cooperative study group Achieving complete remission (CR) in multiple myeloma (MM) translates into extended survival, but two subgroups of patients fall outside this paradigm: cases with unsustained CR, and patients that do not achieve CR but return into a monoclonal gammopathy of undetermined significance (MGUS)-like status with long-term survival. Here, we describe a novel automated flow cytometric classification focused on the analysis of the plasma-cell compartment to identify among newly diagnosed symptomatic MM patients (N ¼ 698) cases with a baseline MGUS-like profile, by comparing them to MGUS (N ¼ 497) patients and validating the classification model in 114 smoldering MM patients. Overall, 59 symptomatic MM patients (8%) showed an MGUS-like profile. Despite achieving similar CR rates after high-dose therapy/autologous stem cell transplantation vs other MM patients, MGUS-like cases had unprecedented longer time-to-progression (TTP) and overall survival (OS; B60% at 10 years; Po0.001). Importantly, MGUS-like MM patients failing to achieve CR showed similar TTP (P ¼ 0.81) and OS (P ¼ 0.24) vs cases attaining CR. This automated classification also identified MGUS patients with shorter TTP (P ¼ 0.001, hazard ratio: 5.53) and ultra-high-risk smoldering MM (median TTP, 15 months). In summary, we have developed a biomarker that identifies a subset of symptomatic MM patients with an occult MGUS-like signature and an excellent outcome, independently of the depth of response.

Elotuzumab is an immunostimulatory, humanized immunoglobulin G1 monoclonal antibody that selectively targets and kills signalling lymphocytic activation molecule family member 7-expressing myeloma cells. We evaluated the safety and tolerability of elotuzumab 10 mg/kg combined with thalidomide 50-200 mg and dexamethasone 40 mg (with/without cyclophosphamide 50 mg) in patients with relapsed/refractory multiple myeloma (RRMM). The primary endpoint was the proportion of grade ≥3 non-haematological adverse events (AEs); other endpoints included the number of dose reductions/discontinuations and efficacy. Forty patients were treated, who had a median of three previous therapies, including bortezomib (98%) and lenalidomide (73%). Grade ≥3 non-haematological AEs were reported in 63% of patients, most commonly asthenia (35%) and peripheral oedema (25%). Six (15%) patients had an infusion reaction. Twenty-six (65%) patients had ≥1 dose reduction/discontinuation due to an AE, none related to elotuzumab. Overall response rate was 38%; median progression-free survival was 3·9 months. Median overall survival was 16·3 months and the 1-year survival rate was 63%. Minimal incremental toxicity was observed with addition of elotuzumab to thalidomide/dexamethasone with or without cyclophosphamide, and efficacy data suggest clinical benefit in a highly pre-treated population. Elotuzumab combined with thalidomide may provide an additional treatment option for patients with RRMM.

We report the characteristics of relapse, treatment response, and outcomes of 145 elderly patients with multiple myeloma in first relapse after front-line treatment with VMP or VTP. Reappearance of CRAB symptoms (113 patients) and more aggressive forms of disease (32 patients) were the most common patterns of relapse. After second-line therapy, 75 (51.7%) patients achieved at partial response and 16 (11%) complete response (CR). Overall survival was longer among patients receiving VMP as front-line induction (21.4 vs. 14.4 months, P=0.037), in patients achieving CR (28.3 vs. 14.8 months; P=0.04), and in patients without aggressive relapse (28.6 vs. 7.6 months; P=0.0007).

After an RIC-allo, FN and early SBI occurred mostly in patients with severe mucositis and early-onset neutropenia, while postengraftment high-dose steroid therapy for acute GVHD was the major RF.

Patient and physician agreement on the most significant symptoms is associated with treatment outcomes and satisfaction with care. Thus, we sought to assess patient and physician agreement on patient-reported quality of life (QoL), and whether patient-related variables predict disagreement. In this cross-sectional, multisite study, patients and physicians completed the FACT-BMT at day 90. Agreement was analyzed with the intraclass coefficient correlation (ICC). Rates of underestimation and overestimation were calculated. Logistic regression models identified predictors of disagreement. We analyzed 96 pairs of questionnaires completed by 96 patients and 11 physicians. The patients' median age was 54 years, 52% were men, and 52% had undergone allogeneic hematopoietic cell transplantation (HCT). The physicians' median age was 42, 64% were men, and they had worked in the HCT field for an average of 12 years. Agreement on QoL was moderate (ICC = .436). Exploratory analyses revealed poor agreement for emotional (ICC = .092) and social (ICC = .270) well-being and moderate agreement for physical (ICC = .457), functional (ICC = .451), and BMT concerns (ICC = .445). Patients' well-being was underestimated by physicians in 41% to 59% of the categories of well-being parameters, and overestimated in 10% to 24%. Patient's anxiety predicted less disagreement in all scales except in social well-being, for which nonsignificant associations were observed. Patient-related variables explained 12% to 19% of the variance in disagreement across well-being scales. Patient and physician agreement on QoL was suboptimal, particularly in emotional and social well-being. The implementation of patient-reported outcomes in the daily care of HCT recipients may contribute to improving patient-centered care.