Pre- and postnatal transplantation of getal mesenchymal stem cells in osteogenesis imperfecta: a two-center experience

Gotherstrom, Cecilia, Westgren, Magnus, Shaw, S.W. Steven, Astrom, Eva, Biswas, Arijit, Byers, Peter H., Mattar, Citra N. Z., Graham, Gail E., Taslimi, Jahan, Ewald, Uwe, Fisk, Nicholas M., Yeoh, Allen E. J., Lin, Ju-Li, Cheng, Po-Jen, Choolani, Mahesh, Le Blanc, Katarina and Chan, Jerry K. Y. (2014) Pre- and postnatal transplantation of getal mesenchymal stem cells in osteogenesis imperfecta: a two-center experience. Stem Cells Translational Medicine, 3 2: 255-264. doi:10.5966/sctm.2013-0090

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Author Gotherstrom, Cecilia
Westgren, Magnus
Shaw, S.W. Steven
Astrom, Eva
Biswas, Arijit
Byers, Peter H.
Mattar, Citra N. Z.
Graham, Gail E.
Taslimi, Jahan
Ewald, Uwe
Fisk, Nicholas M.
Yeoh, Allen E. J.
Lin, Ju-Li
Cheng, Po-Jen
Choolani, Mahesh
Le Blanc, Katarina
Chan, Jerry K. Y.
Title Pre- and postnatal transplantation of getal mesenchymal stem cells in osteogenesis imperfecta: a two-center experience
Journal name Stem Cells Translational Medicine   Check publisher's open access policy
ISSN 2157-6564
2157-6580
Publication date 2014-02-01
Year available 2013
Sub-type Article (original research)
DOI 10.5966/sctm.2013-0090
Open Access Status DOI
Volume 3
Issue 2
Start page 255
End page 264
Total pages 10
Place of publication Durham, NC, United States
Publisher AlphaMed Press
Language eng
Formatted abstract
Osteogenesis imperfecta (OI) can be recognized prenatally with ultrasound. Transplantation of mesenchymal stem cells (MSCs) has the potential to ameliorate skeletal damage. We report the clinical course of two patients with OI who received prenatal human fetal MSC (hfMSC) transplantation and postnatal boosting with same-donor MSCs. We have previously reported on prenatal transplantation for OI type III. This patient was retransplanted with 2.8 × 106 same-donor MSCs per kilogram at 8 years of age, resulting in low-level engraftment in bone and improved linear growth, mobility, and fracture incidence. An infant with an identical mutation who did not receive MSC therapy succumbed at 5 months despite postnatal bisphosphonate therapy. A second fetus with OI type IV was also transplanted with 30 × 106 hfMSCs per kilogram at 31 weeks of gestation and did not suffer any new fractures for the remainder of the pregnancy or during infancy. The patient followed her normal growth velocity until 13 months of age, at which time longitudinal length plateaued. A postnatal infusion of 10 × 106 MSCs per kilogram from the same donor was performed at 19 months of age, resulting in resumption of her growth trajectory. Neither patient demonstrated alloreactivity toward the donor hfMSCs or manifested any evidence of toxicities after transplantation. Our findings suggest that prenatal transplantation of allogeneic hfMSCs in OI appears safe and is of likely clinical benefit and that retransplantation with same-donor cells is feasible. However, the limited experience to date means that it is not possible to be conclusive and that further studies are required.
Keyword Mesenchymal stem cells
Mesenchymal stromal cells
Cell therapy
Osteogenesis imperfecta
Prenatal transplantation
In utero transplantation
Q-Index Code C1
Q-Index Status Confirmed Code
Institutional Status UQ

Document type: Journal Article
Sub-type: Article (original research)
Collections: UQ Centre for Clinical Research Publications
Official 2015 Collection
 
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Citation counts: TR Web of Science Citation Count  Cited 37 times in Thomson Reuters Web of Science Article | Citations
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Created: Fri, 14 Feb 2014, 20:15:53 EST by Roheen Gill on behalf of UQ Centre for Clinical Research