Fanconi anemia (FA) is an autosomal recessive, progressive bone marrow failure disorder characterized by congenital defects and marked cancer predisposition. Hematopoietic stem cell transplant is the therapy of choice for FA patients with progressive pancytopenia. These patients receive multiple transfusions for cytopenias. Oxymetholone has been used with variable success to improve cytopenias. Eltrombopag has been shown to induce bilineage or trilineage hematopoiesis in aplastic anemia and patients with myelodysplastic marrow. We report a case of FA where eltrombopag in conjunction with oxymetholone induced trilineage hematopoiesis and eliminated transfusion requirement before transplant, thereby enhancing favorable outcome after hematopoietic stem cell transplant.
*Rainbow Babies and Children’s Hospital, Division of Pediatric Hematology-Oncology, Case Western University
‡Division of Pathology, University Hospitals Cleveland Medical Center, Cleveland, OH
†HCG-MSR Cancer Centre, Bangalore, Karnataka, India
The authors declare no conflict of interest.
Reprints: Yousif Matloub, MD, Rainbow Babies and Children’s Hospital, Division of Pediatric Hematology-Oncology, Case Western University, 11100 Euclid Ave., RBC 6054, Cleveland, OH 44106 (e-mail: email@example.com).
Received October 8, 2017
Accepted March 10, 2018