2003; baxter - Supplements - Haematologica

[Acquired Inhibitors in Non-Hemophiliacs]review paperAcquired factor VIII and factorIX inhibitors: a survey of ItalianHemophilia CentersFRANCESCO BAUDO, GIANNI MOSTARDA,haematologica 2003; 88(suppl. n. 12):93-99http://www.haematologica.org/free/immunotolerance2001.pdfFRANCESCO DE CATALDO, WRITING COMMITTEEContributing hemophilia centers: Bari, Schiavon M; Bergamo,Finazzi G; Bologna, De Rosa V; Bolzano, Billio A; CastefrancoVeneto, Tagariello G; Catania, Musso R; Catanzaro, Santoro R;Firenze, Linari S; Milano Niguarda, Mostarda G; Milano Policlinico,Santagostino E; Palermo, Caracciolo C; Parma, TagliaferriAR; Pavia, Gamba G; Perugia, Berrettini M; Pescara, Dragani A;Reggio Emilia, Ghirarduzzi A; Roma, Mazzucconi G; Torino,Schinco P; Verona, Franchini M; Vicenza, Castaman G.Objective. To evaluate the clinical problems relatedto the acquired hemophilia syndrome.Study design. Data collected from the ItalianAcquired Hemophilia Register.Results. Ninety-six cases were registered, 95 withanti-factor VIII and 1 with anti-factor IX. Forty-fivecases (46.8%) were idiopathic, 51 (53.2%) associatedwith different clinical conditions. The overallfollow-up is 7 years (range 1-20). In 31 patients(32.2%) bleeding occurred during or after an intervention.A prolonged activated partial thromboplastintime (aPTT) was present in all the patients inwhom the test was carried out but the inhibitor wasidentified on the occasion of a bleed. Twenty-threepatients did not require treatment for bleeding; 66patients received different therapies and in 58patients bleeding was controlled. An anamnesticresponse occurred in 8 cases unrelated to pregnancy.Sixty-five of the ninety patients evaluable forresponse to the initial anti-hemorrhagic therapy arealso evaluable for the effect of the immunosuppressivetherapy used to suppress the inhibitor. The finalresults of induction therapy are 52 (80% ) completeremissions, 6 (9.3%) partial remissions and 7(10.7%) treatment failures. The majority of thepatients who achieved complete remission receivedsteroids. A 2nd remission was obtained in 10/11patients who relapsed with combined therapy. Theoverall mortality was 16.6%.Correspondence: Francesco Baudo, MD, Thrombosis and HemostasisUnit, Niguarda Hospital, p.za Ospedale Maggiore 3,20162 Milan, Italy. Phone: international +39.02.64442970,Fax: international +39.02.66103898. E-mail:md9821@mclink.itConclusions. Correct evaluation of coagulationscreening tests, in particular the prothrombin time,is mandatory. At present combined immunosuppressivetherapy seems to be a reasonable choice forthe treatment of cases unrelated to pregnancy.©2003, Ferrata Storti FoundationKey words: acquired FVIII and FIX inhibitors; survey ofItalian Hemophilia Centers.Acquired hemophilia is a clinical syndromecharacterized by the sudden onset of bleeding,which may be either spontaneous oroccur after surgery or trauma and is usuallysevere (87% of the cases), in patients with a negativefamily or personal history of hemophilia.The depletion of factor VIII (FVIII), and muchless frequently of factor IX (FIX), is immunemediatedby specific autoantibodies. The incidencereported in the literature varies between 1to 4 cases per million/population per year. 1,2 Theincidence increases with age (median age 60-67years) with an equal sex distribution except inthe younger group because of the relation topregnancy. Acquired hemophilia is commonlyassociated with autoimmune (systemic lupuserythematosus, rheumatoid arthritis, asthma)and neoplastic diseases, hypersensitivity to drugsand pregnancy but in 50% of the cases the conditionis idiopathic. 3-5 Common sites of bleedingare the skin (large ecchymoses), the mucosae(epistaxis, gengivorrhagia, metrorrhagia), themuscles and the retro-peritoneum (hematomata);hemarthroses are very rare. The bleeding canarise spontaneously or after a procedure (positioningof an intravenous catheter, surgery,intramuscular injections). If the bleeding occursin critical sites compression problems mayensue. The severity of the bleeding is not proportionallyrelated to the inhibitor titer. 6 Thereported mortality related to bleeding is 8-22%. 2-5The majority of deaths occur within the firstfew weeks after presentation.haematologica vol. 88(supplement n. 12):september 2003