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Paroxysmal Nocturnal Haemoglobinuria (PNH)

A special issue of Journal of Clinical Medicine (ISSN 2077-0383). This special issue belongs to the section "Hematology".

Deadline for manuscript submissions: closed (31 March 2020) | Viewed by 7558

Special Issue Editor


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Guest Editor
Department Haematology, Leeds Teaching Hospitals NHS Trust, Leeds LS9 7TF, UK
Interests: PNH; bone marrow failure; aplastic anaemia; autoimmune haemolytic anaemia

Special Issue Information

Dear Colleagues,

PNH is an acquired disorder that can affect any age group, often targeting young adults. The disease can result in significant morbidity as well as early mortality. Over the last 15 years, the role of complement inhibition has had a significant impact on improving both quality and survival for patients with PNH. Concomitant management of the underlying bone marrow failure is often required and requires understanding of the two disease processes. While terminal complement inhibition has advanced therapy for PNH significantly, there is increasing interest in targeting the complement system proximally to eradicate the extravascular haemolysis that emerges when terminal complement therapeutics are used. This Special Issue will address the most recent and relevant scientific findings in the management of PNH.

Dr. Anita Hill
Guest Editor

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Keywords

  • Intravascular haemolysis
  • Extravascular haemolysis
  • Complement inhibition
  • Bone marrow failure
  • Eculizumab
  • Aplastic anaemia

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Published Papers (1 paper)

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Review

26 pages, 907 KiB  
Review
Complement Inhibition Therapy and Dialytic Strategies in Paroxysmal Nocturnal Hemoglobinuria: The Nephrologist’s Opinion
by Guido Gembillo, Rossella Siligato, Valeria Cernaro and Domenico Santoro
J. Clin. Med. 2020, 9(5), 1261; https://doi.org/10.3390/jcm9051261 - 26 Apr 2020
Cited by 15 | Viewed by 6907
Abstract
Paroxysmal nocturnal hemoglobinuria (PNH) is a rare clonal disease that presents an estimated incidence of 1.3 cases per million per year, with a prevalence of 15.9 cases per million. It is characterized by hemolysis, bone marrow dysfunction with peripheral blood cytopenia, hypercoagulability, thrombosis, [...] Read more.
Paroxysmal nocturnal hemoglobinuria (PNH) is a rare clonal disease that presents an estimated incidence of 1.3 cases per million per year, with a prevalence of 15.9 cases per million. It is characterized by hemolysis, bone marrow dysfunction with peripheral blood cytopenia, hypercoagulability, thrombosis, renal impairment and arterial and pulmonary hypertension. Hemolysis and subsequent hemosiderin accumulation in tubular epithelium cells induce tubular atrophy and interstitial fibrosis. The origin of PNH is the somatic mutation in the X-linked phosphatidylinositol glycan class A (PIG-A) gene located on Xp22: this condition leads to the production of clonal blood cells with a deficiency in those surface proteins that protect against the lytic action of the activated complement system. Despite the increased knowledge of this syndrome, therapies for PNH were still only experimental and symptomatic, until the introduction of the C5 complement blockade agent Eculizumab. A second generation of anti-complement agents is currently under investigation, representing future promising therapeutic strategies for patients affected by PNH. In the case of chronic hemolysis and renal iron deposition, a multidisciplinary approach should be considered to avoid or treat acute tubular injury or acute kidney injury (AKI). New promising perspectives derive from complement inhibitors and iron chelators, as well as more invasive treatments such as immunoadsorption or the use of dedicated hemodialysis filters in the presence of AKI. Full article
(This article belongs to the Special Issue Paroxysmal Nocturnal Haemoglobinuria (PNH))
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