Case Study May Lead the Way for Safer Blood or Marrow Transplants

One patient’s journey undergoing three Blood and Marrow Transplants (BMT) while struggling with myelofibrosis, a bone marrow disease that can lead to acute leukemia, has provided new insights into mechanisms that may reduce some negative transplant side effects. The case study was published as a Letter to the Editor in Bone Marrow Transplantation, an online journal which is part of Nature Publishing Group.

Mutated or abnormal JAK2 is a protein that causes myelofibrosis. The patient underwent two bone marrow transplants and each did not engraft or “take” in the patient. However, after the second transplant, the abnormal JAK2 disappeared and the patient’s own normal blood and marrow cells returned. She did not require transfusions for several months. Unfortunately, the JAK2 and the myelofibrosis returned and the patient needed a third BMT from a different donor. This time the donor cells took permanently, eradicating the JAK2 and the myelofibrosis. Today the patient is alive and doing well. The key finding of this report highlights the ability of the normal donor blood and marrow cells to eliminate the malignant cells while leaving the patient’s normal cells alone, not causing the negative side effect of Graft-versus-Host Disease (GvHD). This is the first case describing a prolonged remission with JAK2 negativity whose transplant initially did not take after BMT.

Philip McCarthy, MD is the senior author of the article and Director, Blood and Marrow Transplant Center, Department of Medicine. He is collaborating with his colleague, Xuefang Cao, MD, PhD, Associate Professor of Oncology in the Department of Immunology, who is leading a project to explore ways to harness this effect without the harmful complication of GvHD.

“This new approach may decrease damage to normal tissue caused by GvHD while increasing the eradication of the underlying leukemia or cancer, thereby allowing physicians to offer BMTs to more patients,” says Dr. McCarthy.