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Children with X-linked severe combined immunodeficiency (SCID-X1) who undergo gene therapy have fewer infections and hospitalizations than those who receive a hematopoietic stem cell transplant (HSCT) from a partially matched donor, according to a study published in Blood.
“Over the last decade, gene therapy has emerged as a viable alternative to a partial matched stem cell transplant for infants with SCID-X1,” said study author Fabien Touzot, MD, PhD, of Necker Children’s Hospital in Paris, France.
“To ensure that we are providing the best alternative therapy possible, we wanted to compare outcomes among infants treated with gene therapy and infants receiving partial matched transplants.”
Dr Touzot and his colleagues studied the medical records of 27 children who received either a partially matched HSCT (n=13) or gene therapy (n=14) for SCID-X1 at Necker Children’s Hospital between 1999 and 2013.
The children receiving half-matched transplants and the children receiving gene therapy had been followed for a median of 6 years and 12 years, respectively.
The researchers compared T-cell development among the patients, as well as key clinical outcomes such as infections and hospitalization.
The 14 children in the gene therapy group developed healthy immune cells faster than the 13 children in the half-matched transplant group. In fact, in the first 6 months after therapy, T-cell counts had reached normal values in 78% of the gene therapy patients, compared to 26% of the HSCT patients.
The more rapid growth of the immune system in gene therapy patients was also associated with faster resolution of disseminated BDG infections. These infections resolved in a median of 11 months in the gene therapy group, compared to 25.5 months in the half-matched transplant group.
Gene therapy patients also had fewer infection-related hospitalizations—3 hospitalizations in 3 patients, compared to 15 hospitalizations in 5 patients from the half-matched HSCT group.
“Our analysis suggests that gene therapy can put these incredibly sick children on the road to defending themselves against infection faster than a half-matched transplant,” Dr Touzot said. “These results suggest that, for patients without a fully matched stem cell donor, gene therapy is the next-best approach.” 
Photo by Chad McNeeley
Children with X-linked severe combined immunodeficiency (SCID-X1) who undergo gene therapy have fewer infections and hospitalizations than those who receive a hematopoietic stem cell transplant (HSCT) from a partially matched donor, according to a study published in Blood.
“Over the last decade, gene therapy has emerged as a viable alternative to a partial matched stem cell transplant for infants with SCID-X1,” said study author Fabien Touzot, MD, PhD, of Necker Children’s Hospital in Paris, France.
“To ensure that we are providing the best alternative therapy possible, we wanted to compare outcomes among infants treated with gene therapy and infants receiving partial matched transplants.”
Dr Touzot and his colleagues studied the medical records of 27 children who received either a partially matched HSCT (n=13) or gene therapy (n=14) for SCID-X1 at Necker Children’s Hospital between 1999 and 2013.
The children receiving half-matched transplants and the children receiving gene therapy had been followed for a median of 6 years and 12 years, respectively.
The researchers compared T-cell development among the patients, as well as key clinical outcomes such as infections and hospitalization.
The 14 children in the gene therapy group developed healthy immune cells faster than the 13 children in the half-matched transplant group. In fact, in the first 6 months after therapy, T-cell counts had reached normal values in 78% of the gene therapy patients, compared to 26% of the HSCT patients.
The more rapid growth of the immune system in gene therapy patients was also associated with faster resolution of disseminated BDG infections. These infections resolved in a median of 11 months in the gene therapy group, compared to 25.5 months in the half-matched transplant group.
Gene therapy patients also had fewer infection-related hospitalizations—3 hospitalizations in 3 patients, compared to 15 hospitalizations in 5 patients from the half-matched HSCT group.
“Our analysis suggests that gene therapy can put these incredibly sick children on the road to defending themselves against infection faster than a half-matched transplant,” Dr Touzot said. “These results suggest that, for patients without a fully matched stem cell donor, gene therapy is the next-best approach.”
Photo by Chad McNeeley
Children with X-linked severe combined immunodeficiency (SCID-X1) who undergo gene therapy have fewer infections and hospitalizations than those who receive a hematopoietic stem cell transplant (HSCT) from a partially matched donor, according to a study published in Blood.
“Over the last decade, gene therapy has emerged as a viable alternative to a partial matched stem cell transplant for infants with SCID-X1,” said study author Fabien Touzot, MD, PhD, of Necker Children’s Hospital in Paris, France.
“To ensure that we are providing the best alternative therapy possible, we wanted to compare outcomes among infants treated with gene therapy and infants receiving partial matched transplants.”
Dr Touzot and his colleagues studied the medical records of 27 children who received either a partially matched HSCT (n=13) or gene therapy (n=14) for SCID-X1 at Necker Children’s Hospital between 1999 and 2013.
The children receiving half-matched transplants and the children receiving gene therapy had been followed for a median of 6 years and 12 years, respectively.
The researchers compared T-cell development among the patients, as well as key clinical outcomes such as infections and hospitalization.
The 14 children in the gene therapy group developed healthy immune cells faster than the 13 children in the half-matched transplant group. In fact, in the first 6 months after therapy, T-cell counts had reached normal values in 78% of the gene therapy patients, compared to 26% of the HSCT patients.
The more rapid growth of the immune system in gene therapy patients was also associated with faster resolution of disseminated BDG infections. These infections resolved in a median of 11 months in the gene therapy group, compared to 25.5 months in the half-matched transplant group.
Gene therapy patients also had fewer infection-related hospitalizations—3 hospitalizations in 3 patients, compared to 15 hospitalizations in 5 patients from the half-matched HSCT group.
“Our analysis suggests that gene therapy can put these incredibly sick children on the road to defending themselves against infection faster than a half-matched transplant,” Dr Touzot said. “These results suggest that, for patients without a fully matched stem cell donor, gene therapy is the next-best approach.”