Quercetin Chemoprevention for Squamous Cell Carcinoma in Patients With Fanconi Anemia

Fanconi anemia (FA) is an autosomal recessive disease characterized by progressive bone
marrow failure, variable congenital abnormalities and a predisposition to malignancy,
particularly acute myeloid leukemia (AML) and squamous cell carcinoma (SCC). Currently, the
only curative treatment option for the hematological complications of FA include
hematopoietic cell transplantation (HCT). The investigators hypothesize that quercetin will
prevent or delay the development of SCC and associated complications, there by ameliorating
or delaying the need for potentially lethal treatment with chemotherapy and/or radiation
therapy for the same.

This study is an open-label, single arm study. This study will enroll approximately 45
post-HCT patients with FA, and approximately 10 patients with FA without history of HCT. In
both groups, patients with or without existing pre-malignant lesions or history of SCC will
be allowed to participate, if they wish so and at the discretion of the PI. All patients will
be treated with oral quercetin for a total of 24 months.

The investigators will determine the efficacy of Quercetin in reducing buccal micronuclei (a
surrogate marker of DNA damage and susceptibility to squamous cell carcinoma due to genomic
instability) in post-HCT patients with fanconi anemia (FA).

Inclusion Criteria:

- Diagnosis of FA

- Able to take enteral medication

- Patients ≥2 years

Exclusion Criteria:

- Renal failure requiring dialysis

- Total bilirubin >3 mg/dl and/or SGPT >200 at time of enrollment

- Patients receiving digoxin therapy, who are unable to discontinue either treatment due
to medical reasons

- Patients who are pregnant or breastfeeding or are at risk of pregnancy or fathering a
baby and are unable to use acceptable methods of birth control during the length of
the study

- Patients who have received quercetin supplementation or other antioxidants within the
last 30 days

- Patients receiving radiation therapy, chemotherapy or immunotherapy for treatment of
SCC.