What is HLH medical?
What is HLH medical?
Hemophagocytic lymphohistiocytosis (HLH) is a rare disease that usually occurs in infants and young children. It may also occur in adults. Children usually inherit the disease. In adults, many different conditions, including infections and cancer, can cause HLH.
What is Gamifant used for?
GAMIFANT is 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.
What causes HLH in adults?
Acquired HLH in adults can be caused by: Viral infections, most often Epstein-Barr virus. Other infections from bacteria or fungi. Some types of cancer, such as T-cell lymphoma.
What is the treatment for HLH?
The goal of therapy for patients with HLH is to suppress life-threatening inflammation by destroying immune cells. Induction therapy based on the HLH-94 protocol consists of a series of weekly treatments with dexamethasone and etoposide (VP-16).
How long does it take to recover from HLH?
Familial HLH is fatal without treatment, with median survival of about two to six months. Chemotherapy and/or immunotherapy temporarily control the disease, but symptoms inevitably return. Stem cell transplant is the only way to cure familial HLH.
What is primary HLH?
Primary, or familial, HLH is caused by problems in genes that control how the immune system kills virus-infected or other abnormal cells in a person’s body. These genes include PRF1, MUNC 13-4, STXBP2 and STX11. Primary HLH also occurs in some closely related inherited diseases.
How is Gamifant administered?
Gamifant is administered as an intravenous infusion over 1 hour twice a week (every 3 to 4 days) until HSCT is performed or unacceptable toxicity.
How do you treat HLH in adults?
Nonetheless, standard treatment of HLH includes the addition of a steroid to etoposide for further suppression of hypercytokinemia and inflammation. Patients with a known predisposition to HLH, recurrent disease, or no clear precipitating cause should ultimately be taken to allogeneic bone marrow transplant.
How long do people with HLH live?
Familial HLH is fatal without treatment, with median survival of about two to six months. Chemotherapy and/or immunotherapy temporarily control the disease, but symptoms inevitably return.