Successful father-to-son stem cell transplantation in a child with hemophagocytic lymphohistiocytosis using a reduced-intensity conditioning regimen.
OBJECTIVE
Hemophagocytic lymphohistiocytosis (HLH) is an uncommon disorder, usually lethal without allogeneic stem cell transplantation (SCT).


METHODS
We report a 9-month-old boy, the first child of consanguineous parents, diagnosed with HLH and neurological involvement demonstrated by magnetic resonance imaging (MRI), who received an allogeneic SCT from his HLA genetically matched father. Transplant was performed after a reduced-intensity conditioning (RIC) regimen consisting of cyclophosphamide, fludarabine, and melphalan. Graft vs. host disease (GVHD) prophylaxis included cyclosporine a and methotrexate.


RESULTS
An absolute neutrophil count of 0.5 x 10(9)/L was documented on day +20 and a platelet count >20 x 10(9)/L was shown by day 33. Full donor chimerism was showed on day +175. A follow-up brain MRI was reported normal. Twenty months after SCT, the child shows no evidence of HLH or GVHD activity, and has a normal psychomotor development.


CONCLUSIONS
Given the reduced toxicity of SCT with RIC, it could represent an attractive transplant method for children with HLH, in whom myeloablation plays no role in disease eradication, and in whom mixed chimerism may be enough to cure the disease.
Gonzalez-Llano
Oscar
O
School of Medicine and University Hospital, Dr. Jose E. Gonzalez, Universidad Autonoma de Nuevo Leon, Monterrey, Mexico. ogzzll25@hotmail.com
Jaime-Pérez
Jose
J
Cantu-Rodríguez
Olga
O
Mancias-Guerra
Consuelo
C
Gutierrez-Aguirre
Homero
H
Herrera-Garza
Jose
J
Gomez-Almaguer
David
D
D000971:Antineoplastic Combined Chemotherapy Protocols / Q000627:therapeutic use; D003131:Combined Modality Therapy; D003520:Cyclophosphamide / Q000008:administration & dosage; D005047:Etoposide / Q000008:administration & dosage; D005190:Family; D006086:Graft vs Host Disease; D006801:Humans; D007223:Infant; D051359:Lymphohistiocytosis, Hemophagocytic / Q000188:drug therapy / Q000473:pathology / Q000601:surgery*; D008279:Magnetic Resonance Imaging; D008297:Male; D008558:Melphalan / Q000008:administration & dosage; D008727:Methotrexate / Q000008:administration & dosage; D033581:Stem Cell Transplantation; D019172:Transplantation Conditioning; D014740:Vidarabine / Q000008:administration & dosage / Q000031:analogs & derivatives
D005047:Etoposide; D003520:Cyclophosphamide; D014740:Vidarabine; C024352:fludarabine; D008558:Melphalan; D008727:Methotrexate