ECTS2013 Poster Presentations Clinical case posters (12 abstracts)
Ten years follow up after prenatal transplantation of fetal mesenchymal stem cell in a patient with severe osteogenesis imperfecta
Cecilia Götherström 1 , Katarina Le Blanc 1, , Eva Åström 1, , Jahan Taslimi 3 , Gail E Graham 4 , Uwe Ewald 3 & Magnus Westgren 1,
1Karolinska Institutet, Stockholm, Sweden; 2Karolinska University Hospital, Stockholm, Sweden; 3Uppsala University Hospital, Uppsala, Sweden; 4Children’s Hospital of Eastern Ontario, Ottawa, Ontario, Canada.
Background: Treatment with multipotent mesenchymal stromal cells (MSC) has the potential to ameliorate mesodermal disorders.
Objective: To treat severe osteogenesis imperfecta (OI) with fetal MSC.
Methods: Ten years ago, we treated a fetus with OI type III (COL1A2: c.3008G>A, p.Gly1003Asp) in utero with fetal HLA-mismatched MSC. The procedure was uncomplicated. At the age of 4 months i.v. pamidronete treatment was started due to new vertebral compressions fractures. Donor cells (range 0.1–16.4%) were detected in the bone at 9 months of age. At 8 years of age soon after a surgery, the patient was re-transplanted with 2.8×106/ kg cells and the effect evaluated.
Results: At 10 years of age, 2 years after the combined surgery and re-transplantation, the patient’s ability to walk has improved. She takes dance classes and participates in modified indoor hockey. Over the last 2 years, her linear growth has improved from −6.5 to −6S.D. Since birth, 12 fractures and 11 vertebral compression fractures have been confirmed. She has developed scoliosis treated with a brace. The patient has no lymphocyte proliferation, anti-FCS abs, anti-HLA I and II abs, anti-IgG or anti-IgM against MSC donor cells. Donor cell engraftment is low (0.003%) and limited to bone.
Conclusion: Our findings suggest that transplantation of allogeneic fetal MSC in OI is safe and re-transplantation is feasible. It is not possible from this single case to conclude on beneficial effects of MSC in OI, but the natural history of this severe form of OI is one of early morbidity and an infant with the same mutation who did not receive MSC treatment succumbed at 5 months of age despite postnatal bisphosphonate therapy.
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