The introduction of proteasome inhibitors and immunomodulatory drugs has led to significant survival improvements in multiple myeloma (MM). 1,2 Two of the most commonly used regimens for initial MM therapy are bortezomib/dexamethasone plus either cyclophosphamide (VCD) or lenalidomide (VRD). 3 Although not compared head-to-head, phase-2 trials results support the use of either regimen for newly diagnosed MM (NDMM). 4-7 While more efficacious than previous classes, these drugs are associated with substantial cost. [8][9][10] In an era focusing on value of care and efficient resource allocation, use of VCD may lower costs by employing only one novel drug, 'reserving' lenalidomide for subsequent line(s) of therapy. This is particularly relevant for MM where no curative treatments exist, and all available therapies may be used along the care continuum. A paucity of comparative effectiveness research on VCD and VRD as initial therapies for MM exists, thus we designed this retrospective, non-interventional, observational cohort study to compare clinical outcomes, healthcare resource utilization (HRU) and total healthcare costs of VCD and VRD as initial MM treatment in the US clinical setting.Medical charts of a random sample of 176 NDMM patients who initiated VCD or VRD at Mayo Clinic, Rochester, or within the US Oncology Network in January 2008-August 2013 were studied. Patients with ≥2 clinic visits and ≥6 months' follow-up from VCD/VRD initiation were included; those treated in clinical trials were excluded. Study was conducted per the Helsinki Declaration, with Institutional Review Board approvals.Best overall response after 4 cycles and end-of-treatment, progression-free (PFS) and overall (OS) survival (overall, 1-and 2-year), adverse events (AEs), HRU, and total costs (US payer's perspective) were evaluated. With a sample size of N=182, there would be ≥80% power to detect a minimum acceptable best response rate of 60% (ie. 0.70 relative response rate), at one-sided =0.05.Comparisons between VCD/VRD employed adjusted logistic regressions for response; chi-square tests for AEs; and Kaplan-Meier methodology and log-rank tests for overall PFS and OS. Adjusted analyses using Cox proportional models compared 1-and 2-year PFS and OS between cohorts. Adjusted Poisson regression models compared incidence rates of HRU between cohorts.Healthcare costs were compared using unadjusted and adjusted cost difference.Adjusted models controlled for treatment site/network, insurance type, ISS stage III disease, stem cell transplant (SCT), renal failure, cardiac disease, diabetes mellitus and bone fractures.Of 176 patients, 101 (57%) received VCD and 75 (43%) VRD in frontline.Baseline characteristics, including comorbidities, were generally similar between cohorts, except for a higher proportion of African Americans in the VRD cohort (9% versus VCD 2%; P=0.03). A higher proportion of patients with renal failure was seen in the VCD cohort (38% versus VRD 19%; P=0.006), likely reflecting 6 Kumar VCD vs VRD outcomes NDMM_Leukemia_23Jun...
A rare, multisystem, plasma cell neoplasm, POEMS (polyradiculoneuropathy, organomegaly, endocrinopathy, M-spike, skin changes) syndrome is characterized by an abundance of proinflammatory and angiogenic cytokines. Patients with POEMS are known to have a high incidence of engraftment syndrome after autologous stem cell transplantation. We conducted a pilot study assessing levels of 30 different pro- and anti-inflammatory cytokines before and serially after transplantation in 18 patients with plasma cell neoplasms: POEMS syndrome (n = 9), multiple myeloma (n = 4), and amyloidosis (n = 5). We show that POEMS patients have higher pretransplantation levels of IL-4, IL-10, IL-13, IFN-α, and EGF as compared with those with non-POEMS plasma cell neoplasms. Higher pre- and posttransplantation IL-13 levels correlated with delayed neutrophil engraftment in POEMS patients. Low posttransplantation IL-1RA levels correlated with engraftment syndrome in both POEMS and non-POEMS patients. We conclude that differences in the peri-transplantation cytokine milieu may explain the higher transplantation morbidity in patients with POEMS syndrome. Our results need validation in a larger cohort.
Background: Modern clinical practice places time restrictions on the amount of information and processing that can be done in a typical office visit. Meeting patient needs within the healthcare system is challenging, contributing to patient (pt) and clinician dissatisfaction and potential suboptimal outcomes. We developed an electronic point-of-care case management system for cancer patients. The system's goal is to obtain patient-reported quality of life (PROQOL) outcomes and provide clinicians with information regarding QOL issues to address. We tested this system in a prospective randomized study against usual care (UC). The primary objective was to determine if PROQOL system would improve mean QOL over time. Methods: Eligible pts were adults with multiple myeloma (MM) or light amyloidosis (AL), seen at Mayo Clinic. Pts were randomized 2:1 to PROQOL system or usual care. PROQOL system was offered prior to every visit. Pts select from various categories about their single biggest concern, and receive a printed list of actionable resources based on selected concern. Clinicians also receive the PROQOL results to review with pts. Providers and pts randomized to PROQOL completed a "was it worth it survey" (WIWI). An 8 item Linear Analogue Self-Assessment was used to assess QOL, the primary endpoint. Secondary endpoints included distribution of choices of most important concern and their relationship to demographic and clinical variables. The study was powered to detect a 0.5 standard deviation difference in QOL between groups. Results: There were 233 patients enrolled (171 MM, 62 AL) in this study between July 1, 2016 and April 17, 2018. Median age was 65 years (range 31-87), and 59% were male. Median time from diagnosis to study consent was 37 months (PROQOL 36.3 versus UC 39.6). Median follow up for all patients was 15 months (range: 0.7-22 months), and median follow up for PROQOL pts was 16 months and 14 months for UC pts (p=0.027). There were 25 pts that have died since enrollment and 208 alive. No statistical significant difference in QOL between PROQOL and UC (median QOL 7 vs 7, p=0.75). The most prevalent main concerns selected were Physical Health (37%) and Cancer and Diagnosis (27%), the pattern remained even when pts were given the option to pick a second major concern 26% and 20% respectively. No other concern was listed more than 8% at baseline. All category selections and percentages are shown in the Figure. The average time to complete the instrument was 6 minutes. Pts found the PROQOL system was worth their time (75%), they would choose it again (87%) and recommend it to others (82%). Eighty-two percent of clinicians did not think the PROQOL tool negatively impacted their practice and 75% believed they may see improvement in pt wellbeing. Discussion: The PROQOL system was well received by pts and clinicians but did not improve QOL relative to UC in this study. The PROQOL demonstrates that majority of patients strongly desire more information regarding ongoing therapy, clinical trials, prognosis and toxicity. Further investigation is needed to understand whether communication is limited by time or mechanism of information delivery, such as verbal or written, or whether it should be undertaken in a separate allied health care visit. Figure. Figure. Disclosures Gertz: spectrum: Consultancy, Honoraria; Abbvie: Consultancy; Research to Practice: Consultancy; annexon: Consultancy; Medscape: Consultancy; Physicians Education Resource: Consultancy; Amgen: Consultancy; janssen: Consultancy; Alnylam: Honoraria; Teva: Consultancy; Apellis: Consultancy; Ionis: Honoraria; celgene: Consultancy; Prothena: Honoraria. Kumar:Novartis: Research Funding; Celgene: Membership on an entity's Board of Directors or advisory committees, Research Funding; Takeda: Membership on an entity's Board of Directors or advisory committees; Janssen: Membership on an entity's Board of Directors or advisory committees, Research Funding; Janssen: Membership on an entity's Board of Directors or advisory committees, Research Funding; AbbVie: Membership on an entity's Board of Directors or advisory committees, Research Funding; KITE: Membership on an entity's Board of Directors or advisory committees, Research Funding; Roche: Research Funding; Oncopeptides: Membership on an entity's Board of Directors or advisory committees; KITE: Membership on an entity's Board of Directors or advisory committees, Research Funding; Celgene: Membership on an entity's Board of Directors or advisory committees, Research Funding; Merck: Membership on an entity's Board of Directors or advisory committees, Research Funding; AbbVie: Membership on an entity's Board of Directors or advisory committees, Research Funding. Dingli:Alexion Pharmaceuticals, Inc.: Other: Participates in the International PNH Registry (for Mayo Clinic, Rochester) for Alexion Pharmaceuticals, Inc.; Millennium Takeda: Research Funding; Millennium Takeda: Research Funding; Alexion Pharmaceuticals, Inc.: Other: Participates in the International PNH Registry (for Mayo Clinic, Rochester) for Alexion Pharmaceuticals, Inc.. Lacy:Celgene: Research Funding. Dispenzieri:Celgene, Takeda, Prothena, Jannsen, Pfizer, Alnylam, GSK: Research Funding.
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