上田 真寿

Daratumumab (DARA) has become a standard care for treating patients with multiple myeloma (MM) and light chain (AL) amyloidosis. The subcutaneous (SC) administration of DARA has been demonstrated to be non-inferior to an intravenous formulation. This retrospective study evaluated patients with MM and/or AL amyloidosis who received SC DARA injections at Tochigi Medical Center Shimotsuga (TMC) and Jichi Medical University (JMU). At TMC, the first dose of DARA was administered on an outpatient basis, whereas at JMU, it was generally administered as an inpatient treatment. At TMC, nurses and pharmacists carefully explained the precautions when administering DARA 1 week in advance. DARA was administered by a physician and patients were observed for 6 h after the first dose. Consequently, outpatient DARA administration was associated with a much shorter median total chair time (345 min) than inpatient treatment (median, 7 days). The incidence of administration-related reactions was low (only one patient in each group). The results revealed that, with aggressive premedication and medical cooperation, the first dose of SC DARA can be safely administered in an outpatient setting.

BACKGROUND: Despite its promising outcome, anti-BCMA CAR-T is the most expensive myeloma treatment that has ever been developed, and its cost-effectiveness is an important issue. OBJECTIVE: This study aimed to assess the cost-effectiveness of anti-BCMA CAR-T in comparison with standard anti-myeloma therapy in RRMM patients. STUDY DESIGN: The model assumed myeloma patients in Japan and the US who have received ≥3 prior lines of anti-myeloma therapies including PIs, IMiDs, and anti-CD38 mAbs. A Markov model was constructed to compare the 'CAR-T' strategy, in which patients receive either idecabtagene vicleucel (ide-cel) or ciltacabtagene autoleucel (cilta-cel) followed by three lines of multiagent chemotherapy after relapse, to the 'no CAR-T' strategy, in which patients only receive chemotherapies. The data from the LocoMMotion, KarMMa, and CARTITUDE-1 trials were extracted. Several assumptions were made regarding long-term progression-free survival (PFS) with CAR-Ts. Extensive scenario analyses were made regarding regimens for 'no CAR-T' strategies. The outcome was an incremental cost-effectiveness ratio (ICER) with willingness-to-pay thresholds of \ 7,500,000 in Japan and $150,000 in the US. RESULTS: When a 5-year PFS of 40% with cilta-cel was assumed, the ICER of the 'CAR-T' versus 'no CAR-T' strategies was \7,603,823 in Japan and $112,191 in the US per QALY over a 10-year time horizon. When a 5-year PFS of 15% with ide-cel was assumed, the ICER was \20,388,711 in Japan and $261,678 in the US per QALY over a 10-year time horizon. The results were highly dependent on the PFS assumption with CAR-T and were robust to changes in most other parameters and scenarios. CONCLUSION: Although anti-BCMA CAR-T can be cost-effective even under current pricing, a high long-term PFS is necessary.