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

doi:10.1097/MPH.0000000000000283

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 journal › Article

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 language	English
Pages (from-to)	322-326
Number of pages	5
Journal	Journal of Pediatric Hematology/Oncology
Volume	37
Issue number	4
DOIs	https://doi.org/10.1097/MPH.0000000000000283
Publication status	Published - 1 Dec 2015
Externally published	Yes

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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

Algeri, M., Comoli, P., Strocchio, L., Perotti, C., Corbella, F., Fante, C. D., 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, 37(4), 322-326. https://doi.org/10.1097/MPH.0000000000000283

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 journal › Article

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.

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10.1097/MPH.0000000000000283