CC BY-NC-ND 4.0 · TH Open 2023; 07(02): e110-e116
DOI: 10.1055/s-0043-1768464
Original Article

Design of a Real-World Observational Study in Previously Untreated and Minimally Treated Hemophilia A Patients: Protect-NOW

Johannes Oldenburg
1   Institute of Experimental Haematology and Transfusion Medicine, University Clinic Bonn, Bonn, Germany
Susan Halimeh
2   Coagulation Centre Rhein-Ruhr, Duisburg, Germany
Georgina W. Hall
3   Oxford Haemophilia and Thrombosis Comprehensive Care Centre, Oxford University Hospital NHS Foundation Trust, Children's Hospital Oxford, Oxford, United Kingdom
Robert Klamroth
4   Department of Haemophilia and Haemostasis, Vivantes Hospital Friedrichshain, Berlin, Germany
Pascual Marco Vera
5   Department of Haematology, University General Hospital of Alicante, Alicante, Spain
Martina Jansen
6   Octapharma Pharmazeutika Produktionsges m.b.H., Vienna, Austria
Mary Mathias
7   Haemophilia Comprehensive Care Centre, Great Ormond Street Hospital for Children, NHS Foundation Trust, London, United Kingdom
› Author Affiliations
Funding The Protect-NOW study is funded by Octapharma AG, Lachen, Switzerland. Medical writing assistance was provided by nspm ltd (Meggen, Switzerland) and funded by Octapharma AG.


Background The efficacy, safety, and immunogenicity of each of Octapharma's factor VIII (FVIII) products, Nuwiq, octanate, and wilate, have been investigated in previously untreated patients (PUPs) with severe hemophilia A in prospective clinical trials. The aim of the Protect-NOW study is to evaluate the effectiveness, safety, and utilization patterns of Nuwiq, octanate, and wilate in PUPs and minimally treated patients (MTPs; <5 exposure days [EDs] to FVIII concentrates or other blood products containing FVIII) with severe hemophilia A in a real-world setting. Real-world data provide valuable information that complement data obtained from interventional clinical trials.

Methods Protect-NOW ( identifier: NCT03695978; ISRCTN identifier: 11492145) is a real-world study in PUPs and MTPs treated with either the human cell line-derived recombinant FVIII Nuwiq (simoctocog alfa) or a plasma-derived FVIII concentrate containing von Willebrand factor (octanate or wilate). It is a prospective and (partly) retrospective, observational, international, noncontrolled, noninterventional study. A total of 140 PUPs and MTPs with severe hemophilia A will be enrolled across around 50 specialized centers worldwide and followed for either 100 EDs or a maximum period of 3 years from ED1. The primary objectives are to assess effectiveness in the prevention and treatment of bleeding episodes and overall safety, including inhibitor development. The secondary objectives are to assess utilization patterns (including dosage and frequency of administration) and the effectiveness in surgical prophylaxis.

Conclusions The Protect-NOW study will provide information on the treatment of PUPs and MTPs in routine clinical practice, which will help guide clinical decision making for treating these patients in the future.

Publication History

Received: 03 January 2023

Accepted: 24 March 2023

Article published online:
10 May 2023

© 2023. The Author(s). This is an open access article published by Thieme under the terms of the Creative Commons Attribution-NonDerivative-NonCommercial License, permitting copying and reproduction so long as the original work is given appropriate credit. Contents may not be used for commercial purposes, or adapted, remixed, transformed or built upon. (

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