Veno-occlusive Hepatic Disease Follow-up
- Author: James L Harper, MD; Chief Editor: Jennifer Reikes Willert, MD more...
Further Inpatient Care
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- The lack of sensitive and specific diagnostic tools warrants prophylactic intervention in patients with veno-occlusive disease (VOD). Several preventive measures have been studied, mostly in small nonrandomized and retrospective studies. Effective prophylaxis with low-dose or low-molecular weight heparin has frequently been reported.[44, 45, 46, 47] However, a large prospective cohort study performed by the European Group for Blood and Marrow Transplantation (EBMT) demonstrated no benefit.
- The efficacy of prostaglandin E1 (PGE1) was investigated, but a prospective trial showed no convincing evidence beyond confirming the known considerable toxicities.[48, 49, 47, 44] Ursodeoxycholic acid was of no benefit in a prospective randomized trial. Antithrombin III (ATIII) was also studied only in small series or in combination with defibrotide.[51, 52] Additionally, none of these drugs demonstrated superior therapeutic efficacy.
- Defibrotide is an effective therapeutic modality that is becoming available. Defibrotide was assessed in a retrospective study of 45 children with veno-occlusive disease. The study showed that, in the subgroup of 22 patients with severe disease, younger age and early start of defibrotide administration was associated with a significantly superior outcome. In a subgroup of patients who underwent transplantation for malignant infantile osteopetrosis, the incidence rate of veno-occlusive disease exceeded 60%. Because of this high incidence of veno-occlusive disease, defibrotide prophylaxis was initiated in 9 consecutive patients who underwent transplantation. In this group, only one patient (11.1%) was diagnosed with moderate veno-occlusive disease.
- A prospective randomized international multicenter trial that intends to conclude if prophylactic defibrotide is superior to therapeutic defibrotide in children at high risk for developing veno-occlusive disease during stem cell transplantation is currently being conducted in Europe (NIH Trial Number: NCT00272948).
- A Cochrane Review concluded that there is low or very low quality evidence that ursodeoxycholic acid may reduce the incidence of hepatic VOD, all-cause mortality and mortality due to VOD in haematopoietic stem cell transplantation recipients. The authors also added that there is insufficient evidence to support the use of heparin, low molecular weight heparin, defibrotide, glutamine, fresh frozen plasma, antithrombin III, and prostaglandin E1.
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- A transplantation-trained physician with experience with this disease should administer primary care for patients at a center with an active hemostasis laboratory.
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- Post–bone marrow transplantation (BMT) veno-occlusive disease may be reduced with pretransplantation screening of both patients and donors.
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- Commonly observed complications include the following:
- Hepatic failure: Some degree of hepatic dysfunction is observed in all cases of post-BMT hepatic veno-occlusive disease; however, in rare severe cases, overt liver failure may be observed.
- Renal failure: This may be secondary to hepatorenal syndrome, as well as direct injury by the vasculopathy. In patients who have undergone transplantation, numerous frequently used nephrotoxic drugs (eg, vancomycin, amphotericin B, cyclosporine) can result in preexisting renal dysfunction and loss of renal function reserve. Separating the effects of the drugs from the effects of veno-occlusive disease may be difficult.
- Pulmonary failure
- Neurologic compromise
- Increased risk of infectious complications due to peritoneal drainage and transfer of an immunodeficient patient to intensive care with no laminar air flow units
- Severe consumptive coagulopathy with an increased risk for thrombosis and bleeding
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