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As many who attended the CGD Symposium at the IDF 2015 National Conference in New Orleans know, Dr. Andrew Gennery gave a riveting presentation regarding the latest advancements in stem cell transplants for chronic granulomatous disease (CGD).
Dr. Gennery is a consultant at Newcastle University, United Kingdom, in hematopoietic stem cell transplantation and pediatric immunology. Thanks to his vast experience and research in treating CGD, he has earned a reputation as a global thought leader in the field.
His research regarding the outcomes of patients following stem cell transplant for primary immunodeficiency diseases (PI) has been published in several highly respected peer-reviewed publications. In addition, his clinical work has led to the development of new methods of T-cell depletion for patients with PI to minimize the occurrence of graft versus host disease in children, a common and serious side effect of stem cell transplants.
Dr. Gennery began his presentation by discussing the considerable risks of stem cell transplant, which despite many advancements, can lead to mortality or long-term complications from graft versus host disease. Remarkably, under Dr. Gennery’s leadership, Newcastle University Hospital has a survival rate of 85%.
“We have been very lucky,” he said quietly and with sincere humility.
In addition to weighing the risks of a bone marrow transplant, Dr. Gennery advised patients to also weigh the risks of not having a bone marrow transplant.
While the survival rate of stem cell transplant for CGD can be as high as 90% at some hospitals, said Dr. Gennery, he pointed out that once patients with CGD reach adulthood, their mortality without transplant increases to 50 percent.
“What we also need to consider, is the quality of life for both groups of patients,” he said.
Dr. Gennery presented the results from a pioneering study he led in 2009, in which parents reported that the quality of life for non-transplanted children was significantly lower than for healthy children. Parents also reported increased emotional difficulties compared to published norms. Meanwhile, transplanted children were not significantly different from healthy norms.
Although Dr. Gennery is a proponent of transplants in younger patients with CGD who have not had an established infection, he also advised that parents and patients carefully consider the hospital before moving forward. While many hospitals may have had success in stem cell transplants for malignant diseases, he recommended choosing a hospital that specializes in transplanting patients with CGD specifically.
“In many cases, it can come down to this,” he said, rubbing his thumb and fingers together in a circular motion, as if sprinkling a pinch of salt. “Hospitals may use the exact same protocol for transplanting patients with CGD, yet their outcomes may be different. It is not unlike a group of chefs, who all use the same recipe to bake a cake. However, based on their level of experience, it will come out differently.”
Thank you, Dr. Gennery, for your insightful presentation and for your commitment to pioneering new research and promising treatments for those living with CGD.
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