Stem Cell Transplant for Bone Marrow Failure Syndromes

• Status: Completed
• Trial end date: 2009-03-01
• Target enrollment: 0
• Participant gender: All

Summary

The researchers hypothesize that it will be possible to perform unrelated bone marrow or cord blood transplants in a safer manner by using less intensive therapy yet still achieve an acceptable level of donor cell engraftment for non-malignant congenital bone marrow failure disorders.

• Phase: Phase 2/Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Masonic Cancer Center, University of Minnesota
• Treatments: Antilymphocyte Serum; Busulfan; Fludarabine; Fludarabine phosphate; Vidarabine

Criteria

Inclusion Criteria:

• Patients eligible for transplantation under this protocol will be <35 years of age, and will be diagnosed with:

• a bone marrow failure syndrome unresponsive to available therapy, including but not limited to Diamond-Blackfan anemia, Shwachman Diamond syndrome or Kostmann's neutropenia but exclusive of aplastic anemia.

• Diamond Blackfan Anemia:

• Patients must show evidence of steroid resistance requiring equivalent of >6 transfusions yearly despite steroid therapy.

• Evidence of developing aplasia or myelodysplasia will also be criteria for transplantation.

• Kostmann's Neutropenia, Shwachman-Diamond syndrome:

• Patients must have been previously diagnosed as having a clinical picture characteristic of Shwachman-Diamond syndrome (exocrine pancreatic insufficiency, growth retardation, metaphyseal dysostosis, neutropenia), or must have a bone marrow aspirate consistent with Kostmann's neutropenia, with no evidence of acute leukemia.

• Patients must have failed therapy with granulocyte-colony stimulating factor (G-CSF), as determined by an inability to maintain an absolute neutrophil count (ANC) >750 cells/ml(3), or manifesting recurrent infections despite G-CSF administration resulting in life threatening infections or repeated hospitalizations (<4 /year).

Exclusion Criteria:

• Patients >35 years of age

• Karnofsky score <70%

• Hepatic dysfunction as determined by bilirubin >3.0, ALT >150, or active hepatitis

• Pulmonary function tests with forced volume vital capacity (FVC) and forced expiratory volume (FEV) <70%; O2 saturation <94%

• Renal dysfunction with glomerular filtration rate (GFR) <30% of predicted.

• Cardiac compromise, with left ejection fraction <45%.

• Severe, stable neurologic impairment.

• Human immunodeficiency virus (HIV) positivity.

• Pregnant or lactating females