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• Extended half-life (EHL) factors increased bleeding protection in clinical trials. • Pharmacokinetics & clinical variables were compared in 2 periods after EHL switch. • EHL achieved improvements in pharmacokinetics, bleeding rates & treatment burden. • Benefits were reported with different EHL products and in all the age subgroups. [ABSTRACT FROM AUTHOR]

Acute myeloid leukemia (AML) with intermediate risk cytogenetics (IRcyto) comprises a variety of biological entities with distinct mutational landscapes that translate into differential risks of relapse and prognosis. Optimal postremission therapy choice in this heterogeneous patient population is currently unsettled. In the current study, we compared outcomes in IRcyto AML recipients of autologous (autoSCT) (n = 312) or allogeneic stem cell transplantation (alloSCT) (n = 279) in first complete remission (CR1). Molecular risk was defined based on CEBPA, NPM1, and FLT3-ITD mutational status, per European LeukemiaNet 2017 criteria. Five-year overall survival (OS) in patients with favorable molecular risk (FRmol) was 62% (95% confidence interval [CI], 50-72) after autoSCT and 66% (95% CI, 41-83) after matched sibling donor (MSD) alloSCT (P = .68). For patients of intermediate molecular risk (IRmol), MSD alloSCT was associated with lower cumulative incidence of relapse (P < .001), as well as with increased nonrelapse mortality (P = .01), as compared to autoSCT. The 5-year OS was 47% (95% CI, 34-58) after autoSCT and 70% (95% CI, 59-79) after MSD alloSCT (P = .02) in this patient subgroup. In a propensity-score matched IRmol subcohort (n = 106), MSD alloSCT was associated with superior leukemia-free survival (hazard ratio [HR] 0.33, P = .004) and increased OS in patients alive 1 year after transplantation (HR 0.20, P = .004). These results indicate that, within IRcyto AML in CR1, autoSCT may be a valid option for FRmol patients, whereas MSD alloSCT should be the preferred postremission strategy in IRmol patients., (Copyright © 2021 The American Society for Transplantation and Cellular Therapy. Published by Elsevier Inc. All rights reserved.)

Extended half-life of factor IX (FIX) demonstrated clinical benefit and lower treatment burden than standard half-life FIX products in clinical trials. We analysed the impact in efficacy, pharmacokinetics (PKs) and costs of the switch from nonacog alfa (rFIX) to albutrepenonacog alfa (rFIX-FP) in the first patient with haemophilia B (HB) treated in Spain outside clinical trials. A 7-year-old boy presented with HB with poor venous access and repetition infections using rFIX, which was switched to rFIX-FP. Prophylaxis was adjusted by PKs using WAPPS-Hemo tailoring from 100 IU/kg/week of rFIX to 80 IU/kg/3 weeks of rFIX-FP. Comparing 6 months before, rFIX-FP reduced 68.5% FIX consumption/kg and 58.3% infusion frequency, but total costs/weight showed a slight increase. Ratio of half-life between rFIX and rFIX-FP was 3.4-3.7. This case report revealed that switch to rFIX-FP decreased frequency and FIX consumption, without adverse events and bleeds., Competing Interests: Competing interests: None declared., (© BMJ Publishing Group Limited 2020. No commercial re-use. See rights and permissions. Published by BMJ.)

Purpose: Long-term therapy of haematology patients has been facilitated by permanent indwelling central venous catheters. We performed a retrospective study to compare the problems occurring with a externalized catheter (Hickman) versus a totally implanted port catheter., Patients and Methods: A total of 171 catheters were placed to 139 haematological patients, 77 patients with Hickman catheters and 94 with totally implanted port catheters. We review our experience in order to identify factors associated with complications., Results: Pneumothorax occurred in one of 171 of the percutaneously placed devices. Other early complications were hematoma 13, and catheter migration out of the vascular tree 8. Late complications included malposition (5.8%), thrombosis (2.9%), septic thrombosis (1.7%) and most notably infection (38.5%). 62 of 77 patients with Hickman catheters developed catheter-related infection (hazard rate infection 7.1/1000 days) compared with 53 of 94 patients with implanted port catheters (hazard rate infection 1.5/1000 days, p < 0.001). Most of infections that occurred were caused by gram-positive organisms but the gram-negative organisms infections resulted in a significantly higher rate of treatment failure and recurrence. A total of 72 catheters were removed of the central line: 36 for infection., Conclusion: We found a significantly increased incidence of catheter-related infection in patients with Hickman catheters. We also observed that the use of intravenous antibiotic prophylaxis prior to catheter insertion did not appear to be beneficial and thrombocytopenia at this moment was a factor in the development of hematoma. The infections due to coagulase-positive staphylococci can be treated successfully without removal of the catheters. However in catheter-related bacteremia due gram-negative organisms there is a chance that the bacteremia will recur if the catheter is not removed.