Allogeneic cord hematopoietic stem cell transplantation in an infant with primary myelofibrosis

Primary myelofibrosis (PMF) is rare in children. An allogeneic hematopoietic stem cell transplantation (HSCT) is the only known curative therapy for severe cases. Here, we report the case of a female infant with PMF treated with allogeneic HSCT using an unrelated cord blood unit. She had successful reversal of her disease, but experienced complications related to transplant. This is the seventh reported case of HSCT for PMF in children, and the second using umbilical cord blood. We conclude that cord HSCT is a useful curative treatment option in children with PMF, but that efforts must be taken to reduce complications.