CLINICAL NEWS
The drug carries a boxed warning for CRS and neurologic events . Fortynine percent of patients in the trial experienced grade 3 or 4 CRS , and 18 percent of patients experienced grade 3 or 4 neurologic events within eight weeks of treatment . There were no incidents of cerebral edema , and the most common neurologic events were encephalopathy ( 34 %), headache ( 37 %), delirium ( 21 %), anxiety ( 13 %), and tremor ( 9 %).
Because of the risk of CRS and neurologic events , the drug was approved with a Risk Evaluation and Mitigation Strategy . to treat CAR T-cell – induced , severe or life-threatening CRS in patients ≥2 years of age . In clinical trials of patients treated with CAR T cells , 69 percent of patients had complete resolution of CRS within two weeks following one or two doses of tocilizumab . ●
Sources : U . S . Food and Drug Administration news release , August 30 , 2017 ; Novartis news release , August 30 , 2017 .
For an in-depth look at tisagenlecleucel and other CAR T-cell therapies in the hematologic malignancies pipeline , see our special edition of ASH Clinical News , “ Focus on Immunotherapies .”
“ Gene and cell therapies hold … the potential to transform medicine and create an inflection point in our ability to treat and even cure many intractable illnesses .”
FACTOR REPLACEMENT MIRRORS THE PROTECTION WITHIN
— SCOTT GOTTLIEB , MD
“ The approval of CAR T-cell therapy for pediatric leukemia marks an important shift in the blood cancer treatment paradigm . … This is a potentially curative therapy in patients whose leukemia is unresponsive to other treatments and represents the latest milestone in the shift away from chemotherapy toward precision medicine ,” said Kenneth Anderson , MD , president of the American Society of Hematology , in a news release . “ While the importance of CAR-T cannot be overstated , this approval only pertains to a small population of children . More research is needed to make this therapy more effective for a broader population , to reduce the severe side effects that patients experience during treatment , and ultimately to find a broader application beyond blood cancers .”
On the same day as the approval , the FDA expanded the indication for tocilizumab , a monoclonal antibody
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References : 1 . Franchini M , Mannucci PM . The history of hemophilia . Semin Thromb Hemost . 2014 ; 40:571-576 . 2 . Peyvandi F , Garagiola I , Young G . The past and future of haemophilia : diagnosis , treatments , and its complications . Lancet . 2016 ; 388:187-197 . 3 . Lenting PJ , van Mourik JA , Mertens K . The life cycle of coagulation factor VIII in view of its structure and function . Blood . 1998 ; 92 ( 11 ): 3983-3996 . 4 . Antovic A , Mikovic D , Elezovic I , Zabczyk M , Hutenby K , Antovic JP . Improvement of fibrin clot structure after factor VIII injection in haemophilia A patients treated on demand . Thromb Haemost . 2013 ; 111 ( 4 ): 656-661 . 5 . Hvas AM , Sørensen HT , Norengaard L , Christiansen K , Ingerslev J , Sørensen B . Tranexamic acid combined with recombinant factor VIII increases clot resistance to accelerated fibrinolysis in severe hemophilia A . J Thromb Haemost . 2007 ; 5:2408-2414 .
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