PMID- 26703084
OWN - NLM
STAT- MEDLINE
DCOM- 20160718
LR  - 20160216
IS  - 1545-5017 (Electronic)
IS  - 1545-5009 (Linking)
VI  - 63
IP  - 4
DP  - 2016 Apr
TI  - Successful Outcome With Fludarabine-Based Conditioning Regimen for Hematopoietic 
      Stem Cell Transplantation From Related Donor in Fanconi Anemia: A Single Center 
      Experience From Turkey.
PG  - 695-700
LID - 10.1002/pbc.25844 [doi]
AB  - BACKGROUND: Fanconi anemia (FA) is a heterogeneous autosomal recessive (and 
      rarely X linked) disorder, which is characterized by congenital malformations, 
      progressive bone marrow failure, and predisposition to malignancies. 
      Hematopoietic stem cell transplantation (HSCT) is the only definitive treatment 
      for the hematological manifestations in FA. PROCEDURE: Twenty-seven patients with 
      FA underwent HSCT using fludarabine (Flu) based regimen at our center between 
      April 2004 and May 2014. One patient who developed acute leukemia before HSCT was 
      excluded from the study. The remaining 26 patients were included. The median age 
      of the patients at the time of transplantation was 9.6 years (range 5.6-17.0 
      years) and male/female ratio was 19/7. Donors were Human leukocyte antigen 
      (HLA)-identical sibling in 18 patients, HLA-identical other relatives in six 
      patients, and HLA 1-antigen mismatched sibling in two patients. Conditioning 
      regimen consisted of Flu, cyclophosphamide, and antithymocyte globulin. RESULTS: 
      All patients engrafted but one developed poor graft function and underwent second 
      HSCT. Acute graft versus host disease (GVHD) (>/=grade 2) occurred in two patients 
      (7.6%) and chronic GVHD in one patient (3.9%). Three patients developed 
      venoocclusive disease (11.5%). Survival rate was 96.2% (25/26) at a median 
      follow-up of 54 months (10-131 months) and all patients who survived were in good 
      clinical condition. None of the patients developed secondary malignancy during 
      the follow-up period. CONCLUSIONS: The present study from Turkey, a middle-income 
      country, shows successful transplant outcome with low toxicity using Flu-based 
      conditioning in patients with FA who underwent HSCT from HLA-related donors.
CI  - (c) 2015 Wiley Periodicals, Inc.
FAU - Kuskonmaz, Baris
AU  - Kuskonmaz B
AD  - Division of Bone Marrow Transplantation, Faculty of Medicine, Department of 
      Pediatrics, Hacettepe University, Ankara, Turkey.
FAU - Unal, Sule
AU  - Unal S
AD  - Division of Hematology, Faculty of Medicine, Department of Pediatrics, Hacettepe 
      University, Ankara, Turkey.
FAU - Bayhan, Turan
AU  - Bayhan T
AD  - Division of Hematology, Faculty of Medicine, Department of Pediatrics, Hacettepe 
      University, Ankara, Turkey.
FAU - Aytac Eyuboglu, Selin
AU  - Aytac Eyuboglu S
AD  - Division of Hematology, Faculty of Medicine, Department of Pediatrics, Hacettepe 
      University, Ankara, Turkey.
FAU - Tavil, Betul
AU  - Tavil B
AD  - Division of Hematology, Faculty of Medicine, Department of Pediatrics, Hacettepe 
      University, Ankara, Turkey.
FAU - Cetin, Mualla
AU  - Cetin M
AD  - Division of Hematology, Faculty of Medicine, Department of Pediatrics, Hacettepe 
      University, Ankara, Turkey.
FAU - Gumruk, Fatma
AU  - Gumruk F
AD  - Division of Hematology, Faculty of Medicine, Department of Pediatrics, Hacettepe 
      University, Ankara, Turkey.
FAU - Uckan Cetinkaya, Duygu
AU  - Uckan Cetinkaya D
AD  - Division of Bone Marrow Transplantation, Faculty of Medicine, Department of 
      Pediatrics, Hacettepe University, Ankara, Turkey.
LA  - eng
PT  - Clinical Trial
PT  - Journal Article
DEP - 20151224
PL  - United States
TA  - Pediatr Blood Cancer
JT  - Pediatric blood & cancer
JID - 101186624
RN  - 0 (Antilymphocyte Serum)
RN  - 0 (Myeloablative Agonists)
RN  - 8N3DW7272P (Cyclophosphamide)
RN  - FA2DM6879K (Vidarabine)
RN  - P2K93U8740 (fludarabine)
SB  - IM
MH  - Adolescent
MH  - Antilymphocyte Serum/administration & dosage/adverse effects
MH  - Child
MH  - Child, Preschool
MH  - Cyclophosphamide/administration & dosage/adverse effects
MH  - Fanconi Anemia/*surgery
MH  - Female
MH  - Graft vs Host Disease/epidemiology
MH  - Hematopoietic Stem Cell Transplantation/*methods
MH  - Humans
MH  - Male
MH  - Myeloablative Agonists/*administration & dosage/adverse effects
MH  - Tissue Donors
MH  - Transplantation Conditioning/*methods
MH  - Turkey
MH  - Vidarabine/administration & dosage/*analogs & derivatives
OTO - NOTNLM
OT  - Fanconi anemia
OT  - fludarabine
OT  - transplantation
EDAT- 2015/12/26 06:00
MHDA- 2016/07/19 06:00
CRDT- 2015/12/26 06:00
PHST- 2015/03/19 00:00 [received]
PHST- 2015/10/20 00:00 [revised]
PHST- 2015/10/22 00:00 [accepted]
PHST- 2015/12/26 06:00 [entrez]
PHST- 2015/12/26 06:00 [pubmed]
PHST- 2016/07/19 06:00 [medline]
AID - 10.1002/pbc.25844 [doi]
PST - ppublish
SO  - Pediatr Blood Cancer. 2016 Apr;63(4):695-700. doi: 10.1002/pbc.25844. Epub 2015 
      Dec 24.