Eculizumab (Intravenous)

Eculizumab is an intravenous drug specified for hemolysis associated with PNH.

Overview

Eculizumab, also known by the brand name Soliris, is suggested for patients who have been diagnosed with PNH, a rare condition that causes the rapid destruction of blood cells.

Eculizumab may also be prescribed for aHUS, a type of kidney disorder. The exception to this rule is when patients have this condition caused by a specific organism.

Eculizumab is moreover indicated for patients with gMG - myasthenia gravis generalized.

Its three main benefits are that, generally, Eculizumab:

1. Reduces the need for blood transfusions
2. Lowers the risk of blood cell destruction
3. Improves quality of life

However, Eculizumab is not beneficial for:

  • Acute Myelogenous Leukemia
  • Aplastic Anemia
  • Blood Clots
  • Myelodysplastic Syndrome

Eculizumab injections are available by prescription only. Most noteworthy, there is limited access to this medicine due to restrictions imposed by the risk assessment program known as REMS.

In a nutshell, the REM program involves doctors:

  • Counseling patients
  • Providing supporting materials to users
  • Verifying that patients are vaccinated
  • Documenting all processes
  • Administering injections in a clinical facility

The primary reason for this program is due to the pronounced risk of acquiring meningococcal infections. The chances of transmission are notable as individuals who take Soliris are at 2,000 times the risk of acquiring the infection.

In the United States, Eculizumab is only accessible to patients who are a part of this REM program.

In medical studies, Soliris was reported as the highest priced drug in the marketplace. A part of the reason for this marked up price is its orphan drug designation.

Conditions Treated?

  • PNH
  • AHUS
  • Generalized Myasthenia Gravis (gMG)

Type Of Medicine?

  • Monoclonal antibody
  • Recombinant humanized monoclonal antibody

Side Effects

Some of the most commonly reported side effects of Soliris include:

  • A pins and needles sensation
  • A severe headache
  • Abnormal heartbeat
  • Appetite changes
  • Blood in the urine
  • Changes in stool color
  • Cold sores
  • Constipation
  • Coughing, sneezing, and a sore throat
  • Drowsiness
  • Feeling nauseous
  • Feeling nervous
  • Flu-like symptoms
  • Insomnia
  • Labored breathing
  • Leg or arm pain
  • Lethargy
  • Nasal congestion
  • Pain in the back
  • Pain while urinating
  • Pain, stiffness, or cramps in the joints or muscles
  • Perspiration
  • Ringing in the ears
  • Runny stools
  • Skin that bruises easily
  • Skin that looks pale
  • Swelling of the joints or glands
  • Trembling
  • Ulcers or sores
  • Unexplained bleeding
  • Vision changes

Dosage

Soliris or Eculizumab is supplied as a concentrated solution for intravenous infusion over 35 minutes. It is generally distributed to certified healthcare providers in cartons containing 10 ml vials.

Soliris is always administered by a trained nurse practitioner. It is infused into the veins via a slow-drip process, where patients are carefully monitored for adverse effects.

Dosage For PNH

To lower the risk of hemolysis, healthcare providers generally write the following dosage amounts for Eculizumab:

  • First Treatment: 600 mg weekly | x 4 Weeks (Via IV infusion lasting 35 minutes)
  • Second Treatment: 900 mg | After one week of the initial four treatments
  • Continuous Treatments: 900 mg | Every two weeks (Bi-monthly)

Dosage For AHUS

To lower the risks associated with this condition, healthcare providers generally write the following dosage amounts for Eculizumab:

  • First Treatment: 900 mg weekly | x 4 weeks (Via IV infusion lasting 35 minutes)
  • Second Treatment: 1200 mg | After one week of the initial four treatments
  • Continuous Treatments: 1200 mg | Every two weeks (Bi-monthly)

Dosage for Generalized Myasthenia Gravis (gMG)- for (anti-acetylcholine receptor (AchR) antibody positive):

  • First Treatment: 900 mg weekly | x 4 weeks (Via IV infusion lasting 35 minutes)
  • Second Treatment: 1200 mg | After one week of the initial four treatments
  • Continuous Treatments: 1200 mg | Every two weeks (Bi-monthly)

Note: In some cases, supplemental plasma infusion or exchange may be required.

How to Prepare

To prepare the IV, healthcare workers must start off with a sterile workspace and wear gloves and other protective gear. Other steps include:

1. Referring to the prescribed amount: Extract the recommended dose for transfer into the IV infusion bag.

2. Mixing: Follow the diluent instructions provided.

3. Capsize: After adding the diluent, the infusion bag should be turned over gently to mix the formula together.

4. Discard unused portions: Any leftovers in the vial should be discarded of and never stored. This is because Eculizumab contains no preservatives.

5. Sit at room temperature: Before injecting the solution into patients, it is very important to adjust the mix to a room temperature of 18-25°C (64-77°F). Do not microwave or place next to areas of heat.

Diluent Instructions

Concentrated solutions can be mixed with equal parts of the following diluents:

  • 5% dextrose in water injection
  • Ringer's injection
  • 0.9% sodium chloride injection
  • 0.45% sodium chloride injection

How to Administer

Some IVs require a one-off process known as the push or bolus method. However, Eculizumab is typically administered via a slow drip method lasting 35 in adults. Nevertheless, the infusion period should never surpass a two-hour timeframe.

Administration in minors differs from adult patients. The healthcare worker generally utilizes a syringe pump or gravity feed and slowly infuses the mix over a 1-4-hour timeframe.

Interactions

Currently, no negative drug interactions have been established with Eculizumab or Soliris.

Underlying Medical Conditions

Eculizumab should not be used in patients with certain pre-existing conditions, including:

  • Current infections: Taking Eculizumab could lower the body's natural ability to fight off new infections.
  • Meningococcal infections: A heightened risk of developing this infection has been found with Eculizumab use.

Warnings

Soliris should be used with caution. It includes a black box warning or insert label with some of the following precautions:

Risk of sensitivities

In rare cases, patients may develop an allergic reaction to Soliris injections. This condition requires immediate medical intervention.

Tell your healthcare provider if you develop the following signs of this condition within the same day of treatment:

  • Chills or fevers
  • Labored breathing
  • Pain in the chest
  • Rashes or hives
  • Skin irritation
  • Swelling in the face

Follow-Up checks

Soliris users should anticipate follow-up checks to be scheduled post administration. Blood tests are generally performed to determine if the medicine is working properly.

Post-treatment checkups for patients with PNH

Following Soliris injections, PNH patients may experience hemolysis. It is critical that patients attend all appointments on time due to this risk. Close monitoring is generally completed by health workers for a minimum of two months following the last administration of Soliris.

Post-treatment checkups for patients with aHUS

Similarly, aHUS patients are generally monitored after Soliris treatments. In these cases, close monitoring lasts three months. The main side effect that medical providers watch for is blood clots forming in small blood vessels. This condition is otherwise known as thrombotic microangiopathy or TMA and the main warning signs are:

  • Tightness or pain in the chest
  • Trouble breathing
  • Mood swings
  • Feeling anxious
  • Feeling depressed
  • Convulsions

Patients who notice any of these signs should contact 911 right away. Do not wait until your next follow-up visit to address these symptoms, as TMA could be fatal if left untreated.

Avoid contact with infectious patients

If you take Soliris, contact with other members of your household who may be sick should be avoided. This is particularly true if someone you come into close contact with on a regular basis has been diagnosed with a meningococcal infection.

To decrease the risk of transmission of a meningococcal infection, your medical worker may schedule a vaccine at least 14 days in advance of the treatment. If you have previously received this immunization, a supplementary dose may be prescribed.

Signs of a meningococcal infection

Meningococcal infections are serious and potentially fatal, and the risk is heightened 2000 times in patients taking Soliris. A meningococcal infection requires prompt medical intervention. Some of the warning signs include:

  • Severe headaches
  • Feeling nauseous
  • Vomiting
  • Spikes in temperature
  • Stiffness in the back or neck
  • A sense of confusion
  • Muscle pain
  • Eye sensitivities

If any of these occur following a dose of Soliris, contact your medical provider or call 911 right away.

Other systemic infections

Patients who take Soliris may also be at a greater risk of acquiring other systemic infections, such as:

  • Streptococcus Pneumoniae
  • H Influenzae

Therefore, care should be taken to avoid others with these and other highly infectious diseases. To elaborate on the warning made earlier, do not stay in the same room as others who have an infection. A doctor can provide more instructions to help keep you safe.

Pregnant and lactating mothers

Eculizumab is not recommended for pregnant or nursing mothers, though insufficient human studies exist. In animal studies, abnormalities in fetal development have been reported. The medicine should be used with caution and patients should inform medical providers if they are pregnant, nursing or plan to be prior to starting treatment with Eculizumab.

Storage

Storage instructions for Eculizumab vary based on whether the vial is sealed or diluted.

Unopened vials:

To store, refrigerate at temperatures of 2-8ºC (36-46ºF). To preserve the quality of the medicine, healthcare workers should never freeze or shake the vial. Additionally, unused vials should be kept away from heat or light.

Unopened cartons

Medical facilities receiving cartons of Eculizumab may store the original and untampered carton at a controlled room temperature not exceeding 25°C (77°F) once, for a period of up to 72 hours or 3 days.

Other storage instructions

Be sure to discard expired vials. The expiration dates are imprinted on the original cartons.

Diluted solutions

Mixed solutions of Eculizumab with diluents may be refrigerated for up to 24 hours at temperatures of 2-8°C (36-46°F).

Summary

Eculizumab is suggested for the prevention of hemolysis or red blood cell destruction associated with PNH. It is also indicated for aHUS and generalized myasthenia gravis (gMG).

The dosage varies based on the condition being treated, as well as the patient's age and weight. A preliminary treatment is given via an infusion bag once weekly for four weeks. One follow-up treatment is required one week later followed by a bi-monthly maintenance dose.

This antibody, however, can only be prescribed by certified doctors who are enrolled in REMS. This is mainly due to users' high risk of acquiring meningococcal infections.

Generally speaking, Eculizumab increases patients' overall quality of life and reduces the need for blood transfusions.