Beneficial effects of eculizumab regardless of prior transfusions or bone marrow disease: Results of the International Paroxysmal Nocturnal Hemoglobinuria Registry

Röth, Alexander; Araten, David J.; Kulasekararaj, Austin; Maciejewski, Jaroslaw P.; Wilson, Amanda; Gustovic, Philippe; Kanakura, Yuzuru

doi:10.1111/ejh.13485

Cited by 6 publications

(5 citation statements)

References 30 publications

(53 reference statements)

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“…Eculizumab protects vulnerable red blood cells that lack CD55 and C59 from complement-mediated hemolysis [ 16 , 17 ]. It is a highly effective monoclonal antibody associated with a 70% reduction in the risk of thrombotic events and significant adverse vascular complications [ 18 ].…”

Section: Discussionmentioning

confidence: 99%

Attack of the Clones: A Patient With Untreated Aplastic Anemia Presenting With Classical Paroxysmal Nocturnal Hemoglobinuria

Rayas¹,

Hassan²,

Hock³

et al. 2023

Cureus

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Paroxysmal nocturnal hemoglobinuria (PNH) is an acquired X-linked, clonal hematopoietic stem cell disease. Patients with PNH may complain of vague symptomatology that contributes to the challenge of its diagnosis. This is especially true in the clinical context of a coinciding hematologic disorder. Aplastic anemia (AA) is an additional immune-mediated illness that results in the destruction of hematopoietic precursors and pancytopenia. The authors encourage screening for PNH clones in patients initially diagnosed with AA, treating underlying hematologic disease to prevent clonal expansion, and further research to investigate the effectiveness of eculizumab in an unusual "classical" PNH secondary to AA with hypercellular bone marrow.

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Section: Discussionmentioning

confidence: 99%

Attack of the Clones: A Patient With Untreated Aplastic Anemia Presenting With Classical Paroxysmal Nocturnal Hemoglobinuria

Rayas¹,

Hassan²,

Hock³

et al. 2023

Cureus

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“…R ): This humanized monoclonal antibody (MA) against complement component 5(C5) from mouse has been very successfully used for the treatment of paroxysmal nocturnal hemoglobinuria (PNH), atypical hemolytic uremic syndrome (aHUS) and neuromyelitis optica. [26,27,28] Bacterial, i.e. neisserial and meningococcal meningitis is one of the important side effects of this therapy; hence, all patients on this medication as chronic therapy must receive meningococcal vaccination [29] Once started for paroxysmal nocturnal hemoglobinuria (PNH), this therapy should not be suddenly stopped as the complement sensitive red cells which accumulates during the therapy may hyperhemolyse in the absence of this inhibitor with attendant complications.…”

Section: Description and Uses Of Monoclonal Antibodiesmentioning

confidence: 99%

Monoclonal antibodies used for management of hematological disorders

Ghosh

2021

JHAS

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Objectives: Monoclonal antibodies (MAs) are increasingly becoming part of therapeutic armamentarium for hematologists and hemato-oncologists. There is paucity of review on majority of these antibodies in one place. The objective of this review is an attempt to fill the gap in paucity of review on majority of these monoclonal antibodies (MAs) in one place. Material and Methods: ‘Pubmed’ and ‘Scopus’ database was explored focusing on monoclonal antibodies (MAs) in clinical hematological practice. Emphasis was given to the more recently published review articles on different monoclonal antibodies (MAs). Results: In the present review, a total of 23 different monoclonal antibodies (MAs) were discussed; some are very frequently used and some rarely. Monoclonal antibodies (MAs) are used for treatment of diverse hematological conditions, i.e. malignant and benign disorders and at various phases of stem cell transplantation. These antibodies were used either alone or in combination with various chemotherapeutic agents, targeted small molecules or as immunoconjugates. Some of the side effect profiles of these antibodies were common and some were unique to the particular monoclonal antibody (MA). Unusual infections or organ dysfunctions were noted. Improved function of antibodies by protein engineering is also advancing rapidly. Dosage, frequency and route of administration depended on the convenience and condition for which the antibody is used. Conclusion: Monoclonal antibodies (MAs) are going to stay for hematological practice. Some amount of familiarity with their usage, advantages, disadvantages and side effects are essential in clinical practice.

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“…These estimates include patients with both clinical and subclinical PNH. However, the actual number of diagnosed patients with PNH with high disease activity and who require treatment is lower: in the Netherlands, there are approximately 140 patients diagnosed with PNH, 80–90 of whom require treatment with a complement C5 inhibitor [ 6 , 7 ]. Though PNH is a chronic disease, it may resolve for some patients, and approximately 5.6–15% of patients with PNH have been found to experience spontaneous remission [ 8 , 9 ].…”

Section: Introductionmentioning

confidence: 99%

Cost-effectiveness of ravulizumab compared with eculizumab for the treatment of paroxysmal nocturnal hemoglobinuria in the Netherlands

Quist¹,

Postma²,

Myrén

et al. 2023

Eur J Health Econ

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Objectives The aim of this study was to evaluate the cost-effectiveness of ravulizumab compared with eculizumab for the treatment of adult patients with paroxysmal nocturnal hemoglobinuria (PNH) in the Netherlands. Methods A cost-effectiveness analysis was conducted based on a Markov cohort model simulating the course of patients with PNH with clinical symptom(s) indicative of high disease activity, or who are clinically stable after having been treated with eculizumab for at least the past six months. Costs, quality of life, and the incremental cost-effectiveness ratio (ICER) were estimated over a lifetime horizon from a Dutch societal perspective. Several additional analyses were performed, including a one-way sensitivity analysis, a probabilistic sensitivity analysis, and scenario analysis. Results When compared with eculizumab, ravulizumab saves €266,833 and 1.57 quality adjusted life years (QALYs) are gained, resulting in a dominant ICER. Drug costs account for the majority of the total costs in both intervention groups. Cost savings were driven by the difference in total treatment costs of ravulizumab compared with eculizumab caused by the reduced administration frequency, accounting for 98% of the total cost savings. The QALY gain with ravulizumab is largely attributable to the improved quality of life associated with less frequent infusions and BTH events. At a willingness-to-pay threshold of €20,000/QALY, there is a 76.6% probability that ravulizumab would be cost-effective. Conclusions The cost reduction and QALY gain associated with the lower rates of BTH and less frequent administration make ravulizumab a cost-saving and clinically beneficial substitute for eculizumab for adults with PNH in the Netherlands.

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Beneficial effects of eculizumab regardless of prior transfusions or bone marrow disease: Results of the International Paroxysmal Nocturnal Hemoglobinuria Registry

Cited by 6 publications

References 30 publications

Attack of the Clones: A Patient With Untreated Aplastic Anemia Presenting With Classical Paroxysmal Nocturnal Hemoglobinuria

Attack of the Clones: A Patient With Untreated Aplastic Anemia Presenting With Classical Paroxysmal Nocturnal Hemoglobinuria

Monoclonal antibodies used for management of hematological disorders

Cost-effectiveness of ravulizumab compared with eculizumab for the treatment of paroxysmal nocturnal hemoglobinuria in the Netherlands

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