Paroxysmal Nocturnal Hemoglobinuria (PNH)

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Treatment Options

PNH treatment depends on the severity of the disease. In some cases, a patient will have few or no symptoms, and close monitoring may be all that is required.

PNH can be treated with monoclonal antibody therapy, which interrupts the activation of complement in the bloodstream, or supportive treatment that includes transfusions for anemia symptoms. Treatment for blood clots includes medications that prevent clotting.

Bone Marrow Transplantation

The only curative treatment for marrow failure associated with PNH is a bone marrow transplant. Fred Hutchinson Cancer Research Center pioneered the use of bone marrow transplantation as a life-saving treatment for leukemia over 40 years ago. The Hutchinson Transplant Program at SCCA, which covers pediatric patients as well, was ranked first in outcomes in a four-year study conducted by the National Marrow Donor Program (NMDP) that measured one-year survival rates of patients among 119 transplant centers in the United States.