Insurance-based disparities impact survival outcomes in Waldenström macroglobulinemia within the United States.

Considerable healthcare resource utilization and financial burden have been associated with the treatment of WM; however, the impact of health insurance status on outcomes has not been previously reported. We conducted a National Cancer Database analysis of newly diagnosed cases of active WM between 2004 and 2017 to evaluate the impact of insurance status on outcomes. For patients <65 years old (n = 1249, male sex: 62.4%, median age: 58 years), significant insurance-based survival differences were observed on multivariable analysis; patients who were uninsured [n = 63; HR 3.11 (95%CI, 1.77-5.45), p < 0.001], on Medicaid [n = 87; HR 1.88 (95% CI, 1.01-3.48), p = 0.045], or on Medicare [n = 122; HR 2.78 (95%CI, 1.76-4.38), p < 0.001], had inferior survival compared to patients with private insurance (n = 977; reference). In patients ≥65 years, no insurance-based survival differences were found (p = 0.10). Overall, significant insurance-based outcome disparities exist in WM. Further work is desperately needed to systematically uncover and address these disparities.

The Impact of Socioeconomic Risk Factors on the Survival Outcomes of Patients With Newly Diagnosed Multiple Myeloma: A Cross-analysis of a Population-based Registry and a Tertiary Care Center.

BACKGROUND: Multiple myeloma (MM) is a heterogeneous clonal plasma cell disorder leading to differences in clinical outcomes such as overall survival (OS) among patients. We hypothesized that with expensive, novel therapeutic agents and paradigm shifts to maintain continuous therapy and improvement in OS, patients with MM are subject to the pressures of financial toxicity and the need for social support, which may be of prognostic importance. MATERIALS AND METHODS: In this study, we examined the records of 122,458 patients from the National Cancer Database (NCDB) to determine the significance of socioeconomic factors such as estimated annual household income and education level, which were based on the patient's ZIP Code and the United States Census Bureau's 5-year report from 2008 to 2012. These socioeconomic factors, in addition to marital status, were then assessed individually and as a cumulative socioeconomic score for prognostic significance in a cohort of 2543 patients treated at a tertiary care center utilizing known biologic risk factors, such as cytogenetic risk, International Staging System classification, and serum lactate dehydrogenase levels. RESULTS: Only marital status and estimated annual household income at diagnosis negatively impacted OS in a univariate analysis, but not in the context of a multivariable analysis incorporating known biologic risk factors. CONCLUSION: Future analyses in other academic and non-academic centers located in urban and rural regions are required to understand the socioeconomic drivers of OS disparity among patients with MM observed nationally.

Disease monitoring with quantitative serum IgA levels provides a more reliable response assessment in multiple myeloma patients.

Unlike IgG monoclonal proteins (MCPs), IgA MCP quantification is unreliable due to beta-migration of IgA MCPs on serum protein electrophoresis (SPEP). The utility of nephelometric quantitative IgA (qIgA) to monitor IgA multiple myeloma (MM) is unclear. We retrospectively studied disease response kinetics using qIgA versus MCPs by SPEP, and developed and validated novel qIgA disease assessment criteria in 491 IgA MM patients. The SPEP MCP nadir occurred a median of 41 (IQR 0-102) days before the qIgA. The median time to achieve a partial response (PR) was shorter using standard IMWG versus qIgA response criteria (32 vs 58 days, p < 0.001). Stratification by qIgA criteria, unlike IMWG criteria, led to clear separation of the progression-free survival curves of patients achieving a PR or very good PR. There was a consistent trend toward earlier detection of disease progression using qIgA versus IMWG progression criteria. In conclusion, monitoring IgA MM using MCP-based IMWG criteria may be falsely reassuring, given that MCP levels on SPEP decrease faster than qIgA levels. The qIgA response criteria more accurately stratify patients based on the progression risk and may detect disease progression earlier, which may lead to more consistent measurement of trial endpoints and improved patient outcomes.

A validated composite organ and hematologic response model for early assessment of treatment outcomes in light chain amyloidosis.

Newly diagnosed AL amyloidosis patients were evaluated to develop a model for early assessment of treatment benefit at 6 months, integrating both hematologic (HR) and organ response (OR) assessment (testing cohort, Mayo: n = 473; validation cohort, Pavia: n = 575). Multiple OR were assessed as follows: All OR (AOR): response in all organs, mixed OR (MOR): response in some organs, no OR (NOR)]. AOR rates at 6 months improved with deepening HR; complete response (CR; 38%, 35%), very good partial response (VGPR; 30%, 26%), and partial response (PR; 16%, 21%), respectively. A composite HR/OR (CHOR) model was developed using incremental scoring based on hazard ratios with scores of 0-3 for HR (0-CR, 1-VGPR, 2-PR, 3-no response) and 0-2 for OR (0-AOR, 1-MOR, 2-NOR). Patients could be divided into two distinct CHOR groups (scores 0-3 and 4-5), with median OS in group 1 and group 2: Not reached vs. 34 months, p < 0.001 [Mayo] and 87 vs. 23 months, p < 0.001 [Pavia]. In conclusion, we developed a model that can assess multiple organs concurrently, and integrate both HR and OR assessments to determine early clinical benefit with treatment, which may be used as a surrogate end-point in trials and to compare outcomes with different therapies.

Assessment of the Need for a Hematology-Specific Fellowship Curriculum for Advanced Practice Providers Using a Needs-Based Survey.

PURPOSE: Subspecialty training programs rarely are available for advanced practice providers (APPs). New curricula are needed to prepare APPs with the skills and knowledge required to deliver high-quality care in hematology and blood and marrow transplantation (BMT). METHODS: A Web-based needs assessment survey was distributed to APPs currently working in hematology and BMT at three Mayo Clinic sites (Rochester, MN; Scottsdale, AZ; and Jacksonville, FL). The survey assessed participants' perceptions of readiness to practice in hematology after completion of their APP education and identified APP learning needs. RESULTS: Of 68 APPs, 49 (72%; 34 nurse practitioners, 15 physician assistants) completed the survey. Twenty-eight APPs (57%) were new graduates, and 17 (35%) had no prior experience in hematology/BMT. All APPs held a master's degree or higher (doctorate, 31%). Thirty-nine (80%) reported that less than 5% of their APP school curriculum was hematology focused. More than 90% felt unprepared to practice in hematology or BMT after their APP education and believed that hematology-specific training could improve their competency as providers and positively affect job satisfaction. APPs reported that they would like more formal training in the following areas of clinical focus: malignant and benign hematologic disorders, hematopathology, palliative care, transfusion medicine, infectious disease, and hematology-related pharmacology. They also preferred the following learning strategies: active learning from patient care, case-based teaching, and experience during hospital rounds. CONCLUSION: This needs-based assessment project confirmed the necessity to develop a hematology-specific fellowship for APPs and helped to optimize the curriculum.

Rapid assessment of hyperdiploidy in plasma cell disorders using a novel multi-parametric flow cytometry method.

Trisomies of odd numbered chromosomes are seen in nearly half of patients with multiple myeloma (MM) and typically correlate with a hyperdiploid state and better overall survival (OS). We compared DNA ploidy of monoclonal plasma cells (as a surrogate for the presence of trisomies) assessed simultaneously by PCPRO (plasma cell proliferative index), a novel method that estimates DNA index by multi-parametric flow cytometry to fluorescence in situ hybridization (FISH) in 1703 patients with plasma cell disorders. The distribution of ploidy was hyperdiploid: 759 (45%), diploid 765 (45%), hypodiploid: 71 (4%), tetraploid/near-tetraploid: 108 (6%). FISH identified trisomies in 82% (621/756) of patients with hyperdiploidy by PCPRO and no trisomy by FISH was observed in 88% (730/834) of patients without hyperdiploidy. 95% (795/834) of patients without hyperdiploidy on PCPRO had one or less trisomy by FISH. Sensitivity and specificity of PCPRO for detecting hyperdiploidy was 86% (621/725) and 84% (730/865), respectively. Sensitivity increased to 94% (579/618) for patients with more than one trisomy. Newly diagnosed MM patients with hyperdiploidy on PCPRO (147/275) had better OS compared to nonhyperdiploid patients (median not reached vs 59 months, P = 0.008) and better progression free survival (median: 33 vs 23 months, P = 0.03). Within the hyperdiploidy group, patients with high-hyperdiploidy (DNA index: 1.19-1.50) versus those with low-hyperdiploidy (DNA index: 1.05-1.18) had superior OS (3 year OS of 88% vs 68% P = 0.03). Ploidy assessment by flow cytometry can provide rapid, valuable prognostic information and also reduces the number of copy number FISH probes required and hence the cost of FISH.

A randomized phase 3 trial of thalidomide and prednisone as maintenance therapy after ASCT in patients with MM with a quality-of-life assessment: the National Cancer Institute of Canada Clinicals Trials Group Myeloma 10 Trial.

We conducted a randomized, controlled trial comparing thalidomide-prednisone as maintenance therapy with observation in 332 patients who had undergone autologous stem cell transplantation with melphalan 200 mg/m2. The primary end point was overall survival (OS); secondary end points were myeloma-specific progression-free survival,progression-free survival, incidence of venous thromboembolism, and health-related quality of life (HRQoL). With a median follow-up of 4.1 years, no differences in OS between thalidomide-prednisone and observation were detected (respective 4-year estimates of 68% vs 60%, respectively; hazard ratio = 0.77; P = .18); thalidomide-prednisone was associated with superior myeloma-specific progression-free survival and progression-free survival (for both outcomes, the 4-year estimates were 32% vs 14%; hazard ratio = 0.56; P < .0001) and more frequent venous thromboembolism (7.3% vs none; P = .0004). Median survival after first disease recurrence was 27.7 months with thalidomide-prednisone and 34.1 months in the observation group. Nine second malignancies were observed with thalidomide-prednisone versus 6 in the observation group. Those allocated to thalidomide-prednisone reported worse HRQoL with respect to cognitive function, dyspnea, constipation, thirst, leg swelling, numbness, dry mouth, and balance problems. We conclude that maintenance therapy with thalidomide-prednisone after autologous stem cell transplantation improves the duration of disease control, but is associated with worsening of patient-reported HRQoL and no detectable OS benefit.

Cost-effectiveness analysis of a risk-adapted algorithm of plerixafor use for autologous peripheral blood stem cell mobilization.

Historically, up to 30% of patients were unable to collect adequate numbers of peripheral blood stem cells (PBSCs) for autologous stem cell transplantation (ASCT). Plerixafor in combination with granulocyte colony-stimulating factor (G-CSF) has shown superior results in mobilizing peripheral blood (PB) CD34+ cells in comparison to G-CSF alone, but its high cost limits general use. We developed and evaluated risk-adapted algorithms for optimal utilization of plerixafor. In plerixafor-1, PBSC mobilization was commenced with G-CSF alone, and if PB CD34 on day 4 or day 5 was <10/µL, plerixafor was administered in the evening, and apheresis commenced the next day. In addition, if on any day, the daily yield was <0.5 × 10(6) CD34/kg, plerixafor was added. Subsequently, the algorithm was revised (plerixafor-2) with lower thresholds. If day-4 PB CD34 <10/µL for single or <20/µL for multiple transplantations, or day-1 yield was <1.5 × 10(6) CD34/kg, or any subsequent daily yield was <0.5 × 10(6) CD34/kg, plerixafor was added. Three time periods were analyzed for results and associated costs: January to December 2008 (baseline cohort; 319 mobilization attempts in 278 patients); February to November 2009 (plerixafor-1; 221 mobilization attempts in 216 patients); and December 2009 to June 2010 (plerixafor-2; 100 mobilization attempts in 98 patients). Plerixafor-2 shows a significant improvement in PB CD34 collection, increased number of patients reaching minimum and optimal goals, fewer days of apheresis, and fewer days of mobilization/collection, albeit at increased costs. In conclusion, although the earlier identification of ineffective PBSC mobilization and initiation of plerixafor (plerixafor-2) increases the per-patient costs of PBSC mobilization, failure rates, days of apheresis, and total days of mobilization/collection are lower.

The well-being and personal wellness promotion strategies of medical oncologists in the North Central Cancer Treatment Group.

PURPOSE: The well-being of oncologists is important to the well-being of their patients. While much is known about oncologist distress, little is known about oncologist well-being. We set out to evaluate oncologist well-being and the personal wellness promotion strategies used by oncologists. PATIENTS AND METHODS: We performed a cross-sectional survey of medical oncologists in the North Central Cancer Treatment Group using a validated instrument to measure quality of life. Study-specific questions explored stressors, wellness promotion strategies and career satisfaction. RESULTS: Of 241 responding oncologists (response rate 61%), 121 (50%) reported high overall well-being. Being age 50 or younger (57 vs. 41%; p = 0.01), male (53 vs. 31%; p = 0.01) and working 60 h or less per week (50 vs. 33%; p = 0.005) were associated with increased overall well-being on bivariate analysis. Ratings of the importance of a number of personal wellness promotion strategies differed for oncologists with high well-being compared with those without high well-being. Developing an approach/philosophy to dealing with death and end-of-life care, using recreation/hobbies/exercise, taking a positive outlook and incorporating a philosophy of balance between personal and professional life were all rated as substantially more important wellness strategies by oncologists with high well-being (p values <0.001). Oncologists with high overall well-being also reported greater career satisfaction. CONCLUSION: Half of medical oncologists experience high overall well-being. Use of specific personal wellness promotion strategies appears to be associated with oncologist well-being. Further investigations of the prevalence, promotion, causes, inequities and clinical impact of physician well-being are needed. .