Outcome of Second Conditioned Allogeneic Stem Cell Transplant in Children with Non-SCID Inborn Errors of Immunity

Graft failure post-allogeneic haematopoietic stem cell transplant (HSCT) is life threatening complication. The Inborn Errors Working Party (IEWP) conducted a retrospective study to examine outcome of second HSCT for children with non-SCID inborn errors of immunity (IEI). 159 children from 37 centres who received a second transplant between 2009-2020 were included in this analysis. The median interval between first and second HSCT was 6.9 months (0.7-155.2 months). The 5-year OS and EFS were 78% and 69%, respectively. Second HSCT for primary graft failure had a significantly lower OS (69%,55-83% versus secondary graft failure, 81%,73-89%; p=0.044) and EFS (52%,37-68% versus secondary graft failure, 75%,67-84%; p<0.001). Viral infections at second HSCT resulted in significantly lower EFS (p=0.030), but not OS (p=0.089). Improved EFS was observed in patients who received MAC/RTC (74%,66-81%) compared to NMA (50%,29-71%, p=0.003), but this association was not observed in OS. Upon multivariable analysis, cord blood was the only independent negative predictor for EFS (HR 4.9,1.3-18.7, p=0.020). The 1-year cumulative incidence (CIN) of all graft failure post-second HSCT was 13% (95% confidence intervals (CI),8-19%). The Day-100 CIN of grade II and grade III-IV aGvHD was 19% (13-25%) and 8% (4-13%), respectively. Whole blood chimerism >90% was reported in 84.3% at last follow-up. We report the first international experience with the largest cohort of second conditioned HSCT in non-SCID IEI to date. This study provides valuable insights into the clinical outcomes following second transplant, identifying key predictors of survival.

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Journal
Blood Advances
Published
2026-03-03
DOI
https://doi.org/10.1182/bloodadvances.2025015971
Primary Topic
Immunodeficiency and Autoimmune Disorders
Type
article
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article

Outcome of Second Conditioned Allogeneic Stem Cell Transplant in Children with Non-SCID Inborn Errors of Immunity

Philippe Lewalle, Krzysztof Kałwak, Wolfgang Schwinger, Alessandra Biffi et al.
Blood Advances
Immunodeficiency and Autoimmune Disorders
article

Outcome of Second Conditioned Allogeneic Stem Cell Transplant in Children with Non-SCID Inborn Errors of Immunity

Philippe Lewalle, Krzysztof Kałwak, Wolfgang Schwinger, Alessandra Biffi, Ansgar Schulz, Matthias Eyrich, Hawazen Saleh Alsaedi, Jochen Buechner, Barış Kuşkonmaz, Bierings Mb, J Winiarski, Tiarlan Sirait, R. Scott Mitchell, P. -G. Schlegel, Mary Slatter, Federica Locatelli, T. Kerre, Patrick Gilbert, Musa KARAKÜKCÜ, Zohreh Nademi, Maura Faraci, Cristina Díaz-de-Heredia, Selim Corbacioglu, Samppa Johannes Ryhänen, Robert Wynn, Alexander Kulagin, Melissa A. Gabriel, Bénédicte Neven, Arjan C Lankester, Peter Bader, Aydan Ikinciogullari, Jolanta Gozdzik, Andrew R Gennery, Michael H Albert, Su-Han Lum, Sujal Ghosh, Tania Nicole Nicole Masmas
article en

Abstract

Graft failure post-allogeneic haematopoietic stem cell transplant (HSCT) is life threatening complication. The Inborn Errors Working Party (IEWP) conducted a retrospective study to examine outcome of second HSCT for children with non-SCID inborn errors of immunity (IEI). 159 children from 37 centres who received a second transplant between 2009-2020 were included in this analysis. The median interval between first and second HSCT was 6.9 months (0.7-155.2 months). The 5-year OS and EFS were 78% and 69%, respectively. Second HSCT for primary graft failure had a significantly lower OS (69%,55-83% versus secondary graft failure, 81%,73-89%; p=0.044) and EFS (52%,37-68% versus secondary graft failure, 75%,67-84%; p<0.001). Viral infections at second HSCT resulted in significantly lower EFS (p=0.030), but not OS (p=0.089). Improved EFS was observed in patients who received MAC/RTC (74%,66-81%) compared to NMA (50%,29-71%, p=0.003), but this association was not observed in OS. Upon multivariable analysis, cord blood was the only independent negative predictor for EFS (HR 4.9,1.3-18.7, p=0.020). The 1-year cumulative incidence (CIN) of all graft failure post-second HSCT was 13% (95% confidence intervals (CI),8-19%). The Day-100 CIN of grade II and grade III-IV aGvHD was 19% (13-25%) and 8% (4-13%), respectively. Whole blood chimerism >90% was reported in 84.3% at last follow-up. We report the first international experience with the largest cohort of second conditioned HSCT in non-SCID IEI to date. This study provides valuable insights into the clinical outcomes following second transplant, identifying key predictors of survival.

Blood Advances
Goethe University Frankfurt (DE), Jagiellonian University (PL), Oslo University Hospital (NO), Hôpital Necker-Enfants Malades (FR), University of Helsinki (FI), University of Padua (IT), Ankara University (TR), Inserm (FR), Medical University of Graz (AT), University of Würzburg (DE), First Pavlov State Medical University of St. Petersburg (RU), Leiden University Medical Center (NL), Helsinki University Hospital (FI), King Faisal Specialist Hospital & Research Centre (SA), Ghent University Hospital (BE), Istituto Giannina Gaslini (IT), Newcastle upon Tyne Hospitals NHS Foundation Trust (GB), Institut Jules Bordet (BE), Children's Hospital at Westmead (AU), Copenhagen University Hospital (DK), Karolinska Institutet (SE), Wroclaw Medical University (PL), Düsseldorf University Hospital (DE), European Society for Blood and Marrow Transplantation (NL), Bambino Gesù Children's Hospital (IT), Sydney Children's Hospital (AU), Universitätsklinikum Würzburg (DE), Princess Máxima Center (NL), Willem-Alexander Kinderziekenhuis (NL), Great North Children's Hospital (GB), Istituti di Ricovero e Cura a Carattere Scientifico (IT), University Hospital Ulm (DE), Royal Manchester Children's Hospital (GB), Heinrich Heine University Düsseldorf (DE), University of Regensburg (DE), Hacettepe University (TR), Ludwig-Maximilians-Universität München (DE), Erciyes University (TR)
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Immunodeficiency and Autoimmune Disorders
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