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hemophagocytic syndrome (HLH) - Clinical Profile, Treatments & Trials | Kisho | Kisho

Rare Disease

hemophagocytic syndrome

MONDO:0015540

Also known as:HLHHemophagocytic LymphohistiocytosisFHL

Find Clinical TrialsTrials

Overview

Hemophagocytic syndrome (HPS) is a rare immune disease and a potentially life-threatening disorder characterized by cytokine storm and overwhelming inflammation causing fever, hepatosplenomegaly, cytopenia, hypertriglyceridemia, hyperferritinemia, and hemophagocytosis in bone marrow, liver, spleen or lymph nodes. It can be either primary due to a genetic defect (primary hemophagocytic lymphohistiocytosis), or secondary to malignancies, to infections, most commonly with viruses such as Epstein-Barr virus or cytomegalovirus, human immunodeficiency virus, or to autoimmune disorders such as systemic lupus erythematosus or adult-onset Still disease (secondary hemophagocytic lymphohistiocytosis).

Treatments1 FDA approved

Clinical Trials25 registered · 7 recruiting

Diagnosis

Biomarker and diagnostic research for hemophagocytic syndrome has been reported in the published literature.

Treatment

Compiled from FDA-approved drug records, orphan drug designations, and FDA Adverse Event Reporting System (FAERS) safety data. GeneReviews management guidance is included where available.

1 FDA-approved treatment is available for hemophagocytic syndrome, including EMAPALUMAB-LZSG (GAMIFANT, approved 2018). An additional 3 compounds hold orphan drug designation.

Approved Therapies

Brand Name	Generic Name	Mechanism	Approved	Market Status

Prognosis

Estimated prevalence: Unknown (Unknown prevalence).

Research Landscape

Sourced from ClinicalTrials.gov and PubMed. Publication types are classified using NLM-curated article metadata; research type breakdowns reflect the nature of published work for this disease.

25 clinical trials registered, 7 recruiting. Interventions under study include drug therapy, other interventions, biologic therapy, and gene therapy. Pipeline includes 1 PHASE4, 3 PHASE3, 4 PHASE2. Research is sponsored by a mix of industry and academic institutions.

NCT ID	Title	Phase	Sponsor	Status
[NCT05416307](https://clinicaltrials.gov/study/NCT05416307)

Data assembled from 5 of 12 sources · Last updated Sep 19, 2026, 2:54 PM UTC

MONDOHPOClinicalTrials.govFDAPubMedHGNCClinVarClinGenGeneReviewsOrphanetOMIMRUSP

Medical References

External Resources

Orphanet

European rare disease database

Orphanet:158032

GARD

Genetic and Rare Diseases Info Center

GARD:20024

Questions for Your Care Team

→What are the benefits and potential side effects of GAMIFANT for managing hemophagocytic syndrome?
→Are any of the 25 active clinical trials a good fit for my situation?
→Are there specialists or centers of excellence for this condition in my area?
→Is there a patient registry I should consider joining?

← Back to All Diseases

Brand Name	Generic Name	Sponsor	Designated	Exclusivity End	Designation Status
fully human IgG1 monoclonal antibody directed against human signal regulatory protein (SIRP)alpha, SIRPbeta1, and SIRPgamma	fully human IgG1 monoclonal antibody directed against human signal regulatory protein (SIRP)alpha, SIRPbeta1, and SIRPgamma	Electra Therapeutics, Inc.	2024	—	Designated
human monoclonal antibody based on an IgG1 lambda framework against human interferon gamma	human monoclonal antibody based on an IgG1 lambda framework against human interferon gamma	Elixiron Immunotherapeutics (Hong Kong) Limited	2022	—	Designated
tadekinig alfa	tadekinig alfa	AB2 Bio Ltd	2017	—	Designated

Research Type	Count	% of Total
Patient case studies	91	55%
Research summaries	26	16%
Disease patterns and progression	18	11%
Clinical study results	9	5%
Other research	7	4%
Testing and diagnosis research	7	4%
Laboratory research	4	2%
New treatment approaches	2	1%

Kisho

hemophagocytic syndrome

Overview

Diagnosis

Treatment

Approved Therapies

Prognosis

Research Landscape

Medical References

External Resources

Orphanet

GARD

Questions for Your Care Team

Questions for Your Care Team

Orphan Drug Designations

Clinical Trials

Recent Publications

hemophagocytic syndrome

Overview

Diagnosis

Treatment

Approved Therapies

Prognosis

Research Landscape

Medical References

External Resources

Orphanet

GARD

Related Diseases

Questions for Your Care Team

Questions for Your Care Team

Orphan Drug Designations

Clinical Trials

Recent Publications