Outcome of Haematopoietic Stem Cell Transplantation in Dyskeratosis Congenita

Outcome of Haematopoietic Stem Cell Transplantation in Dyskeratosis Congenita Publisher Pubmed

Fioredda F¹ ; Iacobelli S² ; Korthof ET³ ; Knol C⁴ ; Van Biezen A⁴ ; Bresters D⁵ ; Veys P⁶ ; Yoshimi A⁷ ; Fagioli F⁸ ; Mats B⁹ ; Zecca M¹⁰ ; Faraci M¹¹ ; Miano M¹ ; Arcuri L¹ Show All Authors

Fioredda F¹
Iacobelli S²
Korthof ET³
Knol C⁴
Van Biezen A⁴
Bresters D⁵
Veys P⁶
Yoshimi A⁷
Fagioli F⁸
Mats B⁹
Zecca M¹⁰
Faraci M¹¹
Miano M¹
Arcuri L¹
Maschan M¹²
Obrien T¹³
Diaz MA¹⁴
Sevilla J¹⁵
Smith O¹⁶
Peffault De Latour R¹⁷
De La Fuente J¹⁸
Or R¹⁹
Van Lint MT²⁰
Tolar J²¹
Aljurf M²²
Fisher A²³
Skorobogatova EV²⁴
Diaz De Heredia C²⁵
Risitano A²⁶
Dalle JH²⁷
Sedlacek P²⁸
Ghavamzadeh A²⁹
Dufour C¹

Source: British Journal of Haematology Published:2018

Abstract

Dyskeratosis congenita (DC) is a genetic multisystem disorder with frequent involvement of the bone marrow. Haematopoietic stem cell transplantation (HSCT) is the only definitive cure to restore haematopoiesis, even though it cannot correct other organ dysfunctions. We collected data on the outcome of HSCT in the largest cohort of DC (n = 94) patients ever studied. Overall survival (OS) and event-free survival (EFS) at 3 years after HSCT were 66% and 62%, respectively. Multivariate analysis showed better outcomes in patients aged less than 20 years and in patients transplanted from a matched, rather than a mismatched, donor. OS and EFS curves tended to decline over time. Early lethal events were infections, whereas organ damage and secondary malignancies appeared afterwards, even a decade after HSCT. A non-myeloablative conditioning regimen appeared to be most advisable. Organ impairment present before HSCT seemed to favour the development of chronic graft-versus-host disease and T-B immune deficiency appeared to enhance pulmonary fibrosis. According to the present data, HSCT in DC is indicated in cases of progressive marrow failure, whereas in patients with pre-existing organ damage, this should be carefully evaluated. Further efforts to investigate treatment alternatives to HSCT should be encouraged. © 2018 British Society for Haematology and John Wiley & Sons Ltd

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Style	Citing Format
MLA	Fioredda F, et al.. "Outcome of Haematopoietic Stem Cell Transplantation in Dyskeratosis Congenita." British Journal of Haematology, vol. 183, no. 1, 2018, pp. 110-118.
APA	Fioredda F, Iacobelli S, Korthof ET, Knol C, Van Biezen A, Bresters D, Veys P, Yoshimi A, Fagioli F, Mats B, Zecca M, Faraci M, Miano M, Arcuri L, Maschan M, Obrien T, Diaz MA, Sevilla J, Smith O, ... Dufour C (2018). Outcome of Haematopoietic Stem Cell Transplantation in Dyskeratosis Congenita. British Journal of Haematology, 183(1), 110-118.
Chicago	Fioredda F, Iacobelli S, Korthof ET, Knol C, Van Biezen A, Bresters D, Veys P, et al.. "Outcome of Haematopoietic Stem Cell Transplantation in Dyskeratosis Congenita." British Journal of Haematology 183, no. 1 (2018): 110-118.
Harvard	Fioredda F et al. (2018) 'Outcome of Haematopoietic Stem Cell Transplantation in Dyskeratosis Congenita', British Journal of Haematology, 183(1), pp. 110-118.
Vancouver	Fioredda F, Iacobelli S, Korthof ET, Knol C, Van Biezen A, Bresters D, et al.. Outcome of Haematopoietic Stem Cell Transplantation in Dyskeratosis Congenita. British Journal of Haematology. 2018;183(1):110-118.
BibTex	@article{ author = {Fioredda F and Iacobelli S and Korthof ET and Knol C and Van Biezen A and Bresters D and Veys P and Yoshimi A and Fagioli F and Mats B and Zecca M and Faraci M and Miano M and Arcuri L and Maschan M and Obrien T and Diaz MA and Sevilla J and Smith O and Peffault De Latour R and De La Fuente J and Or R and Van Lint MT and Tolar J and Aljurf M and Fisher A and Skorobogatova EV and Diaz De Heredia C and Risitano A and Dalle JH and Sedlacek P and Ghavamzadeh A and Dufour C}, title = {Outcome of Haematopoietic Stem Cell Transplantation in Dyskeratosis Congenita}, journal = {British Journal of Haematology}, volume = {183}, number = {1}, pages = {110-118}, year = {2018} }
RIS	TY - JOUR AU - Fioredda F AU - Iacobelli S AU - Korthof ET AU - Knol C AU - Van Biezen A AU - Bresters D AU - Veys P AU - Yoshimi A AU - Fagioli F AU - Mats B AU - Zecca M AU - Faraci M AU - Miano M AU - Arcuri L AU - Maschan M AU - Obrien T AU - Diaz MA AU - Sevilla J AU - Smith O AU - Peffault De Latour R AU - De La Fuente J AU - Or R AU - Van Lint MT AU - Tolar J AU - Aljurf M AU - Fisher A AU - Skorobogatova EV AU - Diaz De Heredia C AU - Risitano A AU - Dalle JH AU - Sedlacek P AU - Ghavamzadeh A AU - Dufour C TI - Outcome of Haematopoietic Stem Cell Transplantation in Dyskeratosis Congenita JO - British Journal of Haematology VL - 183 IS - 1 SP - 110 EP - 118 PY - 2018 ER -

Style

Citing Format

MLA

Fioredda F, et al.. "Outcome of Haematopoietic Stem Cell Transplantation in Dyskeratosis Congenita." British Journal of Haematology, vol. 183, no. 1, 2018, pp. 110-118.

APA

Fioredda F, Iacobelli S, Korthof ET, Knol C, Van Biezen A, Bresters D, Veys P, Yoshimi A, Fagioli F, Mats B, Zecca M, Faraci M, Miano M, Arcuri L, Maschan M, Obrien T, Diaz MA, Sevilla J, Smith O, ... Dufour C (2018). Outcome of Haematopoietic Stem Cell Transplantation in Dyskeratosis Congenita. British Journal of Haematology, 183(1), 110-118.

Chicago

Fioredda F, Iacobelli S, Korthof ET, Knol C, Van Biezen A, Bresters D, Veys P, et al.. "Outcome of Haematopoietic Stem Cell Transplantation in Dyskeratosis Congenita." British Journal of Haematology 183, no. 1 (2018): 110-118.

Harvard

Fioredda F et al. (2018) 'Outcome of Haematopoietic Stem Cell Transplantation in Dyskeratosis Congenita', British Journal of Haematology, 183(1), pp. 110-118.

Vancouver

Fioredda F, Iacobelli S, Korthof ET, Knol C, Van Biezen A, Bresters D, et al.. Outcome of Haematopoietic Stem Cell Transplantation in Dyskeratosis Congenita. British Journal of Haematology. 2018;183(1):110-118.

BibTex

@article{ author = {Fioredda F and Iacobelli S and Korthof ET and Knol C and Van Biezen A and Bresters D and Veys P and Yoshimi A and Fagioli F and Mats B and Zecca M and Faraci M and Miano M and Arcuri L and Maschan M and Obrien T and Diaz MA and Sevilla J and Smith O and Peffault De Latour R and De La Fuente J and Or R and Van Lint MT and Tolar J and Aljurf M and Fisher A and Skorobogatova EV and Diaz De Heredia C and Risitano A and Dalle JH and Sedlacek P and Ghavamzadeh A and Dufour C}, title = {Outcome of Haematopoietic Stem Cell Transplantation in Dyskeratosis Congenita}, journal = {British Journal of Haematology}, volume = {183}, number = {1}, pages = {110-118}, year = {2018} }

RIS

TY - JOUR
AU - Fioredda F
AU - Iacobelli S
AU - Korthof ET
AU - Knol C
AU - Van Biezen A
AU - Bresters D
AU - Veys P
AU - Yoshimi A
AU - Fagioli F
AU - Mats B
AU - Zecca M
AU - Faraci M
AU - Miano M
AU - Arcuri L
AU - Maschan M
AU - Obrien T
AU - Diaz MA
AU - Sevilla J
AU - Smith O
AU - Peffault De Latour R
AU - De La Fuente J
AU - Or R
AU - Van Lint MT
AU - Tolar J
AU - Aljurf M
AU - Fisher A
AU - Skorobogatova EV
AU - Diaz De Heredia C
AU - Risitano A
AU - Dalle JH
AU - Sedlacek P
AU - Ghavamzadeh A
AU - Dufour C
TI - Outcome of Haematopoietic Stem Cell Transplantation in Dyskeratosis Congenita
JO - British Journal of Haematology
VL - 183
IS - 1
SP - 110
EP - 118
PY - 2018
ER -

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