Hepatic veno-occlusive disease (VOD), a common complication of hematopoietic stem cell transplantation (HSCT), is associated with significant morbidity and mortality. The diagnosis is mainly based on clinical criteria that are neither sensitive nor specific enough to establish early diagnosis. CA-125 is a tumor marker, which may also increase in chronic liver disease, especially with ascites formation. Since VOD is known to result from liver injury and is associated with ascites, we examined whether CA-125 can be used as a serum marker for early diagnosis of VOD.

Methods: 74 consecutive patients undergoing HSCT were prospectively followed for clinical signs and symptoms of VOD. Serum samples for CA-125 were taken before conditioning, on day 0 and then every 7 days until patient’s discharge from hospital. CA-125 serum levels were measured by ELISA assay.

Results: 19 of 74 patients undergoing HSCT developed VOD according to Jones criteria. 89% of these patients underwent allogeneic stem cell transplantation, 47% received the regimen containing busulfan/cytoxan and 37% were treated with total body irradiation (TBI) and cytoxan. 26% of the patients had severe VOD. Diagnosis of VOD was established on average on day 7 (range 2–20). No difference in CA-125 was observed pre-transplant between the two groups (VOD versus non-VOD), but CA-125 levels increased in 68% of the patients with VOD as opposed to only 15% of those without VOD (p<0.0001) on average on day 9 post transplant. In addition, the peak CA-125 levels were 149.2u/ml versus 45.6u/ml (p<0.0001), respectively. On day 7 post transplant a cut off level of 43u/ml can detect VOD with specificity and sensitivity of 79% and 72%, respectively. The specificity rose to 94% in patients with normal CA-125 levels pre-transplant.

In conclusion: CA-125 levels rise early in patients developing VOD with a good sensitivity and specificity and can therefore be used as a marker in the diagnosis of VOD. Whether CA-125 is a true predictor for the development of VOD or is a surrogate marker needs to be evaluated prospectively by measuring day serum CA-125 levels starting on day 0. This is currently being evaluated in our center.

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