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Antithymocyte globulin (ATG) is the drug of choice for immunosuppressive therapy (IST) in patients with severe aplastic anemia (SAA) ineligible for allogeneic stem cell transplantation. Recently, rabbit ATG with cyclosporine A has been used as a first-line IST regimen in patients with SAA because of unavailability of horse ATG. We retrospectively analyzed adult SAA patients who were treated with horse ATG ( n = 46) or rabbit ATG ( n = 53) between Feb 2001 and May 2010 to compare hematologic response and survival. Overall response rates at 3, 6, 12, and 18 months were similar in both the horse and rabbit ATG groups: 28.3 versus 35.8 % ( P = 0.421), 39.1 versus 45.3 % ( P = 0.537), 45.7 versus 49.1 % ( P = 0.735), and 47.8 versus 50.9 % ( P = 0.757), respectively. The complete response (CR) rate at 6 months in the horse ATG was significantly superior in comparison with the rabbit ATG (13.0 versus 1.9 %, P = 0.031). But CR rates became similar in both groups after 6 months: 17.4 versus 11.3 % ( P = 0.387) at 12 months and 21.7 versus 22.6 % ( P = 0.914) at 18 months. Lymphocyte depletion after ATG was more profound and protracted in the rabbit ATG group compared to the horse ATG group. Overall survival ( P = 0.460) and failure-free survival ( P = 0.911) were not significantly different between the two groups. Our retrospective study demonstrated that the efficacy of first-line IST with rabbit ATG is similar to that with horse ATG. However, the time from treatment to CR was longer with rabbit ATG than with horse ATG, partly due to more profound and protracted lymphocyte depletion. [ABSTRACT FROM AUTHOR]

The aim of this study was to evaluate the impact of the response to induction therapy on the long-term prognosis of multiple myeloma (MM) after autologous stem cell transplantation (ASCT) in the era of novel agents (NAs). A total of 171 patients who were newly diagnosed with MM and underwent early ASCT were analyzed. One hundred ten had a NA-based induction therapy, and 61 patients had a non-NA-based induction therapy. After a median follow-up of 45.4 months, the 4-year overall survival (OS) and progression-free survival (PFS) from transplantation were 60.5 and 25.5 %, respectively, for the NA-based induction group and 54.6 and 15.6 %, respectively, for the non-NA-based induction group. Multivariate analyses revealed that the patients who had NA-based induction had a significantly shorter OS ( P < 0.001) and PFS ( P < 0.001) when at least a partial response (PR) was not achieved. In patients who did not receive NAs before ASCT, lack of at least a PR to induction therapy was not associated with a survival disadvantage. These findings suggest that, unlike pretransplantation induction before NAs, patients who do not respond to induction treatment using NAs may not derive a benefit from ASCT. The relevance of induction failure differs for corticosteroid- and NA-based induction. [ABSTRACT FROM AUTHOR]

The prognostic impact of extramedullary plasmacytomas (EMPs) on newly diagnosed symptomatic multiple myeloma (MM) was evaluated in the context of treatment approach including autologous stem cell transplantation (ASCT) and chemotherapy alone. A total of 275 consecutive patients with newly diagnosed MM were included, and 54 patients (19.6 %) had EMPs at diagnosis. Patients with initial EMPs were more likely to have myeloma bone disease but favorable laboratory parameters in hemoglobin and β2-microglobulin. Patients were treated with different schemas based on transplant eligibility (154 in ASCT-eligible vs. 121 in ASCT-ineligible). After a median follow-up of 24.6 months (range, 0.2-56.3 months) in survivors, patients with initial EMPs had significantly worse progression-free survival (PFS) ( P = 0.035) and overall survival (OS) ( P = 0.006) compared to those without initial EMPs. In the multivariate analyses, the presence of initial EMPs was an independent prognostic factor for PFS (relative risk (RR) of 2.24, P = 0.024) and OS (RR of 2.47, P = 0.027) in the transplant-ineligible patients, whereas it did not significantly influence PFS ( P = 0.341) or OS ( P = 0.499) in the transplant-eligible patients. However, the adverse impact of EMPs observed in transplant-ineligible patients was attenuated among the patients treated with bortezomib. These data suggest that ASCT can overcome the negative impact of EMPs and highlight the potential efficacy of bortezomib on EMPs in the non-transplant setting. [ABSTRACT FROM AUTHOR]

Cytomegalovirus gastrointestinal (CMV-GI) disease in GI graft-versus-host disease (GI-GVHD) has not been properly evaluated in the era of preemptive therapy for CMV infection. We investigated 103 patients with GI-GVHD who underwent endoscopic biopsies with immunohistochemical staining for CMV. All recipients and/or donors were seropositive for CMV and monitored with a strategy of preemptive therapy based on real-time quantitative polymerase chain reaction. Twenty-six patients (25 %) developed CMV-GI disease, especially in HLA-mismatched transplants ( P = 0.023) and with initial gut involvement of GVHD ( P = 0.009). The CMV-GI diseases were diagnosed at follow-up endoscopies ( n = 10, 39 %), comprising 19 % of 52 patients who underwent follow-up endoscopies, as well as initial endoscopies ( n = 16, 61 %), comprising 16 % of all GI-GVHD patients. In seven cases, either at initial ( n = 5) or follow-up endoscopies ( n = 2), CMV-GI disease was diagnosed in the absence of histopathologic evidence for GI-GVHD. Notably, only 11 patients (42 %) had prior CMV DNAemia before the diagnosis of CMV-GI disease, while 12 (46 %) and three (12 %) had concurrent and no CMV DNAemia, respectively. Sixty-five percent of CMV-GI disease was resolved by additional antiviral therapies, but CMV-GI disease ( P = 0.032) as well as severity of GVHD ( P = 0.001) negatively affected GVHD-specific survival. In conclusion, our data demonstrate that CMV-GI disease was a cause of initial or persistent GI manifestations after the initiation of therapy in a considerable proportion of GI-GVHD. These suggest the necessity of novel strategies to reduce CMV-GI disease as well as an effort to confirm CMV with repeated endoscopies. [ABSTRACT FROM AUTHOR]