Mesenchymal Stem Cell Transplantation for Osteogenesis Imperfecta Patients: A Systematic Review

Khorasani MM, Ebrahimzadeh MH, Dehghani M, Sharafoddin M, Moradi A, Jirofti N

Annals of the New York Academy of Sciences, 2025

12 participants

Abstract

The following systematic review provides an evaluation of the effectiveness of mesenchymal stem cell (MSC) transplantation in the treatment of osteogenesis imperfecta (OI). After reviewing 40 studies, 9 human clinical studies that included case reports or case series were included and resulted in a total of 12 patients with OI. Five studies documented an increase in total body bone mineral (TBBM) content, whereas four studies observed a reduction in fracture rates post-transplantation. Moreover, noticeable improvements in children's growth were recorded. Although both BMSCs and human fetal MSCs (hfMSCs) were found to be effective, studies involving BMSCs were rated higher in terms of methodological quality. Both BMSCs and hfMSCs demonstrated significant improvements in TBBM, growth rates, and fracture reduction, with BMSC studies scoring higher in methodological quality. Future randomized clinical trials with longer follow-ups are necessary.

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Evidence Hub reads this as a systematic review, done in people. Its evidence level is 1 of 5 (Highest · pooled evidence). Pooled evidence from several studies sits at the top of the evidence pyramid. Reported size: 12 participants or records.

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