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

Marci Macaraeg, Maria Proytcheva, Emmanuel Katsanis

Research output: Contribution to journalArticle

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.

Original languageEnglish (US)
Article numbere13587
JournalPediatric transplantation
DOIs
StateAccepted/In press - Jan 1 2019

Keywords

  • congenital dyserythropoietic anemias
  • graft failure
  • haploidentical HCT

ASJC Scopus subject areas

  • Pediatrics, Perinatology, and Child Health
  • Transplantation

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