title: Successful engraftment of haploidentical stem cell transplant for familial haemophagocytic lymphohistiocytosis using both bone marrow and peripheral blood stem cells.
abstract: Familial haemophagocytic lymphohistiocytosis (HLH) is a disease with a very poor prognosis unless patients receive a bone marrow transplant. It is often difficult to find an HLA-matched donor and haploidentical familial donors may be considered. The main complication of this type of transplant is graft rejection. We describe a patient with familial HLH who received a haploidentical transplant using both mobilized peripheral blood and bone marrow stem cells in an attempt to overcome graft rejection by increasing the stem cell dose. The peripheral blood stem cell inoculum was CD34 enriched using a Cellpro column and T-cell depleted by Campath-1M, the patient received conditioning for a matched sibling donor transplant with the addition of Campath 1G. There was rapid and full engraftment and the patient remains disease free at 5 months. This technique may be applicable for other fatal inborn errors in the absence of an HLA-matched donor.
authors.affiliation: Department of Haematology, Institute of Child Health and Great Ormond Street Hospital for Children NHS Trust, London.
authors.forename: P
authors.initials: P
authors.lastname: Collins
authors.forename: M
authors.initials: M
authors.lastname: Watts
authors.forename: M
authors.initials: M
authors.lastname: Brocklesby
authors.forename: B
authors.initials: B
authors.lastname: Gerritsen
authors.forename: P
authors.initials: P
authors.lastname: Veys
mesh_terms: D016026:Bone Marrow Transplantation / Q000379:methods*; D002675:Child, Preschool; D005260:Female; D006085:Graft Survival; D018380:Hematopoietic Stem Cell Transplantation / Q000379:methods*; D015616:Histiocytosis, Non-Langerhans-Cell / Q000235:genetics / Q000628:therapy*; D006801:Humans; D019172:Transplantation Conditioning; D014184:Transplantation, Homologous