Trial Outcomes & Findings for Ruxolitinib Prior to Transplant in Patients With Myelofibrosis (NCT NCT01790295)
NCT ID: NCT01790295
Last Updated: 2019-03-20
Results Overview
The feasibility of combining Ruxolitinib (INC424) with a Reduced intensity conditioning (RIC) regimen likely to produce success post transplantation, success being defined as patient being alive, and without graft failure at day 100-post allogeneic stem cell transplantation (in patients who receive (a) related donor transplant and in those who receive (b) an unrelated donor transplant.
Recruitment status
TERMINATED
Study phase
PHASE2
Target enrollment
21 participants
Primary outcome timeframe
Day 100-post allogeneic stem cell transplantation
Results posted on
2019-03-20
Participant Flow
Recruitment began in February 2013, with first enrollment in November 2013.
Participant milestones
| Measure |
Ruxolitinib Pre- Hematopoietic Cell Transplantation (HCT)
Ruxolitinib Pre- Hematopoietic cell transplantation (HCT) in Patients With Myelofibrosis.
Ruxolitinib (INC424) tablets started 60 days (day -65) prior to start of conditioning chemotherapy. The starting dose of Ruxolitinib was determined according to baseline platelet count and modified according to platelet count at follow-up. The drug was given in the maximum tolerated dose as defined in the protocol for 56 days, followed by 4 days of taper, and stopped completely at the planned start of conditioning therapy (starting on day -5) i.e. 5 days prior to stem cell infusion. The drug was supplied as 5 mg tablets.
|
|---|---|
|
Overall Study
STARTED
|
21
|
|
Overall Study
COMPLETED
|
10
|
|
Overall Study
NOT COMPLETED
|
11
|
Reasons for withdrawal
| Measure |
Ruxolitinib Pre- Hematopoietic Cell Transplantation (HCT)
Ruxolitinib Pre- Hematopoietic cell transplantation (HCT) in Patients With Myelofibrosis.
Ruxolitinib (INC424) tablets started 60 days (day -65) prior to start of conditioning chemotherapy. The starting dose of Ruxolitinib was determined according to baseline platelet count and modified according to platelet count at follow-up. The drug was given in the maximum tolerated dose as defined in the protocol for 56 days, followed by 4 days of taper, and stopped completely at the planned start of conditioning therapy (starting on day -5) i.e. 5 days prior to stem cell infusion. The drug was supplied as 5 mg tablets.
|
|---|---|
|
Overall Study
Adverse Event
|
1
|
|
Overall Study
Death
|
4
|
|
Overall Study
Lack of Efficacy
|
2
|
|
Overall Study
Study closure
|
4
|
Baseline Characteristics
Ruxolitinib Prior to Transplant in Patients With Myelofibrosis
Baseline characteristics by cohort
| Measure |
Ruxolitinib Pre- Hematopoietic Cell Transplantation (HCT)
n=21 Participants
Ruxolitinib Pre- Hematopoietic cell transplantation (HCT) in Patients With Myelofibrosis
Ruxolitinib (INC424) tablets started 60 days (day -65) prior to start of conditioning chemotherapy. The starting dose of Ruxolitinib was determined according to baseline platelet count and modified according to platelet count at follow-up. The drug was given in the maximum tolerated dose as defined in the protocol for 56 days, followed by 4 days of taper, and stopped completely at the planned start of conditioning therapy (starting on day -5) i.e. 5 days prior to stem cell infusion. The drug was supplied as 5 mg tablets.
|
|---|---|
|
Age, Continuous
|
59 years
n=5 Participants
|
|
Sex: Female, Male
Female
|
11 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
10 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
1 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
|
19 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
|
Diagnosis
Primary myelofibrosis
|
10 Participants
n=5 Participants
|
|
Diagnosis
Post-PV myelofibrosis
|
3 Participants
n=5 Participants
|
|
Diagnosis
Post-ET myelofibrosis
|
8 Participants
n=5 Participants
|
|
Donor type
Related donor
|
7 Participants
n=5 Participants
|
|
Donor type
Unrelated donor
|
14 Participants
n=5 Participants
|
|
ECOG status
0
|
9 Participants
n=5 Participants
|
|
ECOG status
1
|
12 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Day 100-post allogeneic stem cell transplantationThe feasibility of combining Ruxolitinib (INC424) with a Reduced intensity conditioning (RIC) regimen likely to produce success post transplantation, success being defined as patient being alive, and without graft failure at day 100-post allogeneic stem cell transplantation (in patients who receive (a) related donor transplant and in those who receive (b) an unrelated donor transplant.
Outcome measures
| Measure |
Ruxolitinib Pre- Hematopoietic Cell Transplantation (HCT)
n=21 Participants
Ruxolitinib Pre- Hematopoietic cell transplantation (HCT) in Patients With Myelofibrosis
Ruxolitinib (INC424) tablets started 60 days (day -65) prior to start of conditioning chemotherapy. The starting dose of Ruxolitinib was determined according to baseline platelet count and modified according to platelet count at follow-up. The drug was given in the maximum tolerated dose as defined in the protocol for 56 days, followed by 4 days of taper, and stopped completely at the planned start of conditioning therapy (starting on day -5) i.e. 5 days prior to stem cell infusion. The drug was supplied as 5 mg tablets.
|
|---|---|
|
Percent of Participants With 100-day Survival Without Graft Failure
|
74 percentage of participants
Interval 48.0 to 88.0
|
SECONDARY outcome
Timeframe: up to 4 yearsNeutrophil recovery will be defined as first of the three consecutive days with neutrophil count ≥0.5 x 109/l.
Outcome measures
| Measure |
Ruxolitinib Pre- Hematopoietic Cell Transplantation (HCT)
n=21 Participants
Ruxolitinib Pre- Hematopoietic cell transplantation (HCT) in Patients With Myelofibrosis
Ruxolitinib (INC424) tablets started 60 days (day -65) prior to start of conditioning chemotherapy. The starting dose of Ruxolitinib was determined according to baseline platelet count and modified according to platelet count at follow-up. The drug was given in the maximum tolerated dose as defined in the protocol for 56 days, followed by 4 days of taper, and stopped completely at the planned start of conditioning therapy (starting on day -5) i.e. 5 days prior to stem cell infusion. The drug was supplied as 5 mg tablets.
|
|---|---|
|
Time to Neutrophil Recovery
|
23 days
Interval 13.0 to 31.0
|
SECONDARY outcome
Timeframe: up to 4 yearsPlatelet recovery will be defined as first of the 7 days with platelet count ≥20 x 109/l, without platelet transfusion support and both maintained for 30 days without transfusion support or myeloid cytokine support.
Outcome measures
| Measure |
Ruxolitinib Pre- Hematopoietic Cell Transplantation (HCT)
n=21 Participants
Ruxolitinib Pre- Hematopoietic cell transplantation (HCT) in Patients With Myelofibrosis
Ruxolitinib (INC424) tablets started 60 days (day -65) prior to start of conditioning chemotherapy. The starting dose of Ruxolitinib was determined according to baseline platelet count and modified according to platelet count at follow-up. The drug was given in the maximum tolerated dose as defined in the protocol for 56 days, followed by 4 days of taper, and stopped completely at the planned start of conditioning therapy (starting on day -5) i.e. 5 days prior to stem cell infusion. The drug was supplied as 5 mg tablets.
|
|---|---|
|
Platelet Recovery
|
30 days
Interval 18.0 to 57.0
|
SECONDARY outcome
Timeframe: 100 daysNRM will be defined as death in first 30 days due to any cause, and subsequently death due to any cause without the recurrence or progression of myelofibrosis. Cumulative incidence of NRM will be calculated taking relapse/progression as competing event.
Outcome measures
| Measure |
Ruxolitinib Pre- Hematopoietic Cell Transplantation (HCT)
n=21 Participants
Ruxolitinib Pre- Hematopoietic cell transplantation (HCT) in Patients With Myelofibrosis
Ruxolitinib (INC424) tablets started 60 days (day -65) prior to start of conditioning chemotherapy. The starting dose of Ruxolitinib was determined according to baseline platelet count and modified according to platelet count at follow-up. The drug was given in the maximum tolerated dose as defined in the protocol for 56 days, followed by 4 days of taper, and stopped completely at the planned start of conditioning therapy (starting on day -5) i.e. 5 days prior to stem cell infusion. The drug was supplied as 5 mg tablets.
|
|---|---|
|
Percent of Participants With Non-relapse Mortality (NRM)
|
16 percentage of participants
Interval 5.0 to 46.0
|
SECONDARY outcome
Timeframe: 1-year post transplantNRM will be defined as death in first 30 days due to any cause, and subsequently death due to any cause without the recurrence or progression of myelofibrosis. Cumulative incidence of NRM will be calculated taking relapse/progression as competing event.
Outcome measures
| Measure |
Ruxolitinib Pre- Hematopoietic Cell Transplantation (HCT)
n=21 Participants
Ruxolitinib Pre- Hematopoietic cell transplantation (HCT) in Patients With Myelofibrosis
Ruxolitinib (INC424) tablets started 60 days (day -65) prior to start of conditioning chemotherapy. The starting dose of Ruxolitinib was determined according to baseline platelet count and modified according to platelet count at follow-up. The drug was given in the maximum tolerated dose as defined in the protocol for 56 days, followed by 4 days of taper, and stopped completely at the planned start of conditioning therapy (starting on day -5) i.e. 5 days prior to stem cell infusion. The drug was supplied as 5 mg tablets.
|
|---|---|
|
Percent of Participants With Non-relapse Mortality (NRM)
|
28 percentage of participants
Interval 13.0 to 61.0
|
SECONDARY outcome
Timeframe: 1-year post transplantAcute and chronic GvHD. GvHD is a potentially serious complication of allogeneic stem cell transplantation. Stage Skin Liver (bilirubin) Gut (stool output/day) 0 No GVHD rash \< 2 mg/dl \< 500 ml/day or persistent nausea. 1. Maculopapular rash\< 25% body surface area (BSA) 2-3 mg/dl 500-999 ml/day 2. Maculopapular rash 25 - 50% BSA 3.1-6 mg/dl 1000-1500 ml/day 3. Maculopapular rash \> 50% BSA 6.1-15 mg/dl Adult: \>1500 ml/day 4. Generalized erythroderma plus bullous formation \>15 mg/dl Severe abdominal pain with or without ileus Grade I Stage 1-2 None None II Stage 3 or Stage 1 or Stage 1 III - Stage 2-3 or Stage 2-4 IV Stage 4 or Stage 4 -
Outcome measures
| Measure |
Ruxolitinib Pre- Hematopoietic Cell Transplantation (HCT)
n=21 Participants
Ruxolitinib Pre- Hematopoietic cell transplantation (HCT) in Patients With Myelofibrosis
Ruxolitinib (INC424) tablets started 60 days (day -65) prior to start of conditioning chemotherapy. The starting dose of Ruxolitinib was determined according to baseline platelet count and modified according to platelet count at follow-up. The drug was given in the maximum tolerated dose as defined in the protocol for 56 days, followed by 4 days of taper, and stopped completely at the planned start of conditioning therapy (starting on day -5) i.e. 5 days prior to stem cell infusion. The drug was supplied as 5 mg tablets.
|
|---|---|
|
Percent of Participants With Graft Versus Host Disease (GvHD)
Acute GvHD (Grade 1/2)
|
48 percentage of participants
Interval 29.0 to 79.0
|
|
Percent of Participants With Graft Versus Host Disease (GvHD)
Acute GvHD (Grade 3)
|
16 percentage of participants
Interval 5.0 to 46.0
|
|
Percent of Participants With Graft Versus Host Disease (GvHD)
Chronic GvHD (Mild)
|
51 percentage of participants
Interval 31.0 to 86.0
|
|
Percent of Participants With Graft Versus Host Disease (GvHD)
Chronic (Moderate)
|
25 percentage of participants
Interval 9.0 to 72.0
|
SECONDARY outcome
Timeframe: 30 days post transplantPopulation: This data was not collected because the study ended early.
Will check percentage of donor versus recipient blood cells to determine efficacy of donor engraftment
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: 60 days post transplantPopulation: This data was not collected because the study ended early.
Will check percentage of donor versus recipient blood cells to determine efficacy of donor engraftment
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: 100 days post transplantPopulation: This data was not collected because the study ended early.
Will check percentage of donor versus recipient blood cells to determine efficacy of donor engraftment
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Day 100 post transplantRemission status according to International Working Group for Myelofibrosis Research and Treatment (IWG-MRT) criteria Remission defined as: Bone marrow:\* Age-adjusted normocellularity; \<5% blasts; ≤grade 1 MF and Peripheral blood: Hemoglobin ≥100 g/L and \<UNL; neutrophil count ≥ 1 × 109/L and \<UNL; Platelet count ≥100 × 109/L and \<UNL; \<2% immature myeloid cells and Clinical: Resolution of disease symptoms; spleen and liver not palpable; no evidence of extramedullary hematopoiesis (EMH)
Outcome measures
| Measure |
Ruxolitinib Pre- Hematopoietic Cell Transplantation (HCT)
n=21 Participants
Ruxolitinib Pre- Hematopoietic cell transplantation (HCT) in Patients With Myelofibrosis
Ruxolitinib (INC424) tablets started 60 days (day -65) prior to start of conditioning chemotherapy. The starting dose of Ruxolitinib was determined according to baseline platelet count and modified according to platelet count at follow-up. The drug was given in the maximum tolerated dose as defined in the protocol for 56 days, followed by 4 days of taper, and stopped completely at the planned start of conditioning therapy (starting on day -5) i.e. 5 days prior to stem cell infusion. The drug was supplied as 5 mg tablets.
|
|---|---|
|
Number of Participants With Remission Status According to IWG-MRT Criteria
|
20 Participants
|
SECONDARY outcome
Timeframe: 6 months post transplantRemission defined as: Bone marrow:\* Age-adjusted normocellularity; \<5% blasts; ≤grade 1 MF† and Peripheral blood: Hemoglobin ≥100 g/L and \<UNL; neutrophil count ≥ 1 × 109/L and \<UNL; Platelet count ≥100 × 109/L and \<UNL; \<2% immature myeloid cells‡ and Clinical: Resolution of disease symptoms; spleen and liver not palpable; no evidence of EMH
Outcome measures
| Measure |
Ruxolitinib Pre- Hematopoietic Cell Transplantation (HCT)
n=21 Participants
Ruxolitinib Pre- Hematopoietic cell transplantation (HCT) in Patients With Myelofibrosis
Ruxolitinib (INC424) tablets started 60 days (day -65) prior to start of conditioning chemotherapy. The starting dose of Ruxolitinib was determined according to baseline platelet count and modified according to platelet count at follow-up. The drug was given in the maximum tolerated dose as defined in the protocol for 56 days, followed by 4 days of taper, and stopped completely at the planned start of conditioning therapy (starting on day -5) i.e. 5 days prior to stem cell infusion. The drug was supplied as 5 mg tablets.
|
|---|---|
|
Number of Participants With Remission Status at 6 Months Post Transplant
|
17 Participants
|
SECONDARY outcome
Timeframe: 12 months post transplantRemission defined as: Bone marrow:\* Age-adjusted normocellularity; \<5% blasts; ≤grade 1 MF† and Peripheral blood: Hemoglobin ≥100 g/L and \<UNL; neutrophil count ≥ 1 × 109/L and \<UNL; Platelet count ≥100 × 109/L and \<UNL; \<2% immature myeloid cells‡ and Clinical: Resolution of disease symptoms; spleen and liver not palpable; no evidence of EMH
Outcome measures
| Measure |
Ruxolitinib Pre- Hematopoietic Cell Transplantation (HCT)
n=21 Participants
Ruxolitinib Pre- Hematopoietic cell transplantation (HCT) in Patients With Myelofibrosis
Ruxolitinib (INC424) tablets started 60 days (day -65) prior to start of conditioning chemotherapy. The starting dose of Ruxolitinib was determined according to baseline platelet count and modified according to platelet count at follow-up. The drug was given in the maximum tolerated dose as defined in the protocol for 56 days, followed by 4 days of taper, and stopped completely at the planned start of conditioning therapy (starting on day -5) i.e. 5 days prior to stem cell infusion. The drug was supplied as 5 mg tablets.
|
|---|---|
|
Number of Participants With Remission Status at 12 Months Post Transplant
|
14 Participants
|
SECONDARY outcome
Timeframe: 1-year post transplantRelapse/progression defined as: Peripheral blood: Hemoglobin ≥100 g/L and \<UNL; neutrophil count ≥1 × 109/L and \<UNL; platelet count ≥100 × 109/L and \<UNL; \<2% immature myeloid cells‡ and Clinical: Resolution of disease symptoms; spleen and liver not palpable; no evidence of EMH or Bone marrow:\* Age-adjusted normocellularity; \<5% blasts; ≤grade 1 MF†, and peripheral blood: Hemoglobin ≥85 but \<100 g/L and \<UNL; neutrophil count ≥1 × 109/L and \<UNL; platelet count ≥50, but \<100 × 109/L and \<UNL; \<2% immature myeloid cells‡ and Clinical: Resolution of disease symptoms; spleen and liver not palpable; no evidence of EMH
Outcome measures
| Measure |
Ruxolitinib Pre- Hematopoietic Cell Transplantation (HCT)
n=21 Participants
Ruxolitinib Pre- Hematopoietic cell transplantation (HCT) in Patients With Myelofibrosis
Ruxolitinib (INC424) tablets started 60 days (day -65) prior to start of conditioning chemotherapy. The starting dose of Ruxolitinib was determined according to baseline platelet count and modified according to platelet count at follow-up. The drug was given in the maximum tolerated dose as defined in the protocol for 56 days, followed by 4 days of taper, and stopped completely at the planned start of conditioning therapy (starting on day -5) i.e. 5 days prior to stem cell infusion. The drug was supplied as 5 mg tablets.
|
|---|---|
|
Number of Participants With Relapse/Progression (Defined as Per IWG-MRT Criteria)
|
7 Participants
|
SECONDARY outcome
Timeframe: 1-year post transplantProgression is defined using Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.0), as a 20% increase in the sum of the longest diameter of target lesions, or a measurable increase in a non-target lesion, or the appearance of new lesions.
Outcome measures
| Measure |
Ruxolitinib Pre- Hematopoietic Cell Transplantation (HCT)
n=21 Participants
Ruxolitinib Pre- Hematopoietic cell transplantation (HCT) in Patients With Myelofibrosis
Ruxolitinib (INC424) tablets started 60 days (day -65) prior to start of conditioning chemotherapy. The starting dose of Ruxolitinib was determined according to baseline platelet count and modified according to platelet count at follow-up. The drug was given in the maximum tolerated dose as defined in the protocol for 56 days, followed by 4 days of taper, and stopped completely at the planned start of conditioning therapy (starting on day -5) i.e. 5 days prior to stem cell infusion. The drug was supplied as 5 mg tablets.
|
|---|---|
|
Number of Participants With Progression-free Survival
|
11 Participants
|
SECONDARY outcome
Timeframe: 1-year post transplantOutcome measures
| Measure |
Ruxolitinib Pre- Hematopoietic Cell Transplantation (HCT)
n=21 Participants
Ruxolitinib Pre- Hematopoietic cell transplantation (HCT) in Patients With Myelofibrosis
Ruxolitinib (INC424) tablets started 60 days (day -65) prior to start of conditioning chemotherapy. The starting dose of Ruxolitinib was determined according to baseline platelet count and modified according to platelet count at follow-up. The drug was given in the maximum tolerated dose as defined in the protocol for 56 days, followed by 4 days of taper, and stopped completely at the planned start of conditioning therapy (starting on day -5) i.e. 5 days prior to stem cell infusion. The drug was supplied as 5 mg tablets.
|
|---|---|
|
Number of Overall Survival
|
15 Participants
|
SECONDARY outcome
Timeframe: baseline and 48 monthsMean change in the Brief Fatigue Inventory score (BFI) from baseline to 48 months to assess impact of allogeneic stem cell transplant on myelofibrosis associated symptoms and overall quality of life. The BFI is a 9 item scored from 0 (no fatigue) -10 (as bad as you can imagine), items are averaged with total score from 0-10, with higher score indicating more fatigue.
Outcome measures
| Measure |
Ruxolitinib Pre- Hematopoietic Cell Transplantation (HCT)
n=21 Participants
Ruxolitinib Pre- Hematopoietic cell transplantation (HCT) in Patients With Myelofibrosis
Ruxolitinib (INC424) tablets started 60 days (day -65) prior to start of conditioning chemotherapy. The starting dose of Ruxolitinib was determined according to baseline platelet count and modified according to platelet count at follow-up. The drug was given in the maximum tolerated dose as defined in the protocol for 56 days, followed by 4 days of taper, and stopped completely at the planned start of conditioning therapy (starting on day -5) i.e. 5 days prior to stem cell infusion. The drug was supplied as 5 mg tablets.
|
|---|---|
|
Mean Change in the Brief Fatigue Inventory Score
|
1.7 score on a scale
Interval 0.25 to 3.08
|
SECONDARY outcome
Timeframe: 30 days post transplantPopulation: This data was not collected because the study ended early.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: 100 days post transplantPopulation: This data was not collected because the study ended early.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: 30 days post transplantPopulation: This data was not collected because the study ended early.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: 100 days post transplantPopulation: This data was not collected because the study ended early.
Outcome measures
Outcome data not reported
Adverse Events
Ruxolitinib Pre- Hematopoietic Cell Transplantation (HCT)
Serious events: 13 serious events
Other events: 20 other events
Deaths: 4 deaths
Serious adverse events
| Measure |
Ruxolitinib Pre- Hematopoietic Cell Transplantation (HCT)
n=21 participants at risk
Ruxolitinib Pre- Hematopoietic cell transplantation (HCT) in Patients With Myelofibrosis
Ruxolitinib (INC424) tablets started 60 days (day -65) prior to start of conditioning chemotherapy. The starting dose of Ruxolitinib was determined according to baseline platelet count and modified according to platelet count at follow-up. The drug was given in the maximum tolerated dose as defined in the protocol for 56 days, followed by 4 days of taper, and stopped completely at the planned start of conditioning therapy (starting on day -5) i.e. 5 days prior to stem cell infusion. The drug was supplied as 5 mg tablets.
|
|---|---|
|
Endocrine disorders
Drug-induced hyperglycemia
|
4.8%
1/21 • up to 4 years
|
|
Gastrointestinal disorders
Diverticulitis NOS
|
4.8%
1/21 • up to 4 years
|
|
Gastrointestinal disorders
Nausea post chemotherapy
|
4.8%
1/21 • up to 4 years
|
|
General disorders
Death sudden
|
4.8%
1/21 • up to 4 years
|
|
General disorders
Neutropenic fever
|
4.8%
1/21 • up to 4 years
|
|
Infections and infestations
Pneumonia
|
19.0%
4/21 • up to 4 years
|
|
Infections and infestations
Sepsis
|
14.3%
3/21 • up to 4 years
|
|
Injury, poisoning and procedural complications
Graft failure
|
19.0%
4/21 • up to 4 years
|
|
Investigations
Elevated liver enzyme levels
|
4.8%
1/21 • up to 4 years
|
|
Investigations
Lymphocyte count decreased
|
4.8%
1/21 • up to 4 years
|
|
Metabolism and nutrition disorders
Hyponatremia
|
4.8%
1/21 • up to 4 years
|
|
Nervous system disorders
Intracranial hemorrhage
|
4.8%
1/21 • up to 4 years
|
|
Nervous system disorders
Spinal epidural hematoma
|
4.8%
1/21 • up to 4 years
|
|
Renal and urinary disorders
Acute renal failure
|
4.8%
1/21 • up to 4 years
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnea
|
4.8%
1/21 • up to 4 years
|
|
General disorders
Disease Progression
|
4.8%
1/21 • up to 4 years
|
|
Respiratory, thoracic and mediastinal disorders
Pneumonia
|
4.8%
1/21 • up to 4 years
|
Other adverse events
| Measure |
Ruxolitinib Pre- Hematopoietic Cell Transplantation (HCT)
n=21 participants at risk
Ruxolitinib Pre- Hematopoietic cell transplantation (HCT) in Patients With Myelofibrosis
Ruxolitinib (INC424) tablets started 60 days (day -65) prior to start of conditioning chemotherapy. The starting dose of Ruxolitinib was determined according to baseline platelet count and modified according to platelet count at follow-up. The drug was given in the maximum tolerated dose as defined in the protocol for 56 days, followed by 4 days of taper, and stopped completely at the planned start of conditioning therapy (starting on day -5) i.e. 5 days prior to stem cell infusion. The drug was supplied as 5 mg tablets.
|
|---|---|
|
Gastrointestinal disorders
Abdominal pain
|
42.9%
9/21 • up to 4 years
|
|
Investigations
Activated partial thromboplastin time
|
23.8%
5/21 • up to 4 years
|
|
Renal and urinary disorders
Acute kidney injury
|
19.0%
4/21 • up to 4 years
|
|
Blood and lymphatic system disorders
Acute myeloid leukemia
|
4.8%
1/21 • up to 4 years
|
|
Renal and urinary disorders
Acute renal failure
|
4.8%
1/21 • up to 4 years
|
|
Nervous system disorders
Agitation
|
4.8%
1/21 • up to 4 years
|
|
Investigations
Alanine aminotransferase increased
|
33.3%
7/21 • up to 4 years
|
|
Investigations
Alkaline phosphatase increased
|
33.3%
7/21 • up to 4 years
|
|
Skin and subcutaneous tissue disorders
Allergic rash
|
4.8%
1/21 • up to 4 years
|
|
Respiratory, thoracic and mediastinal disorders
Allergic rhinitis
|
14.3%
3/21 • up to 4 years
|
|
Immune system disorders
Alloimmunization
|
4.8%
1/21 • up to 4 years
|
|
Gastrointestinal disorders
Anal hemorrhage
|
14.3%
3/21 • up to 4 years
|
|
Blood and lymphatic system disorders
Anemia
|
61.9%
13/21 • up to 4 years
|
|
Musculoskeletal and connective tissue disorders
Ankle edema
|
9.5%
2/21 • up to 4 years
|
|
Metabolism and nutrition disorders
Anorexia
|
33.3%
7/21 • up to 4 years
|
|
Psychiatric disorders
Anxiety
|
9.5%
2/21 • up to 4 years
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
14.3%
3/21 • up to 4 years
|
|
Musculoskeletal and connective tissue disorders
Back pain (without radiation)
|
28.6%
6/21 • up to 4 years
|
|
Gastrointestinal disorders
Bloating
|
23.8%
5/21 • up to 4 years
|
|
Investigations
Blood bilirubin increased
|
23.8%
5/21 • up to 4 years
|
|
Eye disorders
Blurred vision
|
9.5%
2/21 • up to 4 years
|
|
Musculoskeletal and connective tissue disorders
Bone pain
|
9.5%
2/21 • up to 4 years
|
|
Immune system disorders
Bronchospasm
|
4.8%
1/21 • up to 4 years
|
|
Skin and subcutaneous tissue disorders
Bruising
|
23.8%
5/21 • up to 4 years
|
|
Renal and urinary disorders
Burning from urination
|
4.8%
1/21 • up to 4 years
|
|
Nervous system disorders
Burning sensation
|
4.8%
1/21 • up to 4 years
|
|
Gastrointestinal disorders
C.difficile diarrhea
|
4.8%
1/21 • up to 4 years
|
|
Infections and infestations
CMV infection
|
4.8%
1/21 • up to 4 years
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Cardiac neoplasm unspecified
|
4.8%
1/21 • up to 4 years
|
|
Cardiac disorders
Cardiomyopathy
|
4.8%
1/21 • up to 4 years
|
|
Eye disorders
Cataract
|
4.8%
1/21 • up to 4 years
|
|
General disorders
Catheter site erythema
|
4.8%
1/21 • up to 4 years
|
|
Respiratory, thoracic and mediastinal disorders
Chest congestion
|
4.8%
1/21 • up to 4 years
|
|
Cardiac disorders
Chest pain - cardiac
|
4.8%
1/21 • up to 4 years
|
|
General disorders
Chills
|
28.6%
6/21 • up to 4 years
|
|
Renal and urinary disorders
Chronic kidney disease
|
4.8%
1/21 • up to 4 years
|
|
Infections and infestations
Clostridium difficile test positive
|
4.8%
1/21 • up to 4 years
|
|
Nervous system disorders
Confusion
|
9.5%
2/21 • up to 4 years
|
|
Psychiatric disorders
Confusion
|
9.5%
2/21 • up to 4 years
|
|
Eye disorders
Conjunctivitis
|
4.8%
1/21 • up to 4 years
|
|
Gastrointestinal disorders
Constipation
|
19.0%
4/21 • up to 4 years
|
|
Vascular disorders
Contusion
|
4.8%
1/21 • up to 4 years
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
28.6%
6/21 • up to 4 years
|
|
Investigations
Creatinine increased
|
19.0%
4/21 • up to 4 years
|
|
Immune system disorders
Cytokine release syndrome
|
9.5%
2/21 • up to 4 years
|
|
Metabolism and nutrition disorders
Dehydration
|
4.8%
1/21 • up to 4 years
|
|
Psychiatric disorders
Delirium
|
4.8%
1/21 • up to 4 years
|
|
Psychiatric disorders
Depression
|
19.0%
4/21 • up to 4 years
|
|
General disorders
Diaphoresis
|
4.8%
1/21 • up to 4 years
|
|
Gastrointestinal disorders
Diarrhea
|
52.4%
11/21 • up to 4 years
|
|
Nervous system disorders
Dizziness
|
38.1%
8/21 • up to 4 years
|
|
Eye disorders
Double vision
|
4.8%
1/21 • up to 4 years
|
|
Eye disorders
Drug-induced liver disease
|
4.8%
1/21 • up to 4 years
|
|
Eye disorders
Dry eye
|
4.8%
1/21 • up to 4 years
|
|
Gastrointestinal disorders
Dry mouth
|
28.6%
6/21 • up to 4 years
|
|
Skin and subcutaneous tissue disorders
Dry skin
|
28.6%
6/21 • up to 4 years
|
|
Gastrointestinal disorders
Dysgeusia
|
4.8%
1/21 • up to 4 years
|
|
Gastrointestinal disorders
Dyspepsia
|
23.8%
5/21 • up to 4 years
|
|
Gastrointestinal disorders
Dysphagia
|
9.5%
2/21 • up to 4 years
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnea
|
47.6%
10/21 • up to 4 years
|
|
Ear and labyrinth disorders
Ear pain
|
9.5%
2/21 • up to 4 years
|
|
General disorders
Edema extremities
|
38.1%
8/21 • up to 4 years
|
|
Respiratory, thoracic and mediastinal disorders
Epistaxis
|
23.8%
5/21 • up to 4 years
|
|
Skin and subcutaneous tissue disorders
Erythema
|
14.3%
3/21 • up to 4 years
|
|
Skin and subcutaneous tissue disorders
Facial swelling
|
4.8%
1/21 • up to 4 years
|
|
Injury, poisoning and procedural complications
Fall
|
9.5%
2/21 • up to 4 years
|
|
General disorders
Fatigue
|
38.1%
8/21 • up to 4 years
|
|
Blood and lymphatic system disorders
Febrile neutropenia
|
28.6%
6/21 • up to 4 years
|
|
Gastrointestinal disorders
Fecal incontinence
|
9.5%
2/21 • up to 4 years
|
|
General disorders
Fever
|
38.1%
8/21 • up to 4 years
|
|
Investigations
Fibrinogen decreased
|
4.8%
1/21 • up to 4 years
|
|
Gastrointestinal disorders
Flatulence
|
9.5%
2/21 • up to 4 years
|
|
Skin and subcutaneous tissue disorders
Folliculitis
|
9.5%
2/21 • up to 4 years
|
|
Injury, poisoning and procedural complications
Foot injury
|
4.8%
1/21 • up to 4 years
|
|
Metabolism and nutrition disorders
GGT Increased
|
4.8%
1/21 • up to 4 years
|
|
Musculoskeletal and connective tissue disorders
Generalized muscle weakness
|
23.8%
5/21 • up to 4 years
|
|
Metabolism and nutrition disorders
Gout
|
4.8%
1/21 • up to 4 years
|
|
Reproductive system and breast disorders
Gynecomastia
|
4.8%
1/21 • up to 4 years
|
|
Infections and infestations
HSV infection
|
4.8%
1/21 • up to 4 years
|
|
Nervous system disorders
Headache
|
38.1%
8/21 • up to 4 years
|
|
Ear and labyrinth disorders
Hearing impaired
|
4.8%
1/21 • up to 4 years
|
|
Vascular disorders
Hematoma
|
4.8%
1/21 • up to 4 years
|
|
Renal and urinary disorders
Hematuria
|
9.5%
2/21 • up to 4 years
|
|
Investigations
Hemoglobin decreased
|
4.8%
1/21 • up to 4 years
|
|
Blood and lymphatic system disorders
Hemolysis
|
4.8%
1/21 • up to 4 years
|
|
Gastrointestinal disorders
Hemorrhoidal hemorrhage
|
9.5%
2/21 • up to 4 years
|
|
Respiratory, thoracic and mediastinal disorders
Hiccups
|
9.5%
2/21 • up to 4 years
|
|
Respiratory, thoracic and mediastinal disorders
Hoarseness
|
4.8%
1/21 • up to 4 years
|
|
Metabolism and nutrition disorders
Hyperglycemia
|
42.9%
9/21 • up to 4 years
|
|
Metabolism and nutrition disorders
Hyperkalemia
|
38.1%
8/21 • up to 4 years
|
|
Metabolism and nutrition disorders
Hypermagnesemia
|
4.8%
1/21 • up to 4 years
|
|
Metabolism and nutrition disorders
Hypertension
|
28.6%
6/21 • up to 4 years
|
|
Metabolism and nutrition disorders
Hypertriglyceridemia
|
4.8%
1/21 • up to 4 years
|
|
Metabolism and nutrition disorders
Hyperuricemia
|
19.0%
4/21 • up to 4 years
|
|
Metabolism and nutrition disorders
Hypoalbuminemia
|
38.1%
8/21 • up to 4 years
|
|
Metabolism and nutrition disorders
Hypocalcemia
|
14.3%
3/21 • up to 4 years
|
|
Metabolism and nutrition disorders
Hypokalemia
|
23.8%
5/21 • up to 4 years
|
|
Metabolism and nutrition disorders
Hypomagnesaemia
|
47.6%
10/21 • up to 4 years
|
|
Metabolism and nutrition disorders
Hyponatremia
|
28.6%
6/21 • up to 4 years
|
|
Metabolism and nutrition disorders
Hypophosphatemia
|
23.8%
5/21 • up to 4 years
|
|
Vascular disorders
Hypotension
|
9.5%
2/21 • up to 4 years
|
|
Respiratory, thoracic and mediastinal disorders
Hypoxia
|
4.8%
1/21 • up to 4 years
|
|
Investigations
INR increased
|
23.8%
5/21 • up to 4 years
|
|
Investigations
Increased TSH
|
4.8%
1/21 • up to 4 years
|
|
Infections and infestations
Infusion site cellulitis
|
4.8%
1/21 • up to 4 years
|
|
Nervous system disorders
Insomnia
|
9.5%
2/21 • up to 4 years
|
|
Endocrine disorders
Insufficiency adrenal
|
4.8%
1/21 • up to 4 years
|
|
Blood and lymphatic system disorders
Iron deficiency anemia
|
4.8%
1/21 • up to 4 years
|
|
Gastrointestinal disorders
Irritation lips
|
4.8%
1/21 • up to 4 years
|
|
Skin and subcutaneous tissue disorders
Itching
|
4.8%
1/21 • up to 4 years
|
|
Investigations
Laboratory test abnormal
|
4.8%
1/21 • up to 4 years
|
|
Gastrointestinal disorders
Left lower quadrant pain
|
4.8%
1/21 • up to 4 years
|
|
General disorders
Lethargy
|
4.8%
1/21 • up to 4 years
|
|
Blood and lymphatic system disorders
Leukopenia
|
4.8%
1/21 • up to 4 years
|
|
Vascular disorders
Lightheadedness
|
4.8%
1/21 • up to 4 years
|
|
Respiratory, thoracic and mediastinal disorders
Lung infection
|
4.8%
1/21 • up to 4 years
|
|
Investigations
Lymphocyte count decreased
|
23.8%
5/21 • up to 4 years
|
|
Investigations
Lymphocyte count increased
|
4.8%
1/21 • up to 4 years
|
|
Skin and subcutaneous tissue disorders
Maculopapular rash
|
14.3%
3/21 • up to 4 years
|
|
General disorders
Malaise
|
9.5%
2/21 • up to 4 years
|
|
Ear and labyrinth disorders
Middle ear inflammation
|
4.8%
1/21 • up to 4 years
|
|
Gastrointestinal disorders
Mucositis oral
|
38.1%
8/21 • up to 4 years
|
|
Musculoskeletal and connective tissue disorders
Muscle weakness lower limb
|
14.3%
3/21 • up to 4 years
|
|
Respiratory, thoracic and mediastinal disorders
Nasal congestion
|
14.3%
3/21 • up to 4 years
|
|
Gastrointestinal disorders
Nausea
|
66.7%
14/21 • up to 4 years
|
|
Gastrointestinal disorders
Nausea and vomiting
|
4.8%
1/21 • up to 4 years
|
|
Musculoskeletal and connective tissue disorders
Neck pain
|
4.8%
1/21 • up to 4 years
|
|
Investigations
Neutrophil count decreased
|
42.9%
9/21 • up to 4 years
|
|
Eye disorders
Night blindness
|
52.4%
11/21 • up to 4 years
|
|
General disorders
Night sweats
|
9.5%
2/21 • up to 4 years
|
|
General disorders
Non-cardiac chest pain
|
4.8%
1/21 • up to 4 years
|
|
Injury, poisoning and procedural complications
Open wound of foot except toe(s) alone
|
4.8%
1/21 • up to 4 years
|
|
Gastrointestinal disorders
Oral hemorrhage
|
4.8%
1/21 • up to 4 years
|
|
Vascular disorders
Orthostatic hypotension
|
4.8%
1/21 • up to 4 years
|
|
General disorders
Pain
|
19.0%
4/21 • up to 4 years
|
|
Renal and urinary disorders
Painful urination
|
4.8%
1/21 • up to 4 years
|
|
Cardiac disorders
Palpitations
|
4.8%
1/21 • up to 4 years
|
|
Nervous system disorders
Paraplegia
|
4.8%
1/21 • up to 4 years
|
|
Gastrointestinal disorders
Perianal pain
|
4.8%
1/21 • up to 4 years
|
|
Skin and subcutaneous tissue disorders
Periocular rash
|
4.8%
1/21 • up to 4 years
|
|
Nervous system disorders
Peripheral motor neuropathy
|
4.8%
1/21 • up to 4 years
|
|
Nervous system disorders
Peripheral sensory neuropathy
|
4.8%
1/21 • up to 4 years
|
|
Blood and lymphatic system disorders
Petechiae
|
19.0%
4/21 • up to 4 years
|
|
Respiratory, thoracic and mediastinal disorders
Pharyngitis
|
4.8%
1/21 • up to 4 years
|
|
Investigations
Plasma creatinine increased
|
4.8%
1/21 • up to 4 years
|
|
Investigations
Platelet count decreased
|
47.6%
10/21 • up to 4 years
|
|
Respiratory, thoracic and mediastinal disorders
Pleural effusion
|
4.8%
1/21 • up to 4 years
|
|
Respiratory, thoracic and mediastinal disorders
Pleuritic pain
|
4.8%
1/21 • up to 4 years
|
|
Immune system disorders
Polymyalgia rheumatica
|
4.8%
1/21 • up to 4 years
|
|
General disorders
Post procedural discomfort
|
4.8%
1/21 • up to 4 years
|
|
Respiratory, thoracic and mediastinal disorders
Postnasal drip
|
4.8%
1/21 • up to 4 years
|
|
Skin and subcutaneous tissue disorders
Pressure sore
|
4.8%
1/21 • up to 4 years
|
|
Cardiac disorders
Presyncope
|
4.8%
1/21 • up to 4 years
|
|
Skin and subcutaneous tissue disorders
Pruritus
|
23.8%
5/21 • up to 4 years
|
|
Musculoskeletal and connective tissue disorders
Pseudogout
|
4.8%
1/21 • up to 4 years
|
|
Skin and subcutaneous tissue disorders
Rash acneiform
|
4.8%
1/21 • up to 4 years
|
|
Renal and urinary disorders
Renal calculi
|
4.8%
1/21 • up to 4 years
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory failure
|
4.8%
1/21 • up to 4 years
|
|
Infections and infestations
Respiratory syncytial virus infection
|
57.1%
12/21 • up to 4 years
|
|
General disorders
Rigors
|
4.8%
1/21 • up to 4 years
|
|
Eye disorders
Scleral hemorrhage
|
9.5%
2/21 • up to 4 years
|
|
Infections and infestations
Sepsis
|
4.8%
1/21 • up to 4 years
|
|
Cardiac disorders
Sinus tachycardia
|
19.0%
4/21 • up to 4 years
|
|
Respiratory, thoracic and mediastinal disorders
Sinusitis
|
4.8%
1/21 • up to 4 years
|
|
Skin and subcutaneous tissue disorders
Skin desquamation
|
4.8%
1/21 • up to 4 years
|
|
Skin and subcutaneous tissue disorders
Skin hyperpigmentation
|
4.8%
1/21 • up to 4 years
|
|
Infections and infestations
Skin infection
|
4.8%
1/21 • up to 4 years
|
|
Skin and subcutaneous tissue disorders
Skin lesion
|
4.8%
1/21 • up to 4 years
|
|
Skin and subcutaneous tissue disorders
Skin ulcer
|
4.8%
1/21 • up to 4 years
|
|
Respiratory, thoracic and mediastinal disorders
Sneezing
|
4.8%
1/21 • up to 4 years
|
|
Nervous system disorders
Sleep apnea
|
4.8%
1/21 • up to 4 years
|
|
Nervous system disorders
Somnolence
|
9.5%
2/21 • up to 4 years
|
|
Gastrointestinal disorders
Sore throat
|
19.0%
4/21 • up to 4 years
|
|
Respiratory, thoracic and mediastinal disorders
Sore throat
|
19.0%
4/21 • up to 4 years
|
|
Cardiac disorders
Syncope
|
9.5%
2/21 • up to 4 years
|
|
Cardiac disorders
Tachycardia
|
4.8%
1/21 • up to 4 years
|
|
Blood and lymphatic system disorders
Thrombocytopenia
|
19.0%
4/21 • up to 4 years
|
|
Gastrointestinal disorders
Tooth pain
|
4.8%
1/21 • up to 4 years
|
|
Nervous system disorders
Tremor
|
9.5%
2/21 • up to 4 years
|
|
Infections and infestations
Upper respiratory tract infection bacterial
|
14.3%
3/21 • up to 4 years
|
|
Nervous system disorders
Urinary incontinence
|
9.5%
2/21 • up to 4 years
|
|
Renal and urinary disorders
Urinary hesitancy
|
4.8%
1/21 • up to 4 years
|
|
Renal and urinary disorders
Urinary incontinence
|
9.5%
2/21 • up to 4 years
|
|
Infections and infestations
Urinary tract infection
|
9.5%
2/21 • up to 4 years
|
|
Immune system disorders
Urticaria
|
4.8%
1/21 • up to 4 years
|
|
Gastrointestinal disorders
Vomiting
|
33.3%
7/21 • up to 4 years
|
|
General disorders
Weakness
|
4.8%
1/21 • up to 4 years
|
|
Investigations
Weight gain
|
4.8%
1/21 • up to 4 years
|
|
Investigations
Weight loss
|
23.8%
5/21 • up to 4 years
|
|
Respiratory, thoracic and mediastinal disorders
Wheezing
|
19.0%
4/21 • up to 4 years
|
|
Investigations
White blood cell decreased
|
42.9%
9/21 • up to 4 years
|
|
Investigations
Aspartate aminotransferase increased
|
38.1%
8/21 • up to 4 years
|
|
Blood and lymphatic system disorders
Autoimmune hemolytic anemia
|
4.8%
1/21 • up to 4 years
|
|
Skin and subcutaneous tissue disorders
Edema periorbital
|
4.8%
1/21 • up to 4 years
|
|
General disorders
Edematous weight gain
|
4.8%
1/21 • up to 4 years
|
|
Hepatobiliary disorders
Hypoalbuminemia
|
38.1%
8/21 • up to 4 years
|
|
Skin and subcutaneous tissue disorders
Macular Rash
|
4.8%
1/21 • up to 4 years
|
|
Skin and subcutaneous tissue disorders
Petechiae
|
19.0%
4/21 • up to 4 years
|
|
Nervous system disorders
Syncope
|
9.5%
2/21 • up to 4 years
|
|
Investigations
White blood cell count increased
|
4.8%
1/21 • up to 4 years
|
Additional Information
Dr. John Mascarenhas
Icahn School of Medicine at Mount Sinai
Phone: 212-241-3417
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place