Objective: Veno-occlusive disease (VOD) is a serious complication of hematopoietic stem cell transplantation (HSCT). If it is not identified and treated earlier, mortality is high. Combination usage of high-dose methyl prednisolone (MPZ) and defibrotide in VOD treatment have been described in some studies. Here, we present a patient with VOD who responded well to high-dose MPZ.
Case report: 14-month-old girl, diagnosed with thalassemia major, received HSCT from her sibling donor with busulfan and cyclo-phosphamide conditioning. On day +11, the patient experienced painful hepatomegaly and elevated total bilirubin (2.25mg/dL) with 7% weight gain from baseline and respiratory distress while under defibrotide prophylaxis. VOD was diagnosed according to the modified Seattle criteria. Fluid and salt restriction were performed, spironolactone was started, and defibrotide was continued. Due to lack of significant improvement in the patient condition after 4 days of defibrotide, HDM was started at dose of 250mg/m2 per dose every 12h on day +15.
Methodology: A day after MPZ, the patient's condition started to improve. After six doses of methylprednisolone, the dose was reduced to 2mg/kg. Then, the dose was reduced by decreasing to half-dose in three-day periods. The defibrotide was discontinued on day +36, and the patient was discharged on day +45. The patient is currently being followed problem-free after 2 years of transplantation with 100% donor chimerism.
Results: VOD treatment response with high-dose MPZ and defibrotide combination can be better than treatment response with defibrotide alone. The easier and cheaper supply of steroids also prevents the treatment delay. In a study, it was shown that receiving high-dose MPZ without defibrotide was also found to be effective in the VOD treatment. The mortality rate in patients with multiple organ failure symptoms in VOD is between 50% and 100%. However, mortality rate can be decreased by early detection of VOD symptoms such as of painful hepatomegaly, weight gain and ascites. This findings may develop before hyperbilirubinemia especially in pediatric patients. Knowing this is important for early diagnosis and treatment of VOD.
Conclusion: As a conclusion; high-dose MPZ was found to be an effective treatment in VOD even at a dose of 250mg/m2 per dose every 12h in aour patient. High-dose MPZ might be an alternative treatment to defibrotide in early phase VOD. Further studies are needed on the efficacy and dosage of MPZ in VOD.
