ArticleClinical and translational medicine2021
Reiterative infusions of MSCs improve pediatric osteogenesis imperfecta eliciting a pro-osteogenic paracrine response: TERCELOI clinical trial.
Article in Clinical and translational medicine, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT02172885 (Mesenchymal Stem Cell Based Therapy for the Treatment of Osteogenesis Imperfecta), which is not on this map. Cited by 36 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.
Mesenchymal Stem Cell Based Therapy for the Treatment of Osteogenesis Imperfecta
Who cites it
36 citing papers in PubMed, 59 citations in OpenAlex.
- Dose matters: haploinsufficiency in osteogenesis imperfecta.Nature reviews. Endocrinology · 2026Review
- Spatiotemporal Regulation and Lineage Specification in Embryonic Endochondral Ossification.International journal of molecular sciences · 2026Review
- Efficacy of Fetal MSC Therapy for Osteogenesis Imperfecta: A Systematic Review and Meta-Analysis in Mice.Stem cells international · 2026Review
- [Comprehensive considerations for the diagnosis, treatment, and management of osteogenesis imperfecta].Zhongguo dang dai er ke za zhi = Chinese journal of contemporary pediatrics · 2025Review
- Mesenchymal stem cells in treating human diseases: molecular mechanisms and clinical studies.Signal transduction and targeted therapy · 2025Review
- Therapeutic effect of mesenchymal stem cells and their derived exosomes in diseases.Molecular biomedicine · 2025Review
- Comprehensive Review of Osteogenesis Imperfecta: Current Treatments and Future Innovations.Human gene therapy · 2025Review
- Challenges and future perspectives in using mesenchymal stem cells for efficient bone fracture healing.Frontiers in bioengineering and biotechnology · 2025Review
- Medical Management for Fracture Prevention in Children with Osteogenesis Imperfecta.Calcified tissue international · 2024Review
- Current and Developing Pharmacologic Agents for Improving Skeletal Health in Adults with Osteogenesis Imperfecta.Calcified tissue international · 2024Review
- The Role of Mitochondrial Homeostasis in Mesenchymal Stem Cell Therapy-Potential Implications in the Treatment of Osteogenesis Imperfecta.Pharmaceuticals (Basel, Switzerland) · 2024Review
- Article
- Successful transport across continents of GMP-manufactured and cryopreserved culture-expanded human fetal liver-derived mesenchymal stem cells for use in a clinical trial.Regenerative therapy · 2024Article
- New Perspectives of Therapies in Osteogenesis Imperfecta-A Literature Review.Journal of clinical medicine · 2024Review
- Emerging Landscape of Osteogenesis Imperfecta Pathogenesis and Therapeutic Approaches.ACS pharmacology & translational science · 2024Review
- Revolutionizing bone defect healing: the power of mesenchymal stem cells as seeds.Frontiers in bioengineering and biotechnology · 2024Review
- A distinct circular DNA profile intersects with proteome changes in the genotoxic stress-related hSOD1Cell & bioscience · 2023Article
- Regulation of Adipose-Derived Stem Cell Activity by Melatonin Receptors in Terms of Viability and Osteogenic Differentiation.Pharmaceuticals (Basel, Switzerland) · 2023Article
- Review
- Innovations in cell therapy in pediatric diseases: a narrative review.Translational pediatrics · 2023Review
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Authors and funding
14 authors at 6 institutions in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
backgroundOsteogenesis imperfecta (OI) is a rare genetic disease characterized by bone fragility, with a wide range in the severity of clinical manifestations. The majority of cases are due to mutations in the COL1A1 or COL1A2 genes, which encode type I collagen. Mesenchymal stem cells (MSCs), as the progenitors of the osteoblasts, the main type I collagen secreting cell type in the bone, have been proposed and tested as an innovative therapy for OI with promising but transient outcomes.
methodsTo overcome the short-term effect of MSCs therapy, we performed a phase I clinical trial based on reiterative infusions of histocompatible MSCs, administered in a 2.5-year period, in two pediatric patients affected by severe and moderate OI. The aim of this study was to assess the safety and effectiveness of this cell therapy in nonimmunosuppressed OI patients. The host response to MSCs was studied by analyzing the sera from OI patients, collected before, during, and after the cell therapy.
resultsWe first demonstrated that the sequential administration of MSCs was safe and improved the bone parameters and quality of life of OI patients along the cell treatment plus 2-year follow-up period. Moreover, the study of the mechanism of action indicated that MSCs therapy elicited a pro-osteogenic paracrine response in patients, especially noticeable in the patient affected by severe OI.
conclusionsOur results demonstrate the feasibility and potential of reiterative MSCs infusion for two pediatric OI and highlight the paracrine response shown by patients as a consequence of MSCs treatment.
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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.