A Common Bond

BMT patients are two of few known living survivors of Wolman disease

Zuleika Rojas White and Rebecca Owadeyah have something in common beyond their both having a twin sister and love for books. They underwent blood and marrow transplants (BMTs) at University of Minnesota Children’s Hospital, Fairview to treat Wolman disease and are two of only a few known living survivors of the disease in the world.

Wolman disease, also known as acid lipase deficiency, is an inherited metabolic storage disorder that causes accumulation of cholesteryl esters, normally a transport form of cholesterol, and triglycerides, a chemical form in which fats exist in the body, which build up and cause damage in the cells and tissues. Unless treated with a BMT, the disease is fatal by the time a child turns one.

"Currently, the only known treatment for Wolman is blood and marrow transplantation," says Paul Orchard, MD, associate professor of pediatrics in the Division of Hematology-Oncology and BMT and medical director of the Inherited Metabolic and Storage Disease BMT Program at the University of Minnesota.

Zuleika, who received a BMT in 1996, and Rebecca, who received a BMT in 2003, were treated by the late William Krivit, MD, who helped develop the pediatric BMT. He pioneered the research and treatment of many childhood metabolic storage diseases, including Wolman.

Wolman is rare and data are limited about long-term results of BMT treatment for the disease. "We continue to gather data for rare metabolic diseases such as Wolman," says Orchard. "Our focus is to provide the highest-quality health care through research and delivery of new knowledge for the care for children."

The BMT program has treated more Wolman patients than any other BMT program in the world. Zule and Rebecca have never met, but share a connection much deeper than having the same disease and treatment—they share the gift of life they received through transplantation.