ArticleBiologics : targets & therapy2009
Pegademase bovine (PEG-ADA) for the treatment of infants and children with severe combined immunodeficiency (SCID).
Article in Biologics : targets & therapy, 2009. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 59 papers.
What it found
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The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
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Who cites it
59 citing papers in PubMed, 88 citations in OpenAlex.
- Autologous Ex Vivo Lentiviral Gene Therapy for Adenosine Deaminase Deficiency.The New England journal of medicine · 2021Trial
- Defective B cell tolerance in adenosine deaminase deficiency is corrected by gene therapy.The Journal of clinical investigation · 2012Trial
- Trial
- Molecular Survival Strategies Against Kidney Filtration: Implications for Therapeutic Protein Engineering.Biophysica · 2026Article
- Advances and applications of engineered protein therapeutics and their role in precision medicine.Frontiers in bioengineering and biotechnology · 2026Review
- Long-Term Safety and Efficacy of Gene Therapy for Adenosine Deaminase Deficiency.The New England journal of medicine · 2025Observational
- Targeting the Adenosine-Mediated Metabolic Immune Checkpoint with Engineered Probiotic for Enhanced Chemo-Immunotherapy.Advanced science (Weinheim, Baden-Wurttemberg, Germany) · 2025Article
- Multi-Year Registry Study of Elapegademase Treatment in Patients With Adenosine Deaminase Severe Combined Immunodeficiency (ADA-SCID) Requiring Enzyme Replacement Therapy.Journal of clinical immunology · 2025Observational
- Transforming Medicine: Cutting-Edge Applications of Nanoscale Materials in Drug Delivery.ACS nano · 2025Review
- Treatment with Elapegademase Restores Immunity in Infants with Adenosine Deaminase Deficient Severe Combined Immunodeficiency.Journal of clinical immunology · 2024Article
- Research progress on the PEGylation of therapeutic proteins and peptides (TPPs).Frontiers in pharmacology · 2024Review
- Bio-Inspired Nanomaterials for Micro/Nanodevices: A New Era in Biomedical Applications.Micromachines · 2023Review
- Innovative Design of Targeted Nanoparticles: Polymer-Drug Conjugates for Enhanced Cancer Therapy.Pharmaceutics · 2023Review
- PEGylated Recombinant Adenosine Deaminase Maintains Detoxification and Lymphocyte Counts in Patients with ADA-SCID.Journal of clinical immunology · 2023Article
- Safety and efficacy of elapegademase in patients with adenosine deaminase deficiency: A multicenter, open-label, single-arm, phase 3, and postmarketing clinical study.Immunity, inflammation and disease · 2023Review
- Gene therapy for inborn errors of immunity: past, present and future.Nature reviews. Immunology · 2023Review
- Strategies to improve the physicochemical properties of peptide-based drugs.Pharmaceutical research · 2023Review
- Advances in CRISPR/Cas gene therapy for inborn errors of immunity.Frontiers in immunology · 2023Review
- Improved Durability to SARS-CoV-2 Vaccine Immunity following Coimmunization with Molecular Adjuvant Adenosine Deaminase-1.Journal of immunology (Baltimore, Md. : 1950) · 2022Article
- Long-Term Immune Recovery After Hematopoietic Stem Cell Transplantation for ADA Deficiency: a Single-Center Experience.Journal of clinical immunology · 2022Article
Corrections and comments
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Authors and funding
2 authors at 1 institution in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Adenosine deaminase deficiency (ADA) is a rare, inherited disorder of purine metabolism characterized by immunodeficiency, failure to thrive and metabolic abnormalities. A lack of the enzyme ADA allows accumulation of toxic metabolites causing defects of both cell mediated and humoral immunity leading to ADA severe combined immune deficiency (SCID), a condition that can be fatal in early infancy if left untreated. Hematopoietic stem cell transplant is curative but is dependent on a good donor match. Other therapeutic options include enzyme replacement therapy (ERT) with pegademase bovine (PEG-ADA) and more recently gene therapy. PEG-ADA has been used in over 150 patients worldwide and has allowed stabilization of patients awaiting more definitive treatment with hematopoietic stem cell transplant. It affords both metabolic detoxification and protective immune function with patients remaining clinically well, but immune reconstitution is often suboptimal and may not be long lived. We discuss the pharmacokinetics, immune reconstitution, effects on systemic disease and side effects of treatment with PEG-ADA. We also review the long-term outcome of patients receiving ERT and discuss the role of PEG-ADA in the management of infants and children with ADA-SCID, alongside other therapeutic options.
Indexed as
Identifiers
19707420PMC2726071W2122648489What OpenQuestion holds
Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.