Trial Outcomes & Findings for Stem Cell Transplant for Hemoglobinopathy (NCT NCT00176852)
NCT ID: NCT00176852
Last Updated: 2020-02-27
Results Overview
In general, grade 3 equates to moderate, grade 4 to severe and grade 5 to death.
Recruitment status
COMPLETED
Study phase
PHASE2/PHASE3
Target enrollment
22 participants
Primary outcome timeframe
1 year
Results posted on
2020-02-27
Participant Flow
Participant milestones
| Measure |
Full Conditioning (Discontinued / A)
Full Preparative Regimen for subjects with matched donors using Busulfan on Day -8 and -7, Fludarabine on Day -6 through -2, antithymocyte globulin (ATG) on Day -2 through -1, total lymphoid radiation (TLI) on Day -1, stem cell infusion on Day 0.
Busulfan, Fludarabine, ATG, TLI: Busulfan 0.8 mg/kg/dose intravenous (IV) Days -8 and -7 Fludarabine 35 mg/m2 IV Days -6 through -2 Antithymocyte globulin (ATG) 30 mg/kg IV Days -2 and -1 Total lymphoid radiation 300 cGy
|
Busulfan Conditioning (B)
Myeloablative Preparative Regimen for subjects with HLA identical sibling donors consists of Busulfan on day -9 through -6, Cyclophosphamide on day -5 through -2, ATG on day -3 through -1, stem cell infusion on Day 0 and Granulocyte Colony Stimulating Factor on day -3 until ANC \>2500 x 2 days.
Busulfan, Cyclophosphamide, ATG, GCSF: Busulfan 0.8 mg/kg/dose intravenous (IV) Days -9 through -6 Cyclophosphamide 50 mg/kg IV Days -5 through -2 ATG 30 mg/kg IV Day -1 GCSF 5 mcg/kg/day IV until ANC \>2500 x 2 days.
Total Body Irradiation: 300 cGY Day -1
Stem cell infusion: Given Day 0
|
Campath and TBI Conditioning (A2)
Patients with sickle cell disease or thalassemia who do not have an HLA-identical sibling donor or who has pre-existing organ dysfunction making myeloablative condition ineligible will receive Campath on day -10 through -6, Cyclophosphamide on day -7, Fludarabine on day -6 through -2, total body irradiation (TBI) on day -1, stem cell infusion on Day 0.
Campath, Fludarabine, Cyclophosphamide: Receives Campath-1H 0.2 mg/kg Days -10 through -6, Fludarabine 35 mg/m2 intravenous (IV) Days -6 through -2, total body irradiation (TBI) 300 cGy Day -1.
Total Body Irradiation: 300 cGY Day -1
Stem cell infusion: Given Day 0
|
|---|---|---|---|
|
Overall Study
STARTED
|
3
|
5
|
14
|
|
Overall Study
COMPLETED
|
3
|
5
|
14
|
|
Overall Study
NOT COMPLETED
|
0
|
0
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Stem Cell Transplant for Hemoglobinopathy
Baseline characteristics by cohort
| Measure |
Full Conditioning (Discontinued / A)
n=3 Participants
Full Preparative Regimen for subjects with matched donors using Busulfan on Day -8 and -7, Fludarabine on Day -6 through -2, antithymocyte globulin (ATG) on Day -2 through -1, total lymphoid radiation (TLI) on Day -1, stem cell infusion on Day 0.
Busulfan, Fludarabine, ATG, TLI: Busulfan 0.8 mg/kg/dose intravenous (IV) Days -8 and -7 Fludarabine 35 mg/m2 IV Days -6 through -2 Antithymocyte globulin (ATG) 30 mg/kg IV Days -2 and -1 Total lymphoid radiation 300 cGy
|
Busulfan Conditioning (B)
n=5 Participants
Myeloablative Preparative Regimen for subjects with HLA identical sibling donors consists of Busulfan on day -9 through -6, Cyclophosphamide on day -5 through -2, ATG on day -3 through -1, stem cell infusion on Day 0 and Granulocyte Colony Stimulating Factor on day -3 until ANC \>2500 x 2 days.
Busulfan, Cyclophosphamide, ATG, GCSF: Busulfan 0.8 mg/kg/dose intravenous (IV) Days -9 through -6 Cyclophosphamide 50 mg/kg IV Days -5 through -2 ATG 30 mg/kg IV Day -1 GCSF 5 mcg/kg/day IV until ANC \>2500 x 2 days.
Total Body Irradiation: 300 cGY Day -1
Stem cell infusion: Given Day 0
|
Campath and TBI Conditioning (A2)
n=14 Participants
Patients with sickle cell disease or thalassemia who do not have an HLA-identical sibling donor or who has pre-existing organ dysfunction making myeloablative condition ineligible will receive Campath on day -10 through -6, Cyclophosphamide on day -7, Fludarabine on day -6 through -2, total body irradiation (TBI) on day -1, stem cell infusion on Day 0.
Campath, Fludarabine, Cyclophosphamide: Receives Campath-1H 0.2 mg/kg Days -10 through -6, Fludarabine 35 mg/m2 intravenous (IV) Days -6 through -2, total body irradiation (TBI) 300 cGy Day -1.
Total Body Irradiation: 300 cGY Day -1
Stem cell infusion: Given Day 0
|
Total
n=22 Participants
Total of all reporting groups
|
|---|---|---|---|---|
|
Age, Categorical
<=18 years
|
3 Participants
n=5 Participants
|
5 Participants
n=7 Participants
|
12 Participants
n=5 Participants
|
20 Participants
n=4 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
2 Participants
n=5 Participants
|
2 Participants
n=4 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
|
Sex: Female, Male
Female
|
2 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
8 Participants
n=5 Participants
|
11 Participants
n=4 Participants
|
|
Sex: Female, Male
Male
|
1 Participants
n=5 Participants
|
4 Participants
n=7 Participants
|
6 Participants
n=5 Participants
|
11 Participants
n=4 Participants
|
PRIMARY outcome
Timeframe: 1 yearIn general, grade 3 equates to moderate, grade 4 to severe and grade 5 to death.
Outcome measures
| Measure |
RIC Bu/Flu (A) (Discontinued)
n=3 Participants
Full Preparative Regimen for subjects with matched donors using Busulfan on Day -8 and -7, Fludarabine on Day -6 through -2, antithymocyte globulin (ATG) on Day -2 through -1, total lymphoid radiation (TLI) on Day -1, stem cell infusion on Day 0.
Busulfan, Fludarabine, ATG, TLI: Busulfan 0.8 mg/kg/dose intravenous (IV) Days -8 and -7 Fludarabine 35 mg/m2 IV Days -6 through -2 Antithymocyte globulin (ATG) 30 mg/kg IV Days -2 and -1 Total lymphoid radiation 300 cGy
|
MA Bu/Cy (B)
n=5 Participants
Myeloablative Preparative Regimen for subjects with HLA identical sibling donors consists of Busulfan on day -9 through -6, Cyclophosphamide on day -5 through -2, ATG on day -3 through -1, stem cell infusion on Day 0 and Granulocyte Colony Stimulating Factor on day -3 until ANC \>2500 x 2 days.
Busulfan, Cyclophosphamide, ATG, GCSF: Busulfan 0.8 mg/kg/dose intravenous (IV) Days -9 through -6 Cyclophosphamide 50 mg/kg IV Days -5 through -2 ATG 30 mg/kg IV Day -1 GCSF 5 mcg/kg/day IV until ANC \>2500 x 2 days.
Total Body Irradiation: 300 cGY Day -1
Stem cell infusion: Given Day 0
|
RIC Cy/Flu/TBI (A2)
n=14 Participants
Patients with sickle cell disease or thalassemia who do not have an HLA-identical sibling donor or who has pre-existing organ dysfunction making myeloablative condition ineligible will receive Campath on day -10 through -6, Cyclophosphamide on day -7, Fludarabine on day -6 through -2, total body irradiation (TBI) on day -1, stem cell infusion on Day 0.
Campath, Fludarabine, Cyclophosphamide: Receives Campath-1H 0.2 mg/kg Days -10 through -6, Fludarabine 35 mg/m2 intravenous (IV) Days -6 through -2, total body irradiation (TBI) 300 cGy Day -1.
Total Body Irradiation: 300 cGY Day -1
Stem cell infusion: Given Day 0
|
|---|---|---|---|
|
Number of Patients Who Experienced Grade 3-5 Treatment Related Toxicity
|
0 Participants
|
0 Participants
|
2 Participants
|
SECONDARY outcome
Timeframe: 100 daysPopulation: One of the 3 patients treated on Arm A was retreated at Day 40 and was not evaluable. One of the 14 patients on Arm A2 died before 100 days.
The number of patients whose blood and/or bone marrow contains \> 10% donor cells.
Outcome measures
| Measure |
RIC Bu/Flu (A) (Discontinued)
n=2 Participants
Full Preparative Regimen for subjects with matched donors using Busulfan on Day -8 and -7, Fludarabine on Day -6 through -2, antithymocyte globulin (ATG) on Day -2 through -1, total lymphoid radiation (TLI) on Day -1, stem cell infusion on Day 0.
Busulfan, Fludarabine, ATG, TLI: Busulfan 0.8 mg/kg/dose intravenous (IV) Days -8 and -7 Fludarabine 35 mg/m2 IV Days -6 through -2 Antithymocyte globulin (ATG) 30 mg/kg IV Days -2 and -1 Total lymphoid radiation 300 cGy
|
MA Bu/Cy (B)
n=5 Participants
Myeloablative Preparative Regimen for subjects with HLA identical sibling donors consists of Busulfan on day -9 through -6, Cyclophosphamide on day -5 through -2, ATG on day -3 through -1, stem cell infusion on Day 0 and Granulocyte Colony Stimulating Factor on day -3 until ANC \>2500 x 2 days.
Busulfan, Cyclophosphamide, ATG, GCSF: Busulfan 0.8 mg/kg/dose intravenous (IV) Days -9 through -6 Cyclophosphamide 50 mg/kg IV Days -5 through -2 ATG 30 mg/kg IV Day -1 GCSF 5 mcg/kg/day IV until ANC \>2500 x 2 days.
Total Body Irradiation: 300 cGY Day -1
Stem cell infusion: Given Day 0
|
RIC Cy/Flu/TBI (A2)
n=13 Participants
Patients with sickle cell disease or thalassemia who do not have an HLA-identical sibling donor or who has pre-existing organ dysfunction making myeloablative condition ineligible will receive Campath on day -10 through -6, Cyclophosphamide on day -7, Fludarabine on day -6 through -2, total body irradiation (TBI) on day -1, stem cell infusion on Day 0.
Campath, Fludarabine, Cyclophosphamide: Receives Campath-1H 0.2 mg/kg Days -10 through -6, Fludarabine 35 mg/m2 intravenous (IV) Days -6 through -2, total body irradiation (TBI) 300 cGy Day -1.
Total Body Irradiation: 300 cGY Day -1
Stem cell infusion: Given Day 0
|
|---|---|---|---|
|
The Incidence of Chimerism at 100 Days
|
1 Participants
|
5 Participants
|
11 Participants
|
SECONDARY outcome
Timeframe: 6 monthsPopulation: One of the 3 patients treated on Arm A was retreated at Day 40 and was not evaluable. One of the 14 patients on Arm A2 died before 6 months.
The number of patients whose blood and/or bone marrow contains \> 10% donor cells.
Outcome measures
| Measure |
RIC Bu/Flu (A) (Discontinued)
n=2 Participants
Full Preparative Regimen for subjects with matched donors using Busulfan on Day -8 and -7, Fludarabine on Day -6 through -2, antithymocyte globulin (ATG) on Day -2 through -1, total lymphoid radiation (TLI) on Day -1, stem cell infusion on Day 0.
Busulfan, Fludarabine, ATG, TLI: Busulfan 0.8 mg/kg/dose intravenous (IV) Days -8 and -7 Fludarabine 35 mg/m2 IV Days -6 through -2 Antithymocyte globulin (ATG) 30 mg/kg IV Days -2 and -1 Total lymphoid radiation 300 cGy
|
MA Bu/Cy (B)
n=5 Participants
Myeloablative Preparative Regimen for subjects with HLA identical sibling donors consists of Busulfan on day -9 through -6, Cyclophosphamide on day -5 through -2, ATG on day -3 through -1, stem cell infusion on Day 0 and Granulocyte Colony Stimulating Factor on day -3 until ANC \>2500 x 2 days.
Busulfan, Cyclophosphamide, ATG, GCSF: Busulfan 0.8 mg/kg/dose intravenous (IV) Days -9 through -6 Cyclophosphamide 50 mg/kg IV Days -5 through -2 ATG 30 mg/kg IV Day -1 GCSF 5 mcg/kg/day IV until ANC \>2500 x 2 days.
Total Body Irradiation: 300 cGY Day -1
Stem cell infusion: Given Day 0
|
RIC Cy/Flu/TBI (A2)
n=13 Participants
Patients with sickle cell disease or thalassemia who do not have an HLA-identical sibling donor or who has pre-existing organ dysfunction making myeloablative condition ineligible will receive Campath on day -10 through -6, Cyclophosphamide on day -7, Fludarabine on day -6 through -2, total body irradiation (TBI) on day -1, stem cell infusion on Day 0.
Campath, Fludarabine, Cyclophosphamide: Receives Campath-1H 0.2 mg/kg Days -10 through -6, Fludarabine 35 mg/m2 intravenous (IV) Days -6 through -2, total body irradiation (TBI) 300 cGy Day -1.
Total Body Irradiation: 300 cGY Day -1
Stem cell infusion: Given Day 0
|
|---|---|---|---|
|
The Incidence of Chimerism at 6 Months
|
1 Participants
|
5 Participants
|
8 Participants
|
SECONDARY outcome
Timeframe: 1 yearPopulation: One of the 3 patients treated on Arm A was retreated at Day 40 and was not evaluable. One of the 14 patients on Arm A2 died before 1 year.
The number of patients whose blood and/or bone marrow contains \> 10% donor cells.
Outcome measures
| Measure |
RIC Bu/Flu (A) (Discontinued)
n=2 Participants
Full Preparative Regimen for subjects with matched donors using Busulfan on Day -8 and -7, Fludarabine on Day -6 through -2, antithymocyte globulin (ATG) on Day -2 through -1, total lymphoid radiation (TLI) on Day -1, stem cell infusion on Day 0.
Busulfan, Fludarabine, ATG, TLI: Busulfan 0.8 mg/kg/dose intravenous (IV) Days -8 and -7 Fludarabine 35 mg/m2 IV Days -6 through -2 Antithymocyte globulin (ATG) 30 mg/kg IV Days -2 and -1 Total lymphoid radiation 300 cGy
|
MA Bu/Cy (B)
n=5 Participants
Myeloablative Preparative Regimen for subjects with HLA identical sibling donors consists of Busulfan on day -9 through -6, Cyclophosphamide on day -5 through -2, ATG on day -3 through -1, stem cell infusion on Day 0 and Granulocyte Colony Stimulating Factor on day -3 until ANC \>2500 x 2 days.
Busulfan, Cyclophosphamide, ATG, GCSF: Busulfan 0.8 mg/kg/dose intravenous (IV) Days -9 through -6 Cyclophosphamide 50 mg/kg IV Days -5 through -2 ATG 30 mg/kg IV Day -1 GCSF 5 mcg/kg/day IV until ANC \>2500 x 2 days.
Total Body Irradiation: 300 cGY Day -1
Stem cell infusion: Given Day 0
|
RIC Cy/Flu/TBI (A2)
n=13 Participants
Patients with sickle cell disease or thalassemia who do not have an HLA-identical sibling donor or who has pre-existing organ dysfunction making myeloablative condition ineligible will receive Campath on day -10 through -6, Cyclophosphamide on day -7, Fludarabine on day -6 through -2, total body irradiation (TBI) on day -1, stem cell infusion on Day 0.
Campath, Fludarabine, Cyclophosphamide: Receives Campath-1H 0.2 mg/kg Days -10 through -6, Fludarabine 35 mg/m2 intravenous (IV) Days -6 through -2, total body irradiation (TBI) 300 cGy Day -1.
Total Body Irradiation: 300 cGY Day -1
Stem cell infusion: Given Day 0
|
|---|---|---|---|
|
The Incidence of Chimerism at 1 Year
|
1 Participants
|
5 Participants
|
8 Participants
|
SECONDARY outcome
Timeframe: 100 daysThe number of patients who experienced grades 2-4 Acute GVHD. Acute GVHD is when the donated bone marrow or peripheral blood stem cells view the recipient's body as foreign, and the donated cells/bone marrow attack the body. Grades 2-4 equate to mild to severe disease. Symptoms typically appear within weeks after transplant.
Outcome measures
| Measure |
RIC Bu/Flu (A) (Discontinued)
n=3 Participants
Full Preparative Regimen for subjects with matched donors using Busulfan on Day -8 and -7, Fludarabine on Day -6 through -2, antithymocyte globulin (ATG) on Day -2 through -1, total lymphoid radiation (TLI) on Day -1, stem cell infusion on Day 0.
Busulfan, Fludarabine, ATG, TLI: Busulfan 0.8 mg/kg/dose intravenous (IV) Days -8 and -7 Fludarabine 35 mg/m2 IV Days -6 through -2 Antithymocyte globulin (ATG) 30 mg/kg IV Days -2 and -1 Total lymphoid radiation 300 cGy
|
MA Bu/Cy (B)
n=5 Participants
Myeloablative Preparative Regimen for subjects with HLA identical sibling donors consists of Busulfan on day -9 through -6, Cyclophosphamide on day -5 through -2, ATG on day -3 through -1, stem cell infusion on Day 0 and Granulocyte Colony Stimulating Factor on day -3 until ANC \>2500 x 2 days.
Busulfan, Cyclophosphamide, ATG, GCSF: Busulfan 0.8 mg/kg/dose intravenous (IV) Days -9 through -6 Cyclophosphamide 50 mg/kg IV Days -5 through -2 ATG 30 mg/kg IV Day -1 GCSF 5 mcg/kg/day IV until ANC \>2500 x 2 days.
Total Body Irradiation: 300 cGY Day -1
Stem cell infusion: Given Day 0
|
RIC Cy/Flu/TBI (A2)
n=14 Participants
Patients with sickle cell disease or thalassemia who do not have an HLA-identical sibling donor or who has pre-existing organ dysfunction making myeloablative condition ineligible will receive Campath on day -10 through -6, Cyclophosphamide on day -7, Fludarabine on day -6 through -2, total body irradiation (TBI) on day -1, stem cell infusion on Day 0.
Campath, Fludarabine, Cyclophosphamide: Receives Campath-1H 0.2 mg/kg Days -10 through -6, Fludarabine 35 mg/m2 intravenous (IV) Days -6 through -2, total body irradiation (TBI) 300 cGy Day -1.
Total Body Irradiation: 300 cGY Day -1
Stem cell infusion: Given Day 0
|
|---|---|---|---|
|
The Incidence of Grade 2-4 Acute Graft Versus Host Disease (Acute GVHD)
|
0 Participants
|
0 Participants
|
0 Participants
|
SECONDARY outcome
Timeframe: 100 daysThe number of patients who experienced grades 3-4 Acute GVHD. Acute GVHD is when the donated bone marrow or peripheral blood stem cells view the recipient's body as foreign, and the donated cells/bone marrow attack the body. IGrades 3-4 equate to moderate to severe disease. Symptoms typically appear within weeks after transplant.
Outcome measures
| Measure |
RIC Bu/Flu (A) (Discontinued)
n=3 Participants
Full Preparative Regimen for subjects with matched donors using Busulfan on Day -8 and -7, Fludarabine on Day -6 through -2, antithymocyte globulin (ATG) on Day -2 through -1, total lymphoid radiation (TLI) on Day -1, stem cell infusion on Day 0.
Busulfan, Fludarabine, ATG, TLI: Busulfan 0.8 mg/kg/dose intravenous (IV) Days -8 and -7 Fludarabine 35 mg/m2 IV Days -6 through -2 Antithymocyte globulin (ATG) 30 mg/kg IV Days -2 and -1 Total lymphoid radiation 300 cGy
|
MA Bu/Cy (B)
n=5 Participants
Myeloablative Preparative Regimen for subjects with HLA identical sibling donors consists of Busulfan on day -9 through -6, Cyclophosphamide on day -5 through -2, ATG on day -3 through -1, stem cell infusion on Day 0 and Granulocyte Colony Stimulating Factor on day -3 until ANC \>2500 x 2 days.
Busulfan, Cyclophosphamide, ATG, GCSF: Busulfan 0.8 mg/kg/dose intravenous (IV) Days -9 through -6 Cyclophosphamide 50 mg/kg IV Days -5 through -2 ATG 30 mg/kg IV Day -1 GCSF 5 mcg/kg/day IV until ANC \>2500 x 2 days.
Total Body Irradiation: 300 cGY Day -1
Stem cell infusion: Given Day 0
|
RIC Cy/Flu/TBI (A2)
n=14 Participants
Patients with sickle cell disease or thalassemia who do not have an HLA-identical sibling donor or who has pre-existing organ dysfunction making myeloablative condition ineligible will receive Campath on day -10 through -6, Cyclophosphamide on day -7, Fludarabine on day -6 through -2, total body irradiation (TBI) on day -1, stem cell infusion on Day 0.
Campath, Fludarabine, Cyclophosphamide: Receives Campath-1H 0.2 mg/kg Days -10 through -6, Fludarabine 35 mg/m2 intravenous (IV) Days -6 through -2, total body irradiation (TBI) 300 cGy Day -1.
Total Body Irradiation: 300 cGY Day -1
Stem cell infusion: Given Day 0
|
|---|---|---|---|
|
The Incidence of Grade 3-4 Acute Graft Versus Host Disease (Acute GVHD)
|
0 Participants
|
0 Participants
|
0 Participants
|
SECONDARY outcome
Timeframe: 6 monthsThe number of patients who experienced Chronic GVHD. Chronic GVHD is when the donated bone marrow or peripheral blood stem cells view the recipient's body as foreign, and the donated cells/bone marrow attack the body. Chronic GVHD can appear at any time after allogeneic transplant or several years after transplant.
Outcome measures
| Measure |
RIC Bu/Flu (A) (Discontinued)
n=3 Participants
Full Preparative Regimen for subjects with matched donors using Busulfan on Day -8 and -7, Fludarabine on Day -6 through -2, antithymocyte globulin (ATG) on Day -2 through -1, total lymphoid radiation (TLI) on Day -1, stem cell infusion on Day 0.
Busulfan, Fludarabine, ATG, TLI: Busulfan 0.8 mg/kg/dose intravenous (IV) Days -8 and -7 Fludarabine 35 mg/m2 IV Days -6 through -2 Antithymocyte globulin (ATG) 30 mg/kg IV Days -2 and -1 Total lymphoid radiation 300 cGy
|
MA Bu/Cy (B)
n=5 Participants
Myeloablative Preparative Regimen for subjects with HLA identical sibling donors consists of Busulfan on day -9 through -6, Cyclophosphamide on day -5 through -2, ATG on day -3 through -1, stem cell infusion on Day 0 and Granulocyte Colony Stimulating Factor on day -3 until ANC \>2500 x 2 days.
Busulfan, Cyclophosphamide, ATG, GCSF: Busulfan 0.8 mg/kg/dose intravenous (IV) Days -9 through -6 Cyclophosphamide 50 mg/kg IV Days -5 through -2 ATG 30 mg/kg IV Day -1 GCSF 5 mcg/kg/day IV until ANC \>2500 x 2 days.
Total Body Irradiation: 300 cGY Day -1
Stem cell infusion: Given Day 0
|
RIC Cy/Flu/TBI (A2)
n=14 Participants
Patients with sickle cell disease or thalassemia who do not have an HLA-identical sibling donor or who has pre-existing organ dysfunction making myeloablative condition ineligible will receive Campath on day -10 through -6, Cyclophosphamide on day -7, Fludarabine on day -6 through -2, total body irradiation (TBI) on day -1, stem cell infusion on Day 0.
Campath, Fludarabine, Cyclophosphamide: Receives Campath-1H 0.2 mg/kg Days -10 through -6, Fludarabine 35 mg/m2 intravenous (IV) Days -6 through -2, total body irradiation (TBI) 300 cGy Day -1.
Total Body Irradiation: 300 cGY Day -1
Stem cell infusion: Given Day 0
|
|---|---|---|---|
|
The Incidence of Chronic Graft Versus Host Disease (Chronic GVHD)
|
0 Participants
|
0 Participants
|
0 Participants
|
SECONDARY outcome
Timeframe: 1 yearThe number of patients who experienced Chronic GVHD. Chronic GVHD is when the donated bone marrow or peripheral blood stem cells view the recipient's body as foreign, and the donated cells/bone marrow attack the body. Chronic GVHD can appear at any time after allogeneic transplant or several years after transplant.
Outcome measures
| Measure |
RIC Bu/Flu (A) (Discontinued)
n=3 Participants
Full Preparative Regimen for subjects with matched donors using Busulfan on Day -8 and -7, Fludarabine on Day -6 through -2, antithymocyte globulin (ATG) on Day -2 through -1, total lymphoid radiation (TLI) on Day -1, stem cell infusion on Day 0.
Busulfan, Fludarabine, ATG, TLI: Busulfan 0.8 mg/kg/dose intravenous (IV) Days -8 and -7 Fludarabine 35 mg/m2 IV Days -6 through -2 Antithymocyte globulin (ATG) 30 mg/kg IV Days -2 and -1 Total lymphoid radiation 300 cGy
|
MA Bu/Cy (B)
n=5 Participants
Myeloablative Preparative Regimen for subjects with HLA identical sibling donors consists of Busulfan on day -9 through -6, Cyclophosphamide on day -5 through -2, ATG on day -3 through -1, stem cell infusion on Day 0 and Granulocyte Colony Stimulating Factor on day -3 until ANC \>2500 x 2 days.
Busulfan, Cyclophosphamide, ATG, GCSF: Busulfan 0.8 mg/kg/dose intravenous (IV) Days -9 through -6 Cyclophosphamide 50 mg/kg IV Days -5 through -2 ATG 30 mg/kg IV Day -1 GCSF 5 mcg/kg/day IV until ANC \>2500 x 2 days.
Total Body Irradiation: 300 cGY Day -1
Stem cell infusion: Given Day 0
|
RIC Cy/Flu/TBI (A2)
n=14 Participants
Patients with sickle cell disease or thalassemia who do not have an HLA-identical sibling donor or who has pre-existing organ dysfunction making myeloablative condition ineligible will receive Campath on day -10 through -6, Cyclophosphamide on day -7, Fludarabine on day -6 through -2, total body irradiation (TBI) on day -1, stem cell infusion on Day 0.
Campath, Fludarabine, Cyclophosphamide: Receives Campath-1H 0.2 mg/kg Days -10 through -6, Fludarabine 35 mg/m2 intravenous (IV) Days -6 through -2, total body irradiation (TBI) 300 cGy Day -1.
Total Body Irradiation: 300 cGY Day -1
Stem cell infusion: Given Day 0
|
|---|---|---|---|
|
The Incidence of Chronic Graft Versus Host Disease (Chronic GVHD)
|
0 Participants
|
0 Participants
|
0 Participants
|
SECONDARY outcome
Timeframe: pre-transplantThe measure for quality of life used in this study is the Karnofsky Performance Score. The Karnofsky Performance Score runs from 100 to 0, where 100 is "perfect" health and 0 is death.
Outcome measures
| Measure |
RIC Bu/Flu (A) (Discontinued)
n=3 Participants
Full Preparative Regimen for subjects with matched donors using Busulfan on Day -8 and -7, Fludarabine on Day -6 through -2, antithymocyte globulin (ATG) on Day -2 through -1, total lymphoid radiation (TLI) on Day -1, stem cell infusion on Day 0.
Busulfan, Fludarabine, ATG, TLI: Busulfan 0.8 mg/kg/dose intravenous (IV) Days -8 and -7 Fludarabine 35 mg/m2 IV Days -6 through -2 Antithymocyte globulin (ATG) 30 mg/kg IV Days -2 and -1 Total lymphoid radiation 300 cGy
|
MA Bu/Cy (B)
n=5 Participants
Myeloablative Preparative Regimen for subjects with HLA identical sibling donors consists of Busulfan on day -9 through -6, Cyclophosphamide on day -5 through -2, ATG on day -3 through -1, stem cell infusion on Day 0 and Granulocyte Colony Stimulating Factor on day -3 until ANC \>2500 x 2 days.
Busulfan, Cyclophosphamide, ATG, GCSF: Busulfan 0.8 mg/kg/dose intravenous (IV) Days -9 through -6 Cyclophosphamide 50 mg/kg IV Days -5 through -2 ATG 30 mg/kg IV Day -1 GCSF 5 mcg/kg/day IV until ANC \>2500 x 2 days.
Total Body Irradiation: 300 cGY Day -1
Stem cell infusion: Given Day 0
|
RIC Cy/Flu/TBI (A2)
n=14 Participants
Patients with sickle cell disease or thalassemia who do not have an HLA-identical sibling donor or who has pre-existing organ dysfunction making myeloablative condition ineligible will receive Campath on day -10 through -6, Cyclophosphamide on day -7, Fludarabine on day -6 through -2, total body irradiation (TBI) on day -1, stem cell infusion on Day 0.
Campath, Fludarabine, Cyclophosphamide: Receives Campath-1H 0.2 mg/kg Days -10 through -6, Fludarabine 35 mg/m2 intravenous (IV) Days -6 through -2, total body irradiation (TBI) 300 cGy Day -1.
Total Body Irradiation: 300 cGY Day -1
Stem cell infusion: Given Day 0
|
|---|---|---|---|
|
Change in the Patient's Quality of Life as Compared to the Pre-Transplant Assessment
|
100 units on a scale
Interval 100.0 to 100.0
|
98 units on a scale
Interval 90.0 to 100.0
|
99 units on a scale
Interval 90.0 to 100.0
|
SECONDARY outcome
Timeframe: 1 yearPopulation: Two of the 14 patients treated on Arm A2 died before 1 year.
The measure for quality of life used in this study is the Karnofsky Performance Score. The Karnofsky Performance Score runs from 100 to 0, where 100 is "perfect" health and 0 is death.
Outcome measures
| Measure |
RIC Bu/Flu (A) (Discontinued)
n=3 Participants
Full Preparative Regimen for subjects with matched donors using Busulfan on Day -8 and -7, Fludarabine on Day -6 through -2, antithymocyte globulin (ATG) on Day -2 through -1, total lymphoid radiation (TLI) on Day -1, stem cell infusion on Day 0.
Busulfan, Fludarabine, ATG, TLI: Busulfan 0.8 mg/kg/dose intravenous (IV) Days -8 and -7 Fludarabine 35 mg/m2 IV Days -6 through -2 Antithymocyte globulin (ATG) 30 mg/kg IV Days -2 and -1 Total lymphoid radiation 300 cGy
|
MA Bu/Cy (B)
n=5 Participants
Myeloablative Preparative Regimen for subjects with HLA identical sibling donors consists of Busulfan on day -9 through -6, Cyclophosphamide on day -5 through -2, ATG on day -3 through -1, stem cell infusion on Day 0 and Granulocyte Colony Stimulating Factor on day -3 until ANC \>2500 x 2 days.
Busulfan, Cyclophosphamide, ATG, GCSF: Busulfan 0.8 mg/kg/dose intravenous (IV) Days -9 through -6 Cyclophosphamide 50 mg/kg IV Days -5 through -2 ATG 30 mg/kg IV Day -1 GCSF 5 mcg/kg/day IV until ANC \>2500 x 2 days.
Total Body Irradiation: 300 cGY Day -1
Stem cell infusion: Given Day 0
|
RIC Cy/Flu/TBI (A2)
n=12 Participants
Patients with sickle cell disease or thalassemia who do not have an HLA-identical sibling donor or who has pre-existing organ dysfunction making myeloablative condition ineligible will receive Campath on day -10 through -6, Cyclophosphamide on day -7, Fludarabine on day -6 through -2, total body irradiation (TBI) on day -1, stem cell infusion on Day 0.
Campath, Fludarabine, Cyclophosphamide: Receives Campath-1H 0.2 mg/kg Days -10 through -6, Fludarabine 35 mg/m2 intravenous (IV) Days -6 through -2, total body irradiation (TBI) 300 cGy Day -1.
Total Body Irradiation: 300 cGY Day -1
Stem cell infusion: Given Day 0
|
|---|---|---|---|
|
Change in the Patient's Quality of Life as Compared to the Pre-Transplant Assessment
|
97 units on a scale
Interval 90.0 to 100.0
|
100 units on a scale
Interval 100.0 to 100.0
|
100 units on a scale
Interval 80.0 to 100.0
|
SECONDARY outcome
Timeframe: 2 yearsPopulation: Two of the 14 patients treated on Arm A2 died before 2 years and 2 failed their 2 year clinic appointment.
The measure for quality of life used in this study is the Karnofsky Performance Score. The Karnofsky Performance Score runs from 100 to 0, where 100 is "perfect" health and 0 is death.
Outcome measures
| Measure |
RIC Bu/Flu (A) (Discontinued)
n=3 Participants
Full Preparative Regimen for subjects with matched donors using Busulfan on Day -8 and -7, Fludarabine on Day -6 through -2, antithymocyte globulin (ATG) on Day -2 through -1, total lymphoid radiation (TLI) on Day -1, stem cell infusion on Day 0.
Busulfan, Fludarabine, ATG, TLI: Busulfan 0.8 mg/kg/dose intravenous (IV) Days -8 and -7 Fludarabine 35 mg/m2 IV Days -6 through -2 Antithymocyte globulin (ATG) 30 mg/kg IV Days -2 and -1 Total lymphoid radiation 300 cGy
|
MA Bu/Cy (B)
n=5 Participants
Myeloablative Preparative Regimen for subjects with HLA identical sibling donors consists of Busulfan on day -9 through -6, Cyclophosphamide on day -5 through -2, ATG on day -3 through -1, stem cell infusion on Day 0 and Granulocyte Colony Stimulating Factor on day -3 until ANC \>2500 x 2 days.
Busulfan, Cyclophosphamide, ATG, GCSF: Busulfan 0.8 mg/kg/dose intravenous (IV) Days -9 through -6 Cyclophosphamide 50 mg/kg IV Days -5 through -2 ATG 30 mg/kg IV Day -1 GCSF 5 mcg/kg/day IV until ANC \>2500 x 2 days.
Total Body Irradiation: 300 cGY Day -1
Stem cell infusion: Given Day 0
|
RIC Cy/Flu/TBI (A2)
n=10 Participants
Patients with sickle cell disease or thalassemia who do not have an HLA-identical sibling donor or who has pre-existing organ dysfunction making myeloablative condition ineligible will receive Campath on day -10 through -6, Cyclophosphamide on day -7, Fludarabine on day -6 through -2, total body irradiation (TBI) on day -1, stem cell infusion on Day 0.
Campath, Fludarabine, Cyclophosphamide: Receives Campath-1H 0.2 mg/kg Days -10 through -6, Fludarabine 35 mg/m2 intravenous (IV) Days -6 through -2, total body irradiation (TBI) 300 cGy Day -1.
Total Body Irradiation: 300 cGY Day -1
Stem cell infusion: Given Day 0
|
|---|---|---|---|
|
Change in the Patient's Quality of Life as Compared to the Pre-Transplant Assessment
|
100 units on a scale
Interval 90.0 to 100.0
|
100 units on a scale
Interval 100.0 to 100.0
|
100 units on a scale
Interval 90.0 to 100.0
|
SECONDARY outcome
Timeframe: During studyPopulation: data were not collected
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Day 0Population: The Principal Investigator removed this as a study objective and therefore Campath concentrations were not collected.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: 100 daysNumber of patients alive 100 days after transplant.
Outcome measures
| Measure |
RIC Bu/Flu (A) (Discontinued)
n=3 Participants
Full Preparative Regimen for subjects with matched donors using Busulfan on Day -8 and -7, Fludarabine on Day -6 through -2, antithymocyte globulin (ATG) on Day -2 through -1, total lymphoid radiation (TLI) on Day -1, stem cell infusion on Day 0.
Busulfan, Fludarabine, ATG, TLI: Busulfan 0.8 mg/kg/dose intravenous (IV) Days -8 and -7 Fludarabine 35 mg/m2 IV Days -6 through -2 Antithymocyte globulin (ATG) 30 mg/kg IV Days -2 and -1 Total lymphoid radiation 300 cGy
|
MA Bu/Cy (B)
n=5 Participants
Myeloablative Preparative Regimen for subjects with HLA identical sibling donors consists of Busulfan on day -9 through -6, Cyclophosphamide on day -5 through -2, ATG on day -3 through -1, stem cell infusion on Day 0 and Granulocyte Colony Stimulating Factor on day -3 until ANC \>2500 x 2 days.
Busulfan, Cyclophosphamide, ATG, GCSF: Busulfan 0.8 mg/kg/dose intravenous (IV) Days -9 through -6 Cyclophosphamide 50 mg/kg IV Days -5 through -2 ATG 30 mg/kg IV Day -1 GCSF 5 mcg/kg/day IV until ANC \>2500 x 2 days.
Total Body Irradiation: 300 cGY Day -1
Stem cell infusion: Given Day 0
|
RIC Cy/Flu/TBI (A2)
n=14 Participants
Patients with sickle cell disease or thalassemia who do not have an HLA-identical sibling donor or who has pre-existing organ dysfunction making myeloablative condition ineligible will receive Campath on day -10 through -6, Cyclophosphamide on day -7, Fludarabine on day -6 through -2, total body irradiation (TBI) on day -1, stem cell infusion on Day 0.
Campath, Fludarabine, Cyclophosphamide: Receives Campath-1H 0.2 mg/kg Days -10 through -6, Fludarabine 35 mg/m2 intravenous (IV) Days -6 through -2, total body irradiation (TBI) 300 cGy Day -1.
Total Body Irradiation: 300 cGY Day -1
Stem cell infusion: Given Day 0
|
|---|---|---|---|
|
Overall Survival
|
3 Participants
|
5 Participants
|
12 Participants
|
SECONDARY outcome
Timeframe: 1 yearNumber of patients alive 1 year after transplant.
Outcome measures
| Measure |
RIC Bu/Flu (A) (Discontinued)
n=3 Participants
Full Preparative Regimen for subjects with matched donors using Busulfan on Day -8 and -7, Fludarabine on Day -6 through -2, antithymocyte globulin (ATG) on Day -2 through -1, total lymphoid radiation (TLI) on Day -1, stem cell infusion on Day 0.
Busulfan, Fludarabine, ATG, TLI: Busulfan 0.8 mg/kg/dose intravenous (IV) Days -8 and -7 Fludarabine 35 mg/m2 IV Days -6 through -2 Antithymocyte globulin (ATG) 30 mg/kg IV Days -2 and -1 Total lymphoid radiation 300 cGy
|
MA Bu/Cy (B)
n=5 Participants
Myeloablative Preparative Regimen for subjects with HLA identical sibling donors consists of Busulfan on day -9 through -6, Cyclophosphamide on day -5 through -2, ATG on day -3 through -1, stem cell infusion on Day 0 and Granulocyte Colony Stimulating Factor on day -3 until ANC \>2500 x 2 days.
Busulfan, Cyclophosphamide, ATG, GCSF: Busulfan 0.8 mg/kg/dose intravenous (IV) Days -9 through -6 Cyclophosphamide 50 mg/kg IV Days -5 through -2 ATG 30 mg/kg IV Day -1 GCSF 5 mcg/kg/day IV until ANC \>2500 x 2 days.
Total Body Irradiation: 300 cGY Day -1
Stem cell infusion: Given Day 0
|
RIC Cy/Flu/TBI (A2)
n=14 Participants
Patients with sickle cell disease or thalassemia who do not have an HLA-identical sibling donor or who has pre-existing organ dysfunction making myeloablative condition ineligible will receive Campath on day -10 through -6, Cyclophosphamide on day -7, Fludarabine on day -6 through -2, total body irradiation (TBI) on day -1, stem cell infusion on Day 0.
Campath, Fludarabine, Cyclophosphamide: Receives Campath-1H 0.2 mg/kg Days -10 through -6, Fludarabine 35 mg/m2 intravenous (IV) Days -6 through -2, total body irradiation (TBI) 300 cGy Day -1.
Total Body Irradiation: 300 cGY Day -1
Stem cell infusion: Given Day 0
|
|---|---|---|---|
|
Overall Survival
|
3 Participants
|
5 Participants
|
12 Participants
|
SECONDARY outcome
Timeframe: 100 daysNumber of patients alive without disease 100 days after transplant.
Outcome measures
| Measure |
RIC Bu/Flu (A) (Discontinued)
n=3 Participants
Full Preparative Regimen for subjects with matched donors using Busulfan on Day -8 and -7, Fludarabine on Day -6 through -2, antithymocyte globulin (ATG) on Day -2 through -1, total lymphoid radiation (TLI) on Day -1, stem cell infusion on Day 0.
Busulfan, Fludarabine, ATG, TLI: Busulfan 0.8 mg/kg/dose intravenous (IV) Days -8 and -7 Fludarabine 35 mg/m2 IV Days -6 through -2 Antithymocyte globulin (ATG) 30 mg/kg IV Days -2 and -1 Total lymphoid radiation 300 cGy
|
MA Bu/Cy (B)
n=5 Participants
Myeloablative Preparative Regimen for subjects with HLA identical sibling donors consists of Busulfan on day -9 through -6, Cyclophosphamide on day -5 through -2, ATG on day -3 through -1, stem cell infusion on Day 0 and Granulocyte Colony Stimulating Factor on day -3 until ANC \>2500 x 2 days.
Busulfan, Cyclophosphamide, ATG, GCSF: Busulfan 0.8 mg/kg/dose intravenous (IV) Days -9 through -6 Cyclophosphamide 50 mg/kg IV Days -5 through -2 ATG 30 mg/kg IV Day -1 GCSF 5 mcg/kg/day IV until ANC \>2500 x 2 days.
Total Body Irradiation: 300 cGY Day -1
Stem cell infusion: Given Day 0
|
RIC Cy/Flu/TBI (A2)
n=14 Participants
Patients with sickle cell disease or thalassemia who do not have an HLA-identical sibling donor or who has pre-existing organ dysfunction making myeloablative condition ineligible will receive Campath on day -10 through -6, Cyclophosphamide on day -7, Fludarabine on day -6 through -2, total body irradiation (TBI) on day -1, stem cell infusion on Day 0.
Campath, Fludarabine, Cyclophosphamide: Receives Campath-1H 0.2 mg/kg Days -10 through -6, Fludarabine 35 mg/m2 intravenous (IV) Days -6 through -2, total body irradiation (TBI) 300 cGy Day -1.
Total Body Irradiation: 300 cGY Day -1
Stem cell infusion: Given Day 0
|
|---|---|---|---|
|
Disease Free Survival
|
3 Participants
|
5 Participants
|
11 Participants
|
SECONDARY outcome
Timeframe: 1 yearNumber of patients alive without disease 1 year after transplant.
Outcome measures
| Measure |
RIC Bu/Flu (A) (Discontinued)
n=3 Participants
Full Preparative Regimen for subjects with matched donors using Busulfan on Day -8 and -7, Fludarabine on Day -6 through -2, antithymocyte globulin (ATG) on Day -2 through -1, total lymphoid radiation (TLI) on Day -1, stem cell infusion on Day 0.
Busulfan, Fludarabine, ATG, TLI: Busulfan 0.8 mg/kg/dose intravenous (IV) Days -8 and -7 Fludarabine 35 mg/m2 IV Days -6 through -2 Antithymocyte globulin (ATG) 30 mg/kg IV Days -2 and -1 Total lymphoid radiation 300 cGy
|
MA Bu/Cy (B)
n=5 Participants
Myeloablative Preparative Regimen for subjects with HLA identical sibling donors consists of Busulfan on day -9 through -6, Cyclophosphamide on day -5 through -2, ATG on day -3 through -1, stem cell infusion on Day 0 and Granulocyte Colony Stimulating Factor on day -3 until ANC \>2500 x 2 days.
Busulfan, Cyclophosphamide, ATG, GCSF: Busulfan 0.8 mg/kg/dose intravenous (IV) Days -9 through -6 Cyclophosphamide 50 mg/kg IV Days -5 through -2 ATG 30 mg/kg IV Day -1 GCSF 5 mcg/kg/day IV until ANC \>2500 x 2 days.
Total Body Irradiation: 300 cGY Day -1
Stem cell infusion: Given Day 0
|
RIC Cy/Flu/TBI (A2)
n=14 Participants
Patients with sickle cell disease or thalassemia who do not have an HLA-identical sibling donor or who has pre-existing organ dysfunction making myeloablative condition ineligible will receive Campath on day -10 through -6, Cyclophosphamide on day -7, Fludarabine on day -6 through -2, total body irradiation (TBI) on day -1, stem cell infusion on Day 0.
Campath, Fludarabine, Cyclophosphamide: Receives Campath-1H 0.2 mg/kg Days -10 through -6, Fludarabine 35 mg/m2 intravenous (IV) Days -6 through -2, total body irradiation (TBI) 300 cGy Day -1.
Total Body Irradiation: 300 cGY Day -1
Stem cell infusion: Given Day 0
|
|---|---|---|---|
|
Disease Free Survival
|
3 Participants
|
5 Participants
|
11 Participants
|
Adverse Events
Full Conditioning (Discontinued / A)
Serious events: 2 serious events
Other events: 3 other events
Deaths: 0 deaths
Busulfan Conditioning (B)
Serious events: 0 serious events
Other events: 5 other events
Deaths: 0 deaths
Campath and TBI Conditioning (A2)
Serious events: 0 serious events
Other events: 13 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Full Conditioning (Discontinued / A)
n=3 participants at risk
Full Preparative Regimen for subjects with matched donors using Busulfan on Day -8 and -7, Fludarabine on Day -6 through -2, antithymocyte globulin (ATG) on Day -2 through -1, total lymphoid radiation (TLI) on Day -1, stem cell infusion on Day 0.
Busulfan, Fludarabine, ATG, TLI: Busulfan 0.8 mg/kg/dose intravenous (IV) Days -8 and -7 Fludarabine 35 mg/m2 IV Days -6 through -2 Antithymocyte globulin (ATG) 30 mg/kg IV Days -2 and -1 Total lymphoid radiation 300 cGy
|
Busulfan Conditioning (B)
n=5 participants at risk
Myeloablative Preparative Regimen for subjects with HLA identical sibling donors consists of Busulfan on day -9 through -6, Cyclophosphamide on day -5 through -2, ATG on day -3 through -1, stem cell infusion on Day 0 and Granulocyte Colony Stimulating Factor on day -3 until ANC \>2500 x 2 days.
Busulfan, Cyclophosphamide, ATG, GCSF: Busulfan 0.8 mg/kg/dose intravenous (IV) Days -9 through -6 Cyclophosphamide 50 mg/kg IV Days -5 through -2 ATG 30 mg/kg IV Day -1 GCSF 5 mcg/kg/day IV until ANC \>2500 x 2 days.
Total Body Irradiation: 300 cGY Day -1
Stem cell infusion: Given Day 0
|
Campath and TBI Conditioning (A2)
n=14 participants at risk
Patients with sickle cell disease or thalassemia who do not have an HLA-identical sibling donor or who has pre-existing organ dysfunction making myeloablative condition ineligible will receive Campath on day -10 through -6, Cyclophosphamide on day -7, Fludarabine on day -6 through -2, total body irradiation (TBI) on day -1, stem cell infusion on Day 0.
Campath, Fludarabine, Cyclophosphamide: Receives Campath-1H 0.2 mg/kg Days -10 through -6, Fludarabine 35 mg/m2 intravenous (IV) Days -6 through -2, total body irradiation (TBI) 300 cGy Day -1.
Total Body Irradiation: 300 cGY Day -1
Stem cell infusion: Given Day 0
|
|---|---|---|---|
|
General disorders
Primary Graft Failure
|
33.3%
1/3
|
0.00%
0/5
|
0.00%
0/14
|
|
General disorders
Secondary Graft Failure
|
66.7%
2/3
|
0.00%
0/5
|
0.00%
0/14
|
Other adverse events
| Measure |
Full Conditioning (Discontinued / A)
n=3 participants at risk
Full Preparative Regimen for subjects with matched donors using Busulfan on Day -8 and -7, Fludarabine on Day -6 through -2, antithymocyte globulin (ATG) on Day -2 through -1, total lymphoid radiation (TLI) on Day -1, stem cell infusion on Day 0.
Busulfan, Fludarabine, ATG, TLI: Busulfan 0.8 mg/kg/dose intravenous (IV) Days -8 and -7 Fludarabine 35 mg/m2 IV Days -6 through -2 Antithymocyte globulin (ATG) 30 mg/kg IV Days -2 and -1 Total lymphoid radiation 300 cGy
|
Busulfan Conditioning (B)
n=5 participants at risk
Myeloablative Preparative Regimen for subjects with HLA identical sibling donors consists of Busulfan on day -9 through -6, Cyclophosphamide on day -5 through -2, ATG on day -3 through -1, stem cell infusion on Day 0 and Granulocyte Colony Stimulating Factor on day -3 until ANC \>2500 x 2 days.
Busulfan, Cyclophosphamide, ATG, GCSF: Busulfan 0.8 mg/kg/dose intravenous (IV) Days -9 through -6 Cyclophosphamide 50 mg/kg IV Days -5 through -2 ATG 30 mg/kg IV Day -1 GCSF 5 mcg/kg/day IV until ANC \>2500 x 2 days.
Total Body Irradiation: 300 cGY Day -1
Stem cell infusion: Given Day 0
|
Campath and TBI Conditioning (A2)
n=14 participants at risk
Patients with sickle cell disease or thalassemia who do not have an HLA-identical sibling donor or who has pre-existing organ dysfunction making myeloablative condition ineligible will receive Campath on day -10 through -6, Cyclophosphamide on day -7, Fludarabine on day -6 through -2, total body irradiation (TBI) on day -1, stem cell infusion on Day 0.
Campath, Fludarabine, Cyclophosphamide: Receives Campath-1H 0.2 mg/kg Days -10 through -6, Fludarabine 35 mg/m2 intravenous (IV) Days -6 through -2, total body irradiation (TBI) 300 cGy Day -1.
Total Body Irradiation: 300 cGY Day -1
Stem cell infusion: Given Day 0
|
|---|---|---|---|
|
Infections and infestations
Bacterial Infection, Blood
|
66.7%
2/3
|
40.0%
2/5
|
35.7%
5/14
|
|
Infections and infestations
Bacterial Infection, Pulmonary
|
0.00%
0/3
|
20.0%
1/5
|
14.3%
2/14
|
|
Eye disorders
Conjunctival Hemorrhage
|
0.00%
0/3
|
0.00%
0/5
|
14.3%
2/14
|
|
Musculoskeletal and connective tissue disorders
Delayed Growth/Stature
|
100.0%
3/3
|
40.0%
2/5
|
0.00%
0/14
|
|
Infections and infestations
Fungal Infection, Gastrointestinal
|
66.7%
2/3
|
20.0%
1/5
|
7.1%
1/14
|
|
Vascular disorders
Hypertension
|
33.3%
1/3
|
20.0%
1/5
|
57.1%
8/14
|
|
Endocrine disorders
Hypothyroidism
|
100.0%
3/3
|
20.0%
1/5
|
7.1%
1/14
|
|
Cardiac disorders
Pericardial Effusion
|
0.00%
0/3
|
60.0%
3/5
|
21.4%
3/14
|
|
Infections and infestations
Pneumonia
|
33.3%
1/3
|
60.0%
3/5
|
35.7%
5/14
|
|
Renal and urinary disorders
Renal Failure
|
0.00%
0/3
|
0.00%
0/5
|
14.3%
2/14
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory Failure
|
0.00%
0/3
|
0.00%
0/5
|
14.3%
2/14
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Second Malignancy
|
0.00%
0/3
|
0.00%
0/5
|
14.3%
2/14
|
|
Nervous system disorders
Seizure
|
33.3%
1/3
|
0.00%
0/5
|
14.3%
2/14
|
|
Hepatobiliary disorders
Veno-Occlusive Disease
|
0.00%
0/3
|
0.00%
0/5
|
14.3%
2/14
|
|
Infections and infestations
Viral Infection, Blood
|
33.3%
1/3
|
0.00%
0/5
|
35.7%
5/14
|
|
Infections and infestations
Viral Infection, Pulmonary
|
0.00%
0/3
|
40.0%
2/5
|
42.9%
6/14
|
|
Renal and urinary disorders
Cystitis
|
33.3%
1/3
|
20.0%
1/5
|
7.1%
1/14
|
|
Investigations
Elevated Liver Function Tests
|
33.3%
1/3
|
0.00%
0/5
|
0.00%
0/14
|
|
Immune system disorders
Engraftment Syndrome
|
33.3%
1/3
|
0.00%
0/5
|
0.00%
0/14
|
|
Gastrointestinal disorders
Esophageal Varices
|
0.00%
0/3
|
0.00%
0/5
|
7.1%
1/14
|
|
Infections and infestations
Fungal Infection, Blood
|
33.3%
1/3
|
0.00%
0/5
|
7.1%
1/14
|
|
Infections and infestations
Fungal Infection, Genitourinary
|
0.00%
0/3
|
0.00%
0/5
|
7.1%
1/14
|
|
Infections and infestations
Fungal Infection, Pulmonary
|
0.00%
0/3
|
0.00%
0/5
|
7.1%
1/14
|
|
Metabolism and nutrition disorders
Hemochromatosis
|
33.3%
1/3
|
0.00%
0/5
|
0.00%
0/14
|
|
Investigations
Hepatitis
|
33.3%
1/3
|
0.00%
0/5
|
0.00%
0/14
|
|
Endocrine disorders
Hypogonadism
|
33.3%
1/3
|
0.00%
0/5
|
7.1%
1/14
|
|
Cardiac disorders
Intraventricular Septal Hypertrophy
|
0.00%
0/3
|
0.00%
0/5
|
7.1%
1/14
|
|
Musculoskeletal and connective tissue disorders
Joint Dysfunction
|
33.3%
1/3
|
0.00%
0/5
|
0.00%
0/14
|
|
Nervous system disorders
Neuro Toxicity, NOS
|
33.3%
1/3
|
0.00%
0/5
|
0.00%
0/14
|
|
Nervous system disorders
Neuropathy
|
0.00%
0/3
|
20.0%
1/5
|
7.1%
1/14
|
|
Infections and infestations
Otitis Media
|
0.00%
0/3
|
0.00%
0/5
|
7.1%
1/14
|
|
Infections and infestations
Paronychia of Bilateral Great Toes
|
0.00%
0/3
|
0.00%
0/5
|
7.1%
1/14
|
|
Blood and lymphatic system disorders
Polycythemia
|
0.00%
0/3
|
0.00%
0/5
|
7.1%
1/14
|
|
Blood and lymphatic system disorders
Polymorphic Post-Transplant Lymphoproliferative Disorder
|
33.3%
1/3
|
0.00%
0/5
|
0.00%
0/14
|
|
Infections and infestations
Sepsis
|
0.00%
0/3
|
20.0%
1/5
|
0.00%
0/14
|
|
Nervous system disorders
Subarachnoid Hemorrhage
|
0.00%
0/3
|
0.00%
0/5
|
7.1%
1/14
|
|
Vascular disorders
Thrombus, Right Atrium
|
0.00%
0/3
|
0.00%
0/5
|
7.1%
1/14
|
|
Reproductive system and breast disorders
Upper Respiratory Stridor
|
33.3%
1/3
|
0.00%
0/5
|
0.00%
0/14
|
|
Infections and infestations
Viral Infection, Gastrointestinal
|
0.00%
0/3
|
40.0%
2/5
|
42.9%
6/14
|
Additional Information
Dr. Angela Smith
Masonic Cancer Center, University of Minnesota
Phone: 612-626-2778
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place