Even mild hemolysis in paroxysmal nocturnal hemoglobinuria could severely compromise the quality of life due to long-term sustained intolerant fatigue

Abstract

Fatigue is one of the most common symptoms associated with paroxysmal nocturnal hemoglobinuria (PNH), a rare acquired disorder of hematopoietic stem cells. While it directly impairs lifestyle leading to poor quality of life (QOL), it is not well recognized that fatigue could not depend on the disease activity or percentage of glycosylphosphatidylinositol-deficient granulocyte. We describe the case of a 78-year-old woman, with mild hemolysis and a 20-year history of severe sustained fatigue, whose QOL drastically improved with eculizumab followed by ravulizumab. We also used the novel aplastic anemia and PNH specific QOL tool to evaluate multiple statuses of the patient.

Quality of life after immune suppressive therapy in aplastic anemia

“…This questionnaire addresses implications of the disease on physical, psychological and social functioning to get insight in overall QoL of AA or PNH patients. In current published literature only one case-report reports using the QLQ-AA/PNH-54 questionnaire to evaluate QoL of a PNH patient before and after treatment [ 18 ]. Given the low incidence of AA, inclusion of sufficient patient numbers can be challenging.…”

Fatigue and health‐related quality of life in paroxysmal nocturnal haemoglobinuria: A post hoc analysis of the pegcetacoplan PEGASUS trial data

“…Although the exact mechanism that leads to fatigue in PNH is not entirely clear, depletion of nitric oxide due to IVH has been proposed as being central in its manifestation 17 . Historically, fatigue in PNH patients has also been linked to chronic haemolysis 18,19 . Therefore, IVH prevention remains the key goal of PNH therapeutic management, which until recently, has been led by treatment with humanised monoclonal antibodies against complement component 5 (C5) 20 .…”

“…17 Historically, fatigue in PNH patients has also been linked to chronic haemolysis. 18,19 Therefore, IVH prevention remains the key goal of PNH therapeutic management, which until recently, has been led by treatment with humanised monoclonal antibodies against complement component 5 (C5). 20 These therapies effectively reduce IVH as well as complement-mediated thrombosis and associated mortality.…”

The Effect of Respiratory Viral Infections on Breakthrough Hemolysis in Patients with Paroxysmal Nocturnal Hemoglobinuria

“…Early diagnosis and treatment of an acute hemolytic process is critical, as hemolysis itself is a risk factor for thrombosis and organ damage [9]. Furthermore, there is a significant impairment in the quality of life in patients with recurrent or acute hemolytic episodes [10]. It is of particular importance that the propensity for complement-induced lysis is further exaggerated by various situations, both physiological, such as the intravascular acidic state occurring during sleep [7] and pregnancy, and pathological, such as trauma, surgical procedures, vaccinations, and infections [9,10].…”

“…Furthermore, there is a significant impairment in the quality of life in patients with recurrent or acute hemolytic episodes [10]. It is of particular importance that the propensity for complement-induced lysis is further exaggerated by various situations, both physiological, such as the intravascular acidic state occurring during sleep [7] and pregnancy, and pathological, such as trauma, surgical procedures, vaccinations, and infections [9,10]. Eculizumab is a long-acting humanized monoclonal antibody targeted against complement C5, inhibiting its cleavage into C5a and C5b.…”