Dr. Elizabeth Nolan

Dr. Elizabeth Nolan

Clinical Senior Lecturer, Paediatrics


Publications and Further Research Outputs

  • Nabialek T, Pinnamaneni R, Saleemi MS, Nolan B, Corcoran JD, Intra-hepatic haemorrhage and shock during post-natal period, in two brothers with haemophilia, Irish Medical Journal, 107, (8), 2014, p259 - 260Journal Article, 2014, URL
  • Fischer, K. and Iorio, A. and Hollingsworth, R. and Makris, M. and Watson, H. and Rae, J. and Platokouki, H. and Pergantou, H. and Katsarou, O. and Theodossiades, G. and Nomikou, E. and Parra, R. and Alonso, S. and Klamroth, R. and Kubicek, C. and Wilde, J. and Dunkley, T. and Oldenburg, J. and Schmickler, D. and Ackroyd, S. and Mirza, L. and Batorova, A. and Jankovicova, D. and Auerswald, G. and Buehrlen, M. and Penka, M. and Smejkal, P. and Blatny, J. and Zapletal, O. and Hermans, C. and Lambert, C. and Tagariello, G. and Radossi, P. and Nolan, B. and Brady, B. and O'Donnell, J. and Singleton, E. and Thomas, A. and Shea, I. and Morfini, M. and Linari, S. and de Moerloose, P. and Boehlen, F. and Campbell Tait, R. and Brodie, N. and Chalmers, E. and Gibson, A. and Meijer, K. and Tamminga, R. and Lassila, R. and Armstrong, E. and Zulfikar, B. and Ozdemir, N. and Kavakli, K. and Balkan, C. and Jurgutis, R. and Gailiute, N. and Peerlinck, K. and Geet, C.V. and Goudemand, J. and Wibaut, B. and Diniz, M.J. and Antunes, M. and Liesner, R. and Khair, K. and Laffan, M. and Patel, S. and Thynn Yee, T. and Harrington, C. and Pasi, J. and Bowles, L. and Bevan, D. and Madan, B. and Negrier, C. and Ringenbach, S. and Mingot Castellano, M.E. and Astermark, J. and Lindvall, K. and Gatt, A. and Hay, C.R.M. and Grey, P. and Will, A. and Hague, A. and Peyvandi, F. and Mancuso, M.E. and Schved, J.F. and Rousseau, F. and Kurnik, K. and Bidlingmaier, C. and Rocino, A. and Hanley, J. and Talks, K. and Antoniades, M. and Zanon, E. and Bon, E.D. and Lambert, T. and Rothschild, C. and Matingou, M. and Tagliaferri, A. and Rivolta, G.F. and Iorio, A. and Marchesini, E. and Fraga, C. and Cunha-Ribeiro, L. and Carvalho, M. and Komrska, V. and Pindurova, E. and Lejniece, S. and Kluce, G. and Leebeek, F. and Cnossen, M. and Makris, M. and Lockley, C. and Payne, J. and Vidler, V. and Lissitchkov, T. and Raia, P. and Holmstre om, M. and Garipidou, V. and Vakalopoulou, S. and Serban, M. and Mihailov, M.-D. and Schinco, P. and Valeri, F. and Schutgens, R. and Bonanad, S. and Cid, A.R. and Castaman, G. and Pabinger, I. and Reitter, S.-E. and Male, C. and Thom, K. and Windyga, J. and Piorowski, M. and Schmugge, M. and Albisetti, M., FVIII inhibitor development according to concentrate: Data from the EUHASS registry excluding overlap with other studies, Haemophilia, 22, (1), 2016, pe36-e38Journal Article, 2016, DOI , URL
  • Ryan KM, O'Brien K, Regan I, O'Byrne JM, Moore D, Kelly PM, Noel J, Butler J, Nolan B, Kiely PJ, The prevalence of abnormal preoperative coagulation tests in pediatric patients undergoing spinal surgery for scoliosis, Spine J, 15, (6), 2015, p1217 - 1222Journal Article, 2015
  • B. Nolan, B. White, J. Smith, C. O'Reily, B. Fitzpatrick and O. P. Smith, Desmopressin: therapeutic limitations in children and adults with inherited coagulation disorders, British Journal of Haematology, 109, (4), 2000, p865-9Journal Article, 2000, DOI , URL
  • Martin, Therese, Moore, Mary, Melody, Laura, Nolan, Beatrice, Snow, Aisling, Govender, Pradeep, Blackburn, Carol, Going out on a (clotted) limb - an interesting case of paget-schroetter syndrome in a young adolescent, BMJ Publishing Group Ltd and Royal College of Paediatrics and Child Health, 2019Conference Paper, 2019, DOI
  • Bacon, C.L., Singleton, E., Brady, B., White, B., Nolan, B., Gilmore, R.M., Ryan, C., Keohane, C., Vince Jenkins, P. and O'Donnell, Low risk of inhibitor formation in haemophilia A patients following en masse switch in treatment to a third generation full length plasma and albumin-free recombinant factor VIII product (ADVATE®), Haemophilia, 17, 2011, p407 - 411Journal Article, 2011, DOI , URL
  • Sweetman D, Kelly LA, Zareen Z, Nolan B, Murphy J, Boylan G, Donoghue V, Molloy EJ., Coagulation Profiles Are Associated With Early Clinical Outcomes in Neonatal Encephalopathy., Frontiers in pediatrics, 2019Journal Article, 2019, DOI
  • Nolan B., Klukowska A., Shapiro A., Rauch A., Recht M., Ragni M., Curtin J., Gunawardena S., Mukhopadhyay S., Jayawardene D., Winding B., Fischer K., Liesner R., Final results of the PUPs B-LONG study: Evaluating safety and efficacy of rFIXFc in previously untreated patients with hemophilia B, Blood Advances, 5, (13), 2021, p2732 - 2739, p2732-2739Journal Article, 2021, DOI
  • Minna K., Anne M., Beatrice N., Rainer K., Susanna R., Correction of haemostasis can be reduced to four days for CVAD implantation in severe haemophilia A patients: Data from the PedNet study group, Haemophilia, 27, (3), 2021, p392 - 397, p392-397Journal Article, 2021, DOI , URL
  • Hermans C., Mancuso M.E., Nolan B., Pasi K.J., Recombinant factor VIII Fc for the treatment of haemophilia A, European Journal of Haematology, 106, (6), 2021, p745 - 761, p745-761Journal Article, 2021, DOI
  • Pasi K.J., Fischer K., Ragni M., Kulkarni R., Ozelo M.C., Mahlangu J., Shapiro A., P'Ng S., Chambost H., Nolan B., Bennett C., Matsushita T., Winding B., Fruebis J., Yuan H., Rudin D., Oldenburg J., Long-term safety and sustained efficacy for up to 5 years of treatment with recombinant factor IX Fc fusion protein in subjects with haemophilia B: Results from the B-YOND extension study, Haemophilia, 26, (6), 2020, pe262 - e271, pe262-e271Journal Article, 2020, DOI
  • Van Den Berg H.M., Mancuso M.E., Konigs C., D'Oiron R., Platokouki H., Mikkelsen T.S., Motwani J., Nolan B., Santagostino E., ITI treatment is not first-choice treatment in children with hemophilia A and low-responding inhibitors: Evidence from a PedNet study, Thrombosis and Haemostasis, 120, (8), 2020, p1166 - 1172, p1166-1172Journal Article, 2020, DOI
  • Nolan B., Mahlangu J., Pabinger I., Young G., Konkle B.A., Barnes C., Nogami K., Santagostino E., Pasi K.J., Khoo L., Winding B., Yuan H., Fruebis J., Rudin D., Oldenburg J., Recombinant factor VIII Fc fusion protein for the treatment of severe haemophilia A: Final results from the ASPIRE extension study, Haemophilia, 26, (3), 2020, p494 - 502, p494-502Journal Article, 2020, DOI
  • Ahmed S.Z., O'Rourke M., Jenkins V., Regan I., Nolan B., Progressive increase in FIX level in males with haemophilia B Leyden and c.35G > A mutation in early childhood not related to androgen effect, British Journal of Haematology, 189, (6), 2020, pe262 - e265, pe262-e265Journal Article, 2020, DOI
  • Male C., Andersson N.G., Rafowicz A., Liesner R., Kurnik K., Fischer K., Platokouki H., Santagostino E., Chambost H., Nolan B., Konigs C., Kenet G., Ljung R., Van Den Berg H.M., Inhibitor incidence in an unselected cohort of previously untreated patients with severe hemophilia B: A PedNet study, Haematologica, 106, (1), 2021, p123 - 129, p123-129Journal Article, 2021, DOI
  • Hermans C., Giangrande P.L.F., O'Mahony B., De Kleijn P., Bedford M., Batorova A., Blatny J., Jansone K., Astermark J., Crato M., D'Oiron R., Dougall A., Fijnvandraat K., Gronhaug S., Jimenez-Yuste V., Jokic M., Lobet S., Nolan B., Peyvandi F., Ryan A., European principles of inhibitor management in patients with haemophilia: Implications of new treatment options, Orphanet Journal of Rare Diseases, 15, (1), 2020Journal Article, 2020, DOI
  • Andersson N.G., Wu R., Carcao M., Claeyssens-Donadel S., Kobelt R., Liesner R., Makipernaa A., Ranta S., Ljung R., Auerswald G., Barnes C., Chalmers E., Chambost H., Clausen N., Dunn A.L., Escuriola Ettinghausen C., Fischer K., Fijnvandraat K., van Geet C., Hoffmann M., Kavakli K., Kenet G., Konigs C., Kurnik K., Manco-Johnson M., Mancuso M.E., Molinari C., Muntean W., Nolan B., Perez Garrido R., Platokouki E., Rafowicz A., Santagostino E., Shapiro A., Thomas A., Williams M., Long-term follow-up of neonatal intracranial haemorrhage in children with severe haemophilia, British Journal of Haematology, 190, (2), 2020, pe101 - e104, pe101-e104Journal Article, 2020, DOI
  • Male C., Lensing A.W.A., Palumbo J.S., Kumar R., Nurmeev I., Hege K., Bonnet D., Connor P., Hooimeijer H.L., Torres M., Chan A.K.C., Kenet G., Holzhauer S., Santamaria A., Amedro P., Chalmers E., Simioni P., Bhat R.V., Yee D.L., Lvova O., Beyer-Westendorf J., Biss T.T., Martinelli I., Saracco P., Peters M., Kallay K., Gauger C.A., Massicotte M.P., Young G., Pap A.F., Majumder M., Smith W.T., Heubach J.F., Berkowitz S.D., Thelen K., Kubitza D., Crowther M., Prins M.H., Monagle P., Molinari A.C., Nowak Gottl U., Chain J., Robertson J., Thom K., Streif W., Schwarz R., Schmitt K., Grangl G., Van Damme A., Maes P., Labarque V., Petrilli A., Loggeto S., Azeka E., Brandao L., Le D., Sabapathy C., Giordano P., Wu R., Ding J., Huang W., Mao J., Lahteenmaki P., Decramer S., Bernig T., Chada M., Chan G., Kally K., Nolan B., Revel-Vilk S., Tamary H., Levin C., Tormene D., Abbattista M., Artoni A., Ikeyama T., Inuzuka R., Yasukochi S., Morales Soto M., Solis Labastida K.A., Suijker M.H., Bartels M., Tamminga R.Y., Van Ommen C.H., Te Loo D.M., Anjos R., Zubarovskaya L., Popova N., Samochatova E., Belogurova M., Svirin P., Shutova T., Lebedev V., Barbarash O., Koh P.L., Mei J.C., Podracka L., Berrueco R., Fernandez M.F., Frisk T., Grunt S., Rischewski J., Albisetti-Pedroni M., Antmen A., Tokgoz H., Karakas Z., Motwani J., Williams M., Grainger J., Payne J., Richards M., Baird S., Bhatnagar N., Aramburo A., Crary S., Wynn T., Carpenter S., Ahuja S., Goldenberg N., Woods G., Godder K., Scott-Emuakpor A., Roach G., Raffini L., Shah N., Shah S., Thornburg C., Zia A., Berkow R., Rivaroxaban compared with standard anticoagulants for the treatment of acute venous thromboembolism in children: a randomised, controlled, phase 3 trial, The Lancet Haematology, 7, (1), 2020, pe18 - e27, pe18-e27Journal Article, 2020, DOI
  • Andersson N.G., Chalmers E.A., Kenet G., Ljung R., Makipernaa A., Chambost H., Mode of delivery in hemophilia: Vaginal delivery and Cesarean section carry similar risks for intracranial hemorrhages and other major bleeds, Haematologica, 104, (10), 2019, p2100 - 2106, p2100-2106Journal Article, 2019, DOI
  • Eliwan H.O., Watson W.R.G., Regan I., Philbin B., O'hare F.M., Strickland T., O'neill A., O'rourke M., Blanco A., Healy M., Nolan B., Smith O., Molloy E.J., Pediatric intensive care: Immunomodulation with activated protein C ex vivo, Frontiers in Pediatrics, 7, (SEP), 2019Journal Article, 2019, DOI
  • Carcao M., Escuriola-Ettingshausen C., Santagostino E., Oldenburg J., Liesner R., Nolan B., Batorova A., Haya S., Young G., The changing face of immune tolerance induction in haemophilia A with the advent of emicizumab, Haemophilia, 25, (4), 2019, p676 - 684, p676-684Journal Article, 2019, DOI
  • Giangrande P.L.F., Hermans C., O'Mahony B., De Kleijn P., Bedford M., Batorova A., Blatny J., Jansone K., Astermark J., Bok A., Crato M., D'Oiron R., Dougall A., Fijnvandraat K., Gronhaug S., Jimenez-Yuste V., Jokic M., Lobet S., Nolan B., Peyvandi F., Ryan A., European principles of inhibitor management in patients with haemophilia, Orphanet Journal of Rare Diseases, 13, (1), 2018Journal Article, 2018, DOI
  • Andersson N.G., Auerswald G., Barnes C., Carcao M., Dunn A.L., Fijnvandraat K., Hoffmann M., Kavakli K., Kenet G., Kobelt R., Kurnik K., Liesner R., Makipernaa A., Manco-Johnson M.J., Mancuso M.E., Molinari A.C., Nolan B., Perez Garrido R., Petrini P., Platokouki H.E., Shapiro A.D., Wu R., Ljung R., Intracranial haemorrhage in children and adolescents with severe haemophilia A or B " the impact of prophylactic treatment, British Journal of Haematology, 179, (2), 2017, p298 - 307, p298-307Journal Article, 2017, DOI
  • Malone A., Wlodarski M., Nolan B., Smyth L., Smith O.P., Transient myeloproliferative disorder in an infant with PTPN11 mutation, British Journal of Haematology, 177, (1), 2017, p11-Journal Article, 2017, DOI
  • Fischer K., Kulkarni R., Nolan B., Mahlangu J., Rangarajan S., Gambino G., Diao L., Ramirez-Santiago A., Pierce G.F., Allen G., Recombinant factor IX Fc fusion protein in children with haemophilia B (Kids B-LONG): results from a multicentre, non-randomised phase 3 study, The Lancet Haematology, 4, (2), 2017, pe75 - e82, pe75-e82Journal Article, 2017, DOI
  • Pasi K.J., Fischer K., Ragni M., Nolan B., Perry D.J., Kulkarni R., Ozelo M., Mahlangu J., Shapiro A.D., Baker R.I., Bennett C.M., Barnes C., Oldenburg J., Matsushita T., Yuan H., Ramirez-Santiago A., Pierce G.F., Allen G., Mei B., Long-term safety and efficacy of extended-interval prophylaxis with recombinant factor IX Fc fusion protein (rFIXFc) in subjects with haemophilia B, Thrombosis and Haemostasis, 117, (3), 2017, p508 - 518, p508-518Journal Article, 2017, DOI
  • Ljung R., Kenet G., Mancuso M.E., Kaleva V., Rusen L., Tseneklidou-Stoeter D., Michaels L.A., Shah A., Hong W., Maas Enriquez M., Kaleva V., Stoyanova-Deleva A., Blanchette V., Dower N., Clausen N., Kardos M., Kiss C., Zombori M., Nolan B., Kenet G., Santagostino E., Zanon E., Luciani M., Kovalova Z., Rageliene L., Dobaczewski G., Klukowska A., Mlynarkski W., Serban M., Rusen L., Uscatescu V., Kerlin B., Ahuja S., Gruppo R., Singleton T., BAY 81-8973 safety and efficacy for prophylaxis and treatment of bleeds in previously treated children with severe haemophilia A: Results of the LEOPOLD Kids Trial, Haemophilia, 22, (3), 2016, p354 - 360, p354-360Journal Article, 2016, DOI
  • Nolan B., Mahlangu J., Perry D., Young G., Liesner R., Konkle B., Rangarajan S., Brown S., Hanabusa H., Pasi K.J., Pabinger I., Jackson S., Cristiano L.M., Li X., Pierce G.F., Allen G., Long-term safety and efficacy of recombinant factor VIII Fc fusion protein (rFVIIIFc) in subjects with haemophilia A, Haemophilia, 22, (1), 2016, p72 - 80, p72-80Journal Article, 2016, DOI
  • Fischer K., Iorio A., Lassila R., Peyvandi F., Calizzani G., Gatt A., Lambert T., Windyga J., Gilman E.A., Makris M., Inhibitor development in non-severe haemophilia across Europe, Thrombosis and Haemostasis, 114, (4), 2015, p670 - 675Journal Article, 2015, DOI , URL
  • Nijdam A., Kurnik K., Liesner R., Ljung R., Nolan B., Petrini P., Fischer K., How to achieve full prophylaxis in young boys with severe haemophilia A: Different regimens and their effect on early bleeding and venous access, Haemophilia, 21, (4), 2015, p444 - 450Journal Article, 2015, DOI , URL
  • Young G., Mahlangu J., Kulkarni R., Nolan B., Liesner R., Pasi J., Barnes C., Neelakantan S., Gambino G., Cristiano L.M., Pierce G.F., Allen G., Recombinant factor VIII Fc fusion protein for the prevention and treatment of bleeding in children with severe hemophilia A, Journal of Thrombosis and Haemostasis, 13, (6), 2015, p967 - 977, p967-977Journal Article, 2015, DOI
  • Nijdam A., Altisent C., Carcao M.D., Cid A.R., Claeyssens-Donadel S., Kurnik K., Ljung R., Nolan B., Petrini P., Platokouki H., Rafowicz A., Thomas A.E., Fischer K., Bleeding before prophylaxis in severe hemophilia: Paradigm shift over two decades, Haematologica, 100, (3), 2015, pe84 - e86, pe84-e86Journal Article, 2015, DOI
  • Nolan B, Mitchell TH., Autonomic neuropathy with gastropathy and elevated urinary 5-hydroxy indole acetic acid in insulin-dependent diabetes mellitus., The American journal of gastroenterology, 91, (6), 1996, p1286Journal Article, 1996, DOI
  • Mainwaring C.J., Walewska R., Snowden J., Wlnfield D.A., Ng J.P., Chan-Lam D., Nolan B., Booker D.J., Stamps R., Sokol R.J., Fatal cold anti-i autoimmune haemolytic anaemia complicating hairy cell leukaemia, British Journal of Haematology, 109, (3), 2000, p641 - 643Journal Article, 2000, DOI , URL
  • Mitchell T.H., Nolan B., Henry M., Cronin C., Baker H., Greely G., Microalbuminuria in patients with non-insulin-dependent diabetes mellitus relates to nocturnal systolic blood pressure, American Journal of Medicine, 102, (6), 1997, p531 - 535, p531-535Journal Article, 1997, DOI
  • Ng J.-P., Nolan B., Chan-Lam D., Coup A.J., Mckenna D., Successful treatment of aplastic variant of hairy-cell leukemia with deoxycoformycin, Hematology, 7, (4), 2002, p259 - 262Journal Article, 2002, DOI , URL
  • Nolan B., Vidler V., Vora A., Makris M., Lesson of the week: Unsuspected haemophilia in children with a single swollen joint, British Medical Journal, 326, (7381), 2003, p151-152Journal Article, 2003, DOI , URL
  • Hinchliffe R.F., Nolan B., Vora A.J., Stamps R., Neonatal pure red cell aplasia due to anti-M, Archives of Disease in Childhood: Fetal and Neonatal Edition, 91, (6), 2006Journal Article, 2006, DOI
  • Singleton E., Smith J., Kavanagh M., Nolan B., White B., Low risk of inhibitor formation in haemophilia patients after a change in treatment from Chinese hamster ovary cell-produced to baby hamster kidney cell-produced recombinant factor VIII, Thrombosis and Haemostasis, 98, (6), 2007, p1188-1192Journal Article, 2007, DOI , URL
  • Iqbal R, Mulvihill NT, Nolan B, Crean PA., Multivessel coronary thrombosis resulting from heparin induced thrombocytopenia., Irish medical journal, 100, (8), 2007, p569-571Journal Article, 2007, DOI
  • Gleeson, Michelle O†Rourke and Philbin, Brian and Nolan, Beatrice, Poster presentation Application of the US National Institute of Health (NIH) 2008 guidelines for von Willebrand Disease in a National Paediatric Comprehensive Care Centre. Irene E. Regan and Aine Burke, Orla M Cormack, Christopher Fox, Mary E, 2008Journal Article, 2008
  • Gouw, Samantha C ,van der Bom, Johanna G , Ljung, Rolf ,Escuriola, Carmen , Cid, Ana R , Claeyssens-Donadel, Segolene , van Geet, Christel ,Kenet, Gili ,Makipernaa, Anne , Molinari, Angelo Claudio, Muntean, Wolfgang , Kobelt, Rainer , Rivard, George ,Santagostino, Elena , Thomas, Angela , van den Berg, H Marijke ,PedNet and RODIN Study Group, Factor VIII products and inhibitor development in severe hemophilia A, The New England Journal of Medicine, 368, (3), 2013, p231-239Journal Article, 2013, DOI , URL
  • Carcao, Manuel D and van den Berg, H Marijke and Ljung, Rolf and Mancuso, Maria Elisa and {PedNet and the Rodin Study Group, Correlation between phenotype and genotype in a large unselected cohort of children with severe hemophilia A, Blood, 121, (19), 2013, p3946-3952Journal Article, 2013, DOI , URL
  • Gouw, Samantha C and van den Berg, H Marijke and Fischer, Kathelijn and Auerswald, Gunter and Carcao, Manuel and Chalmers, Elizabeth and Chambost, Herve and Kurnik, Karin and Liesner, Ri and Petrini, Pia and Platokouki, Helen and Altisent, Carmen and Oldenburg, Johannes and Nolan, Beatrice and Garrido, Rosario Perez and Mancuso, M Elisa and Rafowicz, Anne and Williams, Mike and Clausen, Niels and Middelburg, Rutger A and Ljung, Rolf and van der Bom, Johanna G and {PedNet and Research of Determinants of INhibitor development (RODIN) Study Group, Intensity of factor VIII treatment and inhibitor development in children with severe hemophilia A: the RODIN study, Blood, 121, (20), 2013, p4046 - 4055Journal Article, 2013, DOI , URL
  • Clausen, N and Petrini, P and Claeyssens-Donadel, S and Gouw, SC and Liesner, R and {PedNet and Research of Determinants of Inhibitor development (RODIN) Study Group, Similar bleeding phenotype in young children with haemophilia A or B: a cohort study, Haemophilia : the official journal of the World Federation of Hemophilia, 20, (6), 2014, p747-755Journal Article, 2014, DOI , URL
  • Regan IE, Brady B, Kavanagh M, Kelly M, Nolan, B, Von willebrand disease reclassification in a national paediatric comprehensive care centre, Archives of Disease in Childhood, Royal College of Paediatrics and Child Health, 9th Europaediatrics Congress, 13-15 June, Dublin, Ireland 2019, Dublin, 2019, 2019, (104 ( Suppl3)), Brirish Medical Journal, 2019Oral Presentation, 2019
  • Mary Kavanagh, Imelda Kelly, Caitriona Ferry, Bridin Brady, Irene Regan, Beatrice Nolan, Review of paediatric patients with severe factor X deficiency in ireland , Archives of Disease in Childhood, Royal College of Paediatrics and Child Health, 9th Europaediatrics Congress, 13-15 June, Dublin, Ireland 2019, Dublin, 2019, Brirish Medical Journal, 2019Poster, 2019, URL
  • V. Dvorakova, B. Nolan, A. Broderick,E.J. MacDermott and R. Watson, Neonatal lupus erythematosus with life-threatening gastrointestinal bleeding, British Journal of Dermatology, 94th Annual Meeting of the British-Association-of-Dermatologists, Glasgow, UK, 1-3 July 014, 171 ( suppl 1), 2014Poster, 2014, URL
  • G. Kenet, B. Nolan, J. Oldenburg, J. Motwani, R. D' Oiron, C. Male, H. Chambost, K. Fischer, C. Van Geet, H.M. van den Berg, PedNet Study Group, Emicizumab Treatment in Pediatric Haemophilia A Patients: >1 Year Safety Based on Real-world Data from the PedNet Cohorts, Research and Practice in Thrombosi s and Hemostasis, ISTH 2021, on line, July 2021, 5 ( suppl 1), 2021Poster, 2021, URL
  • Final Results of PUPs B-LONG Study: Evaluating Safety and Efficacy of rFIXFc in Previously Untreated Patients with Haemophilia B( abstract), B. Nolan, A. Klukowska, A. Shapiro, A. Rauch, M. Recht, M. Ragni, J. Curtin, S. Gunawardena, S. Poloskey, D. Jayawardene, B. Winding, K. Fischer, R. Liesner, Research and Practice in Thrombosis and Hemostasis, ISTH Conference 2020, On line, July 2020, 4 ( suppl1), 2020Poster, 2020, URL
  • Regan I, Pillion C, Riordan M, Nolan B. , Antithrombin Activity Level below 60% in Children Has Implications for Heparin (LMWH) Dosing, Research and Practice in Thrombosis and Haemostasis, ISTH 2020, On line, 4 ( suppl1), 2020Oral Presentation, 2020, URL
  • Saad Z Ahmed, Michelle O'Rourke, Vince Jenkins, Caitriona Keenan, Irene Ellen Regan, Beatrice Nolan, Similar Pattern of Increase in FIX Levels in Female Carriers and Males with Hemophilia B Leyden, Blood , 62nd ASH Annual Meeting and Exposition, On line, 5-8 Dec 2020, 136( Supplement 1), 2020, pp19 - 20Poster, 2020, URL
  • Königs C, Ozelo MC, Dunn A, Kulkarni R, Nolan B, Brown SA, Liesner R, Schiavulli M, Gunawardena S, Mukhopadhyay S, Jayawardene D, Winding B, Carcao M. , Final Results of PUPs A-LONG Study: Evaluating Safety and Efficacy of rFVIIIFc in Previously Untreated Patients with Haemophilia A, Research and Practice in Thrombosis and Haemostasis, ISTH Conference 2020, On line, July 2020, 4 ( suppl1), 2020Oral Presentation, 2020, URL
  • Beatrice Nolan, Anna Klukowska, Amy D Shapiro , Antoine Rauch, Michael Recht, Margaret V. Ragni, Julie Curtin, Sriya Gunawardena, Stacey Poloskey, Deepthi Jayawardene, Bent Winding, Kathelijn Fischerand Raina Liesner, Final Results of PUPs B-LONG Study: Evaluating Safety and Efficacy of rFIXFc in Previously Untreated Patients with Haemophilia B, 62nd ASH Annual Meeting and Exposition, On line, 5-8 Dec 2020, 2020Poster, 2020, URL
  • Christoph Koenigs, Margareth C. Ozelo, Amy Dunn, Roshni Kulkarni, Beatrice Nolan, Simon A. Brown Raina Liesner, Michele Schiavulli, Sriya Gunawardena, Sutirtha Mukhopadhyay, Deepthi Jayawardene, Bent Winding and Manuel Carcao, Final Results of PUPs A-LONG Study: Evaluating Safety and Efficacy of rFVIIIFc in Previously Untreated Patients with Haemophilia A, 62nd ASH Annual Meeting and Exposition, On line, 5-8 Dec 2020, 2020Oral Presentation, 2020, URL
  • Roshni Kulkarni, Beatrice Nolan, Carolyn M. Bennett, Kathelijn Fischer, David J Perry, Christopher Barnes, Huixing Yuan, Alejandra Ramirez-Santiago, Glenn F Pierce, Baisong Mei, Clinical Outcomes in Children with Hemophilia B Treated Long Term with rFIXFc: Interim Results of the B-YOND Extension Study, Blood , ASH Annual Meeting and Exposition, 126, (23), 2015Poster, 2015, URL
  • Margaret Ragni, Roshni Kulkarni, K. John Pasi, Kathelijn Fischer, Johnny Mahlangu, Amy Shapiro, Beatrice Nolan, Johannes Oldenburg, Tadashi Matsushita, Annemieke Willemze, Huixing Yuan, Dan Rudin, B-YOND Final Results Confirm Established Safety, Sustained Efficacy, and Extended Dosing Interval for Up to 4 Years of Treatment With rFIXFc in Previously Treated Subjects With Severe Hemophilia B, Blood, 60th ASH Annual Meeting and Exposition, 2018, 132 ( supplement 1), 2018, pp1214 - 1214Poster, 2018, URL
  • Beatrice Nolan, Johnny Mahlangu, Guy Young, Barbara A Konkle, K. John Pasi, MB, ChB, PhD FRCP, FRCPath, FRCPCH, Johannes Oldenburg, MD PhD, Keiji Nogami, MDPhD, Nikola Tripkovic, Huixing Yuan, Dan Rudin, ASPIRE Final Results Confirm Established Safety and Sustained Efficacy for Up to 4 Years of Treatment With rFVIIIFc in Previously Treated Subjects With Severe Hemophilia A, Blood, 6oth ASH Annual Meeting and Exposition, 2018, 132 ( supplement 1), 2018, pp1192-Poster, 2018, URL
  • Guy Young, Raina Liesner, K John Pasi, Beatrice Nolan, Stefan Lethagen, Lynda M Cristiano, Elisa Tsao, Bent Winding, Johnny Mahlangu, Longitudinal Analysis of Long-Term Safety and Efficacy of Recombinant Factor VIII Fc Fusion Protein (rFVIIIFc) in Previously Treated Children with Severe Hemophilia a, Blood, ASH Annual Meeting and Exposition, 2016, 128, (22), 2016, pp1414-Poster, 2016, URL
  • Michael Wang, MD, K John Pasi, Ingrid Pabinger, Prof, Bryce A. Kerlin, Roshni Kulkarni, MD, Beatrice Nolan, Raina Liesner, Simon A Brown, Hideji Hanabusa, Elisa Tsao, Bent Winding, Stefan Lethagen, Nisha Jain, Long-Term Efficacy and Quality of Life with Recombinant Factor VIII Fc Fusion Protein (rFVIIIFc) Prophylaxis in Pediatric, Adolescent, and Adult Subjects with Target Joints and Severe Hemophilia a, Blood, ASH Annual Meeting and Exposition, 2016, 128, (22), 2016, pp3791-Poster, 2016, URL
  • Jonathan M. Ducore, Kathelijn Fischer, Roshni Kulkarni, Beatrice Nolan, Carolyn M. Bennett, David J Perry, Huixing Yuan, Alejandra Ramirez-Santiago, Francesca Ferrante, Stefan Lethagen, Long Term Safety and Efficacy of Recombinant Factor IX Fc Fusion Protein (rFIXFc) Prophylaxis in Children with Hemophilia B: Interim Results of the B-Yond Extension Study, Blood, ASH Annual Meeting and Exposition, 2016, 128, (22), 2016, pp4976 - 4976Poster, 2016, URL
  • Gibson, L., Cronin, C., Nolan, B., Mitchell, T.H., A case of cerebral malaria, Journal of the Irish Colleges of Physicians and Surgeons, 25, (1), 1996, p17-18Journal Article, 1996
  • White, M., Twomey, A., Donoghue, V., Mac Keogh, L., Nolan, B., O'Carroll, T., Murphy, J.F.A., Molloy, E.J., Decreased fibrinogen levels are associated with severe intraventricular hemorrhage in very low birth weight (VLBW) infants, Journal of Neonatal-Perinatal Medicine, 4, (2), 2011, p133-136Journal Article, 2011, DOI
  • Saad Ahmed, Michelle O'Rourke, Irene Regan, Vince Jerkins, Beatrice Nolan, New insights in to Haemophilia B Leyden, European Haematology Association Annual Congress, On line, 2020, 2020Oral Presentation, 2020
  • Ruth Elizabeth Hunter Nolan1,2, Eibhlin Mc Laughlin1 , Yvonne Duane1 , Ann O' Sullivan2 , Kevin Ryan2 , Niamh O' Connell2 , Beatrice Nolan1, Adolescent feedback on the Haemophilia Transition Programme between Our Lady's Children's Hospital and St. James's Hospital Dublin Ireland. , International Journal of Integrated Care, 17th International Conference on Integrated Care,, Dublin, 8-10 May 2017, 17, (5), 2017Oral Presentation, 2017, URL
  • Swan D., Paran S., Nolan B., Port removal in patients receiving emicizumab prophylaxis: A single centre experience and review of the literature, Haemophilia, 28, (1), 2022, p42 - 45, p42-45Journal Article, 2022, DOI
  • Christoph Königs, Margareth C Ozelo, Amy Dunn, Roshni Kulkarni, Beatrice Nolan, Simon A Brown, Dr, Michele Schiavulli, Sriya Gunawardena, Sutirtha Mukhopadhyay, Deepthi Jayawardene, Bent Winding, Manuel Carcao, First study of extended half-life rFVIIIFc in previously untreated patients with hemophilia A: PUPs A-LONG final results, Blood, 139, (26), 2022, p3699-3707Journal Article, 2022, DOI , URL
  • Hassan Eliwan and Murwan Omer and Ellen McKenna and Lynne A. Kelly and Beatrice Nolan and Irene Regan and Eleanor J. Molloy, Protein C Pathway in Paediatric and Neonatal Sepsis, Frontiers in Pediatrics, 9, 2022Journal Article, 2022, DOI , URL
  • Beatrice Nolan and Michael Recht and Pablo Rendo and Aletta Falk and Meredith Foster and Sandra Casiano and Antoine Rauch and Amy D. Shapiro, Prophylaxis with rFIXFc Reduces the Frequency and Delays Time to First Spontaneous Bleed Event in Previously Untreated Patients with Hemophilia B: A Post Hoc Analysis of the PUPs B-LONG Trial, Blood, 138, (Supplement 1), 2021, p498--498Journal Article, 2021, DOI , URL
  • G. DOYLE, E. MCLAUGHLIN, M. KAVANAGH, R. HUNTER NO-LAN, D. CAWLEY and B. NOLAN, Inherited bleeding disorders: The nursing experience of patient and familycare needs in the pediatric setting, Haemophilia, XXX International Congress of the World Federation of Hemophilia. July 8-12, 2012. Paris, France, Paris, July8-12,2012, 18, (supplement 3), 2012, pp132 - 133Poster, 2012, URL
  • M. KAVANAGH, I. KELLY, E. MCLAUGHLIN, R. HUNTER-NOLAN,D. CAWLEY and B. NOLAN, A survey of trainee's perceptions of the hemophilia nurse specialist vein-training program, Haemophilia, XXX International Congress of the World Federation of Hemophilia, Paris, July 8-12,2012, 18, (Supplement 3), 2012, pp134 - 134Poster, 2012, URL
  • E. MCLAUGHLIN, I. KELLY, M. KAVANAGH, R. HUNTER-NOLAN,D. CAWLEY and B. NOLAN, A survey of parents's perceptions of support received from a hemophilianurse specialist during their child's portacath insertion, Haemophilia, XXX International Congress of the World Federation of Hemophilia,, Paris, July 8-12, 2012, 18, (Supplement 3), 2012, pp135 - 135Poster, 2012, URL
  • H. CHAMBOST, A. RAFOWICZ,Y. GUILLAUME,R. LIESNER,B. NOLAN,M. SHIMA, A. SHAPIRO and R. LJUNG, Perinatal practices in the context of hemophilia carrier: Expert opinion froman international pediatric panel, Haemophilia, XXX International Congress of the World Federation of Hemophilia,, Paris, July 8 to 12 , 2012, 18, (Supplement 3), 2012, pp148 - 148Poster, 2012, URL
  • U. RAO, I. KELLY, M. KAVANAGH, B. BRADY, K. FITZGERALD, B. NOLAN P. FLEMING, Audit of bleeding complications & cost of dental treatment for children with hereditary bleeding disorders using a new treatment protocol, Haemophiia, 6th Congress of the European Association for Haemophilia and Allied Disorders , Warsaw, 6-8 February 2013, 19, (S2), 2013, pp55 - 55Poster, 2013, DOI , URL
  • I E Regan, A Burke, O M Cormack, C Fox, B Nolan, APPLICATION OF THE US NATIONAL INSTITUTE OF HEALTH (NIH) 2008 GUIDELINES FOR VON WILLEBRAND DISEASE IN A NATIONAL PAEDIATRIC COMPREHENSIVE CARE CENTRE., Haematologica, Eurupean Haematology Association 19th Congress, Milan, 12-15 June 2014, 99, (supplement 1), 2014, pp209 - 209Poster, 2014, DOI , URL
  • Hugh O' Reilly, Michael Barrett, Laura Melody, David Rea, Mary O' Regan, Beatrice Nolan, This infant is having a stroke: an illustrative case report, IRISH JOURNAL OF MEDICAL SCIENCE, 2022Journal Article, 2022, DOI
  • Khan A.Z., Kavanagh M., Kelly I., Ferry C., Ahmed S., Nolan B., Intramuscular vaccination in children with haemophilia: A single centre experience and review of the literature, Haemophilia, 2023Journal Article, 2023, DOI
  • Shapiro, Amy D., Kulkarni, Roshni Daniel, Ragni, Margaret V, Chambost, Herve, Mahlangu, Johnny N, Oldenburg, Johannes, Nolan, Beatrice, Ozelo, Margareth C, Foster, Meredith C, Willemze, Annemieke, Barnowski, Chris, Jain, Nisha, Winding, Bent, Dumont, Jennifer, Lethagen, Stefan, Barnes, Chris, Pasi, K John, Post hoc longitudinal assessment of efficacy and safety of recombinant factor IX Fc fusion protein in hemophilia B, Blood Advances, 2023Journal Article, 2023, DOI
  • Kenet, Gili, Nolan, Beatrice, Matsushita, Tadashi, Young, Guy, Quan, Ting, Cano, Viridiana, Sussebach, Christian, Andersson, Shauna, Mei, Baisong, Afonso, Marion, Kavakli, Kaan, Fitusiran Prophylaxis Improves Health-Related Quality of Life in People with Hemophilia a or B, with or without Inhibitors: Results of ATLAS-PPX Study, Blood, 140, (Supplement 1), 2022, p7977-7978Journal Article, 2022, DOI
  • Van Der Zwet, Konrad, de Kovel, Marloes, Motwani, Jayashree, Van Geet, Christel, Nolan, Beatrice, Glosli, Heidi, Kenet, Gili, Fischer, Kathelijn, The Emicizumab Switch: Real-World Data of 251 Pediatric Patients from the Pednet Registry, Blood, 140, (Supplement 1), 2022, p8442-8443Journal Article, 2022, DOI
  • Nolan, B, Hinchcliffe R, Vora A, Neonatal pure red cell aplasia due maternal anti-M, Blood, American Society of Hematology 42nd Annual Meeting, San Francisco , California, USA, Dec 1-5, 2000, 96, (11), 2000, pp8 - 8Meeting Abstract, 2000, URL
  • C. KEENAN, H. EGAN, E. O"SHEA, B. NOLAN, B. WHITE and P. JENKINS, Identification of causative mutations and a possible founder effect withinIrish kindred with the bleeding disorder haemophilia A, Haemophilia, THE XXVIII INTERNATIONAL CONGRESS OF THE WORLD FEDERATION OF HEMOPHILIA, Istanbul, Turkey, 1-5 June 2008, 14, John Wiley and Sons, 2008, pp32-32Meeting Abstract, 2008, DOI , URL
  • O. HALVEY, S. JAMIESON, B. NOLAN, A. O"DWYER,J. COONEY and J. SMITH, Integrated psychosocial services: principles, goals and outcomes, Haemophilia, Abstracts of the XXVIIIth International Congress of the World Federation of Hemophilia, Istanbul, Tukey, June 1-5 , 2008, 14, John Wiley and Sons, 2008, pp149-149Meeting Abstract, 2008, DOI , URL
  • , Audit of bleeding complications & cost ofdental treatment for children with hereditarybleeding disorders using a new treatmentprotocolU. RAO1,*, I. KELLY1, M. KAVANAGH1,B. BRADY1, K. FITZGERALD2, B. NOLAN1,P. FLEMING, Haemophilia, 19, (s2), 2013, p10-82Journal Article, 2013, DOI
  • Eliwan HO, Watson WRG, Melo AM, Kelly LA, Omer M, Jafar A, O'Hare FM, Downey P, Mooney EE, O'Neill A, Blanco A, Regan I, Philbin B, O'Rourke M, Nolan B, Smith O, Molloy EJ., Selective modulation of monocyte and neutrophil responses with activated protein C in preterm infants., J Matern Fetal Neonatal Med, 36, (1), 2023, p2183467Journal Article, 2023, DOI
  • Young G., Lenting P.J., Croteau S.E., Nolan B., Srivastava A., Antithrombin lowering in hemophilia: a closer look at fitusiran, Research and Practice in Thrombosis and Haemostasis, 7, (4), 2023Journal Article, 2023, DOI
  • Susanna Ranta, Jayashree Motwani, Jan Blatny, Martina Bührlen, Manuel Carcao, Hervé Chambost, Carmen Escuriola, Kathelijn Fischer, Mutlu Kartal"Kaess, Marloes de Kovel, Tami Livnat, Christoph Male, Beatrice Nolan, Roseline d'Oiron, Martin Olivieri, Ester Zapotocka, Nadine G Andersson, Christoph Königs, Dilemmas on emicizumab in children with haemophilia A: A survey of strategies from PedNet centres, Haemophilia, 29, (5), 2023, p1291--1298Journal Article, 2023, DOI
  • Barbara A. Konkle, Johannes Oldenburg, John Pasi, Roshni Kulkarni, Beatrice Nolan, Johnny Mahlangu, Guy Young, Simon A. Brown, Ingrid Pabinger, Amy Shapiro, Claude Négrier, Victor Blanchette, Margaret V. Ragni, Jennifer Dumont, Stefan Lethagen, Prophylaxis with a recombinant factor VIII Fc in hemophilia A: long-term follow-up on joint health, efficacy, and safety from phase 3 studies in children and adults, Research and Practice in Thrombosis and Haemostasis, 7, (6), 2023, p102180Journal Article, 2023, DOI
  • , Unsuspected haemophilia in children with a single swollen joint., BMJ (Clinical research ed.), 2003Journal Article, 2003, DOI
  • Jenkins PV, Egan H, Keenan C, O'Shea E, Smith OP, Nolan B, White B, O'Donnell J., Mutation analysis of haemophilia B in the Irish population: increased prevalence caused by founder effect., Haemophilia, 14, (4), 2008, p717-22Journal Article, 2008, DOI
  • Eliwan HO, O'Hare FM, Watson WR, O'Neill A, Molloy EJ, Neonatal Brain Injury and Systemic Inflammation: Modulatin by Activated Protein c ex vivo, Clinical Exp Immunology, 179, (3), 2015, p477 - 484Journal Article, 2015, DOI
  • Beatrice Nolan, Hemophilia A (Case Based Presentation) ISTH Academy. Nolan B. 07/19/18; 223593 Topic: Hemophilia - Clinical, Dublin, 19/07/2018, 2018Invited Talk
  • Beatrice Nolan, ITI in era of Emicizumab, 64th Annual Meeting of Scientific and Standardization Committee of ISTH, Dublin, 20 July 2018, 2018, ISTH SSCInvited Talk
  • Beatrice Nolan, Prophylaxis for children with haemophilia, The Second International Medical Conference on Hemophilia, Thalassaemia and Sickle Cell Disease, Amman, Jordan, 26 -27 September , 2019, Jordanian Ministry of Health, Jordanian Thalassaemia and Hemophilia Society, World Federation of Hemophilia, Thalassemia International FederationInvited Talk
  • Beatrice Nolan, Inhibitors in Haemophilia A, The Third International Medical Conference on Hemophilia, Thalassaemia and Sickle Cell Disease, Amman, Jordan, 2022, Jordanian Ministry of Health, Jordanian Thalassaemia and Hemophilia Society, World Federation of Hemophilia, Thalassemia International FederationInvited Talk

Research Expertise

  • Title
    An Open-Label, Multicenter Evaluation of Safety, Pharmacokinetics, and Efficacy of Recombinant Coagulation Factor VIII Fc Fusion Protein, BIIB031, in the Prevention and Treatment of Bleeding Episodes in Pediatric Subjects With Hemophilia A
    Summary
    The primary objective of the study is to evaluate the safety of Recombinant Coagulation Factor VIII Fc Fusion Protein (rFVIIIFc) in previously treated pediatric subjects with hemophilia A. Secondary objectives of this study in this study population are as follows: to evaluate the efficacy of rFVIIIFc for prevention and treatment of bleeding episodes; to evaluate and assess the pharmacokinetics (PK) of rFVIIIFc; and to evaluate rFVIIIFc consumption for prevention and treatment of bleeding episodes.
    Funding Agency
    Bioverativ Therapeutics Inc.
  • Title
    Study of Recombinant Coagulation Factor IX Fc Fusion Protein, BIIB029, in Previously Treated Pediatric Participants With Hemophilia B
    Summary
    . The primary objective of the study is to evaluate the safety of Recombinant Human Coagulation Factor IX Fc Fusion Protein (rFIXFc) in previously treated pediatric subjects with hemophilia B. Secondary objectives of this study in this study population are as follows: to evaluate the efficacy of rFIXFc for prevention and treatment of bleeding episodes; to evaluate and assess the pharmacokinetics (PK) of rFIXFc; to evaluate rFIXFc consumption for prevention and treatment of bleeding episodes
    Funding Agency
    Bioverative Therapeutics Inc
  • Title
    A 24-month Prospective, Non-interventional, International, Multicentre Study to Describe the Real-world Effectiveness and Usage of Alprolix in Patients With Haemophilia B
    Summary
    Alprolix (rFIXFc) is a recombinant extended half-life coagulation factor product. The purpose of this non-interventional study is to describe the real-world usage and effectiveness of Alprolix in the on-demand and prophylactic treatment of haemophilia B.
    Funding Agency
    Swedish Orphan Biovitrum
  • Title
    BAX 326 (Recombinant Factor IX): Evaluation of Safety, Immunogenicity, and Hemostatic Efficacy in Previously Treated Patients With Severe (FIX Level
    Summary
    The purpose of this BAX 326 Continuation Study is to further investigate incremental recovery over time, the hemostatic efficacy, the safety, immunogenicity, and health-related quality of life (HR QoL) of BAX 326 in previously treated patients (PTPs) with severe and moderately severe hemophilia B who participated in BAX 326 pivotal study 250901 or BAX 326 pediatric study 251101.
    Funding Agency
    CSL Behring
  • Title
    A Multicenter Phase III Uncontrolled Open-label Trial to Evaluate Safety and Efficacy of BAY81-8973 in Children With Severe Hemophilia A Under Prophylaxis Therapy
    Summary
    The primary objective was to evaluate the safety and efficacy of the treatment with BAY81-8973 for prophylaxis and treatment of breakthrough bleeds in children with severe hemophilia A.
    Funding Agency
    Bayer
  • Title
    An Open-Label, Multicenter Evaluation of the Safety and Efficacy of Recombinant Coagulation Factor IX Fc Fusion Protein (rFIXFc; BIIB029) in the Prevention and Treatment of Bleeding in Previously Untreated Patients With Severe Hemophilia B
    Summary
    The primary objective of the study was to evaluate the safety of recombinant coagulation factor IX Fc fusion protein (rFIXFc, BIIB029) in previously untreated patients (PUPs) with severe hemophilia B. Secondary objectives were to evaluate the efficacy of rFIXFc in the prevention and treatment of bleeding episodes in PUPs, and to evaluate rFIXFc consumption for prevention and treatment of bleeding episodes in PUPs.
    Funding Agency
    Bioverativ, a Sanofi company
  • Title
    Safety, Efficacy and PK of BIVV001 in Pediatric Patients With Hemophilia A (XTEND-Kids)
    Summary
    Primary Objective:- To evaluate the safety of BIVV001 in previously treated pediatric subjects with hemophilia A
    Funding Agency
    Bioverativ, a Sanofi company
  • Title
    An Open-Label, Multicenter Evaluation of the Long-Term Safety and Efficacy of Recombinant Human Coagulation Factor VIII Fusion Protein (rFVIIIFc) in the Prevention and Treatment of Bleeding Episodes in Previously Treated Subjects With Hemophilia A
    Summary
    Funding Agency
    Bioverative Therapeutics Inc
  • Title
    Prophylactic Treatment With Recombinant Factor VIIa (rFVIIa, NovoSeven®) in Haemophilia Patients With Inhibitors
    Summary
    This study is conducted in Europe and North and South America. The primary aim of this observational study is to evaluate the frequency and pattern of bleeding episodes in haemophilia patients receiving preventative treatment with activated recombinant human factor VII. The secondary aim is to evaluate which patients are selected for this treatment, the dose and dose intervals used, and the safety of activated recombinant human factor VII when used as prevention. The study also aims to increase understanding of the unmet medical need and clinical relevance of preventative treatment in haemophilia patients.
    Funding Agency
    Novo Nordisk A/S
  • Title
    A Multinational, Open-label, Randomised, Controlled Study to Investigate Efficacy and Safety of NNC0365-3769 (Mim8) in Adults and Adolescents With Haemophilia A With or Without Inhibitors
    Summary
    This study is investigating how Mim8 works compared to other medicines in people with haemophilia A, who either have inhibitors or do not have inhibitors. Mim8 is a new medicine that will be used for prevention of bleeding episodes. Mim8 works by replacing the function of the missing clotting factor VIII (FVIII). When and how often participants will receive Mim8 is dependent on their previous treatment - but is otherwise decided by chance. Mim8 will be injected into a skinfold on the stomach with a thin needle either once a week or once a month. The study will last 54-124 weeks (12-29 months) depending on how long participants will be followed in run-in before they start treatment and if they continue in the follow period or transfer to an open label extension study. Participants will have 12-17 clinic visits.
    Funding Agency
    Novo Nordisk A/S
  • Title
    Single-dose Pilot Study of Oral Rivaroxaban in Pediatric Subjects With Venous Thromboembolism
    Summary
    The first study with rivaroxaban in pediatric subjects is a Phase I study, where the pharmacokinetic/pharmacodynamic (PK/PD) profile of rivaroxaban will be investigated to confirm that the exposure is comparable to adults. This study is a single dose study with multiple PK/PD measurements in pediatric subjects at the end of their Venous Thromboembolism (VTE) treatment.
    Funding Agency
    Bayer
  • Title
    A Non-Controlled, Open-Label, Multicenter, Study of Immune Tolerance Induction Performed With rFVIIIFc Within a Timeframe of 60 Weeks in Severe Haemophilia A Patients With Inhibitors Who Have Failed Previous Immune Tolerance Induction Therapies
    Summary
    The primary purpose of this study is to describe the outcome of Immune Tolerance Induction (ITI) treatment performed with rFVIIIFc within a timeframe of 60 weeks in patients with haemophilia A who have failed previous attempts at tolerization.
    Funding Agency
    Swedish Orphan Biovitrum
  • Title
    An Open Label Study to Determine the Safety and Efficacy of Replacement Factor VIII Protein (Known as rFVIIIFc) in Previously Untreated Males With Severe Hemophilia A
    Summary
    The primary objective of the study was to evaluate the safety of rFVIIIFc (BIIB031) in previously untreated participants (PUPs) with severe hemophilia A. The secondary objectives were to evaluate the efficacy of rFVIIIFc in the prevention and treatment of bleeding episodes in PUPs, to evaluate rFVIIIFc consumption for the prevention and treatment of bleeding episodes in PUPs, and to describe experience with the use of rFVIIIFc for immune tolerance induction (ITI) in participants with inhibitors.
    Funding Agency
    Bioverativ, a Sanofi company
  • Title
    A Chart Review Study of Patients With Haemophilia A With Inhibitors Treated With rFVIIIFc (Elocta®) for Immune Tolerance Induction
    Summary
    Funding Agency
    Swedish Orphan Biovitrum
  • Title
    An Open-Label, Multicenter, Evaluation of the Long-Term Safety and Efficacy of Recombinant Human Coagulation Factor IX Fusion Protein (rFIXFc) in the Prevention and Treatment of Bleeding Episodes in Previously Treated Subjects With Hemophilia B
    Summary
    The primary objective of the study is to evaluate the long-term safety of rFIXFc in participants with hemophilia B.
  • Title
    The European Paediatric Network for Haemophilia Management and the PedNet Haemophilia Registry
    Summary
    Rationale: Haemophilia is a rare disease; to improve knowledge international collaboration is needed. Well-defined clinical data will be collected from complete cohorts in order to prevent selection bias. Objective: To collect data on bleeding during neonatal period, endogenous (genetic) and exogenous (treatment-related) determinants of inhibitor development and long term outcome.
    Funding Agency
    PedNet Hemophilia Research Foundation
  • Title
    Multicenter, Open-label, Active-controlled, Randomized Study to Evaluate the Efficacy and Safety of an age-and Body Weight-adjusted Rivaroxaban Regimen Compared to Standard of Care in Children With Acute Venous Thromboembolism
    Summary
    The purpose of this study is to evaluate comparative efficacy and safety of rivaroxaban to standard of care in children with acute venous thromboembolism.
    Funding Agency
    Bayer
  • Title
    A Study of Fitusiran in Severe Hemophilia A and B Patients Previously Receiving Factor or Bypassing Agent Prophylaxis (ATLAS-PPX)
    Summary
    Primary Objective: To characterize the frequency of bleeding episodes while receiving fitusiran treatment, relative to the frequency of bleeding episodes while receiving factor or bypassing agent (BPA) prophylaxis
    Funding Agency
    Genzyme, a Sanofi Company

Recognition

  • International Society of Thrombosis and Hemostasis
  • Member of European Association of Haemophilia and Allied Disorders
  • Member of Royal Academy of Medicine in Ireland
  • Member of HPRA Panel of External Clinical Experts
  • Member of Royal Society of Medicine
  • Member of European Haematology Association