Trial Outcomes & Findings for Ruxolitinib Pre-, During- and Post-HSCT for Patients With Primary or Secondary Myelofibrosis. (NCT NCT03427866)
NCT ID: NCT03427866
Last Updated: 2026-07-09
Results Overview
The number of participants surviving after one year that have not experienced graft-versus host disease (GVHD) or relapse (GRFS rate)
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
44 participants
Primary outcome timeframe
1 year
Results posted on
2026-07-09
Participant Flow
44 patients with MF were enrolled. 1 withdrew.
There was no pre-assignment.
Participant milestones
| Measure |
MF Eligible Pre-HSCT
* Ruxolitinib will be taken orally at a fixed dose twice every day after transplant
* Dosing will be continuous, with a new cycle scheduled to start every 28 days.
* There will be no break in dosing between cycles
* Ruxolitinib can be administered with or without food.
|
|---|---|
|
Overall Study
STARTED
|
43
|
|
Overall Study
COMPLETED
|
43
|
|
Overall Study
NOT COMPLETED
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
there is just one group. there are therefore no differences in the population.
Baseline characteristics by cohort
| Measure |
MF Eligible For-HSCT
n=43 Participants
* Ruxolitinib will be taken orally at a fixed dose twice every day after transplant
* Dosing will be continuous, with a new cycle scheduled to start every 28 days.
* There will be no break in dosing between cycles
* Ruxolitinib can be administered with or without food.
|
|---|---|
|
Age, Continuous
|
66 years
n=20 Participants • there is just one group. there are therefore no differences in the population.
|
|
Sex: Female, Male
Female
|
16 Participants
n=20 Participants
|
|
Sex: Female, Male
Male
|
27 Participants
n=20 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
1 Participants
n=20 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
37 Participants
n=20 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
5 Participants
n=20 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=20 Participants
|
|
Race (NIH/OMB)
Asian
|
1 Participants
n=20 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=20 Participants
|
|
Race (NIH/OMB)
Black or African American
|
1 Participants
n=20 Participants
|
|
Race (NIH/OMB)
White
|
40 Participants
n=20 Participants
|
|
Race (NIH/OMB)
More than one race
|
1 Participants
n=20 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=20 Participants
|
PRIMARY outcome
Timeframe: 1 yearThe number of participants surviving after one year that have not experienced graft-versus host disease (GVHD) or relapse (GRFS rate)
Outcome measures
| Measure |
MF Eligible for HSCT
n=43 Participants
Patients eligible for HSCT with a diagnosis of MF
|
|---|---|
|
GVHD Free and Relapse Free Survival at 1 Year
|
71 percentage of participants
Interval 55.0 to 83.0
|
SECONDARY outcome
Timeframe: 1 and 2 years1 year and 2 year progression free survival
Outcome measures
| Measure |
MF Eligible for HSCT
n=43 Participants
Patients eligible for HSCT with a diagnosis of MF
|
|---|---|
|
Progression Free Survival
1 Year
|
31 Participants
|
|
Progression Free Survival
2 Year
|
15 Participants
|
SECONDARY outcome
Timeframe: 1 year and 2 year1-year and 2-year overall survival
Outcome measures
| Measure |
MF Eligible for HSCT
n=43 Participants
Patients eligible for HSCT with a diagnosis of MF
|
|---|---|
|
Overall Survival
1 Year
|
34 Participants
|
|
Overall Survival
2 Year
|
18 Participants
|
SECONDARY outcome
Timeframe: 6 monthsCumulative incidence of grades II-IV and II-IV acute GVHD at 6 months after HSCT
Outcome measures
| Measure |
MF Eligible for HSCT
n=43 Participants
Patients eligible for HSCT with a diagnosis of MF
|
|---|---|
|
Cumulative Incidence of aGVHD
|
28 percentage of population at risk
Interval 15.0 to 42.0
|
SECONDARY outcome
Timeframe: 2 yearsCumulative incidence of moderate to severe chronic GVHD at 2 years after HSCT
Outcome measures
| Measure |
MF Eligible for HSCT
n=43 Participants
Patients eligible for HSCT with a diagnosis of MF
|
|---|---|
|
Cumulative Incidence of cGVHD
|
14.5 percentage of events
Interval 6.0 to 28.0
|
SECONDARY outcome
Timeframe: 151 daysEngraftment defined as ANC \>500/ugx3 consecutive measurements and platelets of \>20x10e9/L for three consecutive days.
Outcome measures
| Measure |
MF Eligible for HSCT
n=43 Participants
Patients eligible for HSCT with a diagnosis of MF
|
|---|---|
|
Time to Neutrophil and Platelet Engraftment
ANC
|
15 days
Interval 11.0 to 31.0
|
|
Time to Neutrophil and Platelet Engraftment
Platelet
|
25 days
Interval 9.0 to 151.0
|
SECONDARY outcome
Timeframe: 13 cyclesThe amount of time patients remain on ruxolitinib from transplant until discontinuation.
Outcome measures
| Measure |
MF Eligible for HSCT
n=43 Participants
Patients eligible for HSCT with a diagnosis of MF
|
|---|---|
|
Median Time on Ruxolitinib After HSCT as a Measure of Feasibility
|
13 cycles
Interval 2.0 to 13.0
|
SECONDARY outcome
Timeframe: 24 monthsCumulative incidence of non-relapse mortality (NRM) at 24 months
Outcome measures
| Measure |
MF Eligible for HSCT
n=43 Participants
Patients eligible for HSCT with a diagnosis of MF
|
|---|---|
|
Cumulative Incidence of Non-relapse Mortality (NRM)
|
11.7 percentage of population at risk
Interval 4.3 to 24.0
|
Adverse Events
MF Eligible for HSCT
Serious events: 23 serious events
Other events: 38 other events
Deaths: 8 deaths
Serious adverse events
| Measure |
MF Eligible for HSCT
n=43 participants at risk
MF patients eligible for HSCT
|
|---|---|
|
Respiratory, thoracic and mediastinal disorders
Acute hypoxemic respiratory failure
|
2.3%
1/43 • Number of events 2 • 30 days after end of treatment; median follow up was 27 months (range 1 - 64)
Adverse event reporting for this study does not differ from the definition of adverse event and/or serious adverse event from clinicaltrials.gov. Only those Adverse Events that are grade 3, 4, or 5 per CTCAE version 4.03 must be reported in routine study data submissions to the Overall PI on the toxicity case report forms.
|
|
Renal and urinary disorders
Renal failure
|
2.3%
1/43 • 30 days after end of treatment; median follow up was 27 months (range 1 - 64)
Adverse event reporting for this study does not differ from the definition of adverse event and/or serious adverse event from clinicaltrials.gov. Only those Adverse Events that are grade 3, 4, or 5 per CTCAE version 4.03 must be reported in routine study data submissions to the Overall PI on the toxicity case report forms.
|
|
Vascular disorders
Subdural Hematoma
|
2.3%
1/43 • 30 days after end of treatment; median follow up was 27 months (range 1 - 64)
Adverse event reporting for this study does not differ from the definition of adverse event and/or serious adverse event from clinicaltrials.gov. Only those Adverse Events that are grade 3, 4, or 5 per CTCAE version 4.03 must be reported in routine study data submissions to the Overall PI on the toxicity case report forms.
|
|
Blood and lymphatic system disorders
Platelet count decreased
|
2.3%
1/43 • 30 days after end of treatment; median follow up was 27 months (range 1 - 64)
Adverse event reporting for this study does not differ from the definition of adverse event and/or serious adverse event from clinicaltrials.gov. Only those Adverse Events that are grade 3, 4, or 5 per CTCAE version 4.03 must be reported in routine study data submissions to the Overall PI on the toxicity case report forms.
|
|
Infections and infestations
Sepsis
|
2.3%
1/43 • Number of events 4 • 30 days after end of treatment; median follow up was 27 months (range 1 - 64)
Adverse event reporting for this study does not differ from the definition of adverse event and/or serious adverse event from clinicaltrials.gov. Only those Adverse Events that are grade 3, 4, or 5 per CTCAE version 4.03 must be reported in routine study data submissions to the Overall PI on the toxicity case report forms.
|
|
Skin and subcutaneous tissue disorders
Rash maculo-papular
|
2.3%
1/43 • 30 days after end of treatment; median follow up was 27 months (range 1 - 64)
Adverse event reporting for this study does not differ from the definition of adverse event and/or serious adverse event from clinicaltrials.gov. Only those Adverse Events that are grade 3, 4, or 5 per CTCAE version 4.03 must be reported in routine study data submissions to the Overall PI on the toxicity case report forms.
|
|
General disorders
Fever
|
2.3%
1/43 • Number of events 5 • 30 days after end of treatment; median follow up was 27 months (range 1 - 64)
Adverse event reporting for this study does not differ from the definition of adverse event and/or serious adverse event from clinicaltrials.gov. Only those Adverse Events that are grade 3, 4, or 5 per CTCAE version 4.03 must be reported in routine study data submissions to the Overall PI on the toxicity case report forms.
|
|
Renal and urinary disorders
Acute Kidney Injury
|
2.3%
1/43 • 30 days after end of treatment; median follow up was 27 months (range 1 - 64)
Adverse event reporting for this study does not differ from the definition of adverse event and/or serious adverse event from clinicaltrials.gov. Only those Adverse Events that are grade 3, 4, or 5 per CTCAE version 4.03 must be reported in routine study data submissions to the Overall PI on the toxicity case report forms.
|
|
Gastrointestinal disorders
Abdominal Pain
|
2.3%
1/43 • Number of events 3 • 30 days after end of treatment; median follow up was 27 months (range 1 - 64)
Adverse event reporting for this study does not differ from the definition of adverse event and/or serious adverse event from clinicaltrials.gov. Only those Adverse Events that are grade 3, 4, or 5 per CTCAE version 4.03 must be reported in routine study data submissions to the Overall PI on the toxicity case report forms.
|
|
Musculoskeletal and connective tissue disorders
Back Pain
|
2.3%
1/43 • Number of events 3 • 30 days after end of treatment; median follow up was 27 months (range 1 - 64)
Adverse event reporting for this study does not differ from the definition of adverse event and/or serious adverse event from clinicaltrials.gov. Only those Adverse Events that are grade 3, 4, or 5 per CTCAE version 4.03 must be reported in routine study data submissions to the Overall PI on the toxicity case report forms.
|
|
Infections and infestations
Infection
|
2.3%
1/43 • Number of events 4 • 30 days after end of treatment; median follow up was 27 months (range 1 - 64)
Adverse event reporting for this study does not differ from the definition of adverse event and/or serious adverse event from clinicaltrials.gov. Only those Adverse Events that are grade 3, 4, or 5 per CTCAE version 4.03 must be reported in routine study data submissions to the Overall PI on the toxicity case report forms.
|
|
Blood and lymphatic system disorders
Febrile Neutropenia
|
2.3%
1/43 • Number of events 3 • 30 days after end of treatment; median follow up was 27 months (range 1 - 64)
Adverse event reporting for this study does not differ from the definition of adverse event and/or serious adverse event from clinicaltrials.gov. Only those Adverse Events that are grade 3, 4, or 5 per CTCAE version 4.03 must be reported in routine study data submissions to the Overall PI on the toxicity case report forms.
|
|
Nervous system disorders
Encephalopathy
|
2.3%
1/43 • 30 days after end of treatment; median follow up was 27 months (range 1 - 64)
Adverse event reporting for this study does not differ from the definition of adverse event and/or serious adverse event from clinicaltrials.gov. Only those Adverse Events that are grade 3, 4, or 5 per CTCAE version 4.03 must be reported in routine study data submissions to the Overall PI on the toxicity case report forms.
|
|
Respiratory, thoracic and mediastinal disorders
Hypoxia
|
2.3%
1/43 • Number of events 3 • 30 days after end of treatment; median follow up was 27 months (range 1 - 64)
Adverse event reporting for this study does not differ from the definition of adverse event and/or serious adverse event from clinicaltrials.gov. Only those Adverse Events that are grade 3, 4, or 5 per CTCAE version 4.03 must be reported in routine study data submissions to the Overall PI on the toxicity case report forms.
|
|
Respiratory, thoracic and mediastinal disorders
Mucositis
|
2.3%
1/43 • 30 days after end of treatment; median follow up was 27 months (range 1 - 64)
Adverse event reporting for this study does not differ from the definition of adverse event and/or serious adverse event from clinicaltrials.gov. Only those Adverse Events that are grade 3, 4, or 5 per CTCAE version 4.03 must be reported in routine study data submissions to the Overall PI on the toxicity case report forms.
|
|
Vascular disorders
Hypertension
|
2.3%
1/43 • 30 days after end of treatment; median follow up was 27 months (range 1 - 64)
Adverse event reporting for this study does not differ from the definition of adverse event and/or serious adverse event from clinicaltrials.gov. Only those Adverse Events that are grade 3, 4, or 5 per CTCAE version 4.03 must be reported in routine study data submissions to the Overall PI on the toxicity case report forms.
|
|
Gastrointestinal disorders
Ileus
|
2.3%
1/43 • 30 days after end of treatment; median follow up was 27 months (range 1 - 64)
Adverse event reporting for this study does not differ from the definition of adverse event and/or serious adverse event from clinicaltrials.gov. Only those Adverse Events that are grade 3, 4, or 5 per CTCAE version 4.03 must be reported in routine study data submissions to the Overall PI on the toxicity case report forms.
|
|
Infections and infestations
Urinary Tract Infection
|
2.3%
1/43 • 30 days after end of treatment; median follow up was 27 months (range 1 - 64)
Adverse event reporting for this study does not differ from the definition of adverse event and/or serious adverse event from clinicaltrials.gov. Only those Adverse Events that are grade 3, 4, or 5 per CTCAE version 4.03 must be reported in routine study data submissions to the Overall PI on the toxicity case report forms.
|
|
Nervous system disorders
Syncope
|
2.3%
1/43 • 30 days after end of treatment; median follow up was 27 months (range 1 - 64)
Adverse event reporting for this study does not differ from the definition of adverse event and/or serious adverse event from clinicaltrials.gov. Only those Adverse Events that are grade 3, 4, or 5 per CTCAE version 4.03 must be reported in routine study data submissions to the Overall PI on the toxicity case report forms.
|
|
Metabolism and nutrition disorders
Hypertriglyceridemia
|
2.3%
1/43 • 30 days after end of treatment; median follow up was 27 months (range 1 - 64)
Adverse event reporting for this study does not differ from the definition of adverse event and/or serious adverse event from clinicaltrials.gov. Only those Adverse Events that are grade 3, 4, or 5 per CTCAE version 4.03 must be reported in routine study data submissions to the Overall PI on the toxicity case report forms.
|
|
Gastrointestinal disorders
Nausea
|
2.3%
1/43 • 30 days after end of treatment; median follow up was 27 months (range 1 - 64)
Adverse event reporting for this study does not differ from the definition of adverse event and/or serious adverse event from clinicaltrials.gov. Only those Adverse Events that are grade 3, 4, or 5 per CTCAE version 4.03 must be reported in routine study data submissions to the Overall PI on the toxicity case report forms.
|
|
Gastrointestinal disorders
Vomiting
|
2.3%
1/43 • 30 days after end of treatment; median follow up was 27 months (range 1 - 64)
Adverse event reporting for this study does not differ from the definition of adverse event and/or serious adverse event from clinicaltrials.gov. Only those Adverse Events that are grade 3, 4, or 5 per CTCAE version 4.03 must be reported in routine study data submissions to the Overall PI on the toxicity case report forms.
|
|
Nervous system disorders
Intracranial hemorrhage
|
2.3%
1/43 • 30 days after end of treatment; median follow up was 27 months (range 1 - 64)
Adverse event reporting for this study does not differ from the definition of adverse event and/or serious adverse event from clinicaltrials.gov. Only those Adverse Events that are grade 3, 4, or 5 per CTCAE version 4.03 must be reported in routine study data submissions to the Overall PI on the toxicity case report forms.
|
Other adverse events
| Measure |
MF Eligible for HSCT
n=43 participants at risk
MF patients eligible for HSCT
|
|---|---|
|
Blood and lymphatic system disorders
Anemia
|
44.2%
19/43 • 30 days after end of treatment; median follow up was 27 months (range 1 - 64)
Adverse event reporting for this study does not differ from the definition of adverse event and/or serious adverse event from clinicaltrials.gov. Only those Adverse Events that are grade 3, 4, or 5 per CTCAE version 4.03 must be reported in routine study data submissions to the Overall PI on the toxicity case report forms.
|
|
Investigations
White blood cell decreased
|
37.2%
16/43 • 30 days after end of treatment; median follow up was 27 months (range 1 - 64)
Adverse event reporting for this study does not differ from the definition of adverse event and/or serious adverse event from clinicaltrials.gov. Only those Adverse Events that are grade 3, 4, or 5 per CTCAE version 4.03 must be reported in routine study data submissions to the Overall PI on the toxicity case report forms.
|
|
Investigations
Lymphocyte count decreased
|
16.3%
7/43 • 30 days after end of treatment; median follow up was 27 months (range 1 - 64)
Adverse event reporting for this study does not differ from the definition of adverse event and/or serious adverse event from clinicaltrials.gov. Only those Adverse Events that are grade 3, 4, or 5 per CTCAE version 4.03 must be reported in routine study data submissions to the Overall PI on the toxicity case report forms.
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place