Successful Low Toxicity Hematopoietic Stem Cell Transplantation for High-Risk Adult Chronic Granulomatous Disease Patients
- 15 June 2005
- journal article
- case report
- Published by Ovid Technologies (Wolters Kluwer Health) in Transplantation
- Vol. 79 (11), 1596-1606
- https://doi.org/10.1097/01.tp.0000163466.73485.5e
Abstract
Allogeneic hematopoietic stem cell transplantation for chronic granulomatous disease (CGD) is associated with a significant risk of transplant-related mortality. Adult age, overt infection, and residual inflammatory disease at transplant are major risk factors. Here we report the favorable outcome after bone marrow transplantation in three high-risk adult CGD patients (ages 18, 35, and 39) with severe disease-related complications (overt pneumonia, liver abscess, steroid-dependent granulomatous colitis, diabetes, restrictive lung disease, renal insufficiency, epilepsia). Bone marrow donors were human leukocyte antigen-matched related or unrelated. The conditioning regimen consisted of 2 x 4 mg/kg oral busulphan (d -3, -2), fludarabine 6 x 30 mg/qm (d -7 to -2), rabbit anti-T-cell-globulin (Fresenius) 4 x 10 mg/kg (d -4 to -1). Graft versus host disease prophylaxis consisted of cyclosporine A and mycophenolate-mofetil. Mean neutrophil and platelet engraftment was observed at day +18.5 and +22.5, respectively. All infectious and inflammatory lesions resolved and restrictive lung disease improved. No signs of grade II-IV acute or chronic graft versus host disease were observed. With a follow-up of 12 to 27 months, all patients are alive and well with full donor chimerism, normalized superoxide production, and documented T- and B-cell function. This modified reduced intensity conditioning protocol is a promising treatment modality for high-risk adult CGD patients.Keywords
This publication has 37 references indexed in Scilit:
- Itraconazole to Prevent Fungal Infections in Chronic Granulomatous DiseaseNew England Journal of Medicine, 2003
- Treatment of chronic granulomatous disease with myeloablative conditioning and an unmodified hemopoietic allograft: a survey of the European experience, 1985-2000Blood, 2002
- Diagnostic and therapeutic impact of whole body positron emission tomography using fluorine-18-fluoro-2-deoxy-D-glucose in children with chronic granulomatous diseaseArchives of Disease in Childhood, 2001
- Treatment of Chronic Granulomatous Disease with Nonmyeloablative Conditioning and a T-Cell–Depleted Hematopoietic AllograftNew England Journal of Medicine, 2001
- Chronic Granulomatous Disease: Report on a National Registry of 368 PatientsMedicine, 2000
- Chronic granulomatous disease in Japan: Incidence and natural historyPediatrics International, 1999
- BONE MARROW TRANSPLANTATION FOR BETA-THALASSEMIAHematology/Oncology Clinics of North America, 1999
- Host defense molecule polymorphisms influence the risk for immune-mediated complications in chronic granulomatous disease.JCI Insight, 1998
- A Controlled Trial of Interferon Gamma to Prevent Infection in Chronic Granulomatous DiseaseNew England Journal of Medicine, 1991
- Trimethoprim-Sulfamethoxazole Prophylaxis in the Management of Chronic Granulomatous DiseaseThe Journal of Infectious Diseases, 1990