Effect of stem cell source on long-term chimerism and event-free survival in children with primary immunodeficiency disorders after fludarabine and melphalan.

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Effect of stem cell source on long-term chimerism and event-free survival in children with primary immunodeficiency disorders after fludarabine and melphalan conditioning regimen  Kanchan Rao, MRCPCH, MNAMS, Stuart Adams, PhD, Waseem Qasim, MD, PhD, Zoe Allwood, BSc, MPH, Austen Worth, MRCP, MD, PhD, Juliana Silva, MD, Giovanna Lucchini, MD, Robert Chiesa, MD, Paul Veys, FRCP, FRCPath, Persis Amrolia, PhD, FRCP, FRCPath  Journal of Allergy and Clinical Immunology  Volume 138, Issue 4, Pages 1152-1160 (October 2016) DOI: 10.1016/j.jaci.2016.01.053 Copyright © 2016 American Academy of Allergy, Asthma & Immunology Terms and Conditions

Fig 1 Lineage-specific chimerism in the BM and PBSC groups. Chimerism in the T-cell (T) and myeloid lineage (M) at 1 month, 6 months, and 1 year after HSCT and at last follow-up in the BM and PBSC groups is shown. At all time points, the incidence of MC was higher in the BM group than in the PBSC group, especially in the myeloid lineage. At last follow-up, the incidence of very low-level MC in the myeloid lineage of the BM group was 26% compared with 8% in the PBSC group. *In case of second transplantation or DLI, chimerism immediately before the second procedure is represented here. Journal of Allergy and Clinical Immunology 2016 138, 1152-1160DOI: (10.1016/j.jaci.2016.01.053) Copyright © 2016 American Academy of Allergy, Asthma & Immunology Terms and Conditions

Fig 2 Chimerism according to donor source at last follow-up. A, With BM as the stem cell source, mMUDs had a 40% incidence of very low-level MC, which was most evident in the myeloid lineage. B, With PBSCs as the stem cell source, 100% of mismatched donors achieved complete donor chimerism in all lineages. MSDs also had a 30% incidence of very low-level MC in the myeloid lineage of the BM group. M, Myeloid engraftment; T, T-cell engraftment. Journal of Allergy and Clinical Immunology 2016 138, 1152-1160DOI: (10.1016/j.jaci.2016.01.053) Copyright © 2016 American Academy of Allergy, Asthma & Immunology Terms and Conditions

Fig 3 GVHD after BM and PBSC transplantations. Incidence of significant (grade II or greater), severe aGVHD (grade III and IV), and cGVHD was low with BM transplants. There was a significantly higher incidence of aGVHD and cGVHD with PBSC transplants from mismatched donors. The incidence of GVHD with PBSC transplants from matched donors was low and similar to that in the BM group. The incidence of severe GVHD was only 4% in the matched PBSC setting. Journal of Allergy and Clinical Immunology 2016 138, 1152-1160DOI: (10.1016/j.jaci.2016.01.053) Copyright © 2016 American Academy of Allergy, Asthma & Immunology Terms and Conditions

Fig 4 A-C, Overall survival in the BM and PBSC groups, event-free survival in patients with less than 10% donor chimerism compared with patients with greater than 10% donor chimerism, and survival according to donor type are shown. Survival was very good in the BM and PBSC groups at 76% and 84%, respectively. There was no statistical difference in survival according to donor type. Patients with less than 10% donor chimerism had significantly poorer event-free survival at only 25% compared with 70% in patients with higher levels of chimerism. Journal of Allergy and Clinical Immunology 2016 138, 1152-1160DOI: (10.1016/j.jaci.2016.01.053) Copyright © 2016 American Academy of Allergy, Asthma & Immunology Terms and Conditions