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    Faculty & Disclosures
    Dr Vickie McDonald

    Guy’s & St Thomas’ NHS Foundation Trust, London, UK

    Dr Vickie McDonald is a consultant haematologist at Guy’s and St Thomas’ NHS Foundation Trust, London, UK. Dr McDonald’s main interests are disorders of coagulation, including immune thrombocytopenia (ITP). read more

    Dr McDonald is chief investigator of the UK Adult ITP Registry and principal investigator for several clinical trials for patients with ITP in the UK.

    Dr Vickie McDonald discloses: Advisory board or panel fees from Amgen, Novartis and Sobi. Grants/research support from Grifols (relationship terminated).
    Prof. Waleed Ghanima

    Østfold Hospital, Oslo, Norway

    Prof. Waleed Ghanima is the head of research and a consultant haematologist at Østfold Hospital, Oslo, Norway. He is also a professor in the Institute of Clinical Medicine at the University of Oslo. read more

    Prof. Ghanima gained his medical degree from the College of Medicine, University of Baghdad, Iraq. He joined Østfold Hospital in 1996, becoming a consultant haematologist in 2004, and he assumed his current position as head of research in 2014.

    Prof. Ghanima’s main research field is venous thrombosis and immune thrombocytopenia. He is a member of several professional societies and was President of the Norwegian Society of Haematology from 2013 to 2017. He is well published, with more than 185 articles in national and international peer-reviewed journals, including The New England Journal of Medicine, The Lancet, Blood, Thrombosis and Haemostasis, Haematologica, British Journal of Haematology and Thrombosis Research.

    Prof. Waleed Ghanima discloses: Advisory board or panel fees from Alpine, Amgen, Argenx, Cellphire, Grifols, HI-Bio, Hutchmed, Kedrion, Novartis, Pfizer, Principia Biopharma Inc, Sanofi, Sobi, Takeda and UCB. Grants/research support from Sanofi, Sobi and UCB. Speaker fees from Amgen, Bayer, Bristol Myers Squibb, Grifols, Novartis, Pfizer, Sanofi and Sobi.
    Prof. James B Bussel

    Weill Cornell Medicine, New York, NY, USA

    Prof. James Bussel serves as Emeritus Professor of Pediatrics, Medicine and Obstetrics and Gynecology at Weill Medical College of Cornell University, New York, NY, USA. read more

    Prof. Bussel is affiliated with the New York Presbyterian Hospital and founded and served for many years as the director of the Platelet Disorders Program. Prior to his current appointments, Prof. Bussel served as a haematologist at New York Presbyterian Hospital, Memorial Hospital for Cancer and Allied Diseases, and Lenox Hill Hospital.

    Prof. Bussel is board certified in paediatrics and paediatric haematology/oncology. He specializes in haematology, in particular immune thrombocytopenia (ITP). Throughout his distinguished career, Prof. Bussel has treated rare haematological conditions in both children and adults with unique and extensive expertise in ITP, other thrombocytopenias, including Evans syndrome, and foetal and neonatal alloimmune thrombocytopenia (FNAIT). Prof. Bussel was the first physician in the USA to use intravenous immunoglobulin as a treatment for ITP, which helped to pave the way for FDA approval in this indication and its application as the standard of care for FNAIT-affected pregnancies.

    After receiving his undergraduate degree from Yale University, Prof. Bussel earned his medical degree from Columbia College of Physicians and Surgeons. He completed an internship and residency in paediatrics at Cincinnati Children’s Hospital, followed by a fellowship in paediatric haematology/oncology at New York Hospital-Cornell University Medical Center and Memorial Sloan Kettering Cancer Center, where he became chief fellow. Though his training was in paediatrics, Prof. Bussel’s expertise in these rare conditions has resulted in him routinely treating both children and adults in his practice.

    Among his many achievements, Prof. Bussel has authored over 300 peer-reviewed research publications, over 40 book chapters and contributed to over 140 medical reviews, including authoring nine articles in The New England Journal of Medicine. He is a reviewer for medical journals, such as The New England Journal of Medicine, Blood, Pediatric Blood & Cancer and the British Journal of Haematology, for which he is an associate editor. Prof. Bussel has acted as the primary investigator and co-investigator for many research trials in ITP and has presented both nationally and internationally on topics related to his specialty. Prof. Bussel has received a number of named lectureships. He is the recipient of several prestigious awards including sharing the King Faisal Prize for Medicine in 2012 and receiving the Henry M. Stratton Medal for translational/clinical science from the American Society of Hematology in 2023.

    Prof. James B Bussel discloses: Advisory board or panel fees from Argenx, Alpine-Vertex, Janssen, Rallybio, Sobi and UCB.
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    Rare blood disorders pose a unique set of diagnostic and therapeutic challenges. Their subtle presentation can often mimic more prevalent conditions, leading to delayed diagnoses and treatment. However, recent breakthroughs in precision medicine are transforming the management of these conditions, with a focus on pioneering research in disorders like haemophilia, sickle cell disease, and transthyretin amyloidosis.

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    Rare Diseases Content

    Thrombocytopenia, Rare Diseases, Autoimmune Blood Disorders
    Watch Time: 51 mins
    touchCONGRESS
    Insights from EHA and ISTH 2024: How can we optimize care for patients with chronic ITP?
    CE/CME ACCREDITED Dr Vickie McDonald, Prof. Waleed Ghanima, Prof. James B Bussel Translations available:

    Three experts discuss data presented at EHA and ISTH 2024 on the impact and management of ITP.

    Learning objectives
    Learning Objectives
    • Recognize the impact of ITP on patients and their quality of life
    • Appraise the latest data for current chronic ITP treatments and how they may be applied to clinical practice to optimize patient outcomes
    • Evaluate the latest data for emerging chronic ITP therapies and their potential to impact patient outcomes
    Rare Diseases
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    Phase III APPLY-PNH and APPOINT-PNH trials investigate iptacopan monotherapy in paroxysmal nocturnal haemoglobinuria, 48-week data: Phillip Scheinberg, ASH 2023
    Watch Time: 13 mins

    The 48-week data from the phase III APPLY-PNH and APPOINT-PNH trials investigates iptacopan, a first oral proximal complement inhibitor targeting factor B, in patients with paroxysmal nocturnal haemoglobinuria. We met with Dr Phillip Scheinberg (Hospital A Beneficência Portuguesa, São Paulo, Brazil) to discuss the data, and he reports the proportion of patients with clinical breakthrough […]

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