Successful unrelated bone marrow transplantation for a patient with chronic granulomatous disease and associated resistant pneumonitis and Aspergillus osteomyelitis

We describe the successful treatment of a 20-year-old patient with chronic granulomatous disease (CGD), by unrelated bone marrow transplantation (UBMT ). The patient is relatively old compared to other CGD patients treated wit h BMT. He had had repeated serious infections from early childhood and was diagnosed as CGD, gp91-phox deficiency. Prolonged antibiotic-resistant pneu monitis worsened when the patient was 18 years old. In addition, he suffere d Aspergillus osteomyelitis and acute renal failure due to amphotericin B. He received 94 granulocyte transfusions from 94 adult donors and the infect ions gradually improved. In September 1998, at 20 years of age, he underwen t UBMT from an HLA 6 antigen-matched male donor, with CY and TBI conditioni ng. He received MTX and CsA as prophylaxis against GVHD. No serious complic ations occurred and rapid engraftment was achieved. Acute GVHD (grade 2, at day 19) and chronic GVHD (limited, at day 192) occurred. However, both wer e easily controlled. The patient is alive and well with no late rejection 2 6 months after UBMT.