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Targeting of Antithrombin in Hemophilia A or B with RNAi Therapy
TLDR
Once‐monthly subcutaneous administration of fitusiran resulted in dose‐dependent lowering of the antithrombin level and increased thrombin generation in participants with hemophilia A or B who did not have inhibitory alloantibodies.
Randomized, prospective clinical trial of recombinant factor VIIa for secondary prophylaxis in hemophilia patients with inhibitors
TLDR
Clinically relevant reductions in bleeding frequency during prophylaxis as compared to conventional on‐demand therapy were achieved without raising safety concerns, providing evidence for the concept of secondary rFVIIa prophyllaxis in inhibitor patients with frequent bleeds.
Bendamustine compared with chlorambucil in previously untreated patients with chronic lymphocytic leukaemia: updated results of a randomized phase III trial
TLDR
Bendamustine should be considered as a preferred first‐line option over chlorambucil for CLL patients ineligible for fludarabine, cyclophosphamide and rituximab.
Prophylaxis vs. on‐demand treatment with Nuwiq® (Human‐cl rhFVIII) in adults with severe haemophilia A
Haemophilia A is treated with FVIII, either prophylactically or on demand. Prophylaxis is the gold standard in children and evidence is accumulating in adults.
Randomized, controlled, parallel‐group trial of routine prophylaxis vs. on‐demand treatment with sucrose‐formulated recombinant factor VIII in adults with severe hemophilia A (SPINART)
The benefits of routine prophylaxis vs. on‐demand treatment with factor VIII products have not been evaluated in controlled clinical trials in older patients with hemophilia A.
Phase II study of ISIS 3521, an antisense oligodeoxynucleotide to protein kinase C alpha, in patients with previously treated low-grade non-Hodgkin's lymphoma.
TLDR
ISIS 3521 has demonstrated anti-tumour activity in patients with relapsed low- grade NHL and there may be a potential role for this agent in combination with conventional chemotherapy for advanced low-grade lymphoma, and further trials are warranted.
Phase-I/II study to evaluate dose limiting toxicity, maximum tolerated dose, and tolerability of bendamustine HCl in pre-treated patients with B-chronic lymphocytic leukaemia (Binet stages B and C)
TLDR
First-line use of bendamustine is a safe option for CLL-patients requiring treatment, because it is highly effective, reasonably safe, and does hardly produce cross-resistance against other anti-neoplastic drugs effective in this indication.
Bendamustine Versus Chlorambucil as First-Line Treatment in B Cell Chronic Lymphocytic Leukemia: An Updated Analysis from An International Phase III Study
TLDR
This study has shown that bendamustine offers significantly greater efficacy than chlorambucil, with manageable toxicity, and should be considered as first-line chemotherapy for patients with advanced B-CLL.
Long-acting recombinant coagulation factor IX albumin fusion protein (rIX-FP) in hemophilia B: results of a phase 3 trial.
TLDR
Results indicate rIX-FP is safe and effective for preventing and treating bleeding episodes in patients with hemophilia B at dosing regimens of 40IU/kg weekly and 75 IU/kg every 2 weeks.
Pharmacokinetics, efficacy and safety of BAX326, a novel recombinant factor IX: a prospective, controlled, multicentre phase I/III study in previously treated patients with severe (FIX level <1%) or
TLDR
It is indicated that BAX326 is safe and efficacious in treating bleeds and routine prophylaxis in patients aged 12 years and older with haemophilia B.
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