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ASCO: Is Blincyto Safe and Effective in Pediatric Relapsed or Refractory ALL Patients?

JUNE 05, 2017
James Radke
Blincyto (blinatumomab) is indicated for the treatment of Philadelphia chromosome-negative (Ph-) relapsed or refractory B-cell precursor acute lymphoblastic leukemia (ALL).
 
Based on findings from a phase II trial, the U.S. Food and Drug Administration (FDA) granted an accelerated approval to blinatumomab in 2014 as a treatment for patients with Ph- relapsed/refractory B-precursor ALL.
 
Earlier this year, a supplemental biologics license application (sBLA) was submitted to the FDA for the full regulatory approval of blinatumomab (Blincyto) showing data from the phase III TOWER study, in which the median overall survival (OS) with blinatumomab was 7.7 months versus 4 months with standard chemotherapy.
 
Additionally, the drug was studied in an open-label, expanded access study involving children with relapsed/refractory B-precursor ALL. Data from that study was presented by Locatelli and colleagues at the American Society of Clinical Oncology (ASCO) annual meeting in Chicago this week with the primary objective to assess the adverse event profile of this drug in these patients.
 
Pediatric patients with ≥ 5% blasts and relapsed/refractory B-precursor ALL (refractory, ≥ 2 relapses or relapse after transplant) were given blinatumomab dosed by continuous infusion (4 weeks on/2 weeks off) for up to 5 cycles. For patient with 5-25% blasts the dosing was 15 µg/m2/day. For patients with ≥25% blasts, the dosage was 5 µg/m2/day on day 1−7 in cycle 1, then 15 µg/m2/day). The primary endpoint was incidence of treatment-emergent adverse event (TEAE) and treatment-related adverse events (TRAEs). Key efficacy endpoints were complete response (CR) and minimal residual disease (MRD, by PCR or flow cytometry) in the first 2 cycles, relapse-free survival, overall survival and hematopoietic cell transplantation (HCT) rate. 

Results

Data from the first 40 patients, whose median age was 9 years (range, 1−17 years), were presented at ASCO. Of the 40, 24 had ≥ 2 relapses, 20 relapsed after HCT and 5 were primary refractory. Further, 18 had ≥ 50% blasts and 21 had prior HCT.
 
Regarding safety, Table 1 shows that most patients had a TEAE and the most common TEAEs were pyrexia (78%), cytokine release syndrome (CRS; 23%) vomiting (23%) and anemia (20%).
 
A total of 10 patients interrupted treatment and 2 discontinued due to TRAEs and 13 patients had TRAE that were grade 3 or higher.  Two patients had fatal AEs but were considered unrelated to treatment.

Table 1: Safety and Efficacy
TEAEs All patients
N=40
 
  All Grades
  Grade 3
  Grade 4
  Fatal
 
39
15
12
2
Complete Response (CR) All patients
N=40
 
  < 50% Blasts
  ≥ 50% Blasts
  t (17;19)
 
15
10
2
MRD Responders 
N=25
 
MRD Response < 10-4
  < 50% Blasts
  ≥ 50% Blasts
  t (17;19)
 
19
12
7
2

Reference

Locatelli F, Zugmaier G, Vora A, et al. Blinatumomab use in pediatric patients (pts) with relapsed/refractory B-precursor acute lymphoblastic leukemia (r/r ALL) from an open-label, multicenter, expanded access study. J Clin Oncol. 2017; 35: (suppl; abstr 10530).


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