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Medications for Hemophagocytic Lymphohistiocytosis

Other names: Haemophagocytic Lymphohistiocytosis; Haemophagocytic Syndrome; Hemophagocytic Syndrome; HLH

Hemophagocytic lymphohistiocytosis (HLH)  is a life-threatening condition due to overactive cells of the immune system causing excessive inflammation, which results in damage to tissue and organs especially the liver, brain, skin and bone marrow.

There are two types of HLH, primary HLH and secondary HLH.   Primary HLH is a hereditary condition that is also called  familial hemophagocytic lymphohistiocytosis (FHL).   Secondary HLH or acquired hemophagocytic lymphohistiocytosis usually occurs after a substantial immune reaction for example a systemic infection, malignancy or immunodeficiency.

In the immune system when the T cells and NK cells work correctly their job is to destroy damaged or infected cells within the body.  However in HLH there is a decrease in NK cell activity and an increase in T cells, with a resulting increase in cytokines.  This causes an prolonged macrophage activation and inflammatory reactions which results in the extensive tissue and organ damage and symptoms.

Symptoms of HLH are usually rash, fever, enlarged lymph nodes, enlarged liver and spleen, swollen gums and gastrointestinal problems. If HLH is untreated it can progress to multi-organ failure.  Therapy for HLH is aimed at reducing the life-threatening inflammation.

Drugs used to treat Hemophagocytic Lymphohistiocytosis

The following list of medications are in some way related to, or used in the treatment of this condition.

Drug name Rating Reviews Activity ? Rx/OTC Pregnancy CSA Alcohol
Gamifant Rate Add review
Rx N

Generic name: emapalumab systemic

Drug class: selective immunosuppressants

For consumers: dosage, interactions, side effects

For professionals: Prescribing Information

emapalumab Rate Add review
Rx N

Generic name: emapalumab systemic

Brand name:  Gamifant

Drug class: selective immunosuppressants

For consumers: dosage, interactions, side effects

For professionals: A-Z Drug Facts, AHFS DI Monograph

Legend

Rating For ratings, users were asked how effective they found the medicine while considering positive/adverse effects and ease of use (1 = not effective, 10 = most effective).
Activity Activity is based on recent site visitor activity relative to other medications in the list.
Rx Prescription Only.
OTC Over the Counter.
Rx/OTC Prescription or Over the Counter.
Off-label This medication may not be approved by the FDA for the treatment of this condition.
EUA An Emergency Use Authorization (EUA) allows the FDA to authorize unapproved medical products or unapproved uses of approved medical products to be used in a declared public health emergency when there are no adequate, approved, and available alternatives.
Pregnancy Category
A Adequate and well-controlled studies have failed to demonstrate a risk to the fetus in the first trimester of pregnancy (and there is no evidence of risk in later trimesters).
B Animal reproduction studies have failed to demonstrate a risk to the fetus and there are no adequate and well-controlled studies in pregnant women.
C Animal reproduction studies have shown an adverse effect on the fetus and there are no adequate and well-controlled studies in humans, but potential benefits may warrant use in pregnant women despite potential risks.
D There is positive evidence of human fetal risk based on adverse reaction data from investigational or marketing experience or studies in humans, but potential benefits may warrant use in pregnant women despite potential risks.
X Studies in animals or humans have demonstrated fetal abnormalities and/or there is positive evidence of human fetal risk based on adverse reaction data from investigational or marketing experience, and the risks involved in use in pregnant women clearly outweigh potential benefits.
N FDA has not classified the drug.
Controlled Substances Act (CSA) Schedule
M The drug has multiple schedules. The schedule may depend on the exact dosage form or strength of the medication.
U CSA Schedule is unknown.
N Is not subject to the Controlled Substances Act.
1 Has a high potential for abuse. Has no currently accepted medical use in treatment in the United States. There is a lack of accepted safety for use under medical supervision.
2 Has a high potential for abuse. Has a currently accepted medical use in treatment in the United States or a currently accepted medical use with severe restrictions. Abuse may lead to severe psychological or physical dependence.
3 Has a potential for abuse less than those in schedules 1 and 2. Has a currently accepted medical use in treatment in the United States. Abuse may lead to moderate or low physical dependence or high psychological dependence.
4 Has a low potential for abuse relative to those in schedule 3. It has a currently accepted medical use in treatment in the United States. Abuse may lead to limited physical dependence or psychological dependence relative to those in schedule 3.
5 Has a low potential for abuse relative to those in schedule 4. Has a currently accepted medical use in treatment in the United States. Abuse may lead to limited physical dependence or psychological dependence relative to those in schedule 4.
Alcohol
X Interacts with Alcohol.

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Further information

Always consult your healthcare provider to ensure the information displayed on this page applies to your personal circumstances.