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Thromb Haemost 2015; 114(01): 35-45
DOI: 10.1160/TH14-05-0407
Coagulation and Fibrinolysis
Schattauer GmbH

Benefits of prophylaxis versus on-demand treatment in adolescents and adults with severe haemophilia A: the POTTER study

Annarita Tagliaferri
1   Regional Reference Centre for Inherited Bleeding Disorders, University Hospital of Parma, Parma, Italy
,
Giulio Feola
2   Immunohematology and Hemophilia Center, S. Luca Hospital, Vallo della Lucania, Italy
,
Angelo Claudio Molinari
3   Thrombosis and Hemostasis Unit, Giannina Gaslini Children’s Hospital, Genoa, Italy
,
Cristina Santoro
4   Hematology, Department of Cellular Biotechnology and Haematology, Sapienza University of Rome, Rome, Italy
,
Gianna Franca Rivolta
1   Regional Reference Centre for Inherited Bleeding Disorders, University Hospital of Parma, Parma, Italy
,
Dorina Bianca Cultrera
5   Hemophilia Center, Hematology, Ferrarotto Hospital, Policlinico Vittorio Emanuele University Hospital, Catania, Italy
,
Fabio Gagliano
6   Hemophilia Center, G. Di Cristina Children’s Hospital, Palermo, Italy
,
Ezio Zanon
7   Hemophilia Center, Second Chair of Internal Medicine, University of Padua, Padua, Italy
,
Maria Elisa Mancuso
3   Thrombosis and Hemostasis Unit, Giannina Gaslini Children’s Hospital, Genoa, Italy
,
Lelia Valdrè
9   Angiology and Coagulation Disease Unit, Bologna, Italy
,
Luciana Mameli
10   Thrombosis and Hemostasis Unit, SS Annunziata Hospital, Sassari, Italy
,
Susanna Amoresano
11   Bayer HealthCare, Milan, Italy
,
Prasad Mathew
12   Bayer HealthCare, Whippany, NJ, and University of New Mexico, Albuquerque, NM, USA
,
Antonio Coppola
13   Regional Reference Centre for Coagulation Disorders, Federico II University Hospital, Naples, Italy
,
for the POTTER Study Group › Author Affiliations Financial support: The study was funded by Bayer HealthCare as a phase IV postmarketing surveillance study. Editorial assistance was provided by Complete Healthcare Communications, Inc. (Chadds Ford, PA, USA) and was funded by Bayer HealthCare.
Further Information

Publication History

Received: 05 May 2014

Accepted after major revision: 01 April 2015

Publication Date:
22 November 2017 (online)

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Summary

Rigorous evidence is lacking on long-term outcomes of factor VIII (FVIII) prophylaxis initiated in adolescent or adult patients with severe haemophilia A. The prospective, open-label Prophylaxis versus On-demand Therapy Through Economic Report (POTTER) study (Clinical-Trials.gov NCT01159587) compared long-term late secondary prophylaxis (recombinant FVIII-FS 20–30 IU/kg thrice weekly) with on-demand treatment in patients aged 12 to 55 years with severe haemophilia A. The annual number of joint bleeding episodes (primary endpoint), total bleeding episodes, orthopaedic and radiologic (Pettersson) scores, health-related quality of life (HRQoL), pharmacoeconomic impact, and safety were evaluated over a > 5-year period (2004–2010). Fifty-eight patients were enrolled at 11 centres in Italy; 53 (27 prophylaxis, 26 on demand) were evaluated and stratified into 2 age subgroups (12–25 and 26–55 years). Patients receiving prophylaxis experienced a significantly lower number of joint bleeding episodes vs the on–demand group (annualised bleeding rate, 1.97 vs 16.80 and 2.46 vs 16.71 in younger and older patients, respectively; p=0.0043). Results were similar for total bleeding episodes. Prophylaxis was associated with significantly fewer target joints (p< 0.001), better orthopaedic (p=0.0019) and Pettersson (p=0.0177) scores, better HRQoL, and fewer days of everyday activities lost (p< 0.0001) but required significantly higher FVIII product consumption. The POTTER study is the first prospective, controlled trial documenting long-term benefits of late secondary prophylaxis in adolescents and adults with severe haemophilia A. The benefits of reduced bleeding frequency, improved joint status, and HRQoL may offset the higher FVIII consumption and costs.

Keywords

Bleeding - health-related quality of life - haemophilia - haemophilic arthropathy - prophylaxis

* Investigators of the POTTER Study Group are listed in the Appendix.


 

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