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.

Clinica Pediatrica Università degli Studi di Milano Bicocca Fondazione MBBM Ospedale San Gerardo Monza Italy

Department of Hematology Skanes University Hopsital Lund Sweden

Department of Hematology University Hospital of Children Utrecht the Netherlands

Department of Paediatric Haematology and Oncology Haematopoietic Stem Cell Transplantation Unit Comenius University Children's Hospital Bratislava Slovakia

Department of Paediatric Haematology and Oncology University Hospital Motol Prague Czech Republic

Department of Pediatric Hemato Immunology Hôpital Robert Debré APHP Nord Université de Paris Paris France

Division for Stem Cell Transplantation and Immunology Department for Children and Adolescents University Hospital Frankfurt Goethe University Frankfurt am Main Germany

Pediatric Stem Cell Transplantation Unit Medical Park Antalya Hospital Antalya Turkey

Pediatric Stem Cell Transplantation UZH University of Zurich Zurich Switzerland

Poznan University of Medical Sciences Depratment of Pediatric Oncology Hematology and HSCT Poznan Poland

Rigshospitalet Paediatric Clinic 2 Copenhagen Denmark

St Anna Children's Hospital Universitätsklinik für Kinder und Jugendheilkunde Medizinische Universität Wien Vienna Austria

The Raina Zaizov Pediatric Hematology Oncology Division Schneider Children's Medical Center of Israel Petach Tikva Israel

Willem Alexander Children's Hospital Leiden University Medical Center Leiden the Netherlands

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