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Content Update

November 26, 2019

Voxelotor Approved for Treatment of Sickle Cell Disease: Voxelotor (Oxbryta®) is the first hemoglobin S polymerization inhibitor approved by the FDA and is indicated for adults and children older than 12 for the treatment of sickle cell disease. Sickle cell disease affects approximately 100,000 individuals in the US and is a hereditary condition which results in the production of sickle, or abnormally, shaped hemoglobin and has limited treatment options. In a phase 3 randomized, placebo controlled, double blind trial, two doses of voxelotor were compared to placebo. Those treated with the 1500mg dose had a significantly higher hemoglobin response compared to placebo, 51% (95% CI, 41 - 61) versus 7% (95% CI, 1 - 12). Adverse effects were similar across the study groups and the most common being headache and diarrhea.

Content Update

August 28, 2019

Erdafitnib Approved for FGFR Mutated Bladder Cancers: Erdafitnib (Balversa®) is the first FGFR inhibitor approved by the FDA and is indicated for adults with FGFR3 or FGFR2 mutations in locally advanced and unresectable or metastatic bladder cancer who have progressed on prior therapy. FGFR mutations are common, occurring in up to 60% of bladder cancers. In an open-label, multicenter, Phase II trial, the median progression free survival was 5.5 months (95% CI, 4.2-6.0). The overall response rate to erdafitnib was 40%. The most common adverse effects were hyperphosphatemia, stomatitis and diarrhea.

Content Update

May 7, 2019

ASCO Practice Guidelines on Management of Immune-Related Adverse Events in Patients Treated with Immune Checkpoint Inhibitors: In February 2018, the American Society of Clinical Oncology (ASCO) published a new guideline regarding the management of immune-related adverse events in patients treated with immune checkpoint inhibitor therapy. Recommendations are outlined by organ-specific toxicities with diagnosis and management criteria presented. Differences in treatment approaches exist, but most patients with Grade 1 toxicities may continue immune checkpoint inhibitor therapy. Corticosteroids may be initiated for Grade 3 toxicities and higher and must be tapered over at least 4 to 6 weeks. In severe and refractory cases, other immunosuppressive therapies may be required to control immune-related adverse events. In most cases, immune checkpoint inhibitor therapy is permanently discontinued with Grade 4 toxicities.

Content Update

March 4, 2019

Larotrectinib Approved for NTRK Fusion Cancers: Larotrectinib (Vitrakvi®) is the first disease agnostic small molecule inhibitor approved by the FDA and is indicated for adult and pediatric patients with NTRK fusion cancers. While NTRK fusions are rare, occurring in ~ 1% of all cancers, they are common in some rare cancers such as secretory carcinoma of the breast. Overall response rate in NTRK fusion cancers is 75%. The most common adverse effects are elevations in LFTs and anemia, with serious neuropathies in about 1% of patients. Larotrectinib is ...

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