Blood: Long-term prognosis after gene therapy for patients with adenosine deaminase severe combined immunodeficiency (ADA SCID)

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The effect of gene therapy can be stable for up to 11 years

The effect of gene therapy can be stable for up to 11 years

Analysis of integration sites showed that some significant clones integrated into the chromosomes near the proto-oncogene, but did not cause clinically significant clone proliferation

Patients who lack functional adenosine deaminase activity will develop severe combined immunodeficiency (ADA SCID) and can be treated with ADA enzyme replacement therapy (ERT), allogeneic hematopoietic stem cell transplantation (HSCT) or autologous HSCT with genetically corrected cells ( Gene therapy-GT) treatment

Patients who lack functional adenosine deaminase activity will develop severe combined immunodeficiency (ADA SCID) and can be treated with ADA enzyme replacement therapy (ERT), allogeneic hematopoietic stem cell transplantation (HSCT) or autologous HSCT with genetically corrected cells ( Gene Therapy-GT) Treatment of Immunity

A cohort of 10 ADA SCID patients aged 3 months to 15 years old received GT treatment in a phase II clinical trial from 2009 to 2012

Changes in the counts of different blood cells in 10 patients after GT treatment

Changes in the counts of different blood cells in 10 patients after GT treatment

Following close follow-up for 8-11 years, 9 out of 10 patients obtained effective immune reconstitution sufficient to prevent serious infections and did not require salvage ERT or secondary allogeneic HSCT

After close follow-up for 8-11 years, 9 out of 10 patients obtained effective immune reconstitution enough to prevent serious infections , and did not require salvage ERT or secondary allogeneic HSCT to prevent infection.

Changes of ADA enzyme activity and red blood cell deoxyadenosine nucleotides after GT treatment in 10 patients

Changes of ADA enzyme activity and red blood cell deoxyadenosine nucleotides after GT treatment in 10 patients

Blood cell ADA enzyme activity and adenine metabolite normalization, as well as the level of cellular and humoral immune reconstitution all have a wide range of responses

The prognosis of young patients and those receiving high-dose gene-marked CD34+ cell therapy is generally good

The results of this long-term follow-up study show that gene therapy has a long-lasting effect on ADA SCID patients, but there is also a risk of gRV genotoxicity

Gene therapy has a long-lasting effect in patients with ADA SCID, but there is also a risk of gRV genotoxicity.

Original source:

Reinhardt Bryanna C,Habib Omar,Shaw Kit L et al.

Long-term Outcomes after Gene Therapy for Adenosine Deaminase Severe Combined Immune Deficiency (ADA SCID)

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