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1. Personalized Prophylaxis with myPKFiTCE: A Real-World Cost-Effectiveness Analysis in Haemophilia A Patients

Author

Antonazzo, I, Cortesi, P, Zanon, E, Pasca, S, Morfini, M, Santoro, C, De Cristofaro, R, Di Minno, G, Cozzolino, P, Mantovani, L, Antonazzo I. C., Cortesi P. A., Zanon E., Pasca S., Morfini M., Santoro C., De Cristofaro R., Di Minno G., Cozzolino P., Mantovani L. G., Antonazzo, I, Cortesi, P, Zanon, E, Pasca, S, Morfini, M, Santoro, C, De Cristofaro, R, Di Minno, G, Cozzolino, P, Mantovani, L, Antonazzo I. C., Cortesi P. A., Zanon E., Pasca S., Morfini M., Santoro C., De Cristofaro R., Di Minno G., Cozzolino P., and Mantovani L. G.

Abstract

Background and Objectives: This study aimed to assess the effectiveness and costs associated with pharmacokinetics-driven (PK) prophylaxis based on the myPKFiT® device in patients affected by hemophilia A (HA) in Italy. Materials and Methods: An observational retrospective study was conducted in three Italian hemophilia centers. All patients with moderate or severe HA, aged ≥ 18 years, capable of having PK estimated using the myPKFiT device, and who had had a clinical visit between 1 November 2019 and 31 March 2022 were included. Differences in clinical, treatment, health resources, and cost data were assessed comparing post-PK prophylaxis with pre-PK. The incremental cost-effectiveness ratio (ICER) was estimated as cost (EUR) per bleed avoided. Results: The study enrolled 13 patients with HA. The mean annual bleeding rate decreased by −1.45 (−63.80%, p = 0.0055) after the use of myPKFiT®. Overall, the consumption of FVIII IU increased by 1.73% during follow-up compared to the period prior the use of the myPKFiT. Prophylaxis based on the myPKFiT resulted in an ICER of EUR 5099.89 per bleed avoided. Conclusions: The results of our study support the idea that the use of PK data in clinical practice can be associated with an improvement in the management of patients, as well as clinical outcomes, with a reasonable increase in costs.

Published
2024

2. Recent Advances in the Treatment of Hemophilia: A Review

Author

Marchesini E, Morfini M, and Valentino L

Subjects
replacement therapy, adverse events, pharmacokinetics, extended half-life concentrates, non-replacement therapy, gene therapy, Medicine (General), R5-920
Abstract

Emanuela Marchesini,1 Massimo Morfini,2 Leonard Valentino3,4 1Hemophilia Centre, SC Vascular and Emergency Department, University of Perugia, Perugia, Italy; 2Italian Association of Haemophilia Centres (AICE), Naples, Italy; 3National Hemophilia Foundation, New York, NY, USA; 4Rush University, Chicago, IL, USACorrespondence: Massimo Morfini via Dello Statuto N.1- I-50129, Firenze, ItalyTel +39 348 2306928Fax +39 055-4732 18Email drmassimomorfini@gmail.comAbstract: Progress in hemophilia therapy has been remarkable in the first 20 years of the third millennium, but the innovation began with the description the fractionation of plasma in 1946. The first concentrates followed the discovery of FVIII in the cryoprecipitate of frozen plasma and FIX in the supernatant in the early 1960s, which led to the initial attempts at replacement therapy. Unfortunately, the lack of screening methods for viral pathogens resulted in people with hemophilia (PWH) receiving concentrates contaminated by hepatitis A virus, hepatitis C virus, and human immunodeficiency virus, as these concentrates were made from large industrial pools of plasma derived from thousands of donors. Fortunately, by 1985, viral screening methods and proper virucidal techniques were developed that made concentrates safe. Increasingly pure products followed the introduction of chromatography steps with monoclonal antibodies in the production process. The problem of immunogenicity of exogenously administered concentrates has not yet had a complete solution. The development of alloantibodies against FVIII in about 25– 35% of PWH is the most serious adverse effect of replacement therapy. The next major advance followed the cloning of the F8 gene and later the F9 genes, which paved the way to produce concentrates of factors obtained by the recombinant DNA technology. The injected FVIII and FIX molecules had a relatively short circulating half-life in the plasma of people with hemophilia A and B, approximately 12 and 18 hours, respectively. The ability to prolong the plasma half-life and extend the interval between injections followed the application of methods to conjugate the factor molecule with the fragment crystallizable of IgG1 or albumin or by adding polyethylene glycol, which has led to an increase in the half-life of concentrates, especially for rFIX. The next frontier in hemophilia therapy is the application of durable and potentially curative therapies such as with gene addition therapy. Experiments in hemophilia B have demonstrated durable responses. Unfortunately, the results with gene therapy for hemophilia A have not been as remarkable and the durability must still be demonstrated. Nonetheless, the long-term safety, predictability, durability, and efficacy of gene therapy for hemophilia A and B remain an open question. At present, only healthy adult PWH have been enrolled in gene therapy clinical trials. The application of gene therapy to children and those with pre-existing antibodies against the delivery vector must also be studied before this therapy becomes widespread.Keywords: replacement therapy, adverse events, pharmacokinetics, extended half-life concentrates, non-replacement therapy, gene therapy

Published
2021

3. Patient satisfaction and acceptability of an on-demand and on-prophylaxis device for factor VIII delivery in patients with hemophilia A

Author

Di Minno G, Santagostino E, Morfini M, Ettorre C, Cultrera D, Baldacci E, Russo E, and Gallucci C

Subjects
factor VIII delivery, device, hemophilia A, patient satisfaction, adherence, Medicine (General), R5-920
Abstract

Giovanni Di Minno,1 Elena Santagostino,2 Massimo Morfini,3 Cosimo Ettorre,4 Dorina Cultrera,5 Erminia Baldacci,6 Eleonora Russo,7 Cristiano Gallucci7 On behalf of the Italian FusENGO B1831081 Study Group 1Department of Clinical Medicine and Surgery, Azienda Universitaria Policlinico Federico II, Naples, Italy; 2Hemophilia and Thrombosis Center, IRCCS Fondazione Ca’ Granda, Ospedale Maggiore Policlinico, Milan, Italy; 3Scientific Committee, Italian Association of Haemophilia Centres (AICE), Florence, Italy; 4Haemophilia and Thrombosis Center, Policlinico Giovanni XXIII, Bari, Italy; 5Hematology Unit, Regional Center for Hemophilia, Italy Ospedaliera-Universitaria “Policlinico – Vittorio Emanuele”, Catania, Italy; 6Hemophilia, Thrombosis, and Hematology Center, Dipartimento Biotecnologie cellulari ed Ematologia, Università la Sapienza, Rome, Italy; 7Medical Department, Pfizer srl Rome, Italy Background: FuseNGO is a relatively new device consisting of a prefilled dual-chamber syringe (DCS) that was recently introduced for the reconstitution of recombinant factor VIII. Herein, the DCS device was assessed using five questionnaires with the primary aim of evaluating patient perceptions and preferences.Methods: An observational, non-interventional, longitudinal study on 86 patients with a confirmed diagnosis of hemophilia A was carried out at 21 sites in Italy. Each patient underwent a baseline visit and final study visit within 3–6 months. Patients were administered five questionnaires: HemoPREF; Treatment Satisfaction Questionnaire for Medication (TSQM); VeritasPRO; Hemophilia Well-being Index (HWBI); Work Productivity and Activity Impairment Questionnaire (WPAI) + Classroom Impairment Questions (CIQ): Hemophilia Specific (HS).Results: Compared to baseline, scores for HemoPREF were higher at follow-up; significant increases in the percentage of positive responses were seen for all questions regarding the ease of use (P70% in the VeritasPRO questionnaire, and the majority of patients reported in the HWBI that hemophilia A did not affect their lives in a significant way. The perceived level of overall impairment was 30% as reported in the WPAI + CIQ: HS, indicating little impairment. There were no safety concerns.Conclusion: Considering patient-reported outcomes, the DCS device was associated with easier preparation, storage, disposal of equipment, and overall use. Of particular note, preparation times were reduced by around 50%. The majority of patients were satisfied with the device and overall adherence scores were high. Considering these results, the device has the potential to increase adherence to therapy and, possibly, reduce healthcare costs. Keywords: factor VIII delivery, device, hemophilia A, patient satisfaction, adherence

Published
2019

5. Inhibitor development and mortality in non‐severe hemophilia A

Author

Eckhardt, C.L., Loomans, J.I., van Velzen, A.S., Peters, M., Mauser‐Bunschoten, E.P., Schwaab, R., Mazzucconi, M.G., Tagliaferri, A., Siegmund, B., Reitter‐Pfoertner, S.E., van der Bom, J.G., Fijnvandraat, K., Kamphuisen, P.W., Peerlinck, K., Oldenburg, J., Santagostino, E., Astermark, J., Eckhardt, C.L, van Velzen, A.S, Streefkerk, N., Loomans, J.L., van Eijkelenburg, A., Jansen, A.J., Kruijt, C.C., van Tienoven, B., van Baar, A.C.G., Corten, I.W., Meijer, K., Nijziel, M.R., Dors, N., Hamulyak, K., Beckers, E., Brons, P.P., Laros‐van Gorkom, B.A.P., van Heerde, W.L., Leebeek, F., Kruip, M., Cnossen, M.H., Mauser‐Bunschoten, E., Fischer, K., Smiers, F.J., Hermans, C., Klamroth, R., Escuriola‐Ettingshausen, C., Königs, C., Petrini, P., Holmström, M., Mäkipernaa, A., Male, C., Pabinger, I., Keenan, R.D., Liesner, R., Khair, K., Yee, T.T., Hart, D.P., Rangarajan, S., Mitchell, M., Thompson, G., Haya, S., Moret, A., Cid, A.R., Jimenez‐Yuste, V., Mancuso, M.E., Mazzuconni, M.G., Santoro, C., Morfini, M., Castaman, G., Schinco, P., Rivolta, G.F., Platokouki, H., and McRae, S.

Published
2015
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9. New data from the italian national register of congenital coagulopathies, 2016 annual survey

Author

Abbonizio F., Hassan H. J., Riccioni R., Santagostino E., Arcieri R., Giampaolo A., Contino L., Accorsi A., Caremani A., Ettorre P. C., Giordano P., Lassandro G., Valdre L., Notarangelo L., Aru A. B., Radossi P., Tagariello G., Cultrera D., Iannaccaro P., Santoro R., Biasoli C., Di Gregorio P., Daniele F., Testa S., Serino M. L., Castaman G., Linari S. S., Morfini M., Molinari A. C., Delios G., Cantori I., Franchini M., Caimi M. T., Mancuso M. E., Peyvandi F., Marietta M., Todisco A., Speciale V., Cerbone A. M., Di Minno G., Schiavulli M., Rocino A., Spiezia M. M., Zanon E., Gagliano F., Mansueto M. F., Siragusa S., Coppola A., Quintavalle G., Rivolta G. F., Tagliaferri A., Ambaglio C., Gamba G., Marchesini E., Oliovecchio E., Dragani A., Arbasi M. C., MacChi S., Vincenzi D., Sottilotta G., Pizzini A. M., Luciani M., De Cristofaro R., Baldacci E., Mazzucconi M. G., Santoro C., Mameli L. A., Coluccia A., Marino P., Borchiellini A., Schinco P. C., Messina M., Pollio B., Ricca I., Agostini P., Cristallo A. F., Barillari G., De Angelis V., Mosanghini M. E., Feola G., Bonetti E., Cesaro S., Gandini G., Giuffrida A., Tosetto A., Abbonizio, F., Hassan, H. J., Riccioni, R., Santagostino, E., Arcieri, R., Giampaolo, A., Contino, L., Accorsi, A., Caremani, A., Ettorre, P. C., Giordano, P., Lassandro, G., Valdre, L., Notarangelo, L., Aru, A. B., Radossi, P., Tagariello, G., Cultrera, D., Iannaccaro, P., Santoro, R., Biasoli, C., Di Gregorio, P., Daniele, F., Testa, S., Serino, M. L., Castaman, G., Linari, S. S., Morfini, M., Molinari, A. C., Delios, G., Cantori, I., Franchini, M., Caimi, M. T., Mancuso, M. E., Peyvandi, F., Marietta, M., Todisco, A., Speciale, V., Cerbone, A. M., Di Minno, G., Schiavulli, M., Rocino, A., Spiezia, M. M., Zanon, E., Gagliano, F., Mansueto, M. F., Siragusa, S., Coppola, A., Quintavalle, G., Rivolta, G. F., Tagliaferri, A., Ambaglio, C., Gamba, G., Marchesini, E., Oliovecchio, E., Dragani, A., Arbasi, M. C., Macchi, S., Vincenzi, D., Sottilotta, G., Pizzini, A. M., Luciani, M., De Cristofaro, R., Baldacci, E., Mazzucconi, M. G., Santoro, C., Mameli, L. A., Coluccia, A., Marino, P., Borchiellini, A., Schinco, P. C., Messina, M., Pollio, B., Ricca, I., Agostini, P., Cristallo, A. F., Barillari, G., De Angelis, V., Mosanghini, M. E., Feola, G., Bonetti, E., Cesaro, S., Gandini, G., Giuffrida, A., and Tosetto, A.

Subjects
Blood coagulation disorder, Haemophilia B, Haemophilia A, Von Willebrand's disease, Register
Abstract

Background - In Italy, the National Register of Congenital Coagulopathies (NRCC) collects epidemiological and therapeutic data from patients affected by haemophilia A (HA), haemophilia B (HB), von Willebrand's disease (vWD) and other rare coagulation disorders. Here we present data from the 2016 annual survey. Materials and methods - Data are provided by the Italian Haemophilia Centres, on a voluntary basis. Information flows from every Centre to a web-based platform of the Italian Association of Haemophilia Centres, shared with the Italian National Institute of Health, in accordance with current privacy laws. Patients are classified by diagnosis, disease severity, age, gender and treatment-related complications. Results - In 2016, the total number of patients with congenital coagulopathies in the NRCC was 10,360: 39.8% of these patients had HA, 31.5% had vWD, 8.5% had HB, and 20.2% had less common factor deficiencies. The overall prevalence of HA and HB was 13.9/100,000 males and 3.0/100,000 males, respectively. The overall prevalence of vWD was 5.4/100,000 inhabitants. During 2016, 126 patients had current alloantibodies to factor VIII (FVIII) or factor IX (FIX) and were under treatment with bypassing agents and/or immune tolerance induction. Overall, 388 patients with a history of alloantibodies were recorded in the NRCC of whom 337 with severe HA and 12 with severe HB. Coagulation factor use, evaluated from treatment plans, was approximately 451,000,000 IU of FVIII for HA patients (7.5 IU/inhabitant), and approximately 53,000,000 IU of FIX for HB patients (0.9 IU/inhabitant). Discussion - The prevalences of HA and HB fall within the ranges reported in more developed countries; the consumption of FVIII and FIX was in line with that of other European countries (France, United Kingdom) and Canada. The NRCC, with its bleeding disorder dataset, is a helpful tool for shaping public health policies, as well as planning clinical and epidemiological research projects.

Published
2020

10. Emergency management in patients with haemophilia A and inhibitors on prophylaxis with emicizumab: AICE practical guidance in collaboration with SIBioC, SIMEU, SIMEUP, SIPMeL and SISET

Author

Castaman, G., Santoro, C., Coppola, A., Mancuso, M. E., Santoro, R. C., Bernardini, S., Pugliese, F. R., Lubrano, R., Golato, M., Tripodi, A., Rocino, A., Santagostino, E., Biasoli, C., Borchiellini, A., Catalano, A., Contino, L., Coluccia, A., Cultrera, D., De Cristofaro, R., Di Minno, G., Fabbri, A., Franchini, M., Gamba, G., Chiara, A., Gresele, P., Giampaolo, A., Hassan, H. J., Luciani, M., Marchesini, E., Marino, R., Mazzucconi, M. G., Molinari, A. C., Morfini, M., Notarangelo, L. D., Peccarisi, L., Peyvandi, F., Pollio, B., Rivolta, G. F., Ruggieri, M. P., Sargentini, V., Schiavoni, M., Sciacovelli, L., Serino, M. L., Siragusa, S., Tagliaferri, A., Testa, S., Tosetto, A., Zampogna, S., Zanon, E., Castaman, Giancarlo, Santoro, Cristina, Coppola, Antonio, Mancuso, Maria E, Santoro, Rita C, Bernardini, Sergio, Pugliese, Francesco R, Lubrano, Riccardo, Golato, Maria, Tripodi, Armando, Rocino, Angiola, Santagostino, Elena, Biasoli, Chiara, Borchiellini, Alessandra, Catalano, Alberto, Contino, Laura, Coluccia, Antonella, Cultrera, Dorina, De Cristofaro, Raimondo, Di Minno, Giovanni, Fabbri, Andrea, Franchini, Massimo, Gamba, Gabriella, Giuffrida, Anna Chiara, Gresele, Paolo, Giampaolo, Adele, Hassan, Hamisa J, Luciani, Matteo, Marchesini, Emanuela, Marino, Renato, Mazzucconi, Maria Gabriella, Molinari, Angelo C, Morfini, Massimo, Notarangelo, Lucia D, Peccarisi, Lucia, Peyvandi, Flora, Pollio, Berardino, Rivolta, Gianna Franca, Ruggieri, Maria Pia, Sargentini, Vittorio, Schiavoni, Mario, Sciacovelli, Laura, Serino, Maria Luisa, Siragusa, Sergio, Tagliaferri, Annarita, Testa, Sophie, Tosetto, Alberto, Zampogna, Stefania, Zanon, Ezio, Castaman, G., Santoro, C., Coppola, A., Mancuso, M. E., Santoro, R. C., Bernardini, S., Pugliese, F. R., Lubrano, R., Golato, M., Tripodi, A., Rocino, A., Santagostino, E., Biasoli, C., Borchiellini, A., Catalano, A., Contino, L., Coluccia, A., Cultrera, D., De Cristofaro, R., Di Minno, G., Fabbri, A., Franchini, M., Gamba, G., Chiara, A., Gresele, P., Giampaolo, A., Hassan, H. J., Luciani, M., Marchesini, E., Marino, R., Mazzucconi, M. G., Molinari, A. C., Morfini, M., Notarangelo, L. D., Peccarisi, L., Peyvandi, F., Pollio, B., Rivolta, G. F., Ruggieri, M. P., Sargentini, V., Schiavoni, M., Sciacovelli, L., Serino, M. L., Siragusa, S., Tagliaferri, A., Testa, S., Tosetto, A., Zampogna, S., and Zanon, E.

Subjects
Factor VIII, FVIII inhibitor, Settore BIO/12, Antibodies, Bispecific, Antibodies, Monoclonal, Humanized, Factor VIII, Hemophilia A, Hemorrhage, Hemostatics, Humans, Italy, Quality of Life, FVIII inhibitors, Hemorrhage, Antibodies, Monoclonal, Humanized, Hemophilia A, Antibodies, Hemostatics, bypassing agents, emergency, emicizumab, haemophilia A, Italy, hemic and lymphatic diseases, Monoclonal, Emergency, Haemophilia A, Antibodies, Bispecific, Quality of Life, Humans, Bispecific, Bypassing agents, Emicizumab, Humanized, Bypassing agent, Haemostasis
Abstract

Emicizumab has been approved in several countries for regular prophylaxis in patients with congenital haemophilia A and FVIII inhibitors because it substantially reduces their bleeding risk and improves quality of life. However, although significantly less frequent, some breakthrough bleeds may still occur while on emicizumab, requiring treatment with bypassing or other haemostatic agents. Thrombotic complications have been reported with the associated use of activated prothrombin complex concentrates. In addition, when surgery/invasive procedures are needed while on emicizumab, their management requires multidisciplinary competences and direct supervision by experts in the use of this agent. Given this, and in order to expand the current knowledge on the use of emicizumab and concomitant haemostatic agents, and reduce the risk of complications in this setting, the Italian Association of Haemophilia Centres (AICE) here provides guidance on the management of breakthrough bleeds and surgery in emergency situations in patients with haemophilia A and inhibitors on emicizumab prophylaxis. This paper has been shared with other National Scientific Societies involved in the field.

Published
2020

17. Consensus statements on vaccination in patients with haemophilia—Results from the Italian haemophilia and vaccinations (HEVA) project

Author

Santagostino E., Riva A., Cesaro S., Esposito S., Matino D., Mazzucchelli R. I., Molinari, Angelo Claudio, Mura R., Notarangelo L. D., Tagliaferri A., Di Minno G., Clerici M., Ambaglio C., Brigida Aru A., Baldacci E., Barillari G., Basso M., Bernasconi S., Bertamino M., Bertoni E., Biasoli C., Federica Biguzzi E., Bonetti E., Borchiellini A., Bulgarelli S., Cabibbo S., Cantori I., Castaman G., Castiglia P., Coluccia A., Coppetelli U., Coppola A., Cultrera D., De Candia E., Delios G., Di Gennaro L., Di Gregorio P., Di Minno M., Dragani A., Pietro Ettorre C., Franchini M., Galli M., Gallo G., Giordano P., Giuffrida G., Iannaccaro P., Lassandro G., Lazzareschi I., Linari S., Luciani M., Macchi S., Malcangi G., Malizia R., Marietta M., Marino R., Massoud M., Gabriella Mazzucconi M., Milan M., Morfini M., Napolitano M., Pasca S., Pedrazzi P., Peyvandi F. A., Piscitelli L., Pollio B., Preti P., Quintavalle G., Radossi P., Raso S., Ricca I., Rocino A., Santoro C., Carlotta Santoro R., Sarolo L., Schiavoni M., Schiavulli M., Sciancalepore P., Luisa Serino M., Mario Siragusa S., Sottilotta G., Svahn J., Valdre L., Cristina Vedovati M., Zanon E., Santagostino, E., Riva, A., Cesaro, S., Esposito, S., Matino, D., Mazzucchelli, R. I., Molinari, Angelo Claudio, Mura, R., Notarangelo, L. D., Tagliaferri, A., Di Minno, G., Clerici, M., Ambaglio, C., Brigida Aru, A., Baldacci, E., Barillari, G., Basso, M., Bernasconi, S., Bertamino, M., Bertoni, E., Biasoli, C., Federica Biguzzi, E., Bonetti, E., Borchiellini, A., Bulgarelli, S., Cabibbo, S., Cantori, I., Castaman, G., Castiglia, P., Coluccia, A., Coppetelli, U., Coppola, A., Cultrera, D., De Candia, E., Delios, G., Di Gennaro, L., Di Gregorio, P., Di Minno, M., Dragani, A., Pietro Ettorre, C., Franchini, M., Galli, M., Gallo, G., Giordano, P., Giuffrida, G., Iannaccaro, P., Lassandro, G., Lazzareschi, I., Linari, S., Luciani, M., Macchi, S., Malcangi, G., Malizia, R., Marietta, M., Marino, R., Massoud, M., Gabriella Mazzucconi, M., Milan, M., Morfini, M., Napolitano, M., Pasca, S., Pedrazzi, P., Peyvandi, F. A., Piscitelli, L., Pollio, B., Preti, P., Quintavalle, G., Radossi, P., Raso, S., Ricca, I., Rocino, A., Santoro, C., Carlotta Santoro, R., Sarolo, L., Schiavoni, M., Schiavulli, M., Sciancalepore, P., Luisa Serino, M., Mario Siragusa, S., Sottilotta, G., Svahn, J., Valdre, L., Cristina Vedovati, M., and Zanon, E.

Subjects
Adult, medicine.medical_specialty, Consensus, Delphi Technique, Vaccination schedule, Delphi method, haemophilia, 030204 cardiovascular system & hematology, Hemophilia A, Haemophilia, immunization, Hemophilia B, bleeding disorder, factor VIII inhibitor, vaccination, Child, Evidence-Based Medicine, Humans, Italy, Vaccination, 03 medical and health sciences, 0302 clinical medicine, Vaccine administration, Medicine, In patient, Clinical Haemophilia, Genetics (clinical), business.industry, Original Articles, Hematology, General Medicine, Evidence-based medicine, medicine.disease, Settore MED/38 - PEDIATRIA GENERALE E SPECIALISTICA, Immunization, Family medicine, Original Article, business, 030215 immunology
Abstract

Vaccination against communicable diseases is crucial for disease prevention, but this practice poses challenges to healthcare professionals in patients with haemophilia. Poor knowledge of the vaccination requirements for these patients and safety concerns often result in vaccination delay or avoidance. In order to address this issue, a panel of 11 Italian haemophilia and immunization experts conducted a Delphi consensus process to identify the main concerns regarding the safe use of vaccines in patients with haemophilia. The consensus was based on a literature search of the available evidence, which was used by the experts to design 27 consensus statements. A group of clinicians then rated these statements using the 5‐point Likert‐type scale (1 = strongly disagree; 5 = strongly agree). The main issues identified by the expert panel included vaccination schedule for haemophilic patients; protocol and optimal route of vaccine administration; vaccination of haemophilic patients with antibodies inhibiting coagulation factor VIII (inhibitors); and vaccination and risk of inhibitor development. This manuscript discusses these controversial areas in detail supported by the available literature evidence and provides evidence‐ and consensus‐based recommendations. Overall, participants agreed on most statements, except those addressing the potential role of vaccination in inhibitor formation. Participants agreed that patients with haemophilia should receive vaccinations according to the institutional schedule for individuals without bleeding disorders; however, vaccination of patients with haemophilia requires comprehensive planning, taking into account disease severity, type and route of vaccination, and bleeding risk. Data also suggest vaccination timing does not need to take into consideration when the patient received factor VIII replacement.

Published
2019

18. Bleeding symptoms in patients diagnosed as type 3 von willebrand disease: results from 3WINTERS‐IPS, an international and collaborative cross‐sectional study

Author

Tosetto, A., Badiee, Z., Baghaipour, M., Baronciani, L., Battle, J., Berntorp, E., Bodó, I., Budde, U., Castaman, G., Eikenboom, J.C.J., Eshghi, P., Ettorre, C., Goodeve, A., Goudemand, J., Charles Richard Morris, H., Hoorfar, H., Karimi, M., Keikhaei, B., Lassila, R., Leebeek, F.W.G., Lopez Fernandez, M.F., Mannucci, P.M., Mazzucconi, M.G., Morfini, M., Oldenburg, J., Peake, I., Parra Lòpez, R., Peyvandi, F., Schneppenheim, R., Tiede, A., Toogeh, G., Trossaert, M., Zekavat, O., Zetterberg, E.M.K., and Federici, A.B.

Subjects
congenital, hereditary, and neonatal diseases and abnormalities, hemic and lymphatic diseases
Abstract

Background\ud Type 3 von Willebrand’s disease (VWD) patients present markedly reduced levels of von Willebrand factor and factor VIII. Because of its rarity, the bleeding phenotype of type 3 VWD is poorly described, as compared to type 1 VWD.\ud \ud Aims\ud To evaluate the frequency and the severity of bleeding symptoms across age and sex groups in type 3 patients and to compare these with those observed in type 1 VWD patients; to investigate any possible clustering of bleeding symptoms within type 3 patients.\ud \ud Methods\ud We compared the bleeding phenotype and computed the bleeding score (BS) using the MCMDM‐1VWD bleeding questionnaire in patients enrolled in the 3WINTERS‐IPS and MCMDM‐1VWD studies.\ud \ud Results\ud In 223 unrelated type 3 VWD patients, both the BS and the number of clinically relevant bleeding symptoms were increased in type 3 as compared to type 1 VWD patients (15. vs. 6.0 and 5 vs. 3). Intracranial bleeding, oral cavity, hemarthroses, and deep hematomas were at least five‐fold over‐represented in type 3 VWD. A more severe bleeding phenotype was evident in patients having VWF:Ag levels

Published
2020

19. Product type and the risk of inhibitor development in nonsevere haemophilia A patients

Author

Velzen, A.S. van, Eckhardt, C.L., Peters, M., Oldenburg, J., Cnossen, M., Liesner, R., Morfini, M., Castaman, G., McRae, S., Bom, J.G. van der, Fijnvandraat, K., INSIGHT Consortium, General Paediatrics, ACS - Amsterdam Cardiovascular Sciences, Paediatric Haematology, ACS - Pulmonary hypertension & thrombosis, ARD - Amsterdam Reproduction and Development, and Pediatrics

Subjects
Adult, Male, medicine.medical_specialty, congenital, hereditary, and neonatal diseases and abnormalities, Adolescent, animal diseases, Haemophilia A, haemophilia, Hemophilia A, Haemophilia, 03 medical and health sciences, 0302 clinical medicine, Internal medicine, hemic and lymphatic diseases, Humans, Medicine, antibodies, risk factors, Major complication, Date of birth, Child, Retrospective Studies, Blood Coagulation Factor Inhibitors, business.industry, Platelets, Haemostasis and Thrombosis, Case-control study, Hematology, Odds ratio, Middle Aged, medicine.disease, Confidence interval, Increased risk, factor VIII, Child, Preschool, 030220 oncology & carcinogenesis, business, Research Paper, 030215 immunology
Abstract

Inhibitor development is a major complication of treatment with factor VIII concentrates in nonsevere haemophilia A. It has been suggested that plasma-derived factor VIII (FVIII) concentrates elicit fewer inhibitors than recombinant FVIII concentrates, but studies in severe haemophilia A patients have shown conflicting results. We designed a case-control study to investigate the clinical and genetic risk factors for inhibitor development in nonsevere haemophilia A patients. We investigated whether the type of FVIII concentrate was associated with inhibitor development in nonsevere haemophilia A patients. This nested case-control study includes 75 inhibitor patients and 223 controls, from a source population of the INSIGHT study, including all nonsevere haemophilia A patients (FVIII:C 2-40%) that were treated with FVIII concentrates in 33 European and one Australian centre. Cases and controls were matched for date of birth and cumulative number of exposure days (CED) to FVIII concentrate. A conditional logistic regression model was used to calculate unadjusted and adjusted odds ratios. No increased risk for inhibitor development was found for any type of FVIII concentrate; either when comparing recombinant FVIII concentrates to plasma-derived FVIII concentrates (adjusted odds ratio 0 center dot 96, 95% confidence interval (CI) 0 center dot 36-2 center dot 52) or for specific types of FVIII concentrates.

Published
2020

20. Management of patients with severe haemophilia a without inhibitors on prophylaxis with emicizumab: AICE recommendations with focus on emergency in collaboration with SIBioC, SIMEU, SIMEUP, SIPMeL and SISET

Author

Coppola, A., Castaman, G., Santoro, R. C., Mancuso, M. E., Franchini, M., Marino, R., Rivolta, G. F., Santoro, C., Zanon, E., Sciacovelli, L., Manca, S., Lubrano, R., Golato, M., Tripodi, A., Rocino, A., Bernardini, S., Biasoli, C., Borchiellini, A., Cultrera, D., De Cristofaro, R., Di Minno, G., Gamba, G., Giampaolo, A., Giuffrida, A. C., Giuffrida, C., Gresele, P., Mazzucconi, M. G., Morfini, M., Hassan, H. J., Molinari, A. C., Pollio, B., Sargentini, V., Tagliaferri, A., Tosetto, A., and Zampogna, S.

Subjects
FVIII, Laboratory monitoring, Haemophilia A, haemophilia A, 030204 cardiovascular system & hematology, Antibodies, Monoclonal, Humanized, Hemophilia A, Haemophilia, surgery, 03 medical and health sciences, 0302 clinical medicine, Antibodies, Bispecific, Health care, medicine, bleeding, emergency, emicizumab, Humans, In patient, Genetics (clinical), Emicizumab, Emergency management, business.industry, Hematology, General Medicine, medicine.disease, Severe haemophilia A, Medical emergency, business, 030215 immunology
Abstract

Introduction The factor VIII (FVIII)-mimetic bispecific monoclonal antibody, emicizumab, previously approved for prophylaxis in haemophilia A with inhibitors, has been recently licensed in several countries also in patients with severe haemophilia A (PWSHA) without inhibitors. The introduction of this innovative agent requires the development of specific pathways at Haemophilia Treatment Centres (HTC), particularly regarding laboratory testing and treatment of breakthrough bleeds and invasive procedures/surgeries, even more critical when patients are managed by non-specialist professionals. Limited literature data and clinical experience in PWSHA without inhibitors on emicizumab are currently available. Aim To promote awareness and overcome these challenges, the Italian Association of Haemophilia Centres (AICE) issued a guidance on the management of PWSHA without inhibitors on emicizumab prophylaxis, focused on emergency and shared with other National Scientific Societies in the field. Methods The document, drafted by an AICE expert panel and approved through online consultation, was further revised by a multidisciplinary working group, including members of 5 haemostasis, laboratory and emergency scientific societies. The final version was approved by the Council of each society. Results General recommendations about use of FVIII concentrates for the treatment of bleeding or haemostatic coverage of invasive procedures/surgeries and laboratory monitoring in PWSHA without inhibitors on emicizumab are provided. Specific issues of the management in the emergency room are focused, highlighting the need for direct involvement or formalized supervision by specialist HTC physicians. Conclusions This guidance provides a reference pathway to be implemented in the different healthcare organizations, especially for the challenging emergency management in this setting.

Published
2020

22. Product type and the risk of inhibitor development in nonsevere haemophilia A patients: a case‒control study

Author

Velzen, A.S. (Alice) van, Eckhardt, C.L. (Corien L.), Peters, M.A.D. (Marjolein), Oldenburg, J., Cnossen, M.H. (Marjon), Liesner, R. (Ri), Morfini, M. (Massimo), Castaman, G. (Giancarlo), McRae, S., Bom, J.G. (Anske) van der, Fijnvandraat, K., Velzen, A.S. (Alice) van, Eckhardt, C.L. (Corien L.), Peters, M.A.D. (Marjolein), Oldenburg, J., Cnossen, M.H. (Marjon), Liesner, R. (Ri), Morfini, M. (Massimo), Castaman, G. (Giancarlo), McRae, S., Bom, J.G. (Anske) van der, and Fijnvandraat, K.

Abstract

Inhibitor development is a major complication of treatment with factor VIII concentrates in nonsevere haemophilia A. It has been suggested that plasma-derived factor VIII (FVIII) concentrates elicit fewer inhibitors than recombinant FVIII concentrates, but studies in severe haemophilia A patients have shown conflicting results. We designed a case‒control study to investigate the clinical and genetic risk factors for inhibitor development in nonsevere haemophilia A patients. We investigated whether the type of FVIII concentrate was associated with inhibitor development in nonsevere haemophilia A patients. This nested case‒control study includes 75 inhibitor patients and 223 controls, from a source population of the INSIGHT study, including all nonsevere haemophilia A patients (FVIII:C 2–40%) that were treated with FVIII concentrates in 33 European and one Australian centre. Cases and controls were matched for date of birth and cumulative number of exposure days (CED) to FVIII concentrate. A conditional logistic regression model was used to calculate unadjusted and adjusted odds ratios. No increased risk for inhibitor development was found for any type of FVIII concentrate; either when comparing recombinant FVIII concentrates to plasma-derived FVIII concentrates (adjusted odds ratio 0·96, 95% confidence interval (CI) 0·36–2·52) or for specific types of FVIII concentrates.

Published
2020
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32. Efficacy and safety during formulation switch of a pasteurized VWF/FVIII concentrate: results from an Italian prospective observational study in patients with von Willebrand disease

Author

Castaman, G., Coppola, A., Zanon, E., Boeri, E., Musso, M., Siragusa, S., Federici, A. B., Mancuso, G., Barillari, G., Biasoli, C., Feola, G., Franchini, M., Moratelli, S., Gamba, G., Schinco, P., Valdrè, L., Dragani, A., Mazzucconi, G., Tagliaferri, A., and Morfini, M.

Published
2013
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39. Results of a phase I international clinical trial of recombinant fusion protein linking coagulation factor IX with albumin (rIX-FP) in patients with hemophilia B (PROLONG-9FP): FP-MO-03.2-6

Author

SANTAGOSTINO, E., NEGRIER, C., KLAMROTH, R., TIEDE, A., PABINGER, I., TROSSAERT, M., MARTINOWITZ, U., ALTISENT, C., TAGLIAFERRI, A., ROTHSCHILD, C., ROMÁN, M. Á., LÓPEZ-FERNÁNDEZ, M. F., CASTAMAN, G., LAMBERT, T., VON DEPKA-PRONDZINSKI, M., VOIGT, C., JACOBS, I., and MORFINI, M.

Published
2012

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