The cryopreservation of PBPC components at standard concentrations and 3.3 (1.8-6.2)-fold cell concentrations has no adverse effect on the function of HPCs after thawing.
We have investigated the value of both conventional and quan- apy are shown in Table 1. Mean age was 56 years (range, Material and methodstitative
Between 20-40% of patients with multiple myeloma (MM) present with renal impairment (RI) at diagnosis (Korbet & Schwartz, 2006; Kastritis et al., 2007) and up to 10% of them require dialysis (Dimopoulos et al., 2010). Prognosis in these patients remains significantly worse when compared with patients with normal renal function (Haynes et al., 2010). Daratumumab is an anti-CD38 IgG kappa human monoclonal antibody that has demonstrated superior clinical benefit across lines of therapy, either as monotherapy or when combined with standard-of-care regimens for the treatment of patients with relapsed and refractory MM (RRMM) (Usmani et al., 2016; van de Donk et al., 2016). However, data of daratumumab in patients with RRMM and dialysis-dependant renal failure are lacking. We report the efficacy and safety results from a retrospective Spanish study of daratumumab in patients with RRMM and end-stage RI requiring dialysis. Methods M.J.C. performed the research and contributed to analyzing of data and paper writing. J.D.L.R. was involved in designing the study, analysis, interpretation and reporting of the data, and wrote/reviewed the paper. All authors performed the research and reviewed the draft manuscript and approved the final version for publication.
Key Points• Increased levels of sCD19 protein in the CSF are associated with CNS disease in DLBCL and BL patients at risk of CNS lymphoma.• Presence of lymphoma cells by FCM and/or increased CSF sCD19 levels are related with a poorer EFS and/or OS in DLBCL and BL patients.Flow cytometry (FCM) is more sensitive than conventional cytology for detection of occult leptomeningeal lymphoma; however, some FCM-negative patients show central nervous system (CNS) recurrence. Here, we evaluated the cerebrospinal fluid (CSF) levels of 13 B-cell-associated markers and their contribution to the diagnosis of CNS lymphoma in 91 diffuse large B-cell lymphomas (DLBCL) and 22 Burkitt lymphomas (BLs). From all markers tested, CD19 was the most informative. Thus, higher soluble CD19 (sCD19) levels were associated with a greater frequency of neurological symptoms in DLBCL and BL and with parenchymal CNS lymphoma in DLBCL; sCD19 emerged as a powerful predictor of event-free and overall survival in DLBCL and BL, particularly when combined with FCM detection of CNS disease. These results support the utility of combined FCM detection of lymphoma cells and assessment of sCD19 levels in CSF, for more accurate identification of
Purpose: Early intervention in smoldering multiple myeloma (SMM) requires optimal risk stratification to avoid under and over-treatment. We hypothesized that replacing bone marrow (BM) plasma cells (PCs) for circulating tumor cells (CTCs), and adding immune biomarkers in peripheral blood (PB) for the identification of patients at risk of progression due to lost immune surveillance, could improve the International Myeloma Working Group 20/2/20 model. Experimental Design: We report the outcomes of 150 SMM patients enrolled in the iMMunocell study, in which serial assessment of tumor and immune cells in PB was performed every 6 months for a period of three years since enrollment. Results:Patients with >0.015% vs ≤0.015% CTCs at baseline had a median time-to-progression of 17 months vs not reached (hazard ratio: 4.9, P<.001). Presence of >20% BM PCs had no prognostic value in a multivariate analysis that included serum free light-chain ratio >20, >2g/dL M-protein, and >0.015% CTCs. The 20/2/20 and 20/2/0.015 models yielded similar risk stratification (C-index of 0.76 and 0.78). The combination of the 20/2/0.015 model with an immune risk-score based on the percentages of SLAN+ and SLAN- non-classical monocytes, CD69+HLADR+ cytotoxic NK cells, and CD4+CXCR3+ stem central memory T cells, allowed patient’ stratification into low, intermediate-low, intermediate-high and high-risk disease with 0%, 20%, 39% and 73% rates of progression at two years. Conclusions: This study showed that CTCs outperform BM PCs for assessing tumor burden. Additional analysis in larger series are needed to define a consensus cutoff of CTCs for minimally invasive stratification of SMM.
Objective The presence of plasmacytomas (Ps) in patients with multiple myeloma (MM) is associated with a poor outcome, both in patients treated conventionally and in patients treated with novel agents. Two types of plasmacytomas have being recognized: paraskeletal plasmacytomas (PPs) and extramedullary plasmacytomas (EMPs), being the incidence of EMPs lower but with worse prognosis. Our aim has been to analyze the efficacy of the pomalidomide‐dexamethasone combination in this patient profile. Method In the present study, the efficacy of pomalidomide and dexamethasone in 21 patients from nine hospitals of Catalonia (Spain), with relapsed or refractory MM and Ps, was analyzed. For this purpose, we describe the evolution of paraprotein in serum and urine and the size of plasmacytomas during treatment with pomalidomide‐dexamethasone. Results While 34% of the patients achieved a paraprotein response, only two patients with PPs (9%) responded (RC and PR). There were no responses among patients with EMPs. The median progression‐free survival from the start of treatment with pomalidomide/dexamethasone was only 1.7 months and the median overall survival of 4.5 months. Conclusion In conclusion, pomalidomide and dexamethasone has limited efficacy in patients with advanced MM and soft‐tissue plasmacytomas.
Cunninghamella spp. are unusual opportunistic pathogens that have been identified with increased frequency in immunocompromised patients. Clinical infection by this fungus is almost always devastating and usually fatal. Infections with this group of organisms have been seen most frequently in patients with hematological malignancy. Here we report the case of a patient with acute leukemia who developed multiorganic failure as a consequence of hematological dissemination by Cunninghamella bertholletiae. The case highlights the mortality associated with this fungal infection in immunocompromised patients, confirms the risk factors associated with non-candida fungal infections and shows a clinical presentation mimicking myocardial infarct and cerebrovascular stroke.
Gaucher disease (GD), one of the most common lysosomal disorders (a global population incidence of 1:50,000), is characterized by beta-glucocerebrosidase deficiency. Some studies have demonstrated bone infiltration in up to 80% of patients, even if asymptomatic. Bone disorder remains the main cause of morbidity in these patients, along with osteoporosis, avascular necrosis, and bone infarcts. Enzyme replacement therapy (ERT) has been shown to improve these symptoms. This cross-sectional study included patients with type 1 Gaucher disease (GD1) selected from the Catalan Study Group on GD. Clinical data were collected and a general laboratory workup was performed. Bone mineral density (BMD) was measured at the lumbar spine and hip using dual-energy X-ray absorptiometry (DXA). Patients with bone infarcts or any other focal lesion in the area of indentation visible on imaging were excluded. Bone Material Strength index (BMSi) was measured by bone impact microindentation using an Osteoprobe instrument. Analysis of covariance (ANCOVA) models were fitted to adjust for age, sex, weight, and height. Sixteen patients with GD1 and 29 age- and sex-matched controls were included. GD1 was associated with significantly lower BMSi (adjusted beta -9.30; 95% CI, -15.18 to -3.42; p = 0.004) and reduced lumbar BMD (adjusted beta -0.14; 95% CI, -0.22 to -0.06; p = 0.002) and total hip BMD (adjusted beta -0.09; 95% CI, -0.15 to -0.03; p = 0.006), compared to GD1-free controls. Chitotriosidase levels were negatively correlated with BMSi (linear R = 51.6%, p = 0.004). Bone tissue mechanical characteristics were deteriorated in patients with GD1. BMSi was correlated with chitotriosidase, the marker of GD activity. Bone disorder requires special consideration in this group of patients, and microindentation could be an appropriate tool for assessing and managing their bone health. © 2017 American Society for Bone and Mineral Research.
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