Hematopoietic stem cell transplantation for adolescents and adults with inborn errors of immunity: an EBMT IEWP study
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Hematopoietic stem cell transplantation for adolescents and adults with inborn errors of immunity: an EBMT IEWP study. / Albert, Michael H; Sirait, Tiarlan; Eikema, Dirk-Jan; Bakunina, Katerina; Wehr, Claudia; Suarez, Felipe; Fox, Maria Laura; Mahlaoui, Nizar; Gennery, Andrew R; Lankester, Arjan C; Beier, Rita; Bernardo, Maria Ester; Bigley, Venetia; Lindemans, Caroline A; Burns, Siobhan O; Carpenter, Ben; Dybko, Jaroslaw; Güngör, Tayfun; Hauck, Fabian; Lum, Su Han; Balashov, Dmitry; Meisel, Roland; Moshous, Despina; Schulz, Ansgar; Speckmann, Carsten; Slatter, Mary A; Strahm, Brigitte; Uckan-Cetinkaya, Duygu; Meyts, Isabelle; Vallée, Tanja C; Wynn, Robert; Neven, Bénédicte; Morris, Emma C; Aiuti, Alessandro; Maschan, Alexei; Aljurf, Mahmoud; Gedde-Dahl, Tobias; Gurman, Gunhan; Bordon, Victoria; Kriván, Gergely; Locatelli, Franco; Porta, Fulvio; Valcárcel, David; Beguin, Yves; Faraci, Maura; Kröger, Nicolaus; Kulagin, Aleksandr; Shaw, Peter J; Veelken, Joan Hendrik; Diaz de Heredia, Cristina; Fagioli, Franca; Felber, Matthias; Gruhn, Bernd; Holter, Wolfgang; Rössig, Claudia; Sedlacek, Petr; Apperley, Jane; Ayas, Mouhab; Bodova, Ivana; Choi, Goda; Cornelissen, J J; Sirvent, Anne; Khan, Anjum; Kupesiz, Alphan; Lenhoff, Stig; Ozdogu, Hakan; von der Weid, Nicolas; Rovira, Montserrat; Schots, Rik; Vinh, Donald C; Inborn Errors Working Party of the EBMT.
in: BLOOD, Jahrgang 140, Nr. 14, 06.10.2022, S. 1635-1649.Publikationen: SCORING: Beitrag in Fachzeitschrift/Zeitung › SCORING: Zeitschriftenaufsatz › Forschung › Begutachtung
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TY - JOUR
T1 - Hematopoietic stem cell transplantation for adolescents and adults with inborn errors of immunity: an EBMT IEWP study
AU - Albert, Michael H
AU - Sirait, Tiarlan
AU - Eikema, Dirk-Jan
AU - Bakunina, Katerina
AU - Wehr, Claudia
AU - Suarez, Felipe
AU - Fox, Maria Laura
AU - Mahlaoui, Nizar
AU - Gennery, Andrew R
AU - Lankester, Arjan C
AU - Beier, Rita
AU - Bernardo, Maria Ester
AU - Bigley, Venetia
AU - Lindemans, Caroline A
AU - Burns, Siobhan O
AU - Carpenter, Ben
AU - Dybko, Jaroslaw
AU - Güngör, Tayfun
AU - Hauck, Fabian
AU - Lum, Su Han
AU - Balashov, Dmitry
AU - Meisel, Roland
AU - Moshous, Despina
AU - Schulz, Ansgar
AU - Speckmann, Carsten
AU - Slatter, Mary A
AU - Strahm, Brigitte
AU - Uckan-Cetinkaya, Duygu
AU - Meyts, Isabelle
AU - Vallée, Tanja C
AU - Wynn, Robert
AU - Neven, Bénédicte
AU - Morris, Emma C
AU - Aiuti, Alessandro
AU - Maschan, Alexei
AU - Aljurf, Mahmoud
AU - Gedde-Dahl, Tobias
AU - Gurman, Gunhan
AU - Bordon, Victoria
AU - Kriván, Gergely
AU - Locatelli, Franco
AU - Porta, Fulvio
AU - Valcárcel, David
AU - Beguin, Yves
AU - Faraci, Maura
AU - Kröger, Nicolaus
AU - Kulagin, Aleksandr
AU - Shaw, Peter J
AU - Veelken, Joan Hendrik
AU - Diaz de Heredia, Cristina
AU - Fagioli, Franca
AU - Felber, Matthias
AU - Gruhn, Bernd
AU - Holter, Wolfgang
AU - Rössig, Claudia
AU - Sedlacek, Petr
AU - Apperley, Jane
AU - Ayas, Mouhab
AU - Bodova, Ivana
AU - Choi, Goda
AU - Cornelissen, J J
AU - Sirvent, Anne
AU - Khan, Anjum
AU - Kupesiz, Alphan
AU - Lenhoff, Stig
AU - Ozdogu, Hakan
AU - von der Weid, Nicolas
AU - Rovira, Montserrat
AU - Schots, Rik
AU - Vinh, Donald C
AU - Inborn Errors Working Party of the EBMT
N1 - © 2022 by The American Society of Hematology.
PY - 2022/10/6
Y1 - 2022/10/6
N2 - Allogeneic hematopoietic stem cell transplantation (HSCT) is the gold standard curative therapy for infants and children with many inborn errors of immunity (IEI), but adolescents and adults with IEI are rarely referred for transplant. Lack of published HSCT outcome data outside small, single-center studies and perceived high risk of transplant-related mortality have delayed the adoption of HSCT for IEI patients presenting or developing significant organ damage later in life. This large retrospective, multicenter HSCT outcome study reports on 329 IEI patients (age range, 15-62.5 years at HSCT). Patients underwent first HSCT between 2000 and 2019. Primary endpoints were overall survival (OS) and event-free survival (EFS). We also evaluated the influence of IEI-subgroup and IEI-specific risk factors at HSCT, including infections, bronchiectasis, colitis, malignancy, inflammatory lung disease, splenectomy, hepatic dysfunction, and systemic immunosuppression. At a median follow-up of 44.3 months, the estimated OS at 1 and 5 years post-HSCT for all patients was 78% and 71%, and EFS was 65% and 62%, respectively, with low rates of severe acute (8%) or extensive chronic (7%) graft-versus-host disease. On univariate analysis, OS and EFS were inferior in patients with primary antibody deficiency, bronchiectasis, prior splenectomy, hepatic comorbidity, and higher hematopoietic cell transplant comorbidity index scores. On multivariable analysis, EFS was inferior in those with a higher number of IEI-associated complications. Neither age nor donor had a significant effect on OS or EFS. We have identified age-independent risk factors for adverse outcome, providing much needed evidence to identify which patients are most likely to benefit from HSCT.
AB - Allogeneic hematopoietic stem cell transplantation (HSCT) is the gold standard curative therapy for infants and children with many inborn errors of immunity (IEI), but adolescents and adults with IEI are rarely referred for transplant. Lack of published HSCT outcome data outside small, single-center studies and perceived high risk of transplant-related mortality have delayed the adoption of HSCT for IEI patients presenting or developing significant organ damage later in life. This large retrospective, multicenter HSCT outcome study reports on 329 IEI patients (age range, 15-62.5 years at HSCT). Patients underwent first HSCT between 2000 and 2019. Primary endpoints were overall survival (OS) and event-free survival (EFS). We also evaluated the influence of IEI-subgroup and IEI-specific risk factors at HSCT, including infections, bronchiectasis, colitis, malignancy, inflammatory lung disease, splenectomy, hepatic dysfunction, and systemic immunosuppression. At a median follow-up of 44.3 months, the estimated OS at 1 and 5 years post-HSCT for all patients was 78% and 71%, and EFS was 65% and 62%, respectively, with low rates of severe acute (8%) or extensive chronic (7%) graft-versus-host disease. On univariate analysis, OS and EFS were inferior in patients with primary antibody deficiency, bronchiectasis, prior splenectomy, hepatic comorbidity, and higher hematopoietic cell transplant comorbidity index scores. On multivariable analysis, EFS was inferior in those with a higher number of IEI-associated complications. Neither age nor donor had a significant effect on OS or EFS. We have identified age-independent risk factors for adverse outcome, providing much needed evidence to identify which patients are most likely to benefit from HSCT.
KW - Adolescent
KW - Adult
KW - Bronchiectasis/etiology
KW - Child
KW - Graft vs Host Disease
KW - Hematopoietic Stem Cell Transplantation/adverse effects
KW - Humans
KW - Infant
KW - Middle Aged
KW - Retrospective Studies
KW - Transplantation, Homologous
KW - Young Adult
U2 - 10.1182/blood.2022015506
DO - 10.1182/blood.2022015506
M3 - SCORING: Journal article
C2 - 35344580
VL - 140
SP - 1635
EP - 1649
JO - BLOOD
JF - BLOOD
SN - 0006-4971
IS - 14
ER -