CLINICAL NEWS
EDR was 0.24 for the entire population – lower than the “ excessive use ” threshold .
According to the study ’ s findings , EDR for children was significantly lower than for adults > 30 years old ( 0.18 vs . 0.23 ; p = 0.009 ). However , patients in the transition-age groups and young-adult age groups relied more heavily on ED visits than children ( 0.33 for both vs . 0.18 ; p < 0.001 ).
When the researchers examined temporal patterns in EDR among age groups over the study period , they found that EDR was continuously below the established threshold of 0.33 , though it significantly increased from 0.17 in 2011 to 0.21 in 2015 ( p < 0.001 ).
However , the EDR in the transition-age group continued to increase over time , from 0.23 in 2011 to 0.37 in 2015 ( p = 0.001 ), suggesting that “ further interventions focusing on the transition-age group are needed to improve care and ensure smooth transition from the pediatric to adult health-care systems ,” the authors concluded .
The study was limited by its retrospective nature , and because it was conducted in a single state , the results may not be generalizable to the larger U . S . patient population .
Also , both studies were completed before the FDA approval of L-glutamine oral powder in July 2017 ( the second FDAapproved treatment for SCD ), the availability of which may alter patients ’ incidence and management of painful episodes .
Dr . Ballas and authors report financial relationships with Novartis and Global Blood Therapeutics ; Dr . Singh and authors report no financial conflicts .
REFERENCES
1 . Ballas SK , Kanter J , Agodoa I , et al . Opioid utilization patterns in United States patients with sickle cell disease . Abstract # 130 . Presented at the 2017 American Society of Hematology Annual Meeting , December 9 , 2017 ; Atlanta , GA .
2 . Singh A , Yan K , Brandow AM , Panepinto JA . Longitudinal trend in emergency department reliance for pain among sickle cell disease patients in Wisconsin . Abstract 901 . Presented at the 2017 American Society of Hematology Annual Meeting , December 9 , 2017 ; Atlanta , GA .
Attendees at the 2017 ASH Annual Meeting .
Stephen J . Schuster , MD , speaks at a press briefing .
Tisagenlecleucel Shows High Response Rates in Patients With Relapsed or Refractory Diffuse Large B-Cell Lymphoma
Updated results from the phase II JULIET trial of patients with relapsed or refractory diffuse large B-cell lymphoma ( DLBCL ) presented at the 2017 ASH Annual Meeting confirmed results from an earlier interim analysis : A single infusion of tisagenlecleucel led to good response rates and durable remissions in a patient population that has few to no treatment options available .
As lead author Stephen J . Schuster , MD , from the Abramson Cancer Center at Penn Medicine in Philadelphia , reported during his presentation , 43 of 81 patients ( 53 %) responded to tisagenlecleucel treatment , demonstrating that “ the efficacy we saw in earlier trials was sustained .”
Tisagenlecleucel was the first chimeric antigen receptor ( CAR ) T-cell therapy approved by the U . S . Food and Drug Administration for the treatment of pediatric patients with B-cell precursor acute lymphocytic leukemia ( ALL ). According to Dr . Schuster , it is also a promising therapy for difficult-to-treat DLBCL . “ This is a frustrating patient population because we feel that we can save lives in DLBCL , but not this population ,” he told ASH Clinical News . “ Patients who either don ’ t respond to secondline therapy and are refractory to immunochemotherapy or whose disease has progressed after transplant have no great existing therapy .”
As of March 8 , 2017 ( data cutoff ), 147 patients were enrolled in the trial and 99 ( median age = 56 years ; range = 22-76 years ) were infused with a single dose of tisagenlecleucel ( median dose = 3.1 × 10 8 cells / kg ; range = 0.1-6.0 × 10 8 cells / kg ). Seventy-seven percent of patients had stage III or IV disease at study entry . The median number of prior lines of antineoplastic therapy was three ( range = 1-6 therapies ); 95 percent of patients received two or more therapies , and 51 percent received three or more . Nearly half ( 47 %) had received autologous hematopoietic cell transplantation . Prior to infusion , 93 percent received lymphodepleting chemotherapy with fludarabine , cyclophosphamide , or bendamustine . Dr . Schuster reported findings from the 81 patients infused with tisagenlecleucel who had been followed for at least three months .
Over a median follow-up of 29 months ( range not reported ), the best overall response rate ( ORR ; primary endpoint ) was 53.1 percent ( p < 0.0001 ), which included 32 complete remissions ( CRs ; 39.5 %) and 11 partial remissions ( PRs ; 13.6 %).
Three months after tisagenlecleucel infusion , the ORR was 38 percent
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