Hairy Cell Leukemia With Kidney Failure: Targeted Therapy Opens the Door to Transplant

A 72-year-old man needed a kidney transplant. But during routine testing, doctors discovered a rare blood cancer that threatened his chances of receiving one.

Years of high blood pressure had left his kidneys failing, leaving him with two options: dialysis or a transplant. Then came the diagnosis of hairy cell leukemia, a cancer affecting approximately 1,000 to 1,500 people each year in the United States. With active cancer, he could no longer qualify for a kidney transplant until the disease was under control.

For Dr. Jacqueline Barrientos, chief of hematologic malignancies at Mount Sinai Medical Center in Miami Beach, treating the leukemia presented another challenge: the standard chemotherapy drugs could further damage his already failing kidneys.

After consulting colleagues in the national Hairy Cell Leukemia Consortium, she chose a different approach. Like about 95% of patients with the disease, he carried a BRAF mutation. That made him a candidate for vemurafenib, a targeted oral drug that blocks the pathway driving the cancer. She started him at half the usual dose, then added the antibody rituximab.

The treatment worked. His kidney function declined during therapy, and he began dialysis once a week. Still, two months later, his bone marrow showed no sign of leukemia.

Being cancer-free was not enough. Most transplant centers told him he would need to stay in remission for five years before they would operate. At his age, that wait could cost him his chance at a new kidney. His care team argued that years of dialysis posed a greater risk than the leukemia itself. It took two years to find a center willing to perform the surgery.

Help came from an unexpected place. The patient was a prominent member of his Jewish community. When word spread that he needed a kidney, a young, healthy man he had never met stepped forward to donate one.

Two years after the transplant, he remains in remission. His new kidney is working well. His blood counts, once so low that he required multiple transfusions, are now normal.

“It can be done,” Barrientos said.When a 72-year-old man began testing for a kidney transplant, his doctors found a second problem: hairy cell leukemia, a rare blood cancer diagnosed in only about 1,000 to 1,500 people a year in the United States.

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