Description

Socie et al identified prognostic factors affecting survival in patients with paroxysmal nocturnal hemoglobinuria. These factors can help identify patients who may benefit from more aggressive management or a novel therapy. The authors are from multiple hospitals from across France.


 

Factors associated with a worse survival:

(1) development of thrombosis

(2) development of pancytopenia

(3) progression to myelodysplasia or acute leukemia

(4) diagnosis after 54 years of age

(5) need for additional treatment (with second, third or fourth line therapies)

(6) thrombocytopenia (platelet count < 150,000 per µL) at diagnosis

(7) not evolving from aplastic anemia (evolution from aplastic anemia favorable)

 

where:

• The highest odds ratios were for myelodysplasia/AML (odds ratio 19) and thrombosis (odds ratio 10). The other risk factors had odds ratios ranging form 2.1 to 5.5).

• Initial therapy ranged from none, to blood transfusions, to drugs (androgens, immunosuppression, corticosteroids, danazol) to bone marrow transplantation.

 


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