ESPEYB19 5. Bone, Growth Plate and Mineral Metabolism Novel treatments for rare skeletal disorders (3 abstracts)
5.2. Reiterative infusions of MSCs improve pediatric osteogenesis imperfecta eliciting a pro-osteogenic paracrine response: TERCELOI clinical trial
Infante A , Gener B , Vázquez M , Olivares N , Arrieta A , Grau G , Llano I , Madero L , Bueno AM , Sagastizabal B , Gerovska D , Araúzo-Bravo MJ , Astigarraga I & Rodríguez CI
Clin Transl Med. 2021 Jan;11(1):e265. Abstract: https://pubmed-ncbi-nlm-nih-gov.proxy.kib.ki.se/33463067/
In brief: In order to explore safety, efficacy, and feasibility of repeated infusions of mesenchymal stem cells (MSCs) as a treatment for OI, two pediatric OI patients were treated with repeated infusions of HLA-matched MSCs. The patients tolerated the treatment well and some positive effects on physical functioning and well-being are reported.
Commentary: Infusion of mesenchymal stem cells as a treatment for osteogenesis imperfecta has been considered for more than 20 years. However, enthusiasm has been hampered by findings that engraftment of infused cells into bone is low. Consequently, most studies have focussed on improving the engraftment potential of the infused stem cells.
In this study, two non-immunosuppressed pediatric OI patients were treated with 5 consecutive infusions of related donor MSCs. Both patients tolerated the infusions well with no or minimal side-effects with moderately increased BMD as well as reportedly positive effects on physical functioning and well-being. Due to its small size (two patients) this is obviously a pilot study. Neverthless, both patients tolerated repeated infusion of MHC-matched MSCs well with no immunological side-effects detected and some positive effects on both objective (BMD) and subjective parameters suggest that larger trials would be motivated. This may lead to a new approach using repeated infusions of MSCs isolated from relatives and expanded in vitro as a treatment for moderate and severe OI.
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ESPE Yearbook of Paediatric Endocrinology
ISSN 1662-4009 (Online)
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