Abstract
OBJECTIVES: Multiple myeloma is a malignant neoplasm characterised by uncontrolled growth of monoclonal plasma cells in the bone marrow, leading to the overproduction of nonfunctional intact immunoglobulins or immunoglobulin chains with several complications, including anaemia, bone lesions, infections, hypercalcaemia, renal failure, fatigue, and pain. In recent years, treatment with daratumumab, a monoclonal antibody that binds to the CD38 protein, has proved to be the most effective in slowing the progression of the disease. This mAb acts by inducing the destruction of target cells that express CD38, which are highly prevalent in patients with multiple myeloma. The following study aims to evaluate the response to treatment with daratumumab in patients treated at the Haematology Day Hospital of the Agostino Gemelli Polyclinic in Rome and to compare the overall survival results obtained using Kaplan-Meier curves for patients treated at our hospital with those in registration studies, to evaluate any variability in response. METHODS: First, data was extracted from the Policlinico's anti-cancer therapy management portal, extrapolating all infusions carried out between June 2018 and April 2024. The data for each individual patient was completed also taking additional information from the AIFA (Italian Medicines Agency) portal. Once this database was obtained, we proceeded with the processing of the data, evaluating parameters such as treatment persistence, dose intensity or treatment adherence, follow-up, budget impact, and pharmacovigilance. RESULTS: Real-world OS was generally lower than in clinical trials, highlighting complexities in an unselected patient population with advanced age and comorbidities. Treatment adherence varied markedly with some patients showing persistence up to 2100 days. In relapsed/refractory MM patients treated with DRd protocol, 63-month OS was around 30%, compared to about 50% in corresponding clinical trials (MMY3004), while newly diagnosed patients not eligible for transplant had 30-month OS nearing 75% compared to about 80% in corresponding clinical trials (MMY3007). CONCLUSION: Real-world data therefore offer a more realistic view of the efficacy and safety of treatments because they reflect the use of drugs in everyday clinical practice, on patients who are more heterogeneous than those in trials and allow evidence to be collected more quickly and at lower cost.