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Tacrolimus and Sirolimus as Prophylaxis After Allogenic Non-myeloablative Peripheral Blood Stem Cell Transplantation

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ClinicalTrials.gov Identifier: NCT00282282
Recruitment Status : Completed
First Posted : January 26, 2006
Results First Posted : April 8, 2014
Last Update Posted : May 12, 2014
Sponsor:
Collaborator:
Brigham and Women's Hospital
Information provided by (Responsible Party):
Vincent T. Ho, MD, Dana-Farber Cancer Institute

Study Type Interventional
Study Design Allocation: N/A;   Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Conditions Graft Versus Host Disease
GVHD
Interventions Drug: tacrolimus
Drug: sirolimus
Drug: fludarabine
Drug: busulfex
Enrollment 31
Recruitment Details Patients with hematologic malignancies who were at high risk of complications after conventional transplantation, with 6/6 HLA matched-related donors were approached with information about participating in the study. Participants were approached at either inpatient or outpatient clinics by physicians between 2006 and 2007.
Pre-assignment Details  
Arm/Group Title Tacrolimus and Sirolimus
Hide Arm/Group Description Participants received a tacrolimus and sirolimus graft-versus-host disease (GVHD) prophylaxis regimen. Tacrolimus was given 0.05 mg/kg/day (in 2 daily divided doses) orally starting 3 days before bone marrow transplant with a target serum concentration of 5-10ng/mL. Sirolimus was administered with a 12mg oral loading dose 3 days prior to transplantwith a target serum concentration of 3-12ng/mL. Tapering of tacrolimus and sirolimus doses was encouraged starting 64 days after transplant with a goal of discontinuing immunosuppression therapy approximately 6 months after transplant if there were no signs of GVHD.
Period Title: Overall Study
Started 29 [1]
Completed 29
Not Completed 0
[1]
31 patients enrolled but 2 patients did not go through with transplant
Arm/Group Title Tacrolimus and Sirolimus
Hide Arm/Group Description Participants received a tacrolimus and sirolimus graft-versus-host disease (GVHD) prophylaxis regimen. Tacrolimus was given 0.05 mg/kg/day (in 2 daily divided doses) orally starting 3 days before bone marrow transplant with a target serum concentration of 5-10ng/mL. Sirolimus was administered with a 12mg oral loading dose 3 days prior to transplantwith a target serum concentration of 3-12ng/mL. Tapering of tacrolimus and sirolimus doses was encouraged starting 64 days after transplant with a goal of discontinuing immunosuppression therapy approximately 6 months after transplant if there were no signs of GVHD.
Overall Number of Baseline Participants 29
Hide Baseline Analysis Population Description
[Not Specified]
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 29 participants
<=18 years
0
   0.0%
Between 18 and 65 years
29
 100.0%
>=65 years
0
   0.0%
Age, Continuous  
Median (Full Range)
Unit of measure:  Years
Number Analyzed 29 participants
53
(29 to 64)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 29 participants
Female
12
  41.4%
Male
17
  58.6%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
United States Number Analyzed 29 participants
29
1.Primary Outcome
Title Incidence of Grade II-IV Acute GVHD (aGVHD) Developing by Day 100 Following Non-myeloablative PBSC Transplantation Using Tacrolimus and Sirolimus.
Hide Description All participants received tacrolimus and sirolimus in this one arm study. There were no participants considered unevaluable for this measure (deceased prior to day 100). The total number of people who developed grade II-IV aGVHD before day 100 are reported here.
Time Frame 100 days
Hide Outcome Measure Data
Hide Analysis Population Description
Participants who lived more than 30 days posttransplant were considered evaluable. Incidence of grade II-IV aGVHD was adjusted for participants who had aGVHD off-treatment.
Arm/Group Title Tacrolimus and Sirolimus
Hide Arm/Group Description:
[Not Specified]
Overall Number of Participants Analyzed 29
Measure Type: Number
Unit of Measure: participants
5
2.Secondary Outcome
Title Percentage of Participants With ≥90 Percent Donor-derived Hematopoeisis Around 100 Days Post Transplantation
Hide Description The percentage of participants with ≥90 percent donor-derived hematopoeisis was assessed around day +100 using peripheral blood chimerism.
Time Frame 100 days
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Tacrolimus and Sirolimus
Hide Arm/Group Description:
[Not Specified]
Overall Number of Participants Analyzed 27
Measure Type: Number
Unit of Measure: percentage of participants
78
3.Secondary Outcome
Title Disease Response.
Hide Description Disease response was assessed as 2 year progression-free survival. The median follow-up time was 1.84 years. The percentage of participants with who reached this timepoint with no disease progression are reported.
Time Frame 2 years
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Tacrolimus and Sirolimus GVHD Prophylaxis
Hide Arm/Group Description:
[Not Specified]
Overall Number of Participants Analyzed 29
Measure Type: Number
Number (95% Confidence Interval)
Unit of Measure: percentage of participants
48
(29 to 64)
Time Frame Participants were followed for approximately 2 years post-transplant
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Tacrolimus and Sirolimus
Hide Arm/Group Description Participants received a tacrolimus and sirolimus graft-versus-host disease (GVHD) prophylaxis regimen. Tacrolimus was given 0.05 mg/kg/day (in 2 daily divided doses) orally starting 3 days before bone marrow transplant with a target serum concentration of 5-10ng/mL. Sirolimus was administered with a 12mg oral loading dose 3 days prior to transplantwith a target serum concentration of 3-12ng/mL. Tapering of tacrolimus and sirolimus doses was encouraged starting 64 days after transplant with a goal of discontinuing immunosuppression therapy approximately 6 months after transplant if there were no signs of GVHD.
All-Cause Mortality
Tacrolimus and Sirolimus
Affected / at Risk (%)
Total   --/-- 
Hide Serious Adverse Events
Tacrolimus and Sirolimus
Affected / at Risk (%)
Total   1/29 (3.45%) 
Blood and lymphatic system disorders   
Thrombotic Microangiopathy  1 [1]  1/29 (3.45%) 
Indicates events were collected by systematic assessment
1
Term from vocabulary, CTCAE 3.0
[1]
1 patient had transplantation-associated thrombotic microangiopathy that resolved with the discontinuation of tacrolimus
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
Tacrolimus and Sirolimus
Affected / at Risk (%)
Total   0/29 (0.00%) 
This study is a single-institution study, and as such the data are limited by the small study population.
Certain Agreements
All Principal Investigators ARE employed by the organization sponsoring the study.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Vincent T. Ho, MD Associate Professor of Medicine, Harvard Medical School
Organization: Dana-Farber Cancer Institute
Phone: (617) 632-5938
EMail: vincent_ho@dfci.harvard.edu
Layout table for additonal information
Responsible Party: Vincent T. Ho, MD, Dana-Farber Cancer Institute
ClinicalTrials.gov Identifier: NCT00282282    
Other Study ID Numbers: 05-362
First Submitted: January 24, 2006
First Posted: January 26, 2006
Results First Submitted: January 11, 2013
Results First Posted: April 8, 2014
Last Update Posted: May 12, 2014