Clinical and pharmacological group: & nbsp

Immunosuppressive drugs

Included in the formulation
  • Soliris®
    concentrate d / infusion 
  • Included in the list (Order of the Government of the Russian Federation No. 2782-r of 30.12.2014):

    VED

    АТХ:

    L.04.A.A.25   Ekulizumab

    Pharmacodynamics:

    Ekulizumab suppresses terminal activity of human complement, having a high affinity for its C5-component. As a consequence, the splitting of the C5 component into C5a and C5b is completely blocked and the terminal complex of the complement C5b-9 is formed. In this way, ekulizumab restores the regulation of complement activity in the blood and prevents intravascular hemolysis in patients with paroxysmal nocturnal hemoglobinuria. On the other hand, deficiency of terminal complement is accompanied by an increased incidence of infections with encapsulated microorganisms, mainly meningococcal infection. Wherein ekulizumab supports the content of early complement activation products necessary for opsonization of microorganisms and excretion of immune complexes.

    The appointment to patients is accompanied by a rapid and stable decrease in the terminal activity of the complement. In the majority of patients with paroxysmal nocturnal hemoglobinuria, the concentration of euculizumab in the blood plasma is of the order of 35 μg / ml sufficient for completeinhibition of intravascular hemolysis, induced by terminal activation of complement.

    Pharmacokinetics:

    Metabolism. Human antibodies under the action of lysosomal enzymes in the cells of the reticuloendothelial system are cleaved to small peptides and amino acids.

    Excretion. No special studies have been conducted to evaluate the ways of excretion of ekulizumab. Because of the large molecular weight (148 kD) ekulizumab Not excreted unchanged in the urine. The average clearance is 0.31 ± 0.12 ml / h / kg, the average volume of the distribution is 110.3 ± 17.9 ml / kg, and the mean half-life 11.3 ± 3.4 days. Based on these data, the equilibrium state is reached after 49-56 days.

    The pharmacodynamic activity of ekulizumab is directly proportional to its concentration in the plasma. While maintaining the concentration of ekulizumab in blood plasma ≥ 35 μg / ml, in most patients almost complete blockage of hemolytic activity was noted.

    Indications:

    Paroxysmal nocturnal hemoglobinuria. Efficacy was confirmed only in patients with paroxysmal nocturnal hemoglobinuria, who previously had blood transfusion or its components.

    Atypical hemolytic-uremic syndrome.

    III.D55-D59.D59.3   Hemolytic-uremic syndrome

    III.D55-D59.D59.5   Paroxysmal nocturnal hemoglobinuria [Marietaafy-Mikeli]

    Contraindications:
    • Hypersensitivity to ekulizumab, proteins of mouse origin or other components of the drug.
    • Active infection or bacteriocarrier Neisseria meningitides.
    • Lack of vaccination against Neisseria meningitides.
    • Fixed or suspected congenital deficiency of complement.
    Carefully:

    Active systemic infections, impaired liver and kidney function due to lack of clinical experience.

    Pregnancy and lactation:

    There were no controlled studies in pregnancy.

    It is known that human immunoglobulin G (IgG) passes through the placental barrier, in connection with which ekulizumab is potentially capable of inhibiting the terminal activity of complement in fetal blood. Do not use during pregnancy, except when the benefit to the mother exceeds the possible risk to the fetus.

    Women of reproductive age should use reliable methods of contraception during treatment and within 5 months after its completion.

    It is not established whether the ekulizumab in breast milk, but taking into account the potential undesirable effects of the drug, it is recommended to cancel breastfeeding during treatment with the drug and within 5 months after its completion.

    Dosing and Administration:

    Intravenously drip for 25-45 minutes.

    The course of treatment includes a 5-week initial cycle followed by a cycle of maintenance therapy.

    The initial cycle: 600 mg of the drug once a week for 4 weeks, at the 5th week - 900 mg once a week.

    Supporting phase: 900 mg of the drug is prescribed every 14 ± 2 days.

    For intravenous administration of a diluted solution of the drug, it is necessary to use special infusion systems with controlled delivery. It is not necessary to protect the reconstituted drug solution from light during administration. After the introduction of the drug, patient monitoring should continue for 1 hour. If adverse events develop during the administration of the drug, the infusion rate may be reduced until the injection is completely stopped at the physician's discretion. With a decrease in the rate of administration of the drug, the total infusion time should not exceed 2 hours.

    Side effects:

    Violations from side of the blood and lymphatic system: often thrombocytopenia; infrequently - coagulopathy, agglutination of erythrocytes.

    Benign, malignant and unspecified neoplasms: infrequently - myelodysplastic syndrome, melanoma.

    Infringements from heart: infrequent - a feeling of palpitations.

    Infringements from vessels: infrequently - a hematoma, a decrease in blood pressure, "tides" of blood.

    Infringements from the organ of hearing and labyrinthine disorders: infrequent - ringing in the ears, dizziness.

    Infringements from endocrine system: infrequently hyperthyroidism.

    Infringements from organ of vision: infrequently - irritation of the conjunctiva, vague vision.

    Infringements from GIT: often - abdominal pain, constipation, diarrhea, dyspepsia, nausea, vomiting; infrequently - bloating, gastroesophageal reflux, pain in the gums.

    Infringements from metabolism and nutrition: infrequent - loss of appetite, anorexia.

    Infringements from liver and bile ducts: infrequently, jaundice.

    Infringements from nervous system: very often - headache; often - peripheral dizziness, dysgesia, parasthesia; infrequently - a syncope.

    Infringements from respiratory system, chest and mediastinal organs: often - cough, swelling of the nasal mucosa, pain in the larynx and pharynx; infrequently - hemoptysis, rhinorrhea.

    Violations mentality: infrequent - unusual dreams, anxiety, depression, insomnia, mood swings, sleep disturbances.

    Infringements from kidney and urinary tract: often - dysuria; infrequently - a violation of kidney function.

    Infringements from skin and subcutaneous tissues: often - alopecia, dry skin, itching, rash; infrequently - a hyperhidrosis, petechia, a violation of skin pigmentation, a rash.

    Infringements from musculoskeletal and connective tissue: often - arthralgia, back pain, myalgia, neck pain, pain in the extremities; infrequently - pain in the bones, swelling of the joints, muscle spasms, trismus.

    Infectious and parasitic diseases: often - infections of the upper respiratory tract, gastrointestinal tract and urinary tract, including viral, herpes mucosa of the oral cavity, sepsis, including meningococcal, septic shock; infrequently - infections of the lower respiratory tract, sinusitis, tooth and gum disease infections, abscesses and inflammation of the subcutaneous tissue, fungal infections.

    Infringements from immune system: infrequently, allergic reactions.

    Infringements from genital organs and breast: often - a spontaneous erection; infrequently - violations of the menstrual cycle.

    General disorders and disorders at the site of administration: often - a feeling of discomfort in the chest, chills, weakness, nonspecific reactions at the injection site, swelling, fever; infrequently - pain in the chest, flu-like syndrome, paresthesia at the injection site.

    Laboratory and instrumental data: infrequently - increased activity of aspartate aminotransferase, increased activity of alanine aminotransferase, increased activity of gamma-glutamyltransferase.

    Overdose:

    Cases of an overdose of eculisumab are unknown.

    Interaction:

    Eculizumab may be mixed only with 0.9% sodium chloride solution, 0.45% sodium chloride solution or 5% dextrose (glucose) solution for injection.

    Special instructions:

    The drug should be administered under the supervision of a physician.

    Recommendations for solution preparation and infusion

    Do not mix the drug for intravenous administration with another drug in one syringe or vial!

    Using a sterile syringe with a needle, dial the entire contents of the vial / flasks with the drug and transfer the recommended dose into the vial with the infusion solution.

    Dilute the drug to a final concentration of 5 mg / ml with a 0.9% solution of sodium chloride, 0.45% solution of sodium chloride or 5% solution of dextrose (glucose) for injection.

    The final volume of the reconstituted drug to a final concentration of 5 mg / ml is 120 ml (for a dose of 600 mg) and 180 ml (for a dose of 900 mg).

    The finished solution should be clear and colorless. If the solution is colored or visible inclusions are detected, its use is not allowed.

    The finished solution should not be stored for more than 24 hours at a temperature of 2 to 8 ° C.

    Immediately before administration, the contents of the infusion bottle must be carefully shaken to ensure that the preparation and the solvent are mixed. The temperature of the solution during the injection should be 20-25 ° C.

    Do not inject intravenously!

    The unused drug remaining in the syringe should be immediately disposed of, since its composition does not include preservatives.

    Do not reuse syringe or needles.

    Any unused or consumable material must be disposed of in accordance with local requirements.

    Meningococcal infection: the mechanism of action of the drug involves an increased risk of developing meningococcal disease (Neisseria meningitidis) against the background of its application. Any serotypes, including atypical ones, for example Y, W135 and X can be considered as pathogenic. To reduce the probability of infection, all patients should be vaccinated against meningococcus 2 weeks prior to application of the drug.

    Patients with atypical hemolytic uremic syndrome who received treatment with the drug earlier than 2 weeks after vaccination against meningococcal infection should receive appropriate prophylactic antibiotic therapy within 2 weeks after vaccination. All patients should also be revaccinated according to the existing standards in the Russian Federation. Most preferred is a conjugated tetravalent vaccine against serotypes A, C, Y and W135. In some cases, vaccination does not have a sufficient protective effect.

    When choosing an antibacterial drug for the treatment of this complication, it is necessary to strictly follow the official recommendations.

    All patients should be informed of the early symptoms of meningococcal infection and the need to seek medical help immediately.

    Other systemic infections. The mechanism of action of the drug also suggests the possibility of activation of a latent infection, although clinical studies have not revealed differences in the incidence, severity or localization of infections in patients receiving and placebo. Nevertheless, patients should be warned about the possibility of activating the infection against the background of drug treatment and its possible symptoms.

    Infusion reactions. Intravenous administration of the drug, as well as administration of other protein preparations, may be accompanied by hypersensitivity reactions, including anaphylaxis. Despite the lack of clinical data on the development of such reactions in the treatment of the drug, in the case of a severe infusion reaction, the drug administration should be discontinued and symptomatic therapy should be prescribed.

    Immunogenicity. Low antibody titer was determined in patients both on the background of drug treatment (3.4%) and placebo (4.8%). Patients with atypical hemolytic-uremic syndrome who received ekulizumab, the appearance of antibodies to the drug in 3 cases out of 100 (3%) was recorded. In 1 case out of 100 (1%) patients with atypical hemolytic-uremic syndrome detected the appearance of neutralizing antibodies.No correlation was found between the antibody titer and the clinical efficacy of the drug or its side effect.

    Immunization. Before beginning therapy with the drug for all patients paroxysmal nocturnal hemoglobinuria and atypical hemolytic-uremic syndrome it is recommended to undergo a full vaccination in accordance with the National Calendar of Prophylactic Vaccination. In addition, at least 2 weeks before the start of treatment with the drug, all patients must necessarily be given a meningococcal vaccine, preferably tetravalent conjugate.

    Patients who received treatment with the drug earlier than 2 weeks after vaccination against meningococcal infection should receive appropriate prophylactic antibiotic therapy within 2 weeks after vaccination.

    Patients under the age of 18 should also be vaccinated against hemophilic rods and pneumococci in strict accordance with the national vaccination calendar.

    Anticoagulant therapy. Recommendations for anticoagulant therapy should not be changed in connection with the appointment of the drug.

    Laboratory control in treatment paroxysmal nocturnal hemoglobinuria. In patients paroxysmal nocturnal hemoglobinuria on the background of treatment with a drug to control the expression of intravascular hemolysis, it is necessary to determine the activity of LDH in the blood serum. If it is necessary to correct the dose during the period of maintenance therapy, the frequency of drug administration, determined by the frames (14 ± 2) days, can be increased up to 1 time every 12 days.

    Laboratory control in the treatment of patients with atypical hemolytic-uremic syndrome. Patients with atypical hemolytic uremic syndrome should be monitored for thrombotic microangiopathy by regular monitoring of platelet count, lactate dehydrogenase activity and serum creatinine. If it is necessary to correct the dose during the period of maintenance therapy, the frequency of drug administration, determined by the frames (14 ± 2) days, can be increased up to 1 time every 12 days.

    Termination of treatment of patients with paroxysmal nocturnal hemoglobinuria. Patients for whom drug therapy has been discontinued should be under medical supervision to ensure control of intravascular hemolysis.Signs of severe hemolysis are: LDH activity in the serum is higher than before the beginning of drug therapy, together with one of the following: a decrease of more than 25% in the APG cell population (in the absence of the effect of dilution in case of blood transfusion) for 1 week or earlier; the concentration of hemoglobin is less than 50 g / l or its decrease by more than 40 g / l for 1 week or earlier; the appearance of angina pectoris or an increase in its severity; mental disorders; an increase in the concentration of creatinine in the blood by 50% or thrombosis. The duration of observation of patients after discontinuation of the drug should be at least 8 weeks.

    In case of signs of severe hemolysis after discontinuation of treatment with the drug, it is recommended to appoint blood transfusion (erythrocyte mass) or exchange blood transfusion if, according to flow cytometry, APG cell population> 50% of the total number of erythrocytes; and also prescribe anticoagulants, corticosteroids or resume therapy with the drug. Observations of 16 patients with paroxysmal nocturnal hemoglobinuria, in which drug therapy was discontinued,they did not reveal an increase in intravascular hemolysis intensity.

    Termination of treatment of patients with atypical hemolytic-uremic syndrome. During clinical trials of the drug in patients with atypical hemolytic-uremic syndrome, severe complications of thrombotic microangiopathy after the withdrawal of therapy were observed.

    Patients with atypical hemolytic uremic syndrome who discontinued treatment drug must be under medical supervision to monitor for signs and symptoms of severe complications thrombotic microangiopathy.

    Signs of severe complications thrombotic microangiopathy after drug withdrawal are: (1) any two, or re-detect a change in one of the following: reduction in the number of platelets and 25% lower compared with the initial value or the maximum number of platelets during the drug treatment; an increase in the serum creatinine concentration by 25% or more, as compared to the baseline or minimum level during drug therapy; or an increase in serum LDH activity of 25% or more, as compared with the baseline value or the minimum value oftime of drug therapy; or (2) any of the following symptoms: a change in the psyche or seizures; angina or shortness of breath; thrombosis.

    The duration of follow-up of patients after discontinuation of the drug to detect severe complications of thrombotic microangiopathy should be at least 12 weeks.

    In case of severe complications of thrombotic microangiopathy after discontinuation of treatment with ekulizumab, it is recommended to resume drug therapy, prescribe maintenance treatment with plasmapheresis or exchange plasma transfusions or appropriate specific maintenance therapy, including hemodialysis, pulmonary ventilation or anticoagulant therapy. During clinical trials of the drug in 18 patients with atypical hemolytic-uremic syndrome, drug therapy was abolished (in 5 patients in prospective studies). After missing the next dose of the drug, five patients developed seven serious complications of thrombotic microangiopathy, and in 4 of these 5 patients the therapy was resumed.

    Teaching materials. All doctors who plan to prescribe ekulizumab, should read the "Guidelines for the doctor on the use of the drug." Patients should be instructed that if the body temperature rises above 39 ° C, the development of a headache in combination with fever and / or a feeling of stiff neck or photophobia, they should immediately seek medical help, as this may be signs of a meningococcal infection.

    Excipients. For patients on a strict salt-free diet, it should be borne in mind that each vial of the drug contains 5 mmol of sodium.

    Given the possibility of developing unwanted reactions against the background of drug treatment (for example, headache, dizziness, weakness), you need to take extra care when driving and working with mechanisms.

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