Rare Disease Report


The FDA has approved cabozantinib (Cabometyx) for the treatment of patients with hepatocellular carcinoma who previously received sorafenib (Nexavar).
Only patients with very serious, life-threatening conditions with a risk/benefit ratio is acceptable for use of the therapy at this time.  
Dipanjan Basu, PhD, is testing non-melanoma drugs, like the investigational therapy omipalisib (GSK2126458) in cultured patient cells.
While the absolute risk of therapy-related blood cancers is low, the treatment is often intensive and linked to substantial morbidity.  
The FDA has approved dasatinib (Sprycel) in combination with chemotherapy for the treatment of pediatric patients with newly diagnosed Philadelphia chromosome-positive (Ph+) acute lymphoblastic leukemia (ALL).
The FDA has approved pembrolizumab for the treatment of adult and pediatric patients with recurrent locally advanced or metastatic Merkel cell carcinoma (MCC).
Mohamad Mohty, MD, PhD, discusses the investigational therapy cytotoxic T lymphocytes (CTLs) therapy for the treatment of Epstein-Barr virus associated post-transplant lymphoproliferative disorder (EBVPTLD). 
This rare leukemia form may be treated with ruxolitinib, which appears to be well-tolerated by patients.
Patients with myeloproliferative neoplasms (MPN) with essential thrombocythemia (ET) or polycythemia vera (PV) were found to benefit from hydroxyurea therapy. 
Patients with myeloid/lymphoid neoplasms enrolled in the phase 2 clinical trial demonstrated pemigatinib was generally well-tolerated.  
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