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Primary HLH

A rapidly progressive and fatal disease

Primary hemophagocytic lymphohistiocytosis (HLH) is a rare genetic disorder that can quickly become fatal. Without timely diagnosis and effective treatment, the median survival for patients with primary HLH is under 2 months.1

Primary HLH affects mostly children

Primary HLH typically occurs in infancy and early childhood, manifesting mostly during the first year of life, but it can also occur in teens and adults.1,2

Diagnosis can be challenging

Primary HLH has a variable presentation with symptoms that may include1,3:

IFNγ is a key cytokine in primary HLH

Massive overexpression of IFNγ is central to the cytokine "storm"—an uncontrolled release of inflammatory cytokines and overactivation of phagocytes that give the syndrome its name.4

Treatment has two main goals

 

1.

Immediately bring hyperinflammatory symptoms under control.1,5,6

 

2.

Prepare the patient for hematopoietic stem cell transplant (HSCT).1

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Indication and Usage

Gamifant® (emapalumab-lzsg) is an interferon gamma (IFNγ)–blocking antibody indicated for the treatment of adult and pediatric (newborn and older) patients with primary hemophagocytic lymphohistiocytosis (HLH) with refractory, recurrent, or progressive disease or intolerance with conventional HLH therapy.

Important Safety Information

Before initiating Gamifant, patients should be evaluated for infection, including latent tuberculosis (TB). Prophylaxis for TB should be administered to patients who are at risk for TB or known to have a positive purified protein derivative (PPD) test result or positive IFNγ release assay.

During Gamifant treatment, patients should be monitored for TB, adenovirus, Epstein-Barr virus (EBV), and cytomegalovirus (CMV) every 2 weeks and as clinically indicated.

Patients should be administered prophylaxis for herpes zoster, Pneumocystis jirovecii, and fungal infections prior to Gamifant administration.

Do not administer live or live attenuated vaccines to patients receiving Gamifant and for at least 4 weeks after the last dose of Gamifant. The safety of immunization with live vaccines during or following Gamifant therapy has not been studied.

Infusion-Related Reactions

Infusion-related reactions, including drug eruption, pyrexia, rash, erythema, and hyperhidrosis, were reported with Gamifant treatment in 27% of patients. In one-third of these patients, the infusion-related reaction occurred during the first infusion.

Adverse Reactions

In the pivotal trial, the most commonly reported adverse reactions (≥10%) for Gamifant included infection (56%), hypertension (41%), infusion-related reactions (27%), pyrexia (24%), hypokalemia (15%), constipation (15%), rash (12%), abdominal pain (12%), CMV infection (12%), diarrhea (12%), lymphocytosis (12%), cough (12%), irritability (12%), tachycardia (12%), and tachypnea (12%).

Additional selected adverse reactions (all grades) that were reported in less than 10% of patients treated with Gamifant included vomiting, acute kidney injury, asthenia, bradycardia, dyspnea, gastrointestinal hemorrhage, epistaxis, and peripheral edema.

Please see full Prescribing Information for Gamifant.

You may also contact Sobi at medinfo.us@sobi.com or 866-773-5274.