Hematopoietic stem cell transplantation for Wiskott-Aldrich syndrome: an EBMT inborn errors working party analysis
Identifikátory výsledku
Kód výsledku v IS VaVaI
<a href="https://www.isvavai.cz/riv?ss=detail&h=RIV%2F00064203%3A_____%2F22%3A10438384" target="_blank" >RIV/00064203:_____/22:10438384 - isvavai.cz</a>
Nalezeny alternativní kódy
RIV/00216208:11130/22:10438384
Výsledek na webu
<a href="https://verso.is.cuni.cz/pub/verso.fpl?fname=obd_publikace_handle&handle=_V19l9Xakg" target="_blank" >https://verso.is.cuni.cz/pub/verso.fpl?fname=obd_publikace_handle&handle=_V19l9Xakg</a>
DOI - Digital Object Identifier
<a href="http://dx.doi.org/10.1182/blood.2021014687" target="_blank" >10.1182/blood.2021014687</a>
Alternativní jazyky
Jazyk výsledku
angličtina
Název v původním jazyce
Hematopoietic stem cell transplantation for Wiskott-Aldrich syndrome: an EBMT inborn errors working party analysis
Popis výsledku v původním jazyce
Allogeneic hematopoietic stem cell transplantation (HSCT) is a potentially curative treatment for patients affected by Wiskott-Aldrich syndrome (WAS). Reported HSCT outcomes have improved over time with respect to overall survival, but some studies have identified older age and HSCT from alternative donors as risk factors predicting poorer outcome. We analyzed 197 patients transplanted at EBMT centers between 2006 and 2017, who received conditioning as recommended by the inborn errors working party (IEWP): either busulfan (n=103) or treosulfan (n=94) combined with fludarabine +- thiotepa. After a median follow-up after HSCT of 44.9 months, 176 patients were alive, resulting in a 3-year overall survival of 88.7%, and chronic GVHD-free survival (CRFS; events: death, graft failure, severe chronic GVHD) of 81.7%. Overall survival and CRFS were not significantly impacted by conditioning regimen (busulfan- versus treosulfan-based), donor type (MSD/MFD vs MUD/MMUD vs. MMFD), and period of HSCT (2006-2013 vs. 2014-2017). Patients younger than 5 years at HSCT had a significantly better overall survival. The overall cumulative incidences of grade III-IV acute GVHD and extensive/moderate/severe chronic GVHD were 6.6% and 2.1%, respectively. Patients receiving treosulfan-based conditioning had a higher incidence of graft failure, mixed donor chimerism and more frequently received secondary procedures (2nd HSCT, unconditioned stem cell boost, donor lymphocyte infusion, or splenectomy). In summary, HSCT for WAS with conditioning regimens currently recommended by IEWP results in excellent survival and low rates of GVHD, regardless of donor or stem cell source, but age >=5 years remains a risk factor for overall survival.
Název v anglickém jazyce
Hematopoietic stem cell transplantation for Wiskott-Aldrich syndrome: an EBMT inborn errors working party analysis
Popis výsledku anglicky
Allogeneic hematopoietic stem cell transplantation (HSCT) is a potentially curative treatment for patients affected by Wiskott-Aldrich syndrome (WAS). Reported HSCT outcomes have improved over time with respect to overall survival, but some studies have identified older age and HSCT from alternative donors as risk factors predicting poorer outcome. We analyzed 197 patients transplanted at EBMT centers between 2006 and 2017, who received conditioning as recommended by the inborn errors working party (IEWP): either busulfan (n=103) or treosulfan (n=94) combined with fludarabine +- thiotepa. After a median follow-up after HSCT of 44.9 months, 176 patients were alive, resulting in a 3-year overall survival of 88.7%, and chronic GVHD-free survival (CRFS; events: death, graft failure, severe chronic GVHD) of 81.7%. Overall survival and CRFS were not significantly impacted by conditioning regimen (busulfan- versus treosulfan-based), donor type (MSD/MFD vs MUD/MMUD vs. MMFD), and period of HSCT (2006-2013 vs. 2014-2017). Patients younger than 5 years at HSCT had a significantly better overall survival. The overall cumulative incidences of grade III-IV acute GVHD and extensive/moderate/severe chronic GVHD were 6.6% and 2.1%, respectively. Patients receiving treosulfan-based conditioning had a higher incidence of graft failure, mixed donor chimerism and more frequently received secondary procedures (2nd HSCT, unconditioned stem cell boost, donor lymphocyte infusion, or splenectomy). In summary, HSCT for WAS with conditioning regimens currently recommended by IEWP results in excellent survival and low rates of GVHD, regardless of donor or stem cell source, but age >=5 years remains a risk factor for overall survival.
Klasifikace
Druh
J<sub>imp</sub> - Článek v periodiku v databázi Web of Science
CEP obor
—
OECD FORD obor
30205 - Hematology
Návaznosti výsledku
Projekt
—
Návaznosti
I - Institucionalni podpora na dlouhodoby koncepcni rozvoj vyzkumne organizace
Ostatní
Rok uplatnění
2022
Kód důvěrnosti údajů
S - Úplné a pravdivé údaje o projektu nepodléhají ochraně podle zvláštních právních předpisů
Údaje specifické pro druh výsledku
Název periodika
Blood
ISSN
0006-4971
e-ISSN
1528-0020
Svazek periodika
139
Číslo periodika v rámci svazku
13
Stát vydavatele periodika
US - Spojené státy americké
Počet stran výsledku
14
Strana od-do
2066-2079
Kód UT WoS článku
000783860700017
EID výsledku v databázi Scopus
2-s2.0-85127136275