Outcome of haematopoietic stem cell transplantation in dyskeratosis congenita

Kód výsledku v IS VaVaI

<a href="https://www.isvavai.cz/riv?ss=detail&h=RIV%2F00216208%3A11130%2F18%3A10388889" target="_blank" >RIV/00216208:11130/18:10388889 - isvavai.cz</a>

Nalezeny alternativní kódy

RIV/00064203:_____/18:10388889

Výsledek na webu

<a href="https://doi.org/10.1111/bjh.15495" target="_blank" >https://doi.org/10.1111/bjh.15495</a>

DOI - Digital Object Identifier

<a href="http://dx.doi.org/10.1111/bjh.15495" target="_blank" >10.1111/bjh.15495</a>

Jazyk výsledku

angličtina

Název v původním jazyce

Outcome of haematopoietic stem cell transplantation in dyskeratosis congenita

Popis výsledku v původním jazyce

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 3years after HSCT were 66% and 62%, respectively. Multivariate analysis showed better outcomes in patients aged less than 20years 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.

Název v anglickém jazyce

Outcome of haematopoietic stem cell transplantation in dyskeratosis congenita

Popis výsledku anglicky

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 3years after HSCT were 66% and 62%, respectively. Multivariate analysis showed better outcomes in patients aged less than 20years 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.

Druh

J_imp - Článek v periodiku v databázi Web of Science

CEP obor

—

OECD FORD obor

30204 - Oncology

Projekt

—

Návaznosti

I - Institucionalni podpora na dlouhodoby koncepcni rozvoj vyzkumne organizace

Rok uplatnění

2018

Kód důvěrnosti údajů

S - Úplné a pravdivé údaje o projektu nepodléhají ochraně podle zvláštních právních předpisů

Název periodika

British Journal of Haematology

ISSN

0007-1048

e-ISSN

—

Svazek periodika

183

Číslo periodika v rámci svazku

1

Stát vydavatele periodika

GB - Spojené království Velké Británie a Severního Irska

Počet stran výsledku

9

Strana od-do

110-118

Kód UT WoS článku

000447750900014

EID výsledku v databázi Scopus

2-s2.0-85050608456