International harmonization in performing and reporting minimal residual disease assessment in multiple myeloma trials.

Minimal residual disease (MRD) assessment is incorporated in an increasing number of multiple myeloma (MM) clinical trials as a correlative analysis, an endpoint or even as a determinant of subsequent therapy. There is substantial heterogeneity across clinical trials in how MRD is assessed and reported, creating challenges for data interpretation and for the design of subsequent studies. We convened an international panel of MM investigators to harmonize how MRD should be assessed and reported in MM clinical trials. The panel provides consensus on which MM trials should include MRD, the recommended time points for MRD assessment, and expected analytical validation for MRD assays. We subsequently outlined parameters for reporting MRD results implementing the intention-to-treat principle. The panel provides guidance regarding the incorporation of newer peripheral blood-based and imaging-based approaches to detection of residual disease. Recommendations are summarized in 13 consensus statements that should be followed by sponsors, investigators, editors, and reviewers engaged in designing, performing, and interpreting MM trials.

Financial toxicity in insured patients with multiple myeloma: a cross-sectional pilot study.

BACKGROUND: Financial toxicity is increasingly recognised as adversely affecting the quality of life and medication adherence in patients with cancer in the USA. Patients with multiple myeloma might be particularly vulnerable because of high use of novel treatments and extended treatment duration. METHODS: Between Aug 18, 2014, and Jan 7, 2015, we did a cross-sectional survey of individuals receiving at least 3 months of ongoing treatment for multiple myeloma at a tertiary academic medical centre in the USA. The survey was derived from previous reported studies and included the 11-item COST measure (financial toxicity score range 0-44). A paper survey was offered to eligible patients on arrival for routine follow-up visits, and participants were asked to complete the survey before or after their visit to the clinic. Insurance and treatment data were obtained from patients' electronic health records. FINDINGS: Of 111 patients approached for the study, 100 individuals completed the survey. 59 (59%) of 100 patients reported that treatment costs were higher than expected, 70 (71%) of 99 had at least minor financial burden, and 36 (36%) of 100 reported applying for financial assistance. Use of savings to pay for myeloma treatment was common (43 [46%] of 94 patients) and 21 (21%) of 98 individuals borrowed money to pay for medications. COST scores were highly correlated with patient-reported use of strategies to cope with myeloma treatment expenses. On multivariable analysis, younger age (correlation coefficient ß 0·36, 95% CI 0·15 to 0·56, p=0·00092), non-married status (5·6, 1·5 to 9·6, p=0·0074), longer duration since diagnosis (-4·8, -9·3 to -0·2, p=0·042), and lower household income (US$40 000-79 999: 7·8, 2·7 to 12·9, p=0·0031; ≥$80 000: 11·8, 7·1 to 16·4, p<0·0001) were associated with higher financial burden as measured with the COST score. INTERPRETATION: Patient-reported financial toxicity and use of coping mechanisms were common in our insured population with multiple myeloma. Additional attention to rising treatment costs and cost sharing is needed to address the increasing evidence of financial toxicity affecting patients with cancer. FUNDING: University of Pennsylvania Perelman School of Medicine.

The evolving role of plerixafor in hematopoietic progenitor cell mobilization.

The introduction of plerixafor as a peripheral blood stem cell mobilization agent has allowed more patients with multiple myeloma, non-Hodgkin's lymphoma, and Hodgkin's disease to mobilize sufficient hematopoietic progenitor cells (HPCs) to proceed to autologous transplantation. Because of the high cost of plerixafor, it is not routinely used in all patients undergoing HPC mobilization. If cost were not an issue, an argument could be made that plerixafor could be added to every mobilization regimen, but cost is an issue so in an attempt to be more cost-effective, many centers have limited plerixafor use to patients who have failed or who are predicted to fail collection of adequate numbers of cells by other methods. Additionally, plerixafor is now under investigation both for HPC collection of healthy donors for allogeneic stem cell transplantation and as an adjunct therapy (i.e., chemosensitizing agent) for acute leukemias. This article briefly reviews the role of plerixafor in autologous and allogeneic transplantation as well as its emerging role in the treatment of acute leukemias. Emphasis is placed on the choice of appropriate patients for plerixafor use to assure an adequate stem cell yield while maximizing the cost effectiveness of using plerixafor. The role of prophylactic collections and future areas of research are also presented.

Plerixafor mobilization leads to a lower ratio of CD34+ cells to total nucleated cells which results in greater storage costs.

BACKGROUND: Plerixafor (Mozobil, AMD3100) with granulocyte-colony stimulating factor (G-CSF) mobilizes more CD34+ cells/kg compared to G-CSF alone. Given that plerixafor enhances mobilization of multiple white blood cell lineages, we determined if more storage space is required for products collected from patients mobilized with plerixafor. METHODS: A review of the medical records of 15 patients mobilized with chemotherapy and G-CSF (control) and 14 patients mobilized with plerixafor plus G-CSF (plerixafor) was performed. Data on demographics, baseline characteristics, CD34+ cells/kg, total nucleated cells, total mononuclear cells, total apheresis sessions, and total bags for storage were collected. Mean values were determined and compared using Student's t-test. RESULTS: We found that the proportion of CD34+ cells among total nucleated cells was less in the plerixafor group compared to the control group (P = 0.0427). More nucleated cells (10.7 x 10(10) vs. 7.1 x 10(10), P =0.0452) and mononuclear cells (9.7 x 10(10) vs. 5.9 x 10(10), P = 0.0059) were mobilized with plerixafor plus G-CSF. However, there was no significant difference in CD34+ cells/kg, total CD34+ cells or the proportion of mononuclear cells among total nucleated cells between the two groups. More storage bags were required for the plerixafor group compared to the control group (15 vs. 9, P = 0.0299). CONCLUSION: Mobilization with plerixafor plus G-CSF resulted in a smaller proportion of CD34+ cells collected and a greater number of storage bags. An increase in the number of bags required for stem cell storage may be logistically problematic and will also lead to increased costs for storage of stem cells.