Hemophagocytic syndrome
MONDO:0015540Hemophagocytic 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).
Also known as: HLH, Hemophagocytic Lymphohistiocytosis, hemophagocytic lymphohistiocytosis, hemophagocytic syndrome, FHL, familial erythrophagocytic lymphohistiocytosis, familial hemophagocytic lymphohistiocytosis, familial histiocytic reticulosis
61 clinical trials for this condition and its sub-types.
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Can a cancer drug tame a dangerous immune storm?
Disease control Not yet recruitingThis early study tests the drug ruxolitinib for a rare but serious condition called IEC-HS, which can happen after CAR-T therapy for cancer. The condition causes a dangerous overreaction of the immune system. The trial will include 16 adults and aims to see if the drug is safe an…
Phase: PHASE1 • Sponsor: Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins • Aim: Disease control
Last updated Jun 27, 2026 14:02 UTC
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Selective cell removal may tame stem cell Transplant's dangerous side effect
Disease control Not yet recruitingThis study tests whether removing a specific type of immune cell (naive T cells) from a donor's stem cell graft can prevent graft-versus-host disease (GVHD), a serious complication where donor cells attack the recipient's body. The trial enrolls people with non-malignant blood di…
Phase: PHASE2 • Sponsor: Fred Hutchinson Cancer Center • Aim: Disease control
Last updated Jun 27, 2026 14:01 UTC
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Could a common vitamin a drug tame a deadly immune storm?
Disease control Not yet recruitingThis study tests a drug called all-trans retinoic acid (ATRA) as a first treatment for hemophagocytic lymphohistiocytosis (HLH), a rare but life-threatening condition where the immune system goes into overdrive. ATRA is an oral medication already used for other blood diseases and…
Phase: PHASE2, PHASE3 • Sponsor: Beijing Anzhen Hospital • Aim: Disease control
Last updated Jun 27, 2026 13:02 UTC
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Gene therapy could replace bone marrow transplants for rare immune disease
Disease control Not yet recruitingThis trial tests a gene therapy for FHL3, a rare immune disorder caused by a gene mutation. Instead of a donor stem cell transplant, patients receive their own stem cells that have been corrected with a healthy gene. The goal is to restore immune function while avoiding transplan…
Phase: PHASE1, PHASE2 • Sponsor: Assistance Publique - Hôpitaux de Paris • Aim: Disease control
Last updated Jun 27, 2026 12:36 UTC
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Timing is everything: new trial aims to save lives from immune storm
Disease control Not yet recruitingThis study compares two strategies for starting the drug etoposide in adults with severe hemophagocytic lymphohistiocytosis (HLH) who are in the intensive care unit. HLH is a life-threatening overreaction of the immune system that can cause organ failure. The goal is to see if gi…
Phase: PHASE3 • Sponsor: Assistance Publique - Hôpitaux de Paris • Aim: Disease control
Last updated Jun 27, 2026 12:23 UTC
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New study aims to uncover hidden danger in kawasaki disease
Knowledge-focused Not yet recruitingThis study will observe 150 children with Kawasaki disease to learn more about a rare but serious complication called Macrophage Activation Syndrome (MAS). Researchers will collect data on symptoms, lab results, and treatments across multiple European hospitals. The goal is to be…
Sponsor: Meyer Children's Hospital IRCCS • Aim: Knowledge-focused
Last updated Jun 27, 2026 12:00 UTC