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: 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.
Hematopoietic stem cell transplantation for adolescents and adults with inborn errors of immunity: an EBMT IEWP study
Albert, Michael H
First
;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
Last
2022-01-01
Abstract
: 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.
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Utilizza questo identificativo per citare o creare un link a questo documento: https://hdl.handle.net/2318/1888597
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simulazione ASN
Il report seguente simula gli indicatori relativi alla produzione scientifica in relazione alle soglie ASN 2023-2025 del proprio SC/SSD. Si ricorda che il superamento dei valori soglia (almeno 2 su 3) è requisito necessario ma non sufficiente al conseguimento dell'abilitazione.
La simulazione si basa sui dati IRIS e presenta gli indicatori calcolati alla data indicata sul report. Si ricorda che in sede di domanda ASN presso il MIUR gli indicatori saranno invece calcolati a partire dal 1° gennaio rispettivamente del quinto/decimo/quindicesimo anno precedente la scadenza del quadrimestre di presentazione della domanda (art 2 del DM 598/2018).
In questa simulazione pertanto il valore degli indicatori potrà differire da quello conteggiato all’atto della domanda ASN effettuata presso il MIUR a seguito di:
Correzioni imputabili a eventuali periodi di congedo obbligatorio, che in sede di domanda ASN danno diritto a incrementi percentuali dei valori.
Presenza di eventuali errori di catalogazione e/o dati mancanti in IRIS
Variabilità nel tempo dei valori citazionali (per i settori bibliometrici)
Variabilità della finestra temporale considerata in funzione della sessione di domanda ASN a cui si partecipa.
La presente simulazione è stata realizzata sulla base delle regole riportate nel DM 598/2018 e dell'allegata Tabella A e delle specifiche definite all'interno del Focus Group Cineca relativo al modulo IRIS ER. Il Cineca non si assume alcuna responsabilità in merito all'uso che il diretto interessato o terzi faranno della simulazione.