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Eculizumab


General


Pronunciation

(e kue LIZ oo mab)


Brand Names: U.S.

  • Soliris

Indications


Use: Labeled Indications

Atypical hemolytic uremic syndrome: Treatment of atypical hemolytic uremic syndrome (aHUS) to inhibit complement-mediated thrombotic microangiopathy.

Limitation of use: Eculizumab is not indicated for the treatment of patients with Shiga toxin Escherichia coli-related hemolytic uremic syndrome.

Paroxysmal nocturnal hemoglobinuria: Treatment of paroxysmal nocturnal hemoglobinuria (PNH) to reduce hemolysis.


Contraindications


Unresolved serious Neisseria meningitidis infection; patients not currently vaccinated against Neisseria meningitidis (unless risks of treatment delay outweigh risk of developing a meningococcal infection)


ALERT: U.S. Boxed Warning

Serious meningococcal infection:

Life-threatening and fatal meningococcal infections have occurred in patients treated with eculizumab. Meningococcal infection may become rapidly life-threatening or fatal if not recognized and treated early.

Comply with the most current Advisory Committee on Immunization Practices (ACIP) recommendations for meningococcal vaccination in patients with complement deficiencies.

Immunize patients with a meningococcal vaccine at least 2 weeks prior to administering the first dose of eculizumab, unless the risks of delaying eculizumab therapy outweigh the risk of developing a meningococcal infection.

Monitor patients for early signs of meningococcal infections and evaluate immediately if infection is suspected.

Eculizumab is available only through a restricted program under a Risk Evaluation and Mitigation Strategy (REMS). Under the Soliris REMS, health care providers must enroll in the program. Enrollment in the Soliris REMS program and additional information are available from the manufacturer (1-888-765-4747) or solirisrems.com.


Dosing and Administration


Dosing: Adult

Note: Patients must receive meningococcal vaccine at least 2 weeks prior to treatment initiation; revaccinate according to current guidelines. Treatment should be administered at the recommended time interval although administration may be varied by ‚ ±2 days.

Atypical hemolytic uremic syndrome (aHUS): IV: Induction: 900 mg weekly for 4 doses; Maintenance: 1,200 mg at week 5, then 1,200 mg every 2 weeks thereafter

Supplemental dosing for patients receiving plasmapheresis or plasma exchange: If most recent dose was ≥600 mg, administer 600 mg within 60 minutes after each plasmapheresis or plasma exchange

Supplemental dosing for patients receiving fresh frozen plasma infusion: If most recent dose was ≥300 mg, administer 300 mg within 60 minutes prior to each infusion of fresh frozen plasma

Paroxysmal nocturnal hemoglobinuria (PNH): IV: 600 mg weekly for 4 doses, followed by 900 mg 1 week later; then 900 mg every 2 weeks thereafter


Dosing: Geriatric

Refer to adult dosing.


Dosing: Pediatric

Note: Patients must receive meningococcal vaccine at least 2 weeks prior to treatment initiation; revaccinate according to current guidelines. Treatment should be administered at the recommended time interval although administration may be varied by ‚ ±2 days.

Atypical hemolytic uremic syndrome (aHUS): IV:

Infants ≥2 months, Children, and Adolescents:

5 kg to <10 kg: Induction: 300 mg weekly for 1 dose; Maintenance: 300 mg at week 2, then 300 mg every 3 weeks

10 kg to <20 kg: Induction: 600 mg weekly for 1 dose; Maintenance: 300 mg at week 2, then 300 mg every 2 weeks

20 kg to <30 kg: Induction: 600 mg weekly for 2 doses; Maintenance: 600 mg at week 3, then 600 mg every 2 weeks

30 kg to <40 kg: Induction: 600 mg weekly for 2 doses; Maintenance: 900 mg at week 3, then 900 mg every 2 weeks

≥40 kg: Induction: 900 mg weekly for 4 doses; Maintenance: 1200 mg at week 5, then 1200 mg every 2 weeks

Supplemental dosing for patients receiving plasmapheresis or plasma exchange:

If most recent dose was 300 mg, administer 300 mg within 60 minutes after each plasmapheresis or plasma exchange

If most recent dose was ≥600 mg, administer 600 mg within 60 minutes after each plasmapheresis or plasma exchange

Supplemental dosing for patients receiving fresh frozen plasma infusion: If most recent dose was ≥300 mg, administer 300 mg within 60 minutes prior to each infusion of fresh frozen plasma


Dosing: Renal Impairment

There are no dosage adjustments provided in the manufacturer 's labeling (has not been studied).


Dosing: Hepatic Impairment

There are no dosage adjustments provided in the manufacturer 's labeling (has not been studied).


Reconstitution

Add eculizumab to an empty infusion bag and dilute with an equal volume of D5W, sodium chloride 0.9%, sodium chloride 0.45%, or Ringer 's injection to a final concentration of 5 mg/mL (eg, 300 mg to a total volume of 60 mL, 600 mg in a total volume of 120 mL, 900 mg in a total volume of 180 mL, or 1,200 mg to a total volume of 240 mL). Gently invert bag to mix thoroughly; do not shake.


Administration

IV: Allow to reach room temperature prior to administration. Infuse over 35 minutes in adults and over 1 to 4 hours in pediatric patients; do not administer as an IV push or bolus. Decrease infusion rate or discontinue for infusion reactions; do not exceed a maximum 2-hour duration of infusion in adults. Monitor for at least 1 hour following completion of infusion (for signs/symptoms of infusion reaction).


Storage

Prior to dilution, store intact vials at 2 ‚ °C to 8 ‚ °C (36 ‚ °F to 46 ‚ °F); do not freeze. Vials may be held in the original carton at not more than 25 ‚ °C (77 ‚ °F) for only a single period up to 3 days. Protect from light; do not shake. Following dilution, store at room temperature or refrigerate; use within 24 hours. If refrigerated, allow admixture to reach room temperature prior to administration (do not use a heat source for warming).


Dosage Forms/Strengths


Dosage Forms

Excipient information presented when available (limited, particularly for generics); consult specific product labeling.

Solution, Intravenous [preservative free]:

Soliris: 10 mg/mL (30 mL)


Drug Interactions

BCG (Intravesical): Immunosuppressants may diminish the therapeutic effect of BCG (Intravesical). Avoid combination

Belimumab: Monoclonal Antibodies may enhance the adverse/toxic effect of Belimumab. Avoid combination

Coccidioides immitis Skin Test: Immunosuppressants may diminish the diagnostic effect of Coccidioides immitis Skin Test. Monitor therapy

Denosumab: May enhance the adverse/toxic effect of Immunosuppressants. Specifically, the risk for serious infections may be increased. Monitor therapy

Echinacea: May diminish the therapeutic effect of Immunosuppressants. Consider therapy modification

Fingolimod: Immunosuppressants may enhance the immunosuppressive effect of Fingolimod. Management: Avoid the concomitant use of fingolimod and other immunosuppressants when possible. If combined, monitor patients closely for additive immunosuppressant effects (eg, infections). Consider therapy modification

Leflunomide: Immunosuppressants may enhance the adverse/toxic effect of Leflunomide. Specifically, the risk for hematologic toxicity such as pancytopenia, agranulocytosis, and/or thrombocytopenia may be increased. Management: Consider not using a leflunomide loading dose in patients receiving other immunosuppressants. Patients receiving both leflunomide and another immunosuppressant should be monitored for bone marrow suppression at least monthly. Consider therapy modification

Natalizumab: Immunosuppressants may enhance the adverse/toxic effect of Natalizumab. Specifically, the risk of concurrent infection may be increased. Avoid combination

Nivolumab: Immunosuppressants may diminish the therapeutic effect of Nivolumab. Consider therapy modification

Pimecrolimus: May enhance the adverse/toxic effect of Immunosuppressants. Avoid combination

Roflumilast: May enhance the immunosuppressive effect of Immunosuppressants. Consider therapy modification

Sipuleucel-T: Immunosuppressants may diminish the therapeutic effect of Sipuleucel-T. Monitor therapy

Tacrolimus (Topical): May enhance the adverse/toxic effect of Immunosuppressants. Avoid combination

Tofacitinib: Immunosuppressants may enhance the immunosuppressive effect of Tofacitinib. Management: Concurrent use with antirheumatic doses of methotrexate or nonbiologic disease modifying antirheumatic drugs (DMARDs) is permitted, and this warning seems particularly focused on more potent immunosuppressants. Avoid combination

Trastuzumab: May enhance the neutropenic effect of Immunosuppressants. Monitor therapy

Vaccines (Inactivated): Immunosuppressants may diminish the therapeutic effect of Vaccines (Inactivated). Management: Vaccine efficacy may be reduced. Complete all age-appropriate vaccinations at least 2 weeks prior to starting an immunosuppressant. If vaccinated during immunosuppressant therapy, revaccinate at least 3 months after immunosuppressant discontinuation. Consider therapy modification

Vaccines (Live): Immunosuppressants may enhance the adverse/toxic effect of Vaccines (Live). Immunosuppressants may diminish the therapeutic effect of Vaccines (Live). Management: Avoid use of live organism vaccines with immunosuppressants; live-attenuated vaccines should not be given for at least 3 months after immunosuppressants. Avoid combination


Monitoring Parameters

CBC with differential, lactic dehydrogenase (LDH), serum creatinine, AST, urinalysis; early signs/symptoms of meningococcal infection; signs and symptoms of infusion reaction (during infusion and for 1 hour after infusion complete).

After discontinuation:

aHUS: Signs/symptoms of thrombotic microangiopathy (TMA) complications (monitor for at least 12 weeks after treatment discontinuation), including angina, dyspnea, mental status changes, seizure, or thrombosis; occurrence of two or repeated measurement of any one of the following: Serum creatinine elevation ( ≥25% from baseline or nadir), serum LDH elevation ( ≥25% from baseline or nadir), thrombocytopenia (platelet decrease by ≥25% compared to baseline or peak).

PNH: Signs and symptoms of intravascular hemolysis (monitor for at least 8 weeks after discontinuation), including anemia, fatigue, pain, dark urine, dyspnea, or thrombosis.


Adverse Reactions


Frequency reported for adolescent and adult patients ≥13 years unless otherwise noted.

>10%:

Cardiovascular: Hypertension (aHUS: 17% to 59%; infants, children, and adolescents 5 months through 17 years: 18%), peripheral edema (20% to 29%), tachycardia (aHUS: children 21%), hypotension (12% to 20%)

Central nervous system: Headache (37% to 50%; serious: 2%; infants, children, and adolescents 5 months through 17 years: 18%), insomnia (10% to 24%), fatigue (7% to 20%)

Dermatologic: Skin rash (infants ≥5 months, children, adolescents, and adults 12% to 18%), pruritus (6% to 15%)

Endocrine & metabolic: Hypokalemia (10% to 18%)

Gastrointestinal: Diarrhea (32% to 47%; infants, children, and adolescents 2 months through 17 years: 32%), vomiting (15% to 47%; infants, children, and adolescents 2 months through 17 years: 21% to 27%), nausea (12% to 40%), abdominal pain (15% to 30%), gastroenteritis (5% to 18%), dyspepsia (infants, children, and adolescents 5 months through 17 years: 14%)

Genitourinary: Urinary tract infection (15% to 35%; infants, children, and adolescents 5 months through 17 years: 18%), uropathy (infants, children, and adolescents 5 months through 17 years: 18%), proteinuria (5% to 12%)

Hematologic & oncologic: Anemia (17% to 35%; serious: 2%), neoplasm (6% to 30%), leukopenia (16% to 24%)

Local: Catheter infection (infants, children, and adolescents 5 months through 17 years: 14%)

Neuromuscular & skeletal: Weakness (15% to 20%), back pain (5% to 19%), arthralgia (6% to 17%), muscle spasm (infants, children, and adolescents 5 months through 17 years: 14%), limb pain (7% to 11%)

Ophthalmic: Eye disease (10% to 29%; infants, children, and adolescents 5 months through 17 years: 14%)

Renal: Renal insufficiency (15% to 29%)

Respiratory: Nasopharyngitis (18% to 55%; infants, children, and adolescents 5 months through 17 years: 27%), upper respiratory tract infection (infants ≥2 months, children, adolescents, and adults 5% to 40%), cough (infants ≥5 months, children, adolescents, and adults 12% to 36%), nasal congestion (aHUS: children 21%), rhinitis (infants, children, and adolescents 5 months through 17 years: 18%), bronchitis (10% to 18%), oropharyngeal pain (infants, children, and adolescents 5 months through 17 years: 14%)

Miscellaneous: Fever (infants, children, and adolescents 2 months through 17 years: 47% to 50%; adults 17% to 25%)

1% to 10%:

Gastrointestinal: Constipation (7%)

Immunologic: Antibody development (2% to 3%; neutralizing: 1%)

Infection: Herpes virus infection (7%), viral infection (serious: 2%), meningococcal infection ( ≤1%)

Neuromuscular & skeletal: Myalgia (7%)

Respiratory: Respiratory tract infection (7%), sinusitis (7%), flu-like symptoms (5%)

<1% (Limited to important or life-threatening): Aspergillosis, cholangitis, endometritis, hematoma (mild), infusion related reaction, pyelonephritis


Warnings/Precautions


Concerns related to adverse effects:

- Infections: In addition to meningitis, the risk of other infections, especially encapsulated bacteria (eg, Streptococcus pneumoniae, H. influenzae) is increased with eculizumab treatment (because eculizumab blocks terminal complement activation). Aspergillus infections have occurred in immunocompromised and neutropenic patients. Children should receive vaccination for prevention of S. pneumoniae, H. influenzae according to current ACIP guidelines. Use with caution when administering to a patient with a systemic infection.

- Infusion reactions: Infusion reactions, including anaphylaxis or hypersensitivity, may occur; interrupt infusion for severe reaction (eg, cardiovascular instability, respiratory compromise). Continue monitoring for 1 hour after completion of infusion.

- Meningococcal infection: [US Boxed Warning]: Meningococcal (Neisseria meningitides) infections have occurred in patients receiving eculizumab; may be fatal or life-threatening if not detected and treated promptly. Monitor closely for early signs of meningococcal infection; evaluate and treat promptly if suspected. Follow current meningococcal immunization recommendations for patients with complement deficiencies. Vaccinate with meningococcal vaccine at least 2 weeks prior to initiation of treatment (unless the risks of delaying eculizumab outweigh the risk of developing meningococcal infection); revaccinate according to current guidelines. Polyvalent meningococcal vaccines are recommended. If urgent treatment is necessary in an unvaccinated patient, administer meningococcal vaccine as soon as possible. Although the risk/benefits of prophylactic meningococcal antibiotic therapy have not been determined, prophylactic antibiotics were administered in clinical studies until at least 2 weeks after vaccination. Meningococcal infections developed in some patients despite vaccination. Discontinue eculizumab during the treatment of serious meningococcal infections.

Concurrent drug therapy issues:

- Anticoagulation: In clinical trials, anticoagulant therapy was continued in patients who were receiving these agents (due to history of or risk for thromboembolism) prior to initiation of eculizumab. The effect of anticoagulant therapy withdrawal is unknown. Treatment with eculizumab should not alter anticoagulation management.

- Drug-drug interactions: Potentially significant interactions may exist, requiring dose or frequency adjustment, additional monitoring, and/or selection of alternative therapy. Consult drug interactions database for more detailed information.

Other warnings/precautions:

- Discontinuation in aHUS: Monitor patients for at least 12 weeks after treatment discontinuation for signs/symptoms of thrombotic microangiopathy (TMA) complications. Signs/symptoms of TMA include angina, dyspnea, mental status changes, seizure, or thrombosis; occurrence of two or repeated measurement of any one of the following: Serum creatinine elevation ( ≥25% from baseline or nadir), serum LDH elevation ( ≥25% from baseline or nadir), thrombocytopenia (platelet decrease by ≥25% compared to baseline or peak). If TMA complications occur after stopping eculizumab, consider reinitiation of treatment, plasmapheresis, plasma exchange, fresh frozen plasma infusion, and/or appropriate organ-specific measures.

- Discontinuation in PNH: Patients with PNH who discontinue eculizumab treatment may be at increased risk for serious hemolysis; monitor closely for at least 8 weeks after treatment discontinuation.

- Immunizations: Patients should be up to date with all immunizations before initiating therapy.

- REMS program: [US Boxed Warning]: Access is restricted through a REMS program. Prescribers must be enrolled in the program; enrollment and additional information is available at 1-888-765-4747 or solirisrems.com. Counsel patients on the risk of meningococcal infection; ensure patients are vaccinated and provide educational materials.


Pregnancy Risk Factor

C


Pregnancy Considerations

Adverse events were observed in animal reproduction studies. Eculizumab crosses the placenta and can be detected in cord blood. Pregnant women with PNH and their fetuses have high rates of morbidity and mortality during pregnancy and the postpartum period. Treatment of PNH with eculizumab has been shown to increase fetal survival and decrease maternal complications (Kelly 2015). Use of eculizumab for the treatment of a HUS in pregnancy has also been described (Ardissino 2013).


Actions


Pharmacology

Terminal complement-mediated intravascular hemolysis is a key clinical feature of paroxysmal nocturnal hemoglobinuria (PNH); blocking the formation of membrane attack complex (MAC) results in stabilization of hemoglobin and a reduction in the need for RBC transfusions. Impairment of complement activity regulation leads to uncontrolled complement activation in atypical hemolytic uremic syndrome (aHUS). Eculizumab is a humanized monoclonal IgG antibody that binds to complement protein C5, preventing cleavage into C5a and C5b. Blocking the formation of C5b inhibits the subsequent formation of terminal complex C5b-9 or MAC.


Distribution

PNH: 7.7 L; aHUS: 6.14 L


Onset of Action

PNH: Reduced hemolysis: ≤1 week


Half-Life Elimination

PNH: ~11 days (range: ~8 to 15 days); aHUS: ~12 days (during plasma exchange the half-life is reduced to 1.26 hours)


Patient and Family Education


Patient Education

- Discuss specific use of drug and side effects with patient as it relates to treatment. (HCAHPS: During this hospital stay, were you given any medicine that you had not taken before? Before giving you any new medicine, how often did hospital staff tell you what the medicine was for? How often did hospital staff describe possible side effects in a way you could understand?)

- Patient may experience rhinitis, pharyngitis, rhinorrhea, back pain, nausea, vomiting, diarrhea, abdominal pain, constipation, muscle pain, joint pain, muscle spasm, pain in arms or legs, or insomnia. Have patient report immediately to prescriber signs of infection, signs of meningococcal infection, signs of low potassium (muscle pain or weakness, muscle cramps, or an abnormal heartbeat), signs of severe cerebrovascular disease (change in strength on one side is greater than the other, trouble speaking or thinking, change in balance, or change in eyesight), signs of DVT (edema, warmth, numbness, change in color, or pain in the extremities), coughing up blood, shortness of breath, bruising, bleeding, seizures, urinary retention, change in amount of urine passed, dark urine, angina, tachycardia, severe dizziness, passing out, severe headache, swelling of arm or leg, or loss of strength and energy (HCAHPS).

- Educate patient about signs of a significant reaction (eg, wheezing; chest tightness; fever; itching; bad cough; blue skin color; seizures; or swelling of face, lips, tongue, or throat). Note: This is not a comprehensive list of all side effects. Patient should consult prescriber for additional questions.

Intended Use and Disclaimer: Should not be printed and given to patients. This information is intended to serve as a concise initial reference for healthcare professionals to use when discussing medications with a patient. You must ultimately rely on your own discretion, experience and judgment in diagnosing, treating and advising patients.

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