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What Is Known and Objective: High costs of novel agents increasingly put pressure on limited healthcare budgets. Demonstration of their real-world costs and cost-effectiveness is often required for reimbursement. However, few published economic evaluations of novel agents for multiple myeloma exist. Moreover, existing cost analyses were heavily based on conventionally treated patients. We investigated real-world health care costs of relapsed/refractory multiple myeloma in Dutch daily practice., Methods: A retrospective medical chart review was conducted for 139 patients treated between January 2001 and May 2009. Total monthly costs attributable to each cost component were described across all regimens and for bortezomib-, thalidomide- and lenalidomide-based treatment regimens., Results: Mean monthly total costs (€3,981) varied depending on the sequence of therapy (range: €442-€31,318). Significant cost drivers across all regimens included costs of therapy and hospital admissions. The acquisition costs for novel agents in particular accounted for 32% of mean total monthly costs. Prognostic factors associated with increased mean total monthly costs in multivariate regression analysis included low platelet counts (P = 0·01) and worsening performance status (P < 0·001). Mean total monthly costs of bortezomib- and lenalidomide-based regimens were significantly higher than those for thalidomide-based regimens in second, third and fourth treatment line., What Is New and Conclusions: Real-world costs during treatment of relapsed/refractory multiple myeloma vary greatly. Cost drivers include hospital admissions and acquisition costs of novel agents. Costs also vary by prognostic factors and treatment-related resource use. Future studies assessing the costs of combination therapy consisting of two or more novel agents are encouraged., (© 2012 Blackwell Publishing Ltd.)

Thalidomide with melphalan/prednisone (MPT) was defined as standard treatment in elderly patients with multiple myeloma (MM) based on five randomized trials. In one of these trials, HOVON49, a prospective health-related quality-of-life (HRQoL) study was initiated in order to assess the impact of thalidomide on QoL. Patients aged >65 years with newly diagnosed MM were randomized to receive melphalan plus prednisone (MP) or MPT, followed by thalidomide maintenance in the MPT arm. Two hundred eighty-four patients were included in this side study (MP, n=149; MPT n=135). HRQoL was assessed with the EORTC Core QoL Questionnaire (QLQ-C30) and the myeloma-specific module (QLQ-MY24) at baseline and at predetermined intervals during treatment. The QLQ-C30 subscales physical function (P=0.044) and constipation (P<0.001) showed an improvement during induction in favour of the MP arm. During thalidomide maintenance, the scores for the QLQ-MY24 paraesthesia became significantly higher in the MPT arm (P<0.001). The QLQ-C30 subscales pain (P=0.12), insomnia (P=0.068), appetite loss (P=0.074) and the QLQ-MY24 item sick (P=0.086) scored marginally better during thalidomide maintenance. The overall QoL-scale QLQ-C30-HRQoL showed a significant time trend towards more favourable mean values during protocol treatment without differences between MP and MPT. For the QLQ-C30 subscales emotional function and future perspectives, difference in favour of the MPT arm from the start of treatment was observed (P=0.018 and P=0.045, respectively) with no significant 'time × arm' interaction, indicating a persistent better patient perspective with MPT treatment. This study shows that the higher frequency of toxicity associated with MPT does not translate into a negative effect on HRQoL and that MPT holds a better patient perspective.

Objectives: To investigate the subjective well-being of patients with newly diagnosed multiple myeloma who were treated in a tandem transplantation programme., Methods: Fifty-one patients participated in the prospective, longitudinal questionnaire study. The EORTC QLQ-C30 and the EuroQol-5D were administered 2 wk after completion of vincristine, adriamycin and dexamethason/vincristine, adriamycin and methyl prednison (VAD/VAMP) chemotherapy, both at hospital discharge after treatment with high-dose melphalan (HDM) and 1 month after this hospital discharge, at hospital admission, at the day of hospital discharge for peripheral stem cell transplantation (PSCT) and at 6 and 12 months following discharge after PSCT., Results: Overall, patients' functioning improved during treatment and follow-up, with significant decreases shortly following PSCT. Shortly after HDM and PSCT, patients reported a considerable increase in levels of soreness in the mouth (+26/+36 points on a scale ranging form 0 to 100; P < 0.01), change of taste (+23/+21 points; P < 0.05/NS), nausea/vomiting (+26/+27 points; P < 0.01/< 0.05), appetite loss (+40/+43 points; P < 0.001) and diarrhoea (+25/+36 points; P < 0.01). However, none of these symptoms persisted during follow-up., Conclusion: The intensive treatment programme was subjectively being well tolerated by the majority of patients. The duration of declined quality of life after administration of HDM seemed to be short. The duration of subjective recovery after PSCT remained uncertain, but in any case was present at the 6 month follow-up. Together with the rather good results in survival, the evaluation of quality of life invites further exploration of double transplantations in multiple myeloma., ((c) Blackwell Munksgaard 2005)

A prospective randomised phase III study in patients < or =65 years old with previously untreated multiple myeloma (MM), intensive chemotherapy followed by myeloablative chemotherapy and autologous stem-cell rescue was compared with intensive chemotherapy alone. This economic evaluation was based on detailed data from patient charts and hospital information systems. In the intention-to-treat analysis, mean total treatment and follow-up costs of the myeloablative treatment arm were 81,643 euros compared to 68,802 euros for the chemotherapy arm (P=0.09). Costs per quality-adjusted life year were 51,357 euros versus 37,328 euros. In the clinical study, no significant differences were found in overall survival after a median follow-up of 33 months from randomisation. Intensive chemotherapy is regarded as standard therapy for younger patients with previously untreated MM. Cost-effectiveness of myeloma therapy after 3 years of follow up seems not to be favoured by myeloablative treatment with autologous stem-cell rescue.

In a retrospective study, we calculated the treatment and follow-up costs of patients with newly diagnosed multiple myeloma. The total treatment programme consisted of eight phases: VAD or VAMP chemotherapy, follow-up I, high-dose melphalan followed by transplantation of whole blood, follow-up II, collection of peripheral blood progenitor cells by leukapheresis, follow-up III, high-dose chemotherapy (busulfan/cyclophosphamide) followed by reinfusion of peripheral stem cells and follow-up IV (until 3 months from hospital discharge after peripheral stem cell transplantation). For each phase the average costs were calculated for all patients who were on treatment/follow-up in each particular phase. The total average cumulative costs of treatment and follow-up of all patients amounted to US$49850. Considering only the patients who completed the total treatment programme as it was scheduled, the average total treatment and follow-up costs were US$44800. The average costs of treatment and follow-up of patients who did not complete the programme as it was scheduled (patients who died, patients who were withdrawn from treatment and patients who received additional treatment) were US$57025.

In a retrospective study, we calculated the treatment costs of 26 patients, who received either high dose melphalan combined with granulocyte colony-stimulating factor (G-CSF; filgrastim)(n = 7) or without G-CSF (n = 11) or alternatively, peripheral blood progenitor cell reinfusion (PBPC) mobilised by G-CSF following high dose melphalan. In comparison with the control group, a shortening of the pancytopenic period and platelet recovery was noticed in the PBPC group. This resulted in a reduction in hospital costs, diagnostics, laboratory services, total parenteral nutrition and transfusions. The average costs per treatment in the PBPC group amounted to about US$ 17,908 as compared to US$ 32,223 in the control group, implying a cost reduction of 44% when changing to PBPC reinfusion.