322.

DISORDERS OF COAGULATION OR FIBRINOLYSIS: POSTER I | NOVEMBER 29, 2018

Impact of Treatment Regimen with Moroctocog Alfa

(AF-CC) on Bleeding Frequency in Pediatric Aged
Subjects with Moderately Severe to Severe Hemophilia A
1 2 *,3
Mark P. Smith, MD FRACP, FRCPA, Jeremy Rupon, MD, Yasser Wali, MD,
*,4 *,2 *,2 2
Hala Remawi, MD, Joan Korth-Bradley, PharmD, PhD, Lynne Smith, MBA, Pablo Rendo, MD
1
Canterbury Health Laboratories, Christchurch, NZL
2
Pfizer, Collegeville, PA
3
Sultan Qaboos University, Muscat, Oman
4
King Abdullah University Hospital,, Amman, Jordan

Blood (2018) 132 (Supplement 1) : 1205.

http://doi.org/10.1182/blood-2018-99-111891

Abstract
Moroctocog alfa (AF-CC) is a B-domain deleted recombinant factor VIII product for the treatment of
patients with hemophilia A. It has previously been studied in pediatric aged hemophilia patients, however
direct comparisons between treatment regimens have not been evaluated. Importantly, as prophylaxis
has been shown to be superior to on-demand treatment, comparisons between different prophylaxis
regimens are needed. Reducing the frequency of infusions without sacrificing efficacy may reduce the
burden of hemophilia management. This study was an open label, multicenter, randomized crossover
study of two routine prophylaxis (RP) regimens of moroctocog alfa (AF-CC) compared to on-demand
(OD) therapy in 2 cohorts of children with hemophilia A. The primary objective was to demonstrate that
prophylaxis reduces ABR relative to OD therapy. A secondary objective was to compare the efficacy of
two different RP regimens. Additional objectives focused on FVIII recovery and safety.

Study Design: The study was open to previously treated patients (≥20 exposure days (ED) to any FVIII
replacement product) with moderately severe to severe hemophilia A (<2% FVIII) aged 6 months to <16
years. The study enrolled 2 cohorts of subjects who were treated in two segments. The first cohort started
with OD treatment for 6 months (segment 1) followed by 1 year of RP of 25 international units (IU)/kg
every other day (segment 2). Subjects in the second cohort were randomized to one of 2 RP regimens
for 1 year (segment 1) and then crossed over to a second RP regimen for an additional year (segment
2). The RP regimens were high frequency (HF) (25 IU/kg every other day) and low frequency (LF) (45 IU/
kg twice weekly). Recovery assessment after administration of 50 IU/kg of moroctocog alfa (AF-CC) was
optional.

Results: A total of 51 subjects were enrolled in the study: 9 subjects (age 2.4-5.9 yr; median 4.9 yr) in the
OD group, 18 subjects (age 1.1-12.7 yr; median 4.7 yr) received LF followed by HF, and 24 subjects (age
1.2 to 9.6 yr; median 4.6 yr) received HF followed by LF. The mean (standard deviation [SD]) ABR for the
first cohort (n=9) during the OD segment was 47.0(32.2) and during the RP segment (HF) was 1.5(2.2). In
those subjects from cohort 2 that completed both segments (n=38), the mean (standard deviation) ABR
for HF was 2.2(4.1) and for LF was 3.3(5.3). Based on a prospectively defined equivalence limit of (-3,
3) bleeds per year, equivalence between these two regimens was demonstrated as the 90% CI for the
difference, (0.03, 2.22) fell within this range. The mean (SD) recovery in 6 children aged 3.7 to 5.8 yrs
was 1.44(0.61) IU/dL/IU/kg. Three subjects tested positive for FVIII inhibitors, however 2 were considered
to be false positives for an overall rate of 2%. There were no new safety signals that emerged during this
study.

Conclusion: This study met its primary objective of demonstrating that RP reduces the ABR compared to
OD. Notably, LF prophylaxis with moroctocog alfa (AF-CC) is as efficacious as a HF prophylaxis regimen.
This has important implications as less frequent infusions can improve adherence and the quality of life
for patients with hemophilia. Additionally, recovery was as expected in this pediatric population and no
new safety signals emerged. Moroctocog alfa (AF-CC) is safe and efficacious for routine prophylaxis
in pediatric patients with hemophilia A and a low frequency (twice weekly) prophylaxis regimen is as
efficacious as every other day prophylaxis.

Disclosures

Rupon: Pfizer: Employment. Korth-Bradley: Pfizer Inc.: Employment. Smith: Pfizer: Employment.
Rendo: Pfizer: Employment.

Author notes
* Asterisk with author names denotes non-ASH members.

© 2018 by the American Society of Hematology