Arthritis Drug Tofacitinib Approved for Severe Ulcerative Colitis | FDA

The US FDA has approved tofacitinib (Xeljanz, Pfizer) for the treatment of moderate-severe ulcerative colitis (UC).

Tofacitinib

  • Janus kinase inhibitor (blocks STAT transcription factor activation and inflammatory signaling)
  • First oral agent approved for chronic use in UC
  • 10 mg tofacitinib PO BID induced remission in 17-18% of patients (vs. 8.2% placebo) after 8 weeks
  • Of patients that responded after 8 week trial, 40.6% who continued 10-mg tofacitinib for 52 weeks achieved sustained remission (versus 11.1% placebo)
  • Adverse events (common):  arthralgia, diarrhea, elevated cholesterol, headache, shingles, rash, and upper respiratory infection
  • Adverse events (severe): malignancy (e.g., lymphoma), serious opportunistic infections (FDA black-box warnings)

Continue reading “Arthritis Drug Tofacitinib Approved for Severe Ulcerative Colitis | FDA”

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Fluticasone Propionate vs. Fluticasone Furoate: What’s the Difference?

Fluticasone Propionate and Fluticasone Furoate: Ester Cousins

Fluticasone propionate (FP) and fluticasone fuorate (FF) are both synthetic glucocorticoid drugs used in the prevention of asthma exacerbations and for the treatment of allergy symptoms and rhinitis. Continue reading “Fluticasone Propionate vs. Fluticasone Furoate: What’s the Difference?”

Fluticasone Furoate (Flonase Sensimist®, Arnuity Ellipta®)

Overview

Fluticasone furoate is a synthetic corticosteroid with clinical applications in the treatment of asthma and allergy symptoms.  Continue reading “Fluticasone Furoate (Flonase Sensimist®, Arnuity Ellipta®)”

Fluticasone Propionate (Flonase Allergy Relief®, Flovent®, ArmonAir RespiClick® Cutivate®)

Overview

Fluticasone propionate is a synthetic corticosteroid with clinical applications in the treatment of asthma, allergy symptoms, non-allergic rhinitis, and dermatologic conditions.  Continue reading “Fluticasone Propionate (Flonase Allergy Relief®, Flovent®, ArmonAir RespiClick® Cutivate®)”

Anti-IL-1β Therapy For Genetic Fever Syndromes Eliminates Flares in Nearly Half of Patients | NEJM

Study

  • Clinical Trial: CLUSTER
  • Authors: Benedetti, Penades et al
  • Journal: New England Journal of Medicine
  • Date: May 17th, 2018

Conclusions

  • Subcutaneous canakinumab, an anti-IL-1β antibody, eliminated flares in 23-53% of patients with one of three autoinflammatory fever syndromes (FMF, MKD, TRAPS) for up to 40 weeks
  • IL-1β common mediator in FMF, MKD, TRAPS pathogenesis
  • Increased risk of infection w/ canakinumab use

Continue reading “Anti-IL-1β Therapy For Genetic Fever Syndromes Eliminates Flares in Nearly Half of Patients | NEJM”

Ibuprofen (Motrin®, Caldolor®, Advil®)

Overview

Ibuprofen is a non-steroidal anti-inflammatory drug (NSAID) and non-selective cyclooxygenase inhibitor indicated for the treatment of mild-moderate pain, fever, and arthritis.  Its analgesic, antipyretic, and anti-inflammatory properties are thought to be mediated through inhibition of the cyclooxygenase-2 (COX-2) enzyme.  COX-2 is upregulated by cells during the inflammatory response, resulting in increased production of PGE2 and leading to tissue inflammation, fluid extravasation, pyrexia, pain, and vasodilation.  Inhibition of prostaglandin synthesis by ibuprofen blocks inflammatory signaling pathways and decreases pain and body temperature. Continue reading “Ibuprofen (Motrin®, Caldolor®, Advil®)”

Novel Enzyme Therapy Approved for Genetic Disease Phenylketonuria | FDA

The US FDA has approved pegvaliase (Palynziq®, BioMarin Inc) for the treatment of phenylketonurea (PKU) uncontrolled by conventional therapy.

Pegvaliase

  • Pegylated recombinant bacterial Anabaena variabilis phenylalanine ammonia lyase
  • Subcutanous daily injection, 5-60 mg
  • 51.1% decrease in blood Phe levels after 12 months of therapy
  • Half of patients on treatment achieve blood Phe level < 120 µmol/L, the upper limit of normal range, within 2 years
  • Adverse events (common): arthralgia (70.5%), injection-site reaction (62.1%), headache (47.1%)
  • Adverse events (severe): anaphylaxis (4.6%)
  • Current treatment: dietary phenylalanine restriction, sapropterin in THB-responsive PKU

Continue reading “Novel Enzyme Therapy Approved for Genetic Disease Phenylketonuria | FDA”

Cannabidiol Reduces Number of Seizures in Lennox-Gastaut Syndrome, Rare Epilepsy Disorder| NEJM

Publication

  • Study: GWPCARE3
  • Authors: Devinsky, Zuberi et al
  • Journal: New England Journal of Medicine
  • Date: May 17th, 2018

Conclusions

  • Cannabidiol add-on to conventional antiepileptic therapy reduced drop seizure frequency in patients with Lennox–Gastaut syndrome
  • Both 10 mg/kg and 20 mg/kg cannabidiol effective in reducing number of seizures
  • Dose-dependent increase in adverse events relative to placebo

Continue reading “Cannabidiol Reduces Number of Seizures in Lennox-Gastaut Syndrome, Rare Epilepsy Disorder| NEJM”

Levothyroxine (Synthroid®, Levoxyl®, Tirosint®, Unithroid®)

Overview

Levothyroxine is a thyroid receptor agonist commonly prescribed for hypothyroidism.  It is a synthetically manufactured levo-isomer of the endogenous thyroxine (T4) hormone, and is converted peripherally to triiodothyronine (T3) hormone.  T3 binds to thyroid hormone receptors (TR) and can induce either transcriptional repression or derepression depending on the molecular and cellular context.  These transcriptional changes give rise to an increased sensitivity to catecholamines (e.g., raising heart rate and blood pressure) and increasing metabolic activity.  Pituitary thyroid-stimulating hormone (TSH) is downregulated by levothyroxine administration.

Continue reading “Levothyroxine (Synthroid®, Levoxyl®, Tirosint®, Unithroid®)”

Platelet-Boosting Drug Approved to Prevent Surgical Bleeding in Cirrhosis Patients | FDA

The US FDA has approved the thrombopoietin receptor agonist avatrombopag (Doptelet®, AkaRx Inc) for the prevention of bleeding events in patients with chronic liver disease and thrombocytopenia undergoing a planned medical or dental procedure.

Avatrombopag

  • Thrombopoietin receptor agonist, stimulates platelet production
  • Decreases serious bleed risk 2-3 fold in patients w/ severe thrombocytopenia (<50*109/L) and chronic liver disease undergoing invasive procedures for up to 7 days after procedure
  • Most common adverse events: fever, abdominal pain, fatigue, edema, nausea
  • Current treatment: pre-procedural platelet transfusion 
  • May increase risk of thromboembolism in some patients

Continue reading “Platelet-Boosting Drug Approved to Prevent Surgical Bleeding in Cirrhosis Patients | FDA”