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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 two main goals:
Subdue the cytokine storm
Immediately bring the cytokine storm under control. 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 are not FDA-approved for primary HLH, are aimed at immune supression.5,6
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
A targeted Treatment
Gamifant® (emapalumab-lzsg) is the first and only treatment designed specifically for primary HLH.It works by targeting IFNγ, a central cytokine in the pathogenesis of the disease.8-10
See how it works