Management of Catastrophic Antiphosphopholipid Syndrome with Eculizumab

Blood ◽  
2016 ◽  
Vol 128 (22) ◽  
pp. 2603-2603 ◽  
Author(s):  
Ami Chitalia ◽  
David Swoboda ◽  
Catherine Broome
Keyword(s):  
Skin Biopsy ◽  
Unfractionated Heparin ◽  
Antiphospholipid Syndrome ◽  
Thrombotic Microangiopathy ◽  
Atypical Hemolytic Uremic Syndrome ◽  
Coagulation Cascade ◽  
Antiphospholipid Antibody ◽  
Clinical Recurrence ◽  
Elevated Liver Enzymes ◽  
Wide Range

Abstract Catastrophic antiphospholipid syndrome (CAPS) is a rare, often fatal phenomenon. Patients present with a wide range of symptomatology including thrombotic microangiopathy, cytopenias and end organ damage. The mortality rate of CAPS is as high as 33% in spite of the use of combination therapies including steroids, anticoagulation, plasma exchange (PEX) and intravenous immunoglobulin (Cervera CA 2009; Espinosa G 2011; Bucciarelli S 2006). CAPS is believed to be a disorder of complement-mediated inflammation which results in tissue injury. The proposed mechanism of the thrombotic microangiopathyisthe interaction of the coagulation cascade and complement (Mehdi AA 2010). Multiple murine-based studies demonstrate the contribution of C5a to antiphospholipid antibody-mediated intravascular thrombosis (Fischetti F 2005; Pierangeli SS 2005; Giannakopoulos B 2013). Eculizumab is a humanized monoclonal antibody that binds to C5 and inhibits its cleavage to C5a and C5b. It is FDA approved for the treatment of the complement mediated disorders paroxysmal nocturnal hemoglobinuria and atypical hemolytic uremic syndrome. We present 3 cases of CAPS treated with eculizumab at our institution. Their individual diagnostic criteria and background characteristics are outlined in Table 1. Case 1: 41-year-old male with history of antiphospholipid syndrome on chronic anticoagulation presented with fevers and ventilatory-dependent respiratory failure. Upon transfer to our institution, he developed thrombocytopenia and left hand mottling; doppler revealed a radial artery thrombus. Biopsy of the involved area of skin indicated thrombotic microangiopathy. Pulse dose steroids and unfractionated heparin were initiated. He was extubated within 24 hours and fever resolved. He remained dyspneic and mildly thrombocytopenic and therefore rituximab was initiated. Oxygen requirements, liver dysfunction, and thrombocytopenia resolved. As the prednisone dose was tapered, new microangiopathic lesions were noted. The decision was made to start eculizumab which is ongoing as a maintenance dose every two weeks with no clinical recurrence of CAPS. Case 2: 68-year-old male presented with priapism. On day 2 of hospitalization, he was found to have left lower extremity (LLE) DVTs and received unfractionated heparin. On day 4, he developed fever and thrombocytopenia and was noted to have a cold LLE. Angiography demonstrated occlusion of the posterior tibial artery and heparin was discontinued in favor of an alteplase drip. The patient was subsequently transferred to our institution. Doppler demonstrated multiple DVTs in both lower extremities. Evaluation for HIT was negative. Lupus anticoagulant (LA) was strongly positive raising concern for CAPS. Steroids and PEX were initiated. However, emergency fasciotomy of the RLE was needed to relieve compartment syndrome. Skin biopsy demonstrated microangiopathy. Due to lack of clinical improvement, PEX was discontinued and eculizumab was initiated. On day 3 post-eculizumab initiation, there was evidence of regression of skin mottling. Eculizumab was redosed on day 5. Steroids were tapered and he received 2 additional doses of eculizumab. No additional thrombotic complications occurred. He eventually died during this hospitalization due to septic shock. Case 3: 37-year-old female presented with fever, hypoxia, and painful feet. She was hypotensive and labs demonstrated leukopenia, thrombocytopenia, elevated liver enzymes, and acute kidney injury. Vasopressors and antibiotics were initiated. Over the next 24 hours she developed mottling of her feet, face and breasts. The diagnosis of CAPS was made based on clinical criteria and a positive LA. Unfractionated heparin, steroids and eculizumab were initiated. Dialysis was started due to renal failure. Skin biopsy showed microangiopathy. Marked improvement of skin mottling was noted after 3 doses of eculizumab and renal function normalized after 7 doses. She was discharged with a slow steroid taper, therapeutic enoxaparin, and maintenance eculizumab. Complement blockade with eculizumab is safe and effective in patients with CAPS. These cases suggest that utilizing therapy to inhibit the complement pathway may be an integral component in treating CAPS more effectively. Future directions should include a randomized clinical trial to evaluate eculizumab as part of combination therapy for CAPS. Disclosures Broome: True North Therapeutics: Honoraria; Alexion Pharmaceuricals: Honoraria.

scholarly journals Case of an unusual diagnosis of primary antiphospholipid syndrome with multiple clinical complications

2020 ◽  
Vol 2020 (12) ◽  
Author(s):  
Stathis Tsiakas ◽  
Chrysanthi Skalioti ◽  
Paraskevi Kotsi ◽  
Ioannis Boletis ◽  
Smaragdi Marinaki
Keyword(s):  
Antiphospholipid Syndrome ◽  
Thrombotic Event ◽  
Renal Involvement ◽  
Clinical Manifestations ◽  
Young Male ◽  
Multiple Organ ◽  
Systemic Autoimmune Disease ◽  
Antiphospholipid Antibody ◽  
Primary Antiphospholipid Syndrome ◽  
Wide Range

ABSTRACT Antiphospholipid syndrome (APS) is a systemic autoimmune disease defined by the presence of antiphospholipid antibodies in association with thrombotic events and/or obstetric complications. Renal involvement is not infrequent in both primary and secondary APS. Kidney manifestations comprise a wide range of clinical features, including hypertension, major renal vessel thrombosis or microvascular endothelial injury, also described as APS nephropathy. In the absence of a thrombotic event, clinical manifestations of APS are often non-specific. We recently encountered a case of primary APS in a young male with newly diagnosed hypertension and renal impairment. The diagnosis of APS was initially suspected by his kidney biopsy findings, when electron microscopy examination showed the features of chronic microangiopathy, and was later confirmed by a triple positive antiphospholipid antibody profile and multiple organ involvement.

scholarly journals Extracellular Vesicles Tune the Immune System in Renal Disease: A Focus on Systemic Lupus Erythematosus, Antiphospholipid Syndrome, Thrombotic Microangiopathy and ANCA-Vasculitis

2021 ◽  
Vol 22 (8) ◽  
pp. 4194
Author(s):  
Martina Mazzariol ◽  
Giovanni Camussi ◽  
Maria Felice Brizzi
Keyword(s):  
Systemic Lupus Erythematosus ◽  
Immune System ◽  
Autoimmune Diseases ◽  
Extracellular Vesicles ◽  
Antiphospholipid Syndrome ◽  
Lupus Erythematosus ◽  
Thrombotic Microangiopathy ◽  
Coagulation Cascade ◽  
Renal Diseases ◽  
Systemic Lupus

Extracellular vesicles (EV) are microparticles released in biological fluids by different cell types, both in physiological and pathological conditions. Owing to their ability to carry and transfer biomolecules, EV are mediators of cell-to-cell communication and are involved in the pathogenesis of several diseases. The ability of EV to modulate the immune system, the coagulation cascade, the angiogenetic process, and to drive endothelial dysfunction plays a crucial role in the pathophysiology of both autoimmune and renal diseases. Recent studies have demonstrated the involvement of EV in the control of renal homeostasis by acting as intercellular signaling molecules, mediators of inflammation and tissue regeneration. Moreover, circulating EV and urinary EV secreted by renal cells have been investigated as potential early biomarkers of renal injury. In the present review, we discuss the recent findings on the involvement of EV in autoimmunity and in renal intercellular communication. We focused on EV-mediated interaction between the immune system and the kidney in autoimmune diseases displaying common renal damage, such as antiphospholipid syndrome, systemic lupus erythematosus, thrombotic microangiopathy, and vasculitis. Although further studies are needed to extend our knowledge on EV in renal pathology, a deeper investigation of the impact of EV in kidney autoimmune diseases may also provide insight into renal biological processes. Furthermore, EV may represent promising biomarkers of renal diseases with potential future applications as diagnostic and therapeutic tools.

scholarly journals Severe Vitamin B12 Deficiency in Pregnancy Mimicking HELLP Syndrome

2019 ◽  
Vol 2019 ◽  
pp. 1-4
Author(s):  
Shravya Govindappagari ◽  
Michelle Nguyen ◽  
Megha Gupta ◽  
Ramy M. Hanna ◽  
Richard M. Burwick
Keyword(s):  
Vitamin B12 ◽  
Pernicious Anemia ◽  
Thrombotic Microangiopathy ◽  
Hellp Syndrome ◽  
Atypical Hemolytic Uremic Syndrome ◽  
Vitamin B12 Deficiency ◽  
Severe Vitamin ◽  
Elevated Liver Enzymes ◽  
B12 Deficiency ◽  
In Pregnancy

Severe vitamin B12 deficiency may present with hematologic abnormalities that mimic thrombotic microangiopathy disorders such as hemolysis, elevated liver enzymes, and low platelet count (HELLP) syndrome. We report a patient diagnosed with severe vitamin B12 deficiency, following termination of pregnancy for suspected preeclampsia and HELLP syndrome at 21 weeks’ gestation. When hemolysis and thrombocytopenia persisted after delivery, testing was performed to rule out other etiologies of thrombotic microangiopathy, including atypical hemolytic uremic syndrome, thrombotic thrombocytopenic purpura, and vitamin B12 deficiency. This work-up revealed undetectable vitamin B12 levels and presence of intrinsic factor antibodies, consistent with pernicious anemia. Parenteral B12 supplementation was initiated, with subsequent improvement in hematologic parameters. Our case emphasizes the importance of screening for B12 deficiency in pregnancy, especially in at-risk women with unexplained anemia or thrombocytopenia. Moreover, providers should consider B12 deficiency and pernicious anemia in the differential diagnosis of pregnancy-associated thrombotic microangiopathy.

Thrombotic Microangiopathy Due to Catastrophic Antiphospholipid Antibody Syndrome Confirmed on Skin Biopsy and Treated with Eculizumab

2017 ◽  
Vol 1 (2) ◽  
pp. e1-e7 ◽  
Author(s):  
Ramy M. Hanna ◽  
Mina Hanna ◽  
Brent Larson ◽  
Eduardo A. Lopez ◽  
James Wilson ◽  
...  
Keyword(s):  
Skin Biopsy ◽  
Thrombotic Microangiopathy ◽  
Antiphospholipid Antibody ◽  
Antiphospholipid Antibody Syndrome

scholarly journals A Challenging Case of Atypical Hemolytic Uremic Syndrome in a Young African American Patient

Blood ◽  
2021 ◽  
Vol 138 (Supplement 1) ◽  
pp. 4225-4225
Author(s):  
Urwat Til Vusqa ◽  
Palash Asawa ◽  
Yazan Samhouri ◽  
Rama Bhagavatula ◽  
Robert B. Kaplan
Keyword(s):  
Blood Pressure ◽  
African American ◽  
Hemolytic Uremic Syndrome ◽  
Thrombotic Microangiopathy ◽  
Atypical Hemolytic Uremic Syndrome ◽  
Malignant Hypertension ◽  
Coagulation Cascade ◽  
Complement Factors ◽  
Uremic Syndrome ◽  
Terminal Complement

Abstract Background It is known that malignant hypertension (mHTN) and thrombotic microangiopathy (TMA) commonly coexist. Deciding which phenomenon preceded the other remains a clinical dilemma, specifically in African American patients. However, making that determination is of utmost importance because the management will be different, and that can have dramatic effects on prognosis and outcomes. Herein, we report a case of atypical hemolytic uremic syndrome (aHUS) presenting as mHTN. Case Presentation A 35-year-old African American male with known history of hypertension, presented with nausea, vomiting, and diarrhea for four days. He also reported fatigue and exertional shortness of breath. Upon presentation, his blood pressure was 260/160 mmHg, otherwise physical exam was unremarkable. Initial work up showed hemoglobin of 8.8 g/dL (baseline 13.5), platelet count of 21,000/mL (baseline 250,000), serum creatinine of 16.99 mg/dL (baseline 0.99), MCV (84 fl), increased reticulocyte production index (3.58), increased LDH (1709 U/L), undetectable haptoglobin, and numerous schistocytes on peripheral blood smear. He was admitted as a case of hypertensive emergency and TMA. IV labetalol and hemodialysis were started. Given his gastrointestinal symptoms; stool for Shigella and E.Coli O157:H7 were checked and they were negative. Given the severity of his hematologic derangements and difficult to control blood pressure, we decided to proceed with renal biopsy to rule out primary aHUS which showed thrombotic microangiopathy, global glomerulosclerosis, moderate interstitial fibrosis and tubular atrophy suggestive of aHUS or rheumatologic disorders like systemic sclerosis and arguing against malignant HTN as the sole player. ANA and anti-Scl-70 antibodies were negative. Final impression was aHUS by exclusion, and patient received meningococcal vaccines (Menactra and Bexsero) in preparation to start eculizumab. aHUS genetic panel was sent which came back equivocal as it showed mutations of unknown significance (homozygous missense mutation in the MASP2 gene and 2 heterozygous mutations in the C2 gene). He was started on eculizumab 900 mg weekly for 4 weeks then 1200 mg biweekly starting week 5. He was seen in the office 2 months after initial presentation and receiving 5 doses of Eculizumab. His kidney function showed improvement with > 2 liters of urine output daily, blood pressure was better controlled. A decision by nephrology was made to give him a break from dialysis and remains dialysis-free a year later. Discussion aHUS is a rare disorder with an estimated prevalence of seven per one million children in Europe. It causes uninhibited activation of complement factors that leads to renal endothelial damage and activation of coagulation cascade leading to TMA. The diagnosis of aHUS requires the fulfillment of the classical triad (microangiopathic hemolytic anemia, thrombocytopenia, and acute renal failure) with a positive gene mutation or antibodies to complement factors. However, absence of these mutations or antibodies, as in the presented case, do not exclude the diagnosis. The early diagnosis of aHUS is necessary for treatment with eculizumab, a monoclonal antibody against C5 to block the terminal complement cascade. Kidney biopsy can be helpful in equivocal cases especially if it shows only the typical changes of malignant hypertension which essentially rules out aHUS. Hypertension with concurrent TMA is treated with strict BP control which is often enough to resolve TMA features and restores renal function, at least partially. On the contrary, aHUS causing severe HTN needs more sophisticated testing and blockade of the terminal complement component to improve outcome; that's why the distinction of which one is the primary process is of utmost importance. Our case emphasizes the importance of having low threshold for testing for aHUS in patients with mHTN and TMA, especially in African American patients where malignant HTN is known to happen more commonly, and to notice the subtle hints that may help in this distinction, such as profound hemolysis or thrombocytopenia out of proportion to what one would expect from mHTN alone. Early recognition of aHUS may save a patient's kidney. Disclosures No relevant conflicts of interest to declare.

scholarly journals Atypical Hemolytic Uremic Syndrome Presenting as Acute Heart Failure—A Rare Presentation: Diagnosis Supported by Skin Biopsy

2019 ◽  
Vol 7 ◽  
pp. 232470961984290
Author(s):  
Asim Kichloo ◽  
Savneek Singh Chugh ◽  
Sanjeev Gupta ◽  
Jay Pandav ◽  
Praveen Chander
Keyword(s):  
Heart Failure ◽  
Skin Biopsy ◽  
Hemolytic Uremic Syndrome ◽  
Thrombotic Microangiopathy ◽  
Atypical Hemolytic Uremic Syndrome ◽  
Systolic Heart Failure ◽  
Kidney Injury ◽  
Rare Presentation ◽  
History Of ◽  
Uremic Syndrome

Atypical hemolytic uremic syndrome (aHUS) is a rare disorder of uncontrolled complement activation that manifests classically as anemia, thrombocytopenia, and renal failure, although extrarenal manifestations are observed in 20% of the patient most of which involving central nervous system, with relatively rare involvement of the heart. In this article, we report the case of a 24-year-old male with no history of heart disease presenting with acute systolic heart failure along with microangiopathic hemolytic anemia, thrombocytopenia, and acute kidney injury. Given his presentation of thrombotic microangiopathy (TMA), along with laboratory results significant for low haptoglobin, platelets, hemoglobin, C3, C4, CH50, and normal ADAMTS13 levels, with no diarrhea and negative STEC polymerase chain reaction in stool, aHUS diagnosis was established with strong clinical suspicion, and immediate initiation of treatment was advised. Kidney biopsy to confirm diagnosis of aHUS was inadvisable because of thrombocytopenia, so the skin biopsy of a rash on his arm was done, which came to be consistent with thrombotic microangiopathy. Our case highlights a relatively rare association between aHUS and cardiac involvement, and the use of skin biopsy to support diagnosis of aHUS in patients who cannot undergo renal biopsy because of thrombocytopenia.

scholarly journals Management of thrombotic microangiopathy in pregnancy and postpartum: report from an international working group

Blood ◽  
2020 ◽  
Vol 136 (19) ◽  
pp. 2103-2117
Author(s):  
Fadi Fakhouri ◽  
Marie Scully ◽  
François Provôt ◽  
Miquel Blasco ◽  
Paul Coppo ◽  
...  
Keyword(s):  
Thrombotic Microangiopathy ◽  
Working Group ◽  
Current Knowledge ◽  
Atypical Hemolytic Uremic Syndrome ◽  
Specific Treatment ◽  
Clinical Approach ◽  
Elevated Liver Enzymes ◽  
Pregnancy And Postpartum ◽  
Multidisciplinary Working ◽  
In Pregnancy

Abstract Pregnancy and postpartum are high-risk periods for different forms of thrombotic microangiopathy (TMA). However, the management of pregnancy-associated TMA remains ill defined. This report, by an international multidisciplinary working group of obstetricians, nephrologists, hematologists, intensivists, neonatologists, and complement biologists, summarizes the current knowledge of these potentially severe disorders and proposes a practical clinical approach to diagnose and manage an episode of pregnancy-associated TMA. This approach takes into account the timing of TMA in pregnancy or postpartum, coexisting symptoms, first-line laboratory workup, and probability-based assessment of possible causes of pregnancy-associated TMA. Its aims are: to rule thrombotic thrombocytopenic purpura (TTP) in or out, with urgency, using ADAMTS13 activity testing; to consider alternative disorders with features of TMA (preeclampsia/eclampsia; hemolysis elevated liver enzymes low platelets syndrome; antiphospholipid syndrome); or, ultimately, to diagnose complement-mediated atypical hemolytic uremic syndrome (aHUS; a diagnosis of exclusion). Although they are rare, diagnosing TTP and aHUS associated with pregnancy, and postpartum, is paramount as both require urgent specific treatment.

scholarly journals Thrombotic Microangiopathy After Kidney Transplantation: An Underdiagnosed and Potentially Reversible Entity

2021 ◽  
Vol 8 ◽  
Author(s):  
Ana Ávila ◽  
Eva Gavela ◽  
Asunción Sancho
Keyword(s):  
Renal Failure ◽  
Thrombotic Microangiopathy ◽  
De Novo ◽  
Organ Procurement ◽  
Ischemia Reperfusion ◽  
Atypical Hemolytic Uremic Syndrome ◽  
Antiphospholipid Antibody ◽  
Antiphospholipid Antibody Syndrome ◽  
Post Transplant ◽  
Antibody Mediated Rejection

Thrombotic microangiopathy is a rare but serious complication that affects kidney transplant recipients. It appears in 0.8–14% of transplanted patients and negatively affects graft and patient survival. It can appear in a systemic form, with hemolytic microangiopathic anemia, thrombocytopenia, and renal failure, or in a localized form, with progressive renal failure, proteinuria, or arterial hypertension. Post-transplant thrombotic microangiopathy is classified as recurrent atypical hemolytic uremic syndrome or de novo thrombotic microangiopathy. De novo thrombotic microangiopathy accounts for the majority of cases. Distinguishing between the 2 conditions can be difficult, given there is an overlap between them. Complement overactivation is the cornerstone of all post-transplant thrombotic microangiopathies, and has been demonstrated in the context of organ procurement, ischemia-reperfusion phenomena, immunosuppressive drugs, antibody-mediated rejection, viral infections, and post-transplant relapse of antiphospholipid antibody syndrome. Although treatment of the causative agents is usually the first line of treatment, this approach might not be sufficient. Plasma exchange typically resolves hematologic abnormalities but does not improve renal function. Complement blockade with eculizumab has been shown to be an effective therapy in post-transplant thrombotic microangiopathy, but it is necessary to define which patients can benefit from this therapy and when and how eculizumab should be used.

scholarly journals A retrospective review of antiphospholipid syndrome from a South Asian country

2021 ◽  
Author(s):  
Muhammad Zain Mushtaq ◽  
Syed Ahsan Ali ◽  
Zaibunnisa Sattar ◽  
Saad Bin Zafar Mahmood ◽  
Tazein Amber ◽  
...  
Keyword(s):  
Venous Thrombosis ◽  
South Asian ◽  
Antiphospholipid Syndrome ◽  
Tertiary Care ◽  
Asian Country ◽  
Immunoglobulin M ◽  
Antiphospholipid Antibody ◽  
Care Hospital ◽  
South Asian Country ◽  
Wide Range

Objectives: This study aims to investigate clinical presentations, antiphospholipid antibody patterns and their levels, therapeutic regimens, and outcomes in patients with antiphospholipid syndrome (APS) admitted to a tertiary care hospital of a South Asian country. Patients and methods: Between January 2009 and December 2019, a total of 216 patients with APS (8 males, 208 females; median age: 35.7±6.9 years; range, 20 to 76 years) who either fulfilled the modified Sydney criteria or those who satisfied only clinical criteria along with positive antiphospholipid antibody on at least one occasion (probable APS) were retrospectively analyzed. Results: The majority of the patients (n=183, 84.7%) had obstetric complications, followed by venous thrombosis in 23 (10.8%) patients. Recurrent early abortions in 126 (58.6%) and deep venous thrombosis in 16 (7.4%) patients were the most prevalent obstetrical and venous events, respectively, whereas limb gangrene in seven (3.3%) and ischemic stroke in seven (3.3%) were the most common arterial events. A total of 190 (88%) patients had primary APS, while 26 (12%) had secondary APS. Systemic lupus erythematosus was the frequent association with secondary APS found in 19 (73%) patients. Immunoglobulin M (IgM) anticardiolipin antibody was present in 173 (65.0%) patients, being the most commonly reported antibody. Probable catastrophic APS was found in four (1.9%) patients. Majority of the patients (n=190, 87.9%) were treated with a combination of acetylsalicylic acid and low-molecular-weight heparin. Single mortality was observed in our study population due to complications related to catastrophic APS. Conclusion: Antiphospholipid syndrome has a wide range of thrombotic and obstetrical manifestations with important variations in different regions of the world. There is a significant morbidity and mortality related to APS, despite treatment with anticoagulation and; therefore, describing prognostic markers and optimal therapeutic interventions is pivotal to prevent complications.

scholarly journals Thrombotic microangiopathy in rheumatology: a link between thrombosis and autoimmunity

2020 ◽  
Vol 92 (5) ◽  
pp. 4-14
Author(s):  
Evgeny L. Nasonov ◽  
Tatyana M. Reshetnyak ◽  
Zemfira S. Alekberova
Keyword(s):  
Systemic Lupus Erythematosus ◽  
Antiphospholipid Syndrome ◽  
Rheumatic Diseases ◽  
Lupus Erythematosus ◽  
Thrombotic Microangiopathy ◽  
Human Diseases ◽  
Inflammatory Rheumatic Diseases ◽  
Systemic Lupus ◽  
Close Link ◽  
Wide Range

Uncontrolled hypercoagulation and inflammation (thromboinflammation), which are both independent and closely related and amplifying each other pathological processes, form the basis for pathogenesis of a wide range of diseases and complications, including immuno-inflammatory (autoimmune) rheumatic diseases, with the development of potentially fatal injuries of internal organs. Thrombotic microangiopathy is one of the most prominent prototypes of thromboinflammatory pathological conditions. The close link between environmental factors, hemostasis genetic defects and the complement system, inflammation and autoimmunity as pathogenetic mechanisms of microthrombosis draws particular attention to studying thrombotic microangiopathy in immuno-inflammatory rheumatic diseases, primarily systemic lupus erythematosus, antiphospholipid syndrome and scleroderma renal crisis. In future, these studies may be important for expanding the idea of the role of autoimmune mechanisms in pathogenesis of critical hemostasis disorders in human diseases, and for developing new approaches to therapy. Recently, special attention has been paid to the treatment of systemic lupus erythematosus and antiphospholipid syndrome with eculizumab, which is humanized monoclonal IgG2/4k antibody that blocks the complement component C5a and the membrane attack complex (C5b-9) formation, and which is registered for the treatment of atypical hemolytic uremic syndrome, paroxysmal nocturnal hemoglobinuria, as well as severe forms of myasthenia gravis and neuromyelitis optica. Further studies in this direction will create prerequisites for improving the prognosis not only in patients with orphan disorders, but also for widespread human diseases.

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