Miscellaneous Hematological Agents
The researchers found that the MFS/ATR1a-null mice showed unabated aortic root enlargement and remained responsive to losartan, despite being hypotensive and resistant to AngII vasopressor effects.
Data were included for 2,821 children with 5,014 person-years. The researchers found that 18 percent of children received ≥300 days of antibiotics.
After completion of the initial bevacizumab treatment cycle, there was a significant reduction in epistaxis severity scores and RBC transfusion requirements.
At 90 days, the prevalence of venous thromboembolism varied from 0.3 to 1.37% for all patients and from 0.75 to 3.86% for hospitalized patients.
The researchers observed significant associations for lupus anticoagulant (48% positive in cases versus 5% in controls), protein C deficiency (50 versus 8%), and combined thrombophilias (62 versus 31%) with calciphylaxis among all patients.
Endari is indicated to reduce the acute complications of sickle cell disease in adult and pediatric patients 5 years of age and older.
"Warfarin use may have broad anticancer potential in a large, population-based cohort of persons older than 50 years," the authors write.
"While lenalidomide therapy does produce hematological responses in patients with MDS/MPN-RS-T, these are often suboptimal, without significant changes in bone marrow morphology," the authors found.
Physicians need to be able to explain concepts of autologous and allogeneic use of cord blood units, as well as benefits and limitations of blood banking and transplantation.
Receipt of red blood cell transfusion from ever-pregnant donor tied to increased risk for males.
The trial, L-PLUS2, included 215 patients who were randomized 1:1 to either 3mg of lusutrombopag or placebo for up to 7 days.
The researchers found that in transplanted patients, the 30-year overall survival (OS) was 82.6±2.7% and thalassemia-free survival (TFS) was 77.8±2.9% compared with OS of 85.3±2.7% in CT patients (P=NS).
Erythropoietic protoporphyria (EPP), the most common form of porphyria (a group of blood disorders), affects the body's ability to make heme resulting in symptoms of chronic anemia. A patient's skin can become very sensitive to light, resulting in disfiguring blisters.
Burosumab is being developed in a collaboration between Ultragenyx and Kyowa Hakko Kirin Pharmaceuticals, based on a license agreement between both companies.
The FDA has accepted for review the BLA resubmission for AndexXa (andexanet alfa; Portola) for the reversal of the anticoagulant effects of Factor Xa inhibitors in patients experiencing uncontrolled or life-threatening bleeding.
"By using our novel nanochip technology, injured or compromised organs can be replaced. We have shown that skin is a fertile land where we can grow the elements of any organ that is declining," said Dr. Chandan Sen.
The application includes data from the open-label, Phase 2 VE-BASKET study; non-randomized, basket study investigating the use of Zelboraf in patients with BRAF V600 mutation-positive cancers and other diseases, including 22 patients with ECD.
The safety and efficacy of Endari were evaluated in a randomized clinical trial involving patients aged 5-58 years with sickle cell disease who had ≥2 painful crises within 12 months prior to trial enrollment.
Synthetic Biotic medicines utilize synthetic biology to reprogram probiotic bacteria to perform critical functions that compensate for those missing or damaged due to a particular disease.
Both the tablets and granules should not be taken with aluminum-containing antacid products. The drug labeling contains additional information for patients who are on current chelation therapy with Exjade (deferasirox) tablets for oral suspension and converting to Jadenu.
Wechsler, ME et al. "Mepolizumab or Placebo for Eosinophilic Granulomatosis with Polyangiitis". DOI: 10.1056/NEJMOA1702079