Trial Outcomes & Findings for Steroid Treatment for Hypereosinophilic Syndrome (NCT NCT01524536)
NCT ID: NCT01524536
Last Updated: 2022-02-15
Results Overview
Response to glucocorticoid challenge as assessed by the percentage of the eosinophil count 24 hours post-glucocorticoid challenge relative to the Baseline AEC for each participant. Measure Description (%): Percent of baseline AEC is defined as the Absolute Eosinophil Count at 24 hours divided by the Absolute Eosinophil Count at Baseline \*100.
Recruitment status
COMPLETED
Study phase
PHASE4
Target enrollment
26 participants
Primary outcome timeframe
24 hours
Results posted on
2022-02-15
Participant Flow
26 participants were consented to protocol but three did not meet the eligibility criteria so did not start study.
Participant milestones
| Measure |
Steroid Challenge
A single oral dose of prednisone (1 mg/kg rounded to the nearest 5mg) was administered to all participants. The following day, participant began glucocorticoid therapy of prednisone 30 mg oral daily for one week followed by a standardized taper until a minimally effective dose was achieved or participant tapered to prednisone 5 mg oral daily.
|
|---|---|
|
Overall Study
STARTED
|
23
|
|
Overall Study
COMPLETED
|
23
|
|
Overall Study
NOT COMPLETED
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Steroid Treatment for Hypereosinophilic Syndrome
Baseline characteristics by cohort
| Measure |
Steroid Challenge
n=23 Participants
A single oral dose of prednisone (1 mg/kg rounded to the nearest 5mg) was administered to all participants. The following day, participant began glucocorticoid therapy of prednisone 30 mg oral daily for one week followed by a standardized taper until a minimally effective dose was achieved or participant tapered to prednisone 5 mg oral daily.
|
|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
22 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
1 Participants
n=5 Participants
|
|
Sex: Female, Male
Female
|
12 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
11 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
6 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
17 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
2 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
1 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
15 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
1 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
4 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: 24 hoursPopulation: All participants who received the single challenge dose of prednisone
Response to glucocorticoid challenge as assessed by the percentage of the eosinophil count 24 hours post-glucocorticoid challenge relative to the Baseline AEC for each participant. Measure Description (%): Percent of baseline AEC is defined as the Absolute Eosinophil Count at 24 hours divided by the Absolute Eosinophil Count at Baseline \*100.
Outcome measures
| Measure |
Steroid Challenge - Responders
n=16 Participants
A single oral dose of prednisone (1 mg/kg rounded to the nearest 5mg) was administered to all participants. The following day, participant began glucocorticoid therapy of prednisone 30 mg oral daily for one week followed by a standardized taper until a minimally effective dose was achieved or participant tapered to prednisone 5 mg oral daily.
|
Steroid Challenge - Non-responders
n=7 Participants
A single oral dose of prednisone (1 mg/kg rounded to the nearest 5mg) was administered to all participants. The following day, participant began glucocorticoid therapy of prednisone 30 mg oral daily for one week followed by a standardized taper until a minimally effective dose was achieved or participant tapered to prednisone 5 mg oral daily.
|
Steroid Challenge - Glucocorticoid Unresponsiveness
Participants with hypereosinophilic syndromes (HES) that were not responsive to prednisone defined as no decline of absolute eosinophil count (AEC) below 1000/mm\^3 after one week of prednisone 30 mg daily, followed by 2 days of prednisone 60 mg daily
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|---|---|---|---|
|
Mean Percent Change in Eosinophil Count After Glucocorticoid Challenge
|
-69.6 percentage of eosinophil count
Interval -80.1 to -59.1
|
-13.6 percentage of eosinophil count
Interval -35.2 to 9.13
|
—
|
SECONDARY outcome
Timeframe: Baseline (Day 1)Population: All participants who received the prednisone challenge and completed dose course post challenge
Participants with idiopathic hypereosinophilic syndromes (IHES) that achieved glucocorticoid responsiveness, suboptimal responsiveness, or without response.
Outcome measures
| Measure |
Steroid Challenge - Responders
n=11 Participants
A single oral dose of prednisone (1 mg/kg rounded to the nearest 5mg) was administered to all participants. The following day, participant began glucocorticoid therapy of prednisone 30 mg oral daily for one week followed by a standardized taper until a minimally effective dose was achieved or participant tapered to prednisone 5 mg oral daily.
|
Steroid Challenge - Non-responders
n=5 Participants
A single oral dose of prednisone (1 mg/kg rounded to the nearest 5mg) was administered to all participants. The following day, participant began glucocorticoid therapy of prednisone 30 mg oral daily for one week followed by a standardized taper until a minimally effective dose was achieved or participant tapered to prednisone 5 mg oral daily.
|
Steroid Challenge - Glucocorticoid Unresponsiveness
n=7 Participants
Participants with hypereosinophilic syndromes (HES) that were not responsive to prednisone defined as no decline of absolute eosinophil count (AEC) below 1000/mm\^3 after one week of prednisone 30 mg daily, followed by 2 days of prednisone 60 mg daily
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|---|---|---|---|
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Participants With Glucocorticoid Responsiveness - IHES Variant
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6 Participants
|
2 Participants
|
2 Participants
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SECONDARY outcome
Timeframe: Baseline (Day 1)Population: All participants who received the prednisone challenge and completed dose course post challenge
Participants with lymphoid hypereosinophilic syndromes (LHES) that achieved glucocorticoid responsiveness, suboptimal responsiveness, or without response.
Outcome measures
| Measure |
Steroid Challenge - Responders
n=11 Participants
A single oral dose of prednisone (1 mg/kg rounded to the nearest 5mg) was administered to all participants. The following day, participant began glucocorticoid therapy of prednisone 30 mg oral daily for one week followed by a standardized taper until a minimally effective dose was achieved or participant tapered to prednisone 5 mg oral daily.
|
Steroid Challenge - Non-responders
n=5 Participants
A single oral dose of prednisone (1 mg/kg rounded to the nearest 5mg) was administered to all participants. The following day, participant began glucocorticoid therapy of prednisone 30 mg oral daily for one week followed by a standardized taper until a minimally effective dose was achieved or participant tapered to prednisone 5 mg oral daily.
|
Steroid Challenge - Glucocorticoid Unresponsiveness
n=7 Participants
Participants with hypereosinophilic syndromes (HES) that were not responsive to prednisone defined as no decline of absolute eosinophil count (AEC) below 1000/mm\^3 after one week of prednisone 30 mg daily, followed by 2 days of prednisone 60 mg daily
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|---|---|---|---|
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Participants With Glucocorticoid Responsiveness - LHES Variant
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2 Participants
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1 Participants
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2 Participants
|
SECONDARY outcome
Timeframe: Baseline (Day 1)Population: All participants who received the prednisone challenge and completed dose course post challenge
Participants with myeloid hypereosinophilic syndromes (MHES) that achieved glucocorticoid responsiveness, suboptimal responsiveness, or without response.
Outcome measures
| Measure |
Steroid Challenge - Responders
n=11 Participants
A single oral dose of prednisone (1 mg/kg rounded to the nearest 5mg) was administered to all participants. The following day, participant began glucocorticoid therapy of prednisone 30 mg oral daily for one week followed by a standardized taper until a minimally effective dose was achieved or participant tapered to prednisone 5 mg oral daily.
|
Steroid Challenge - Non-responders
n=5 Participants
A single oral dose of prednisone (1 mg/kg rounded to the nearest 5mg) was administered to all participants. The following day, participant began glucocorticoid therapy of prednisone 30 mg oral daily for one week followed by a standardized taper until a minimally effective dose was achieved or participant tapered to prednisone 5 mg oral daily.
|
Steroid Challenge - Glucocorticoid Unresponsiveness
n=7 Participants
Participants with hypereosinophilic syndromes (HES) that were not responsive to prednisone defined as no decline of absolute eosinophil count (AEC) below 1000/mm\^3 after one week of prednisone 30 mg daily, followed by 2 days of prednisone 60 mg daily
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|---|---|---|---|
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Participants With Glucocorticoid Responsiveness - MHES Variant
|
0 Participants
|
0 Participants
|
1 Participants
|
SECONDARY outcome
Timeframe: Baseline (Day 1)Population: All participants who received the prednisone challenge and completed dose course post challenge
Participants with overlap hypereosinophilic syndromes that achieved glucocorticoid responsiveness, suboptimal responsiveness, or without response.
Outcome measures
| Measure |
Steroid Challenge - Responders
n=11 Participants
A single oral dose of prednisone (1 mg/kg rounded to the nearest 5mg) was administered to all participants. The following day, participant began glucocorticoid therapy of prednisone 30 mg oral daily for one week followed by a standardized taper until a minimally effective dose was achieved or participant tapered to prednisone 5 mg oral daily.
|
Steroid Challenge - Non-responders
n=5 Participants
A single oral dose of prednisone (1 mg/kg rounded to the nearest 5mg) was administered to all participants. The following day, participant began glucocorticoid therapy of prednisone 30 mg oral daily for one week followed by a standardized taper until a minimally effective dose was achieved or participant tapered to prednisone 5 mg oral daily.
|
Steroid Challenge - Glucocorticoid Unresponsiveness
n=7 Participants
Participants with hypereosinophilic syndromes (HES) that were not responsive to prednisone defined as no decline of absolute eosinophil count (AEC) below 1000/mm\^3 after one week of prednisone 30 mg daily, followed by 2 days of prednisone 60 mg daily
|
|---|---|---|---|
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Participants With Glucocorticoid Responsiveness - Overlap HES Subtypes
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3 Participants
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2 Participants
|
1 Participants
|
SECONDARY outcome
Timeframe: Baseline (Day 1)Population: All participants who received the prednisone challenge and completed dose course post challenge
Mean baseline immunoglobulin E (IgE) level in participants prior to initiation of glucocorticoid
Outcome measures
| Measure |
Steroid Challenge - Responders
n=11 Participants
A single oral dose of prednisone (1 mg/kg rounded to the nearest 5mg) was administered to all participants. The following day, participant began glucocorticoid therapy of prednisone 30 mg oral daily for one week followed by a standardized taper until a minimally effective dose was achieved or participant tapered to prednisone 5 mg oral daily.
|
Steroid Challenge - Non-responders
n=5 Participants
A single oral dose of prednisone (1 mg/kg rounded to the nearest 5mg) was administered to all participants. The following day, participant began glucocorticoid therapy of prednisone 30 mg oral daily for one week followed by a standardized taper until a minimally effective dose was achieved or participant tapered to prednisone 5 mg oral daily.
|
Steroid Challenge - Glucocorticoid Unresponsiveness
n=7 Participants
Participants with hypereosinophilic syndromes (HES) that were not responsive to prednisone defined as no decline of absolute eosinophil count (AEC) below 1000/mm\^3 after one week of prednisone 30 mg daily, followed by 2 days of prednisone 60 mg daily
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|---|---|---|---|
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Mean Baseline IgE Level
|
285.2 IU/mL
Interval 50.74 to 1603.0
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1171.5 IU/mL
Interval 69.23 to 19824.0
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434.8 IU/mL
Interval 27.49 to 6877.0
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SECONDARY outcome
Timeframe: Baseline (Day 1)Population: All participants who received the prednisone challenge and completed dose course post challenge
Mean baseline absolute eosinophil count in participants prior to initiation of glucocorticoids
Outcome measures
| Measure |
Steroid Challenge - Responders
n=11 Participants
A single oral dose of prednisone (1 mg/kg rounded to the nearest 5mg) was administered to all participants. The following day, participant began glucocorticoid therapy of prednisone 30 mg oral daily for one week followed by a standardized taper until a minimally effective dose was achieved or participant tapered to prednisone 5 mg oral daily.
|
Steroid Challenge - Non-responders
n=5 Participants
A single oral dose of prednisone (1 mg/kg rounded to the nearest 5mg) was administered to all participants. The following day, participant began glucocorticoid therapy of prednisone 30 mg oral daily for one week followed by a standardized taper until a minimally effective dose was achieved or participant tapered to prednisone 5 mg oral daily.
|
Steroid Challenge - Glucocorticoid Unresponsiveness
n=7 Participants
Participants with hypereosinophilic syndromes (HES) that were not responsive to prednisone defined as no decline of absolute eosinophil count (AEC) below 1000/mm\^3 after one week of prednisone 30 mg daily, followed by 2 days of prednisone 60 mg daily
|
|---|---|---|---|
|
Mean Baseline Absolute Eosinophil Count
|
2.81 10^3 cells per microliter
Interval 2.097 to 3.771
|
3.85 10^3 cells per microliter
Interval 2.036 to 7.313
|
5.90 10^3 cells per microliter
Interval 2.772 to 12.59
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SECONDARY outcome
Timeframe: Baseline (Day 1)Population: All participants who received the prednisone challenge and completed dose course post challenge
Cardiac involvement in participants with hypereosinophilic syndromes (HES)
Outcome measures
| Measure |
Steroid Challenge - Responders
n=11 Participants
A single oral dose of prednisone (1 mg/kg rounded to the nearest 5mg) was administered to all participants. The following day, participant began glucocorticoid therapy of prednisone 30 mg oral daily for one week followed by a standardized taper until a minimally effective dose was achieved or participant tapered to prednisone 5 mg oral daily.
|
Steroid Challenge - Non-responders
n=5 Participants
A single oral dose of prednisone (1 mg/kg rounded to the nearest 5mg) was administered to all participants. The following day, participant began glucocorticoid therapy of prednisone 30 mg oral daily for one week followed by a standardized taper until a minimally effective dose was achieved or participant tapered to prednisone 5 mg oral daily.
|
Steroid Challenge - Glucocorticoid Unresponsiveness
n=7 Participants
Participants with hypereosinophilic syndromes (HES) that were not responsive to prednisone defined as no decline of absolute eosinophil count (AEC) below 1000/mm\^3 after one week of prednisone 30 mg daily, followed by 2 days of prednisone 60 mg daily
|
|---|---|---|---|
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Participants With Glucocorticoid Responsiveness - Cardiac Involvement
|
1 Participants
|
0 Participants
|
4 Participants
|
SECONDARY outcome
Timeframe: Baseline (Day 1)Population: All participants who received the prednisone challenge and completed dose course post challenge
Pulmonary involvement in participants with hypereosinophilic syndromes (HES)
Outcome measures
| Measure |
Steroid Challenge - Responders
n=11 Participants
A single oral dose of prednisone (1 mg/kg rounded to the nearest 5mg) was administered to all participants. The following day, participant began glucocorticoid therapy of prednisone 30 mg oral daily for one week followed by a standardized taper until a minimally effective dose was achieved or participant tapered to prednisone 5 mg oral daily.
|
Steroid Challenge - Non-responders
n=5 Participants
A single oral dose of prednisone (1 mg/kg rounded to the nearest 5mg) was administered to all participants. The following day, participant began glucocorticoid therapy of prednisone 30 mg oral daily for one week followed by a standardized taper until a minimally effective dose was achieved or participant tapered to prednisone 5 mg oral daily.
|
Steroid Challenge - Glucocorticoid Unresponsiveness
n=7 Participants
Participants with hypereosinophilic syndromes (HES) that were not responsive to prednisone defined as no decline of absolute eosinophil count (AEC) below 1000/mm\^3 after one week of prednisone 30 mg daily, followed by 2 days of prednisone 60 mg daily
|
|---|---|---|---|
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Participants With Glucocorticoid Responsiveness - Pulmonary Involvement
|
4 Participants
|
3 Participants
|
3 Participants
|
Adverse Events
Steroid Challenge
Serious events: 0 serious events
Other events: 7 other events
Deaths: 0 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Steroid Challenge
n=23 participants at risk
A single oral dose of prednisone (1 mg/kg rounded to the nearest 5mg) was administered to all participants. The following day, participant began glucocorticoid therapy of prednisone 30 mg oral daily for one week followed by a standardized taper until a minimally effective dose was achieved or participant tapered to prednisone 5 mg oral daily.
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|---|---|
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Eye disorders
Glaucoma
|
4.3%
1/23 • 20 weeks
|
|
Eye disorders
Vitreous floaters
|
4.3%
1/23 • 20 weeks
|
|
General disorders
Swelling face
|
4.3%
1/23 • 20 weeks
|
|
General disorders
Thirst
|
4.3%
1/23 • 20 weeks
|
|
Investigations
Weight increased
|
8.7%
2/23 • 20 weeks
|
|
Metabolism and nutrition disorders
Fluid retention
|
4.3%
1/23 • 20 weeks
|
|
Metabolism and nutrition disorders
Increased appetite
|
8.7%
2/23 • 20 weeks
|
|
Nervous system disorders
Dizziness
|
4.3%
1/23 • 20 weeks
|
|
Nervous system disorders
Headache
|
4.3%
1/23 • 20 weeks
|
|
Psychiatric disorders
Insomnia
|
4.3%
1/23 • 20 weeks
|
|
Renal and urinary disorders
Polyuria
|
4.3%
1/23 • 20 weeks
|
|
Skin and subcutaneous tissue disorders
Dermatitis acneiform
|
4.3%
1/23 • 20 weeks
|
Additional Information
Paneez Khoury
National Institute of Allergy and Infectious Diseases
Phone: +1 301 402 3773
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place