Trial Outcomes & Findings for Allogeneic Stem Cell Transplantation for Myelofibrosis and Myelodysplastic Syndrome (NCT NCT00475020)
NCT ID: NCT00475020
Last Updated: 2019-05-21
Results Overview
To evaluate the safety of Fludarabine/Busulfan as conditioned regimen for allogeneic stem cell transplantation in patients with myelofibrosis/myelodysplastic syndrome at 100 days post-transplant
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
63 participants
Primary outcome timeframe
Non-relapse mortality at 100 days post-transplant
Results posted on
2019-05-21
Participant Flow
Participant milestones
| Measure |
Participants With Myelofibrosis and Myelodysplastic Syndrome
Allogeneic stem cell transplantation (SCT) with preparative regimen of fludarabine and busulfan in myelofibrosis and myelodysplastic syndrome
|
|---|---|
|
Overall Study
STARTED
|
63
|
|
Overall Study
COMPLETED
|
58
|
|
Overall Study
NOT COMPLETED
|
5
|
Reasons for withdrawal
| Measure |
Participants With Myelofibrosis and Myelodysplastic Syndrome
Allogeneic stem cell transplantation (SCT) with preparative regimen of fludarabine and busulfan in myelofibrosis and myelodysplastic syndrome
|
|---|---|
|
Overall Study
Physician Decision
|
3
|
|
Overall Study
Insurance Denial
|
1
|
|
Overall Study
Donor not able to donate the last minute
|
1
|
Baseline Characteristics
Allogeneic Stem Cell Transplantation for Myelofibrosis and Myelodysplastic Syndrome
Baseline characteristics by cohort
| Measure |
Participants With Myelofibrosis and Myelodysplastic Syndrome
n=58 Participants
Allogeneic stem cell transplantation (SCT) with preparative regimen of fludarabine and busulfan in myelofibrosis and myelodysplastic syndrome
|
|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=99 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
45 Participants
n=99 Participants
|
|
Age, Categorical
>=65 years
|
13 Participants
n=99 Participants
|
|
Age, Continuous
|
59 years
n=99 Participants
|
|
Sex: Female, Male
Female
|
26 Participants
n=99 Participants
|
|
Sex: Female, Male
Male
|
32 Participants
n=99 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=99 Participants
|
|
Race (NIH/OMB)
Asian
|
1 Participants
n=99 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=99 Participants
|
|
Race (NIH/OMB)
Black or African American
|
4 Participants
n=99 Participants
|
|
Race (NIH/OMB)
White
|
53 Participants
n=99 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=99 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=99 Participants
|
|
Region of Enrollment
United States
|
58 participants
n=99 Participants
|
PRIMARY outcome
Timeframe: Non-relapse mortality at 100 days post-transplantTo evaluate the safety of Fludarabine/Busulfan as conditioned regimen for allogeneic stem cell transplantation in patients with myelofibrosis/myelodysplastic syndrome at 100 days post-transplant
Outcome measures
| Measure |
Participants With Myelofibrosis and Myelodysplastic Syndrome
n=58 Participants
Allogeneic stem cell transplantation (SCT) with preparative regimen of fludarabine and busulfan in myelofibrosis and myelodysplastic syndrome
|
|---|---|
|
Rate of Non-relapse Mortality at 100 Days Post-transplant
Infections
|
1 Participants
|
|
Rate of Non-relapse Mortality at 100 Days Post-transplant
Organ Failures
|
2 Participants
|
|
Rate of Non-relapse Mortality at 100 Days Post-transplant
Sudden death: likely due to ischemic cardiac event
|
1 Participants
|
SECONDARY outcome
Timeframe: Up to 3 years post-transplantEfficacy Assessed as Number of Participants with Overall Survival, Leukemia Progression, Primary Graft Failure and Complete Hematological Response. Primary graft failure is defined as failure to achieve an ANC \>/= 0.5 x 10 (9)/L for 3 consecutive days and evidence of donor chimerism by Day +28. Complete hematological response is defined by hemoglobin \>/= 120 g/L; or achievement of transfusion independence, with stable Hb \> 110 g/L, for RBC transfusion-dependent participants; Spleen not palpable; platelet count 150 x 10 (9)/L; White blood cell 4 x 10 (9)/L to 10 x 10(9)/L.
Outcome measures
| Measure |
Participants With Myelofibrosis and Myelodysplastic Syndrome
n=58 Participants
Allogeneic stem cell transplantation (SCT) with preparative regimen of fludarabine and busulfan in myelofibrosis and myelodysplastic syndrome
|
|---|---|
|
Efficacy of This Therapy 3 Years Post-transplant
Overall survival at 3 years
|
25 Participants
|
|
Efficacy of This Therapy 3 Years Post-transplant
Leukemia progression
|
27 Participants
|
|
Efficacy of This Therapy 3 Years Post-transplant
Primary graft failures
|
0 Participants
|
|
Efficacy of This Therapy 3 Years Post-transplant
Complete hematological remission
|
44 Participants
|
Adverse Events
Participants With Myelofibrosis and Myelodysplastic Syndrome
Serious events: 18 serious events
Other events: 58 other events
Deaths: 8 deaths
Serious adverse events
| Measure |
Participants With Myelofibrosis and Myelodysplastic Syndrome
n=58 participants at risk
Allogeneic stem cell transplantation (SCT) with preparative regimen of fludarabine and busulfan in myelofibrosis and myelodysplastic syndrome
|
|---|---|
|
Gastrointestinal disorders
GI GvHD
|
3.4%
2/58 • January 2006 to May 2012
|
|
Skin and subcutaneous tissue disorders
Skin GvHD
|
1.7%
1/58 • January 2006 to May 2012
|
|
Infections and infestations
Infectious pneumonia
|
5.2%
3/58 • January 2006 to May 2012
|
|
Hepatobiliary disorders
Veno-occlusive disease
|
3.4%
2/58 • January 2006 to May 2012
|
|
Blood and lymphatic system disorders
Liver GvHD
|
1.7%
1/58 • January 2006 to May 2012
|
|
Blood and lymphatic system disorders
Secondary graft failure
|
10.3%
6/58 • January 2006 to May 2012
|
|
Blood and lymphatic system disorders
Hemolytic anemia
|
1.7%
1/58 • January 2006 to May 2012
|
|
Blood and lymphatic system disorders
Thrombocytopenia
|
5.2%
3/58 • January 2006 to May 2012
|
|
Renal and urinary disorders
Renal failure
|
3.4%
2/58 • January 2006 to May 2012
|
|
Hepatobiliary disorders
Elevated transminitis
|
10.3%
6/58 • January 2006 to May 2012
|
|
Infections and infestations
Septic shock
|
1.7%
1/58 • January 2006 to May 2012
|
Other adverse events
| Measure |
Participants With Myelofibrosis and Myelodysplastic Syndrome
n=58 participants at risk
Allogeneic stem cell transplantation (SCT) with preparative regimen of fludarabine and busulfan in myelofibrosis and myelodysplastic syndrome
|
|---|---|
|
Infections and infestations
Infections
|
58.6%
34/58 • January 2006 to May 2012
|
|
Blood and lymphatic system disorders
Poor graft function
|
1.7%
1/58 • January 2006 to May 2012
|
|
Blood and lymphatic system disorders
ABO incompatibility
|
1.7%
1/58 • January 2006 to May 2012
|
|
Investigations
ATG induced fevers
|
41.4%
24/58 • January 2006 to May 2012
|
|
Investigations
Fluid overload
|
37.9%
22/58 • January 2006 to May 2012
|
|
Gastrointestinal disorders
GI GvHD
|
10.3%
6/58 • January 2006 to May 2012
|
|
Gastrointestinal disorders
Mucositis
|
74.1%
43/58 • January 2006 to May 2012
|
|
Gastrointestinal disorders
Upper GI GvHD
|
19.0%
11/58 • January 2006 to May 2012
|
|
Renal and urinary disorders
Renal insufficiency
|
13.8%
8/58 • January 2006 to May 2012
|
|
Hepatobiliary disorders
Liver GvHD
|
8.6%
5/58 • January 2006 to May 2012
|
|
Hepatobiliary disorders
Veno-occlusive disease
|
1.7%
1/58 • January 2006 to May 2012
|
|
Investigations
Neutropenic fevers
|
24.1%
14/58 • January 2006 to May 2012
|
|
General disorders
Headaches
|
8.6%
5/58 • January 2006 to May 2012
|
|
Infections and infestations
Infectious pneumonia
|
15.5%
9/58 • January 2006 to May 2012
|
|
Respiratory, thoracic and mediastinal disorders
ATG induced shortness of breath
|
3.4%
2/58 • January 2006 to May 2012
|
|
Skin and subcutaneous tissue disorders
Skin GvHD
|
24.1%
14/58 • January 2006 to May 2012
|
|
Cardiac disorders
Hypertension
|
5.2%
3/58 • January 2006 to May 2012
|
|
Hepatobiliary disorders
Ascites
|
3.4%
2/58 • January 2006 to May 2012
|
|
Gastrointestinal disorders
Diarrhea
|
19.0%
11/58 • January 2006 to May 2012
|
|
Renal and urinary disorders
Hemorrhagic cystits
|
3.4%
2/58 • January 2006 to May 2012
|
|
Gastrointestinal disorders
Nausea
|
87.9%
51/58 • January 2006 to May 2012
|
|
Infections and infestations
BK virus associated hemorrhagic cystitis
|
8.6%
5/58 • January 2006 to May 2012
|
|
Hepatobiliary disorders
Elevated transminitis
|
34.5%
20/58 • January 2006 to May 2012
|
|
Nervous system disorders
Altered mental status changes
|
5.2%
3/58 • January 2006 to May 2012
|
|
Investigations
Cytokine release syndrome
|
3.4%
2/58 • January 2006 to May 2012
|
|
Skin and subcutaneous tissue disorders
Cellulitis
|
3.4%
2/58 • January 2006 to May 2012
|
|
Skin and subcutaneous tissue disorders
ATG induced skin rash
|
1.7%
1/58 • January 2006 to May 2012
|
|
Investigations
Fevers
|
6.9%
4/58 • January 2006 to May 2012
|
Additional Information
Popat,Uday,M.D. / Stem Cell Transplantation
UT MD Anderson Cancer Center
Phone: 713-745-3055
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place