title: Fludarabine-based reduced-intensity conditioning regimen for hematopoietic stem cell transplantation in primary hemophagocytic lymphohistiocytosis.
abstract: OBJECTIVE
  Primary hemophagocytic lymphohistiocytosis (HLH) is a life-threatening condition that clinically characterized by fever, hepatosplenomegaly, and cytopenia. Hematopoietic stem cell transplantation (HSCT) is the only curative treatment option for patients diagnosed with primary HLH.
  
  
  METHODS
  In this prospective study, we analyzed the outcome of 10 pediatric patients with primary HLH who had received HSCT, using reduced-intensity conditioning (RIC) regimen from 2007 to 2012. The median age at transplantation was 22.6 months (range: 6-60). All of the patients received the same RIC regimen based on the use of fludarabine in combination with melphalan and horse antithymocyte globulin (ATG). Cyclosporine and methylprednisolone were used as graft-vs.-host disease (GvHD) prophylaxis.
  
  
  RESULTS
  Hematopoietic engraftment occurred in all patients. At the present time, 8 patients with a median follow-up of 39 months are still alive and all of them are disease free. Acute and chronic GvHD developed in 6 and 2 patients, retrospectively. Two patients died of sepsis and chronic GvHD during the study.
  
  
  CONCLUSIONS
  Because of pretransplant infections caused by underlying immunodeficiency in patients with primary HLH, the use of less toxic regimen with RIC seems to be highly effective in this regard. Recipients of RIC transplant, with either full or mixed chimerism, had a long-term survival rate with no manifestation of primary HLH symptoms.
authors: Hematology-Oncology and Stem Cell Transplantation Research Center, Tehran University of Medical Sciences, Tehran, Iran.
authors: Amir Ali
authors: AA
authors: Hamidieh
authors: Zahra
authors: Z
authors: Pourpak
authors: Susan
authors: S
authors: Hashemi
authors: Kolsoum
authors: K
authors: Yari
authors: Mohammad Reza
authors: MR
authors: Fazlollahi
authors: Masoud
authors: M
authors: Movahedi
authors: Maryam
authors: M
authors: Behfar
authors: Mostafa
authors: M
authors: Moin
authors: Ardeshir
authors: A
authors: Ghavamzadeh
mesh_terms: D000971:Antineoplastic Combined Chemotherapy Protocols / Q000627:therapeutic use; D002648:Child; D002675:Child, Preschool; D005260:Female; D005500:Follow-Up Studies; D006086:Graft vs Host Disease; D018380:Hematopoietic Stem Cell Transplantation / Q000009:adverse effects; D006801:Humans; D007223:Infant; D051359:Lymphohistiocytosis, Hemophagocytic / Q000175:diagnosis / Q000628:therapy*; D008297:Male; D019653:Myeloablative Agonists / Q000008:administration & dosage; D011446:Prospective Studies; D018183:Transplantation Chimera; D019172:Transplantation Conditioning; D016896:Treatment Outcome; D014740:Vidarabine / Q000008:administration & dosage / Q000031:analogs & derivatives
chemical_list: D019653:Myeloablative Agonists; D014740:Vidarabine; C024352:fludarabine
keywords: hematopoietic stem cell transplantation; primary hemophagocytic lymphohistiocytosis; reduced-intensity conditioning