Stem Cell Transplantation for Fanconi Anemia

• Status: Terminated
• Trial end date: 2008-12-01
• Target enrollment: 0
• Participant gender: All

Summary

The purpose of this study is to determine whether thymic shielding during total body irradiation can be given and whether it will reduce the risk of infections in Fanconi Anemia patients undergoing alternate donor (not a matched sibling) stem cell transplants.

• Phase: Phase 1/Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Masonic Cancer Center, University of Minnesota
• Treatments: Cyclophosphamide; Fludarabine; Fludarabine phosphate

Criteria

Inclusion Criteria:

• Patients must be less than (<) 18 years of age with a diagnosis of Fanconi anemia.

• Patients must have an HLA-A, B, DRB1 identical unrelated donor or less than or equal to (≤)1 antigen mismatched related (non-HLA-matched sibling) or <1 antigen mismatched unrelated UCB donor. Patients and donors will be typed for HLA-A and B using serological or molecular techniques and for DRB1 using high resolution molecular typing.

• Patients with FA must have aplastic anemia (AA), myelodysplastic syndrome without excess blasts, or high risk genotype as defined below.

• Aplastic anemia is defined as having at least one of the following when not receiving growth factors or transfusions

• Platelet count <20 x 10^9/L

• ANC <5 x 10^8/L

• Hgb <8 g/dL

• Myelodysplastic syndrome with multilineage dysplasia with or without chromosomal anomalies

• High risk genotype (e.g. IVS-4 or exon 14 FANCC mutations, or BRCA1 or 2 mutations)

• Adequate major organ function including

• Cardiac: ejection fraction greater than (>)45%

• Hepatic: bilirubin, AST/ALT, ALP <2 x normal

• Karnofsky performance status >70% or Lansky performance status >50%

• Women of child-bearing age must be using adequate birth control and have a negative pregnancy test

Exclusion Criteria:

• Available HLA-genotypically identical related donor

• History of gram negative sepsis or systemic fungal infection (proven or suspected based on radiographic studies)

• Refractory anemia with excess blasts, or leukemia

• Active central nervous system (CNS) leukemia at time of hematopoietic cell transplant (HCT)

• History of squamous cell carcinoma of the head/neck/cervix within 2 years of HCT

• Pregnant or lactating female

• Prior radiation therapy preventing use of total body irradiation (TBI) 450 centigray (cGy)