Immunoablative Mini Transplant (Hematopoietic Peripheral Blood Stem Cell Transplant [HPBSC])



Status:Completed
Conditions:Skin Cancer, Cancer, Blood Cancer, Hematology
Therapuetic Areas:Hematology, Oncology
Healthy:No
Age Range:Any - 21
Updated:4/2/2016
Start Date:March 2000
End Date:March 2015
Contact:Reggie Duerst, MD
Email:rduerst@luriechildrens.org
Phone:312-227-4090

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Immunoablative Protocol for Allogeneic Related and Unrelated Hematopoietic Peripheral Blood Stem Cell Transplant (HPBSC)

The purpose of this research study is to evaluate the effectiveness of transplantation of
high doses of peripheral blood stem cells (stem cells are special cells found in the blood
and bone marrow that produce new blood cells) after treatment with non-myeloablative
chemotherapy (not toxic to the bone marrow). In addition, this study will assess the side
effects of the transplant.

The standard treatment in many disorders of the bone marrow is high dose chemotherapy and
whole-body radiation treatment followed by the stem cell transplant. This type of transplant
not only suppresses or kills off the immune system, but is very toxic to the bone marrow.
This study uses a chemotherapy regimen that will suppress the patient's immune system;
however, it is non-myeloablative (not toxic to the bone marrow). It does not use whole-body
radiation treatment. This approach can minimize the short- and long-term effects of
transplantation. Other studies have shown that using chemotherapy followed by bone marrow
transplantation without whole-body radiation can produce similar results as treatment with
whole-body radiation.

Patients will be given chemotherapy with Fludarabine and Busulfan prior to the stem cell
transplant. This treatment not only destroys diseased cells, but it also kills normal bone
marrow cells. Following this experimental treatment, the patient will be given the stem
cells through a central venous catheter (tube inserted in a vein). When the healthy stem
cells are given to the patient, they will replace the destroyed bone marrow cells and
produce new blood cells. The Allogeneic (not one's own) stem cells used in this experimental
transplant will be obtained from a related matched donor or from an unrelated matched donor
located through the National Marrow Donor Program.

Inclusion Criteria:

- Patients with recurrent solid tumors

- Patients with malignant melanoma

- Patients with hematological malignancies.

- Chronic myelogenous leukemia in chronic or accelerated phase, to include chronic
myelomonocytic leukemia (juvenile chronic myelogenous leukemia [JCML] or CMML).

- Acute lymphoblastic leukemia (ALL)

- First remission high-risk ALL (Ph+ with initial high white blood cell
[WBC]; t (4-11) in infants less than 1 year and CALLA negative)

- Second or subsequent remission ALL or isolated extramedullary disease on or
off therapy.

- Acute non-lymphocytic leukemia (ANLL)

- Patients with ANLL in first remission who have a matched sibling donor.

- ANLL in second remission, or patients who only achieve an initial partial
remission < 15% blasts, or early relapse.

- Myelodysplastic syndromes (MDS): refractory anemia (RA), refractory anemia with
excess blasts (RAEB), refractory anemia with excess blasts in transformation
(RAEB-T) and CMML/JCML.

- Selected immunodeficiencies:

- Wiskott-Aldrich syndrome.

- Severe combined immunodeficiency variants that require ablation.

- Hyper-IGM syndrome.

- Other immune deficiencies after approval from the medical director.

- Bone marrow failure syndromes (single or multiple hematopoietic lines)

- Venous access: A double lumen central vascular access device or its equivalent will
be required for all patients entered on the protocol.

- Informed consent: The donor and the patient and/or the patient's legally authorized
guardian must acknowledge in writing that consent to become a study subject has been
obtained in accordance with the institutional policy approved by the United States
(U.S.) Department of Health and Human Services.

- Patient organ function requirements:

- Adequate renal function: serum creatinine < 2 x normal, or creatinine clearance
calculated by Schwartz formula, of glomerular filtration rate (GFR) > 40
ml/min/1.73m2, or an equivalent GFR as determined by the institutional normal
range.

- Adequate liver function: total bilirubin (ALT)
- Adequate cardiac function: shortening fraction of > 24% by echocardiogram, or
ejection fraction of > 30% by radionuclide angiogram.

- Adequate pulmonary function: DLCO, FEV1 / FVC > 30% by pulmonary function test.
For children who are uncooperative for pulmonary function tests and have no
evidence of dyspnea at rest or exercise intolerance, pulse oximetry > 94% on
room air is considered acceptable.

- Performance status: Lansky >/= 60% for children Karnofsky > 60% status for those > 16 years of age.

Exclusion Criteria:

- Patients who are pregnant

- Inability to find a suitable donor for the patient

- Patient is HIV-positive

- Patient has active Hepatitis B

- Disease progression or relapse prior to HPC infusion
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