Stem Cell Transplantation for Fanconi Anemia
Fanconi Anemia
About this trial
This is an interventional treatment trial for Fanconi Anemia focused on measuring Stem Cell Transplant, Thymic Shielding, Total Body Irradiation, Chemotherapy
Eligibility 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)
Sites / Locations
- Masonic Cancer Center, University of Minnesota
Arms of the Study
Arm 1
Experimental
HSCT Patients
Patients who received total body irradiation (450 cGy [centigray]) with thymic shielding prior to chemotherapy regimen and Hematopoietic Stem Cell Transplant (HSCT)