X-linked lymphoproliferative disease due to SAP/SH2D1A deficiency: a multicenter study on the manifestations, management and outcome of the disease by.

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Presentation transcript:

X-linked lymphoproliferative disease due to SAP/SH2D1A deficiency: a multicenter study on the manifestations, management and outcome of the disease by Claire Booth, Kimberly C. Gilmour, Paul Veys, Andrew R. Gennery, Mary A. Slatter, Helen Chapel, Paul T. Heath, Colin G. Steward, Owen Smith, Anna O'Meara, Hilary Kerrigan, Nizar Mahlaoui, Marina Cavazzana-Calvo, Alain Fischer, Despina Moshous, Stephane Blanche, Jana Pachlopnik Schmid, Sylvain Latour, Genevieve de Saint-Basile, Michael Albert, Gundula Notheis, Nikolaus Rieber, Brigitte Strahm, Henrike Ritterbusch, Arjan Lankester, Nico G. Hartwig, Isabelle Meyts, Alessandro Plebani, Annarosa Soresina, Andrea Finocchi, Claudio Pignata, Emilia Cirillo, Sonia Bonanomi, Christina Peters, Krzysztof Kalwak, Srdjan Pasic, Petr Sedlacek, Janez Jazbec, Hirokazu Kanegane, Kim E. Nichols, I. Celine Hanson, Neena Kapoor, Elie Haddad, Morton Cowan, Sharon Choo, Joanne Smart, Peter D. Arkwright, and Hubert B. Gaspar Blood Volume 117(1):53-62 January 6, 2011 ©2011 by American Society of Hematology

Outcome of patients with HLH during course of disease. Outcome of patients with HLH during course of disease. Survival of patients who present with HLH—patients who remain untransplanted have a poor survival outcome with only 18.8% survival. By contrast the survival of those who undergo transplant is higher at 50%. Claire Booth et al. Blood 2011;117:53-62 ©2011 by American Society of Hematology

Survival in XLP1 related to different variables. Survival in XLP1 related to different variables. (A) Overall survival of transplanted versus untransplanted patients. In the transplanted group this represents time from presentation and not transplant. (B) Survival according to donor source. (C) Survival after HSCT with relation to presence of HLH before transplant. (D) Survival according to age at transplant. Claire Booth et al. Blood 2011;117:53-62 ©2011 by American Society of Hematology

Outcome of patients with SAP/SH2D1A mutations. Claire Booth et al. Blood 2011;117:53-62 ©2011 by American Society of Hematology