ReviewJournal of clinical immunology2017
How We Manage Adenosine Deaminase-Deficient Severe Combined Immune Deficiency (ADA SCID).
Review in Journal of clinical immunology, 2017. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 21 papers.
What it found
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
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.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
21 citing papers in PubMed, 45 citations in OpenAlex.
- Outcomes following treatment for ADA-deficient severe combined immunodeficiency: a report from the PIDTC.Blood · 2022Trial
- Review
- 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
- The possible role of the nucleoside adenosine in countering skin aging: A review.BioFactors (Oxford, England) · 2022Review
- Current landscape of gene-editing technology in biomedicine: Applications, advantages, challenges, and perspectives.MedComm · 2022Review
- Precision medicine: The use of tailored therapy in primary immunodeficiencies.Frontiers in immunology · 2022Review
- Review of Treatment for Adenosine Deaminase Deficiency (ADA) Severe Combined Immunodeficiency (SCID).Therapeutics and clinical risk management · 2022Review
- Adenosine Deaminase (ADA)-Deficient Severe Combined Immune Deficiency (SCID) in the US Immunodeficiency Network (USIDNet) Registry.Journal of clinical immunology · 2020Article
- Past, Present, and Future of The Journal of Clinical Immunology, the International Journal of Inborn Errors of Immunity.Journal of clinical immunology · 2020Article
- Preclinical Development of Autologous Hematopoietic Stem Cell-Based Gene Therapy for Immune Deficiencies: A Journey from Mouse Cage to Bed Side.Pharmaceutics · 2020Review
- Comparison of elapegademase and pegademase in ADA-deficient patients and mice.Clinical and experimental immunology · 2020Article
- Gene therapy and genome editing for primary immunodeficiency diseases.Genes & diseases · 2020Review
- Editorial, Journal of Clinical Immunology.Journal of clinical immunology · 2019Article
- StrimvelisPharmacoEconomics - open · 2019Review
- Family-Based Next-Generation Sequencing Study Identifies anOmics : a journal of integrative biology · 2019Article
- Consensus approach for the management of severe combined immune deficiency caused by adenosine deaminase deficiency.The Journal of allergy and clinical immunology · 2019Review
- Enzymatic Transition States and Drug Design.Chemical reviews · 2018Review
- Adenosine deaminase deficiency: a review.Orphanet journal of rare diseases · 2018Review
- Gene therapy in rare diseases: the benefits and challenges of developing a patient-centric registry for Strimvelis in ADA-SCID.Orphanet journal of rare diseases · 2018Article
- Long-Term Outcome of Adenosine Deaminase-Deficient Patients-a Single-Center Experience.Journal of clinical immunology · 2017Article
Corrections and comments
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Authors and funding
2 authors at 2 institutions in 2 countries.
Funding
Abstract
Adenosine deaminase-deficient severe combined immune deficiency (ADA SCID) accounts for 10-15% of cases of human SCID. From what was once a uniformly fatal disease, the prognosis for infants with ADA SCID has improved greatly based on the development of multiple therapeutic options, coupled with more frequent early diagnosis due to implementation of newborn screening for SCID. We review the various treatment approaches for ADA SCID including allogeneic hematopoietic stem cell transplantation (HSCT) from a human leukocyte antigen-matched sibling or family member or from a matched unrelated donor or a haplo-identical donor, autologous HSCT with gene correction of the hematopoietic stem cells (gene therapy-GT), and enzyme replacement therapy (ERT) with polyethylene glycol-conjugated adenosine deaminase. Based on growing evidence of safety and efficacy from GT, we propose a treatment algorithm for patients with ADA SCID that recommends HSCT from a matched family donor, when available, as a first choice, followed by GT as the next option, with allogeneic HSCT from an unrelated or haplo-identical donor or long-term ERT as other options.
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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.