Steroid Treatment for Hypereosinophilic Syndrome
| Status: | Recruiting |
|---|---|
| Conditions: | Blood Cancer, Hematology |
| Therapuetic Areas: | Hematology, Oncology |
| Healthy: | No |
| Age Range: | 7 - 100 |
| Updated: | 3/31/2019 |
| Start Date: | February 16, 2012 |
| End Date: | December 31, 2023 |
| Contact: | Nicole C Holland-Thomas, R.N. |
| Email: | hollandnc@mail.nih.gov |
| Phone: | (301) 402-5969 |
Assessment of Glucocorticoid Responsiveness and Mechanisms of Resistance in Hypereosinophilic Syndromes
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 associated with chronic eosinophilic leukemia
- Children less than 18 years old who weigh less than 48kg or 106lb
Design:
- 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
morning.
- 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.
- 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 associated with chronic eosinophilic leukemia
- Children less than 18 years old who weigh less than 48kg or 106lb
Design:
- 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
morning.
- 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.
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 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.
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 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.
- SUBJECT INCLUSION CRITERIA:
Subjects on Protocol #94-I-0079 will be eligible for participation in the study only if all
of the following criteria apply:
1. Subject meets diagnostic criteria for HES (AEC >1500/microL, absence of a secondary
cause and signs and/or symptoms attributable to the eosinophilia)
2. Willingness to perform the timed steroid challenge
3. Appropriate candidate for GC treatment after challenge
4. Willingness to have samples stored for future research
SUBJECT EXCLUSION CRITERIA:
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
excluded).
3. AEC less than or equal to 1500/microl at the time of enrollment.
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 evaluation.
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.
We found this trial at
1
site
9000 Rockville Pike
Bethesda, Maryland 20892
Bethesda, Maryland 20892
Phone: 800-411-1222
Click here to add this to my saved trials
