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Reduced Intensity Stem Cell Transplant in Children and Young Adults Utilizing Photopheresis, Fludarabine, and Busulfan

Phase 1/Phase 2
Open (Enrolling)
Leukemia, Cancer

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Trial Information

Reduced Intensity Stem Cell Transplant in Children and Young Adults Utilizing Photopheresis, Fludarabine, and Busulfan

This study tests the feasibility of a reduced intensity preparative regimen for stem cell
transplant including extracorporeal photopheresis (ECP), busulfan, and fludarabine in
patients with leukemia, lymphoma, and certain non-malignant diseases. The current reduced
intensity protocol includes busulfan, fludarabine, and anti-thymocyte immunoglobulin. ECP
is currently used in diseases such as chronic GVHD and cutaneous T cell lymphoma. The
mechanism of ECP has not been defined. It is hypothesized that exposure of white blood
cells to ultraviolet light with 8-methoxypsoralen initiates an apoptotic cellular cascade.
Apoptotic cells are recognized and removed by the reticuloendothelial system, initiating the
secretion of anti-inflammatory cytokines and the reduction of proinflammatory cytokines.
Antigen presenting cells then regulate immune responses through the induction of tolerance.

Here we incorporate the use of ECP, fludarabine, and busulfan in the preparative regimen,
followed by ECP as prophylaxis for acute graft versus host disease. We hypothesize that
photopheresis is safe and feasible, and patients will have similar rates of engraftment with
less GVHD as those treated with current reduced intensity protocols. The use of ECP prior
to transplant provides immunosuppression promoting host engraftment. Furthermore, the
introduction of ECP following transplant may be able to induce tolerance thereby reducing
rates of GVHD.

Inclusion Criteria:

- Weight > 25 kg

- Patients with acute lymphoblastic leukemia (ALL) who are in CR (complete remission; <
5% blasts in bone marrow and no active central nervous system disease) who:

- Are in second remission with an initial remission of < 36 months.

- Patients with "high risk" disease in CR1, defined by karyotype abnormalities
such as presence of (9;22) translocation, monosomy 7, or monosomy 5; and/or
patients with slow initial response (initial remission not reached within four
weeks from diagnosis).

- Are in third (or subsequent) remission

- Experience isolated extramedullary relapse while on therapy

- Have experienced relapse following myeloablative stem cell transplant

- Are WT1+ following induction therapy

- Patients with acute myelogenous leukemia (AML) who:

- Are in first remission and remain WT1 positive.

- Are in second remission

- Are in initial partial remission (< 20% blasts in bone marrow)

- Experience relapse following myeloablative stem cell transplant

- Patients with relapsed lymphoma whose residual disease appears to be chemo-responsive
and non-bulky (< 5 cm largest diameter)

- Patients with chronic myelogenous leukemia (CML) in chronic phase who:

- Don't achieve remission (molecular or cytogenetic) by 1 year of diagnosis with
therapy (imatinib mesylate or interferon)

- Have a rising quantitative bcr/abl on imatinib mesylate (molecular relapse)

- Had developed accelerated phase regardless of therapy but are now back in second
chronic phase

- Patients with recurrent solid tumors (neuroblastoma, Ewing's sarcoma, melanoma,

- Patients with myelodysplastic syndrome

- Patients with refractory anemia (RA) and refractory anemia with excess blasts (RAEB)
are eligible, but refractory anemia with excess blasts in transformation (RAEB-T)
patients are only eligible if treated to < 20% blasts with chemotherapy

- Patients with selected immunodeficiencies such as Wiskott-Aldrich syndrome or
hyper-IgM syndrome

- Patients with metabolic diseases such as Niemann-Pick or adrenoleukodystrophy

- Patients with bone marrow failure syndromes, including aplastic anemia

- Adequate venous access

Type of Study:


Study Design:

Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:

To determine the feasibility of using photopheresis as the backbone of a reduced intensity transplant regimen to reduce transplant related mortality and acute and chronic graft versus host disease (GVHD)

Outcome Time Frame:

Throughout Treatment

Safety Issue:


Principal Investigator

Jennifer Schneiderman, MD, MS

Investigator Role:

Principal Investigator

Investigator Affiliation:

Ann & Robert H Lurie Children's Hospital of Chicago


United States: Institutional Review Board

Study ID:




Start Date:

July 2005

Completion Date:

January 2010

Related Keywords:

  • Leukemia
  • Cancer
  • cancer
  • hemoglobinopathy
  • immune deficiency
  • photopheresis
  • ECP
  • extracorporeal photopheresis
  • Leukemia



Children's Memorial Hospital Chicago, Illinois  60614