Controversies in multiple myeloma: Evidence-based update.

Area of Science:

• Hematology
• Oncology
• Clinical Pharmacology

Background:

• The US Food and Drug Administration (FDA) approved 10 new drugs for multiple myeloma (MM) over the past two decades.
• New highly effective anti-myeloma agents present complex clinical decision-making challenges.
• Existing evidence for treatment strategies in MM is often based on single-arm trials or comparisons to older regimens.

Purpose of the Study:

• To review evidence behind conflicting treatment strategies in four controversial clinical settings in multiple myeloma (MM).
• To provide evidence-based recommendations from myeloma experts on complex MM treatment decisions.
• To address the evolving landscape of MM therapeutics, including smoldering MM, induction, transplant, and maintenance therapy.

Main Methods:

• Selection of four controversial clinical settings in multiple myeloma (MM) treatment.
• Comprehensive review of existing evidence for conflicting treatment strategies.
• Expert panel discussions to formulate evidence-based recommendations.

Main Results:

• Treatment of smoldering multiple myeloma (SMM) remains investigational, though lenalidomide showed survival benefit in high-risk patients.
• Contemporary induction regimens for newly diagnosed MM often lack robust comparative trial data.
• Novel regimens achieve deep responses, potentially altering the role of autologous stem cell transplant (ASCT).
• Maintenance therapy in MM requires redefined risk-benefit profiles with accumulating long-term data.

Conclusions:

• The optimal management of multiple myeloma (MM) requires careful consideration of emerging evidence and expert consensus.
• Individualized treatment strategies are crucial, particularly regarding smoldering MM, induction, ASCT, and maintenance therapy.
• Further research and high-quality trials are needed to clarify optimal MM treatment pathways.