Alexion to screen for paroxysmal nocturnal hemoglobinuria in patients with bone marrow failure disorders

By E. Janene Geiss

Philadelphia, June 16 - Alexion Pharmaceuticals Inc. said Friday that it would begin a multi-center study to examine the frequency of paroxysmal nocturnal hemogobinuria (PNH) in patients with aplastic anemia, myelodysplastic syndrome and other bone marrow failure disorders.

Research suggests that many patients with PNH go undiagnosed for years and that many patients have PNH in association with bone marrow failure disorders, according to a company news release.

Although the hemolytic blood disorder PNH is associated with bone marrow disorders, it is often misdiagnosed or undetected in these patients, officials said.

Current published guidelines recommend that patients with aplastic anemia, refractory anemia-myelodysplastic syndromes and patients with any one of the following - venous thrombosis involving unusual sites, hemoglobin in the urine, intravascular hemolysis without antibodies, or episodic abdominal or swallowing pain and hemolysis - should be screened for PNH.

The test for PNH requires only a small sample of the patient's blood from a vein in the arm.

The announcement of the study coincides with the launch of an Alexion-sponsored web site, PNHSource.com, which will serve as the first PNH-specific, online informational resource for physicians, patients and caregivers, officials said.

The study expects to engage about 200 hematologists to enroll an estimated 2,000 patients.

Patients with any evidence of aplastic anemia, myelodysplastic syndrome or other bone marrow failure disorders will be asked to give informed consent to enter into the study's screening phase.

The trial will mark the first systematic effort to determine the presence of the PNH blood cell clone in patients with aplastic anemia, myelodysplastic syndrome or other bone marrow failure disorders.

Enrollment is expected to begin this summer in the United States and is then expected to expand to Europe and Asia.

PNH is a rare, severe anemia that is associated with significant morbidity and early mortality. Based upon scientific investigations and presentations of the prevalence of patients diagnosed with abnormal PNH cells in their blood, it is estimated that about 8,000 to 10,000 people in North America and Western Europe suffer from PNH.

It is caused by a hematopoietic stem cell mutation resulting in a deficiency of CD59, a complement inhibitor protein that blocks the formation of the terminal complement complex on the blood cell surface and prevents hemolysis.

Alexion is a Cheshire, Conn., biotechnology company.

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