SynthesisInternational journal of environmental research and public health2020
The Value of Case Reports in Systematic Reviews from Rare Diseases. The Example of Enzyme Replacement Therapy (ERT) in Patients with Mucopolysaccharidosis Type II (MPS-II).
Synthesis in International journal of environmental research and public health, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 23 papers, 8 of them syntheses that pooled it.
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.
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Who cites it
23 citing papers in PubMed, 8 syntheses or guidelines pooled it, 27 citations in OpenAlex.
- Hepatic superscan on [Abdominal radiology (New York) · 2026Pooled it
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- Epidemiological and Clinical Characteristics of Urogenital System Cystic Echinococcosis: A Systematic Review.Balkan medical journal · 2025Pooled it
- Safety of Dupilumab Therapy for Atopic Dermatitis during Pregnancy: A Systematic Review and Meta-analysis.Acta dermato-venereologica · 2025Pooled it
- The prevalence of hepatic and thyroid toxicity associated with imatinib treatment of chronic myeloid leukaemia: a systematic review.International journal of clinical pharmacy · 2024Pooled it
- Symptomatic intraventricular choroid plexus cysts. Illustrative case and systematic review.Neurosurgical review · 2023Pooled it
- Simultaneous ipsilateral floating hip and knee: the double floating extremity-a systematic review and proposal of a treatment algorithm.European journal of trauma and emergency surgery : official publication of the European Trauma Society · 2023Pooled it
- Sirolimus treatment for paediatric head and neck lymphatic malformations: a systematic review.European archives of oto-rhino-laryngology : official journal of the European Federation of Oto-Rhino-Laryngological Societies (EUFOS) : affiliated with the German Society for Oto-Rhino-Laryngology - Head and Neck Surgery · 2023Pooled it
- From Immunization to Autoimmunity: A Deep Dive Into Post-COVID-19 Vaccine Reactions-A Narrative Review.Health science reports · 2026Article
- Finding Case Report Nuggets: A Web-Based Tool for Mining and Enhancing the Value of Clinical Case Reports.medRxiv : the preprint server for health sciences · 2025Article
- Electroconvulsive Therapy for Obsessive-Compulsive Symptoms in Schizophrenia Spectrum Disorders: A Systematic Review.Cureus · 2025Review
- Mental Ill-Health in young people with systemic autoinflammatory disease - a scoping review.Rheumatology international · 2025Article
- Identifying global trends from case reports of fibrodysplasia ossificans progressiva: a scoping review.Therapeutic advances in musculoskeletal disease · 2025Article
- Systemic treatment of recessive dystrophic epidermolysis bullosa with mesenchymal stromal cells: a scoping review of the literature and conclusions for future clinical research.The Journal of dermatological treatment · 2024Article
- Revision Surgery for Shoulder Infection after Arthroscopic Rotator Cuff Repair: Functional Outcomes and Eradication Rate-A Systematic Review.Healthcare (Basel, Switzerland) · 2024Review
- THE VALUE OF N-OF-1 DATA IN ZOOLOGICAL MEDICINE: A METHODOLOGICAL REVIEW.Journal of zoo and wildlife medicine : official publication of the American Association of Zoo Veterinarians · 2023Review
- Electromagnetic radiation therapy for Parkinson's disease tremor reduction- systematic reviews and Bayesian meta-analyses for comparing the effectiveness of electric, magnetic and light stimulation methods.Journal of neuroengineering and rehabilitation · 2023Review
- Rehabilitation in Patients Diagnosed with Arthrogryposis Multiplex Congenita: A Systematic Review.Children (Basel, Switzerland) · 2023Review
- Photobiomodulation Therapy for Multiple Painful Fixed Drug Eruptions: The First Case Report.Journal of lasers in medical sciences · 2023Article
- A single-arm study design with non-inferiority and superiority time-to-event endpoints: a tool for proof-of-concept and de-intensification strategies in breast cancer.Frontiers in oncology · 2023Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
8 authors at 4 institutions in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
backgroundCase reports are usually excluded from systematic reviews. Patients with rare diseases are more dependent on novel individualized strategies than patients with common diseases. We reviewed and summarized the novelties reported by case reports in mucopolysaccharidosis type II (MPS-II) patients treated with enzyme replacement therapy (ERT).
methodsWe selected the case reports included in a previous meta-analysis of patients with MPS-II treated with ERT. Later clinical studies evaluating the same topic of those case reports were reported. Our primary aim was to summarize novelties reported in previous case reports. Secondary objectives analyzed the number of novelties evaluated in subsequent clinical studies and the time elapsed between the publication of the case report to the publication of the clinical study.
resultsWe identified 11 innovative proposals in case reports that had not been previously considered in clinical studies. Only two (18.2%) were analyzed in subsequent nonrandomized cohort studies. The other nine novelties (81.8%) were analyzed in later case reports (five) or were not included in ulterior studies (four) after more than five years from their first publication.
conclusionsCase reports should be included in systematic reviews of rare disease to obtain a comprehensive summary of the state of research and offer valuable information for healthcare practitioners.
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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.