Allogeneic hematopoietic cell transplantation for XIAP deficiency: an international survey reveals poor outcomes

Result code in IS VaVaI

<a href="https://www.isvavai.cz/riv?ss=detail&h=RIV%2F00064203%3A_____%2F13%3A10209601" target="_blank" >RIV/00064203:_____/13:10209601 - isvavai.cz</a>

Alternative codes found

RIV/00216208:11130/13:10209601

Result on the web

<a href="http://dx.doi.org/10.1182/blood-2012-06-432500" target="_blank" >http://dx.doi.org/10.1182/blood-2012-06-432500</a>

DOI - Digital Object Identifier

<a href="http://dx.doi.org/10.1182/blood-2012-06-432500" target="_blank" >10.1182/blood-2012-06-432500</a>

Result language

angličtina

Original language name

Allogeneic hematopoietic cell transplantation for XIAP deficiency: an international survey reveals poor outcomes

Original language description

There have been no studies on patient outcome after allogeneic hematopoietic cell transplantation (HCT) in patients with X-linked inhibitor of apoptosis (XIAP) deficiency. To estimate the success of HCT, we conducted an international survey of transplantation outcomes. Data were reported for 19 patients. Seven patients received busulfan-containing myeloablative conditioning (MAC) regimens. Eleven patients underwent reduced intensity conditioning (RIC) regimens predominantly consisting of alemtuzumab, fludarabine, and melphalan. One patient received an intermediate-intensity regimen. Survival was poor in the MAC group, with only 1 patient surviving (14%). Most deaths were from transplantation-related toxicities, including venoocclusive disease and pulmonary hemorrhage. Of the 11 patients who received RIC, 6 are currently surviving at a median of 570 days after HCT (55%). Preparative regimen and HLH activity affected outcomes, and of RIC patients reported to be in remission from HLH, sur

Czech name

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Czech description

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Type

J_x - Unclassified - Peer-reviewed scientific article (Jimp, Jsc and Jost)

CEP classification

FD - Oncology and haematology

OECD FORD branch

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Project

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Continuities

I - Institucionalni podpora na dlouhodoby koncepcni rozvoj vyzkumne organizace

Publication year

2013

Confidentiality

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

Name of the periodical

Blood

ISSN

0006-4971

e-ISSN

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Volume of the periodical

121

Issue of the periodical within the volume

6

Country of publishing house

US - UNITED STATES

Number of pages

7

Pages from-to

877-883

UT code for WoS article

000314868800008

EID of the result in the Scopus database

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