On Location ASH Annual Meeting
dromes (MDS; 15%) with <10 percent marrow blasts. Patients
with AML and ALL were in first or subsequent complete
remission at the time of enrollment.
All patients received human leukocyte antigen–
matched unrelated products (mobilized peripheral blood
[PB] = 80%; bone marrow [BM] = 20%) and received
myeloablative conditioning with one of three possible
regimens:
• cyclophosphamide and total body irradiation (Cy/
TBI): 27%
• busulfan and cyclophosphamide: 33%
• busulfan and fludarabine: 40%
Standard treatment included tacrolimus one day before
alloHCT and methotrexate prophylaxis on days one, three,
six, and 11 post-alloHCT. Patients were randomized to
receive either placebo (n=128) or ATLG 20 mg/kg daily for
the three days prior to alloHCT (n=126).
“Treatment arms were balanced with respect to age,
diagnosis, remission status, cytogenetics, graft source (PB
or BM), cytomegalovirus serostatus, and conditioning
regimen,” Dr. Soiffer and authors noted.
After a median follow-up of 745 days (range = 61-
1,425 days), investigators found no significant difference
in rates of moderate-to-severe cGVHD-free survival
between ATLG- and placebo-treated patients (48% vs.
44%, respectively; p=0.57) or the combined endpoint of
GVHD- and relapse-free survival (38% vs. 40%, respec-
tively; p=0.85).
Patients who received ATLG also appeared to have sig-
nificantly worse survival than those who received placebo:
PAGE 4
Opening the possibilities for your
younger patients
Proven efficacy and safety in previously treated children
with prophylaxis using as few as 2 infusions per week 1
LEOPOLD Kids Trial—Part A 1
Study description
Multinational, open-label, prospective trial evaluating
the pharmacokinetics, efficacy, safety, and perioperative
management of bleeding with KOVALTRY ®
Previously treated male patients aged 0 to <6 years (n=25)
and aged 6 to 12 years (n=26) with severe hemophilia A
(<1% FVIII) (n=51) studied for 6 months
Dosing regimens were determined by the investigators to
meet individual patients’ needs
Dosing
Primary endpoint
Pharmacokinetics
2x/week prophylaxis: 25-50 IU/kg
3x/week or every-other-day (EOD) prophylaxis: 25-50 IU/kg
Regimen 0 to <6 years 6 to 12 years
2x/week 36% (n=9) 50% (n=13)
3x/week or EOD 64% (n=16) 50% (n=13)
Annualized number of total bleeds measured during routine
prophylaxis, within 48 hours of previous prophylaxis treatment
After a single 50 IU/kg dose of KOVALTRY ® , the demonstrated
half-life [mean ± standard deviation (SD)], using the chromogenic
assay, in 18 previously treated patients 0 to 12 years of age was:
0 to <6 years
(n=7)
12.1 ± 2.7 hours
6 to <12 years
(n=10)
12.0 ± 2.1 hours
SELECTED IMPORTANT SAFETY INFORMATION
Hemophilic patients with cardiovascular risk factors or diseases may be at the same risk to
develop cardiovascular events as non-hemophilic patients when clotting has been normalized by
treatment with Factor VIII.
Please see additional Important Safety Information on following pages.
For additional important risk and use information, please see Brief Summary on following pages.
T:1
S: