Bone marrow transplantation and alternatives for adenosine deaminase deficiency.
Adenosine deaminase (ADA)-deficient severe combined immunodeficiency (SCID) comprises approximately 10% to 15% of all cases of SCID. The clinical effects of ADA deficiency are manifest most dramatically in the immune system, where it leads to severe lymphopenia. Although hematopoietic stem cell transplantation remains the mainstay of treatment for ADA-deficient SCID, 2 other treatment options are available, namely enzyme replacement therapy with PEG-ADA and autologous hematopoietic stem cell gene therapy. In this article the author reviews the available data on treatment by these different options, and offers an overview on when each of the different treatment options should be used.
Gaspar
H Bobby
HB
Centre for Immunodeficiency, Molecular Immunology Unit, UCL Institute of Child Health, 30 Guilford Street, London WC1N 1EH, UK. h.gaspar@ich.ucl.ac.uk
D000243:Adenosine Deaminase / Q000235:genetics* / Q000378:metabolism / Q000627:therapeutic use; D000818:Animals; D016026:Bone Marrow Transplantation; D002986:Clinical Trials as Topic; D056947:Enzyme Replacement Therapy; D015316:Genetic Therapy; D018380:Hematopoietic Stem Cell Transplantation; D006801:Humans; D007391:International Cooperation; D008231:Lymphopenia; D016511:Severe Combined Immunodeficiency / Q000235:genetics / Q000503:physiopathology / Q000628:therapy*
D000243:Adenosine Deaminase; C051409:pegademase bovine