Clinical Trial: Steroid Treatment for Hypereosinophilic Syndrome
Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional
Official Title: Assessment of Glucocorticoid Responsiveness and Mechanisms of Resistance in Hypereosinophilic Syndromes
Brief Summary:
Background:
- 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.
Objectives:
- 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.
Eligibility:
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
Detailed Summary: 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/PDGFRA-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 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 4 and 24 hours post-challenge. Mechanisms and in vitro correlates of GC resistance will also be explored.
Sponsor: National Institute of Allergy and Infectious Diseases (NIAID)
Current 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 [ Time Frame: completion of Study ]
Original Primary Outcome: GC responsiveness: defined as the lowest dose of prednisone at which eosinophils remain < 1000/microl with control of symptoms, or dose at which eosinophils rebound after taper, or if there is no response, the challenge dose.
Current Secondary Outcome:
- To define a group of GC non-responders and/or suboptimal responders for further study of the mechanisms and biologic correlates of GC resistance in HES [ Time Frame: 7-24 weeks post challenge. ]
- To delineate some of the mechanisms of decreased GC responsiveness in GC-resistant subjects and to assess in-vitro correlates of therapeutic responsiveness in HES [ Time Frame: After a sufficient amount of patients from each patient typed has been enrolled and completed steroid taper ]
Original Secondary Outcome: Clinical factors associated with GC responsiveness (including HES variant, pattern of organ involvement and laboratory parameters) will be explored.
Information By: National Institutes of Health Clinical Center (CC)
Dates:
Date Received: February 1, 2012
Date Started: November 22, 2011
Date Completion: December 31, 2019
Last Updated: May 12, 2017
Last Verified: May 4, 2017
