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macrophage activation syndrome (MAS) - Clinical Profile, Genes & Research | Kisho | Kisho

Rare Disease

macrophage activation syndrome

MONDO:0015545

Also known as:MASreactive hemophagocytic lymphohistiocytosis

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Overview

A complication of rheumatic disease that is caused by excessive activation and uncontrolled proliferation of T lymphocytes and well-differentiated macrophages. It is characterized by fever, pancytopenia, liver insufficiency, coagulopathy and neurologic symptoms.

TreatmentsNo approved treatments · 1 in pipeline

Clinical Trials5 registered · 1 recruiting

Diagnosis

Biomarker and diagnostic research for macrophage activation 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.

No approved treatments are currently available for macrophage activation syndrome. An additional 1 compound holds orphan drug designation.

Orphan Drug Designations

While no drugs are FDA-approved specifically for macrophage activation syndrome, some of the following designated compounds may be used off-label in clinical practice. Treatment decisions should be made in consultation with a specialist familiar with this condition.

The following drugs have received orphan drug designation from the FDA for macrophage activation syndrome. Orphan designation reflects regulatory interest and does not indicate approval for treatment.

Brand Name	Generic Name	Sponsor

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.

5 clinical trials registered, 1 recruiting. Interventions under study include other interventions, drug therapy, and biologic therapy. Pipeline includes 1 PHASE2, 1 PHASE1. Research is primarily sponsored by academic and government institutions.

NCT ID	Title	Phase	Sponsor	Status
[NCT01966367](https://clinicaltrials.gov/study/NCT01966367)

Data assembled from 5 of 12 sources · Last updated Sep 19, 2026, 2:42 AM UTC

MONDOHPOClinicalTrials.govFDAPubMedHGNCClinVarClinGenGeneReviewsOrphanetOMIMRUSP

Medical References

External Resources

Orphanet

European rare disease database

Orphanet:158061

GARD

Genetic and Rare Diseases Info Center

GARD:12124

Questions for Your Care Team

→Are any of the 5 active clinical trials a good fit for my situation?
→What is the current development status of dusquetide, and when might it become available?
→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

Research Type	Count	% of Total
Research summaries	45	28%
Patient case studies	38	24%
Laboratory research	20	13%
Disease patterns and progression	19	12%
Testing and diagnosis research	13	8%
Other research	12	8%
Clinical study results	10	6%
New treatment approaches	2	1%

Kisho

macrophage activation syndrome

Overview

Diagnosis

Treatment

Orphan Drug Designations

Prognosis

Research Landscape

Medical References

External Resources

Orphanet

GARD

Questions for Your Care Team

Questions for Your Care Team

Clinical Trials

Recent Publications

Latest News & Developments

A systematic review of juvenile dermatomyositis complicated by macrophage activation syndrome.

EBV induced Evans syndrome in a patient of SLE related MAS: A case report.

Treatment outcomes and predictors of macrophage activation syndrome in adult-onset Still's disease: a 24 year single-centre experience.

macrophage activation syndrome

Overview

Diagnosis

Treatment

Orphan Drug Designations

Prognosis

Research Landscape

Medical References

External Resources

Orphanet

GARD

Related Diseases

Questions for Your Care Team

Questions for Your Care Team

Clinical Trials

Recent Publications

Latest News & Developments

A systematic review of juvenile dermatomyositis complicated by macrophage activation syndrome.

EBV induced Evans syndrome in a patient of SLE related MAS: A case report.

Treatment outcomes and predictors of macrophage activation syndrome in adult-onset Still's disease: a 24 year single-centre experience.