BJH - volume 15, issue 8, december 2024
G. Vermeersch MD, T. Devos MD, PhD
SUMMARY
Paroxysmal nocturnal haemoglobinuria (PNH) is characterised by the deficiency of glycosylphosphatidylinositol (GPI) anchored proteins, such as CD55 and CD59, on hematopoietic cells resulting in uncontrolled complement activation and haemolysis. Ravulizumab is a new monoclonal antibody with high affinity for complement factor C5 and hereby inhibits complement activation. Due to its significant longer half-life time compared to eculizumab, another C5 targeting complement inhibitor, ravulizumab, is administered intravenously once every eight weeks. Since November 2023, ravulizumab is reimbursed in Belgium for patients with haemolysis and showing clinical symptoms of high disease burden, as well as for clinically stable patients who are treated with eculizumab for at least six months.
(BELG J HEMATOL 2024;15(8):318–24)
Read more
BJH - volume 13, issue 6, october 2022
G. Vermeersch MD, W. Janssens MD, PhD, S. Bos MD, M. Garmyn MD, PhD, J. Maertens MD, PhD
SUMMARY
Yellow Nail Syndrome (YNS) is a rare entity characterised by the triad of nail discolouration, lung manifestations/sinusitis and lymphoedema. With ongoing debate, the exact aetiological mechanisms of YNS remain unknown. YNS is associated with various conditions such as malignancies, autoimmune and immuno-deficiency diseases. Some authors consider YNS as a paraneoplastic phenomenon due to its association with malignancies. Here we report the first patient presenting with the typical triad of YNS and a consecutive diagnosis of acute myeloid leukaemia with recurrent genetic abnormalities (KMT2A-PTD). Nail symptoms showed partial recovery after initiation of chemotherapy. Currently, the patient is off therapy and remains in first complete remission. More research to identify the exact pathophysiological mechanism and clinical significance of YNS is needed.
(BELG J HEMATOL 2022;13(6):249–52)
Read more
Top
