Transfusion independence after repeated haploidentical hematopoietic cell transplants in a patient with congenital dyserythropoietic anemia type II and hemosiderosis.

Journal: Pediatric Transplantation
Treatment Used: haploidentical bone marrow transplantation with PT-CY
Number of Patients: 1
MediFind Summary

Overview: This report describes the case of a twelve year old male with severe hemosiderosis, disturbed bone marrow (BM) microenvironment which lead to experience graft failures.

Conclusion: After receiving three hematopoietic cell transplantation (HCT) the patients was able to receive complete donor chimerism and transfusion independence. Researchers assessed the risk associated with performing haploidentical HCT in older pediatric patients with severe hemosiderosis.

Abstract

Matched 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.

Authors
Marci Macaraeg, Maria Proytcheva, Emmanuel Katsanis

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