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Kenneth H. Shain, MD, PhD, discusses the evolution of treatment for patients with newly diagnosed multiple myeloma and how physicians are leveraging data with chimeric antigen receptor T-cell therapy and minimal residual disease negativity to improve outcomes.

We headed to Tampa, Florida for a State of the Science Summit on Hematologic Malignancies. The meeting covered updates in follicular lymphoma, chronic lymphocytic leukemia, mantle cell lymphoma, multiple myeloma, acute lymphoblastic leukemia, chronic myeloid leukemia, acute myeloid leukemia, myeloproliferative neoplasms, and CAR T-cell therapy.

The first patient has been treated in a phase 2b dose-confirmation study of AMT-061, an investigational gene therapy for the treatment of patients with severe and moderately severe hemophilia B. Once the dosing of AMT-061 is confirmed, the safety and efficacy of the therapy will be evaluated in the global phase 3 HOPE-B clinical trial.

During a meeting of the Medicare Evidence Development and Coverage Advisory Committee (MEDCAC), panelists heard from chimeric antigen receptor (CAR) T therapy drug makers, health researchers, and policy makers, and mostly endorsed including patient-reported outcomes (PROs) in its final national coverage analysis decision, expected next year.