Spleen tyrosine kinase (Syk) signaling is central to phagocytosis‐based, antibody‐mediated platelet destruction in adults with immune thrombocytopenia (ITP). Fostamatinib, an oral Syk inhibitor, produced sustained on‐treatment responses in a phase 2 ITP study. In two parallel, phase 3, multicenter, randomized, double‐blind, placebo‐controlled trials (FIT1 and FIT2), patients with persistent/chronic ITP were randomized 2:1 to fostamatinib (n = 101) or placebo (n = 49) at 100 mg BID for 24 weeks with a dose increase in nonresponders to 150 mg BID after 4 weeks. The primary endpoint was stable response (platelets ≥50 000/μL at ≥4 of 6 biweekly visits, weeks 14‐24, without rescue therapy). Baseline median platelet count was 16 000/μL; median duration of ITP was 8.5 years. Stable responses occurred in 18% of patients on fostamatinib vs. 2% on placebo (P = .0003). Overall responses (defined retrospectively as ≥1 platelet count ≥50 000/μL within the first 12 weeks on treatment) occurred in 43% of patients on fostamatinib vs. 14% on placebo (P = .0006). Median time to response was 15 days (on 100 mg bid), and 83% responded within 8 weeks. The most common adverse events were diarrhea (31% on fostamatinib vs. 15% on placebo), hypertension (28% vs. 13%), nausea (19% vs. 8%), dizziness (11% vs. 8%), and ALT increase (11% vs. 0%). Most events were mild or moderate and resolved spontaneously or with medical management (antihypertensive, anti‐motility agents). Fostamatinib produced clinically‐meaningful responses in ITP patients including those who failed splenectomy, thrombopoietic agents, and/or rituximab. Fostamatinib is a novel ITP treatment option that targets an important mechanism of ITP pathogenesis.
The results of our prospective study have revealed great diversity in the treatment regimens used to manage MM in real-life practice. This diversity was linked to factors such as novel agent accessibility and evolving treatment recommendations. Our results provide insight into associated clinical benefits.
IntroductionThe Public Access to Defibrillation (PAD) program was designed to raise the awareness of sudden cardiac death (SCA) pre-hospital management among the community. The goal of the following research was to confirm the final impact of the Polish PAD program on various resident groups that differ by age, training and education level.Material and methodsThe trial total number of participants reached 404 people from three cities divided into two groups. In group one (n = 295) were randomly selected people inside the trial area and in group two (n = 109) we had individuals who were theoretically trained in basic life support (BLS) algorithms, including the use of an automatic external defibrillator (AED). The research method was based on two different questionnaires completed by participants from each group.ResultsThe greatest knowledge of SCA, as well as the use of AED, and the best practical skills, were possessed by the residents of cities with a population over 100 000, aged between 18 and 30 years, who completed secondary or higher education (31.7%). The group with the smallest knowledge about SCA lived in the country (10.7%). The second group with little knowledge of the subject consisted mostly of individuals with primary education (4.19%) or professional abilities and over 50 years old (2.16%).ConclusionsThere must be some actions taken in order to increase the community awareness concerning automatic defibrillation. Training on AED and the possibility of practical exercise needs to be organized and should be conducted especially among residents of the countryside and people under 30 or over 50 years old.
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