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Treosulfan, Fludarabine Conditioning for HSCT in Children with Primary Immunodeficiency: UK Experience

Slatter, MA; Rao, K; Abd Hamid, IJ; Nademi, Z; Chiesa, R; Elfeky, R; Pearce, MS; ... Veys, P; + view all (2018) Treosulfan, Fludarabine Conditioning for HSCT in Children with Primary Immunodeficiency: UK Experience. Biology of Blood and Marrow Transplantation , 24 (3) pp. 529-536. 10.1016/j.bbmt.2017.11.009. Green open access

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Abstract

We previously published results of 70 children who received treosulfan with cyclophosphamide (30) or fludarabine (40) before haematopoietic stem cell transplantation (HSCT) for Primary Immunodeficiency (PID). Toxicity was lower and T cell chimerism better in those receiving fludarabine, but numbers were relatively small and follow-up short. We now report outcome of 160 children who received homogeneous conditioning with treosulfan, fludarabine mostly with alemtuzumab (n=124). Median age at transplant was 1.36 years (0.09-18.25). Donors were: matched unrelated, 73; 1-3 antigen mismatched unrelated, 54; matched sibling, 12; other matched family, 17; haploidentical, 4. Stem cell source was: peripheral blood stem cells (PBSCs), 70; Bone marrow, 49; Cord Blood, 41. Median follow up was 4.3 years (0.8-9.4). Overall survival was 83%. There was no veno- occlusive disease. Seventy-four (46%) had acute GVHD, but only 14(9%) greater than grade II. Four patients were successfully retransplanted for graft loss or poor immune reconstitution. One further patient who rejected the graft, died. There was no association between T cell chimerism > 95% and stem cell source, but a significant association with myeloid chimerism > 95% and use of PBSC without an increased risk of significant GVHD compared to other sources. All 11 patients with severe combined immunodeficiency diagnosed at birth are alive with up to 8.7 years follow up. Long-term studies are required to determine late gonadotoxic effects and pharmacokinetic studies are needed to identify whether specific targeting is advantageous. The combination of treosulfan, fludarabine and alemtuzumab gives excellent results in HSCT for PID.

Type: Article
Title: Treosulfan, Fludarabine Conditioning for HSCT in Children with Primary Immunodeficiency: UK Experience
Location: United States
Open access status: An open access version is available from UCL Discovery
DOI: 10.1016/j.bbmt.2017.11.009
Publisher version: http://dx.doi.org/10.1016/j.bbmt.2017.11.009
Language: English
Additional information: This version is the author accepted manuscript. For information on re-use, please refer to the publisher’s terms and conditions.
Keywords: Chimerism, Fludarabine, Haematopoietic stem cell transplantation, Primary Immunodeficiency, Treosulfan
UCL classification: UCL
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Population Health Sciences > UCL GOS Institute of Child Health
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Population Health Sciences > UCL GOS Institute of Child Health > Developmental Neurosciences Dept
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Population Health Sciences > UCL GOS Institute of Child Health > Infection, Immunity and Inflammation Dept
URI: https://discovery.ucl.ac.uk/id/eprint/10038524
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