Successful T-cell-depleted Haploidentical Hematopoietic Stem Cell Transplantation in a Child with Dyskeratosis Congenita after a Fludarabine-based Conditioning Regimen

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Abstract

Summary: Allogeneic hematopoietic stem cell transplantation (HSCT) is the only cure for marrow failure associated with dyskeratosis congenita (DC). Data on transplants from alternative donors are limited. We describe a boy with DC and severe aplastic anemia who underwent haploidentical T-cell depleted HSCT using a reduced-intensity conditioning regimen. He underwent engraftment without toxicity or GVHD. His posttransplant course was complicated by EBV reactivation, treated with rituximab and EBVspecific T lymphocytes. After 26 months, he is in complete chimerism, with normal blood count and no sign of GVHD or pulmonary dysfunction. To the best of our knowledge, this is the first report of DC successfully treated with allogeneic HSCT from a haploidentical family donor.

Original languageEnglish
Pages (from-to)322-326
Number of pages5
JournalJournal of Pediatric Hematology/Oncology
Volume37
Issue number4
DOIs
Publication statusPublished - Dec 1 2015

Keywords

  • allogeneic stem cell transplantation
  • constitutional bone marrow failure
  • dyskeratosis congenita
  • reduced intensity
  • T-cell depletion

ASJC Scopus subject areas

  • Pediatrics, Perinatology, and Child Health
  • Oncology
  • Hematology
  • Medicine(all)

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