Patient-Reported Outcomes in Patients with Multiple Myeloma

Chicago—A broad perspective of disease burden and impact of treatment may be gained utilizing patient-reported outcomes (PROs) such as health-related quality of life (HRQoL). For patients with multiple myeloma (MM) and other hematologic malignancies, HRQoL has been measured recently via prospective observational studies and randomized clinical trials.

Connect^® MM is a registry created to provide insights into disease burden and treatment impact in patients with symptomatic newly diagnosed MM (NDMM); it is an ongoing, multicenter, prospective, observational registry of patients with NDMM (diagnosis within 2 months of enrollment). Community, academic, and military/veterans institutions in the United States created the registry in September 2009. Currently, the registry includes 1494 patients enrolled at 228 centers.

Utilizing data from Connect MM, researchers recently conducted a study to assess the change in PROs of patients with NDMM who met CRAB (calcium elevation, renal insufficiency, anemia, and lytic bone lesions) criteria between baseline and 1 year, relative to their International Staging System (ISS) stage and Eastern Cooperative Oncology Group Performance Status (ECOG PS) score. Results were presented during a poster session at the ASCO meeting. The poster was titled Changes in Patient-Reported Outcomes in Patients Diagnosed with and Treated for Multiple Myeloma in the Connect^® MM Registry.

Baseline demographics, ECOG PS score, and ISS stage were provided by clinicians. PROs were collected at baseline and at 1 year, using 3 validated instruments: (1) Brief Pain Inventory (BPI), measuring the existence and intensity of pain on a scale from 0 (no pain) to 10 (worst pain); (2) EQ-5D, assessing general HRQoL, overall and within 5 domain-specific areas (1. mobility, 2. self-care, 3. usual activities, 4. pain/discomfort, and 5. anxiety/depression) measured on a scale of 1 (no problems) to 3 (extreme problems); and (3) Functional Assessment of Cancer Therapy-Multiple Myeloma (FACT-MM), evaluating cancer-specific (FACT-G) and MM-specific (FACT-MM) HRQoL (higher scores indicate better HRQoL). Domains include physical, social/family, emotional, and functional well-being.

The study cohort included 636 patients enrolled at 189 centers. Mean age of the eligible patients was 66 years and 58% were male; 81% were treated in community centers, 17% in academic institutions, and 2% in military/veterans centers.

Statistically significant improvements in overall HRQoL were observed across all ISS stages, with no significant difference between stages. HRQoL functional ability improved in 4 of 5 FACT domains (all except social/family; all others P<.001), and in 4 of 5 EQ-5D domains (all except pain/discomfort; all others P=.01 to P<.0001).

Likewise, improvements in FACT-MM and FACT-G scores were observed among cohorts with ECOG PS scores 1 to 3 (P=.02 to P=.005). Patients with poorer ECOG PS scores tended to have greater improvement in EQ-5D domains of mobility, self-care, and usual activities.

There was significant improvement in the average pain score over 1 year: mean change from baseline to 1 year, -0.4 (P=.0005). However, there were no statistically significant differences observed by ISS stage or ECOG PS score.

The authors concluded by noting, “Connect MM data analysis showed that the overall HRQoL of patients with NDMM meeting CRAB criteria improved between baseline and 1 year, with a consistent benefit observed across patients with different ISS states and ECOG PS scores.”

They added, “Additional analyses are planned to examine which disease- and treatment-related factors are associated with these HRQoL improvements. A better understanding of these factors may allow clinicians and patients to make more informed decisions regarding disease management.”

Support for this analysis was provided by Celgene Corporation.