Treatment with emapalumab in an ADA-SCID patient with refractory hemophagocytic lymphohistiocytosis-related graft failure and disseminated BCGitis.

We report a case of secondary HLH-related GF in the context of HLA-haploidentical HSCT successfully treated with emapalumab in the presence of concomitant life-threatening infections, including disseminated tuberculosis (TB). A 4 years old girl with Adenosine Deaminase-Severe Combined Immunodeficiency complicated by disseminated TB came to our attention for ex-vivo hematopoietic stem cell-gene therapy. After engraftment failure of gene corrected cells, she received two HLA-haploidentical T-cell depleted HSCT from the father, both failed due to GF related to concomitant multiple infections and secondary HLH. Emapalumab administration allowed to control HLH, as well as to prevent GF after a third haplo-HSCT from the mother. Remarkably, all infections improved with antimicrobial medications and disseminated TB did not show any reactivation. This seminal case supports emapalumab use for treatment of secondary HLH and prevention of GF in patients undergoing haplo-HSCT even in the presence of multiple infections, including TB.
PMID: 32817285 [PubMed - as supplied by publisher]

Source: Haematologica - August 12, 2020 Category: Hematology Authors: Tucci F, Gallo V, Barzaghi F, Ferrua F, Migliavacca M, Calbi V, Doglio M, Fratini ES, Karakas Z, Guner S, Zambelli M, Parisi C, Milani R, Gattillo S, Mazzi B, Oltolini C, Barbera M, Baldoli C, Cirillo DM, Asnaghi V, De Min C, Cicalese MP, Ciceri F, Aiuti Tags: Haematologica Source Type: research