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Primary hemophagocytic lymphohistiocytosis (HLH) is a rare genetic disease characterized by life-threatening inflammatory symptoms.1,2 Though primary HLH can affect adults and teenagers, the majority of patients are very young children—often in the first months or years of their lives.1,3 Due to the rapidly progressive nature of the disease, immediate therapy is required to temper the “cytokine storm”—a massive release of cytokines that leads to the signs and symptoms of primary HLH.1,4
Primary HLH presents as a heterogeneous syndrome of rapidly progressive, life-threatening inflammatory symptoms, including1,2:
To date, general consensus in the scientific community on primary HLH is as follows1,3:
|Mortality rate||High mortality rate|
|Age of presentation||Typically infants/early childhood; documented cases also exist in teens and adults|
|Cause||Testable genetic mutation|
|Family history||May be positive for the disease|
|Resolution||Usually requires hematopoietic stem cell transplant (HSCT)|
The symptoms of primary HLH, along with their differing levels of severity, combine to form a broad spectrum of disease presentation that varies from patient to patient and within the same patient over time. Although scientific consensus of the appropriate diagnostic criteria continues to evolve, 2 recognized options for confirming a diagnosis of primary HLH exist2,3,5,6:
In 1994, as part of the HLH-94 clinical trial, the Histiocyte Society proposed a standard definition of HLH. Click to expand this version.
When it comes to treating primary HLH, there is no time to wait. Prior to receiving the results of a genetic test, ancillary testing and flow cytometry can be used, where available, to help differentiate between primary and secondary HLH. This approach helps find the triggers or underlying causes of HLH and prevents misdiagnosis.1,8,9
Consider running the following tests9:
Consider checking for decreased levels of 9,10:
IFNγ is central to the "cytokine storm"—an uncontrolled release of inflammatory cytokines and overactivation of phagocytes that give the syndrome its name.10Learn more
Primary hemophagocytic lymphohistiocytosis (HLH) is a rare hyperinflammatory condition that mostly affects children, but can also occur in adults and teenagers.1-4
Gamifant reduces the inflammatory symptoms of primary HLH by neutralizing IFNγ.7
Gamifant is given as an intravenous infusion twice per week until the patient no longer requires therapy for the treatment of HLH or until hematopoietic stem cell transplantation (HSCT) is performed.7
Download the Start Form now or contact Gamifant Patient Services at 833-597-6530.