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Getting patients to transplant

For patients with primary hemophagocytic lymphohistiocytosis (HLH), a successful hematopoietic stem cell transplant (HSCT) is the ultimate goal. Meanwhile, keeping inflammatory symptoms under control with the proper treatment is crucial.1-3

Treatment has 2 main goals:

Subdue the cytokine storm

Immediately bring the cytokine storm under control to prevent irreversible organ damage. Subsequent treatment depends on symptom presentation and confirmation of diagnosis.1-3

Condition for transplant

Condition the patient for HSCT in a way that optimizes their long-term prognosis.1

Data show that 50% of patients fail to reach HSCT due to inadequate response to conventional treatments.4

The treatment landscape

Over the past 25 years, few advancements have been made in targeted HLH therapy.5

Conventional treatments—which were not specifically developed for primary HLH, and which are not FDA-approved for the condition—are aimed at immune suppression.1,6

When a patient responds unsatisfactorily to conventional treatment, cannot tolerate it, or relapses, there has been no standard alternative. HSCT is always the goal, but physicians may have little choice but to rush poorly conditioned patients into the procedure, or forgo transplant altogether.1,5,7

There has been a clear need for a targeted option with an established safety profile to treat patients with primary HLH.1

A targeted Treatment

Gamifant® (emapalumab-lzsg) is the first and only treatment designed specifically for primary HLH. It works by targeting IFNγ, a central and upstream cytokine in the pathogenesis of the disease.8-10

See how it works