In utero transplantation: baby steps towards an effective therapy
- M O Muench
- Bone Marrow Transplantation
We read the in utero transplantation review by Dr Muench with interest. This article describes the successful use of the procedure to transplant foetal patients with immunodeficiency, and attempts to correct other conditions. However, in order for a procedure to gain widespread acceptance it needs to demonstrate significant benefit over those procedures that it is intended to replace. In utero transplantation can only be offered to those families in which the diagnosis has previously been made. In immunodeficient patients, this applies to families who have had previously affected children. In families with severe combined immunodeficiency (SCID), the alternative to in utero stem cell transplantation is immediate diagnosis at birth by examination of the umbilical cord lymphocyte phenotype, followed by immediate transfer to a centre performing stem cell transplantation for immunodeficiency. Haematopoietic stem cell transplantation performed in the neonatal period avoids the morbidity and cost of treating postnatal acquired infection and failure to thrive, associated with late diagnosis of SCID.