Co-transfusion of haplo-identical hematopoietic and mesenchymal stromal cells to treat a patient with severe aplastic.
A 3-year-old girl with severe aplastic anemia (SAA) that was unresponsive to steroid, cyclosporine and filgrastim treatments received bone marrow (BM) mesenchymal stromal cells (MSC; 1.25 x 10(6)/kg), granulocyte colony-stimulating factor (G-CSF)-mobilized BM and peripheral blood stem cell grafts from her father. Prior to stem cell transplantation, she had experienced repeated bacterial infections and received 44 blood transfusions during 8 months after diagnosis. The conditioning regimen consisted of fludarabine, cyclophosphamide and busulfan, and prophylaxis of acute graft-versus-host disease (GvHD) was performed by administration of anti-CD25 monoclonal antibody, cyclosporine A, methotrexate, mycophenolate mofetil and anti-thymocyte globulin. The patient achieved rapid hematopoietic engraftment of donor origin and no acute or chronic GvHD was observed. She is now alive with a good performance status, and the dose of cyclosporine A is being tapered. The novel regimen described here might be a suitable option for children with SAA who lack immediate access to HLA-matched sources.
Wang
Hengxiang
H
Wang
Zhidong
Z
Xue
Mei
M
Liu
Jing
J
Yan
Hongmin
H
Guo
Zikuan
Z
D000741:Anemia, Aplastic / Q000235:genetics / Q000276:immunology / Q000473:pathology / Q000503:physiopathology / Q000628:therapy*; D000911:Antibodies, Monoclonal / Q000627:therapeutic use; D001803:Blood Transfusion; D001854:Bone Marrow Cells / Q000166:cytology / Q000187:drug effects / Q000502:physiology*; D002675:Child, Preschool; D018572:Disease-Free Survival; D005220:Fathers; D005260:Female; D016179:Granulocyte Colony-Stimulating Factor / Q000008:administration & dosage; D006680:HLA Antigens / Q000276:immunology; D019650:Hematopoietic Stem Cell Mobilization; D018380:Hematopoietic Stem Cell Transplantation; D006801:Humans; D007108:Immune Tolerance; D007166:Immunosuppressive Agents / Q000627:therapeutic use; D053645:Interleukin-2 Receptor alpha Subunit / Q000276:immunology; D008297:Male; D045164:Mesenchymal Stem Cell Transplantation; D019653:Myeloablative Agonists / Q000627:therapeutic use; D019172:Transplantation Conditioning
D000911:Antibodies, Monoclonal; D006680:HLA Antigens; D007166:Immunosuppressive Agents; D053645:Interleukin-2 Receptor alpha Subunit; D019653:Myeloablative Agonists; D016179:Granulocyte Colony-Stimulating Factor