Successful CD34(+) progenitor cell transplantation in juvenile chronic myelogenous leukemia from HLA-mismatched father

Purpose Juvenile chronic myelogenous leukemia (JCML) has a poor prognosis, and stem cell transplantation (SCT) from an HLA-matched donor is the only c urative treatment. CD34(+) progenitor cell transplantation from HLA-mismatc hed family members for JCML patients who lack HLA-matched donors is an alte rnative approach to the treatment of this disease, and we present the first successful case reported to date. Patient and methods A 30-month-old boy with JCML received CD34(+) cell tran splantation from his HLA-mismatched father with the following conditioning regimen: TBI (12 Gy) in six fractions, thiotepa (150 mg/m(2)) every 12h x 4 doses (days 5 and 4) and melphalan (70mg/m(2))x 2 doses (days 3 and 2). By using a continuous flow blood cell separator, after four days of subcutane ous granulocyte-colony stimulating factor (G-CSF) injection, CD34(+) cells were collected. Results The number of CD34(+) cells infused was 5.0x10(6)/kg. Engraftment w as rapid and grade I acute graft versus host disease (GVHD) and chronic GVH D developed, both of which responded to steroid therapy. The patient is cur rently in complete remission (18 months post-transplant). Conclusion CD34(+) progenitor cell transplantation is a feasible alternativ e method of SCT for JCML patients, particularly when an HLA-matched donor i s not available.