Paroxysmal nocturnal hemoglobinuria

Common Name(s)

Paroxysmal nocturnal hemoglobinuria

Paroxysmal nocturnal hemoglobinuria (PNH) is an acquired disorder that leads to the premature death and impaired production of blood cells. It can occur at any age, but is usually diagnosed in young adulthood. People with PNH have recurring episodes of symptoms due to hemolysis, which may be triggered by stresses on the body such as infections or physical exertion. This results in a deficiency of various types of blood cells and can cause signs and symptoms such as fatigue, weakness, abnormally pale skin (pallor), shortness of breath, and an increased heart rate. People with PNH may also be prone to infections and abnormal blood clotting (thrombosis) or hemorrhage, and are at increased risk of developing leukemia. It is caused by acquired, rather than inherited, mutations in the PIGA gene; the condition is not passed down to children of affected individuals. Sometimes, people who have been treated for aplastic anemia may develop PNH. The treatment of PNH is largely based on symptoms; stem cell transplantation is typically reserved for severe cases of PNH with aplastic anemia or those whose develop leukemia.
 

Advocacy and Support Organizations

 

Condition Specific Organizations

Following organizations serve the condition "Paroxysmal nocturnal hemoglobinuria" for support, advocacy or research.

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Aplastic Anemia & MDS International Foundation

The Aplastic Anemia & MDS International Foundation (AA&MDSIF) is the world's leading nonprofit health organization dedicated to supporting patients and families living with aplastic anemia, myelodysplastic syndromes (MDS), paroxysmal nocturnal hemoglobinuria (PNH), and related bone marrow failure diseases. AA&MDSIF provides answers, support and hope to thousands of patients and their families around the world.

Last Updated: 29 Apr 2014

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Advocacy and Support Organizations

 

Condition Specific Organizations

Following organizations serve the condition "Paroxysmal nocturnal hemoglobinuria" for support, advocacy or research.

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Aplastic Anemia & MDS International Foundation

The Aplastic Anemia & MDS International Foundation (AA&MDSIF) is the world's leading nonprofit health organization dedicated to supporting patients and families living with aplastic anemia, myelodysplastic syndromes (MDS), paroxysmal nocturnal hemoglobinuria (PNH), and related bone marrow failure diseases. AA&MDSIF provides answers, support and hope to thousands of patients and their families around the world.

http://www.aamds.org/

Last Updated: 29 Apr 2014

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Scientific Literature

Articles from the PubMed Database

Research articles describe the outcome of a single study. They are the published results of original research.
The terms "Paroxysmal nocturnal hemoglobinuria" returned 390 free, full-text research articles on human participants. First 3 results:

Severe Infection of Pseudomonas aeruginosa during Eculizumab Therapy for Paroxysmal Nocturnal Hemoglobinuria.
 

Author(s): Toru Kawakami, Hideyuki Nakazawa, Yukifumi Kurasawa, Hitoshi Sakai, Sayaka Nishina, Noriko Senoo, Yasushi Senoo, Fumihiro Ishida

Journal: Intern. Med.. 2018 Jan;57(1):127-130.

 

Eculizumab is the complement inhibitor administered to ameliorate intravascular hemolysis in paroxysmal nocturnal hemoglobinuria. Whether or not the inhibitory mechanism may also increase the susceptibility to non-Neisserial infection is unclear. A 73-year old woman presented with ...

Last Updated: 31 Dec 1969

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Distinct clinical characteristics of paroxysmal nocturnal hemoglobinuria in patients in Southern Taiwan: A multicenter investigation.
 

Author(s): Hui-Ching Wang, Ching-Yuan Kuo, I-Ting Liu, Tsai-Yun Chen, Yu-Hsiang Chang, Shyh-Jer Lin, Shih-Feng Cho, Yi-Chang Liu, Ta-Chih Liu, Sheng-Fung Lin, Chao-Sung Chang

Journal: Kaohsiung J. Med. Sci.. 2017 Aug;33(8):405-410.

 

Paroxysmal nocturnal hemoglobinuria (PNH) is an extremely rare acquired disorder. The aim of this study was to investigate the demographics, clinical manifestations, and outcomes of PNH patients in southern Taiwan. Data on PNH patients diagnosed over a 30-year period (1985-2015) were ...

Last Updated: 31 Dec 1969

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T Cell Transcriptomes from Paroxysmal Nocturnal Hemoglobinuria Patients Reveal Novel Signaling Pathways.
 

Author(s): Kohei Hosokawa, Sachiko Kajigaya, Keyvan Keyvanfar, Wangmin Qiao, Yanling Xie, Danielle M Townsley, Xingmin Feng, Neal S Young

Journal: J. Immunol.. 2017 07;199(2):477-488.

 

Paroxysmal nocturnal hemoglobinuria (PNH) is a rare acquired disorder originating from hematopoietic stem cells and is a life-threating disease characterized by intravascular hemolysis, bone marrow (BM) failure, and venous thrombosis. The etiology of PNH is a somatic mutation in the ...

Last Updated: 31 Dec 1969

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Reviews from the PubMed Database

Review articles summarize what is currently known about a disease. They discuss research previously published by others.
The terms "Paroxysmal nocturnal hemoglobinuria" returned 35 free, full-text review articles on human participants. First 3 results:

A Hispanic female patient with heartburn: A rare presentation of Paroxysmal Nocturnal Hemoglobinuria.
 

Author(s): Luis A Figueroa-Jiménez, Amy Lee González-Márquez, Ricardo Alicea-Guevara, Mónica Santiago-Casiano, Maryknoll de la Paz-López, Luis Negrón-Garcia, Luis Báez-Diaz, William Cáceres-Pérkins

Journal: Bol Asoc Med P R. ;107(3):9-12.

 

Paroxysmal nocturnal hemoglobinuria (PNH) is a non-malignant, acquired clonal hematopoietic stem cell disease that can present with bone marrow failure, hemolytic anemia, smooth muscle dystonias, and thrombosis. We present a case of a 32 year-old-female, G2P2A0 with no past medical ...

Last Updated: 31 Dec 1969

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Paroxysmal nocturnal hemoglobinuria: a complement-mediated hemolytic anemia.
 

Author(s): Amy E DeZern, Robert A Brodsky

Journal: Hematol. Oncol. Clin. North Am.. 2015 Jun;29(3):479-94.

 

Paroxysmal nocturnal hemoglobinuria is manifests with a chronic hemolytic anemia from uncontrolled complement activation, a propensity for thrombosis and marrow failure. The hemolysis is largely mediated by the alternative pathway of complement. Clinical manifestations result from ...

Last Updated: 31 Dec 1969

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Cerebral venous thrombosis in paroxysmal nocturnal hemoglobinuria: a series of 15 cases and review of the literature.
 

Author(s): Elodie Meppiel, Isabelle Crassard, Régis Peffault de Latour, Sophie de Guibert, Louis Terriou, Hugues Chabriat, Gérard Socié, Marie-Germaine Bousser

Journal: Medicine (Baltimore). 2015 Jan;94(1):e362.

 

Paroxysmal nocturnal hemoglobinuria (PNH) is a rare acquired disorder of hematopoietic stem cells characterized by hemolytic anemia, marrow failure, and a high incidence of life-threatening venous thrombosis. Cerebral venous system is the second most frequent location of thrombosis ...

Last Updated: 31 Dec 1969

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Clinical Trial Information This information is provided by ClinicalTrials.gov

Paroxysmal Nocturnal Hemoglobinuria (PNH) Registry
 

Status: Recruiting

Condition Summary: Paroxysmal Nocturnal Hemoglobinuria

 

Last Updated: 22 Feb 2018

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A Treatment Study of ACH-0144471 in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) With Inadequate Response to Eculizumab
 

Status: Recruiting

Condition Summary: Paroxysmal Nocturnal Hemoglobinuria (PNH)

 

Last Updated: 19 Jun 2018

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Study of Safety, Efficacy, Tolerability, Pharmacokinetics and Pharmacodynamics of LNP023 in in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)
 

Status: Recruiting

Condition Summary: Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)

 

Last Updated: 30 Jul 2018

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