# Target trial emulation of carfilzomib safety among patients with relapsed/refractory multiple myeloma using a nationwide observational data in Korea

## TL;DR

This study evaluates carfilzomib's safety in Korean patients with multiple myeloma using real-world data, finding increased risks of certain adverse events compared to another treatment.

## Contribution

The study provides real-world safety data for carfilzomib in Korean patients with relapsed/refractory multiple myeloma using a nationwide observational database.

## Key findings

- KRd treatment was associated with higher risks of hematologic adverse events like anemia and thrombocytopenia compared to Rd.
- Cardiovascular events such as heart failure and hypertension were significantly more common in the KRd group.
- The safety profile of carfilzomib in Korean patients was consistent with previous RCTs but highlighted increased cardiovascular risks.

## Abstract

Carfilzomib, commonly used for relapsed/refractory multiple myeloma (RRMM), has been associated with various adverse events in randomized controlled trials (RCTs). However, real-world safety data for a more diverse population are needed, as carfilzomib received expedited approval. This study aimed to evaluate carfilzomib’s safety in Korea by comparing new users of KRd (carfilzomib, lenalidomide, and dexamethasone) to Rd (lenalidomide and dexamethasone) using a nationwide administrative claims database.

The retrospective cohort study utilized target trial emulation, focusing on adverse events in various organ systems similar to the ASPIRE trial.

This study included 4,580 RRMM patients between 2007 and 2020, and the KRd group showed significantly higher risks of hematologic adverse events (anemia, neutropenia, thrombocytopenia) and some non-hematologic adverse events (cough, hypokalemia, constipation, hypertension, heart failure) compared to the Rd group. Among non-hematologic adverse events, cardiovascular events (heart failure [HR 2.04; 95% CI 1.24–3.35], hypertension [HR 1.58; 95% CI 1.15–2.17]) had the highest risk in the KRd group.

The safety profile of carfilzomib in Korean patients was similar to previous RCTs. Therefore, caution should be exercised when using carfilzomib in Asian individuals with RRMM due to the increased risk of cardiovascular adverse events.

The online version contains supplementary material available at 10.1007/s00432-024-05800-8.