Nonmyeloablative Bone Marrow Transplant from HLA-Identical Sibling for Chronic Granulomatous Disease

• Published in: The Japanese Journal of Pediatric Hematology Vol. 20; no. 1; pp. 34 - 38
• Main Authors: NUNOI, Hiroyuki, MIZUKAMI, Tomoyuki, HAMAMOTO, Kazuko, FUJITA, Naoto, KOBAYASHI, Masao, ONO, Mayuko
• Format: Journal Article
• Language: Japanese
• Published: THE JAPANESE SOCIETY OF PEDIATRIC HEMATOLOGY/ONCOLOGY 2006; 特定非営利活動法人 日本小児血液・がん学会
• Subjects: bone marrow transplantation; chronic granulomatous disease; donor lymphocyte infusion; nonmyeloablative stem cell transplantation
• ISSN: 0913-8706; 1884-4723
• DOI: 10.11412/jjph1987.20.34

Chronic granulomatous disease (CGD) is an inherited disorder caused by the abnormalities of NADPH oxidase in phagocytic cells. Stem cell transplantation (SCT) from an HLA-identical donor is an effective treatment for patients with CGD. However, SCT with myeloablative conditioning carries a high incidence of serious complications and death. We report a 21-year-old man with CGD who was successfully treated with allogeneic bone marrow transplantation (alloBMT) from his HLA-identical brother following a nonmyeloablative conditioning regimen consisting of cyclophosphamide, fludarabine and a low dose of total body irradiation. Engraftment was rapidly achieved without any toxicity or complication. Partial donor chimerism following alloBMT was converted to complete donor chimerism and nomal oxidase activity of neutrophils was induced by several donor lymphocyte infusions (DLI) from his HLA-identical brother. At 30 months after transplant, the patient is in excellent clinical and hematological condition, maintaining complete chimerism. We suggest that alloBMT preceded by nonmyeloablative conditioning and followed by DLI may constitute a successful mode of therapy for patients with CGD.