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Comparing the timing of etoposide and dexamethasone treatment for patients with severe sporadic hemophagocytic lymphohistiocytosis in intensive care.

Verified siteRegistered drugNo placebo
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What is this trial about?

A plain-language summary of the goals, design and what participants do

This study focuses on patients with Hemophagocytic lymphohistiocytosis, a rare and serious condition where the immune system becomes overactive and causes excessive inflammation. This inflammation can lead to organ failure, which occurs when vital organs like the liver, kidneys, or lungs stop working correctly. The study is being conducted to compare two different ways of starting a treatment called etoposide in patients who are receiving intensive care. All patients in the study also receive dexamethasone, a medication used to reduce inflammation.

The first group will receive etoposide very early, within 12 hours of being admitted to the study. The second group will follow a delayed strategy, where the medication is only started if the patient's condition does not improve after 48 hours. During the course of the study, researchers will monitor how these different timing strategies affect the development or worsening of organ problems. The study will also look at various aspects of recovery, such as the length of time spent in the hospital and the need for support from machines like a ventilator, which helps a person breathe.

The research process

The trial runs in 4 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    <b>study inclusion</b>

    Participation in the trial begins upon inclusion for the treatment of hemophagocytic lymphohistiocytosis (HLH), which is a rare condition where the immune system becomes overactive.

  2. Step 2

    <b>treatment strategies</b>

    One of two treatment strategies for the administration of etoposide (a medication given through an intravenous infusion or injection into a vein) will be followed.

    The first strategy involves the administration of etoposide at a dose of 100 mg/m2 (milligrams per square meter of body surface area) within 12 hours of inclusion.

    The second strategy involves a delayed administration of etoposide. in this approach, the medication is only started if there is no improvement or if the condition worsens after 48 hours.

    dexamethasone (a medication given through intravenous use) at a dose of 10 mg/m2 is also used as a background treatment.

  3. Step 3

    <b>monitoring and evaluation</b>

    The progression of organ failure is monitored using a scoring system called the modified Sequential Organ Failure Assessment (SOFA) score. this score tracks how well different organ systems are functioning, excluding blood-related components.

    An event is recorded if there is an increase of at least 1 point in at least two organ systems compared to the first day of inclusion.

    Various health indicators are tracked, including the need for a ventilator (a machine to help with breathing), the need for catecholamines (medications used to support blood pressure), and the need for renal replacement therapy (a procedure such as dialysis to perform the functions of the kidneys).

    Blood levels, such as fibrinogen (a protein that helps blood clot), ferritin (a protein that stores iron), and triglycerides (a type of fat in the blood), are monitored to track changes during the stay in the intensive care unit.

    The HScore, which is a tool used to assess the activity of the disease, is evaluated on days 2, 7, and 14.

  4. Step 4

    <b>follow-up period</b>

    The period of observation for survival and other health outcomes lasts for a maximum of 60 days after inclusion.

Who can join the trial?

11 criteria

  • You must be an adult, meaning you have reached the age of legal maturity.
  • You must have a confirmed diagnosis of Hemophagocytic lymphohistiocytosis (HLH), which is a rare and serious condition where the immune system becomes overactive and attacks the body.
  • To confirm the diagnosis, you must meet at least 5 out of 6 specific medical signs, which include fever, an enlarged spleen (the organ that filters blood), cytopenias (a low count of different types of blood cells), high levels of triglycerides (fats in the blood) or low levels of fibrinogen (a protein that helps blood clot), evidence of hemophagocytosis (specialized immune cells eating other blood cells) in the bone marrow or organs, or very high levels of ferritin (a protein that stores iron).
  • The diagnosis must be officially confirmed by a multidisciplinary team, which is a group of different medical specialists working together.
  • This must be your first episode of this condition.
  • You must be admitted to the intensive care unit, a special hospital area for patients who need constant, close monitoring and advanced support.
  • You must be experiencing one or more types of organ failure, which means one of your body's vital organs is not working correctly.
  • Circulatory failure requirements include very low blood pressure, high levels of lactate (a substance produced when the body's tissues do not get enough oxygen), or the need for catecholamines (strong medicines used to raise blood pressure).
  • Respiratory failure requirements include needing significant oxygen therapy (extra oxygen supplied to the body), non-invasive ventilation (a mask that helps you breathe), high-flow nasal cannula oxygen therapy (a device that delivers high amounts of oxygen through the nose), or invasive mechanical ventilation (a breathing tube placed in the airway).
  • Renal failure requirements include kidney problems where creatinine (a waste product in the blood) is much higher than your normal level, or a very low urine output (the amount of urine your body produces) over 12 hours.
  • Neurological failure requirements include a Glasgow Coma Scale (GCS) score of 13 or less, which is a medical tool used to measure a person's level of consciousness and awareness.

Who cannot join the trial?

12 criteria

  • Patients who are in a critical state of distributive shock, which is a condition where blood vessels lose their tone and blood pressure drops dangerously low, and who require very high doses of noradrenaline (a medicine used to raise blood pressure) and are at immediate risk of death.
  • People who have recently received a live attenuated vaccine, which is a type of vaccine that uses a weakened version of a germ.
  • Anyone with an allergy or medical reason not to use dexamethasone (a type of steroid medicine) or any of the other ingredients used to make it.
  • Patients who develop severe acute malnutrition (SAM), which is a serious state of extreme weight loss and lack of nutrients, within 15 days of receiving chemotherapy for cancer or blood diseases.
  • People who are already participating in another medical research study involving an intervention.
  • Situations where it is not possible to give the medicine etoposide (a type of chemotherapy drug) within 12 hours.
  • Patients who have already received etoposide before being admitted to the intensive care unit.
  • Anyone with an allergy to etoposide or any of the other ingredients used to make it.
  • Patients who do not have social security coverage.
  • Patients who are under legal guardianship, meaning someone else has been legally appointed to make decisions for them.
  • Minors, which refers to anyone under the legal age of adulthood.
  • Women who are pregnant or lactating (breastfeeding).
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Investigated drugs

  • Etoposide

    is a type of chemotherapy medicine that is given through a vein to help treat certain severe conditions by affecting how cells grow and multiply.

  • Dexamethasone

    is a type of steroid medicine that is used to reduce swelling and calm an overactive immune system.

What is already known about the treatment

  • Etoposide

    This medication is an anticancer drug administered through an intravenous injection or infusion into a vein. It is a well-established treatment used to manage various types of cancer and certain severe immune system disorders like hemophagocytic lymphohistiocytosis. At a molecular level, it works by interfering with the way DNA replicates within cells, which prevents fast-growing or harmful cells from multiplying. It is classified as a topoisomerase inhibitor, a type of chemotherapy that stops cells from repairing their genetic material.

  • Dexamethasone

    This is a potent steroid medication given through an intravenous injection into the bloodstream. It is widely used in medical practice to reduce swelling, calm an overactive immune system, and treat various inflammatory conditions. It works by mimicking natural hormones in the body to block the production of chemicals that cause inflammation. Pharmacologically, it is classified as a glucocorticoid, which helps control the body's immune response.

Investigated diseases

Hemophagocytic lymphohistiocytosis - This condition occurs when the immune system becomes overactive and begins to attack the body's own cells. It involves an excessive inflammatory response that can lead to widespread damage. As the condition progresses, the immune cells may begin to consume various blood cells within the organs. This process often results in the dysfunction of several major organ systems.
Trial detailsLast updated 7 Oct 2026
Age18+ yearsPhasePhase IIITrial ID2024-511807-41-01Protocol codeAPHP230874Estimated enrolment176 patientsSponsorAssistance Publique Hopitaux De Paris

sourced from the EU Clinical Trials Register and site verification

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