Thrombotic thrombocytopenic purpura (TTP) is a fatal disease in which platelet-rich microthrombi cause end-organ ischemia and damage. TTP is caused by markedly reduced ADAMTS13 (a disintegrin and metalloproteinase with a thrombospondin type 1 motif, member 13) activity. ADAMTS13 autoantibodies (autoAbs) are the major cause of immune TTP (iTTP), determining ADAMTS13 deficiency. The pathophysiology of such autoAbs as well as their prognostic role are continuous objects of scientific studies in iTTP fields. This review aims to provide clinicians with the basic information and updates on autoAbs’ structure and function, how they are typically detected in the laboratory and their prognostic implications. This information could be useful in clinical practice and contribute to future research implementations on this specific topic.

Background/Objectives: After an initial course of anticoagulation for deep vein thrombosis (DVT), identifying patients at higher risk of recurrence remains a clinical challenge. The role of residual vein thrombosis (RVT) in this setting is still debated, as most available evidence derives from retrospective studies or from the Warfarin era. We conducted a study to evaluate the incidence of RVT in patients treated with direct oral anticoagulants (DOACs) and to identify the clinical factors associated with its persistence. We also compared the outcomes from the two most prescribed drugs in Italy, Apixaban and Rivaroxaban. Methods: A total of 113 patients with newly diagnosed DVT underwent follow-up visits at 6 weeks (T1), 3 months (T2) and 6 months (T3) after diagnosis. RVT was assessed by compression ultrasonography and clinical, family and pathological history data were collected. Ninety-six patients were included in the final statistical analysis. Results: RVT was detected in 68.2%, 52.1% and 37.7% of patients at T1, T2 and T3, respectively. Factors significantly associated with RVT at T2 were male sex, femoral vein involvement and a family history of DVT. No significant differences were observed between Apixaban and Rivaroxaban. Prior episodes of thrombosis, smoking, diabetes and obesity were not associated with RVT at 3 months. Conclusions: Our findings confirm that RVT rates progressively decrease over time, as previously observed in the Coumarins era, but suggest a stronger early response to DOACs, particularly during the first three months of therapy. Moreover, DOACs appear to provide more effective protection in patients with risk factors for venous disease.

Background: Acquired hemophilia A (AHA) is a rare bleeding disease due to autoantibodies directed against clotting factor VIII (FVIII). Treatment of AHA consists of inhibitor eradication with immunosuppressive therapy (IST) and prompt control of bleeding obtained with bypassing agents or recombinant porcine FVIII (rpFVIII). The latter has recently been licensed for management of acute bleeding in AHA. Unlike treatment with bypassing agents, rpFVIII can be monitored to provide a successful hemostatic effect and avoid overtreatment. Correlation between rpFVIII inhibitor titers and efficacy of rpFVIII treatment remains a matter of debate. Methods: We report three cases of AHA in which rpFVIII was successfully used with an unconventional schedule despite the presence of medium–high titers of the rpFVIII. The modified Nijmegen–Bethesda inhibitor assay (NBA) was used to dose porcine FVIII inhibitors. Result: The presence of rpFVIII inhibitors prior to the exposition to susoctocog-alfa, that may suggest a cross-reactivity with human FVIII inhibitors, did not affect hemostasis. Conclusion: In our experience, rpFVIII demonstrates safety and efficacy in the presence of rpFVIII inhibitors and using an unconventional schedule in both the perioperative and outpatient settings. Laboratory measurement of inhibitors against rpFVIII during treatment is described for the first time.

Background/Objectives: The approach to physical activity in people with hemophilia (PwH) is still conditioned by many difficulties. Thus, a prospective observational pilot study has been carried out aiming to evaluate how an adequate and controlled training program can slow down the onset or evolution of arthropathy and improve musculoskeletal health and quality of life. Methods: Performed from April 2022 to April 2023, this study involved nine severe hemophilic A and B patients, aged > 18 years old, on regular prophylaxis with replacement products. Participants, without changing the usual prophylaxis schedule and maintaining a trough level of at least 20% FVIII/FIX before training, were involved in physical activity twice a week. Results: After 12 months, no increase in annual bleeding ratio (ABR) was observed, and baseline joint status (as assessable by HEAD US score, HJHS, and NRS) was maintained. Even if not statistically significant, a trend toward improvement in mean HEAD US score (15.55 vs. 13.11) and HJHS (14.4 vs. 11) from baseline was observed. Some of the physical tests performed showed a significant improvement at 6 months and 12 months from baseline (5 Rep Sit to Stand, Sit and Reach, and 6-minute Walking Test), meaning an improvement in leg strength, dorsal flexibility, and aerobic resistance. Conclusions: This is the first pilot study evaluating at 360 degrees the safety and impact of a controlled physical activity in PwH. No participant experienced bleedings or a worsening in joint status, but they experienced an improvement in articular functionality. Without changing the usual prophylaxis, scheduling training sessions according to individual pharmacokinetics turned out to be a safe and a cost-effective approach.