An observational study of once-weekly carfilzomib in patients with multiple myeloma in Japan (Weekly-CAR study)
Creators: name: Abe, Yu
name: Kubonishi, Shiro
name: Ri, Masaki
name: Iino, Masaki
name: Sunami, Kazutaka
name: Ito, Tomoki
name: Fukaya, Masafumi
name: Kitano, Toshiyuki
name: Ikeda, Sho
name: Ota, Shuichi
name: Kuroi, Taiga
name: Iriyama, Noriyoshi
name: Jo, Tatsuro
name: Adachi, Masaaki
name: Akahane, Daigo
name: Kai, Tatsuyuki
name: Kohara, Yoichi
name: Kadowaki, Norimitsu
name: Katayama, Teruaki
Publisher: name: Taylor & Francis
Year: 2024
Subjects: subject: Medicine
subject: Genetics
subject: FOS: Biological sciences
subject: Pharmacology
subject: Immunology
subject: FOS: Clinical medicine
subject: Cancer
subject: Science Policy
subject: Hematology
subject: Infectious Diseases
subject: FOS: Health sciences

description: <b>Background:</b> The ARROW study demonstrated that once-weekly carfilzomib and dexamethasone (wKd) therapy significantly prolonged progression-free survival compared with twice-weekly carfilzomib and dexamethasone therapy in relapsed or refractory multiple myeloma patients. <b>Aim:</b> To describe the treatment patterns, effectiveness and safety of wKd therapy in real-world settings in Japan. <b>Methods:</b> We investigated data from the medical records of 126 Japanese patients with relapsed or refractory multiple myeloma. <b>Results:</b> The overall response rate was 66.3%. The median progression-free survival was 9.5 months. The incidence of treatment-emergent adverse events of any grade and grade ≥3 were 45.8 and 20.8%, respectively. <b>Conclusion:</b> There were no new or unexpected safety signals in this study. This study demonstrated the effectiveness and safety profiles of wKd therapy in Japan. Carfilzomib became available for daily clinical practice as a drug for cancer of bone marrow (multiple myeloma) that comes back or does not respond to previous drug (relapsed or refractory). This drug was approved in the USA in 2012, and in Japan in 2016. In this study, we looked at how once-weekly carfilzomib works and how safe it is in real-life situations in Japan. We screened 126 patients with relapsed or refractory multiple myeloma in Japan. The median age of the patients was 70 years, with 25% being over 75 years. This study also included some patients who were not in the best overall health, had a history of many treatments or had heart complications. In 66.3% of patients, the cancer had disappeared or the extent of the cancer had reduced after treatment. Side effects and serious side effects occurred in 45.8 and 14.2% of patients, respectively. The most common side effects were low levels of blood platelets (9.2%), high blood pressure (5.8%), loose or watery stools (5.0%), fever (5.0%), and low levels of red blood cells (4.2%). Heart disorders occurred in five patients. But all patients recovered or improved with treatment such as blood pressure lowering drugs and diuretics. These results showed that once-weekly carfilzomib works well and is safe in real-world settings in Japan. This information can help us think about how to pick the right patients and handle heart disease risks when using carfilzomib treatment. Once-weekly carfilzomib in combination with dexamethasone (wKd) therapy is approved for the treatment of relapsed or refractory multiple myeloma (RRMM). Although the ARROW study demonstrated that wKd therapy significantly prolonged progression-free survival compared with twice-weekly carfilzomib and dexamethasone (20/27 mg/m<sup>2</sup>) therapy in RRMM patients, there are few reports based on real-world data for wKd therapy in Japanese patients. This Weekly-CAR study is an observational study that evaluated real-world clinical data on wKd treatment in Japanese patients with RRMM. In this study, the overall response rate and median progression-free survival were 66.3% and 9.5 months, respectively. There were no new or unexpected safety signals in this study. Additionally, this study demonstrated the effectiveness and safety in patients who were not recruitable to the clinical trial of wKd therapy, for example those with an Eastern Cooperative Oncology Group performance status ≥2, or those with 1 or ≥4 prior regimens. These data demonstrated the effectiveness and safety profiles of wKd therapy in real-world settings in Japan.