Baseline characteristics and disease burden in patients in the International Paroxysmal Nocturnal Hemoglobinuria Registry
Hubert Schrezenmeier,
Petra Muus,
Gérard Socié,
Jeffrey Szer,
Alvaro Urbano-Ispizua,
Jaroslaw P. Maciejewski,
Robert A. Brodsky,
Monica Bessler,
Yuzuru Kanakura,
Wendell Rosse,
Gus Khursigara,
Camille Bedrosian,
Peter Hillmen
Affiliations
Hubert Schrezenmeier
Institute of Clinical Transfusion Medicine and Immunogenetics, German Red Cross Blood Transfusion Service Baden-Württemberg–Hessen, and Institute of Transfusion Medicine, University of Ulm, Germany
Petra Muus
Radboud University Medical Centre, Nijmegen, The Netherlands
Gérard Socié
Hôpital Saint-Louis and Institut National de la Santé et de la Recherche Médicale, Paris, France
Jeffrey Szer
Royal Melbourne Hospital, Australia
Alvaro Urbano-Ispizua
Hospital Clinic, University of Barcelona, Institute of Research Josep Carreras, Barcelona, Spain
Jaroslaw P. Maciejewski
Taussig Cancer Center, Cleveland Clinic, Cleveland, OH, USA
Robert A. Brodsky
Johns Hopkins University School of Medicine, Baltimore, MD, USA
Monica Bessler
Department of Hematology, University of Pennsylvania School of Medicine, and Children’s Hospital of Philadelphia, PA, USA
Yuzuru Kanakura
Osaka University Graduate School of Medicine, Japan
Wendell Rosse
Duke University Medical Center, Durham, NC, USA
Gus Khursigara
Alexion Pharmaceuticals, Inc., Cheshire, CT, USA
Camille Bedrosian
Alexion Pharmaceuticals, Inc., Cheshire, CT, USA
Peter Hillmen
Department of Haematology, St James’ University Hospital, Leeds, UK
Paroxysmal nocturnal hemoglobinuria is a rare, acquired disease associated with hemolytic anemia, bone marrow failure, thrombosis, and, frequently, poor quality of life. The International PNH Registry is a worldwide, observational, non-interventional study collecting safety, effectiveness, and quality-of-life data from patients with a confirmed paroxysmal nocturnal hemoglobinuria diagnosis or detectable paroxysmal nocturnal hemoglobinuria clone, irrespective of treatment. In addition to evaluating the long-term safety and effectiveness of eculizumab in a global population, the registry aims to improve diagnosis, optimize patient management and outcomes, and enhance the understanding of the natural history of paroxysmal nocturnal hemoglobinuria. Here we report the characteristics of the first 1610 patients enrolled. Median disease duration was 4.6 years. Median granulocyte paroxysmal nocturnal hemoglobinuria clone size was 68.1% (range 0.01–100%). Overall, 16% of patients had a history of thrombotic events and 14% a history of impaired renal function. Therapies included anticoagulation (31%), immunosuppression (19%), and eculizumab (25%). Frequently reported symptoms included fatigue (80%), dyspnea (64%), hemoglobinuria (62%), abdominal pain (44%), and chest pain (33%). Patients suffered from poor quality of life; 23% of patients had been hospitalized due to paroxysmal nocturnal hemoglobinuria-related complications and 17% stated that paroxysmal nocturnal hemoglobinuria was the reason they were not working or were working less. This international registry will provide an ongoing, valuable resource to further the clinical understanding of paroxysmal nocturnal hemoglobinuria.
