Adenosine deaminase (ADA) enzyme deficiency results in severe combined immunodeficiency (SCID), a fatal autosomal recessive inherited immune disorder. Strimvelis (or GSK2696273) is a gene therapy intended for patients with ADA-SCID and for whom no suitable human leukocyte antigen (HLA) matched related stem cell donor is available. This therapy aims to restore ADA function in hematopoietic cell lineages, and in doing so prevents the pathology caused by purine metabolites (i.e., impaired immune function). This registry evaluates the long term safety and effectiveness outcomes of subjects who have received Strimvelis (or GSK2696273).
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Overall survival
Timeframe: After 15 years of follow-up, it will continue to be solicited every 2 years until the registry closes
Intervention free survival
Timeframe: Up to 15 years
Number of subjects with the use of medications/treatments of interest
Timeframe: Up to 15 years
Absolute peripheral lymphocyte for Immune reconstitution assessment
Timeframe: Up to 15 years
Absolute cluster of differentiation (CD)3+ T-cell for Immune reconstitution assessment
Timeframe: Up to 15 years
Absolute CD19+ B-cell counts for Immune reconstitution assessment
Timeframe: Up to 15 years
Phytohaemagglutinin (PHA) and anti CD-3 as a measure for T cell function
Timeframe: Up to 15 years
Growth percentile in body height
Timeframe: Up to 15 years
Growth percentile in body weight
Timeframe: Up to 15 years
Deoxyadenosine nucleotides (dAXP) levels in red blood cells for the measurement of systemic metabolite detoxification
Timeframe: Up to 15 years
Vector copy number measured in peripheral blood mononuclear cells (PBMCs)
Timeframe: Up to 15 years
Number of subjects with severe infections
Timeframe: Up to 15 years
Percentage of subjects with severe infections
Timeframe: Up to 15 years
Length of hospital stay
Timeframe: Up to 15 years
Number of subjects with non-immunological manifestations of ADA SCID
Timeframe: Up to 15 years
Pediatric development and quality of life data
Timeframe: Up to 15 years
Scores for Pediatric Quality of Life Questionnaire (Peds-QL)
Timeframe: Up to 15 years
Scores for Ages and Stages Questionnaire-3[ASQ-3]
Timeframe: Up to 15 years
Number of subjects with adverse events of interest
Timeframe: Up to 15 years (oncogenesis will continue to be solicited every 2 years until the registry closes)
Number of subjects with any adverse events (AEs) and any serious adverse events (SAEs) as a safety measure
Timeframe: Up to 15 years
Number of subjects with abnormal clinical laboratory blood test results as a safety measure
Timeframe: Up to 15 years
Number of subjects with fertility and pregnancy related outcomes
Timeframe: After 15 years of follow-up, it will continue to be solicited every 2 years until the registry closes
Data from Retroviral Insertion Site (RIS) analysis and replication competent retrovirus (RCR)
Timeframe: Up to 15 years