Diagnosis and management of PNH: Review and recommendations from a Belgian expert panel

Autor:	Nancy Boeckx, Bernard Chatelain, Bérangère Devalet, Stef Meers, Timothy Devos, André Gothot, Christian Chatelain, Dries Deeren
Rok vydání:	2018
Předmět:	medicine.medical_specialty Quality Assurance Health Care haematopoietic stem cell transplantation Hemoglobinuria Paroxysmal Disease paroxysmal nocturnal haemoglobinuria registry 03 medical and health sciences Complement inhibitor 0302 clinical medicine Quality of life Belgium Risk Factors hemic and lymphatic diseases medicine Humans Registries Intensive care medicine Expert Testimony thrombosis Cause of death business.industry Diagnostic Tests Routine flow cytometry Disease Management Hematology General Medicine Eculizumab Haemolysis medicine.disease Thrombosis 030220 oncology & carcinogenesis eculizumab haemolysis Paroxysmal nocturnal haemoglobinuria Symptom Assessment business 030215 immunology medicine.drug Follow-Up Studies
Zdroj:	European journal of haematology. 101(6)
ISSN:	1600-0609
Popis:	Despite its considerable morbidity and mortality, paroxysmal nocturnal haemoglobinuria (PNH) is still underdiagnosed. Patients with PNH can suffer from cardiovascular, gastrointestinal, neurological or haematological symptoms and refer to several specialists. The aim of this paper is to review the diagnosis and the management of PNH patients, with the primary focus on identifying high-risk groups. Additionally, the implementation and prognostic value of the defined high-risk groups will be commented on and the management of PNH patients is discussed from a Belgian perspective. Finally, based on the available data, recommendations are provided. Eculizumab is a potent C5 complement inhibitor and reduces intravascular haemolysis and thrombosis in PNH patients and improves their quality of life. As thrombosis is the main cause of death in PNH patients, identifying high-risk PNH patients in need of therapy is essential. Currently, novel complement inhibitors are in development and the first data seem promising. Another challenge in PNH is to identify new markers to assess the thrombotic risk to achieve a better risk-based prophylactic anti-thrombotic management. Finally, because of the low prevalence of the disease, PNH patients should be included in the prospective PNH registry, which will offer new insights on the natural course of the disease and the impact of treatment of PNH. ispartof: EUROPEAN JOURNAL OF HAEMATOLOGY vol:101 issue:6 pages:737-749 ispartof: location:England status: published
Databáze:	OpenAIRE
Externí odkaz:	https://explore.openaire.eu/search/publication?articleId=doi_dedup___::002b3d8e4c6aadc616132810fd9c8d29 https://pubmed.ncbi.nlm.nih.gov/30171728 Zobrazit plný text záznamu Plný text ve formátu PDF Plný text ve formátu HTML
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