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Irradiation-free re-conditioning in children following graft failure of a T cell-depleted graft from a haploidentical parent

Bone Marrow Transplantation volume 56, pages 1452–1454 (2021)Cite this article

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Haplo-identical stem cell transplantation (hHSCT) is an option for patients who require a stem cell transplantation, but lack an HLA-identical sibling or HLA-matched unrelated donor [1,2,3]. Various modes of hHSCT are currently employed [4]. Here, we focus on transplantation of T cell-depleted HLA-haploidentical stem cells (TCD hHSCT) from a parent to their child. This maneuver is associated with a risk of graft failure in 10% of these procedures. Graft rejection represents a life-threatening complication and requires timely re-transplantation [5]. Teltschik et al. reported on a cohort of 16 pediatric patients who underwent a second TCD hHSCT using a reconditioning regimen containing total nodal irradiation prior to the re-transplantation [6]. Although localized irradiation represents a toxicity-reduced approach, various reasons argue to avoid irradiation in pediatric transplant. In the present retrospective study, we identified nine patients with graft failure after first TCD hHSCT who received a salvage TCD hHSCT from the other parent preceded by an irradiation-free lymphodepleting reconditioning regimen. All of these patients achieved sustained engraftment and complete donor chimerism.

We report on a cohort of nine children who underwent T cell-depleted HLA-haploidentical stem cell transplantation from a parent between 2011 and 2019. Median age at first transplantation was 2.3 years (range 3 months to 10 years). We reviewed seven female and two male patients, whose indication for the transplantation was a malignancy in four cases (two cases of myelodysplastic syndrome, one case of each, acute lymphoblastic leukemia and neuroblastoma). In five patients, indication for HSCT was based on non-malignant diseases, i.e., mucopolysaccharidosis Type I Hurler in three cases and hemophagocytic lymphohistiocytosis in two children. Five patients had secondary graft failure and four patients showed non-engraftment after their first TCD hHSCT. In all cases, one parent donated for the first transplantation, and the other parent for the re-transplantation.

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Authors and Affiliations

  1. Division for Pediatric Stem Cell Transplantation and Immunology, Clinic for Pediatric Hematology and Oncology, University Medical Center Hamburg Eppendorf, Hamburg, Germany

    Susanne Aydin, Anne Kruchen, Katharina Wustrau, Johanna Schrum & Ingo Müller

  2. Department I-General Pediatrics, Haematology/Oncology, University Hospital Tübingen-Children’s Hospital, Tübingen, Germany

    Michaela Doering

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  1. Susanne Aydin
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  2. Anne Kruchen
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  3. Katharina Wustrau
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  6. Ingo Müller
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Correspondence to Ingo Müller.

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Aydin, S., Kruchen, A., Wustrau, K. et al. Irradiation-free re-conditioning in children following graft failure of a T cell-depleted graft from a haploidentical parent. Bone Marrow Transplant 56, 1452–1454 (2021). https://doi.org/10.1038/s41409-020-01196-4

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