ArticleThe Journal of allergy and clinical immunology2009
Improving cellular therapy for primary immune deficiency diseases: recognition, diagnosis, and management.
Article in The Journal of allergy and clinical immunology, 2009. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to 4 registered trials, which are not on this map. Cited by 54 papers.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
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.
A Prospective Natural History Study of Diagnosis, Treatment and Outcomes of Children With SCID Disorders (RDCRN PIDTC-6901)
A Retrospective and Cross-Sectional Analysis of Patients Treated for SCID Since January 1,1968 (RDCRN PIDTC-6902)
Analysis of Patients Treated for Wiskott-Aldrich Syndrome Since January 1, 1990 (RDCRN PIDTC-6904)
Analysis of Patients Treated for Chronic Granulomatous Disease Since January 1, 1995
Who cites it
54 citing papers in PubMed, 129 citations in OpenAlex.
- Autologous Ex Vivo Lentiviral Gene Therapy for Adenosine Deaminase Deficiency.The New England journal of medicine · 2021Trial
- Newborn Screening Followed By Early Treatment is Essential to Improve Survival in SCID.Journal of clinical immunology · 2025Article
- Somatic Genetic Reversion of a Duplication Event in IL2RG in Siblings.Journal of clinical immunology · 2023Article
- Severe Combined Immunodeficiency-Classification, Microbiology Association and Treatment.Microorganisms · 2023Review
- The diagnosis of severe combined immunodeficiency: Implementation of the PIDTC 2022 Definitions.The Journal of allergy and clinical immunology · 2023Article
- The diagnosis of severe combined immunodeficiency (SCID): The Primary Immune Deficiency Treatment Consortium (PIDTC) 2022 Definitions.The Journal of allergy and clinical immunology · 2023Article
- Positive Newborn Screening for Severe Combined Immunodeficiency: What Should the Pediatrician Do?Clinical medicine insights. Pediatrics · 2023Review
- SCID and Other Inborn Errors of Immunity with Low TRECs - the Brazilian Experience.Journal of clinical immunology · 2022Article
- Severe Combined Immunodeficiency with De Novo Duchenne Muscular Dystrophy Mutation.JPGN reports · 2022Article
- Defining the Clinical, Emotional, Social, and Financial Burden of Congenital Athymia.Advances in therapy · 2021Article
- Congenital Athymia: Genetic Etiologies, Clinical Manifestations, Diagnosis, and Treatment.Journal of clinical immunology · 2021Review
- Clinical and Immunological Features of 96 Moroccan Children with SCID Phenotype: Two Decades' Experience.Journal of clinical immunology · 2021Article
- A Critical Review on the Standardization and Quality Assessment of Nonfunctional Laboratory Tests Frequently Used to Identify Inborn Errors of Immunity.Frontiers in immunology · 2021Review
- Infections in Infants with SCID: Isolation, Infection Screening, and Prophylaxis in PIDTC Centers.Journal of clinical immunology · 2021Article
- Primary Immunodeficiencies: Diseases of Children and Adults - A Review.Advances in experimental medicine and biology · 2021Review
- Successful Preclinical Development of Gene Therapy for Recombinase-Activating Gene-1-Deficient SCID.Molecular therapy. Methods & clinical development · 2020Article
- Psychosocial services for primary immunodeficiency disorder families during hematopoietic cell transplantation: A descriptive study.Palliative & supportive care · 2019Article
- Consensus approach for the management of severe combined immune deficiency caused by adenosine deaminase deficiency.The Journal of allergy and clinical immunology · 2019Review
- Article
- Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
17 authors at 15 institutions in 3 countries.
Funding
Abstract
More than 20 North American academic centers account for the majority of hematopoietic stem cell transplantation (HCT) procedures for primary immunodeficiency diseases (PIDs), with smaller numbers performed at additional sites. Given the importance of a timely diagnosis of these rare diseases and the diversity of practice sites, there is a need for guidance as to best practices in management of patients with PIDs before, during, and in follow-up for definitive treatment. In this conference report of immune deficiency experts and HCT physicians who care for patients with PIDs, we present expert guidance for (1) PID diagnoses that are indications for HCT, including severe combined immunodeficiency disease (SCID), combined immunodeficiency disease, and other non-SCID diseases; (2) the critical importance of a high degree of suspicion of the primary care physician and timeliness of diagnosis for PIDs; (3) the need for rapid referral to an immune deficiency expert, center with experience in HCT, or both for patients with PIDs; (4) medical management of a child with suspicion of SCID/combined immunodeficiency disease while confirming the diagnosis, including infectious disease management and workup; (5) the posttransplantation follow-up visit schedule; (6) antimicrobial prophylaxis after transplantation, including gamma globulin administration; and (7) important indications for return to the transplantation center after discharge. Finally, we discuss the role of high-quality databases in treatment of PIDs and HCT as an element of the infrastructure that will be needed for productive multicenter clinical trials in these rare diseases.
Indexed as
Identifiers
What 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.