A prospective observational study of 915 patients with systemic AL amyloidosis treated with upfront bortezomib
| Autor: | Darren Foard, Sajitha Sachchithanantham, Cristina Quarta, Shameem Mahmood, Faye Sharpley, Philip N. Hawkins, Oliver C Cohen, Richa Manwani, Helen J. Lachmann, Carol J. Whelan, Marianna Fontana, Julian D. Gillmore, Ashutosh D. Wechalekar |
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| Rok vydání: | 2019 |
| Předmět: |
Adult
Male medicine.medical_specialty Immunology Biochemistry Gastroenterology Disease-Free Survival Bortezomib 03 medical and health sciences 0302 clinical medicine Internal medicine medicine AL amyloidosis Humans Immunoglobulin Light-chain Amyloidosis Prospective Studies Prospective cohort study Survival rate Aged Aged 80 and over business.industry Amyloidosis Cell Biology Hematology Middle Aged medicine.disease Survival Rate Clinical trial 030220 oncology & carcinogenesis Female Observational study Response Duration business 030215 immunology medicine.drug |
| Zdroj: | Blood. 134:2271-2280 |
| ISSN: | 1528-0020 0006-4971 |
| Popis: | Bortezomib is a standard therapy in light-chain amyloidosis (AL), but little is known about response duration. A difference in involved amyloidogenic and uninvolved serum-free light chains (dFLC)10 mg/L (low dFLC response) predicts survival in AL patients with low presenting dFLC (20-50 mg/L). We report outcomes in the largest AL cohort treated with upfront bortezomib and explore the impact of posttreatment dFLC10 mg/L ("stringent dFLC response"). A total of 915 newly diagnosed AL patients treated with bortezomib and assessed at our center were included. Hematologic responses, 6-month dFLC, organ responses, overall survival (OS), and time-to-next-treatment (TNT) (excluded patients who died without starting second-line treatment) were evaluated. Overall response rate (intent-to-treat) was 65%, with 49% complete response (CR)/very good partial response/low dFLC response and with a stringent dFLC response, dFLC 10-40 mg/L, and dFLC40 mg/L was 30%, 22%, and 48%, respectively. Median OS was 72 months. A total of 289 patients died without progressing to second-line treatment. Median TNT was not reached, and 55% had not progressed to further treatment at 7 years. Patients with stringent dFLC responses had significantly better OS and TNT than did those with lesser responses. A total of 72% of CR patients did not progress to further treatment at 3 years compared with 84% with stringent dFLC responses. Cardiac responses were better in those with stringent dFLC responses (61%) compared with lesser responses (45%; P = .005). Upfront bortezomib confers durable hematologic responses. A stringent dFLC response predicts prolonged TNT and impressive organ responses. |
| Databáze: | OpenAIRE |
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