Stem Cell Transplantation for Patients With Hematologic Malignancies

• Status: Completed
• Trial end date: 2009-01-01
• Target enrollment: 0
• Participant gender: All

Summary

Childhood leukemias which cannot be cured by chemotherapy alone may be effectively treated by allogeneic bone marrow transplantation. Moreover, for patients with chronic myelogenous leukemia (CML), allogeneic hematopoietic stem cell transplantation (HSCT) is the only proven curative modality of treatment. Patients who have received hematopoietic stem cells from an HLA matched sibling donor have proven to be less at risk for disease relapse and regimen related toxicity. However, about 70% of patients in need of HSCT do not have an HLA matched sibling donor. This necessitates the search for alternative donors, which may increase the risk of a poor outcome.

The nature of the hematopoietic stem cell graft has been implicated as a primary factor determining these outcomes. The standard stem cell graft has been unmanipulated bone marrow, but recently several advantages of T-lymphocyte depleted bone marrow and mobilized peripheral blood progenitor cells (PBPC) have been demonstrated. However, T-cell depletion may increase the risk of infectious complications and leukemic recurrence while an unmanipulated stem cell graft may increase the risk of graft vs. host disease (GVHD). A key element in long range strategies in improving outcomes for patients undergoing matched unrelated donor (MUD) HSCT is to provide the optimal graft.

The primary objective of this clinical trial is to estimate the incidence of acute GVHD in pediatric patients with hematologic malignancies who receive HSCT with an unmanipulated marrow graft. The results of this study can be used as the foundation for future trials related to engineering unrelated donor graft.

• Phase: Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: St. Jude Children's Research Hospital
• Treatments: Antibodies

Criteria

Inclusion Criteria:

• All patients lacking an HLA identical sibling for whom an unrelated donor matched at 6/6 HLA loci is formally requested within about 3 months of search initiation

• Age greater than or equal to 24 months, but less than 21 years for new patients.

• Diagnosis of one of the following high risk hematological malignancies:

• Acute lymphoblastic leukemia (in second or subsequent remission or high risk in first remission)

• Acute myeloid leukemia (in relapse or remission)

• Secondary AML/MDS

• Chronic myeloid leukemia

• Juvenile myelomonocytic leukemia

• Myelodysplastic syndrome

• Paroxysmal nocturnal hemoglobinuria

• Non-Hodgkin's lymphoma (in second or subsequent remission)

Exclusion Criteria:

• Patients with symptomatic cardiac disease, or evidence of significant cardiac disease by echocardiogram, or cardiac shortening fraction below 25%

• Patients with renal creatinine clearance < 40ml/min/1.73m^2

• Patients with FVC < 40% predicted or pulse oximetry less than or equal to 92% on room air if unable to perform pulmonary function tests

• Patients with direct bilirubin > 3 mg/dl

• Patients with SGPT > 500 U/L

• Patients with a Karnofsky or Lansky performance score < 70

• Patients who have received a previous allogeneic stem cell transplant

• Patients with a known allergy to rabbit or murine products

• Patients with isolated extramedullary leukemic relapse, including isolated CNS or testicular recurrence