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2016 ; 22
(7
): 1306-1312
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gab.com Text
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English Wikipedia
Defibrotide for Treatment of Severe Veno-Occlusive Disease in Pediatrics and
Adults: An Exploratory Analysis Using Data from the Center for International
Blood and Marrow Transplant Research
#MMPMID27108694
Strouse C
; Richardson P
; Prentice G
; Korman S
; Hume R
; Nejadnik B
; Horowitz MM
; Saber W
Biol Blood Marrow Transplant
2016[Jul]; 22
(7
): 1306-1312
PMID27108694
show ga
Veno-occlusive disease (VOD) is an early and serious complication of
hematopoietic cell transplantation (HCT) that is associated with inferior
survival, particularly when it is complicated by multiorgan failure (severe VOD).
We evaluated the efficacy of defibrotide in the treatment of severe VOD using
observational data from the Center for International Blood and Marrow Transplant
Research (CIBMTR). Eight thousand three hundred forty-one patients treated by HCT
between 2008 and 2011 were identified from the CIBMTR clinical database; 3.2% met
criteria for VOD and 1.2% met criteria for severe VOD. Patients with a diagnosis
of VOD as reported to the CIBMTR by their transplanting centers, who had no prior
history of cirrhosis, and who had a maximum total bilirubin level > 2.0 mg/dL by
day +100 post-HCT were selected for study. Severe VOD was defined as VOD
occurring in the setting of renal impairment requiring dialysis or any
noninfectious pulmonary abnormality. Patients with severe VOD were divided into 2
groups for analysis: those treated with defibrotide (n = 41) and those not
treated with defibrotide (n = 55). Patients in the nondefibrotide group were
older, were more likely to be male, were more likely to have a history of
previous fungal infection, and had a higher proportion of clinically significant
pre-existing disease or organ impairment. Survival rate at day +100 was 39% (95%
CI, 24.8% to 54.3%) in patients receiving defibrotide and 30.9% (95% CI, 19.5% to
43.6%) in those not receiving defibrotide. Resolution rate of VOD at day +100 was
51% in the defibrotide group and 29% in the nondefibrotide group (difference,
22.1%; 95% CI, 2.6% to 42%). The results of our study are consistent with
previously reported experiences with defibrotide, confirm the poor outcome of
this syndrome, and suggest defibrotide is effective in the treatment of severe
VOD.