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

Mattia Algeri, Patrizia Comoli, Luisa Strocchio, Cesare Perotti, Franco Corbella, Claudia Del Fante, Ambrogia Baio, Giovanna Giorgiani, Antonella Gurrado, Elisa Accornero, Chiara Cugno, Andrea Pession, Marco Zecca

Research output: Contribution to journalArticle

7 Citations (Scopus)

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 - 1 Dec 2015
Externally publishedYes

Fingerprint

Dyskeratosis Congenita
Hematopoietic Stem Cell Transplantation
T-Lymphocytes
Tissue Donors
Chimerism
Aplastic Anemia
Human Herpesvirus 4
Bone Marrow
Transplants
Lung
fludarabine
Conditioning (Psychology)

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)

Cite this

Successful T-cell-depleted Haploidentical Hematopoietic Stem Cell Transplantation in a Child with Dyskeratosis Congenita after a Fludarabine-based Conditioning Regimen. / Algeri, Mattia; Comoli, Patrizia; Strocchio, Luisa; Perotti, Cesare; Corbella, Franco; Fante, Claudia Del; Baio, Ambrogia; Giorgiani, Giovanna; Gurrado, Antonella; Accornero, Elisa; Cugno, Chiara; Pession, Andrea; Zecca, Marco.

In: Journal of Pediatric Hematology/Oncology, Vol. 37, No. 4, 01.12.2015, p. 322-326.

Research output: Contribution to journalArticle

Algeri, M, Comoli, P, Strocchio, L, Perotti, C, Corbella, F, Fante, CD, Baio, A, Giorgiani, G, Gurrado, A, Accornero, E, Cugno, C, Pession, A & Zecca, M 2015, 'Successful T-cell-depleted Haploidentical Hematopoietic Stem Cell Transplantation in a Child with Dyskeratosis Congenita after a Fludarabine-based Conditioning Regimen', Journal of Pediatric Hematology/Oncology, vol. 37, no. 4, pp. 322-326. https://doi.org/10.1097/MPH.0000000000000283
Algeri, Mattia ; Comoli, Patrizia ; Strocchio, Luisa ; Perotti, Cesare ; Corbella, Franco ; Fante, Claudia Del ; Baio, Ambrogia ; Giorgiani, Giovanna ; Gurrado, Antonella ; Accornero, Elisa ; Cugno, Chiara ; Pession, Andrea ; Zecca, Marco. / Successful T-cell-depleted Haploidentical Hematopoietic Stem Cell Transplantation in a Child with Dyskeratosis Congenita after a Fludarabine-based Conditioning Regimen. In: Journal of Pediatric Hematology/Oncology. 2015 ; Vol. 37, No. 4. pp. 322-326.
@article{ce190c3e9d5640cfbc3b4cd1156d8864,
title = "Successful T-cell-depleted Haploidentical Hematopoietic Stem Cell Transplantation in a Child with Dyskeratosis Congenita after a Fludarabine-based Conditioning Regimen",
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.",
keywords = "allogeneic stem cell transplantation, constitutional bone marrow failure, dyskeratosis congenita, reduced intensity, T-cell depletion",
author = "Mattia Algeri and Patrizia Comoli and Luisa Strocchio and Cesare Perotti and Franco Corbella and Fante, {Claudia Del} and Ambrogia Baio and Giovanna Giorgiani and Antonella Gurrado and Elisa Accornero and Chiara Cugno and Andrea Pession and Marco Zecca",
year = "2015",
month = "12",
day = "1",
doi = "10.1097/MPH.0000000000000283",
language = "English",
volume = "37",
pages = "322--326",
journal = "Journal of Pediatric Hematology/Oncology",
issn = "1077-4114",
publisher = "Lippincott Williams and Wilkins",
number = "4",

}

TY - JOUR

T1 - Successful T-cell-depleted Haploidentical Hematopoietic Stem Cell Transplantation in a Child with Dyskeratosis Congenita after a Fludarabine-based Conditioning Regimen

AU - Algeri, Mattia

AU - Comoli, Patrizia

AU - Strocchio, Luisa

AU - Perotti, Cesare

AU - Corbella, Franco

AU - Fante, Claudia Del

AU - Baio, Ambrogia

AU - Giorgiani, Giovanna

AU - Gurrado, Antonella

AU - Accornero, Elisa

AU - Cugno, Chiara

AU - Pession, Andrea

AU - Zecca, Marco

PY - 2015/12/1

Y1 - 2015/12/1

N2 - 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.

AB - 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.

KW - allogeneic stem cell transplantation

KW - constitutional bone marrow failure

KW - dyskeratosis congenita

KW - reduced intensity

KW - T-cell depletion

UR - http://www.scopus.com/inward/record.url?scp=84937965607&partnerID=8YFLogxK

UR - http://www.scopus.com/inward/citedby.url?scp=84937965607&partnerID=8YFLogxK

U2 - 10.1097/MPH.0000000000000283

DO - 10.1097/MPH.0000000000000283

M3 - Article

VL - 37

SP - 322

EP - 326

JO - Journal of Pediatric Hematology/Oncology

JF - Journal of Pediatric Hematology/Oncology

SN - 1077-4114

IS - 4

ER -