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Molecularly Guided Therapy Dramatically Improves Survival in High‑Risk Myeloma

Regulators are reviewing the MMProfiler SKY92 gene‑expression test that would let the NHS identify high‑risk patients for tailored treatment.

Overview

  • Long‑term results published in Lancet Oncology show about 70% overall survival at roughly six years for patients on the Optimum (MUK Nine) risk‑adapted regimen versus about 40% with standard care and 54% progression‑free at six years versus 18% in usual care.
  • The randomised trial enrolled 107 newly diagnosed high‑risk myeloma patients and used a gene‑expression classifier to select those for intensified treatment.
  • Trial treatment combined an early high‑intensity induction—autologous stem cell transplant plus daratumumab, cyclophosphamide, bortezomib, lenalidomide and dexamethasone—with prolonged combination maintenance while disease remained controlled.
  • A SKY92‑defined subgroup had especially large benefit, with 62% alive and progression‑free at six years compared with 20% on conventional therapy, showing the test can pinpoint who gains most from the approach.
  • The MMProfiler SKY92 test is not yet routine in the NHS and is under regulatory review, so wider patient access will depend on regulator approval, health‑service capacity for molecular testing, further validation and commissioning decisions; patients on the trial such as Ken Theobold report extended survival and maintained quality of life on maintenance therapy.