Indications for hematopoietic cell transplantation for children with severe congenital neutropenia.

Severe congenital neutropenia (SCN) is a hematologic condition characterized by arrested maturation of myelopoiesis at the promyelocyte stage of development (1). With appropriate treatment using recombinant human granulocyte colony-stimulating factor (r-HuG-CSF), patients with SCN are now surviving well past infancy. With longer survival, the high risk of developing myelodysplastic syndrome (MDS)/acute myeloid leukemia (AML) has become remarkedly clear (2). Hematopoietic cell transplant (HCT) is the only curative option for these patients, but the outcome is poor once MDS/AML has developed. In this issue of the journal, Oshima et al. (3) report an analysis of 18 patients with SCN in Japan who underwent HCT (1989–2005) because of the lack of or a partial response to treatment with r-HuG-CSF prior to malignant transformation to MDS/AML.