Transfusion independence after repeated haploidentical hematopoietic cell transplants in a patient with congenital dyserythropoietic anemia type II and hemosiderosis
AffiliationUniv Arizona, Dept Pediat
Univ Arizona, Dept Pathol
Univ Arizona, Dept Med
Univ Arizona, Dept Immunobiol
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CitationMacaraeg M, Proytcheva M, Katsanis E. Transfusion independence after repeated haploidentical hematopoietic cell transplants in a patient with congenital dyserythropoietic anemia type II and hemosiderosis. Pediatr Transplant. 2019;00:e13587. https://doi.org/10.1111/petr.13587
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AbstractMatched related or unrelated donor allogeneic HCT has occasionally been applied in patients with severe CDA type II and proven to be curative. We report on the first patient with CDA to undergo haploidentical bone marrow transplantation with PT-CY. A 12-year-old boy with severe hemosiderosis, and a, consequently, disturbed BM microenvironment, developed recurrent graft failures and required salvage with two additional haploidentical HCTs. He achieved complete donor chimerism and transfusion independence after the third HCT. Our case underscores the risks associated with performing haploidentical HCT in older pediatric patients with CDA and severe chronic iron overload.
Note12 month embargo; published online: 17 September 2019
VersionFinal accepted manuscript