HOVON 104, long-term follow-up of bortezomib-dexamethasone induction therapy followed by autologous stem cell transplantation in newly diagnosed AL amyloidosis patients.

The HOVON 104 studied bortezomib-dexamethasone induction therapy and autologous stem cell transplantation in 50 patients, of whom 35 received an autologous stem cell transplantation (ASCT). We demonstrate a 5-year overall survival (OS) of 73% and progression-free survival (PFS) of 52% for all 50 patients with a median follow-up of 61.3 months. For the 35 transplanted patients, calculated from the date of ASCT, the 5-year OS and PFS were 91% and 68%, respectively. After ASCT, the rate of organ response improved over time but stabilized around 3 years. A complete cardiac response was seen in around 60% of patients and remained stable from 2 years onward. Reaching complete renal response was slower over time and achieved by 61% of the renal-affected patients at 5 years. We confirm the excellent outcomes after ASCT and demonstrate a 60% complete organ response with longer follow-up.
Competing Interests: MCM Research Funding: Beigene Consultancy: Janssen Cilag, BMS, GSK, CDR‐life, Speakers Bureau: Siemens, Janssen Cilag, all paid to the institution, UH Speakers Bureau: Janssen Cilag, Pfizer, Alnylam, Akcea, Prothena, Astra Zeneca, Hospitality: Janssen, Prothena, Pfizer. Advisory Boards: Pfizer, Prothena, Janssen, Alexion. MJK Honoraria: BMS/Celgene, Kite/Gilead, Novartis, Roche; Consulting or Advisory Role: BMS/Celgene, Kite/Gilead, Miltenyi, Biotech, Novartis, Takeda, Adicet Bio and Miltenyi Biomedicine; Research Funding: Kite/Gilead, all paid to the institution. SZ Research funding: Janssen Cilag, Advisory board: Janssen Cilag, BMS, Sanofi, Oncopeptides, Amgen, all paid to the institution. AB Advisory board: Janssen Pharmaceuticals, Amgen, Celgene, BMS, Takeda. PS Research funding: Janssen Pharmaceuticals, Amgen, Celgene, Karyopharm. Advisory board: Janssen Pharmaceuticals, Pfizer, BMS. SOS Research support: Janssen, Prothena, Sanofi, Neurimmune Advisory boards: Janssen, Telix and Prothena Honoraria: AstraZeneca, Alexion, Sobi, Janssen, Takeda, Pfizer, Prothena. KN, PFY, SC, LT, KLW, GMJB, and HK declare no conflict of interest.
(© 2024 The Author(s). eJHaem published by British Society for Haematology and John Wiley & Sons Ltd.)