NCT01623167 · OUTCOME

Rate of Complete Hematologic Response

Eltrombopag With Standard Immunosuppression for Severe Aplastic Anemia · Source last updated 2026-04-13

Results reported by the registry submitting party. Population, time horizon, units and missing values must be read together.

Measure: COUNT_OF_PARTICIPANTS
Unit: Participants
Interval / dispersion: Not reported
Time frame: 6 months

What was measured

Rate of complete hematologic response at six months for cohorts 1, 2 and 3.

Groups in this outcome

hATG, CsA, EPAG Day 14 to Month 6

Receive horse ATG days 1- 4, receive CsA day 1 to month 6, and receive eltrombopag day 14 to month 6 hATG/CsA /eltrombopag -Cohort 1: hATG (standard of care) administered for 4 days, CsA (standard of care) administered starting day 1 for 6 months, eltrombopag (experimental) administered Day 14 to month 6

hATG, CsA, EPAG Day 14 to Month 3

Receive horse ATG days 1- 4, receive CsA day 1 to month 6, and receive eltrombopag day 14 to month 3 hATG/CsA /eltrombopag Cohort 2: hATG (standard of care) administered for 4 days, CsA (standard of care) administered starting day 1 for 6 months, eltrombopag (experimental) administered Day 14 to month 3

hATG, CsA (Dose Reduced), EPAG Day 1 to Month 6

Receive horse ATG days 1- 4, receive CsA day 1 to month 6 at higher dose, then reduced dose for 18months, and receive eltrombopag day 1 to month 6 hATG/CsA /eltrombopag Cohort 3: hATG (standard of care) administered for 4 days, CsA (standard of care) administered starting day 1 for 6 months at higher dose, then reduced dose for 18 months, eltrombopag (experimental) administered Day 1 to month 6

Group identifiers and denominators belong to this outcome only. They may differ in another outcome or in safety reporting.

Analysis denominator · Participants

Group	Source count
hATG, CsA, EPAG Day 14 to Month 6	30
hATG, CsA, EPAG Day 14 to Month 3	31
hATG, CsA (Dose Reduced), EPAG Day 1 to Month 6	31

Reported measurements

Source class 1

No Response

Values in Participants · Interval/dispersion: Not reported
Group	Value	Spread	Lower	Upper	Comment
hATG, CsA, EPAG Day 14 to Month 6	3	Not reported	Not reported	Not reported	Not reported
hATG, CsA, EPAG Day 14 to Month 3	2	Not reported	Not reported	Not reported	Not reported
hATG, CsA (Dose Reduced), EPAG Day 1 to Month 6	1	Not reported	Not reported	Not reported	Not reported

Partial Response

Values in Participants · Interval/dispersion: Not reported
Group	Value	Spread	Lower	Upper	Comment
hATG, CsA, EPAG Day 14 to Month 6	14	Not reported	Not reported	Not reported	Not reported
hATG, CsA, EPAG Day 14 to Month 3	19	Not reported	Not reported	Not reported	Not reported
hATG, CsA (Dose Reduced), EPAG Day 1 to Month 6	11	Not reported	Not reported	Not reported	Not reported

Complete Response

Values in Participants · Interval/dispersion: Not reported
Group	Value	Spread	Lower	Upper	Comment
hATG, CsA, EPAG Day 14 to Month 6	10	Not reported	Not reported	Not reported	Not reported
hATG, CsA, EPAG Day 14 to Month 3	8	Not reported	Not reported	Not reported	Not reported
hATG, CsA (Dose Reduced), EPAG Day 1 to Month 6	18	Not reported	Not reported	Not reported	Not reported

Off study/Not Evaluable

Values in Participants · Interval/dispersion: Not reported
Group	Value	Spread	Lower	Upper	Comment
hATG, CsA, EPAG Day 14 to Month 6	3	Not reported	Not reported	Not reported	Not reported
hATG, CsA, EPAG Day 14 to Month 3	2	Not reported	Not reported	Not reported	Not reported
hATG, CsA (Dose Reduced), EPAG Day 1 to Month 6	1	Not reported	Not reported	Not reported	Not reported

Complete source fields

classes

categories
measurements
group Id
OG000
value
3
group Id
OG001
value
2
group Id
OG002
value
1
title
No Response
measurements
group Id
OG000
value
14
group Id
OG001
value
19
group Id
OG002
value
11
title
Partial Response
measurements
group Id
OG000
value
10
group Id
OG001
value
8
group Id
OG002
value
18
title
Complete Response
measurements
group Id
OG000
value
3
group Id
OG001
value
2
group Id
OG002
value
1
title
Off study/Not Evaluable

denoms

counts
group Id
OG000
value
30
group Id
OG001
value
31
group Id
OG002
value
31
units
Participants

description

Rate of complete hematologic response at six months for cohorts 1, 2 and 3.

groups

description
Receive horse ATG days 1- 4, receive CsA day 1 to month 6, and receive eltrombopag day 14 to month 6 hATG/CsA /eltrombopag -Cohort 1: hATG (standard of care) administered for 4 days, CsA (standard of care) administered starting day 1 for 6 months, eltrombopag (experimental) administered Day 14 to month 6
id
OG000
title
hATG, CsA, EPAG Day 14 to Month 6
description
Receive horse ATG days 1- 4, receive CsA day 1 to month 6, and receive eltrombopag day 14 to month 3 hATG/CsA /eltrombopag Cohort 2: hATG (standard of care) administered for 4 days, CsA (standard of care) administered starting day 1 for 6 months, eltrombopag (experimental) administered Day 14 to month 3
id
OG001
title
hATG, CsA, EPAG Day 14 to Month 3
description
Receive horse ATG days 1- 4, receive CsA day 1 to month 6 at higher dose, then reduced dose for 18months, and receive eltrombopag day 1 to month 6 hATG/CsA /eltrombopag Cohort 3: hATG (standard of care) administered for 4 days, CsA (standard of care) administered starting day 1 for 6 months at higher dose, then reduced dose for 18 months, eltrombopag (experimental) administered Day 1 to month 6
id
OG002
title
hATG, CsA (Dose Reduced), EPAG Day 1 to Month 6

param Type

COUNT_OF_PARTICIPANTS

reporting Status

POSTED

time Frame

6 months

title

Rate of Complete Hematologic Response

type

PRIMARY

unit Of Measure

Participants

Download exact source JSON → · Snapshot ctgov-results-191f4516d4760045304b24a0

Preserved source evidence · Not medical advice