The two boys in a sixth floor room at Boston Children’s Hospital – 4-year-old Zachary in bed playing a bike race game on an iPad and 2-year-old Gabriel asleep on his mother’s shoulder – are recovering from stem cell transplants for a pre-leukemia condition so rare in pediatrics that only 50 other cases have been reported globally. The brothers are among the seven children that Dr. Inga Hofmann has treated for myelofibrosis, a blood disorder that usually strikes older adults. This is why the Kaiser boys and their family moved here in February 2015 from Ohio to be treated at the Dana-Farber/Boston Children’s Cancer and Blood Disorders Center.
“Myelofibrosis is rare even in adults, but it is much more common in adults than in children,” Hofmann says. “Very little is known about children with the condition. The concern has always been that it could lead to bone marrow failure or leukemia.”
The Kaisers’ saga began at Zachary’s 2-year-old check-up in July 2013. A routine hemoglobin check showed low blood cell levels, and Zachary was hospitalized in Columbus for a week. His mother, Candice Kaiser, was nine months pregnant with Gabriel at the time.
Preliminary results of a bone marrow biopsy raised concern that Zachary might have leukemia, but that was quickly ruled out. After a year of testing, doctors finally reached a diagnosis: myelofibrosis. Zachary began a course of transfusions to control the bone marrow disorder, which causes scarring of the marrow because the disease disrupts the body’s production of blood cells.