Multicenter experience in hematopoietic stem cell transplantation for serious complications of common variable immunodeficiency  Claudia Wehr, MD, Andrew R. Gennery, MD, Caroline Lindemans, MD, PhD, Ansgar Schulz, MD, Manfred Hoenig, MD, Reinhard Marks, MD, Mike Recher, MD, Bernd Gruhn, MD, Andreas Holbro, MD, Ingmar Heijnen, PhD, Deborah Meyer, BSc, Goetz Grigoleit, MD, Hermann Einsele, MD, Ulrich Baumann, MD, Thorsten Witte, MD, Karl-Walter Sykora, MD, Sigune Goldacker, MD, Lorena Regairaz, MD, Serap Aksoylar, MD, Ömur Ardeniz, MD, Marco Zecca, MD, Przemyslaw Zdziarski, MD, Isabelle Meyts, MD, Susanne Matthes- Martin, MD, Kohsuke Imai, MD, Chikako Kamae, MD, Adele Fielding, MD, Suranjith Seneviratne, MD, Nizar Mahlaoui, MD, MSc, MPH, Mary A. Slatter, MD, Tayfun Güngör, MD, Peter D. Arkwright, MD, Joris van Montfrans, MD, Kathleen E. Sullivan, MD, PhD, Bodo Grimbacher, MD, Andrew Cant, MD, Hans-Hartmut Peter, MD, Juergen Finke, MD, H. Bobby Gaspar, MD, Klaus Warnatz, MD, Marta Rizzi, MD, PhD  Journal of Allergy and Clinical Immunology  Volume 135, Issue 4, Pages 988-997.e6 (April 2015) DOI: 10.1016/j.jaci.2014.11.029 Copyright © 2014 American Academy of Allergy, Asthma & Immunology Terms and Conditions

Fig 1 Indication for HSCT in the whole CVID cohort (25 patients; A), patients with CVID without signs of cellular immunodeficiency (14 [56%] of 25 patients; B), and patients with CVID with signs of cellular immunodeficiency (11 [44%] of 25 patients; C). Journal of Allergy and Clinical Immunology 2015 135, 988-997.e6DOI: (10.1016/j.jaci.2014.11.029) Copyright © 2014 American Academy of Allergy, Asthma & Immunology Terms and Conditions

Fig 2 Comorbidities and mortality after HSCT. A and B, Survival (Fig 2, A) and HCT-CI (Fig 2, B), according to subgroups (defined in the the “Indication for transplantation, conditioning and stem cell source” Results section). ID, Immunodeficiency. C, Mortality according to pretransplantation PID-related complications. CNS, Central nervous system; Tx, transplantation. D and E, Survival curve (Fig 2, D) and HCT-CI (Fig 2, E) stratified per indications. For survival curves, ticks indicate the last follow-up of surviving patients. HCT-CI: Dark gray, high risk of treatment-related mortality; light gray, intermediate risk of treatment-related mortality; white, low risk of treatment-related mortality. Journal of Allergy and Clinical Immunology 2015 135, 988-997.e6DOI: (10.1016/j.jaci.2014.11.029) Copyright © 2014 American Academy of Allergy, Asthma & Immunology Terms and Conditions

Fig 3 GvHD. A, Occurrence of mild (light blue), severe (indigo), or absent (dark blue) GvHD in the whole CVID cohort and outcome of patients with severe GvHD. B and C, GvHD according to donor type (P = .9694; Fig 3, B) and stem cell source (P = .603; Fig 3, C). Solid symbols, Patients with CVID without signs of cellular immunodeficiency; open symbols, patients with CVID with signs of cellular immunodeficiency. CB, Cord blood; mMUD, Mismatched unrelated donor; MRD, matched related donor; MUD, matched unrelated donor. Journal of Allergy and Clinical Immunology 2015 135, 988-997.e6DOI: (10.1016/j.jaci.2014.11.029) Copyright © 2014 American Academy of Allergy, Asthma & Immunology Terms and Conditions

Fig 4 Reconstitution of the B-cell compartment. A, Percentage of patients withdrawn from immunoglobulin replacement (off Ig) over time. Ticks indicate last follow-up. B, B-cell numbers at last follow-up in patients still dependent on (on Ig subst.) and patients independent of (off Ig subst.) immunoglobulin substitution. Error bars show means ± SEMs. The shaded area indicates normal range. Journal of Allergy and Clinical Immunology 2015 135, 988-997.e6DOI: (10.1016/j.jaci.2014.11.029) Copyright © 2014 American Academy of Allergy, Asthma & Immunology Terms and Conditions

Fig E1 GvHD, comorbidity index, and mortality. A-C, HCT-CI stratified for survival, GvHD, and donor type. HCT-CI: dark gray, high risk of transplant-related mortality; light gray, intermediate risk of transplant-related mortality; and white, low risk of transplant-related mortality. D, Karnofsky index before transplantation (Tx) in deceased versus surviving patients. E and F, Survival curve of patients with CVID after HSCT stratified according to year of transplantation (before and from 2000 onward; Fig E1, E) and according to pediatric (age at transplantation <18 years) and adult (age at transplantation ≥18 years) status (Fig E1, F). The number of patients in each group is indicated in parentheses. G and H, Occurrence and outcome of GvHD stratified according to absent (dark blue), mild (light blue), and severe (indigo) GvHD in patients with CVID without cellular immunodeficiency (Fig E1, G) and in patients with CVID with signs of cellular immunodeficiency (Fig E1, H). Journal of Allergy and Clinical Immunology 2015 135, 988-997.e6DOI: (10.1016/j.jaci.2014.11.029) Copyright © 2014 American Academy of Allergy, Asthma & Immunology Terms and Conditions