Addition of High-Dose Cytarabine to Fludarabine-Based Conditioning for Hematopoietic Stem Cell Transplantation for Treating Fanconi Anemia Patients with Advanced Myeloid Malignancy: A Single-Center Experience and Literature Review

• Fanconi anemia patients with advanced myeloid malignancy have a poor prognosis
• Four such Fanconi anemia patients survived with high-dose cytarabine plus fludarabine-based conditioning
• High-dose cytarabine could feasibly be added to fludarabine-based conditioning in Fanconi anemia patients
• High-dose cytarabine plus fludarabine-based conditioning may be effective for Fanconi anemia patients with leukemia

The complication of Fanconi anemia (FA) with acute leukemia is rare and challenging to treat because of high relapse rates, despite the improved outcome of hematopoietic stem cell transplantation with fludarabine-based conditioning for treating FA patients with hematological abnormalities. We added high-dose cytarabine to fludarabine-based conditioning to promote an enhanced antitumor effect and successfully subjected 4 patients with FA, including 3 with acute leukemia, to hematopoietic stem cell transplantation. All patients remain alive without treatment-related mortality or evidence of disease. Adding high-dose cytarabine to fludarabine-based conditioning may be tolerable and effective for treating FA patients with acute leukemia.