Beyond patient benefit: clinical development in hemophilia

Author:

Auerswald Günter1,Šalek Silva Zupančić2,Benson Gary3,Elezović Ivo4,Lambert Thierry5,Morfini Massimo6,Pasi John7,Remor Eduardo8,Santagostino Elena9,Salaj Peter10,Ljung Rolf11

Affiliation:

1. Klinikum Bremen-Mitte, Professor Hess Children’s Hospital, Bremen, Germany

2. National Haemophilia and Thrombophilia Centre, Division of Haematology, Department of Internal Medicine, University Hospital Centre Rebro, Zagreb, Croatia

3. Northern Ireland Haemophilia Comprehensive Care Centre, Belfast, Northern Ireland, UK

4. Institute of HaematologyClinical Centre of Serbia, School of Medicine, University of Belgrade, Belgrade, Serbia

5. Hemophilic Care CenterBicêtre AP-HP Hospital and Faculté de Médecine Paris XI, Paris, France

6. Agency for Hemophilia – Reference Center for Inherited Bleeding Disorders of Tuscany, Department of Emergency and Reception, Azienda Ospedaliero Universitaria Careggi, Florence, Italy

7. Haemophilia Centre, Barts and the London School of Medicine and Dentistry, London, UK

8. Department of Psychobiology and HealthFaculty of Psychology, Universidad Autónoma de Madrid, Madrid, Spain

9. IRCCS Cà Granda Foundation, Maggiore Hospital Policlinico and University of Milan, Italy

10. Institute of Hematology and Blood Transfusion, Prague, Czech Republic

11. Lund University, Department of Paediatrics and Malmö Centre for Thrombosis and Haemostasis, Skåne University Hospital, Malmö, Sweden

Publisher

Informa UK Limited

Subject

Hematology,Hematology

Reference45 articles.

1. Paediatric haemophilia with inhibitors: existing management options, treatment gaps and unmet needs

2. Meeting unmet needs in inhibitor patients

3. ICH Topic E 6 (R1). Guideline for Good Clinical Practice (CPMP/ICH/135/9). Available at http://www.ema.europa.eu/docs/en_GB/document_library/Scientific_guideline/2009/09/WC500002874.pdf. Accessed July 12, 2011.

4. Challenges of defining reliable clinical surrogate end points in haemophilia trials: a critical review

5. Musculoskeletal measurement tools from the International Prophylaxis Study Group (IPSG)

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