Trial Outcomes & Findings for Study of Fludarabine Based Conditioning for Allogeneic Stem Cell Transplantation for Myelofibrosis (NCT NCT00572897)
NCT ID: NCT00572897
Last Updated: 2017-05-15
Results Overview
Number of participants alive at 2 years who are progression-free
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
66 participants
Primary outcome timeframe
2 years
Results posted on
2017-05-15
Participant Flow
Patients were recruited from 2007 to 2011 at 11 centers affiliated with the Myeloproliferative Disorders Research Consortium (MPD-RC)
Participant milestones
| Measure |
Sibling Donor
Patients received a stem cell transplant from sibling
|
Unrelated Donor
Patients received a stem cell transplant from an unrelated donor
|
|---|---|---|
|
Overall Study
STARTED
|
32
|
34
|
|
Overall Study
COMPLETED
|
32
|
34
|
|
Overall Study
NOT COMPLETED
|
0
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Study of Fludarabine Based Conditioning for Allogeneic Stem Cell Transplantation for Myelofibrosis
Baseline characteristics by cohort
| Measure |
Sibling Donor
n=32 Participants
Patients received a stem cell transplant from sibling
|
Unrelated Donor
n=34 Participants
Patients received a stem cell transplant from an unrelated donor
|
Total
n=66 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
55 years
n=5 Participants
|
56 years
n=7 Participants
|
55 years
n=5 Participants
|
|
Sex: Female, Male
Female
|
13 Participants
n=5 Participants
|
15 Participants
n=7 Participants
|
28 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
19 Participants
n=5 Participants
|
19 Participants
n=7 Participants
|
38 Participants
n=5 Participants
|
|
Diagnosis
PMF
|
14 participants
n=5 Participants
|
25 participants
n=7 Participants
|
39 participants
n=5 Participants
|
|
Diagnosis
PV-MF
|
3 participants
n=5 Participants
|
5 participants
n=7 Participants
|
8 participants
n=5 Participants
|
|
Diagnosis
ET-MF
|
15 participants
n=5 Participants
|
4 participants
n=7 Participants
|
19 participants
n=5 Participants
|
|
Lille score
0
|
3 participants
n=5 Participants
|
0 participants
n=7 Participants
|
3 participants
n=5 Participants
|
|
Lille score
1
|
20 participants
n=5 Participants
|
23 participants
n=7 Participants
|
43 participants
n=5 Participants
|
|
Lille score
2
|
9 participants
n=5 Participants
|
11 participants
n=7 Participants
|
20 participants
n=5 Participants
|
|
Patient: donor gender
Female:Female
|
6 participants
n=5 Participants
|
7 participants
n=7 Participants
|
13 participants
n=5 Participants
|
|
Patient: donor gender
Male:Male
|
7 participants
n=5 Participants
|
8 participants
n=7 Participants
|
15 participants
n=5 Participants
|
|
Patient: donor gender
Male:Female
|
10 participants
n=5 Participants
|
6 participants
n=7 Participants
|
16 participants
n=5 Participants
|
|
Patient: donor gender
Female:Male
|
9 participants
n=5 Participants
|
13 participants
n=7 Participants
|
22 participants
n=5 Participants
|
|
JAK-2V617F
Positive
|
12 participants
n=5 Participants
|
18 participants
n=7 Participants
|
30 participants
n=5 Participants
|
|
JAK-2V617F
Negative
|
17 participants
n=5 Participants
|
16 participants
n=7 Participants
|
33 participants
n=5 Participants
|
|
JAK-2V617F
Unknown
|
3 participants
n=5 Participants
|
0 participants
n=7 Participants
|
3 participants
n=5 Participants
|
|
Bone Marrow Fibrosis
Grade 1
|
0 participants
n=5 Participants
|
2 participants
n=7 Participants
|
2 participants
n=5 Participants
|
|
Bone Marrow Fibrosis
Grade 2
|
2 participants
n=5 Participants
|
6 participants
n=7 Participants
|
8 participants
n=5 Participants
|
|
Bone Marrow Fibrosis
Grade 3
|
18 participants
n=5 Participants
|
23 participants
n=7 Participants
|
41 participants
n=5 Participants
|
|
Bone Marrow Fibrosis
Unknown
|
12 participants
n=5 Participants
|
3 participants
n=7 Participants
|
15 participants
n=5 Participants
|
|
Splenomegaly
Yes
|
24 participants
n=5 Participants
|
28 participants
n=7 Participants
|
52 participants
n=5 Participants
|
|
Splenomegaly
No
|
3 participants
n=5 Participants
|
1 participants
n=7 Participants
|
4 participants
n=5 Participants
|
|
Splenomegaly
Splenectomy
|
5 participants
n=5 Participants
|
5 participants
n=7 Participants
|
10 participants
n=5 Participants
|
|
Karyotype
Normal
|
14 participants
n=5 Participants
|
14 participants
n=7 Participants
|
28 participants
n=5 Participants
|
|
Karyotype
One abnormality
|
7 participants
n=5 Participants
|
9 participants
n=7 Participants
|
16 participants
n=5 Participants
|
|
Karyotype
Complex abnormality
|
5 participants
n=5 Participants
|
0 participants
n=7 Participants
|
5 participants
n=5 Participants
|
|
Karyotype
Unknown
|
6 participants
n=5 Participants
|
11 participants
n=7 Participants
|
17 participants
n=5 Participants
|
|
Stem cell source
Peripheral blood
|
26 participants
n=5 Participants
|
31 participants
n=7 Participants
|
57 participants
n=5 Participants
|
|
Stem cell source
Bone Marrow
|
6 participants
n=5 Participants
|
3 participants
n=7 Participants
|
9 participants
n=5 Participants
|
|
HLA match
Full HLA matched
|
30 participants
n=5 Participants
|
25 participants
n=7 Participants
|
55 participants
n=5 Participants
|
|
HLA match
HLA 1 Ag mismatched, no allele mismatched
|
2 participants
n=5 Participants
|
4 participants
n=7 Participants
|
6 participants
n=5 Participants
|
|
HLA match
HLA Ag matched, 1 0r 2 alleles mismatched
|
0 participants
n=5 Participants
|
5 participants
n=7 Participants
|
5 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: 2 yearsNumber of participants alive at 2 years who are progression-free
Outcome measures
| Measure |
Sibling Donor
n=32 Participants
Patients received a stem cell transplant from sibling
|
Unrelated Donor
n=34 Participants
Patients received a stem cell transplant from an unrelated donor
|
|---|---|---|
|
The Primary Endpoint is Progression-free Survival.
|
24 participants
|
11 participants
|
SECONDARY outcome
Timeframe: 180 daysPopulation: Clinical responses were assessed according to the IWG-MRT 2006 criteria in 46 patients (29 sibling and 17 unrelated transplants) who survived at least 180 days.
assessed according to the IWG Criteria
Outcome measures
| Measure |
Sibling Donor
n=29 Participants
Patients received a stem cell transplant from sibling
|
Unrelated Donor
n=17 Participants
Patients received a stem cell transplant from an unrelated donor
|
|---|---|---|
|
Response Outcomes
clinical complete response
|
7 participants
|
6 participants
|
|
Response Outcomes
clinical partial response
|
8 participants
|
1 participants
|
|
Response Outcomes
clinical improvement
|
11 participants
|
5 participants
|
|
Response Outcomes
stable disease
|
2 participants
|
4 participants
|
|
Response Outcomes
progressive disease
|
0 participants
|
1 participants
|
SECONDARY outcome
Timeframe: 73 monthsThe number of patients alive at last follow-up.
Outcome measures
| Measure |
Sibling Donor
n=32 Participants
Patients received a stem cell transplant from sibling
|
Unrelated Donor
n=34 Participants
Patients received a stem cell transplant from an unrelated donor
|
|---|---|---|
|
Overall Survival
|
25 participants
|
11 participants
|
SECONDARY outcome
Timeframe: 2 yearsPatients with ANC ≥0.5 × 10\^9/L
Outcome measures
| Measure |
Sibling Donor
n=32 Participants
Patients received a stem cell transplant from sibling
|
Unrelated Donor
n=34 Participants
Patients received a stem cell transplant from an unrelated donor
|
|---|---|---|
|
Absolute Neutrophil Count (ANC)
yes
|
31 participants
|
26 participants
|
|
Absolute Neutrophil Count (ANC)
no
|
1 participants
|
8 participants
|
SECONDARY outcome
Timeframe: 2 yearsPatients with PLT ≥20 × 109/L
Outcome measures
| Measure |
Sibling Donor
n=32 Participants
Patients received a stem cell transplant from sibling
|
Unrelated Donor
n=34 Participants
Patients received a stem cell transplant from an unrelated donor
|
|---|---|---|
|
PLT
yes
|
28 participants
|
20 participants
|
|
PLT
no
|
4 participants
|
14 participants
|
SECONDARY outcome
Timeframe: 2 yearsTransplant-related Mortality including Graft-versus-host disease (GVHD)
Outcome measures
| Measure |
Sibling Donor
n=32 Participants
Patients received a stem cell transplant from sibling
|
Unrelated Donor
n=34 Participants
Patients received a stem cell transplant from an unrelated donor
|
|---|---|---|
|
Transplant-related Mortality
yes
|
3 participants
|
20 participants
|
|
Transplant-related Mortality
no
|
29 participants
|
14 participants
|
Adverse Events
Sibling Donor
Serious events: 8 serious events
Other events: 0 other events
Deaths: 0 deaths
Unrelated Donor
Serious events: 23 serious events
Other events: 0 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Sibling Donor
n=32 participants at risk
Patients received a stem cell transplant from sibling
|
Unrelated Donor
n=34 participants at risk
Patients received a stem cell transplant from an unrelated donor
|
|---|---|---|
|
Blood and lymphatic system disorders
Death
|
3.1%
1/32 • Number of events 1
all adverse events reported under serious adverse events. other non-serious adverse events were not separated out.
|
8.8%
3/34 • Number of events 3
all adverse events reported under serious adverse events. other non-serious adverse events were not separated out.
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Death
|
3.1%
1/32 • Number of events 1
all adverse events reported under serious adverse events. other non-serious adverse events were not separated out.
|
0.00%
0/34
all adverse events reported under serious adverse events. other non-serious adverse events were not separated out.
|
|
Immune system disorders
Death
|
9.4%
3/32 • Number of events 3
all adverse events reported under serious adverse events. other non-serious adverse events were not separated out.
|
14.7%
5/34 • Number of events 5
all adverse events reported under serious adverse events. other non-serious adverse events were not separated out.
|
|
Respiratory, thoracic and mediastinal disorders
Death
|
3.1%
1/32 • Number of events 1
all adverse events reported under serious adverse events. other non-serious adverse events were not separated out.
|
5.9%
2/34 • Number of events 2
all adverse events reported under serious adverse events. other non-serious adverse events were not separated out.
|
|
Cardiac disorders
Death
|
3.1%
1/32 • Number of events 1
all adverse events reported under serious adverse events. other non-serious adverse events were not separated out.
|
0.00%
0/34
all adverse events reported under serious adverse events. other non-serious adverse events were not separated out.
|
|
Surgical and medical procedures
Death
|
0.00%
0/32
all adverse events reported under serious adverse events. other non-serious adverse events were not separated out.
|
2.9%
1/34 • Number of events 1
all adverse events reported under serious adverse events. other non-serious adverse events were not separated out.
|
|
Renal and urinary disorders
Death
|
0.00%
0/32
all adverse events reported under serious adverse events. other non-serious adverse events were not separated out.
|
5.9%
2/34 • Number of events 2
all adverse events reported under serious adverse events. other non-serious adverse events were not separated out.
|
|
Vascular disorders
Death
|
0.00%
0/32
all adverse events reported under serious adverse events. other non-serious adverse events were not separated out.
|
2.9%
1/34 • Number of events 1
all adverse events reported under serious adverse events. other non-serious adverse events were not separated out.
|
|
Infections and infestations
Death
|
0.00%
0/32
all adverse events reported under serious adverse events. other non-serious adverse events were not separated out.
|
2.9%
1/34 • Number of events 1
all adverse events reported under serious adverse events. other non-serious adverse events were not separated out.
|
Other adverse events
Adverse event data not reported
Additional Information
Dr. Damiano Rondelli
Univeristy of Illinois Hospital & Health Sciences System
Phone: 312-996-6179
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place