title: A prospective survey on incidence, risk factors and therapy of hepatic veno-occlusive disease in children after hematopoietic stem cell transplantation.
abstract: OBJECTIVE
  Veno-occlusive disease (VOD) is one of the most frequent complications after stem cell transplantation. We conducted a prospective survey of 244 hematopoietic stem cell transplants in children to determine the incidence of VOD, its main risk factors, treatment and effect on the transplant.
  
  
  METHODS
  Two hundred and forty-four hematopoietic stem cell transplants (HSCT) performed in 220 pediatric patients from 1993 to 2003 were evaluated. The series included 127 males and 93 females with a median age of 6.7 years at the time of transplantation.
  
  
  RESULTS
  VOD was diagnosed following 26 of the 244 transplants (cumulative incidence 11%), but a higher incidence was found in patients with at least one known risk factor for VOD (cumulative incidence 20%). In multivariate analysis, risk factors for VOD were age < 6.7 years; type of VOD prophylaxis, and busulphan-containing conditioning regimens. Routine treatment of VOD was based on supportive care and, starting from 1999, defibrotide was used. All patients were monitored with daily Doppler ultrasound-(US) for early diagnosis of inversion of portal blood flow. Twelve patients developed inversion of portal flow (9 had severe VOD; 3 had moderate VOD) and were promptly started on fibrinolytic and anticoagulant therapy with heparin and recombinant tissue plasminogen activator (rt-PA). Hepatic flow reverted to normal in all 12 patients; only 4 patients ultimately developed multiorgan failure and died. The transplant-related-mortality (TRM) rate in patients with or without inversion of portal flow was 33% vs 7%, (p=0.1). The TRM in patients with or without VOD was 19% vs 8% (p=0.001).
  
  
  CONCLUSIONS
  This study showed that younger age, type of VOD prophylaxis, and busulphan-based conditioning regimens are independent risk factors for VOD. Inversion of portal flow was found in 9 of 10 patients with severe VOD. Doppler US monitoring may be helpful in early identification of the patients with VOD-induced inversion of portal flow who might benefit from therapy with heparin and rt-PA.
authors.affiliation: Clinic of Pediatric Hematology Oncology Department of Pediatrics, University of Padova, Via Giustiniani 3, 35128 Padova, Italy. simone.cesaro @unipd.it
authors.forename: Simone
authors.initials: S
authors.lastname: Cesaro
authors.forename: Marta
authors.initials: M
authors.lastname: Pillon
authors.forename: Enrico
authors.initials: E
authors.lastname: Talenti
authors.forename: Tiziana
authors.initials: T
authors.lastname: Toffolutti
authors.forename: Elisabetta
authors.initials: E
authors.lastname: Calore
authors.forename: Gloria
authors.initials: G
authors.lastname: Tridello
authors.forename: Liliana
authors.initials: L
authors.lastname: Strugo
authors.forename: Roberta
authors.initials: R
authors.lastname: Destro
authors.forename: Maria Vittoria
authors.initials: MV
authors.lastname: Gazzola
authors.forename: Stefania
authors.initials: S
authors.lastname: Varotto
authors.forename: Gabriella
authors.initials: G
authors.lastname: Errigo
authors.forename: Modesto
authors.initials: M
authors.lastname: Carli
authors.forename: Luigi
authors.initials: L
authors.lastname: Zanesco
authors.forename: Chiara
authors.initials: C
authors.lastname: Messina
mesh_terms: D000293:Adolescent; D000367:Age Factors; D002648:Child; D002675:Child, Preschool; D005260:Female; D006306:Health Surveys; D018380:Hematopoietic Stem Cell Transplantation / Q000009:adverse effects; D006504:Hepatic Veno-Occlusive Disease / Q000453:epidemiology* / Q000209:etiology / Q000628:therapy*; D006801:Humans; D015994:Incidence; D007223:Infant; D008297:Male; D011446:Prospective Studies; D012307:Risk Factors