relapsed low-grade non-Hodgkin's lymphoma (NHL)., Methods: This was a retrospective analysis of direct costs of drug delivery (acquisition plus administration) of relapsed low-grade NHL in 424 patients in Canada, Germany, and Italy. Results were expressed as an average treatment cost per patient for six cycles of chemotherapy. Exchange rates used were $1 (Canada)= currency 0.672, 1 DM (Germany)= currency 0.511, and 1 Lit (Italy)= currency 0.000517., Results: Direct costs of drug delivery were greater for inpatients receiving fludarabine (Canada currency 12,669; Italy currency 13,027) than for CHOP (Canada currency 7856; Germany currency 7218; Italy currency 4251) or COP/CVP (Canada currency 7360; Germany currency 8449). Treatment administration setting was a major cost driver with inpatient treatment up to 9-fold more expensive than the same regimen given to outpatients. Drug administration costs comprised the largest proportion of the total for each regimen in the inpatient setting (69-98%). Costs of drug delivery in the outpatient setting were 10% to 65% of those in the inpatient setting. Again, fludarabine was more expensive (Italy currency 8493; Canada currency 7269) than CHOP (Canada currency 4403; Germany currency 2150; Italy currency 1264) and COP/CVP (Canada currency 3009; Germany currency 867). Administration costs were 2.5- to 15-fold higher for inpatients compared to outpatients., Conclusions: Costs of drug administration are a major driver for total direct treatment costs in the treatment of relapsed low-grade NHL and are at least as important as drug acquisition costs. Drug administration practices, in terms of inpatient or outpatient treatment, are a major factor in determining overall direct costs. Therapeutic strategies, which offer shortened treatment duration and/or a simple mode of administration, are likely to be economically attractive.