Steroid Treatment for Hypereosinophilic Syndrome

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Brief Title

Steroid Treatment for Hypereosinophilic Syndrome

Official Title

Assessment of Glucocorticoid Responsiveness and Mechanisms of Resistance in Hypereosinophilic Syndromes

Brief Summary


      - Hypereosinophilic syndrome (HES) is a disorder in which the body has too many eosinophils
      (a type of white blood cell). Too many eosinophils in HES can cause damage to the heart,
      nerves, or skin. Certain drugs can help lower eosinophil counts to prevent tissue damage.
      Corticosteroids, such as prednisone, are used for initial therapy in this disorder. Although
      most people respond to prednisone, some people develop side effects from it, or do not
      respond very well to treatment. Better ways of determining the dose to give could help to
      decide on the best therapy for HES.


        -  To determine whether a single-dose of prednisone can be used to predict which people
           with hypereosinophilia respond to treatment.

        -  To study lack of response to steroid treatment in people with HES.


      Inclusion criteria:

        -  Individuals with hypereosinophilic syndrome with high eosinophil counts.

        -  Individuals who are willing to have blood drawn before and after getting steroids.

      Exclusion criteria:

        -  Individuals who are on more than 10mg of prednisone (or similar drug)

        -  Individuals with hypereosinophilic syndrome who are on other medications that could
           interfere with the study

        -  Women who are pregnant or breast-feeding

        -  Individuals who have a known gene mutation associated with chronic eosinophilic leukemia

        -  Children less than 18 years old who weigh less than 48kg or 106lb


        -  Participants will have a screening visit with a physical exam and medical history. Blood
           and urine samples will be collected.

        -  Participants will have a single dose of the steroid prednisone by mouth in the morning.
           Blood samples will be collected 2, 4, 24 hours after this dose.

        -  On the day after the steroid dose, participants will provide another blood sample in the

        -  Participants will start to take prednisone daily when they return home. Blood samples
           will be collected weekly at the participant s doctor s office. The dose of prednisone
           will be lowered depending on the weekly eosinophil count. We will try to get each person
           on the lowest dose of prednisone possible that will control the disorder. Participants
           who do not respond or have severe side effects will be taken off prednisone. Other
           treatments will be considered for people who do not respond to steroids. The goal is to
           evaluate the response to prednisone. Our research will try to figure out why some people
           do not respond to steroids. Most people will complete the study within 6 to 16 weeks,
           depending on their response to prednisone.

Detailed Description

      This study aims to develop a model to determine whether a single, oral, weight-based dose of
      glucocorticoid (GC) can predict clinical and biologic response to GC s over the long term in
      subjects with hypereosinophilic syndrome (HES). Subjects with FIP1L1/PDGFRalpha-negative HES,
      who are symptomatic with eosinophil count >1500/microL and receiving less than or equal to10
      mg prednisone daily, will be enrolled. A single oral dose of prednisone (1 mg/kg rounded to
      the nearest 5mg) will be administered. Eosinophil count and various laboratory parameters
      will be assessed at 2 hours, 4 hours and 24 hours following prednisone administration
      (investigators will be blinded to the results of the eosinophil counts). The subjects will
      then begin GC therapy at 30 mg prednisone daily followed by a standardized taper. The lowest
      dose of GC at which symptoms and eosinophilia are controlled will be compared to the change
      in eosinophil count at 2, 4 and 24 hours post-challenge. Mechanisms and in vitro correlates
      of GC resistance will also be explored.

Study Phase

Phase 4

Study Type


Primary Outcome

To develop a model to determine whether a single dose steroid challenge can be used to predict GC responsiveness in subjects with HES.





Study Arms / Comparison Groups

Description:  All subjects will receive a single oral dose of prednison (1 mg/kg rounded to the neares 5 mg). The subjects will then begin GC therapy at 30 mg prednisone daily followed by a standardized taper.


* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information

Recruitment Status


Estimated Enrollment


Start Date

February 16, 2012

Completion Date

December 10, 2020

Primary Completion Date

December 10, 2020

Eligibility Criteria


        Subjects on Protocol #94-I-0079 will be eligible for participation in the study only if all
        of the following criteria apply:

          1. Subjects must be 7 years of age or older to enroll

          2. Subject meets diagnostic criteria for HES (AEC >1500/microL, absence of a secondary
             cause and signs and/or symptoms attributable to the eosinophilia)

          3. AEC greater than 1500 microL obtained within 14 days prior to enrollment

          4. Willingness to perform the timed steroid challenge

          5. Appropriate candidate for GC treatment after challenge

          6. Willingness to have samples stored for future research


        A subject will not be eligible to participate in the study if any of the following apply:

          1. Receiving >10 mg prednisone or equivalent at the time of enrollment.

          2. Receiving less than or equal to 10 mg of prednisone or equivalent but have not been on
             a fixed dose for at least 3 weeks (subjects on a current corticosteroid taper will be

          3. AEC less than or equal to 1500/microl on the day of the steroid challenge

          4. Use of immunomodulatory medications, (other than less than or equal to 10 mg/day
             prednisone) including but not limited to biologics, within the past 6 months.

          5. Pregnant at the time of screening.

          6. Have a known mutation in the FIP1L1-PDGFR gene.

          7. Any condition that, in the opinion of the investigator, places the subject at undue
             risk by participating in the protocol.

          8. Weight less than 48 kg (106 lbs) in subjects less than 18 years of age.




7 Years - 100 Years

Accepts Healthy Volunteers



Paneez Khoury, M.D., , 

Location Countries

United States

Location Countries

United States

Administrative Informations



Organization ID


Secondary IDs


Responsible Party


Study Sponsor

National Institute of Allergy and Infectious Diseases (NIAID)

Study Sponsor

Paneez Khoury, M.D., Principal Investigator, National Institute of Allergy and Infectious Diseases (NIAID)

Verification Date

February 22, 2021