Allogeneic Reduced-Intensity Hematopoietic Stem Cell Transplantation for Chronic Granulomatous Disease: a Single-Center Prospective Trial.
Mark PartaCorin KellyNana KwatemaaNarda TheobaldDiane HilligossJing QinDouglas B KuhnsChrista ZerbeSteven M HollandHarry MalechElizabeth M KangPublished in: Journal of clinical immunology (2017)
A reduced-intensity allogeneic transplant protocol based on alemtuzumab and busulfan with sirolimus GVHD prophylaxis produced high rates of successful engraftment and minimal regimen-related toxicity. Prolonged clinical follow-up has confirmed its efficacy in ameliorating CGD-related disease. Outcomes were not acceptable with donor cell infusion rescue of cause with poor graft function.
Keyphrases
- stem cell transplantation
- hematopoietic stem cell
- bone marrow
- randomized controlled trial
- single cell
- allogeneic hematopoietic stem cell transplantation
- study protocol
- acute myeloid leukemia
- clinical trial
- low dose
- cell therapy
- oxidative stress
- type diabetes
- stem cells
- metabolic syndrome
- mesenchymal stem cells
- weight loss
- idiopathic pulmonary fibrosis
- systemic sclerosis