MUKnine OPTIMUM protocol: a screening study to identify high-risk patients with multiple myeloma suitable for novel treatment approaches combined with a phase II study evaluating optimised combination of biological therapy in newly diagnosed high-risk multiple myeloma and plasma cell leukaemia.

摘要: Introduction Multiple myeloma (MM) is a plasma cell tumour with over 5800 new cases each year in the UK. The introduction of biological therapies has improved outcomes for majority patients MM, but approximately 20% characterised by genetic changes which confer significantly poorer prognosis, generally termed high-risk (HR) MM. It important to diagnose these early and identify more effective first-line treatment options patients. Methods analysis Myeloma UK nine OPTIMUM trial (MUKnine) evaluates novel strategies HRMM. Patients suspected or newly diagnosed fit intensive therapy, are offered participation screening protocol (MUKnine a), primary endpoint proportion molecular performed within 8 weeks. identified as molecularly HR invited into phase II, single-arm, multicentre b) investigating an schedule comprising bortezomib, lenalidomide, daratumumab, low-dose cyclophosphamide dexamethasone, single high-dose melphalan autologous stem transplantation (ASCT) followed combination consolidation maintenance therapy. MUKnine b endpoints minimal residual disease (MRD) at day 100 post-ASCT progression-free survival. Secondary include response, safety quality life. uses Bayesian decision rule determine if this strategy sufficiently active further study. not having receive standard up cohort Exploratory studies longitudinal whole-body diffusion-weighted MRI imaging MRD testing. Ethics dissemination approval London South East Research Committee (Ref: 17/LO/0022, 17/LO/0023). Results will be submitted publication peer-reviewed journal. Trial registration number ISRCTN16847817, May 2017; Pre-results.