Successful T-cell-depleted haploidentical hematopoietic stem cell transplantation in a child with dyskeratosis congenita after a fludarabine-based conditioning regimen.
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.
*Oncoematologia Pediatrica ‡Struttura Complessa di Radioterapia Oncologica, Fondazione IRCCS Policlinico San Matteo †Servizio di Immunoematologia e Medicina Trasfusionale, Fondazione IRCCS Policlinico San Matteo, Pavia §Clinica Pediatrica, Policlinico Sant'Orsola, Bologna, Italy.
Mattia
M
Algeri
Patrizia
P
Comoli
Luisa
L
Strocchio
Cesare
C
Perotti
Franco
F
Corbella
Claudia
C
Del Fante
Ambrogia
A
Baio
Giovanna
G
Giorgiani
Antonella
A
Gurrado
Elisa
E
Accornero
Chiara
C
Cugno
Andrea
A
Pession
Marco
M
Zecca
D002648:Child; D019871:Dyskeratosis Congenita / Q000628:therapy*; D018380:Hematopoietic Stem Cell Transplantation; D006801:Humans; D008212:Lymphocyte Depletion; D008297:Male; D013601:T-Lymphocytes / Q000276:immunology*; D019172:Transplantation Conditioning; D014740:Vidarabine / Q000031:analogs & derivatives* / Q000627:therapeutic use; D014916:Whole-Body Irradiation
D014740:Vidarabine; C024352:fludarabine