322.

DISORDERS OF COAGULATION OR FIBRINOLYSIS | NOVEMBER 13, 2019

Real-World Effectiveness and Safety of BAY 94-9027 (Damoctocog

Alfa Pegol) in Previously Treated Patients with Hemophilia A
(HEM-POWR): Online Patient Portal and LIFE-ACTIVE Sub-Study
1 *,2 *,3 4
Johannes Oldenburg, María Teresa Alvarez Román, Giancarlo Castaman, Maissaa Janbain, MD,
5 6 *,7 *,8 *,9
Tadashi Matsushita, Karina Meijer, Sabine Friedl, Martin Sanabria, Mark Reding
1
University Clinic Bonn, Bonn, Germany
2
Hospital Universitario La Paz, Madrid, Spain
3
Careggi University Hospital, Firenze, Italy
4
Tulane University Health Sciences Center, New Orleans, LA
5
Nagoya University Hospital, Nagoya, Japan
6
University Medical Center Groningen, Groningen, Netherlands
7
Bayer AG, Berlin, Germany
8
Bayer, Basel, Switzerland
9
University of Minnesota Medical Center, Minneapolis,

bloodjournal Blood blood (2019) 134 (Supplement_1) : 4943.

http://doi.org/10.1182/blood-2019-128140

Background and Rationale:

BAY 94-9027 (damoctocog alfa pegol) is a site-specifically PEGylated B-domain deleted recombinant
factor VIII (FVIII) with an extended half-life, approved for prophylaxis or treatment of bleeds in previously

treated patients (PTPs) aged ≥12 with hemophilia A. The efficacy and safety of BAY 94-9027 was
demonstrated in two phase II/III clinical studies in PTPs with severe hemophilia A, however, real-world
data are still being gathered. The aim of the HEM-POWR study is to assess the effectiveness and long-
term safety of BAY 94-9027 in the real-world clinical setting. Patients will be introduced to an online
patient portal that provides study information as well as access to eDiaries and electronic patient-reported
outcomes (ePROs) to patients to facilitate retention over the duration of the study. Patients will also be
given the opportunity to participate in LIFE-ACTIVE, a sub-study analyzing the relationship between the
patients' regular daily activity and the efficacy parameters collected during HEM-POWR.
Here we present the features of the patient portal and describe the LIFE-ACTIVE sub-study design.

Study Design and Methods:

HEM-POWR (NCT03932201) is a multinational, multicenter, non-interventional, open-label, prospective,

phase IV, cohort study. It aims to enroll ≥200 PTPs with hemophilia A receiving BAY 94-9027 (on-
demand, prophylaxis, or intermittent prophylaxis [as per local label]). Key exclusion criteria are presence

or history of FVIII inhibitor (≥0.6 Bethesda units), diagnosis of any bleeding or coagulation disorder other
than hemophilia A, or treatment with immune tolerance induction at enrollment. The primary objective
of HEM-POWR is to assess the effectiveness of prophylaxis with BAY 94-9027 in the real-world setting
through the collection of total bleeding events and analysis of annualized bleeding rate. Secondary
objectives include long-term safety, joint health, location and number of target joints, hemostasis during
surgery and PROs.

Patient enrollment, adherence and retention can be difficult in observational hemophilia studies. The
patient portal for this study aims to overcome these challenges by providing study- and product-related
information. It also aims to lessen the burden for patients in the study by providing e-solutions to collect
their study data, including the ability to complete the study diary, and PRO measures online. The portal
also includes videos explaining the study and study procedures, and is country-customized with links to
relevant websites.

Patients participating in LIFE-ACTIVE will be asked to wear an ActiGraph CP Insight activity-tracking

smart watch continually for four 30-day periods, at their initial visit and then at months 12, 24 and 36.
Measurements recorded will include physical activity intensity and duration, general mobility, and sleep
quality and duration. All data will be transferred to a secure, cloud-based system and patients will not be
aware of the values measured by the device.

Participating countries include, but may not be limited to Austria, Belgium/Luxemburg, Canada, Colombia,
Finland, Germany, Greece, Italy, Japan, Netherlands, Portugal, Saudi Arabia, Denmark, Norway,
Sweden, Slovenia, Spain, Switzerland, Taiwan, and USA. The study will run from 2019 until 2025, with an

observation period of ≥60 months.

Disclosures
Oldenburg: Octapharma: Consultancy, Research Funding, Speakers Bureau; NovoNordisk:
Consultancy, Honoraria, Research Funding; Bayer: Consultancy, Research Funding, Speakers Bureau;
Grifols: Consultancy, Speakers Bureau; Pfizer: Consultancy, Speakers Bureau; Roche: Consultancy,
Speakers Bureau; CSL Behring: Consultancy, Research Funding, Speakers Bureau; Takeda (Shire):
Consultancy, Research Funding, Speakers Bureau; Chugai: Consultancy, Speakers Bureau; Biotest:
Consultancy, Research Funding, Speakers Bureau; Swedish Orphan Biovitrum: Consultancy, Speakers
Bureau. Alvarez Román: CSL Behring: Speakers Bureau; Amgen: Speakers Bureau; Novartis:
Speakers Bureau; Sobi: Speakers Bureau; Bayer: Speakers Bureau; Novo Nordisk: Speakers Bureau;
Roche: Speakers Bureau; Shire (Takeda): Research Funding, Speakers Bureau. Castaman: Shire:
Speakers Bureau; Uniqure Kedrion: Speakers Bureau; Pfizer: Research Funding; CSL Behring:
Research Funding, Speakers Bureau; Bayer: Speakers Bureau; Novo Nordisk: Speakers Bureau; Roche:
Consultancy, Honoraria, Speakers Bureau; Sobi: Research Funding, Speakers Bureau. Janbain: Shire
(Vonvendi): Speakers Bureau; Genentech: Consultancy, Honoraria; Bayer: Consultancy, Honoraria;
CSL Behring: Consultancy, Honoraria; Shire: Consultancy, Honoraria; HTRS-MRA (Bioverativ Sanofi):
Research Funding. Matsushita: uniQure: Consultancy, Honoraria; CSL: Consultancy, Honoraria;
Bioverative: Research Funding; Pfizer: Consultancy, Honoraria; KM biologists: Consultancy, Honoraria,
Research Funding; Novo Nordisk: Consultancy, Honoraria. Meijer: Sanquin: Research Funding; Pfizer,
Sanquin, Uniqure: Research Funding; Uniqure, BMS, Aspen, Boehringer Ingelheim, Sanquin, Bayer:
Consultancy, Honoraria; Bayer: Research Funding. Sanabria: Bayer: Employment. Reding: Novo
Nordisk: Consultancy, Honoraria, Speakers Bureau; Bayer: Consultancy, Honoraria, Research Funding,
Speakers Bureau; Sanofi Genzyme: Consultancy, Honoraria, Speakers Bureau; Biomarin: Research
Funding; Takeda: Consultancy, Honoraria, Speakers Bureau.

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

© 2019 by the American Society of Hematology