Paroxysmal nocturnal haemoglobinuria at Oslo University Hospital 2000-2010.

Abstract

BACKGROUND:

Paroxysmal nocturnal haemoglobinuria (PNH) is a rare haematological disease characterised by chronic haemolysis, pancytopenia and venous thrombosis. The condition is attributable to a lack of control of complement attack on erythrocytes, thrombocytes and leukocytes, and can be diagnosed by means of flow cytometry. In this quality assurance study, we have reviewed information from the medical records of all patients tested for PNH using flow cytometry at our laboratory over a ten-year period.

MATERIAL AND METHOD:

In the period 2000-2010 a total of 28 patients were tested for PNH using flow cytometry at the Department of Immunology and Transfusion Medicine, Oslo University Hospital. We have reviewed the results of these examinations retrospectively together with information from medical records and transfusion data for the patients concerned.

RESULTS:

Flow cytometry identified 22 patients with PNH: four with classic disease and 18 with PNH secondary to another bone marrow disease. Five patients had atypical thrombosis. Seventeen patients received antithymocyte globulin or drug treatment; of these, six recovered from their bone marrow disease, while six died and five had a need for long-term transfusion. Five patients with life-threatening bone marrow disease underwent allogeneic stem cell transplantation, three of whom died. Six of 22 patients received eculizumab; the need for transfusion has been reduced or eliminated in three patients treated with eculizumab over a longer period.

INTERPRETATION:

Flow cytometry identified PNH in a majority of patients from whom we obtained samples. Most patients had a PNH clone secondary to bone marrow failure. Atypical thrombosis should be borne in mind as an indication for the test. Treatment with eculizumab is relevant for selected patients with PNH.

• Authors: Lise Sofie H Nissen-Meyer, Geir E Tjønnfjord, Elzbieta Golebiowska, Jens Kjeldsen-Kragh, Çiğdem Akalın Akkök
• Journal: Tidsskrift for den Norske laegeforening : tidsskrift for praktisk medicin, ny raekke (Tidsskr Nor Laegeforen) Vol. 135 Issue 11 Pg. 1039-43 (Jun 16 2015) ISSN: 0807-7096 [Electronic] Norway
• PMID: 26080779 (Publication Type: Journal Article)

Chemical References

• Antibodies, Monoclonal, Humanized
• Antilymphocyte Serum
• CD55 Antigens
• CD59 Antigens
• eculizumab

Topics

• Antibodies, Monoclonal, Humanized (therapeutic use)
• Antilymphocyte Serum (therapeutic use)
• Bone Marrow Diseases (complications, drug therapy, surgery)
• CD55 Antigens (blood)
• CD59 Antigens (blood)
• Female
• Flow Cytometry
• Hemoglobinuria, Paroxysmal (blood, diagnosis, drug therapy, etiology)
• Humans
• Male
• Norway
• Quality Assurance, Health Care
• Retrospective Studies