CLINICAL NEWS
trials – including the trial described in
this paper – we believe that initiating
treatment with 150 mg/day in adults is
indicated to improve the likelihood of a
faster response.”
In this report, Dr. Dunbar and col-
leagues reviewed data from this initial
phase II trial (“study 1”), which enrolled 43
patients (median age = 45 years; range =
17-77 years), with data from a subsequent
phase II trial (“study 2”) of 40 patients
(median age = 50 years; range = 4-79 years)
whose disease did not respond to rab-
bit or horse antithymocyte globulin with
cyclosporine.
Demographics and clinical charac-
teristics were similar between the two
study groups, although patients in study
1 had received more immunosuppressive
therapies and more were receiving platelet
transfusions at baseline.
In study 2, eltrombopag dosing was
stratified by age:
• ≥12 years: 150 mg/day
• 6-11 years: 75 mg/day
• 2-5 years: 2.5 mg/kg/day
Unlike the earlier trial, responses (defined as
platelets >100×10 9 /L, hemoglobin 10 g/dL,
absolute neutrophil count >1,000/uL)
NOW APPROVED
Effi cacy in treatment-naïve patients (N=29) 1
72%
CR/CRc
rate*
90%
ORR
45%
bridged to stem cell
transplantation
* CRc = clinical complete response; defi ned as complete response with residual skin abnormality not indicative of active disease. 2
CR = complete response; ORR = overall response rate.
Median duration of CR/CRc has not yet been reached in treatment-naïve
patients (range, 1.3-32.2+ months). 1
Median overall survival has not yet been reached; median follow-up 23.0 months
(range, 0.2-41.0+ months). 1
In the pivotal cohort, treatment-naïve patients (N=13) achieved a 54% CR/CRc rate,
77% ORR, and 46% bridged to stem cell transplantation. 1,2
ELZONRIS TM (tagraxofusp-erzs) was evaluated in treatment-naïve and previously-treated patients with BPDCN in
an open-label, multicenter clinical study (N=44). The pivotal cohort consisted of 13 treatment-naïve patients. 1,2
VISIT ELZONRIS.COM/HCP FOR MORE INFORMATION.
Hypersensitivity Reactions ADVERSE REACTIONS:
• ELZONRIS can cause severe hypersensitivity reactions. Grade 3
or higher events were reported in 10% of patients in clinical
trials. Monitor patients for hypersensitivity reactions during
treatment with ELZONRIS. Interrupt ELZONRIS infusion and
provide supportive care as needed if a hypersensitivity
reaction should occur. If the reaction is severe, discontinue
ELZONRIS permanently
Hepatotoxicity
• Elevations in liver enzymes can occur with ELZONRIS.
Grade 3 or higher elevations in liver enzymes occurred
in approximately 40% of patients in clinical trials
• Monitor alanine aminotransferase (ALT) and aspartate
aminotransferase (AST) prior to each infusion with ELZONRIS.
Temporarily withhold ELZONRIS if the transaminases rise to
greater than 5 times the upper limit of normal (ULN) and
resume treatment upon normalization or when resolved The most common adverse reactions in the clinical trials
(incidence ≥ 30%) are capillary leak syndrome, nausea, fatigue,
peripheral edema, pyrexia, and weight increase. The most
common laboratory abnormalities (incidence ≥ 50%) are
decreases in albumin, platelets, hemoglobin, calcium,
sodium, and increases in glucose, ALT, and AST.
Please see Brief Summary of full Prescribing Information,
including Boxed WARNING, on the following pages.
To report SUSPECTED ADVERSE REACTIONS, contact Stemline
Therapeutics, Inc. at 1- 877-332-7961 or contact the FDA at
1- 800-FDA-1088 or www.fda.gov/medwatch.
References: 1. Data on fi le. Stemline Therapeutics, Inc. 2. ELZONRIS [prescribing
information]. New York, NY, US: Stemline Therapeutics, Inc.; December 2018.
were assessed at 12 weeks and 24 weeks.
Patients whose disease responded to el-
trombopag continued with therapy on an
extension phase until their blood counts
stabilized (defined as no further improve-
ment for >6 months) or until achieve-
ment of transfusion independence. The
researchers also performed a bone marrow
aspiration and biopsy with cytogenetics at
baseline, 12 weeks, and 24 weeks of treat-
ment, and every six months in responders