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Occurrence of long-term effects after hematopoietic stem cell transplantation in children affected by acute leukemia receiving either busulfan or total body irradiation: results of an AIEOP (Associazione Italiana Ematologia Oncologia Pediatrica) retrospective study

Bone Marrow Transplantation volume 55, pages 1918–1927 (2020)Cite this article

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Abstract

Patients given allogeneic hematopoietic stem cell transplantation (alloHSCT) present an increased incidence of long-term toxicities that can be attributed to the preparative regimen. We retrospectively analyzed in a population of 670 children receiving allo-HSCT for acute leukemia the occurrence of different late effects in function of the choice made between total body irradiation (TBI) and busulfan, as part of the preparative regimen. In univariable analysis, we found that patients treated with TBI developed cataract in 24% of the cases compared with 4% in patients treated with BU (p = 0.0001) and that the incidence of secondary malignant neoplasia (SMN) was higher in patients treated with TBI (18%) as compared with those prepared to the allograft with a Bu-based regimen (0%) (p = 0.019). Conditioning regimen did not show a statistically significant correlation with the occurrence of all the other investigated late effects. In multivariable analysis, TBI remained associated with the occurrence of cataracts (Relative Risk: 0.33 p = 0.012) and secondary malignancies (Relative Risk 3.96 × 10e−6 p < 0.001); however, other variables, as GvHD and disease type, were also correlated with these long-term sequels, indicating that in our study population the preparative regimen is not the only factor influencing the incidence of these complications.

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Fig. 1: Cumulative Incidence of long-term effects.

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Acknowledgements

This work was supported by Associazione Donatrici Italiane Sangue Cordone Ombelicale (ADISCO) sezione Piemonte, Associazione Italiana contro le Leucemie, i Linfomi e il Mieloma (AIL) Sezione di Torino e Fondazione Umberto Veronesi.

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Authors and Affiliations

  1. Pediatric Onco-Hematology, Stem Cell Transplantation and Cellular Therapy Division, Regina Margherita Children’s Hospital, Torino, Italy

    Francesco Saglio, Paola Quarello & Franca Fagioli

  2. University of Turin, Turin, Italy

    Francesco Saglio, Paola Quarello & Franca Fagioli

  3. Pediatric Hematology/Oncology, Fondazione IRCCS Policlinico San Matteo, Pavia, Italy

    Marco Zecca & Giovanna Giorgiani

  4. Department of Pediatric Hematology and Oncology, IRCCS, Ospedale Bambino Gesù, Rome, Italy

    Daria Pagliara & Franco Locatelli

  5. Bone Marrow Transplantation Unit, Pediatric Department of Milano-Bicocca University, Monza e Brianza per il Bambino e la sua Mamma Foundation, Monza, Italy

    Adriana Balduzzi

  6. Pediatric Hematology-Oncology Unit, Department of Women’s and Children’s Health, Azienda Ospedaliera-University of Padova, Padova, Italy

    Elisabetta Calore

  7. Transplantation Unit, Department of Pediatric Oncology, Meyer Children’s Hospital, Florence, Italy

    Claudio Favre

  8. Hematopoietic Stem Cell Unit, Department of Hematology-Oncology, IRCSS-Istituto G. Gaslini, Genova, Italy

    Maura Faraci

  9. Oncology, Hematology and Hematopoietic Stem Cell Transplant Program, U.O. Pediatrics- S, Orsola-Malpighi University of Bologna, Bologna, Italy

    Arcangelo Prete

  10. Department of Hemato-Oncology, Santobono-Pausilipon Hospital, Bone Marrow Transplant Unit, Napoli, Italy

    Francesco Paolo Tambaro

  11. Sapienza University of Rome, Rome, Italy

    Franco Locatelli

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  1. Francesco Saglio
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Correspondence to Francesco Saglio.

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Saglio, F., Zecca, M., Pagliara, D. et al. Occurrence of long-term effects after hematopoietic stem cell transplantation in children affected by acute leukemia receiving either busulfan or total body irradiation: results of an AIEOP (Associazione Italiana Ematologia Oncologia Pediatrica) retrospective study. Bone Marrow Transplant 55, 1918–1927 (2020). https://doi.org/10.1038/s41409-020-0806-8

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