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

被引：6

作者：

Algeri, Marria ^{[1
]}

Comoli, Patrizia ^{[1
]}

Strocchio, Luisa ^{[1
]}

Perotti, Cesare ^{[3
]}

Corbella, Franco ^{[2
]}

Del Fante, Claudia ^{[3
]}

Baio, Ambrogia ^{[2
]}

Giorgiani, Giovanna ^{[1
]}

Gurrado, Antonella ^{[1
]}

Accornero, Elisa ^{[1
]}

Cugno, Chiara ^{[1
]}

Pession, Andrea ^{[4
]}

Zecca, Marco ^{[1
]}

机构：

[1] Fdn IRCCS Policlin San Matteo, Oncoematol Pediat, I-27100 Pavia, Italy

[2] Fdn IRCCS Policlin San Matteo, Struttura Complessa Radioterapia Oncol, I-27100 Pavia, Italy

[3] Fdn IRCCS Policlin San Matteo, Serv Immunoematol & Med Trasfus, I-27100 Pavia, Italy

[4] Policlin St Orsola, Pediat Clin, Bologna, Italy

来源：

JOURNAL OF PEDIATRIC HEMATOLOGY ONCOLOGY | 2015年 / 37卷 / 04期

关键词：

allogeneic stem cell transplantation; dyskeratosis congenita; constitutional bone marrow failure; T-cell depletion; reduced intensity; CORD BLOOD TRANSPLANTATION; BONE-MARROW-TRANSPLANTATION; FANCONI-ANEMIA; ANTITHYMOCYTE GLOBULIN; PATIENT; CYCLOPHOSPHAMIDE; FAILURE; ENGRAFTMENT; INJECTION; DISEASE;

D O I：

10.1097/MPH.0000000000000283

中图分类号：

R73 [肿瘤学];

学科分类号：

100214 ;

摘要：

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 EBV-specific 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.

引用

页码：322 / 326

页数：5

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