Last updated on February 2018

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


Are you eligible to participate in this study?

You may be eligible for this study if you meet the following criteria:

  • Conditions: childhood ALL | Chronic myeloid leukemia | melanoma | Hematologic Malignancy | Cancer
  • Age: - 21 Years
  • Gender: Male or Female

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-Immunoglobulin M (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 </= 2 x normal; and Aspartate aminotransferase (AST) or Alanine aminotransferase (ALT) </= 4 x normal.
  • Adequate cardiac function: shortening fraction of > 24% by echocardiogram, or ejection fraction of > 30% by radionuclide angiogram.
  • Adequate pulmonary function: Diffusion Lung Capacity Carbon Monoxide (DLCO), Forced Expiratory Volume in 1 second (FEV1) / Forced Vital Capacity (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 Score >/= 60% for children </= 16 years of age; or 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

Recruitment Status: Open


Brief Description Eligibility Contact Research Team


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