Bourbon et al describe a series of 19 patients from France with VEXAS syndrome, including a novel genetic aberration in UBA1, and the outcomes of treatment for those with and those without myelodysplasia.

on behalf of the Lyon BK virus Study group 5 BK virus (BKV) reactivation has been increasingly associated with the occurrence of late-onset hemorrhagic cystitis (HC) after allogeneic hematopoietic SCT (allo-HSCT) resulting in morbidity and sometimes mortality. We investigated the incidence, risk factors and outcome of BKV-HC in 323 consecutive adult patients undergoing allo-HSCT over a 5-year period. BK viremia values for HC staging were evaluated, as well as the medico-economic impact of the complication. Forty-three patients developed BKV-HC. In univariate analysis, young age (P ¼ 0.028), unrelated donor (P ¼ 0.0178), stem cell source (P ¼ 0.0001), HLA mismatching (P ¼ 0.0022) and BU in conditioning regimen (P ¼ 0.01) were associated with a higher risk of developing BKV-HC. In multivariate analysis, patients receiving cord blood units (CBUs) (P ¼ 0.0005) and peripheral blood stem cells (P ¼ 0.011) represented high-risk subgroups for developing BKV-HC. BK viremia was directly correlated to HC severity (P ¼ 0.011) with a 3 to 6-log peak being likely associated with grades 3 or 4 HC. No correlation was found between BKV-HC and acute graft versus host disease or mortality rate. Patients with BKV-HC required a significantly longer duration of hospitalization (Po0.0001), more RBC (P ¼ 0.0003) and platelet transfusions (Po0.0001). Over the 5-year study period, the financial cost of the complication was evaluated at h2 376 076 ($3 088 899). Strategies to prevent the occurrence of late-onset BKV-HC after allo-HSCT are urgently needed, especially in CBU and peripheral blood stem cell recipients. BK viremia correlates with the severity of the disease. Prospective studies are required to test prophylactic approaches.

Intravascular hemolysis in Paroxysmal nocturnal hemoglobinuria (PNH) can effectively be controlled with eculizumab, a humanized monoclonal antibody that binds complement protein C5. We report here a retrospective comparison study between 123 patients treated with eculizumab in the recent period (>2005) and 191 historical controls (from the French registry). Overall survival (OS) at 6 years was 92% (95%CI, 87 to 98) in the eculizumab cohort versus 80% (95%CI 70 to 91) in historical controls diagnosed after 1985 (HR 0.38 [0.15 to 0.94], P 5 0.037). There were significantly fewer thrombotic events (TEs) in the group of patients treated with eculizumab (4% [1-10]) as compared to the historical cohort (27% [20][21][22][23][24][25][26][27][28][29][30][31][32][33][34]). However, we found that TEs may still occur after the initiation of eculizumab treatment and that previous TEs still have a negative impact on survival. Evolutions to myelodysplastic syndrome or acute leukemia were similar in both cohorts. There was less evolution to aplastic anemia in the treatment group. In multivariate analysis, absence of a previous TE and treatment with eculizumab were associated with a better OS. Treatment with eculizumab improves overall survival in classic PNH patients without increasing the risk of clonal evolution.

The authors' full names, academic degrees, and affiliations are listed in the Appendix. Dr. Peffault de Latour can be contacted at regis .