A Consistent Lack of Consistency: Definitions, Evidentiary Expectations and Potential Use of Meaningful Change Data in Clinical Outcome Assessments Across Stakeholders. Results from a DIA Working Group Literature Review and Survey.

Background: Clinical outcome assessments (COAs) measure how patients feel or function and can be used to understand which patients experience benefits of treatment and which do not. Interpretation of COA data is influenced by how meaningful change is defined. We aimed to compare how different stakeholders define, assess, and use meaningful change for decisions that impact patients.

Overall and complete response rates as potential surrogates for overall survival in relapsed/refractory multiple myeloma.

The correlation between response and survival has not been well-studied in relapsed or refractory multiple myeloma (RRMM). A systematic literature review of Medline, Embase and Cochrane databases (2010-06/2020) and relevant congresses (2018-2020) was performed to identify randomized clinical trials in RRMM reporting median overall survival (mOS), progression-free survival and response end points. The relationship between mOS and response end points was analyzed using Pearson's product-moment correlation.

Characterizing the Patient Journey in Multiple Myeloma: Qualitative Review.

Background: The patient experience of multiple myeloma (MM) is multifaceted and varies substantially between individuals. Current published information on the patient perspective and treatment of MM is limited, making it difficult to gain insights into patient needs regarding the condition.

Objective: In this review, a combined research method approach (ie, the review of published literature and social media posts) was undertaken to provide insight into patients' perspectives on the burden and treatment of MM, the impact of the COVID-19 pandemic, and the impact of MM on caregivers of patients with MM.

Response rates and minimal residual disease outcomes as potential surrogates for progression-free survival in newly diagnosed multiple myeloma.

Progression-free survival (PFS) is a common primary endpoint in newly diagnosed multiple myeloma (NDMM). Patients with NDMM typically have longer PFS and are more likely to achieve minimal residual disease (MRD) or complete response (CR) compared to patients with relapsed or refractory multiple myeloma. Response-based surrogate endpoints may hold value given the longer follow-up time required to evaluate PFS in NDMM.

Efficacy of non-intensive therapies approved for relapsed/refractory acute myeloid leukemia: a systematic literature review.

relapsed and/or refractory acute myeloid leukemia (rrAML) has limited treatment options for patients not eligible ('unfit') to receive intensive chemotherapy-based interventions. The authors aimed to summarize outcomes for licensed therapies in this setting. A systematic literature review identified licensed therapies in this setting.