Allogeneic Hematopoietic Stem Cell Transplantation (NST) for Patients With Systemic Sclerosis



Status:Completed
Conditions:Skin and Soft Tissue Infections, Neurology, Dermatology
Therapuetic Areas:Dermatology / Plastic Surgery, Neurology
Healthy:No
Age Range:Any
Updated:11/18/2012
Start Date:May 2005
End Date:December 2014
Contact:Dzemila Spahovic, MD
Email:d-spahovic@northwestern.edu
Phone:312-908-0059

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Non-myeloablative Allogeneic Hematopoietic Stem Cell Transplantation (NST) for Patients With Systemic Sclerosis


Scleroderma is disease believed to be due to immune cells, cells which normally protect the
body but are now causing damage to the body. There has not been any treatment that has been
effective in treating this disease. The likelihood of progression of the disease to severe
disability and death is high. This study is designed to examine whether treating patients
with high dose Cyclophosphamide and Fludarabine (drugs which reduce the function of your
immune system) and CAMPATH-1H (a protein that kills the immune cells that are thought to be
causing the disease), followed by return of blood stem cells that have been previously
collected from patients brother or sister will stop or reverse the disease. The purpose of
the Cyclophosphamide, Fludarabine and CAMPATH-1H is to decrease immune system. The purpose
of the stem cell infusion is to restore blood production, which will be severely impaired by
the Cyclophosphamide, Fludarabine and CAMPATH-1H, and to produce a normal immune system that
will no longer attack the body.


Patient Inclusion Criteria:

1. Age 18 to 55 years old

2. An established diagnosis of scleroderma (84)

3. Diffuse cutaneous scleroderma with involvement proximal to the elbow or knee and a
Rodnan score (see Appendix III) of > 14 (85)

And one of the following:

1. DLCO < 80% of predicted or decrease in lung function (TLC, DLCO or FEV1) of 10% or
more over 12 months

2. Active alveolitis on bronchoalveolar lavage

3. Pulmonary fibrosis or alveolitis on CT scan or CXR

4. Elevated ESR greater than or equal to 25mm/hour confirmed on a second occasion at
least two weeks apart without evidence of active infectious process.

5. Abnormal EKG (low QRS voltage, or ventricular hypertrophy) or left ventricle (LV)
diastolic dysfunction (expressed by an inverted E/A ratio which represents early and
late filling of the LV during atrial contraction) or LV wall thickness

6. Since pulmonary disease independent of skin score (NEJM, 2006, 345:25 2655-2709)
carries a poor prognosis, patient may be enroled for only lung involvement defined as
active alveolitis on BAL or ground-glass opacity on CT, a DLCO < 80% predicted or
decrease in lung function (TLC), DLCO, FVC) of 10% or more in last 12 months.

Patient Exclusion Criteria:

1. Poor performance status (ECOG > or =2) at the time of entry, unless due to disease.

2. Significant end organ damage such as:

1. LVEF <40% or deterioration of LVEF during exercise test on MUGA or
echocardiogram

2. Untreated life-threatening arrhythmia

3. Active ischemic heart disease or heart failure

4. DLCO less than 45% of predicted value, unless due to disease.

5. Pulmonary hypertension (estimated systolic pulmonary arterial pressure >40 mmHg
by Doppler echocardiography or measurement by pulmonary arterial catheter)

6. Serum creatinine > 2.0 mg/dl

7. Liver cirrhosis, transaminases >3x of normal limits or bilirubin >2.0 unless due
to Gilberts disease

3. HIV positive

4. Uncontrolled diabetes mellitus or any other illness that in the opinion of the
investigators would jeopardize the ability of the patient to tolerate aggressive
treatment

5. Prior history of malignancy except localized basal cell or squamous skin cancer.
Other malignancies for which the patient is judged to be cured by local surgical
therapy, such as (but not limited to) head and neck cancer, or stage I or II breast
cancer will be considered on an individual basis.

6. Positive pregnancy test, inability or unable to pursue effective means of birth
control, failure to willingly accept or comprehend irreversible sterility as a side
effect of therapy

7. Psychiatric illness or mental deficiency making compliance with treatment or informed
consent impossible

8. Inability to give informed consent

9. Age <18 or > 55 years old

Donor Inclusion Criteria:

1. Donor must be a HLA identical sibling or HLA matched cord blood donor

2. If donor is HLA matched sibling, donor must be > 18 years of age and less than 50
years old

3. If multiple HLA matched donors are available, preference will be given to same sex,
same CMV status, or in the case of cord blood higher nucleated cell count

4. If donor is HLA matched cord blood, cord blood stem cells will be obtained from the
New York Blood Center Cord Blood Registry (Tel 212-570-3230) which is an
internationally recognized registry or, if a match is not available, from Stemcyte
(626-821-9860) which is a commercial registry that specializes in minority donors.
One unit of HLA matched cord blood unit will be infused on day zero

Donor Exclusion Criteria:

1. Age > 50 years old or <18 years old

2. HIV positive

3. Active ischemic heart disease or heart failure

4. Acute or chronic active hepatitis

5. Uncontrolled diabetes mellitus or any other illness that in the opinion of the
investigators would jeopardize the ability of the donor to tolerate stem cell
collection

6. Prior history of malignancy except localized basal cell or squamous skin cancer.
Other malignancies for which the patient is judged to be cured by local surgical
therapy, such as (but not limited to) head and neck cancer, or stage I or II breast
cancer will be considered on an individual basis.

7. Positive pregnancy test

8. Psychiatric illness or mental deficiency making compliance with treatment or informed
consent impossible

9. Major hematological abnormalities such as platelet count less than 100,000/ul, ANC
less than 1000/ul

10. If donor is sibling must collect a minimum of 2. 106CD34+ cells/kg to proceed to
transplant

11. If donor is cord blood unit(s) then a minimum number of nucleated cells available
must be more than 2 x 107 /kg. To achieve this number of nucleated cells, two units
of HLA matched cord blood may be utilized. (Wagner JE Blood. 2005 Feb
1;105(3):1343-7)
We found this trial at
1
site
303 E Chicago Ave
Chicago, Illinois 60611
(312) 503-8194
Northwestern University Feinberg School of Medicine Northwestern University Feinberg School of Medicine, founded in 1859,...
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from
Chicago, IL
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