Emapalumab Brand Name– Gamifant
What is Emapalumab
Emapalumab is an interferon gamma blocking antibody used for the treatment of primary hemophagocytic lymphohistiocytosis (HLH) in adult and pediatric patients with refractory, recurrent, or progressive disease or intolerance to conventional HLH therapy.
Primary HLH is a rare and life-threatening condition that typically affects children. In patients with HLH, the body’s immune cells do not function properly.
The cells become overactive, leading to inflammation and damage to the body’s organs, including the liver, brain, and bone marrow.
Patients with primary HLH usually develop symptoms, which may include fever, enlarged liver or spleen, and cytopenia, within the first months or years of life. Emapalumab was studied in a clinical trial of 27 pediatric patients (median age 1 year old) with suspected or confirmed primary HLH with either refractory, recurrent, or progressive disease during conventional therapy or who were intolerant to conventional therapy.
The overall response rate, defined as achievement of either a complete or partial response or HLH improvement, was 63% (95% CI: 0.42, 0.81; p = 0.013) at the end of treatment.
Additionally, 70% (19/27) of patients proceeded to hematopoietic stem cell transplantation (HSCT). Infections were the most common adverse reaction reported during the clinical trial.
Prior to treatment, evaluate patients for latent tuberculosis and administer prophylaxis to patients at risk for tuberculosis.
Additionally, administer prophylaxis for Herpes Zoster, Pneumocystis jirovecii, and fungal infections prior to emapalumab initiation.
During emapalumab treatment, routine monitoring for tuberculosis, adenovirus, Epstein-Barr virus, and cytomegalovirus is recommended.
Indications
- hemophagocytic lymphohistiocytosis
Side Effects
- abdominal pain
- antibody formation
- asthenia
- bradycardia
- constipation
- cough
- diarrhea
- dyspnea
- epistaxis
- erythema
- fever
- hyperhidrosis
- hypertension
- hypokalemia
- infection
- infusion-related reactions
- irritability
- lymphocytosis
- peripheral edema
- rash
- sinus tachycardia
- tachypnea
- vomiting
Monitoring Parameters
- D-dimer
- ferritin
- fibrinogen
- platelet count
- tuberculin skin test
- WBC
Contraindications
- breast-feeding
- infection
- infusion-related reactions
- pregnancy
- vaccination
Interactions
- Alfentanil
- Bacillus Calmette-Guerin Vaccine, BCG
- Belladonna Alkaloids; Ergotamine; Phenobarbital
- Caffeine; Ergotamine
- Carbamazepine
- Cisapride
- Cyclosporine
- Dextromethorphan; Quinidine
- Dihydroergotamine
- Ergotamine
- Ethosuximide
- Everolimus
- Fentanyl
- Influenza Virus Vaccine
- Intranasal Influenza Vaccine
- Live Vaccines
- Measles Virus; Mumps Virus; Rubella Virus; Varicella Virus Vaccine, Live
- Measles/Mumps/Rubella Vaccines, MMR
- Paclitaxel
- Phenytoin
- Pimozide
- Quinidine
- Rotavirus Vaccine
- Rubella Virus Vaccine Live
- Sirolimus
- Smallpox and Monkeypox Vaccine, Live, Nonreplicating
- Smallpox Vaccine, Vaccinia Vaccine
- Tacrolimus
- Theophylline, Aminophylline
- Thioridazine
- Tizanidine
- Typhoid Vaccine
- Varicella-Zoster Virus Vaccine, Live
- Warfarin
- Yellow Fever Vaccine, Live
