Eculizumab in refractory catastrophic antiphospholipid syndrome: a case report and systematic review of the literature

Tinti, Maria Giulia; Carnevale, Vincenzo; Inglese, Michele; Molinaro, Francesca; Bernal, Marco A. Bustamante; Migliore, Alberto; Cata, Angelo De

doi:10.1007/s10238-019-00565-8

Cited by 53 publications

(53 citation statements)

References 32 publications

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“…Their characteristics are presented in Table 3. 1–10 All patients, including the one described in this study, fulfilled the diagnostic criteria of definitive (four criteria present) or probable (three criteria present) CAPS. Eight out of 11 patients were diagnosed with primary CAPS, while the other three had CAPS in the setting of SLE.…”

Section: Resultsmentioning

confidence: 99%

Thrombotic microangiopathy in the course of catastrophic antiphospholipid syndrome successfully treated with eculizumab: case report and systematic review of the literature

Skoczyńska

Crowther

Chowaniec

et al. 2020

Lupus

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Objective The purpose of this study was to characterize the role of eculizumab, a monoclonal antibody against the terminal complement component C5, in patients with catastrophic antiphospholipid syndrome (CAPS). Methods We present a case report of a patient with systemic lupus erythematosus (SLE) and CAPS treated with eculizumab, as well as results of a systematic review of the literature. Results Including our patient, we identified 11 case reports of patients with CAPS treated with eculizumab. All of them had partial or total remission of symptoms. Conclusion Data on eculizumab efficacy in CAPS are promising but are limited to single case reports. More studies are needed to develop evidence-based recommendations for eculizumab use in CAPS.

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Section: Resultsmentioning

confidence: 99%

Thrombotic microangiopathy in the course of catastrophic antiphospholipid syndrome successfully treated with eculizumab: case report and systematic review of the literature

Skoczyńska

Crowther

Chowaniec

et al. 2020

Lupus

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“…It is currently used in the treatment of paroxysmal nocturnal hemoglobinuria (PNH), atypical uremic syndrome (aHUS), generalized myasthenia gravis (gMG), and neuromyelitis optica spectrum disorder (NMOSD) [13]. There have been case reports showing the success of eculizumab in the treatment of refractory CAPS [14][15][16][17][18][19]. Our patient was started on an induction dose of 600 mg with undetectable complement levels which suggested appropriate medication activity.…”

Section: Discussionmentioning

confidence: 91%

Therapeutic Challenges with Catastrophic Antiphospholipid Antibody Syndrome: A Case Report

Harne

Sampat

Bisen

et al. 2020

Cureus

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Antiphospholipid antibody syndrome (APS) is a multisystem disorder characterized by thromboembolic events in the presence of antiphospholipid antibodies (APLA). Catastrophic antiphospholipid antibody syndrome (CAPS) is an uncommon variant of APS which is associated with widespread coagulopathy that predominantly affects small vessels. Despite maximal treatment, CAPS has a very high mortality rate. We present a case of a 42-year-old woman with a history of APS who presented to our hospital with complaints of epistaxis, hemoptysis, menorrhagia, and shortness of breath. She was diagnosed with CAPS and developed multiorgan failure and sepsis. Despite maximal treatment with immune modulators, she unfortunately succumbed. With this case, we highlight the importance of early recognition of CAPS and review various treatment modalities that have been proven beneficial. Despite these modalities, CAPS remains a therapeutic challenge in many cases and has a high mortality rate.

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“…However, like with AAV, there is increasing evidence that complement is of importance (Salmon and Girardi, 2004;Meroni et al, 2016). Particularly when this condition exacerbates to a severe catastrophic APS (CAPS), a systemic life-threatening condition, complement inhibitor therapy has been very efficient (Tinti et al, 2019) and was most likely lifesaving in a patient with recurrent CAPS who repeatedly responded on complement inhibition (Barratt-Due et al., 2016). Based on what we know about the pathophysiology and what is published, we suggest that AAV and CAPS are reasonably good candidates on the list of diseases to be treated with complement inhibitors in the future.…”

Section: Therapeutic Complement Inhibitionmentioning

confidence: 99%

Therapeutic Targeting of the Complement System: From Rare Diseases to Pandemics

2021

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The complement system was discovered at the end of the 19th century as a heat-labile plasma component that “complemented” the antibodies in killing microbes, hence the name “complement.” Complement is also part of the innate immune system, protecting the host by recognition of pathogen-associated molecular patterns. However, complement is multifunctional far beyond infectious defense. It contributes to organ development, such as sculpting neuron synapses, promoting tissue regeneration and repair, and rapidly engaging and synergizing with a number of processes, including hemostasis leading to thromboinflammation. Complement is a double-edged sword. Although it usually protects the host, it may cause tissue damage when dysregulated or overactivated, such as in the systemic inflammatory reaction seen in trauma and sepsis and severe coronavirus disease 2019 (COVID-19). Damage-associated molecular patterns generated during ischemia-reperfusion injuries (myocardial infarction, stroke, and transplant dysfunction) and in chronic neurologic and rheumatic disease activate complement, thereby increasing damaging inflammation. Despite the long list of diseases with potential for ameliorating complement modulation, only a few rare diseases are approved for clinical treatment targeting complement. Those currently being efficiently treated include paroxysmal nocturnal hemoglobinuria, atypical hemolytic-uremic syndrome, myasthenia gravis, and neuromyelitis optica spectrum disorders. Rare diseases, unfortunately, preclude robust clinical trials. The increasing evidence for complement as a pathogenetic driver in many more common diseases suggests an opportunity for future complement therapy, which, however, requires robust clinical trials; one ongoing example is COVID-19 disease. The current review aims to discuss complement in disease pathogenesis and discuss future pharmacological strategies to treat these diseases with complement-targeted therapies. Significance Statement The complement system is the host’s defense friend by protecting it from invading pathogens, promoting tissue repair, and maintaining homeostasis. Complement is a double-edged sword, since when dysregulated or overactivated it becomes the host’s enemy, leading to tissue damage, organ failure, and, in worst case, death. A number of acute and chronic diseases are candidates for pharmacological treatment to avoid complement-dependent damage, ranging from the well established treatment for rare diseases to possible future treatment of large patient groups like the pandemic coronavirus disease 2019.

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Eculizumab in refractory catastrophic antiphospholipid syndrome: a case report and systematic review of the literature

Cited by 53 publications

References 32 publications

Thrombotic microangiopathy in the course of catastrophic antiphospholipid syndrome successfully treated with eculizumab: case report and systematic review of the literature

Thrombotic microangiopathy in the course of catastrophic antiphospholipid syndrome successfully treated with eculizumab: case report and systematic review of the literature

Therapeutic Challenges with Catastrophic Antiphospholipid Antibody Syndrome: A Case Report

Therapeutic Targeting of the Complement System: From Rare Diseases to Pandemics

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