Drugs of today
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Giant cell tumor of bone (GCTB) is an osteolytic, usually benign neoplasm characterized by infiltration with osteoclast-like giant cells, and the osteoclast differentiation factor receptor activator of nuclear factor kappa-B ligand (RANKL) is heavily involved in its pathogenesis. Denosumab belongs to a new class of drugs that inhibit RANKL. ⋯ On the basis of this data, the FDA approved denosumab for the treatment of patients whose GCTB is unresectable, or when surgery is likely to result in severe morbidity. Ongoing questions remain, including the optimal scheduling, patient selection, use in the adjuvant setting and long-term toxicity concerns.
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Lower respiratory tract infections are usually treated with systemic antibiotics, but sometimes in certain conditions inhaled antibiotics may also be used. In cystic fibrosis (CF) for example, inhaled tobramycin is used to treat chronic airways infection due to Pseudomonas aeruginosa, and in other conditions such as ventilator-associated pneumonia, inhaled colistin is used as add-on therapy to reduce the risk of side effects of prolonged systemic exposure. Other inhaled antibiotics are currently under development, and inhaled amikacin is one such example. The two formulations of inhaled amikacin currently under clinical development may have two different therapeutic trajectories: the nebulized liposomal formulation is the first once-daily aminoglycoside for CF patients, whereas the free nebulized amikacin may be used in addition to systemic therapy in ventilator-associated pneumonia patients exploiting reduced systemic exposure to amikacin as a therapeutic advantage, especially in patients with renal impairment.
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Glucagon-like peptide 2 (GLP-2) decreases gastric and intestinal motility, reduces gastric secretions, promotes intestinal growth and improves post-resection structural and functional adaptation in short bowel syndrome (SBS). Teduglutide, an analogue of GLP-2, has a prolonged half-life and provides intestinotrophic effects with once-daily subcutaneous injection in patients with SBS. This monograph reviews the preclinical and clinical data that provide the scientific rationale for the use of teduglutide in this orphan condition. ⋯ Following its positive regulatory review and approval by the European Medicines Agency and the U. S. Food and Drug Administration in 2012, teduglutide has moved from the research setting to clinical practice, offering a new treatment paradigm for this burdensome and potentially life-threatening condition.
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Multiple myeloma (MM) is an incurable disease characterized by the proliferation of plasma cells. The survival in MM patients has improved significantly in the past decade due to the introduction of novel agents. ⋯ Although the development of novel agents has improved the outcomes of MM treatment, most of the patients will still relapse and become refractory to therapy due to development of drug resistance. A better understanding of the biological mechanisms of MM progression, including cellular and molecular events in the MM cells and in their bone marrow microenvironment, is warranted to provide new therapeutic targets and develop new drugs and therapeutic strategies to treat MM.
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A multivesicular liposomal formulation of bupivacaine which can provide prolonged postsurgical pain relief has been developed. Two pivotal placebo-controlled phase III trials, one in hemorrhoidectomy and one in bunionectomy procedures, showed that wound infiltration of the suspension at the end of surgery could provide analgesia for up to 72 hours, reduce the amount of opiate rescue medications, the time to their first use and increase patient satisfaction. ⋯ S. health economic study showed benefits (versus patient-controlled analgesia) for the preparation in decreasing total opioid use, length of hospital stay and total hospital costs. Further larger studies are warranted, in particular using bupivacaine hydrochloride as the comparator.