Publication Date:
2005-11-16
Description:
In hemolytic paroxysmal nocturnal hemoglobinuria (PNH) patients, hemoglobin levels are typically maintained by transfusions, without which severe anemia can occur. The terminal complement inhibitor eculizumab was previously shown to effectively control intravascular hemolysis and the need for transfusions in PNH patients. We now report on compassionate use of eculizumab in a severely anemic Jehovah’s Witness PNH patient. RF is a 61 year old Hispanic woman with a 10 year history of PNH and s/p nephrectomy. In Jan 2004 she was hospitalized for a severe hemolytic paroxysm with jaundice, weakness, lower abdominal pain, vomiting, hemoglobinuria, LDH of 15,000 IU/L, hemoglobin (Hgb) of 5–6 g/dL, and 40–50,000/mm3 platelets. Flow cytometry showed 41% RBC and 70% granulocyte PNH clone sizes. To her prednisone (80 mg qd), Procrit 40,000 U thrice per week and coumadin 2.5 mg qd were added. In Feb, she improved somewhat after high dose IVIG but remained icteric and had a bed-chair existence. In mid-March, her Hgb was 5.1 g/dL, LDH 5000 IU/L, and haptoglobin
Print ISSN:
0006-4971
Electronic ISSN:
1528-0020
Topics:
Biology
,
Medicine
Permalink