Polyethylene glycol-conjugated adenosine deaminase (ADA) therapy provides temporary immune reconstitution to a child with delayed-onset ADA deficiency

E Lainka, MS Hershfield, I Santisteban… - Clinical and Vaccine …, 2005 - Am Soc Microbiol
E Lainka, MS Hershfield, I Santisteban, P Bali, A Seibt, J Neubert, W Friedrich, T Niehues
Clinical and Vaccine Immunology, 2005Am Soc Microbiol
We describe the effects of polyethylene glycol-conjugated adenosine deaminase (ADA)
replacement therapy on lymphocyte counts, activation, apoptosis, proliferation, and cytokine
secretion in a 14-month-old girl with “delayed-onset” ADA deficiency and marked
immunodysregulation. Pretreatment lymphopenia affected T cells (CD4, 150/μl; CD8,
459/μl), B cells (16/μl), and NK cells (55/μl). T cells were uniformly activated and largely
apoptotic (CD4, 59%; CD8, 82%); and T-cell-dependent cytokine levels in plasma were …
Abstract
We describe the effects of polyethylene glycol-conjugated adenosine deaminase (ADA) replacement therapy on lymphocyte counts, activation, apoptosis, proliferation, and cytokine secretion in a 14-month-old girl with “delayed-onset” ADA deficiency and marked immunodysregulation. Pretreatment lymphopenia affected T cells (CD4, 150/μl; CD8, 459/μl), B cells (16/μl), and NK cells (55/μl). T cells were uniformly activated and largely apoptotic (CD4, 59%; CD8, 82%); and T-cell-dependent cytokine levels in plasma were elevated, including the levels of interleukin 2 (IL-2; 26 pg/ml), IL-4 (81 pg/ml), IL-5 (46 pg/ml), gamma interferon (1,430 pg/ml), tumor necrosis factor alpha (210 pg/ml), and IL-10 (168 pg/ml). Mitogen-stimulated peripheral blood mononuclear cells show reduced IL-2 secretion and proliferation. During the first 5 months of therapy there was clinical improvement and partial immune reconstitution, with nearly normal lymphocyte subset numbers, reduced T-cell activation and CD4-cell apoptosis, and decreased plasma cytokine levels. In parallel, IL-2 secretion and the lymphocyte mitogenic response improved. Between 4 and 7 months, immunoglobulin G antibodies to bovine ADA developed and resulted in the complete reversal of immune recovery.
American Society for Microbiology