Modern management of patients with paroxysmal nocturnal hemoglobinuria

Since the advent of therapeutic complement inhibition, the management of patients with paroxysmal nocturnal haemoglobinuria (PNH) has improved significantly, leading to reduced risk of thrombosis, transfusion-independence, improved pregnancy outcomes, and even affording near-normal survival in many. Despite these gains, ongoing issues remain, such as persistence of fatigue and infection risk, the potential of breakthrough haemolysis. Some patients experience an iatrogenic extravascular haemolysis, which can be clinically significant. Proximal complement inhibition has vastly improved this effect, and can provide alternative treatment schedules and modes of delivery for patients. Shared decision-making with patients is crucial to ensure that the complement inhibitor prescribed properly suits their disease characteristics, comorbidities, and lifestyle to ensure proper adherence. Greater focus has been given to development of disease-specific patient reported outcomes to better reflect the patient journey and to holistically assess response to modern treatment options.Despite the major strides forward in PNH management with the advent of complement inhibition, several unmet needs remain. There is also variability in practice globally, due at least in part to variable access to testing and therapeutic options. The Research Topic will focus on these various aspects of disease management, treatment of complications, special populations, disease monitoring, shared decision-making, and validated assessment of patient-reported outcomes, including but not limited to: &#8226 The complexities of patient choice across therapies &#8226 The use of complement inhibition in special populations (e.g. pregnancy, concomitant marrow failure) &#8226 The exploration of breakthrough haemolysis risks and management&#8226 The validation of novel patient-reported outcome measures&#8226 The variable access to complement inhibitors globally Topic editors: Christopher Patriquin, Louise Arnold Source: https://www.frontiersin.org/research-topics/81860/modern-management-of-patients-with-paroxysmal-nocturnal-hemoglobinuria