XVIII Congress of the Italian Society for Hemostasis and Thrombosis Research, Rome, Sept. 30-Oct. 3, <strong>2004</strong>1<strong>89</strong>PO-185QUALITY OF LIFE IS ASSOCIATED TO ORTHOPEDIC STATUS INHEMOPHILIACS WITH INHIBITORSScalone L,* Gringeri A,° Mannucci PM,°Mantovani LG* for the COCIS Investigators*Center of Pharmacoeco<strong>no</strong>mics, University of Milan,Italy; °IRCCS Maggiore Hospital, Milan, ItalyAim. We evaluated Quality of Life (QoL) in hemophiliapeople with inhibitors and investigated if QoLis associated to patients’ clinical status. Methods: TheCOCIS (Cost Of Care of InhibitorS) study was a longitudinalstudy involving 52 hemophiliacs (median age35, 15-64) with inhibitors, 98% high responders(>5BU), sequentially enrolled at 11 Italian HemophiliaCare Centers. Information on demographics, clinicalstatus, health-care resources consumption wasprospectively collected. QoL was investigated bymeans of generic instruments: results from EuroQol-5D are reported. Results. The majority of patientsreported some/moderate problems in domainspain/discomfort (72%), mobility (66%) and usualactivities (54%). Around one third declaredsome/moderate problems with anxiety/depression(34%) and self-care (32%). No more than 6% ofpatients reported severe problems in any of thedomains. The EQ-VAS was <strong>no</strong>rmally distributed, witha median value of 66 (30-95). Patients with moresevere orthopedic impairment perceived more problemswith physical component of QoL, while <strong>no</strong> associationwas found with anxiety or depression. TheOrthopedic Joint Score, a measure of the orthopedicfunctioning, significantly correlated with EQ-VAS(Pearson’s R is -0.57, p
190PostersAt the last follow-up, 11 patients with severe hemophilia,treated on demand, showed a mean orthopedicjoint score of 4 and a mean number of hemartrhosesper year of 2.3; 9 patients with moderatehaemophilia showed a mean orthopedic joint scoreof 1.1 and a mean number of haemartrhoses per yearof 1.5. Sixteen patients began prophylaxis at a medianage of 6.5 (range 2.1-15.6). Nine out of 16received prophylaxis for a period 45 weeks per year.Before starting prophylaxis and at last control, mea<strong>no</strong>rthopaedic joint score was 3.9 and 0.66 respectively(p=0.0069), and mean number of haemartrhosesper year was 3.5 and 0.47 respectively (p=0.00035).Seven/16 received prophylaxis for a period < 45weeks per year; they showed at last control a mea<strong>no</strong>rthopaedic joint score of 1.2 and a mean number ofhaemarthroses per year of 2.5. Two patients are <strong>no</strong>tevaluable because in immu<strong>no</strong>tolerance program.Prophylaxis in severe haemophilia patients results ina significantly better orthopaedic outcome than theone observed in severe and moderate hemophiliapatients on demand treatment. In order to obtaingreatest efficacy, patients on prophylaxis must betreated without discontinuation.PO-188KIDNEY AND BLADDER STONES IN HAEMOPHILIA PATIENTSMusso R, Cultrera D,* Chiarenza A,* D'Arpa S,Cipolla N, Burgio N, Giustolisi R*Haemophilia and Thrombosis Regional ReferenceCenter, *Division of Clinical Haematology, Universityof Catania, ItalyTo date, there are few data about kidney stones(KS) and bladder stones (BS) in haemophilia patientsand it is still unk<strong>no</strong>wn if these complications fall inthe general population prevalence or should be considereda new emergent concern in haemophilia. Westudied our large cohort of haemophiliacs to collectin an uniform way clinical data regarding KS and BSprevalence. One hundred and twenty two [males 109,females 15 including severe Factor XI:C deficiency(n=8) and obligate symptomatic carriers (n=5)patients, with a mean age 27.07 years] were followed.KS (n=16) and BS (n=7) were found in 19 (13males, 6 females) for a total prevalence of 15.8% vs5-10% in the general population. We observed ayounger age (23.4 years) in hemophiliac patientscompared with <strong>no</strong>rmal population (peak of frequencybetween 40-70 years). From our observation, it isnecessary to evaluate a larger haemophilia patientnumber of different Centres to confirm our data. Amore dept research on this pathology will be usefulto k<strong>no</strong>w which other factors among the anamnesticand/or recurrent hematuria, substitution therapiesor antiphibri<strong>no</strong>litics agents may play a key role inthe pathogenesis of these complications. If thesedata would be confirm, a new chapter will be addedin hemophilia related pathologies.PO-1<strong>89</strong>IN VIVO RECOVERY (IVR), EFFICACY AND SAFETY OFSOLVENT/DETERGENT (S/D) TREATED PLASMA IN PATIENTSWITH RARE INHERITED COAGULATION DISORDERS (RICDs)Mancuso ME,* Santagosti<strong>no</strong> E,* Morfini M,^Schiavoni M,° Tagliaferri A, # Barillari G,°°Mannucci PM**Angelo Bianchi Bo<strong>no</strong>mi Hemophilia and ThrombosisCenter, IRCCS Maggiore Hospital, Milan, Italy;^Hemophilia Center, Careggi Hospital, Florence,Italy; °Hemophilia & Thrombosis Center, PoliclinicoHospital, Bari, Italy; # Hemophilia Center, ParmaHospital, Parma, Italy; °°Hemophilia Center, S. Mariadella Misericordia Hospital, Udine, ItalyVirus-inactivated concentrates are <strong>no</strong>t availablefor most RICDs and virus-inactivated plasma is recommendedas therapeutic alternative. This prospectivemulticenter study evaluated the efficacy andsafety of S/D plasma (Octaplas, Kedrion, Italy), theIVR of the deficient factors and the dose regimensrequired for RICDs. Seven-teen patients wereenrolled (age: 13-65 yr; M/F: 6/11): 8 had severedeficiencies (1 afibri<strong>no</strong>genemia, 2 FV, 1 FX, 4 FXI)and 9 had mild deficiencies (2 FV:20-27%; 6 FV/FVI-II:12-20/6-47%; 1 FXI:30%). Seronegative patientsfor HIV, HCV, HBV, HAV and parvovirus B19 were 17,14, 7, 5, and 1, respectively. Reasons for treatment:delivery (2), mi<strong>no</strong>r surgery (5) and major surgery (10).Octaplas (6-29 mL/Kg) was given at 6-96h intervals,according to factor half-lives and clinical features fora mean treatment duration of 5 days. FVIII and/orDDAVP were associated for replacement of FV/FVIIIdeficiency. Mean IVRs: FV 1.8 dL/Kg (0.4-2.9), FVIII0.8 dL/Kg (0.4-1.5), FXI 1.3 dL/Kg (0.6-1.8), FX 1.8dL/Kg and fibri<strong>no</strong>gen 1.7 dL/Kg. Two perioperativehaemorrhages, in a patient maintaining FXI levels20-41% and in a patient with FV 43%, were controlledby surgical haemostasis and further Octaplasadministrations. Postoperative bleeding in a FV/FVI-II deficient patient occurred when FVIII was 18% andit was controlled by giving FVIII instead of DDAVP.Rash during Octaplas infusion occurred in 1/3patients who had had reactions to FFP. No viral seroconversionwas observed during one-year follow-up.Octaplas was safe and effective in patients withRICDs and it should be preferred when virus-inactivatedconcentrates are <strong>no</strong>t available.haematologica vol. <strong>89</strong>(suppl. n. 8):september <strong>2004</strong>
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