In June, results from the Phase II OPTIMUM/MUK nine clinical trial were presented at the European Haematology Association Annual Meeting and the American Society of Clinical Oncology Annual Conference.

This trial is the first to use state-of-the-art diagnostics and genetic profiling to screen for ultra-high risk myeloma patients to find better treatments for this hard-to-treat group of myeloma patients.

Following the initial screening of 472 newly diagnosed myeloma patients, 107 ultra-high risk myeloma patients were recruited to the treatment phase of the trial.

Ultra-high risk was defined through genomic testing, gene expression profiling, the presence of plasma cell leukaemia.

Trial participants received an intensive five-drug induction treatment, Dara-CVRd (daratumumab (Darzalex®), cyclophosphamide, bortezomib (Velcade®), lenalidomide (Revlimid®) and dexamethasone) followed by high-dose therapy and stem cell transplantation (HDT-SCT). This was augmented with bortezomib, followed by Dara-VR(d) consolidation for 18 cycles and Dara-R maintenance.

The overall response rate (ORR) following induction was 94%, with 80% of patients achieving very good partial response or greater (≥ VGPR) and 41% achieving minimal residual disease (MRD)-negative status.

At day 100 post HDT-SCT the ORR was 83% with 79% achieving ≥ VGPR and 64% MRD-negativity.

The most frequent severe adverse events during induction were neutropenia (21%), thrombocytopenia (12%) and infection (12%).

These results show that treatment with a five-drug induction treatment (Dara-CVRd) and stem cell transplant is effective and well tolerated in ultra-high risk myeloma patients.

However, it also shows that even with this intensive treatment, a small number of patients relapsed early. This highlights the clear need for more research and focused drug development for this hard-to-treat group of myeloma patients.