Haematologica 2004;89: supplement no. 8 - Supplements ...

XVIII Congress of the Italian Society for Hemostasis and Thrombosis Research, Rome, Sept. 30-Oct. 3, 2004187the involvement of different experts (patients, physicians,nurses) in order to have a standardised andvalid questionnaire. The results of the qualitativestudy show already that the Haem-A-QoL is wellacceptedby adult haemophiliacs.PO-181SEVERE BLEEDING TENDENCY IN A PATIENT WITH MILDHEMOPHILIA B, FACTOR X DEFICIENCY AND LIVER CIRRHOSIS:SUCCESSFUL PROPHYLAXIS WITH PROTHROMBIN COMPLEXCONCENTRATECimino E, Coppola A, Sorrentino P,* Varricchione N,Di Minno M, Lombardini D, Tufano A, Di Minno GRegional Reference Centre for Coagulation Disease,and *Hepatology Unit, Dep. of Clinical and ExperimentalMedicine, “Federico II” University, Naples,ItalyA 59 yr-old man with a history of mild hemophiliaB was referred to our Institution because ofincreasing severity of a bleeding tendency, includingtwo hemorrhages from gastro-esophageal varices.On admission the patient, who had a long-lastinghistory of chronic hepatitis C, showed a clear-cutclinical picture of cirrhosis with severe portal hypertension.An unusual prolonged PT and aPTT andreduced Factor (F) X levels (10%) led to the diagnosisof combined mild FX deficiency. This was confirmedin his two sons. In several occasions FIX concentrateinfusions were unable to stop joint bleedingand the patient needed hospitalization and freshfrozen plasma or prothrombin complex concentrate(PCC) infusions. Prophylaxis with 40 IU/Kg FIX concentratetwice or thrice weekly did not reduce thepatient’s bleeding tendency: this led to progressivejoint disability. A prophylaxis regimen with 30 IU/KgPCC (Uman Complex D.I, Kedrion, Italy) was startedon April 2003, after a new episode of gastric bleeding.The patient did not show any symptom or signof cardiovascular disease nor significant risk factorsand thrombophilic abnormalities. To reduce bleedingand hospitalization frequency and to enable physicaltherapy for joint function recovery, a similarschedule was also prescribed at home. Clinical andlaboratory follow-up was carried out weekly andthen monthly; mild increase of D-Dimer was detectedbetween 4 and 24 hours after PCC infusion. However,no adverse event was documented over 1-yrtreatment. To date no further significant bleedingepisode occurred and the patient’s quality of life significantlyimproved. Thus, PCC prophylaxis has beenhelpful to manage the bleeding tendency in thispatient whose severity had been increased by thecoexistence of more genetic and acquired coagulationabnormalities.PO-182TGF-ETA TO EVALUATE ANTIVIRAL THERAPY IN HEMOPHILICPATIENTS WITH HCV-RELATED CHRONIC HEPATITISCoppola A, Tarantino G,* Conca P,* Cimino E,Sorrentino P,* Ariello M,* Scopacasa F,^Grimaldi E,^ Di Minno GRegional Reference Centre for Coagulation Disease,and *Sector of Hepatology in Internal Medicine,Dep. of Clinical and Experimental Medicine; ^Dep.Of Biochemistry and Medical Biotechnology;Federico II University, Naples, ItalyChronic hepatitis C (CHC) and its complicationsare a major problem in young-adult hemophiliacs.Treatment with interferon and ribavirin has beenshown effective in reducing hepatic fibrosis (HF). Dueto the limitations of ordinarily performing liver biopsiesin hemophiliacs, few data are available in thissetting. Recently, various serum substances reflectingliver fibrogenesis have been discovered. Amongthem, transforming growth factor-β (TGF-β) plays apivotal role, by stimulating the expression of extracellularmatrix components, inhibiting collagenasecomponents, and promoting the activation of fatstoringhepatic stellate cells towards a myofibroblastphenotype. We evaluated serum levels of this surrogatemarker of HF (ELISA) in 11 patients with hemophiliaA, aged 29.1±6.3 yrs, with CHC (genotypes1/4), comparable for clinical features and duration ofinfection (25.5±4.4 yrs), undergoing treatment (allnaïve) with peg-interferon α2b+ribavirin (canonicaldose) at baseline, after 6 mo. and 12 mo. (end) oftreatment. As expected, patients showed significantlyhigher TGF-β levels than age-matched controls(44.5 ng/mL, 28.4-48.1 vs. 28.3, 23.2-34.1,median and 95%CI; p=0.004). When compared tobaseline, irrespective of treatment outcome (3patients were non-responders/breakthrough, 8 weresustained responders–SR; for the latter, samples 6mo. after the end of treatment were also collected),TGF-β was significantly reduced after 6 mo. and at12 mo. (37.0 ng/mL, 22.6-44.1 and 27.0 ng/mL, 23.9-43.6, respectively; p