PMID- 27872734
OWN - NLM
STAT- PubMed-not-MEDLINE
LR  - 20200930
IS  - 2035-3006 (Print)
IS  - 2035-3006 (Electronic)
IS  - 2035-3006 (Linking)
VI  - 8
IP  - 1
DP  - 2016
TI  - Allogeneic Hematopoietic Stem Cell Transplantation for Adult Patients with 
      Fanconi Anemia.
PG  - e2016054
LID - e2016054
AB  - BACKGROUND AND OBJECTIVES: Fanconi anemia (FA) is a rare genetic disorder caused 
      by an impaired DNA repair mechanism which leads to an increased tendency toward 
      malignancies and progressive bone marrow failure. The only curative management 
      available for hematologic abnormalities in FA patients is hematopoietic stem cell 
      transplantation (HSCT). This study aimed to report the results of HSCT in adult 
      or adolescent FA patients. PATIENTS AND METHODS: Twenty FA patients with ages of 
      16 or more who underwent HSCT between 2002 and 2015 enrolled in this study. The 
      stem cell source was peripheral blood, and all patients had a full human 
      leukocyte antigen (HLA) matched donor, 19 patients had a sibling donor, and one 
      had full matched other related. Indications for HSCT were severe bone marrow 
      failure or dependence on blood products transfusion and failure of medical 
      treatment to sustain peripheral blood elements at an acceptable level. RESULTS: 
      Eleven patients were female and 9 male (55% and 45%). Mean age was 24.05 years. 
      Mortality rate was 50% (n=10), and the leading cause of death was graft versus 
      host disease (GVHD) which occurred in 5 patients (4 cases from acute GVHD and one 
      from chronic GVHD). Survival analysis showed an overall 5-year survival of 53.63% 
      (95% confidence interval: 29.53%-72.74%) and 13 year survival of 45.96 % (95% 
      confidence interval: 22.08%-67.03%) among patients. CONCLUSION: HSCT is the only 
      curative management for bone marrow failure in FA patients. But the high rate of 
      mortality and morbidity in adolescent and adult patients makes it a challenging 
      issue.
FAU - Fumani, Hosein Kamranzadeh
AU  - Fumani HK
AD  - Hematology, Oncology and Stem Cell Transplantation Research Center; Tehran 
      University of Medical Sciences, Tehran, Iran.
FAU - Zokaasadi, Mohammad
AU  - Zokaasadi M
AD  - Hematology, Oncology and Stem Cell Transplantation Research Center; Tehran 
      University of Medical Sciences, Tehran, Iran.
FAU - Kasaeian, Amir
AU  - Kasaeian A
AD  - Hematology, Oncology and Stem Cell Transplantation Research Center; Tehran 
      University of Medical Sciences, Tehran, Iran.
FAU - Alimoghaddam, Kamran
AU  - Alimoghaddam K
AD  - Hematology, Oncology and Stem Cell Transplantation Research Center; Tehran 
      University of Medical Sciences, Tehran, Iran.
FAU - Mousavi, Asadollah
AU  - Mousavi A
AD  - Hematology, Oncology and Stem Cell Transplantation Research Center; Tehran 
      University of Medical Sciences, Tehran, Iran.
FAU - Bahar, Babak
AU  - Bahar B
AD  - Hematology, Oncology and Stem Cell Transplantation Research Center; Tehran 
      University of Medical Sciences, Tehran, Iran.
FAU - Vaezi, Mohammad
AU  - Vaezi M
AD  - Hematology, Oncology and Stem Cell Transplantation Research Center; Tehran 
      University of Medical Sciences, Tehran, Iran.
FAU - Ghavamzadeh, Ardeshir
AU  - Ghavamzadeh A
AD  - Hematology, Oncology and Stem Cell Transplantation Research Center; Tehran 
      University of Medical Sciences, Tehran, Iran.
LA  - eng
PT  - Journal Article
DEP - 20161101
PL  - Italy
TA  - Mediterr J Hematol Infect Dis
JT  - Mediterranean journal of hematology and infectious diseases
JID - 101530512
PMC - PMC5111526
EDAT- 2016/11/23 06:00
MHDA- 2016/11/23 06:01
PMCR- 2016/01/01
CRDT- 2016/11/23 06:00
PHST- 2016/08/03 00:00 [received]
PHST- 2016/10/10 00:00 [accepted]
PHST- 2016/11/23 06:00 [entrez]
PHST- 2016/11/23 06:00 [pubmed]
PHST- 2016/11/23 06:01 [medline]
PHST- 2016/01/01 00:00 [pmc-release]
AID - mjhid-8-1-e2016054 [pii]
AID - 10.4084/MJHID.2016.054 [doi]
PST - epublish
SO  - Mediterr J Hematol Infect Dis. 2016 Nov 1;8(1):e2016054. doi: 
      10.4084/MJHID.2016.054. eCollection 2016.