Abstract
Allogeneic HSCT represents the only potentially curative treatment for very high risk (VHR) ALL. Two consecutive international prospective studies, ALL-SCT-(I)BFM 2003 and 2007 were conducted in 1150 pediatric patients. 569 presented with VHR disease leading to any kind of HSCT. All patients >2 year old were transplanted after TBI-based MAC. The median follow-up was 5 years. 463 patients were transplanted from matched donor (MD) and 106 from mismatched donor (MMD). 214 were in CR1. Stem cell source was unmanipulated BM for 330 patients, unmanipulated PBSC for 135, ex vivo T-cell depleted PBSC for 62 and cord-blood for 26. There were more advanced disease, more ex vivo T-cell depletion, and more chemotherapy based conditioning regimen for patients transplanted from MMD as compared to those transplanted from MSD or MD. Median follow up (reversed Kaplan Meier estimator) was 4.99 years, median follow up of survivals was 4.88, range (0.01–11.72) years. The 4-year CI of extensive cGvHD was 13 ± 2% and 17 ± 4% (p = NS) for the patients transplanted from MD and MMD, respectively. 4-year EFS was statistically better for patients transplanted from MD (60 ± 2% vs. 42 ± 5%, p < 0.001) for the whole cohort. This difference does not exist if considering separately patients treated in the most recent study. There was no difference in 4-year CI of relapse. The 4-year NRM was lower for patients transplanted from MD (9 ± 1% vs. 23 ± 4%, p < 0.001). In multivariate analysis, donor-type appears as a negative risk-factor for OS, EFS, and NRM. This paper demonstrates the impact of donor type on overall results of allogeneic stem cell transplantation for very-high risk pediatric acute lymphoblastic leukemia with worse results when using MMD stem cell source.
Highlights
Allogeneic hematopoietic stem cell transplantation (HSCT) was developed as an additional treatment for patients presenting with high risk malignant hematological diseases and may represent the only curative option for very high risk acute lymphoblastic leukemia
Many different parameters play a role into the final results measured by both overall survival (OS), event-free survival (EFS), non relapse mortality (NRM) and more recently defined graft-versus-hostrelapse-free-survival such as disease profile risk at diagnosis, disease status at the time of HSCT (CR1/CR2 vs beyond CR2), donor type, stem cell source (Bone Marrow (BM), Peripheral Blood Stem Cell (PBMC), Cord Blood Unit (CBU)), conditioning regimen (TBI-based, chemo-based, myelo-ablative conditioning regimen vs. reduced toxicity conditioning regimen vs. less intensive conditioning regimen i.e., non myéloablative conditioning regimen and reduced intensity ones)
There were more advanced disease, more ex vivo T-cell depletion, and more chemotherapy based conditioning regimen for patients transplanted from mismatched donor (MMD) as compared to those transplanted from MSD or matched donor (MD)
Summary
Allogeneic hematopoietic stem cell transplantation (HSCT) was developed as an additional treatment for patients presenting with high risk malignant hematological diseases and may represent the only curative option for very high risk acute lymphoblastic leukemia. Extended author information available on the last page of the article procedure success before applying HSCT. Many different parameters play a role into the final results measured by both overall survival (OS), event-free survival (EFS), non relapse mortality (NRM) and more recently defined graft-versus-hostrelapse-free-survival such as disease profile risk at diagnosis, disease status at the time of HSCT (CR1/CR2 vs beyond CR2), donor type (sibling, full-HLA compatible donor, haplodonor), stem cell source (Bone Marrow (BM), Peripheral Blood Stem Cell (PBMC), Cord Blood Unit (CBU)), conditioning regimen (TBI-based, chemo-based, myelo-ablative conditioning regimen vs reduced toxicity conditioning regimen vs less intensive conditioning regimen i.e., non myéloablative conditioning regimen and reduced intensity ones). It’s mainly based on biological and molecular characteristics at time of diagnosis and on minimal residual disease (MRD) level after one or two courses of chemotherapy
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