Papers by Dr. Francesco Demartis
Haemophilia, Apr 24, 2017
Introduction: A room temperature stable formulation of recombinant activated factor VII (NovoSeve... more Introduction: A room temperature stable formulation of recombinant activated factor VII (NovoSeven â), allowing convenient storage and therefore improved treatment access, has been developed. Bioequivalence to the previous NovoSeven â was demonstrated in healthy humans, leading to European approval (2008). Although no confirmed cases of neutralising antibodies to rFVIIa in patients with haemophilia A or B have been observed with the original formulation, changes in formulation or storage condition may alter immunogenicity. Aim: SMART-7 TM was designed to investigate the safety of NovoSeven â in a real-world setting in patients with haemophilia A or B with inhibitors. Methods: Study medication was not provided by the sponsor, and treatment was at the discretion of the treating physician, in accordance with the local label. Patient baseline information was collected at enrolment. Information on safety, drug exposure and bleeding episodes was collected and FVII antibody screening was encouraged at baseline and performed at the investigator's discretion. Results: Fifty-one patients were enrolled and 31 completed the study. Forty-one adverse events (AEs) were reported in 23 patients; 25 AEs in 14 patients were serious. No thromboembolic events were observed. Although four cases of reduced therapeutic response were reported, FVII antibody screening was negative. Forty-eight patients experienced 618 bleeding episodes and 93.4% of 609 evaluated bleeds were stopped by treatment. Of the 538 bleeding episodes treated with NovoSeven â monotherapy, 94.2% stopped by end of treatment. Conclusion: Data collected during the SMART-7 TM study revealed no treatment-related safety issues and no FVII-binding antibodies for patients treated with NovoSeven â under real-world conditions.
Cambridge …, 1998
Page 1. Contributors Monica Arruda is a candidate for the BSN/MSN in the University of Penn-sylva... more Page 1. Contributors Monica Arruda is a candidate for the BSN/MSN in the University of Penn-sylvania School of Nursing and Senior Research Assistant in the Center for Bioethics at Penn. Her previous work has focused on the commercialization of genetic testing. ...
Journal of Clinical Medicine
Emicizumab is a humanized recombinant bispecific antibody, bridging together activated factor IX ... more Emicizumab is a humanized recombinant bispecific antibody, bridging together activated factor IX (FIXa) and factor X (FX), thus mimicking the activity of FVIII in vivo. Emicizumab is designed for long-term prophylaxis in patients with severe hemophilia A with and without inhibitors. This approach provides constant protection, with significant reduction in bleeding rate and improved quality of life. However, protection provided by emicizumab is not absolute, and clotting factor concentrates (FVIII, rFVIIa, aPCC) may be necessary for post-traumatic bleeding or surgery, with a potential thrombotic risk or difficulty in preventing bleeding. Real world evidence is still scanty, especially for managing major surgery. In this study, 75 surgeries were managed in 28 patients (27 major procedures in 15 patients and 48 minor procedures in 20 patients. In 17 patients without inhibitors, 30 minor surgeries were carried out by using FVIII in 5, with only a bleeding event, which was successfully t...
Journal of Clinical Medicine
Background: Intracranial hemorrhage (ICH) is a highly serious event in patients with haemophilia ... more Background: Intracranial hemorrhage (ICH) is a highly serious event in patients with haemophilia (PWH) which leads to disability and in some cases to death. ICH occurs among all ages but is particularly frequent in newborns. Aim: The primary aim was to assess the incidence and mortality due to ICH in an Italian population of PWH. Secondary aims were to evaluate the risk factors for ICH, the role of prophylaxis, and the clinical management of patients presenting ICH. Methods: A retrospective-prospective registry was established in the network of the Italian Association of Haemophilia Centers to collect all ICHs in PWH from 2009 to 2019 reporting clinical features, treatments, and outcomes. Results: Forty-six ICHs were collected from 13 Centers. The ICHs occurred in 15 children (10 < 2 years), and in 31 adults, 45.2% of them with mild hemophilia. Overall, 60.9% patients had severe haemophilia (15/15 children). Overall ICH incidence (×1000 person/year) was 0.360 (0.270–0.480 95% CI)...
Blood, 2016
Introduction: SMART-7™ (NCT01220141) was a prospective, post-authorization, single-arm, multinati... more Introduction: SMART-7™ (NCT01220141) was a prospective, post-authorization, single-arm, multinational, multi-center, non-interventional study investigating the safety and effectiveness of room temperature stable rFVIIa (NovoSeven®) in patients with hemophilia A or B with inhibitors in a real-world setting. Here, we present a subgroup analysis of the hemostatic response of bleeding episodes to treatment with rFVIIa. Methods: Study medication was not provided; use was at the discretion of the treating physician in accordance with the local label. Bleeding history was collected at the initiation visit. Information on bleeding episodes, including home treatment, was recorded in patient diaries during the study. Patients evaluated the status of bleeding episodes after treatment as…
TH Open, 2017
Treating hemophilia A or B patients with inhibitors is particularly challenging, as they do not r... more Treating hemophilia A or B patients with inhibitors is particularly challenging, as they do not respond to replacement therapy with factor VIII or factor IX concentrates. A room temperature–stable formulation of recombinant activated factor VII (rFVIIa; NovoSeven®), which provides improved convenience and treatment access to patients compared with the earlier formulation of rFVIIa, was shown to be safe and effective in a post-authorization, multinational, observational study (Study Monitoring Antibodies against Room Temperature–stable factor 7 [SMART-7™]). In post hoc, subgroup analyses of SMART-7™ data, the hemostatic response following rFVIIa monotherapy in patients with hemophilia A or B with inhibitors by time to first treatment and in different age cohorts was assessed. A total of 482/618 bleeding episodes treated with rFVIIa monotherapy and with (1) valid efficacy assessment, (2) no missing time for bleed start, (3) no missing time for any dose administration, and (4) valid ti...
Cambridge Quarterly of Healthcare Ethics, 1998
On August 1, 1996, due to the expiration of the five-year preservation limit provided by British ... more On August 1, 1996, due to the expiration of the five-year preservation limit provided by British law for unclaimed and legally unusable frozen embryos, 3,300 embryos were thawed and discarded. In Italy the news of this impending event triggered many reactions among scholars as well as the general population. In Massa, a little town in Tuscany, a most unusual response arose. Two hundred women banded together and asked to carry out a prenatal adoption. Their purpose in making this request was to avoid what they believed to be mass infanticide. Many of the women were married and already had children. They belonged to a local Catholic association. Nonetheless, their reaction was their own response to numerous appeals to respect life by the Catholic Church worldwide and by Italian Catholic thinkers especially.
AIDS, 1994
ObjectiveTo characterize a Kaposi's sarcoma (KS) cell line established from a tumor biopsy fr... more ObjectiveTo characterize a Kaposi's sarcoma (KS) cell line established from a tumor biopsy from the oral mucosa of an iatrogenically immunosuppressed HIV-negative man. MethodsCells were placed in culture and evaluated by a variety of biologic, serologic, karyotypic, and immunologic procedures. Electron microscopic examination was performed. The ability to produce tumors in nude mice was evaluated, and the nature of the cells within the tumor determined. Assays for urokinase plasminogen activator type (uPA), plasminogen activator inhibitor-1 (PAI-1) and the urokinase receptor (uPAR) were conducted. ResultsThe SLK cell line has an endothelial cell morphology with very little anaplasia. The karyotype indicates diploid phenotype of human origin. Immunohistochemical and electron microscopic examinations confirmed the endothelial nature of this cell line. No viruses were detected. The tumors induced in nude mice showed hypervascularization, with characteristics of KS. The cell line produces uPA and PAI-1, and also expresses uPAR. ConclusionsThe SLK cell line is of endothelial cell origin and the first human cell line to induce KS-like tumors in recipient animals. The expression of urokinase and its receptor suggests a paracrine and autocrine interaction that may be important for the growth of the tumor. The SLK line should be valuable for studies of KS pathogenesis and therapeutic approaches to this malignancy.
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Papers by Dr. Francesco Demartis