Summary40 Hemophilia Centers from the USA, Canada, Europe and Japan referred to the International Registry 204 patients with haemophilia A, treated by Immune Tolerance (IT) Protocols over the past two decades because of the presence of an inhibitor to FVIII. 82% of the patients were high responders, while IT was started with low levels of inhibitor (<10 BU) in most (57.3%) of the cases. 69 patients (33.8%) were given the highest FVIII dosage (≥200 IU/kg/day), 71 (34.8%) intermediate dosages (50 - <200) and 64 (31.4%) the lowest dosages (<50). Of 158 patients persevering with treatment long enough to judge the outcome thereof, 107 (67.7%) achieved tolerance, 12 (7,6%) had a partial response, while 39 (24.7%) did not respond. Multivariate logistic regression analysis showed that two variables were independently associated with the highest probability of success: the use of high dose protocols (≥100 IU/kg/day) (p <.0001) and the presence of low levels of inhibitor (<10 BU) at enrollment (p = .004). The Kaplan-Meier inhibitor-free survival curve showed that tolerance is longlasting: only 1 out of 107 patients relapsed and the longest documented tolerant patient has been inhibitor-free for 16 years. 129 hemophiliacs were HIV Ab-negative at enrollment; of the 118 HIV-screened after the treatment, 18 (13.9%) were found to be HIV Ab-positive. IT can indeed modify the natural history of inhibitors to FVIII in hemophilia.

No abstract

No abstract

No abstract

No abstract

We report our experience with danazol in the treatment of patients with refractory immune thrombocytopenic purpura (ITP). The effects of this drug were investigated in 10 patients, 6 males and 4 females, aged from 40 to 85 years, (median 58 years), with a platelet count below 50 × 10⁹/1. The patients had previously been treated with steroids; one of them had also been unsuccessfully splenectomized. Danazol was administered at a dosage of 600 mg/day for 3 months. Before and after treatment, detection of antiplatelet antibodies was performed. Seven patients were treated for 3 months. One of them showed a transient increase of platelet count, in the others, no significant rise was noted. Six patients experienced side effects during treatment. We think that danazol does not appear to be an alternative therapeutical approach in refractory ITP.

No abstract

A study evaluating the risk of a commercial factor VIII (FVIII) concentrate's transmitting the human immunodeficiency virus (HIV) was carried out on hemophiliacs, by using multiple serological markers and the polymerase chain reaction (PCR). Twenty-nine hemophiliacs, negative for HIV antibodies, were treated for 18 months with a concentrate that had been inactivated by solvent-detergent. HIV-1 antibodies and antigen were assayed during the follow-up period. At the end of the study, all patients were also tested by the HIV 1 + 2 combined antibody assay; Western blot (WB) antibody analysis; and in eight cases, by an HIV-1 PCR technique. Patients received a yearly median FVIII dose of 35,330 IU (range 3,300-306,000); the median number of lots given to each patient was 6 (1-45). During the follow-up period and at the end of the study, HIV-1 antibodies and antigen were not detected in any of the subjects. The HIV 1 + 2 combined assay and WB analysis carried out only at the end of the study were negative. HIV-1 PCR was negative in all the tested patients. This study has shown that this solvent-detergent-treated FVIII concentrate did not transmit HIV.