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 | |
| 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
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
}
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 -