Successful treatment of invasive aspergillosis in chronic granulomatous disease by granulocyte transfusions followed by peripheral blood stem cell transplantation

Chronic granulomatous disease (CGD) is a primary immunodeficiency disorder characterized by impaired microbial killing and susceptibility to bacterial and fungal infections. Cure of the disease can be achieved by stem cell tr ansplantation when performed early in its course, and before severe infecti ons have developed. Invasive aspergillosis constitutes a very high risk for transplantation. We report a 4-year-old boy with X-linked CGD who underwen t successful HLA-identical peripheral blood stem cell (PBSC) transplantatio n during invasive pulmonary aspergillosis and osteomyelitis of the left fou rth rib, which was unresponsive to antifungal treatment. During the 2 month s prior to the transplant he received G-CSF-mobilized granulocyte transfusi ons (GTX) from unrelated donors three times a week in addition to the antif ungal treatment. This resulted in clinical improvement in his respiratory s tatus. He also received GTX during the aplastic period after the conditioni ng regimen, until he had engrafted. Post-transplant superoxide generation t est revealed that neutrophil function was within normal range. One year pos t transplant the CT scan showed almost complete clearance of the pulmonary infiltrates and a marked improvement in the osteomyelitic process. Based on other reports and our own experience, GTX can serve as important treatment in patients with CGD who have failed conventional anti-fungal treatment an d for whom stem cell transplantation is the only chance for cure.